Glycogen Storage Disease Type II Pompe Disease
Conditions
Brief summary
Primary Objective: Long-term safety and pharmacokinetics (PK) of avalglucosidase alfa Secondary Objective: Long-term effect of avalglucosidase alfa on pharmacodynamic variables
Detailed description
The planned duration of the study for each participant was initially 6 years. Each participant continued with the study until the participant withdrew, the Investigator withdrew the participant, or the Sponsor terminated the study. An additional follow-up phase began after the participant has completed the 6-year study period, and lasted until avalglucosidase alfa was approved in the participant's country, except in the United Kingdom (UK), Germany and Denmark, where the duration of the additional follow-up phase was up to the approval in the country or limited to a maximum of 2 years, whichever occurred first (ie, for participants in the UK, Germany and Denmark, the total study duration per participant was 8 years at the maximum including the initial 6-year period and the additional 2-year follow-up).
Interventions
Pharmaceutical form: lyophilized powder reconstituted for infusion Route of administration: intravenous
Sponsors
Genzyme, a Sanofi Company
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Healthy volunteers
No
Inclusion criteria
Participants with Pompe disease who previously completed an avalglucosidase study. The participant and/or their parent/legal guardian is willing and able to provide signed informed consent, and the participant, if \<18 years of age, was willing to provide assent if deemed able to do so. The participant (and participant's legal guardian if participant is \<18 years of age) must have had the ability to comply with the clinical protocol. The participant, if female and of childbearing potential, had to have a negative pregnancy test \[urine beta-human chorionic gonadotropin\] at baseline.
Exclusion criteria
The participant was concurrently participating in another clinical study using investigational treatment. The participant, in the opinion of the Investigator, was unable to adhere to the requirements of the study. The participant had clinically significant organic disease (with the exception of symptoms relating to Pompe disease), including clinically significant cardiovascular, hepatic, pulmonary, neurologic, or renal disease, or other medical condition, serious intercurrent illness, or extenuating circumstance that, in the opinion of the Investigator, precluded participation in the study or potentially decreases survival. The above information was not intended to contain all considerations relevant to a participant's potential participation in a clinical trial.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Apparent Volume of Distribution Steady-State (Vss) of Avalglucosidase Alfa | Predose (prior to infusion), end of the infusion and at 1, 4, 8, 12, and 24 hours post-dose on Week 312 | Vss was calculated using the following equation: Vz= CLss/λz. The non-compartmental PK analysis was performed. |
| Number of Participants With Body Weight Increased/Decreased | From first dose of IMP up to 4 weeks after the last treatment administration of the IMP, a maximum up to 458 weeks | Body weight was measured in kilograms and collected in the electronic case report forms every 3 months throughout the duration of the study, as well as at the end of study visit. |
| Number of Participants With Potentially Clinically Significant 12-Lead Electrocardiogram (ECG) Abnormalities | From first dose of IMP up to 4 weeks after the last treatment administration of the IMP, a maximum up to 458 weeks | Standard 12-lead ECGs were recorded after at least 15 minutes in the supine position using an electrocardiographic device. The following were assessed: heart rate, rhythm, interval between the peaks of successive QRS complexes (RR), interval from the beginning of the P wave until the beginning of the QRS complex (PR), interval from start of the Q wave to the end of the S wave (QRS), interval between the start of the Q wave and the end of the T wave (QT), QT interval corrected for heart rate (QTc) automatic correction evaluation (by the ECG device), QRS axis, R voltage V6, voltage V1, left ventricular hypertrophy criteria, right ventricular hypertrophy criteria, repolarization charges, and overall cardiac impression for each participant. |
| Number of Participants With Antidrug Antibodies (ADA) Status, Positive or Negative | From first dose of IMP up to 4 weeks after the last treatment administration of the IMP, a maximum up to 458 weeks | ADA negative was defined as ADAs are not detected (that is, negative in screening assay or reactive in screening but negative in confirmatory assay). ADA positive was defined as ADA was detected (that is, an assay signal equal to or greater than the cut-point in the screening assay and was tested positive in the confirmatory assay). |
| Maximum Observed Plasma Concentration (Cmax) of Avalglucosidase Alfa | Predose (prior to infusion), end of the infusion and at 1, 4, 8, 12, and 24 hours post-dose on Week 312 | Cmax was defined as maximum plasma concentration observed. The non-compartmental pharmacokinetic (PK) analysis was performed. |
| Area Under the Plasma Concentration Versus Time Curve From Time Zero to the Real Time (AUClast) of Avalglucosidase Alfa | Predose (prior to infusion), end of the infusion and at 1, 4, 8, 12, and 24 hours post-dose on Week 312 | AUClast was calculated using the trapezoidal method from time zero to the real time. The non-compartmental PK analysis was performed. |
| Terminal Half-Life (t1/2z) of Avalglucosidase Alfa | Predose (prior to infusion), end of the infusion and at 1, 4, 8, 12, and 24 hours post-dose on Week 312 | t1/2z was calculated according to the following equation: t1/2z = 0.693/λz. Where, λz is the slope of the regression line of the terminal phase of the plasma concentration versus time curve. Half-life was calculated by taking the regression of at least 3 points. The non-compartmental PK analysis was performed. |
| Apparent Total Body Clearance Steady-State (CLss) of Avalglucosidase Alfa | Predose (prior to infusion), end of the infusion and at 1, 4, 8, 12, and 24 hours post-dose on Week 312 | CLss was calculated using the following equation: CLss= dose/AUC. The non-compartmental PK analysis was performed. |
| Time Corresponding to the Last Concentration (Tlast) of Avalglucosidase Alfa | Predose (prior to infusion), end of the infusion and at 1, 4, 8, 12, and 24 hours post-dose on Week 312 | Tlast was defined as time corresponding to the last concentration above the limit of quantification, Clast. The non-compartmental PK analysis was performed. |
| Number of Participants With Treatment-Emergent Adverse Events (TEAEs), Treatment-Emergent Serious Adverse Events (TESAEs), Infusion Associated Reactions (IARs) and Deaths | From first dose of IMP up to 4 weeks after the last treatment administration of the IMP, a maximum up to 458 weeks | An adverse event (AE) is any untoward medical occurrence in a participant or clinical investigation participant administered a pharmaceutical product and which does not necessarily have to have a causal relationship with the treatment. An serious AE (SAE) is any untoward medical occurrence that results: death or life-threatening or inpatient hospitalization or prolongation of existing hospitalization or persistent or significant disability or congenital anomaly or medically important event. TEAEs are defined as AEs that develop or worsen during the on-treatment period (that is, from the time of first dose of IMP up to 4 weeks after the last administration of the IMP). Protocol-defined IARs were defined as AEs that occur during either the infusion or the post-infusion observation period (that is, up to 2 hours or longer following the infusion as per the Investigator's discretion) which were deemed to be related or possibly related to the IMP. |
| Number of Participants With Clinically Significant Physical Examination Abnormalities | From first dose of IMP up to 4 weeks after the last treatment administration of the IMP, a maximum up to 458 weeks | Physical examination included, at a minimum, an assessment of the participant's general appearance; skin; head, eyes, ears, nose, and throat; examinations of lymph nodes, abdomen, extremities/joints, neurological and mental status; heart and respiratory auscultation; peripheral arterial pulse; and pupil, knee, achilles, and plantar reflexes. |
| Number of Participants With Potentially Clinically Significant Abnormalities in Biochemistry | From first dose of IMP up to 4 weeks after the last treatment administration of the IMP, a maximum up to 458 weeks | Blood samples were collected to determine the clinical chemistry laboratory abnormalities. |
| Number of Participants With Potentially Clinically Significant Abnormalities in Hematology | From first dose of IMP up to 4 weeks after the last treatment administration of the IMP, a maximum up to 458 weeks | Blood samples were collected to determine the hematology laboratory significant abnormalities. |
| Change From Baseline in Urine BUN up to Last IMP Administration | Baseline (Day 1) and last on-treatment values (up to 454 weeks) | Last on-treatment (LOT) values were collected at or just prior to the last IMP administration. |
| Change From Baseline in Urine Hyaline Casts up to Last IMP Administration | Baseline (Day 1) and last on-treatment values (up to 454 weeks) | The LOT values were collected at or just prior to the last IMP administration. |
| Change From Baseline in Urine Leukocytes [White Blood Cell (WBC)] up to Last IMP Administration | Baseline (Day 1) and last on-treatment values (up to 454 weeks) | The LOT values were collected at or just prior to the last IMP administration. |
| Change From Baseline in Urine Specific Gravity up to Last IMP Administration | Baseline (Day 1) and last on-treatment values (up to 454 weeks) | The LOT values were collected at or just prior to the last IMP administration. |
| Change From Baseline in Urine pH up to Last IMP Administration | Baseline (Day 1) and last on-treatment values (up to 454 weeks) | The LOT values were collected at or just prior to the last IMP administration. |
| Number of Participants With Potentially Clinically Significant Vital Signs Abnormalities | From first dose of IMP up to 4 weeks after the last treatment administration of the IMP, a maximum up to 458 weeks | Participants vital signs were examined to determine the abnormalities. Vital signs included heart rate, systolic and diastolic blood pressure. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Change From Baseline in Dixon Fat Fraction of Skeletal Muscle Magnetic Resonance Imaging (MRI) Up to Week 442 | Baseline (Day 1) and Weeks 104 and 442 | Skeletal muscle MRI performed prior to the muscle needle or open biopsy procedure using both qualitative (T1) and quantitative (T2, dixon) modalities to assess disease severity and detect treatment effects. The T1 weighted axial data was analyzed using the mercuri scale, which determines degree of intact muscle and fatty replacement, providing a qualitative measure of overall disease severity. Trophicity changes were evaluated for 5 muscle groups, including the upper leg muscles (quadriceps, hamstring) and the lower leg muscles (triceps, extensors, fibularis). Three-point dixon imaging provided quantification of fat content in muscles \[fat fraction (FF)\]. |
| Change From Baseline in Index of Real Muscle Mass (IRMM) of Skeletal Muscle Magnetic Resonance Imaging (MRI) Up to Week 442 | Baseline (Day 1) and Weeks 104 and 442 | Skeletal muscle MRI performed prior to the muscle needle or open biopsy procedure using both qualitative (T1) and quantitative (T2, dixon) modalities to assess disease severity and detect treatment effects. The T1 weighted axial data was analyzed using the mercuri scale, which determines degree of intact muscle and fatty replacement, providing a qualitative measure of overall disease severity. Trophicity changes were evaluated for 5 muscle groups, including the upper leg muscles (quadriceps, hamstring) and the lower leg muscles (triceps, extensors, fibularis). The FF was combined with the CSA measurements trophicity to provide an IRMM in mm\^2 (that is, IRMM = CSA x \[1 - FF\]). A negative change from baseline value in IRMM of skeletal muscle MRI indicates muscle loss (worse outcome) and a positive change from baseline value indicates muscle gain (better outcome). |
| Change From Baseline in T2 of Skeletal Muscle Magnetic Resonance Imaging (MRI) Up to Week 442 | Baseline (Day 1) and Weeks 104 and 442 | Skeletal muscle MRI performed prior to the muscle needle or open biopsy procedure using both qualitative (T1) and quantitative (T2, dixon) modalities to assess disease severity and detect treatment effects. The T1 weighted axial data was analyzed using the mercuri scale, which determines degree of intact muscle and fatty replacement, providing a qualitative measure of overall disease severity. Trophicity changes were evaluated for 5 muscle groups, including the upper leg muscles (quadriceps, hamstring) and the lower leg muscles (triceps, extensors, fibularis). The T2 multi-slice multi-spin echo and B1 mapping provided a quantitative measure of disease activity (edema, inflammation) within muscles. |
| Change From Baseline in T2 With B1 of Skeletal Muscle Magnetic Resonance Imaging (MRI) Up to Week 442 | Baseline (Day 1) and Weeks 104 and 442 | Skeletal muscle MRI performed prior to the muscle needle or open biopsy procedure using both qualitative (T1) and quantitative (T2, dixon) modalities to assess disease severity and detect treatment effects. The T1 weighted axial data was analyzed using the mercuri scale, which determines degree of intact muscle and fatty replacement, providing a qualitative measure of overall disease severity. Trophicity changes were evaluated for 5 muscle groups, including the upper leg muscles (quadriceps, hamstring) and the lower leg muscles (triceps, extensors, fibularis). The T2 multi-slice multi-spin echo and B1 mapping provided a quantitative measure of disease activity (edema, inflammation) within muscles. |
| Change From Baseline in Skeletal Muscle Biopsy Up to Week 312 | Baseline (Day 1) and Weeks 27, 104, 208, 260 and 312 | Skeletal muscle needle or open biopsy was performed on the lower extremity (quadriceps) muscle to assess glycogen content. The MRI appearance of the muscle was used to determine the level (axial slice position) that the biopsy procedure should target (avoiding fatty replaced tissue). Glycogen content was measured by histomorphometric analysis or severity grading to determine how effectively avalglucosidase alfa was able to remove glycogen from muscle. |
| Change From Baseline in Urinary Glucose Tetrasaccharide (Hex4) Level Up to Week 442 | Baseline (Day 1) and Weeks 1, 3, 5, 7, 9, 11, 13, 15, 17, 19, 21, 23, 25, 27, 52, 78, 104, 130, 156, 182, 208, 234, 260, 286, 312, 338, 364, 390, 416 and 442 | The Hex4, a tetraglucose oligomer, has been shown to be elevated in the urine of participants with Pompe disease. Hence, determination of Hex4 levels may be a means by which the efficacy of treatments were monitored. Urine samples were collected prior to IMP infusion for the assessment of urinary Hex4 concentrations. |
| Change From Baseline in Cross-Sectional Area (CSA) of Skeletal Muscle Magnetic Resonance Imaging (MRI) Up to Week 442 | Baseline (Day 1) and Weeks 104 and 442 | Skeletal muscle MRI performed prior to the muscle needle or open biopsy procedure using both qualitative (T1) and quantitative (T2, dixon) modalities to assess disease severity and detect treatment effects. The T1 weighted axial data was analyzed using the mercuri scale, which determines degree of intact muscle and fatty replacement, providing a qualitative measure of overall disease severity. Trophicity changes were evaluated for 5 muscle groups, including the upper leg muscles (quadriceps, hamstring) and the lower leg muscles (triceps, extensors, fibularis). The measured area of each muscle group, CSA was provided. |
Countries
Belgium, Denmark, France, Germany, Netherlands, United Kingdom, United States
Participant flow
Recruitment details
The study was conducted at 17 centers in 7 countries. A total of 21 participants completed the open-label study TDR12857 (NCT01898364). Of which, 19 participants were enrolled in this study. There was no screening period in this study.
Pre-assignment details
Participants who completed the open-label TDR12857 study were part of this study. This was an open-label study. As prespecified, data were analyzed and presented combinedly for studies TDR12857 and LTS13769.
Participants by arm
| Arm | Count |
|---|---|
| Group 1: Avalglucosidase Alfa 5 mg/kg Participants who were treatment-naïve to alglucosidase alfa received avalglucosidase alfa 5 mg/kg IV infusion qow for up to 454 weeks. | 4 |
| Group 1: Avalglucosidase Alfa 10 mg/kg Participants who were treatment-naïve to alglucosidase alfa received avalglucosidase alfa 10 mg/kg IV infusion qow for up to 432 weeks. | 3 |
| Group 1: Avalglucosidase Alfa 20 mg/kg Participants who were treatment-naïve to alglucosidase alfa received avalglucosidase alfa 20 mg/kg IV infusion qow for up to 421 weeks. | 3 |
| Group 2: Avalglucosidase Alfa 5 mg/kg Participants who were previously treated with alglucosidase alfa received avalglucosidase alfa 5 mg/kg IV infusion qow for up to 424 weeks. | 4 |
| Group 2: Avalglucosidase Alfa 10 mg/kg Participants who were previously treated with alglucosidase alfa received avalglucosidase alfa 10 mg/kg IV infusion qow for up to 450 weeks. | 4 |
| Group 2: Avalglucosidase Alfa 20 mg/kg Participants who were previously treated with alglucosidase alfa received avalglucosidase alfa 20 mg/kg IV infusion qow for up to 445 weeks. | 6 |
| Total | 24 |
Withdrawals & dropouts
| Period | Reason | FG000 | FG001 | FG002 | FG003 | FG004 | FG005 |
|---|---|---|---|---|---|---|---|
| Period 1: TDR12857 Study | Adverse Event | 1 | 0 | 0 | 0 | 0 | 0 |
| Period 1: TDR12857 Study | Wishes To Withdraw | 0 | 0 | 0 | 1 | 0 | 1 |
| Period 2: LTS13769 Study | Adverse Event | 0 | 0 | 1 | 0 | 0 | 0 |
| Period 2: LTS13769 Study | Other | 0 | 0 | 0 | 0 | 0 | 1 |
| Period 2: LTS13769 Study | Wishes To Withdraw | 0 | 1 | 0 | 0 | 1 | 0 |
Baseline characteristics
| Characteristic | Group 1: Avalglucosidase Alfa 5 mg/kg | Group 1: Avalglucosidase Alfa 20 mg/kg | Group 1: Avalglucosidase Alfa 10 mg/kg | Group 2: Avalglucosidase Alfa 5 mg/kg | Group 2: Avalglucosidase Alfa 10 mg/kg | Group 2: Avalglucosidase Alfa 20 mg/kg | Total |
|---|---|---|---|---|---|---|---|
| Age, Categorical <=18 years | 0 Participants | 0 Participants | 0 Participants | 0 Participants | 0 Participants | 0 Participants | 0 Participants |
| Age, Categorical >=65 years | 1 Participants | 1 Participants | 0 Participants | 1 Participants | 0 Participants | 1 Participants | 4 Participants |
| Age, Categorical Between 18 and 65 years | 3 Participants | 2 Participants | 3 Participants | 3 Participants | 4 Participants | 5 Participants | 20 Participants |
| Race/Ethnicity, Customized Black or African American | 0 Participants | 0 Participants | 0 Participants | 0 Participants | 1 Participants | 0 Participants | 1 Participants |
| Race/Ethnicity, Customized Multiple | 0 Participants | 0 Participants | 1 Participants | 0 Participants | 0 Participants | 0 Participants | 1 Participants |
| Race/Ethnicity, Customized Not Reported | 0 Participants | 0 Participants | 1 Participants | 0 Participants | 0 Participants | 0 Participants | 1 Participants |
| Race/Ethnicity, Customized White | 4 Participants | 3 Participants | 1 Participants | 4 Participants | 3 Participants | 6 Participants | 21 Participants |
| Sex: Female, Male Female | 3 Participants | 1 Participants | 3 Participants | 2 Participants | 1 Participants | 2 Participants | 12 Participants |
| Sex: Female, Male Male | 1 Participants | 2 Participants | 0 Participants | 2 Participants | 3 Participants | 4 Participants | 12 Participants |
Adverse events
| Event type | EG000 affected / at risk | EG001 affected / at risk | EG002 affected / at risk | EG003 affected / at risk | EG004 affected / at risk | EG005 affected / at risk |
|---|---|---|---|---|---|---|
| deaths Total, all-cause mortality | 0 / 4 | 0 / 3 | 1 / 3 | 0 / 4 | 0 / 4 | 0 / 6 |
| other Total, other adverse events | 4 / 4 | 3 / 3 | 3 / 3 | 4 / 4 | 4 / 4 | 6 / 6 |
| serious Total, serious adverse events | 3 / 4 | 1 / 3 | 1 / 3 | 1 / 4 | 1 / 4 | 2 / 6 |
Outcome results
Primary
Apparent Total Body Clearance Steady-State (CLss) of Avalglucosidase Alfa
CLss was calculated using the following equation: CLss= dose/AUC. The non-compartmental PK analysis was performed.
Time frame: Predose (prior to infusion), end of the infusion and at 1, 4, 8, 12, and 24 hours post-dose on Week 312
Population: The PK analysis set included enrolled participants without any critical deviations related to IMP administration, and for whom any PK data were available. As pre-specified, results for this outcome measure are presented combinedly for all dosage groups (5, 10 and 20 mg/kg) under corresponding reporting groups, Group 1: (Avalglucosidase Alfa) and Group 2: (Avalglucosidase Alfa). Overall Number of Participants Analyzed = Number of participants contributed to the analysis for this outcome measure.
| Arm | Measure | Value (GEOMETRIC_MEAN) | Dispersion |
|---|---|---|---|
| Group 1: Avalglucosidase Alfa 5 mg/kg | Apparent Total Body Clearance Steady-State (CLss) of Avalglucosidase Alfa | 0.90 liter/hour | Geometric Coefficient of Variation 24 |
| Group 1: Avalglucosidase Alfa 10 mg/kg | Apparent Total Body Clearance Steady-State (CLss) of Avalglucosidase Alfa | 1.06 liter/hour | Geometric Coefficient of Variation 12 |
Primary
Apparent Volume of Distribution Steady-State (Vss) of Avalglucosidase Alfa
Vss was calculated using the following equation: Vz= CLss/λz. The non-compartmental PK analysis was performed.
Time frame: Predose (prior to infusion), end of the infusion and at 1, 4, 8, 12, and 24 hours post-dose on Week 312
Population: The PK analysis set included enrolled participants without any critical deviations related to IMP administration, and for whom any PK data were available. As pre-specified, results for this outcome measure are presented combinedly for all dosage groups (5, 10 and 20 mg/kg) under corresponding reporting groups, Group 1: (Avalglucosidase Alfa) and Group 2: (Avalglucosidase Alfa). Overall Number of Participants Analyzed = Number of participants contributed to the analysis for this outcome measure.
| Arm | Measure | Value (GEOMETRIC_MEAN) | Dispersion |
|---|---|---|---|
| Group 1: Avalglucosidase Alfa 5 mg/kg | Apparent Volume of Distribution Steady-State (Vss) of Avalglucosidase Alfa | 3.04 liter | Geometric Coefficient of Variation 13 |
| Group 1: Avalglucosidase Alfa 10 mg/kg | Apparent Volume of Distribution Steady-State (Vss) of Avalglucosidase Alfa | 3.8 liter | Geometric Coefficient of Variation 24 |
Primary
Area Under the Plasma Concentration Versus Time Curve From Time Zero to the Real Time (AUClast) of Avalglucosidase Alfa
AUClast was calculated using the trapezoidal method from time zero to the real time. The non-compartmental PK analysis was performed.
Time frame: Predose (prior to infusion), end of the infusion and at 1, 4, 8, 12, and 24 hours post-dose on Week 312
Population: The PK analysis set included enrolled participants without any critical deviations related to IMP administration, and for whom any PK data were available. As pre-specified, results for this outcome measure are presented combinedly for all dosage groups (5, 10 and 20 mg/kg) under corresponding reporting groups, Group 1: (Avalglucosidase Alfa) and Group 2: (Avalglucosidase Alfa). Overall Number of Participants Analyzed = Number of participants contributed to the analysis for this outcome measure.
| Arm | Measure | Value (GEOMETRIC_MEAN) | Dispersion |
|---|---|---|---|
| Group 1: Avalglucosidase Alfa 5 mg/kg | Area Under the Plasma Concentration Versus Time Curve From Time Zero to the Real Time (AUClast) of Avalglucosidase Alfa | 1350 hour*mcg/mL | Geometric Coefficient of Variation 24 |
| Group 1: Avalglucosidase Alfa 10 mg/kg | Area Under the Plasma Concentration Versus Time Curve From Time Zero to the Real Time (AUClast) of Avalglucosidase Alfa | 1290 hour*mcg/mL | Geometric Coefficient of Variation 21 |
Primary
Change From Baseline in Urine BUN up to Last IMP Administration
Last on-treatment (LOT) values were collected at or just prior to the last IMP administration.
Time frame: Baseline (Day 1) and last on-treatment values (up to 454 weeks)
Population: The safety analysis set included all participants who received at least 1 complete infusion of IMP.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Group 1: Avalglucosidase Alfa 5 mg/kg | Change From Baseline in Urine BUN up to Last IMP Administration | 60.8 millimoles per liter (mmol/L) | Standard Deviation 121 |
| Group 1: Avalglucosidase Alfa 10 mg/kg | Change From Baseline in Urine BUN up to Last IMP Administration | -17.6 millimoles per liter (mmol/L) | Standard Deviation 268.8 |
| Group 1: Avalglucosidase Alfa 20 mg/kg | Change From Baseline in Urine BUN up to Last IMP Administration | 179.5 millimoles per liter (mmol/L) | Standard Deviation 157.7 |
| Group 2: Avalglucosidase Alfa 5 mg/kg | Change From Baseline in Urine BUN up to Last IMP Administration | 72.9 millimoles per liter (mmol/L) | Standard Deviation 25.8 |
| Group 2: Avalglucosidase Alfa 10 mg/kg | Change From Baseline in Urine BUN up to Last IMP Administration | 37.1 millimoles per liter (mmol/L) | Standard Deviation 186.5 |
| Group 2: Avalglucosidase Alfa 20 mg/kg | Change From Baseline in Urine BUN up to Last IMP Administration | 118.8 millimoles per liter (mmol/L) | Standard Deviation 134.8 |
Primary
Change From Baseline in Urine Hyaline Casts up to Last IMP Administration
The LOT values were collected at or just prior to the last IMP administration.
Time frame: Baseline (Day 1) and last on-treatment values (up to 454 weeks)
Population: The safety analysis set included all participants who received at least 1 complete infusion of IMP. Only participants with data collected for LOT value are reported.
| Arm | Measure | Value (MEAN) |
|---|---|---|
| Group 1: Avalglucosidase Alfa 10 mg/kg | Change From Baseline in Urine Hyaline Casts up to Last IMP Administration | 2.0 casts per low-power field (casts/lpf) |
Primary
Change From Baseline in Urine Leukocytes [White Blood Cell (WBC)] up to Last IMP Administration
The LOT values were collected at or just prior to the last IMP administration.
Time frame: Baseline (Day 1) and last on-treatment values (up to 454 weeks)
Population: The safety analysis set included all participants who received at least 1 complete infusion of IMP. Only participants with data collected for LOT value are reported.
| Arm | Measure | Value (MEAN) |
|---|---|---|
| Group 1: Avalglucosidase Alfa 10 mg/kg | Change From Baseline in Urine Leukocytes [White Blood Cell (WBC)] up to Last IMP Administration | -1.0 WBC per high power field (WBC/HPF) |
Primary
Change From Baseline in Urine pH up to Last IMP Administration
The LOT values were collected at or just prior to the last IMP administration.
Time frame: Baseline (Day 1) and last on-treatment values (up to 454 weeks)
Population: The safety analysis set included all participants who received at least 1 complete infusion of IMP. Only participants with data collected for LOT value are reported.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Group 1: Avalglucosidase Alfa 5 mg/kg | Change From Baseline in Urine pH up to Last IMP Administration | -0.4 pH score | Standard Deviation 1.3 |
| Group 1: Avalglucosidase Alfa 10 mg/kg | Change From Baseline in Urine pH up to Last IMP Administration | 0.2 pH score | Standard Deviation 1 |
| Group 1: Avalglucosidase Alfa 20 mg/kg | Change From Baseline in Urine pH up to Last IMP Administration | -0.3 pH score | Standard Deviation 1.1 |
| Group 2: Avalglucosidase Alfa 5 mg/kg | Change From Baseline in Urine pH up to Last IMP Administration | 0.1 pH score | Standard Deviation 0.5 |
| Group 2: Avalglucosidase Alfa 10 mg/kg | Change From Baseline in Urine pH up to Last IMP Administration | -0.8 pH score | Standard Deviation 0.4 |
| Group 2: Avalglucosidase Alfa 20 mg/kg | Change From Baseline in Urine pH up to Last IMP Administration | -0.3 pH score | Standard Deviation 0.9 |
Primary
Change From Baseline in Urine Specific Gravity up to Last IMP Administration
The LOT values were collected at or just prior to the last IMP administration.
Time frame: Baseline (Day 1) and last on-treatment values (up to 454 weeks)
Population: The safety analysis set included all participants who received at least 1 complete infusion of IMP. Only participants with data collected for LOT value are reported.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Group 1: Avalglucosidase Alfa 5 mg/kg | Change From Baseline in Urine Specific Gravity up to Last IMP Administration | 0.0 ratio | Standard Deviation 0 |
| Group 1: Avalglucosidase Alfa 10 mg/kg | Change From Baseline in Urine Specific Gravity up to Last IMP Administration | 0.0 ratio | Standard Deviation 0 |
| Group 1: Avalglucosidase Alfa 20 mg/kg | Change From Baseline in Urine Specific Gravity up to Last IMP Administration | 0.0 ratio | Standard Deviation 0 |
| Group 2: Avalglucosidase Alfa 5 mg/kg | Change From Baseline in Urine Specific Gravity up to Last IMP Administration | 0.0 ratio | Standard Deviation 0.1 |
| Group 2: Avalglucosidase Alfa 10 mg/kg | Change From Baseline in Urine Specific Gravity up to Last IMP Administration | 0.0 ratio | Standard Deviation 0 |
| Group 2: Avalglucosidase Alfa 20 mg/kg | Change From Baseline in Urine Specific Gravity up to Last IMP Administration | 0.0 ratio | Standard Deviation 0 |
Primary
Maximum Observed Plasma Concentration (Cmax) of Avalglucosidase Alfa
Cmax was defined as maximum plasma concentration observed. The non-compartmental pharmacokinetic (PK) analysis was performed.
Time frame: Predose (prior to infusion), end of the infusion and at 1, 4, 8, 12, and 24 hours post-dose on Week 312
Population: The PK analysis set included enrolled participants without any critical deviations related to IMP administration, and for whom any PK data were available. As pre-specified, results for this outcome measure are presented combinedly for all the dosage groups (5, 10 and 20 mg/kg) under the corresponding reporting groups, Group 1: (Avalglucosidase Alfa) and Group 2: (Avalglucosidase Alfa).
| Arm | Measure | Value (GEOMETRIC_MEAN) | Dispersion |
|---|---|---|---|
| Group 1: Avalglucosidase Alfa 5 mg/kg | Maximum Observed Plasma Concentration (Cmax) of Avalglucosidase Alfa | 269 microgram per milliliter (mcg/mL) | Geometric Coefficient of Variation 16 |
| Group 1: Avalglucosidase Alfa 10 mg/kg | Maximum Observed Plasma Concentration (Cmax) of Avalglucosidase Alfa | 234 microgram per milliliter (mcg/mL) | Geometric Coefficient of Variation 24 |
Primary
Number of Participants With Antidrug Antibodies (ADA) Status, Positive or Negative
ADA negative was defined as ADAs are not detected (that is, negative in screening assay or reactive in screening but negative in confirmatory assay). ADA positive was defined as ADA was detected (that is, an assay signal equal to or greater than the cut-point in the screening assay and was tested positive in the confirmatory assay).
Time frame: From first dose of IMP up to 4 weeks after the last treatment administration of the IMP, a maximum up to 458 weeks
Population: The Pharmacodynamic (PD) analysis set included enrolled participants without any critical deviations related to IMP administration, and for whom any PD data were available.
| Arm | Measure | Group | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|---|
| Group 1: Avalglucosidase Alfa 5 mg/kg | Number of Participants With Antidrug Antibodies (ADA) Status, Positive or Negative | ADA positive post-baseline | 4 Participants |
| Group 1: Avalglucosidase Alfa 5 mg/kg | Number of Participants With Antidrug Antibodies (ADA) Status, Positive or Negative | ADA positive at baseline | 0 Participants |
| Group 1: Avalglucosidase Alfa 5 mg/kg | Number of Participants With Antidrug Antibodies (ADA) Status, Positive or Negative | ADA negative | 0 Participants |
| Group 1: Avalglucosidase Alfa 10 mg/kg | Number of Participants With Antidrug Antibodies (ADA) Status, Positive or Negative | ADA negative | 1 Participants |
| Group 1: Avalglucosidase Alfa 10 mg/kg | Number of Participants With Antidrug Antibodies (ADA) Status, Positive or Negative | ADA positive at baseline | 0 Participants |
| Group 1: Avalglucosidase Alfa 10 mg/kg | Number of Participants With Antidrug Antibodies (ADA) Status, Positive or Negative | ADA positive post-baseline | 2 Participants |
| Group 1: Avalglucosidase Alfa 20 mg/kg | Number of Participants With Antidrug Antibodies (ADA) Status, Positive or Negative | ADA positive post-baseline | 3 Participants |
| Group 1: Avalglucosidase Alfa 20 mg/kg | Number of Participants With Antidrug Antibodies (ADA) Status, Positive or Negative | ADA negative | 0 Participants |
| Group 1: Avalglucosidase Alfa 20 mg/kg | Number of Participants With Antidrug Antibodies (ADA) Status, Positive or Negative | ADA positive at baseline | 0 Participants |
| Group 2: Avalglucosidase Alfa 5 mg/kg | Number of Participants With Antidrug Antibodies (ADA) Status, Positive or Negative | ADA positive at baseline | 1 Participants |
| Group 2: Avalglucosidase Alfa 5 mg/kg | Number of Participants With Antidrug Antibodies (ADA) Status, Positive or Negative | ADA negative | 1 Participants |
| Group 2: Avalglucosidase Alfa 5 mg/kg | Number of Participants With Antidrug Antibodies (ADA) Status, Positive or Negative | ADA positive post-baseline | 2 Participants |
| Group 2: Avalglucosidase Alfa 10 mg/kg | Number of Participants With Antidrug Antibodies (ADA) Status, Positive or Negative | ADA negative | 1 Participants |
| Group 2: Avalglucosidase Alfa 10 mg/kg | Number of Participants With Antidrug Antibodies (ADA) Status, Positive or Negative | ADA positive at baseline | 3 Participants |
| Group 2: Avalglucosidase Alfa 10 mg/kg | Number of Participants With Antidrug Antibodies (ADA) Status, Positive or Negative | ADA positive post-baseline | 0 Participants |
| Group 2: Avalglucosidase Alfa 20 mg/kg | Number of Participants With Antidrug Antibodies (ADA) Status, Positive or Negative | ADA negative | 3 Participants |
| Group 2: Avalglucosidase Alfa 20 mg/kg | Number of Participants With Antidrug Antibodies (ADA) Status, Positive or Negative | ADA positive post-baseline | 2 Participants |
| Group 2: Avalglucosidase Alfa 20 mg/kg | Number of Participants With Antidrug Antibodies (ADA) Status, Positive or Negative | ADA positive at baseline | 1 Participants |
Primary
Number of Participants With Body Weight Increased/Decreased
Body weight was measured in kilograms and collected in the electronic case report forms every 3 months throughout the duration of the study, as well as at the end of study visit.
Time frame: From first dose of IMP up to 4 weeks after the last treatment administration of the IMP, a maximum up to 458 weeks
Population: The safety analysis set included all participants who received at least 1 complete infusion of IMP.
| Arm | Measure | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|
| Group 1: Avalglucosidase Alfa 5 mg/kg | Number of Participants With Body Weight Increased/Decreased | 1 Participants |
| Group 1: Avalglucosidase Alfa 10 mg/kg | Number of Participants With Body Weight Increased/Decreased | 0 Participants |
| Group 1: Avalglucosidase Alfa 20 mg/kg | Number of Participants With Body Weight Increased/Decreased | 0 Participants |
| Group 2: Avalglucosidase Alfa 5 mg/kg | Number of Participants With Body Weight Increased/Decreased | 1 Participants |
| Group 2: Avalglucosidase Alfa 10 mg/kg | Number of Participants With Body Weight Increased/Decreased | 0 Participants |
| Group 2: Avalglucosidase Alfa 20 mg/kg | Number of Participants With Body Weight Increased/Decreased | 2 Participants |
Primary
Number of Participants With Clinically Significant Physical Examination Abnormalities
Physical examination included, at a minimum, an assessment of the participant's general appearance; skin; head, eyes, ears, nose, and throat; examinations of lymph nodes, abdomen, extremities/joints, neurological and mental status; heart and respiratory auscultation; peripheral arterial pulse; and pupil, knee, achilles, and plantar reflexes.
Time frame: From first dose of IMP up to 4 weeks after the last treatment administration of the IMP, a maximum up to 458 weeks
Population: The safety analysis set included all participants who received at least 1 complete infusion of IMP.
| Arm | Measure | Group | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|---|
| Group 1: Avalglucosidase Alfa 5 mg/kg | Number of Participants With Clinically Significant Physical Examination Abnormalities | Lymph Nodes | 0 Participants |
| Group 1: Avalglucosidase Alfa 5 mg/kg | Number of Participants With Clinically Significant Physical Examination Abnormalities | Neurological Status | 0 Participants |
| Group 1: Avalglucosidase Alfa 5 mg/kg | Number of Participants With Clinically Significant Physical Examination Abnormalities | Knee Reflexes | 0 Participants |
| Group 1: Avalglucosidase Alfa 5 mg/kg | Number of Participants With Clinically Significant Physical Examination Abnormalities | Achilles Reflexes | 0 Participants |
| Group 1: Avalglucosidase Alfa 5 mg/kg | Number of Participants With Clinically Significant Physical Examination Abnormalities | General Appearance | 0 Participants |
| Group 1: Avalglucosidase Alfa 10 mg/kg | Number of Participants With Clinically Significant Physical Examination Abnormalities | Neurological Status | 0 Participants |
| Group 1: Avalglucosidase Alfa 10 mg/kg | Number of Participants With Clinically Significant Physical Examination Abnormalities | General Appearance | 0 Participants |
| Group 1: Avalglucosidase Alfa 10 mg/kg | Number of Participants With Clinically Significant Physical Examination Abnormalities | Knee Reflexes | 0 Participants |
| Group 1: Avalglucosidase Alfa 10 mg/kg | Number of Participants With Clinically Significant Physical Examination Abnormalities | Lymph Nodes | 0 Participants |
| Group 1: Avalglucosidase Alfa 10 mg/kg | Number of Participants With Clinically Significant Physical Examination Abnormalities | Achilles Reflexes | 0 Participants |
| Group 1: Avalglucosidase Alfa 20 mg/kg | Number of Participants With Clinically Significant Physical Examination Abnormalities | Neurological Status | 0 Participants |
| Group 1: Avalglucosidase Alfa 20 mg/kg | Number of Participants With Clinically Significant Physical Examination Abnormalities | Lymph Nodes | 0 Participants |
| Group 1: Avalglucosidase Alfa 20 mg/kg | Number of Participants With Clinically Significant Physical Examination Abnormalities | Achilles Reflexes | 0 Participants |
| Group 1: Avalglucosidase Alfa 20 mg/kg | Number of Participants With Clinically Significant Physical Examination Abnormalities | General Appearance | 0 Participants |
| Group 1: Avalglucosidase Alfa 20 mg/kg | Number of Participants With Clinically Significant Physical Examination Abnormalities | Knee Reflexes | 0 Participants |
| Group 2: Avalglucosidase Alfa 5 mg/kg | Number of Participants With Clinically Significant Physical Examination Abnormalities | Knee Reflexes | 0 Participants |
| Group 2: Avalglucosidase Alfa 5 mg/kg | Number of Participants With Clinically Significant Physical Examination Abnormalities | Achilles Reflexes | 0 Participants |
| Group 2: Avalglucosidase Alfa 5 mg/kg | Number of Participants With Clinically Significant Physical Examination Abnormalities | Lymph Nodes | 0 Participants |
| Group 2: Avalglucosidase Alfa 5 mg/kg | Number of Participants With Clinically Significant Physical Examination Abnormalities | Neurological Status | 0 Participants |
| Group 2: Avalglucosidase Alfa 5 mg/kg | Number of Participants With Clinically Significant Physical Examination Abnormalities | General Appearance | 0 Participants |
| Group 2: Avalglucosidase Alfa 10 mg/kg | Number of Participants With Clinically Significant Physical Examination Abnormalities | Lymph Nodes | 0 Participants |
| Group 2: Avalglucosidase Alfa 10 mg/kg | Number of Participants With Clinically Significant Physical Examination Abnormalities | General Appearance | 0 Participants |
| Group 2: Avalglucosidase Alfa 10 mg/kg | Number of Participants With Clinically Significant Physical Examination Abnormalities | Neurological Status | 2 Participants |
| Group 2: Avalglucosidase Alfa 10 mg/kg | Number of Participants With Clinically Significant Physical Examination Abnormalities | Knee Reflexes | 0 Participants |
| Group 2: Avalglucosidase Alfa 10 mg/kg | Number of Participants With Clinically Significant Physical Examination Abnormalities | Achilles Reflexes | 0 Participants |
| Group 2: Avalglucosidase Alfa 20 mg/kg | Number of Participants With Clinically Significant Physical Examination Abnormalities | Neurological Status | 1 Participants |
| Group 2: Avalglucosidase Alfa 20 mg/kg | Number of Participants With Clinically Significant Physical Examination Abnormalities | Knee Reflexes | 1 Participants |
| Group 2: Avalglucosidase Alfa 20 mg/kg | Number of Participants With Clinically Significant Physical Examination Abnormalities | Achilles Reflexes | 1 Participants |
| Group 2: Avalglucosidase Alfa 20 mg/kg | Number of Participants With Clinically Significant Physical Examination Abnormalities | General Appearance | 1 Participants |
| Group 2: Avalglucosidase Alfa 20 mg/kg | Number of Participants With Clinically Significant Physical Examination Abnormalities | Lymph Nodes | 1 Participants |
Primary
Number of Participants With Potentially Clinically Significant 12-Lead Electrocardiogram (ECG) Abnormalities
Standard 12-lead ECGs were recorded after at least 15 minutes in the supine position using an electrocardiographic device. The following were assessed: heart rate, rhythm, interval between the peaks of successive QRS complexes (RR), interval from the beginning of the P wave until the beginning of the QRS complex (PR), interval from start of the Q wave to the end of the S wave (QRS), interval between the start of the Q wave and the end of the T wave (QT), QT interval corrected for heart rate (QTc) automatic correction evaluation (by the ECG device), QRS axis, R voltage V6, voltage V1, left ventricular hypertrophy criteria, right ventricular hypertrophy criteria, repolarization charges, and overall cardiac impression for each participant.
Time frame: From first dose of IMP up to 4 weeks after the last treatment administration of the IMP, a maximum up to 458 weeks
Population: The safety analysis set included all participants who received at least 1 complete infusion of IMP.
| Arm | Measure | Group | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|---|
| Group 1: Avalglucosidase Alfa 5 mg/kg | Number of Participants With Potentially Clinically Significant 12-Lead Electrocardiogram (ECG) Abnormalities | QRS Duration: >110 msec and increase from baseline >=25% | 0 Participants |
| Group 1: Avalglucosidase Alfa 5 mg/kg | Number of Participants With Potentially Clinically Significant 12-Lead Electrocardiogram (ECG) Abnormalities | HR: >100 bpm and increase from baseline >=20 bpm | 0 Participants |
| Group 1: Avalglucosidase Alfa 5 mg/kg | Number of Participants With Potentially Clinically Significant 12-Lead Electrocardiogram (ECG) Abnormalities | PR Duration: >240 msec | 0 Participants |
| Group 1: Avalglucosidase Alfa 5 mg/kg | Number of Participants With Potentially Clinically Significant 12-Lead Electrocardiogram (ECG) Abnormalities | QTcB - Bazett's Correction Formula: >480 msec | 0 Participants |
| Group 1: Avalglucosidase Alfa 5 mg/kg | Number of Participants With Potentially Clinically Significant 12-Lead Electrocardiogram (ECG) Abnormalities | HR: >100 bpm | 0 Participants |
| Group 1: Avalglucosidase Alfa 5 mg/kg | Number of Participants With Potentially Clinically Significant 12-Lead Electrocardiogram (ECG) Abnormalities | HR: >90 bpm and increase from baseline >=20 bpm | 0 Participants |
| Group 1: Avalglucosidase Alfa 5 mg/kg | Number of Participants With Potentially Clinically Significant 12-Lead Electrocardiogram (ECG) Abnormalities | QTcF - Fridericia's Correction Formula: >500 msec | 0 Participants |
| Group 1: Avalglucosidase Alfa 5 mg/kg | Number of Participants With Potentially Clinically Significant 12-Lead Electrocardiogram (ECG) Abnormalities | QTcB - Bazett's Correction Formula: >450 msec | 1 Participants |
| Group 1: Avalglucosidase Alfa 5 mg/kg | Number of Participants With Potentially Clinically Significant 12-Lead Electrocardiogram (ECG) Abnormalities | PR Duration: >220 msec | 0 Participants |
| Group 1: Avalglucosidase Alfa 5 mg/kg | Number of Participants With Potentially Clinically Significant 12-Lead Electrocardiogram (ECG) Abnormalities | QTcB - Bazett's Correction Formula: >500 msec | 0 Participants |
| Group 1: Avalglucosidase Alfa 5 mg/kg | Number of Participants With Potentially Clinically Significant 12-Lead Electrocardiogram (ECG) Abnormalities | HR: <50 bpm | 1 Participants |
| Group 1: Avalglucosidase Alfa 5 mg/kg | Number of Participants With Potentially Clinically Significant 12-Lead Electrocardiogram (ECG) Abnormalities | PR Duration: > 200 millisecond (msec) | 0 Participants |
| Group 1: Avalglucosidase Alfa 5 mg/kg | Number of Participants With Potentially Clinically Significant 12-Lead Electrocardiogram (ECG) Abnormalities | QTcB - Bazett's Correction Formula: Increase from baseline (30-60) msec | 0 Participants |
| Group 1: Avalglucosidase Alfa 5 mg/kg | Number of Participants With Potentially Clinically Significant 12-Lead Electrocardiogram (ECG) Abnormalities | HR: <50 bpm and decrease from baseline >=20 bpm | 0 Participants |
| Group 1: Avalglucosidase Alfa 5 mg/kg | Number of Participants With Potentially Clinically Significant 12-Lead Electrocardiogram (ECG) Abnormalities | PR Duration: >240 msec and increase from baseline >=25% | 0 Participants |
| Group 1: Avalglucosidase Alfa 5 mg/kg | Number of Participants With Potentially Clinically Significant 12-Lead Electrocardiogram (ECG) Abnormalities | HR: <40 bpm | 0 Participants |
| Group 1: Avalglucosidase Alfa 5 mg/kg | Number of Participants With Potentially Clinically Significant 12-Lead Electrocardiogram (ECG) Abnormalities | HR: <30 bpm | 0 Participants |
| Group 1: Avalglucosidase Alfa 5 mg/kg | Number of Participants With Potentially Clinically Significant 12-Lead Electrocardiogram (ECG) Abnormalities | QTcF - Fridericia's Correction Formula: Increase from baseline >60 msec | 0 Participants |
| Group 1: Avalglucosidase Alfa 5 mg/kg | Number of Participants With Potentially Clinically Significant 12-Lead Electrocardiogram (ECG) Abnormalities | PR Duration: >220 msec and increase from baseline >=25% | 0 Participants |
| Group 1: Avalglucosidase Alfa 5 mg/kg | Number of Participants With Potentially Clinically Significant 12-Lead Electrocardiogram (ECG) Abnormalities | QTcF - Fridericia's Correction Formula: >480 msec | 0 Participants |
| Group 1: Avalglucosidase Alfa 5 mg/kg | Number of Participants With Potentially Clinically Significant 12-Lead Electrocardiogram (ECG) Abnormalities | HR: <40 bpm and decrease from baseline >=20 bpm | 0 Participants |
| Group 1: Avalglucosidase Alfa 5 mg/kg | Number of Participants With Potentially Clinically Significant 12-Lead Electrocardiogram (ECG) Abnormalities | QRS Duration: >110 msec | 0 Participants |
| Group 1: Avalglucosidase Alfa 5 mg/kg | Number of Participants With Potentially Clinically Significant 12-Lead Electrocardiogram (ECG) Abnormalities | QTcF - Fridericia's Correction Formula: Increase from baseline (30-60) msec | 0 Participants |
| Group 1: Avalglucosidase Alfa 5 mg/kg | Number of Participants With Potentially Clinically Significant 12-Lead Electrocardiogram (ECG) Abnormalities | QTcF - Fridericia's Correction Formula: >450 msec | 0 Participants |
| Group 1: Avalglucosidase Alfa 5 mg/kg | Number of Participants With Potentially Clinically Significant 12-Lead Electrocardiogram (ECG) Abnormalities | HR: >90 bpm | 0 Participants |
| Group 1: Avalglucosidase Alfa 5 mg/kg | Number of Participants With Potentially Clinically Significant 12-Lead Electrocardiogram (ECG) Abnormalities | HR: >120 bpm and increase from baseline >=20 bpm | 0 Participants |
| Group 1: Avalglucosidase Alfa 5 mg/kg | Number of Participants With Potentially Clinically Significant 12-Lead Electrocardiogram (ECG) Abnormalities | HR: <30 bpm and decrease from baseline >=20 bpm | 0 Participants |
| Group 1: Avalglucosidase Alfa 5 mg/kg | Number of Participants With Potentially Clinically Significant 12-Lead Electrocardiogram (ECG) Abnormalities | QTcB - Bazett's Correction Formula: Increase from baseline >60 msec | 0 Participants |
| Group 1: Avalglucosidase Alfa 5 mg/kg | Number of Participants With Potentially Clinically Significant 12-Lead Electrocardiogram (ECG) Abnormalities | HR: >120 bpm | 0 Participants |
| Group 1: Avalglucosidase Alfa 5 mg/kg | Number of Participants With Potentially Clinically Significant 12-Lead Electrocardiogram (ECG) Abnormalities | QRS Duration: >120 msec | 0 Participants |
| Group 1: Avalglucosidase Alfa 5 mg/kg | Number of Participants With Potentially Clinically Significant 12-Lead Electrocardiogram (ECG) Abnormalities | PR Duration: >200 msec and increase from baseline >=25% | 0 Participants |
| Group 1: Avalglucosidase Alfa 5 mg/kg | Number of Participants With Potentially Clinically Significant 12-Lead Electrocardiogram (ECG) Abnormalities | QRS Duration: >120 msec and increase from baseline >=25% | 0 Participants |
| Group 1: Avalglucosidase Alfa 10 mg/kg | Number of Participants With Potentially Clinically Significant 12-Lead Electrocardiogram (ECG) Abnormalities | QRS Duration: >110 msec and increase from baseline >=25% | 0 Participants |
| Group 1: Avalglucosidase Alfa 10 mg/kg | Number of Participants With Potentially Clinically Significant 12-Lead Electrocardiogram (ECG) Abnormalities | QRS Duration: >120 msec | 1 Participants |
| Group 1: Avalglucosidase Alfa 10 mg/kg | Number of Participants With Potentially Clinically Significant 12-Lead Electrocardiogram (ECG) Abnormalities | HR: <40 bpm and decrease from baseline >=20 bpm | 0 Participants |
| Group 1: Avalglucosidase Alfa 10 mg/kg | Number of Participants With Potentially Clinically Significant 12-Lead Electrocardiogram (ECG) Abnormalities | PR Duration: >240 msec | 0 Participants |
| Group 1: Avalglucosidase Alfa 10 mg/kg | Number of Participants With Potentially Clinically Significant 12-Lead Electrocardiogram (ECG) Abnormalities | PR Duration: >200 msec and increase from baseline >=25% | 1 Participants |
| Group 1: Avalglucosidase Alfa 10 mg/kg | Number of Participants With Potentially Clinically Significant 12-Lead Electrocardiogram (ECG) Abnormalities | PR Duration: >220 msec and increase from baseline >=25% | 0 Participants |
| Group 1: Avalglucosidase Alfa 10 mg/kg | Number of Participants With Potentially Clinically Significant 12-Lead Electrocardiogram (ECG) Abnormalities | HR: >100 bpm | 0 Participants |
| Group 1: Avalglucosidase Alfa 10 mg/kg | Number of Participants With Potentially Clinically Significant 12-Lead Electrocardiogram (ECG) Abnormalities | HR: <30 bpm | 0 Participants |
| Group 1: Avalglucosidase Alfa 10 mg/kg | Number of Participants With Potentially Clinically Significant 12-Lead Electrocardiogram (ECG) Abnormalities | QTcB - Bazett's Correction Formula: Increase from baseline >60 msec | 0 Participants |
| Group 1: Avalglucosidase Alfa 10 mg/kg | Number of Participants With Potentially Clinically Significant 12-Lead Electrocardiogram (ECG) Abnormalities | QTcB - Bazett's Correction Formula: >480 msec | 0 Participants |
| Group 1: Avalglucosidase Alfa 10 mg/kg | Number of Participants With Potentially Clinically Significant 12-Lead Electrocardiogram (ECG) Abnormalities | QTcF - Fridericia's Correction Formula: >500 msec | 0 Participants |
| Group 1: Avalglucosidase Alfa 10 mg/kg | Number of Participants With Potentially Clinically Significant 12-Lead Electrocardiogram (ECG) Abnormalities | HR: >120 bpm and increase from baseline >=20 bpm | 0 Participants |
| Group 1: Avalglucosidase Alfa 10 mg/kg | Number of Participants With Potentially Clinically Significant 12-Lead Electrocardiogram (ECG) Abnormalities | HR: >90 bpm and increase from baseline >=20 bpm | 2 Participants |
| Group 1: Avalglucosidase Alfa 10 mg/kg | Number of Participants With Potentially Clinically Significant 12-Lead Electrocardiogram (ECG) Abnormalities | QTcB - Bazett's Correction Formula: >450 msec | 1 Participants |
| Group 1: Avalglucosidase Alfa 10 mg/kg | Number of Participants With Potentially Clinically Significant 12-Lead Electrocardiogram (ECG) Abnormalities | QTcF - Fridericia's Correction Formula: >450 msec | 1 Participants |
| Group 1: Avalglucosidase Alfa 10 mg/kg | Number of Participants With Potentially Clinically Significant 12-Lead Electrocardiogram (ECG) Abnormalities | QTcF - Fridericia's Correction Formula: Increase from baseline (30-60) msec | 1 Participants |
| Group 1: Avalglucosidase Alfa 10 mg/kg | Number of Participants With Potentially Clinically Significant 12-Lead Electrocardiogram (ECG) Abnormalities | PR Duration: >220 msec | 0 Participants |
| Group 1: Avalglucosidase Alfa 10 mg/kg | Number of Participants With Potentially Clinically Significant 12-Lead Electrocardiogram (ECG) Abnormalities | HR: <30 bpm and decrease from baseline >=20 bpm | 0 Participants |
| Group 1: Avalglucosidase Alfa 10 mg/kg | Number of Participants With Potentially Clinically Significant 12-Lead Electrocardiogram (ECG) Abnormalities | QRS Duration: >110 msec | 0 Participants |
| Group 1: Avalglucosidase Alfa 10 mg/kg | Number of Participants With Potentially Clinically Significant 12-Lead Electrocardiogram (ECG) Abnormalities | HR: <50 bpm | 0 Participants |
| Group 1: Avalglucosidase Alfa 10 mg/kg | Number of Participants With Potentially Clinically Significant 12-Lead Electrocardiogram (ECG) Abnormalities | QTcF - Fridericia's Correction Formula: >480 msec | 0 Participants |
| Group 1: Avalglucosidase Alfa 10 mg/kg | Number of Participants With Potentially Clinically Significant 12-Lead Electrocardiogram (ECG) Abnormalities | HR: >120 bpm | 0 Participants |
| Group 1: Avalglucosidase Alfa 10 mg/kg | Number of Participants With Potentially Clinically Significant 12-Lead Electrocardiogram (ECG) Abnormalities | QTcB - Bazett's Correction Formula: Increase from baseline (30-60) msec | 2 Participants |
| Group 1: Avalglucosidase Alfa 10 mg/kg | Number of Participants With Potentially Clinically Significant 12-Lead Electrocardiogram (ECG) Abnormalities | HR: >90 bpm | 2 Participants |
| Group 1: Avalglucosidase Alfa 10 mg/kg | Number of Participants With Potentially Clinically Significant 12-Lead Electrocardiogram (ECG) Abnormalities | QRS Duration: >120 msec and increase from baseline >=25% | 0 Participants |
| Group 1: Avalglucosidase Alfa 10 mg/kg | Number of Participants With Potentially Clinically Significant 12-Lead Electrocardiogram (ECG) Abnormalities | HR: <50 bpm and decrease from baseline >=20 bpm | 0 Participants |
| Group 1: Avalglucosidase Alfa 10 mg/kg | Number of Participants With Potentially Clinically Significant 12-Lead Electrocardiogram (ECG) Abnormalities | HR: >100 bpm and increase from baseline >=20 bpm | 0 Participants |
| Group 1: Avalglucosidase Alfa 10 mg/kg | Number of Participants With Potentially Clinically Significant 12-Lead Electrocardiogram (ECG) Abnormalities | PR Duration: > 200 millisecond (msec) | 1 Participants |
| Group 1: Avalglucosidase Alfa 10 mg/kg | Number of Participants With Potentially Clinically Significant 12-Lead Electrocardiogram (ECG) Abnormalities | PR Duration: >240 msec and increase from baseline >=25% | 0 Participants |
| Group 1: Avalglucosidase Alfa 10 mg/kg | Number of Participants With Potentially Clinically Significant 12-Lead Electrocardiogram (ECG) Abnormalities | QTcB - Bazett's Correction Formula: >500 msec | 0 Participants |
| Group 1: Avalglucosidase Alfa 10 mg/kg | Number of Participants With Potentially Clinically Significant 12-Lead Electrocardiogram (ECG) Abnormalities | QTcF - Fridericia's Correction Formula: Increase from baseline >60 msec | 0 Participants |
| Group 1: Avalglucosidase Alfa 10 mg/kg | Number of Participants With Potentially Clinically Significant 12-Lead Electrocardiogram (ECG) Abnormalities | HR: <40 bpm | 0 Participants |
| Group 1: Avalglucosidase Alfa 20 mg/kg | Number of Participants With Potentially Clinically Significant 12-Lead Electrocardiogram (ECG) Abnormalities | HR: >100 bpm and increase from baseline >=20 bpm | 0 Participants |
| Group 1: Avalglucosidase Alfa 20 mg/kg | Number of Participants With Potentially Clinically Significant 12-Lead Electrocardiogram (ECG) Abnormalities | PR Duration: > 200 millisecond (msec) | 1 Participants |
| Group 1: Avalglucosidase Alfa 20 mg/kg | Number of Participants With Potentially Clinically Significant 12-Lead Electrocardiogram (ECG) Abnormalities | HR: <40 bpm and decrease from baseline >=20 bpm | 0 Participants |
| Group 1: Avalglucosidase Alfa 20 mg/kg | Number of Participants With Potentially Clinically Significant 12-Lead Electrocardiogram (ECG) Abnormalities | HR: >90 bpm | 0 Participants |
| Group 1: Avalglucosidase Alfa 20 mg/kg | Number of Participants With Potentially Clinically Significant 12-Lead Electrocardiogram (ECG) Abnormalities | HR: <30 bpm | 0 Participants |
| Group 1: Avalglucosidase Alfa 20 mg/kg | Number of Participants With Potentially Clinically Significant 12-Lead Electrocardiogram (ECG) Abnormalities | HR: <30 bpm and decrease from baseline >=20 bpm | 0 Participants |
| Group 1: Avalglucosidase Alfa 20 mg/kg | Number of Participants With Potentially Clinically Significant 12-Lead Electrocardiogram (ECG) Abnormalities | QTcF - Fridericia's Correction Formula: >500 msec | 0 Participants |
| Group 1: Avalglucosidase Alfa 20 mg/kg | Number of Participants With Potentially Clinically Significant 12-Lead Electrocardiogram (ECG) Abnormalities | PR Duration: >200 msec and increase from baseline >=25% | 0 Participants |
| Group 1: Avalglucosidase Alfa 20 mg/kg | Number of Participants With Potentially Clinically Significant 12-Lead Electrocardiogram (ECG) Abnormalities | QTcF - Fridericia's Correction Formula: Increase from baseline >60 msec | 0 Participants |
| Group 1: Avalglucosidase Alfa 20 mg/kg | Number of Participants With Potentially Clinically Significant 12-Lead Electrocardiogram (ECG) Abnormalities | QTcF - Fridericia's Correction Formula: >480 msec | 0 Participants |
| Group 1: Avalglucosidase Alfa 20 mg/kg | Number of Participants With Potentially Clinically Significant 12-Lead Electrocardiogram (ECG) Abnormalities | PR Duration: >240 msec and increase from baseline >=25% | 0 Participants |
| Group 1: Avalglucosidase Alfa 20 mg/kg | Number of Participants With Potentially Clinically Significant 12-Lead Electrocardiogram (ECG) Abnormalities | PR Duration: >220 msec and increase from baseline >=25% | 0 Participants |
| Group 1: Avalglucosidase Alfa 20 mg/kg | Number of Participants With Potentially Clinically Significant 12-Lead Electrocardiogram (ECG) Abnormalities | QTcF - Fridericia's Correction Formula: >450 msec | 0 Participants |
| Group 1: Avalglucosidase Alfa 20 mg/kg | Number of Participants With Potentially Clinically Significant 12-Lead Electrocardiogram (ECG) Abnormalities | QRS Duration: >110 msec | 1 Participants |
| Group 1: Avalglucosidase Alfa 20 mg/kg | Number of Participants With Potentially Clinically Significant 12-Lead Electrocardiogram (ECG) Abnormalities | PR Duration: >220 msec | 0 Participants |
| Group 1: Avalglucosidase Alfa 20 mg/kg | Number of Participants With Potentially Clinically Significant 12-Lead Electrocardiogram (ECG) Abnormalities | QTcB - Bazett's Correction Formula: Increase from baseline >60 msec | 0 Participants |
| Group 1: Avalglucosidase Alfa 20 mg/kg | Number of Participants With Potentially Clinically Significant 12-Lead Electrocardiogram (ECG) Abnormalities | QRS Duration: >110 msec and increase from baseline >=25% | 0 Participants |
| Group 1: Avalglucosidase Alfa 20 mg/kg | Number of Participants With Potentially Clinically Significant 12-Lead Electrocardiogram (ECG) Abnormalities | HR: >120 bpm | 0 Participants |
| Group 1: Avalglucosidase Alfa 20 mg/kg | Number of Participants With Potentially Clinically Significant 12-Lead Electrocardiogram (ECG) Abnormalities | QRS Duration: >120 msec | 1 Participants |
| Group 1: Avalglucosidase Alfa 20 mg/kg | Number of Participants With Potentially Clinically Significant 12-Lead Electrocardiogram (ECG) Abnormalities | HR: >120 bpm and increase from baseline >=20 bpm | 0 Participants |
| Group 1: Avalglucosidase Alfa 20 mg/kg | Number of Participants With Potentially Clinically Significant 12-Lead Electrocardiogram (ECG) Abnormalities | HR: <40 bpm | 0 Participants |
| Group 1: Avalglucosidase Alfa 20 mg/kg | Number of Participants With Potentially Clinically Significant 12-Lead Electrocardiogram (ECG) Abnormalities | PR Duration: >240 msec | 0 Participants |
| Group 1: Avalglucosidase Alfa 20 mg/kg | Number of Participants With Potentially Clinically Significant 12-Lead Electrocardiogram (ECG) Abnormalities | QTcF - Fridericia's Correction Formula: Increase from baseline (30-60) msec | 0 Participants |
| Group 1: Avalglucosidase Alfa 20 mg/kg | Number of Participants With Potentially Clinically Significant 12-Lead Electrocardiogram (ECG) Abnormalities | QRS Duration: >120 msec and increase from baseline >=25% | 1 Participants |
| Group 1: Avalglucosidase Alfa 20 mg/kg | Number of Participants With Potentially Clinically Significant 12-Lead Electrocardiogram (ECG) Abnormalities | HR: >90 bpm and increase from baseline >=20 bpm | 0 Participants |
| Group 1: Avalglucosidase Alfa 20 mg/kg | Number of Participants With Potentially Clinically Significant 12-Lead Electrocardiogram (ECG) Abnormalities | QTcB - Bazett's Correction Formula: >480 msec | 0 Participants |
| Group 1: Avalglucosidase Alfa 20 mg/kg | Number of Participants With Potentially Clinically Significant 12-Lead Electrocardiogram (ECG) Abnormalities | QTcB - Bazett's Correction Formula: >450 msec | 1 Participants |
| Group 1: Avalglucosidase Alfa 20 mg/kg | Number of Participants With Potentially Clinically Significant 12-Lead Electrocardiogram (ECG) Abnormalities | HR: >100 bpm | 1 Participants |
| Group 1: Avalglucosidase Alfa 20 mg/kg | Number of Participants With Potentially Clinically Significant 12-Lead Electrocardiogram (ECG) Abnormalities | QTcB - Bazett's Correction Formula: Increase from baseline (30-60) msec | 1 Participants |
| Group 1: Avalglucosidase Alfa 20 mg/kg | Number of Participants With Potentially Clinically Significant 12-Lead Electrocardiogram (ECG) Abnormalities | HR: <50 bpm | 0 Participants |
| Group 1: Avalglucosidase Alfa 20 mg/kg | Number of Participants With Potentially Clinically Significant 12-Lead Electrocardiogram (ECG) Abnormalities | HR: <50 bpm and decrease from baseline >=20 bpm | 0 Participants |
| Group 1: Avalglucosidase Alfa 20 mg/kg | Number of Participants With Potentially Clinically Significant 12-Lead Electrocardiogram (ECG) Abnormalities | QTcB - Bazett's Correction Formula: >500 msec | 0 Participants |
| Group 2: Avalglucosidase Alfa 5 mg/kg | Number of Participants With Potentially Clinically Significant 12-Lead Electrocardiogram (ECG) Abnormalities | QTcB - Bazett's Correction Formula: >450 msec | 2 Participants |
| Group 2: Avalglucosidase Alfa 5 mg/kg | Number of Participants With Potentially Clinically Significant 12-Lead Electrocardiogram (ECG) Abnormalities | QTcB - Bazett's Correction Formula: >480 msec | 0 Participants |
| Group 2: Avalglucosidase Alfa 5 mg/kg | Number of Participants With Potentially Clinically Significant 12-Lead Electrocardiogram (ECG) Abnormalities | HR: >120 bpm and increase from baseline >=20 bpm | 0 Participants |
| Group 2: Avalglucosidase Alfa 5 mg/kg | Number of Participants With Potentially Clinically Significant 12-Lead Electrocardiogram (ECG) Abnormalities | PR Duration: > 200 millisecond (msec) | 0 Participants |
| Group 2: Avalglucosidase Alfa 5 mg/kg | Number of Participants With Potentially Clinically Significant 12-Lead Electrocardiogram (ECG) Abnormalities | PR Duration: >200 msec and increase from baseline >=25% | 0 Participants |
| Group 2: Avalglucosidase Alfa 5 mg/kg | Number of Participants With Potentially Clinically Significant 12-Lead Electrocardiogram (ECG) Abnormalities | PR Duration: >240 msec and increase from baseline >=25% | 0 Participants |
| Group 2: Avalglucosidase Alfa 5 mg/kg | Number of Participants With Potentially Clinically Significant 12-Lead Electrocardiogram (ECG) Abnormalities | PR Duration: >220 msec and increase from baseline >=25% | 1 Participants |
| Group 2: Avalglucosidase Alfa 5 mg/kg | Number of Participants With Potentially Clinically Significant 12-Lead Electrocardiogram (ECG) Abnormalities | QRS Duration: >110 msec | 0 Participants |
| Group 2: Avalglucosidase Alfa 5 mg/kg | Number of Participants With Potentially Clinically Significant 12-Lead Electrocardiogram (ECG) Abnormalities | PR Duration: >220 msec | 1 Participants |
| Group 2: Avalglucosidase Alfa 5 mg/kg | Number of Participants With Potentially Clinically Significant 12-Lead Electrocardiogram (ECG) Abnormalities | QRS Duration: >110 msec and increase from baseline >=25% | 0 Participants |
| Group 2: Avalglucosidase Alfa 5 mg/kg | Number of Participants With Potentially Clinically Significant 12-Lead Electrocardiogram (ECG) Abnormalities | QRS Duration: >120 msec | 1 Participants |
| Group 2: Avalglucosidase Alfa 5 mg/kg | Number of Participants With Potentially Clinically Significant 12-Lead Electrocardiogram (ECG) Abnormalities | QRS Duration: >120 msec and increase from baseline >=25% | 0 Participants |
| Group 2: Avalglucosidase Alfa 5 mg/kg | Number of Participants With Potentially Clinically Significant 12-Lead Electrocardiogram (ECG) Abnormalities | PR Duration: >240 msec | 0 Participants |
| Group 2: Avalglucosidase Alfa 5 mg/kg | Number of Participants With Potentially Clinically Significant 12-Lead Electrocardiogram (ECG) Abnormalities | QTcB - Bazett's Correction Formula: Increase from baseline (30-60) msec | 1 Participants |
| Group 2: Avalglucosidase Alfa 5 mg/kg | Number of Participants With Potentially Clinically Significant 12-Lead Electrocardiogram (ECG) Abnormalities | HR: <50 bpm | 0 Participants |
| Group 2: Avalglucosidase Alfa 5 mg/kg | Number of Participants With Potentially Clinically Significant 12-Lead Electrocardiogram (ECG) Abnormalities | HR: <50 bpm and decrease from baseline >=20 bpm | 0 Participants |
| Group 2: Avalglucosidase Alfa 5 mg/kg | Number of Participants With Potentially Clinically Significant 12-Lead Electrocardiogram (ECG) Abnormalities | HR: <40 bpm | 0 Participants |
| Group 2: Avalglucosidase Alfa 5 mg/kg | Number of Participants With Potentially Clinically Significant 12-Lead Electrocardiogram (ECG) Abnormalities | HR: <40 bpm and decrease from baseline >=20 bpm | 0 Participants |
| Group 2: Avalglucosidase Alfa 5 mg/kg | Number of Participants With Potentially Clinically Significant 12-Lead Electrocardiogram (ECG) Abnormalities | HR: <30 bpm | 0 Participants |
| Group 2: Avalglucosidase Alfa 5 mg/kg | Number of Participants With Potentially Clinically Significant 12-Lead Electrocardiogram (ECG) Abnormalities | HR: <30 bpm and decrease from baseline >=20 bpm | 0 Participants |
| Group 2: Avalglucosidase Alfa 5 mg/kg | Number of Participants With Potentially Clinically Significant 12-Lead Electrocardiogram (ECG) Abnormalities | HR: >90 bpm | 0 Participants |
| Group 2: Avalglucosidase Alfa 5 mg/kg | Number of Participants With Potentially Clinically Significant 12-Lead Electrocardiogram (ECG) Abnormalities | QTcB - Bazett's Correction Formula: >500 msec | 0 Participants |
| Group 2: Avalglucosidase Alfa 5 mg/kg | Number of Participants With Potentially Clinically Significant 12-Lead Electrocardiogram (ECG) Abnormalities | HR: >90 bpm and increase from baseline >=20 bpm | 0 Participants |
| Group 2: Avalglucosidase Alfa 5 mg/kg | Number of Participants With Potentially Clinically Significant 12-Lead Electrocardiogram (ECG) Abnormalities | HR: >100 bpm | 0 Participants |
| Group 2: Avalglucosidase Alfa 5 mg/kg | Number of Participants With Potentially Clinically Significant 12-Lead Electrocardiogram (ECG) Abnormalities | HR: >100 bpm and increase from baseline >=20 bpm | 0 Participants |
| Group 2: Avalglucosidase Alfa 5 mg/kg | Number of Participants With Potentially Clinically Significant 12-Lead Electrocardiogram (ECG) Abnormalities | HR: >120 bpm | 0 Participants |
| Group 2: Avalglucosidase Alfa 5 mg/kg | Number of Participants With Potentially Clinically Significant 12-Lead Electrocardiogram (ECG) Abnormalities | QTcB - Bazett's Correction Formula: Increase from baseline >60 msec | 0 Participants |
| Group 2: Avalglucosidase Alfa 5 mg/kg | Number of Participants With Potentially Clinically Significant 12-Lead Electrocardiogram (ECG) Abnormalities | QTcF - Fridericia's Correction Formula: >450 msec | 1 Participants |
| Group 2: Avalglucosidase Alfa 5 mg/kg | Number of Participants With Potentially Clinically Significant 12-Lead Electrocardiogram (ECG) Abnormalities | QTcF - Fridericia's Correction Formula: >480 msec | 0 Participants |
| Group 2: Avalglucosidase Alfa 5 mg/kg | Number of Participants With Potentially Clinically Significant 12-Lead Electrocardiogram (ECG) Abnormalities | QTcF - Fridericia's Correction Formula: >500 msec | 0 Participants |
| Group 2: Avalglucosidase Alfa 5 mg/kg | Number of Participants With Potentially Clinically Significant 12-Lead Electrocardiogram (ECG) Abnormalities | QTcF - Fridericia's Correction Formula: Increase from baseline (30-60) msec | 0 Participants |
| Group 2: Avalglucosidase Alfa 5 mg/kg | Number of Participants With Potentially Clinically Significant 12-Lead Electrocardiogram (ECG) Abnormalities | QTcF - Fridericia's Correction Formula: Increase from baseline >60 msec | 0 Participants |
| Group 2: Avalglucosidase Alfa 10 mg/kg | Number of Participants With Potentially Clinically Significant 12-Lead Electrocardiogram (ECG) Abnormalities | PR Duration: >200 msec and increase from baseline >=25% | 0 Participants |
| Group 2: Avalglucosidase Alfa 10 mg/kg | Number of Participants With Potentially Clinically Significant 12-Lead Electrocardiogram (ECG) Abnormalities | QTcB - Bazett's Correction Formula: >500 msec | 0 Participants |
| Group 2: Avalglucosidase Alfa 10 mg/kg | Number of Participants With Potentially Clinically Significant 12-Lead Electrocardiogram (ECG) Abnormalities | HR: <50 bpm | 1 Participants |
| Group 2: Avalglucosidase Alfa 10 mg/kg | Number of Participants With Potentially Clinically Significant 12-Lead Electrocardiogram (ECG) Abnormalities | QTcB - Bazett's Correction Formula: Increase from baseline (30-60) msec | 1 Participants |
| Group 2: Avalglucosidase Alfa 10 mg/kg | Number of Participants With Potentially Clinically Significant 12-Lead Electrocardiogram (ECG) Abnormalities | HR: >120 bpm and increase from baseline >=20 bpm | 0 Participants |
| Group 2: Avalglucosidase Alfa 10 mg/kg | Number of Participants With Potentially Clinically Significant 12-Lead Electrocardiogram (ECG) Abnormalities | QTcF - Fridericia's Correction Formula: Increase from baseline >60 msec | 1 Participants |
| Group 2: Avalglucosidase Alfa 10 mg/kg | Number of Participants With Potentially Clinically Significant 12-Lead Electrocardiogram (ECG) Abnormalities | HR: >90 bpm and increase from baseline >=20 bpm | 0 Participants |
| Group 2: Avalglucosidase Alfa 10 mg/kg | Number of Participants With Potentially Clinically Significant 12-Lead Electrocardiogram (ECG) Abnormalities | QRS Duration: >120 msec and increase from baseline >=25% | 0 Participants |
| Group 2: Avalglucosidase Alfa 10 mg/kg | Number of Participants With Potentially Clinically Significant 12-Lead Electrocardiogram (ECG) Abnormalities | HR: >100 bpm | 0 Participants |
| Group 2: Avalglucosidase Alfa 10 mg/kg | Number of Participants With Potentially Clinically Significant 12-Lead Electrocardiogram (ECG) Abnormalities | QRS Duration: >120 msec | 0 Participants |
| Group 2: Avalglucosidase Alfa 10 mg/kg | Number of Participants With Potentially Clinically Significant 12-Lead Electrocardiogram (ECG) Abnormalities | QTcF - Fridericia's Correction Formula: Increase from baseline (30-60) msec | 0 Participants |
| Group 2: Avalglucosidase Alfa 10 mg/kg | Number of Participants With Potentially Clinically Significant 12-Lead Electrocardiogram (ECG) Abnormalities | HR: >100 bpm and increase from baseline >=20 bpm | 0 Participants |
| Group 2: Avalglucosidase Alfa 10 mg/kg | Number of Participants With Potentially Clinically Significant 12-Lead Electrocardiogram (ECG) Abnormalities | QRS Duration: >110 msec and increase from baseline >=25% | 0 Participants |
| Group 2: Avalglucosidase Alfa 10 mg/kg | Number of Participants With Potentially Clinically Significant 12-Lead Electrocardiogram (ECG) Abnormalities | HR: >120 bpm | 0 Participants |
| Group 2: Avalglucosidase Alfa 10 mg/kg | Number of Participants With Potentially Clinically Significant 12-Lead Electrocardiogram (ECG) Abnormalities | PR Duration: >220 msec | 0 Participants |
| Group 2: Avalglucosidase Alfa 10 mg/kg | Number of Participants With Potentially Clinically Significant 12-Lead Electrocardiogram (ECG) Abnormalities | QTcB - Bazett's Correction Formula: >480 msec | 0 Participants |
| Group 2: Avalglucosidase Alfa 10 mg/kg | Number of Participants With Potentially Clinically Significant 12-Lead Electrocardiogram (ECG) Abnormalities | QTcB - Bazett's Correction Formula: Increase from baseline >60 msec | 1 Participants |
| Group 2: Avalglucosidase Alfa 10 mg/kg | Number of Participants With Potentially Clinically Significant 12-Lead Electrocardiogram (ECG) Abnormalities | QRS Duration: >110 msec | 3 Participants |
| Group 2: Avalglucosidase Alfa 10 mg/kg | Number of Participants With Potentially Clinically Significant 12-Lead Electrocardiogram (ECG) Abnormalities | PR Duration: >240 msec and increase from baseline >=25% | 0 Participants |
| Group 2: Avalglucosidase Alfa 10 mg/kg | Number of Participants With Potentially Clinically Significant 12-Lead Electrocardiogram (ECG) Abnormalities | QTcF - Fridericia's Correction Formula: >450 msec | 1 Participants |
| Group 2: Avalglucosidase Alfa 10 mg/kg | Number of Participants With Potentially Clinically Significant 12-Lead Electrocardiogram (ECG) Abnormalities | PR Duration: >220 msec and increase from baseline >=25% | 0 Participants |
| Group 2: Avalglucosidase Alfa 10 mg/kg | Number of Participants With Potentially Clinically Significant 12-Lead Electrocardiogram (ECG) Abnormalities | PR Duration: >240 msec | 0 Participants |
| Group 2: Avalglucosidase Alfa 10 mg/kg | Number of Participants With Potentially Clinically Significant 12-Lead Electrocardiogram (ECG) Abnormalities | QTcB - Bazett's Correction Formula: >450 msec | 1 Participants |
| Group 2: Avalglucosidase Alfa 10 mg/kg | Number of Participants With Potentially Clinically Significant 12-Lead Electrocardiogram (ECG) Abnormalities | QTcF - Fridericia's Correction Formula: >480 msec | 0 Participants |
| Group 2: Avalglucosidase Alfa 10 mg/kg | Number of Participants With Potentially Clinically Significant 12-Lead Electrocardiogram (ECG) Abnormalities | HR: <50 bpm and decrease from baseline >=20 bpm | 0 Participants |
| Group 2: Avalglucosidase Alfa 10 mg/kg | Number of Participants With Potentially Clinically Significant 12-Lead Electrocardiogram (ECG) Abnormalities | QTcF - Fridericia's Correction Formula: >500 msec | 0 Participants |
| Group 2: Avalglucosidase Alfa 10 mg/kg | Number of Participants With Potentially Clinically Significant 12-Lead Electrocardiogram (ECG) Abnormalities | HR: <30 bpm and decrease from baseline >=20 bpm | 0 Participants |
| Group 2: Avalglucosidase Alfa 10 mg/kg | Number of Participants With Potentially Clinically Significant 12-Lead Electrocardiogram (ECG) Abnormalities | HR: <30 bpm | 0 Participants |
| Group 2: Avalglucosidase Alfa 10 mg/kg | Number of Participants With Potentially Clinically Significant 12-Lead Electrocardiogram (ECG) Abnormalities | PR Duration: > 200 millisecond (msec) | 0 Participants |
| Group 2: Avalglucosidase Alfa 10 mg/kg | Number of Participants With Potentially Clinically Significant 12-Lead Electrocardiogram (ECG) Abnormalities | HR: >90 bpm | 0 Participants |
| Group 2: Avalglucosidase Alfa 10 mg/kg | Number of Participants With Potentially Clinically Significant 12-Lead Electrocardiogram (ECG) Abnormalities | HR: <40 bpm and decrease from baseline >=20 bpm | 0 Participants |
| Group 2: Avalglucosidase Alfa 10 mg/kg | Number of Participants With Potentially Clinically Significant 12-Lead Electrocardiogram (ECG) Abnormalities | HR: <40 bpm | 0 Participants |
| Group 2: Avalglucosidase Alfa 20 mg/kg | Number of Participants With Potentially Clinically Significant 12-Lead Electrocardiogram (ECG) Abnormalities | PR Duration: > 200 millisecond (msec) | 1 Participants |
| Group 2: Avalglucosidase Alfa 20 mg/kg | Number of Participants With Potentially Clinically Significant 12-Lead Electrocardiogram (ECG) Abnormalities | HR: >120 bpm and increase from baseline >=20 bpm | 0 Participants |
| Group 2: Avalglucosidase Alfa 20 mg/kg | Number of Participants With Potentially Clinically Significant 12-Lead Electrocardiogram (ECG) Abnormalities | QTcB - Bazett's Correction Formula: Increase from baseline >60 msec | 0 Participants |
| Group 2: Avalglucosidase Alfa 20 mg/kg | Number of Participants With Potentially Clinically Significant 12-Lead Electrocardiogram (ECG) Abnormalities | QTcB - Bazett's Correction Formula: >500 msec | 0 Participants |
| Group 2: Avalglucosidase Alfa 20 mg/kg | Number of Participants With Potentially Clinically Significant 12-Lead Electrocardiogram (ECG) Abnormalities | HR: <50 bpm | 0 Participants |
| Group 2: Avalglucosidase Alfa 20 mg/kg | Number of Participants With Potentially Clinically Significant 12-Lead Electrocardiogram (ECG) Abnormalities | QTcF - Fridericia's Correction Formula: Increase from baseline >60 msec | 0 Participants |
| Group 2: Avalglucosidase Alfa 20 mg/kg | Number of Participants With Potentially Clinically Significant 12-Lead Electrocardiogram (ECG) Abnormalities | QTcB - Bazett's Correction Formula: Increase from baseline (30-60) msec | 4 Participants |
| Group 2: Avalglucosidase Alfa 20 mg/kg | Number of Participants With Potentially Clinically Significant 12-Lead Electrocardiogram (ECG) Abnormalities | PR Duration: >240 msec and increase from baseline >=25% | 0 Participants |
| Group 2: Avalglucosidase Alfa 20 mg/kg | Number of Participants With Potentially Clinically Significant 12-Lead Electrocardiogram (ECG) Abnormalities | QTcB - Bazett's Correction Formula: >480 msec | 0 Participants |
| Group 2: Avalglucosidase Alfa 20 mg/kg | Number of Participants With Potentially Clinically Significant 12-Lead Electrocardiogram (ECG) Abnormalities | HR: <50 bpm and decrease from baseline >=20 bpm | 0 Participants |
| Group 2: Avalglucosidase Alfa 20 mg/kg | Number of Participants With Potentially Clinically Significant 12-Lead Electrocardiogram (ECG) Abnormalities | QTcB - Bazett's Correction Formula: >450 msec | 3 Participants |
| Group 2: Avalglucosidase Alfa 20 mg/kg | Number of Participants With Potentially Clinically Significant 12-Lead Electrocardiogram (ECG) Abnormalities | QTcF - Fridericia's Correction Formula: >500 msec | 0 Participants |
| Group 2: Avalglucosidase Alfa 20 mg/kg | Number of Participants With Potentially Clinically Significant 12-Lead Electrocardiogram (ECG) Abnormalities | HR: >90 bpm and increase from baseline >=20 bpm | 0 Participants |
| Group 2: Avalglucosidase Alfa 20 mg/kg | Number of Participants With Potentially Clinically Significant 12-Lead Electrocardiogram (ECG) Abnormalities | QRS Duration: >120 msec and increase from baseline >=25% | 0 Participants |
| Group 2: Avalglucosidase Alfa 20 mg/kg | Number of Participants With Potentially Clinically Significant 12-Lead Electrocardiogram (ECG) Abnormalities | QTcF - Fridericia's Correction Formula: >450 msec | 0 Participants |
| Group 2: Avalglucosidase Alfa 20 mg/kg | Number of Participants With Potentially Clinically Significant 12-Lead Electrocardiogram (ECG) Abnormalities | HR: <40 bpm | 0 Participants |
| Group 2: Avalglucosidase Alfa 20 mg/kg | Number of Participants With Potentially Clinically Significant 12-Lead Electrocardiogram (ECG) Abnormalities | HR: >100 bpm | 0 Participants |
| Group 2: Avalglucosidase Alfa 20 mg/kg | Number of Participants With Potentially Clinically Significant 12-Lead Electrocardiogram (ECG) Abnormalities | PR Duration: >220 msec and increase from baseline >=25% | 0 Participants |
| Group 2: Avalglucosidase Alfa 20 mg/kg | Number of Participants With Potentially Clinically Significant 12-Lead Electrocardiogram (ECG) Abnormalities | QRS Duration: >120 msec | 0 Participants |
| Group 2: Avalglucosidase Alfa 20 mg/kg | Number of Participants With Potentially Clinically Significant 12-Lead Electrocardiogram (ECG) Abnormalities | PR Duration: >240 msec | 0 Participants |
| Group 2: Avalglucosidase Alfa 20 mg/kg | Number of Participants With Potentially Clinically Significant 12-Lead Electrocardiogram (ECG) Abnormalities | HR: <30 bpm and decrease from baseline >=20 bpm | 0 Participants |
| Group 2: Avalglucosidase Alfa 20 mg/kg | Number of Participants With Potentially Clinically Significant 12-Lead Electrocardiogram (ECG) Abnormalities | HR: <30 bpm | 0 Participants |
| Group 2: Avalglucosidase Alfa 20 mg/kg | Number of Participants With Potentially Clinically Significant 12-Lead Electrocardiogram (ECG) Abnormalities | HR: >100 bpm and increase from baseline >=20 bpm | 0 Participants |
| Group 2: Avalglucosidase Alfa 20 mg/kg | Number of Participants With Potentially Clinically Significant 12-Lead Electrocardiogram (ECG) Abnormalities | QRS Duration: >110 msec and increase from baseline >=25% | 0 Participants |
| Group 2: Avalglucosidase Alfa 20 mg/kg | Number of Participants With Potentially Clinically Significant 12-Lead Electrocardiogram (ECG) Abnormalities | HR: >90 bpm | 2 Participants |
| Group 2: Avalglucosidase Alfa 20 mg/kg | Number of Participants With Potentially Clinically Significant 12-Lead Electrocardiogram (ECG) Abnormalities | PR Duration: >220 msec | 0 Participants |
| Group 2: Avalglucosidase Alfa 20 mg/kg | Number of Participants With Potentially Clinically Significant 12-Lead Electrocardiogram (ECG) Abnormalities | QTcF - Fridericia's Correction Formula: Increase from baseline (30-60) msec | 1 Participants |
| Group 2: Avalglucosidase Alfa 20 mg/kg | Number of Participants With Potentially Clinically Significant 12-Lead Electrocardiogram (ECG) Abnormalities | QTcF - Fridericia's Correction Formula: >480 msec | 0 Participants |
| Group 2: Avalglucosidase Alfa 20 mg/kg | Number of Participants With Potentially Clinically Significant 12-Lead Electrocardiogram (ECG) Abnormalities | HR: >120 bpm | 0 Participants |
| Group 2: Avalglucosidase Alfa 20 mg/kg | Number of Participants With Potentially Clinically Significant 12-Lead Electrocardiogram (ECG) Abnormalities | PR Duration: >200 msec and increase from baseline >=25% | 0 Participants |
| Group 2: Avalglucosidase Alfa 20 mg/kg | Number of Participants With Potentially Clinically Significant 12-Lead Electrocardiogram (ECG) Abnormalities | QRS Duration: >110 msec | 4 Participants |
| Group 2: Avalglucosidase Alfa 20 mg/kg | Number of Participants With Potentially Clinically Significant 12-Lead Electrocardiogram (ECG) Abnormalities | HR: <40 bpm and decrease from baseline >=20 bpm | 0 Participants |
Primary
Number of Participants With Potentially Clinically Significant Abnormalities in Biochemistry
Blood samples were collected to determine the clinical chemistry laboratory abnormalities.
Time frame: From first dose of IMP up to 4 weeks after the last treatment administration of the IMP, a maximum up to 458 weeks
Population: The safety analysis set included all participants who received at least 1 complete infusion of IMP.
| Arm | Measure | Group | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|---|
| Group 1: Avalglucosidase Alfa 5 mg/kg | Number of Participants With Potentially Clinically Significant Abnormalities in Biochemistry | Creatinine: >=30% change from baseline | 0 Participants |
| Group 1: Avalglucosidase Alfa 5 mg/kg | Number of Participants With Potentially Clinically Significant Abnormalities in Biochemistry | Alanine Aminotransferase > 3 ULN and Bilirubin > 2 ULN | 0 Participants |
| Group 1: Avalglucosidase Alfa 5 mg/kg | Number of Participants With Potentially Clinically Significant Abnormalities in Biochemistry | Creatinine: >=100% change from baseline | 0 Participants |
| Group 1: Avalglucosidase Alfa 5 mg/kg | Number of Participants With Potentially Clinically Significant Abnormalities in Biochemistry | Alanine Aminotransferase: >5 ULN | 0 Participants |
| Group 1: Avalglucosidase Alfa 5 mg/kg | Number of Participants With Potentially Clinically Significant Abnormalities in Biochemistry | Sodium: >=160 mmol/L | 0 Participants |
| Group 1: Avalglucosidase Alfa 5 mg/kg | Number of Participants With Potentially Clinically Significant Abnormalities in Biochemistry | Total Bilirubin: >2 ULN | 0 Participants |
| Group 1: Avalglucosidase Alfa 5 mg/kg | Number of Participants With Potentially Clinically Significant Abnormalities in Biochemistry | Albumin: <=25 gram per liter (g/L) | 0 Participants |
| Group 1: Avalglucosidase Alfa 5 mg/kg | Number of Participants With Potentially Clinically Significant Abnormalities in Biochemistry | Glucose: >=11.1 mmol/L (unfasted); >=7 mmol/L (fasted) | 2 Participants |
| Group 1: Avalglucosidase Alfa 5 mg/kg | Number of Participants With Potentially Clinically Significant Abnormalities in Biochemistry | Alanine Aminotransferase: >3 ULN | 0 Participants |
| Group 1: Avalglucosidase Alfa 5 mg/kg | Number of Participants With Potentially Clinically Significant Abnormalities in Biochemistry | Glucose: <=3.9 millimoles per liter (mmol/L) and < lower limit of normal (LLN) | 2 Participants |
| Group 1: Avalglucosidase Alfa 5 mg/kg | Number of Participants With Potentially Clinically Significant Abnormalities in Biochemistry | Potassium: <3 mmol/L | 0 Participants |
| Group 1: Avalglucosidase Alfa 5 mg/kg | Number of Participants With Potentially Clinically Significant Abnormalities in Biochemistry | Direct Bilirubin > 35% Bilirubin and Bilirubin > 1.5 ULN | 0 Participants |
| Group 1: Avalglucosidase Alfa 5 mg/kg | Number of Participants With Potentially Clinically Significant Abnormalities in Biochemistry | Aspartate Aminotransferase: >10 ULN | 0 Participants |
| Group 1: Avalglucosidase Alfa 5 mg/kg | Number of Participants With Potentially Clinically Significant Abnormalities in Biochemistry | Total Bilirubin: >1.5 ULN | 1 Participants |
| Group 1: Avalglucosidase Alfa 5 mg/kg | Number of Participants With Potentially Clinically Significant Abnormalities in Biochemistry | Aspartate Aminotransferase: >5 ULN | 0 Participants |
| Group 1: Avalglucosidase Alfa 5 mg/kg | Number of Participants With Potentially Clinically Significant Abnormalities in Biochemistry | Chloride: >115 mmol/L | 0 Participants |
| Group 1: Avalglucosidase Alfa 5 mg/kg | Number of Participants With Potentially Clinically Significant Abnormalities in Biochemistry | Potassium: >=5.5 mmol/L | 0 Participants |
| Group 1: Avalglucosidase Alfa 5 mg/kg | Number of Participants With Potentially Clinically Significant Abnormalities in Biochemistry | Alkaline Phosphatase: >1.5 ULN | 0 Participants |
| Group 1: Avalglucosidase Alfa 5 mg/kg | Number of Participants With Potentially Clinically Significant Abnormalities in Biochemistry | Alanine Aminotransferase: >10 ULN | 0 Participants |
| Group 1: Avalglucosidase Alfa 5 mg/kg | Number of Participants With Potentially Clinically Significant Abnormalities in Biochemistry | Aspartate Aminotransferase: >3 upper limit of normal (ULN) | 0 Participants |
| Group 1: Avalglucosidase Alfa 5 mg/kg | Number of Participants With Potentially Clinically Significant Abnormalities in Biochemistry | Sodium: <=129 mmol/L | 0 Participants |
| Group 1: Avalglucosidase Alfa 5 mg/kg | Number of Participants With Potentially Clinically Significant Abnormalities in Biochemistry | Urea Nitrogen: >=17 mmol/L | 0 Participants |
| Group 1: Avalglucosidase Alfa 5 mg/kg | Number of Participants With Potentially Clinically Significant Abnormalities in Biochemistry | Chloride: <80 mmol/L | 0 Participants |
| Group 1: Avalglucosidase Alfa 5 mg/kg | Number of Participants With Potentially Clinically Significant Abnormalities in Biochemistry | Creatinine: >=150 micromoles/L (Adults) | 0 Participants |
| Group 1: Avalglucosidase Alfa 10 mg/kg | Number of Participants With Potentially Clinically Significant Abnormalities in Biochemistry | Chloride: <80 mmol/L | 0 Participants |
| Group 1: Avalglucosidase Alfa 10 mg/kg | Number of Participants With Potentially Clinically Significant Abnormalities in Biochemistry | Creatinine: >=100% change from baseline | 0 Participants |
| Group 1: Avalglucosidase Alfa 10 mg/kg | Number of Participants With Potentially Clinically Significant Abnormalities in Biochemistry | Creatinine: >=150 micromoles/L (Adults) | 0 Participants |
| Group 1: Avalglucosidase Alfa 10 mg/kg | Number of Participants With Potentially Clinically Significant Abnormalities in Biochemistry | Chloride: >115 mmol/L | 0 Participants |
| Group 1: Avalglucosidase Alfa 10 mg/kg | Number of Participants With Potentially Clinically Significant Abnormalities in Biochemistry | Alanine Aminotransferase > 3 ULN and Bilirubin > 2 ULN | 0 Participants |
| Group 1: Avalglucosidase Alfa 10 mg/kg | Number of Participants With Potentially Clinically Significant Abnormalities in Biochemistry | Albumin: <=25 gram per liter (g/L) | 0 Participants |
| Group 1: Avalglucosidase Alfa 10 mg/kg | Number of Participants With Potentially Clinically Significant Abnormalities in Biochemistry | Glucose: <=3.9 millimoles per liter (mmol/L) and < lower limit of normal (LLN) | 2 Participants |
| Group 1: Avalglucosidase Alfa 10 mg/kg | Number of Participants With Potentially Clinically Significant Abnormalities in Biochemistry | Direct Bilirubin > 35% Bilirubin and Bilirubin > 1.5 ULN | 0 Participants |
| Group 1: Avalglucosidase Alfa 10 mg/kg | Number of Participants With Potentially Clinically Significant Abnormalities in Biochemistry | Total Bilirubin: >2 ULN | 0 Participants |
| Group 1: Avalglucosidase Alfa 10 mg/kg | Number of Participants With Potentially Clinically Significant Abnormalities in Biochemistry | Total Bilirubin: >1.5 ULN | 0 Participants |
| Group 1: Avalglucosidase Alfa 10 mg/kg | Number of Participants With Potentially Clinically Significant Abnormalities in Biochemistry | Glucose: >=11.1 mmol/L (unfasted); >=7 mmol/L (fasted) | 0 Participants |
| Group 1: Avalglucosidase Alfa 10 mg/kg | Number of Participants With Potentially Clinically Significant Abnormalities in Biochemistry | Alkaline Phosphatase: >1.5 ULN | 0 Participants |
| Group 1: Avalglucosidase Alfa 10 mg/kg | Number of Participants With Potentially Clinically Significant Abnormalities in Biochemistry | Sodium: <=129 mmol/L | 0 Participants |
| Group 1: Avalglucosidase Alfa 10 mg/kg | Number of Participants With Potentially Clinically Significant Abnormalities in Biochemistry | Alanine Aminotransferase: >5 ULN | 1 Participants |
| Group 1: Avalglucosidase Alfa 10 mg/kg | Number of Participants With Potentially Clinically Significant Abnormalities in Biochemistry | Sodium: >=160 mmol/L | 0 Participants |
| Group 1: Avalglucosidase Alfa 10 mg/kg | Number of Participants With Potentially Clinically Significant Abnormalities in Biochemistry | Creatinine: >=30% change from baseline | 0 Participants |
| Group 1: Avalglucosidase Alfa 10 mg/kg | Number of Participants With Potentially Clinically Significant Abnormalities in Biochemistry | Alanine Aminotransferase: >3 ULN | 2 Participants |
| Group 1: Avalglucosidase Alfa 10 mg/kg | Number of Participants With Potentially Clinically Significant Abnormalities in Biochemistry | Aspartate Aminotransferase: >10 ULN | 0 Participants |
| Group 1: Avalglucosidase Alfa 10 mg/kg | Number of Participants With Potentially Clinically Significant Abnormalities in Biochemistry | Potassium: <3 mmol/L | 0 Participants |
| Group 1: Avalglucosidase Alfa 10 mg/kg | Number of Participants With Potentially Clinically Significant Abnormalities in Biochemistry | Aspartate Aminotransferase: >5 ULN | 1 Participants |
| Group 1: Avalglucosidase Alfa 10 mg/kg | Number of Participants With Potentially Clinically Significant Abnormalities in Biochemistry | Aspartate Aminotransferase: >3 upper limit of normal (ULN) | 2 Participants |
| Group 1: Avalglucosidase Alfa 10 mg/kg | Number of Participants With Potentially Clinically Significant Abnormalities in Biochemistry | Potassium: >=5.5 mmol/L | 0 Participants |
| Group 1: Avalglucosidase Alfa 10 mg/kg | Number of Participants With Potentially Clinically Significant Abnormalities in Biochemistry | Urea Nitrogen: >=17 mmol/L | 0 Participants |
| Group 1: Avalglucosidase Alfa 10 mg/kg | Number of Participants With Potentially Clinically Significant Abnormalities in Biochemistry | Alanine Aminotransferase: >10 ULN | 0 Participants |
| Group 1: Avalglucosidase Alfa 20 mg/kg | Number of Participants With Potentially Clinically Significant Abnormalities in Biochemistry | Potassium: <3 mmol/L | 0 Participants |
| Group 1: Avalglucosidase Alfa 20 mg/kg | Number of Participants With Potentially Clinically Significant Abnormalities in Biochemistry | Alanine Aminotransferase > 3 ULN and Bilirubin > 2 ULN | 0 Participants |
| Group 1: Avalglucosidase Alfa 20 mg/kg | Number of Participants With Potentially Clinically Significant Abnormalities in Biochemistry | Glucose: <=3.9 millimoles per liter (mmol/L) and < lower limit of normal (LLN) | 2 Participants |
| Group 1: Avalglucosidase Alfa 20 mg/kg | Number of Participants With Potentially Clinically Significant Abnormalities in Biochemistry | Glucose: >=11.1 mmol/L (unfasted); >=7 mmol/L (fasted) | 1 Participants |
| Group 1: Avalglucosidase Alfa 20 mg/kg | Number of Participants With Potentially Clinically Significant Abnormalities in Biochemistry | Sodium: <=129 mmol/L | 0 Participants |
| Group 1: Avalglucosidase Alfa 20 mg/kg | Number of Participants With Potentially Clinically Significant Abnormalities in Biochemistry | Sodium: >=160 mmol/L | 0 Participants |
| Group 1: Avalglucosidase Alfa 20 mg/kg | Number of Participants With Potentially Clinically Significant Abnormalities in Biochemistry | Potassium: >=5.5 mmol/L | 0 Participants |
| Group 1: Avalglucosidase Alfa 20 mg/kg | Number of Participants With Potentially Clinically Significant Abnormalities in Biochemistry | Chloride: <80 mmol/L | 0 Participants |
| Group 1: Avalglucosidase Alfa 20 mg/kg | Number of Participants With Potentially Clinically Significant Abnormalities in Biochemistry | Chloride: >115 mmol/L | 0 Participants |
| Group 1: Avalglucosidase Alfa 20 mg/kg | Number of Participants With Potentially Clinically Significant Abnormalities in Biochemistry | Albumin: <=25 gram per liter (g/L) | 0 Participants |
| Group 1: Avalglucosidase Alfa 20 mg/kg | Number of Participants With Potentially Clinically Significant Abnormalities in Biochemistry | Creatinine: >=150 micromoles/L (Adults) | 0 Participants |
| Group 1: Avalglucosidase Alfa 20 mg/kg | Number of Participants With Potentially Clinically Significant Abnormalities in Biochemistry | Creatinine: >=100% change from baseline | 0 Participants |
| Group 1: Avalglucosidase Alfa 20 mg/kg | Number of Participants With Potentially Clinically Significant Abnormalities in Biochemistry | Creatinine: >=30% change from baseline | 0 Participants |
| Group 1: Avalglucosidase Alfa 20 mg/kg | Number of Participants With Potentially Clinically Significant Abnormalities in Biochemistry | Urea Nitrogen: >=17 mmol/L | 1 Participants |
| Group 1: Avalglucosidase Alfa 20 mg/kg | Number of Participants With Potentially Clinically Significant Abnormalities in Biochemistry | Aspartate Aminotransferase: >3 upper limit of normal (ULN) | 0 Participants |
| Group 1: Avalglucosidase Alfa 20 mg/kg | Number of Participants With Potentially Clinically Significant Abnormalities in Biochemistry | Aspartate Aminotransferase: >5 ULN | 0 Participants |
| Group 1: Avalglucosidase Alfa 20 mg/kg | Number of Participants With Potentially Clinically Significant Abnormalities in Biochemistry | Aspartate Aminotransferase: >10 ULN | 0 Participants |
| Group 1: Avalglucosidase Alfa 20 mg/kg | Number of Participants With Potentially Clinically Significant Abnormalities in Biochemistry | Alanine Aminotransferase: >3 ULN | 0 Participants |
| Group 1: Avalglucosidase Alfa 20 mg/kg | Number of Participants With Potentially Clinically Significant Abnormalities in Biochemistry | Alanine Aminotransferase: >5 ULN | 0 Participants |
| Group 1: Avalglucosidase Alfa 20 mg/kg | Number of Participants With Potentially Clinically Significant Abnormalities in Biochemistry | Alanine Aminotransferase: >10 ULN | 0 Participants |
| Group 1: Avalglucosidase Alfa 20 mg/kg | Number of Participants With Potentially Clinically Significant Abnormalities in Biochemistry | Alkaline Phosphatase: >1.5 ULN | 0 Participants |
| Group 1: Avalglucosidase Alfa 20 mg/kg | Number of Participants With Potentially Clinically Significant Abnormalities in Biochemistry | Total Bilirubin: >1.5 ULN | 1 Participants |
| Group 1: Avalglucosidase Alfa 20 mg/kg | Number of Participants With Potentially Clinically Significant Abnormalities in Biochemistry | Total Bilirubin: >2 ULN | 0 Participants |
| Group 1: Avalglucosidase Alfa 20 mg/kg | Number of Participants With Potentially Clinically Significant Abnormalities in Biochemistry | Direct Bilirubin > 35% Bilirubin and Bilirubin > 1.5 ULN | 0 Participants |
| Group 2: Avalglucosidase Alfa 5 mg/kg | Number of Participants With Potentially Clinically Significant Abnormalities in Biochemistry | Albumin: <=25 gram per liter (g/L) | 0 Participants |
| Group 2: Avalglucosidase Alfa 5 mg/kg | Number of Participants With Potentially Clinically Significant Abnormalities in Biochemistry | Aspartate Aminotransferase: >10 ULN | 0 Participants |
| Group 2: Avalglucosidase Alfa 5 mg/kg | Number of Participants With Potentially Clinically Significant Abnormalities in Biochemistry | Aspartate Aminotransferase: >5 ULN | 0 Participants |
| Group 2: Avalglucosidase Alfa 5 mg/kg | Number of Participants With Potentially Clinically Significant Abnormalities in Biochemistry | Creatinine: >=30% change from baseline | 1 Participants |
| Group 2: Avalglucosidase Alfa 5 mg/kg | Number of Participants With Potentially Clinically Significant Abnormalities in Biochemistry | Glucose: <=3.9 millimoles per liter (mmol/L) and < lower limit of normal (LLN) | 1 Participants |
| Group 2: Avalglucosidase Alfa 5 mg/kg | Number of Participants With Potentially Clinically Significant Abnormalities in Biochemistry | Sodium: >=160 mmol/L | 0 Participants |
| Group 2: Avalglucosidase Alfa 5 mg/kg | Number of Participants With Potentially Clinically Significant Abnormalities in Biochemistry | Potassium: >=5.5 mmol/L | 0 Participants |
| Group 2: Avalglucosidase Alfa 5 mg/kg | Number of Participants With Potentially Clinically Significant Abnormalities in Biochemistry | Alanine Aminotransferase: >3 ULN | 1 Participants |
| Group 2: Avalglucosidase Alfa 5 mg/kg | Number of Participants With Potentially Clinically Significant Abnormalities in Biochemistry | Chloride: >115 mmol/L | 0 Participants |
| Group 2: Avalglucosidase Alfa 5 mg/kg | Number of Participants With Potentially Clinically Significant Abnormalities in Biochemistry | Urea Nitrogen: >=17 mmol/L | 0 Participants |
| Group 2: Avalglucosidase Alfa 5 mg/kg | Number of Participants With Potentially Clinically Significant Abnormalities in Biochemistry | Glucose: >=11.1 mmol/L (unfasted); >=7 mmol/L (fasted) | 1 Participants |
| Group 2: Avalglucosidase Alfa 5 mg/kg | Number of Participants With Potentially Clinically Significant Abnormalities in Biochemistry | Creatinine: >=150 micromoles/L (Adults) | 0 Participants |
| Group 2: Avalglucosidase Alfa 5 mg/kg | Number of Participants With Potentially Clinically Significant Abnormalities in Biochemistry | Aspartate Aminotransferase: >3 upper limit of normal (ULN) | 1 Participants |
| Group 2: Avalglucosidase Alfa 5 mg/kg | Number of Participants With Potentially Clinically Significant Abnormalities in Biochemistry | Alanine Aminotransferase: >5 ULN | 0 Participants |
| Group 2: Avalglucosidase Alfa 5 mg/kg | Number of Participants With Potentially Clinically Significant Abnormalities in Biochemistry | Alkaline Phosphatase: >1.5 ULN | 0 Participants |
| Group 2: Avalglucosidase Alfa 5 mg/kg | Number of Participants With Potentially Clinically Significant Abnormalities in Biochemistry | Chloride: <80 mmol/L | 0 Participants |
| Group 2: Avalglucosidase Alfa 5 mg/kg | Number of Participants With Potentially Clinically Significant Abnormalities in Biochemistry | Direct Bilirubin > 35% Bilirubin and Bilirubin > 1.5 ULN | 0 Participants |
| Group 2: Avalglucosidase Alfa 5 mg/kg | Number of Participants With Potentially Clinically Significant Abnormalities in Biochemistry | Alanine Aminotransferase: >10 ULN | 0 Participants |
| Group 2: Avalglucosidase Alfa 5 mg/kg | Number of Participants With Potentially Clinically Significant Abnormalities in Biochemistry | Creatinine: >=100% change from baseline | 0 Participants |
| Group 2: Avalglucosidase Alfa 5 mg/kg | Number of Participants With Potentially Clinically Significant Abnormalities in Biochemistry | Total Bilirubin: >2 ULN | 0 Participants |
| Group 2: Avalglucosidase Alfa 5 mg/kg | Number of Participants With Potentially Clinically Significant Abnormalities in Biochemistry | Sodium: <=129 mmol/L | 1 Participants |
| Group 2: Avalglucosidase Alfa 5 mg/kg | Number of Participants With Potentially Clinically Significant Abnormalities in Biochemistry | Alanine Aminotransferase > 3 ULN and Bilirubin > 2 ULN | 0 Participants |
| Group 2: Avalglucosidase Alfa 5 mg/kg | Number of Participants With Potentially Clinically Significant Abnormalities in Biochemistry | Total Bilirubin: >1.5 ULN | 0 Participants |
| Group 2: Avalglucosidase Alfa 5 mg/kg | Number of Participants With Potentially Clinically Significant Abnormalities in Biochemistry | Potassium: <3 mmol/L | 1 Participants |
| Group 2: Avalglucosidase Alfa 10 mg/kg | Number of Participants With Potentially Clinically Significant Abnormalities in Biochemistry | Potassium: <3 mmol/L | 1 Participants |
| Group 2: Avalglucosidase Alfa 10 mg/kg | Number of Participants With Potentially Clinically Significant Abnormalities in Biochemistry | Glucose: >=11.1 mmol/L (unfasted); >=7 mmol/L (fasted) | 1 Participants |
| Group 2: Avalglucosidase Alfa 10 mg/kg | Number of Participants With Potentially Clinically Significant Abnormalities in Biochemistry | Alkaline Phosphatase: >1.5 ULN | 0 Participants |
| Group 2: Avalglucosidase Alfa 10 mg/kg | Number of Participants With Potentially Clinically Significant Abnormalities in Biochemistry | Aspartate Aminotransferase: >5 ULN | 0 Participants |
| Group 2: Avalglucosidase Alfa 10 mg/kg | Number of Participants With Potentially Clinically Significant Abnormalities in Biochemistry | Chloride: >115 mmol/L | 0 Participants |
| Group 2: Avalglucosidase Alfa 10 mg/kg | Number of Participants With Potentially Clinically Significant Abnormalities in Biochemistry | Albumin: <=25 gram per liter (g/L) | 0 Participants |
| Group 2: Avalglucosidase Alfa 10 mg/kg | Number of Participants With Potentially Clinically Significant Abnormalities in Biochemistry | Total Bilirubin: >1.5 ULN | 0 Participants |
| Group 2: Avalglucosidase Alfa 10 mg/kg | Number of Participants With Potentially Clinically Significant Abnormalities in Biochemistry | Chloride: <80 mmol/L | 0 Participants |
| Group 2: Avalglucosidase Alfa 10 mg/kg | Number of Participants With Potentially Clinically Significant Abnormalities in Biochemistry | Urea Nitrogen: >=17 mmol/L | 0 Participants |
| Group 2: Avalglucosidase Alfa 10 mg/kg | Number of Participants With Potentially Clinically Significant Abnormalities in Biochemistry | Creatinine: >=100% change from baseline | 0 Participants |
| Group 2: Avalglucosidase Alfa 10 mg/kg | Number of Participants With Potentially Clinically Significant Abnormalities in Biochemistry | Glucose: <=3.9 millimoles per liter (mmol/L) and < lower limit of normal (LLN) | 2 Participants |
| Group 2: Avalglucosidase Alfa 10 mg/kg | Number of Participants With Potentially Clinically Significant Abnormalities in Biochemistry | Total Bilirubin: >2 ULN | 0 Participants |
| Group 2: Avalglucosidase Alfa 10 mg/kg | Number of Participants With Potentially Clinically Significant Abnormalities in Biochemistry | Aspartate Aminotransferase: >10 ULN | 0 Participants |
| Group 2: Avalglucosidase Alfa 10 mg/kg | Number of Participants With Potentially Clinically Significant Abnormalities in Biochemistry | Sodium: >=160 mmol/L | 0 Participants |
| Group 2: Avalglucosidase Alfa 10 mg/kg | Number of Participants With Potentially Clinically Significant Abnormalities in Biochemistry | Alanine Aminotransferase: >3 ULN | 0 Participants |
| Group 2: Avalglucosidase Alfa 10 mg/kg | Number of Participants With Potentially Clinically Significant Abnormalities in Biochemistry | Creatinine: >=30% change from baseline | 0 Participants |
| Group 2: Avalglucosidase Alfa 10 mg/kg | Number of Participants With Potentially Clinically Significant Abnormalities in Biochemistry | Direct Bilirubin > 35% Bilirubin and Bilirubin > 1.5 ULN | 0 Participants |
| Group 2: Avalglucosidase Alfa 10 mg/kg | Number of Participants With Potentially Clinically Significant Abnormalities in Biochemistry | Sodium: <=129 mmol/L | 0 Participants |
| Group 2: Avalglucosidase Alfa 10 mg/kg | Number of Participants With Potentially Clinically Significant Abnormalities in Biochemistry | Alanine Aminotransferase: >5 ULN | 0 Participants |
| Group 2: Avalglucosidase Alfa 10 mg/kg | Number of Participants With Potentially Clinically Significant Abnormalities in Biochemistry | Aspartate Aminotransferase: >3 upper limit of normal (ULN) | 0 Participants |
| Group 2: Avalglucosidase Alfa 10 mg/kg | Number of Participants With Potentially Clinically Significant Abnormalities in Biochemistry | Creatinine: >=150 micromoles/L (Adults) | 0 Participants |
| Group 2: Avalglucosidase Alfa 10 mg/kg | Number of Participants With Potentially Clinically Significant Abnormalities in Biochemistry | Alanine Aminotransferase: >10 ULN | 0 Participants |
| Group 2: Avalglucosidase Alfa 10 mg/kg | Number of Participants With Potentially Clinically Significant Abnormalities in Biochemistry | Potassium: >=5.5 mmol/L | 0 Participants |
| Group 2: Avalglucosidase Alfa 10 mg/kg | Number of Participants With Potentially Clinically Significant Abnormalities in Biochemistry | Alanine Aminotransferase > 3 ULN and Bilirubin > 2 ULN | 0 Participants |
| Group 2: Avalglucosidase Alfa 20 mg/kg | Number of Participants With Potentially Clinically Significant Abnormalities in Biochemistry | Creatinine: >=100% change from baseline | 2 Participants |
| Group 2: Avalglucosidase Alfa 20 mg/kg | Number of Participants With Potentially Clinically Significant Abnormalities in Biochemistry | Creatinine: >=30% change from baseline | 2 Participants |
| Group 2: Avalglucosidase Alfa 20 mg/kg | Number of Participants With Potentially Clinically Significant Abnormalities in Biochemistry | Potassium: >=5.5 mmol/L | 1 Participants |
| Group 2: Avalglucosidase Alfa 20 mg/kg | Number of Participants With Potentially Clinically Significant Abnormalities in Biochemistry | Urea Nitrogen: >=17 mmol/L | 0 Participants |
| Group 2: Avalglucosidase Alfa 20 mg/kg | Number of Participants With Potentially Clinically Significant Abnormalities in Biochemistry | Potassium: <3 mmol/L | 0 Participants |
| Group 2: Avalglucosidase Alfa 20 mg/kg | Number of Participants With Potentially Clinically Significant Abnormalities in Biochemistry | Aspartate Aminotransferase: >3 upper limit of normal (ULN) | 0 Participants |
| Group 2: Avalglucosidase Alfa 20 mg/kg | Number of Participants With Potentially Clinically Significant Abnormalities in Biochemistry | Aspartate Aminotransferase: >5 ULN | 0 Participants |
| Group 2: Avalglucosidase Alfa 20 mg/kg | Number of Participants With Potentially Clinically Significant Abnormalities in Biochemistry | Chloride: >115 mmol/L | 0 Participants |
| Group 2: Avalglucosidase Alfa 20 mg/kg | Number of Participants With Potentially Clinically Significant Abnormalities in Biochemistry | Sodium: >=160 mmol/L | 0 Participants |
| Group 2: Avalglucosidase Alfa 20 mg/kg | Number of Participants With Potentially Clinically Significant Abnormalities in Biochemistry | Aspartate Aminotransferase: >10 ULN | 0 Participants |
| Group 2: Avalglucosidase Alfa 20 mg/kg | Number of Participants With Potentially Clinically Significant Abnormalities in Biochemistry | Total Bilirubin: >2 ULN | 0 Participants |
| Group 2: Avalglucosidase Alfa 20 mg/kg | Number of Participants With Potentially Clinically Significant Abnormalities in Biochemistry | Alanine Aminotransferase: >3 ULN | 0 Participants |
| Group 2: Avalglucosidase Alfa 20 mg/kg | Number of Participants With Potentially Clinically Significant Abnormalities in Biochemistry | Sodium: <=129 mmol/L | 1 Participants |
| Group 2: Avalglucosidase Alfa 20 mg/kg | Number of Participants With Potentially Clinically Significant Abnormalities in Biochemistry | Creatinine: >=150 micromoles/L (Adults) | 1 Participants |
| Group 2: Avalglucosidase Alfa 20 mg/kg | Number of Participants With Potentially Clinically Significant Abnormalities in Biochemistry | Alanine Aminotransferase: >5 ULN | 0 Participants |
| Group 2: Avalglucosidase Alfa 20 mg/kg | Number of Participants With Potentially Clinically Significant Abnormalities in Biochemistry | Alanine Aminotransferase: >10 ULN | 0 Participants |
| Group 2: Avalglucosidase Alfa 20 mg/kg | Number of Participants With Potentially Clinically Significant Abnormalities in Biochemistry | Alanine Aminotransferase > 3 ULN and Bilirubin > 2 ULN | 0 Participants |
| Group 2: Avalglucosidase Alfa 20 mg/kg | Number of Participants With Potentially Clinically Significant Abnormalities in Biochemistry | Glucose: >=11.1 mmol/L (unfasted); >=7 mmol/L (fasted) | 3 Participants |
| Group 2: Avalglucosidase Alfa 20 mg/kg | Number of Participants With Potentially Clinically Significant Abnormalities in Biochemistry | Direct Bilirubin > 35% Bilirubin and Bilirubin > 1.5 ULN | 0 Participants |
| Group 2: Avalglucosidase Alfa 20 mg/kg | Number of Participants With Potentially Clinically Significant Abnormalities in Biochemistry | Alkaline Phosphatase: >1.5 ULN | 0 Participants |
| Group 2: Avalglucosidase Alfa 20 mg/kg | Number of Participants With Potentially Clinically Significant Abnormalities in Biochemistry | Glucose: <=3.9 millimoles per liter (mmol/L) and < lower limit of normal (LLN) | 1 Participants |
| Group 2: Avalglucosidase Alfa 20 mg/kg | Number of Participants With Potentially Clinically Significant Abnormalities in Biochemistry | Albumin: <=25 gram per liter (g/L) | 0 Participants |
| Group 2: Avalglucosidase Alfa 20 mg/kg | Number of Participants With Potentially Clinically Significant Abnormalities in Biochemistry | Chloride: <80 mmol/L | 0 Participants |
| Group 2: Avalglucosidase Alfa 20 mg/kg | Number of Participants With Potentially Clinically Significant Abnormalities in Biochemistry | Total Bilirubin: >1.5 ULN | 2 Participants |
Primary
Number of Participants With Potentially Clinically Significant Abnormalities in Hematology
Blood samples were collected to determine the hematology laboratory significant abnormalities.
Time frame: From first dose of IMP up to 4 weeks after the last treatment administration of the IMP, a maximum up to 458 weeks
Population: The safety analysis set included all participants who received at least 1 complete infusion of IMP.
| Arm | Measure | Group | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|---|
| Group 1: Avalglucosidase Alfa 5 mg/kg | Number of Participants With Potentially Clinically Significant Abnormalities in Hematology | Leukocyte Count: <3x10^9/L (Non-Black); <2x10^9/L (Black) | 0 Participants |
| Group 1: Avalglucosidase Alfa 5 mg/kg | Number of Participants With Potentially Clinically Significant Abnormalities in Hematology | Platelet Count: >=700x10^9/L | 0 Participants |
| Group 1: Avalglucosidase Alfa 5 mg/kg | Number of Participants With Potentially Clinically Significant Abnormalities in Hematology | Lymphocytes: >4x10^9/L | 0 Participants |
| Group 1: Avalglucosidase Alfa 5 mg/kg | Number of Participants With Potentially Clinically Significant Abnormalities in Hematology | Hematocrit: <=0.37 volume per volume (v/v) (Male); <=0.32 v/v (Female) | 1 Participants |
| Group 1: Avalglucosidase Alfa 5 mg/kg | Number of Participants With Potentially Clinically Significant Abnormalities in Hematology | Leukocyte Count: >=16x10^9/L | 0 Participants |
| Group 1: Avalglucosidase Alfa 5 mg/kg | Number of Participants With Potentially Clinically Significant Abnormalities in Hematology | Neutrophils: <1.5x10^9/L (Non-Black); <1x10^9/L (Black) | 0 Participants |
| Group 1: Avalglucosidase Alfa 5 mg/kg | Number of Participants With Potentially Clinically Significant Abnormalities in Hematology | Hematocrit: >=0.55 v/v (Male); >=0.5 v/v (Female) | 0 Participants |
| Group 1: Avalglucosidase Alfa 5 mg/kg | Number of Participants With Potentially Clinically Significant Abnormalities in Hematology | Erythrocyte Count: >=6x10^12/L | 0 Participants |
| Group 1: Avalglucosidase Alfa 5 mg/kg | Number of Participants With Potentially Clinically Significant Abnormalities in Hematology | Basophils: >0.1x10^9/L | 1 Participants |
| Group 1: Avalglucosidase Alfa 5 mg/kg | Number of Participants With Potentially Clinically Significant Abnormalities in Hematology | Hemoglobin: <=115 g/L (Male); <=95 g/L (Female) | 1 Participants |
| Group 1: Avalglucosidase Alfa 5 mg/kg | Number of Participants With Potentially Clinically Significant Abnormalities in Hematology | Eosinophils: >0.5x10^9/L or >ULN (if ULN >=0.5x10^9/L) | 0 Participants |
| Group 1: Avalglucosidase Alfa 5 mg/kg | Number of Participants With Potentially Clinically Significant Abnormalities in Hematology | Hemoglobin: >=185 g/L (Male); >=165 g/L (Female) | 0 Participants |
| Group 1: Avalglucosidase Alfa 5 mg/kg | Number of Participants With Potentially Clinically Significant Abnormalities in Hematology | Hemoglobin: Decrease from baseline >=20 g/L | 1 Participants |
| Group 1: Avalglucosidase Alfa 5 mg/kg | Number of Participants With Potentially Clinically Significant Abnormalities in Hematology | Monocytes: >0.7x10^9/L | 1 Participants |
| Group 1: Avalglucosidase Alfa 5 mg/kg | Number of Participants With Potentially Clinically Significant Abnormalities in Hematology | Platelet Count: <100x10^9/L | 0 Participants |
| Group 1: Avalglucosidase Alfa 10 mg/kg | Number of Participants With Potentially Clinically Significant Abnormalities in Hematology | Leukocyte Count: >=16x10^9/L | 0 Participants |
| Group 1: Avalglucosidase Alfa 10 mg/kg | Number of Participants With Potentially Clinically Significant Abnormalities in Hematology | Monocytes: >0.7x10^9/L | 0 Participants |
| Group 1: Avalglucosidase Alfa 10 mg/kg | Number of Participants With Potentially Clinically Significant Abnormalities in Hematology | Basophils: >0.1x10^9/L | 0 Participants |
| Group 1: Avalglucosidase Alfa 10 mg/kg | Number of Participants With Potentially Clinically Significant Abnormalities in Hematology | Hemoglobin: Decrease from baseline >=20 g/L | 1 Participants |
| Group 1: Avalglucosidase Alfa 10 mg/kg | Number of Participants With Potentially Clinically Significant Abnormalities in Hematology | Hemoglobin: <=115 g/L (Male); <=95 g/L (Female) | 0 Participants |
| Group 1: Avalglucosidase Alfa 10 mg/kg | Number of Participants With Potentially Clinically Significant Abnormalities in Hematology | Lymphocytes: >4x10^9/L | 1 Participants |
| Group 1: Avalglucosidase Alfa 10 mg/kg | Number of Participants With Potentially Clinically Significant Abnormalities in Hematology | Hematocrit: <=0.37 volume per volume (v/v) (Male); <=0.32 v/v (Female) | 0 Participants |
| Group 1: Avalglucosidase Alfa 10 mg/kg | Number of Participants With Potentially Clinically Significant Abnormalities in Hematology | Neutrophils: <1.5x10^9/L (Non-Black); <1x10^9/L (Black) | 0 Participants |
| Group 1: Avalglucosidase Alfa 10 mg/kg | Number of Participants With Potentially Clinically Significant Abnormalities in Hematology | Hematocrit: >=0.55 v/v (Male); >=0.5 v/v (Female) | 0 Participants |
| Group 1: Avalglucosidase Alfa 10 mg/kg | Number of Participants With Potentially Clinically Significant Abnormalities in Hematology | Eosinophils: >0.5x10^9/L or >ULN (if ULN >=0.5x10^9/L) | 0 Participants |
| Group 1: Avalglucosidase Alfa 10 mg/kg | Number of Participants With Potentially Clinically Significant Abnormalities in Hematology | Platelet Count: <100x10^9/L | 0 Participants |
| Group 1: Avalglucosidase Alfa 10 mg/kg | Number of Participants With Potentially Clinically Significant Abnormalities in Hematology | Platelet Count: >=700x10^9/L | 0 Participants |
| Group 1: Avalglucosidase Alfa 10 mg/kg | Number of Participants With Potentially Clinically Significant Abnormalities in Hematology | Hemoglobin: >=185 g/L (Male); >=165 g/L (Female) | 0 Participants |
| Group 1: Avalglucosidase Alfa 10 mg/kg | Number of Participants With Potentially Clinically Significant Abnormalities in Hematology | Leukocyte Count: <3x10^9/L (Non-Black); <2x10^9/L (Black) | 0 Participants |
| Group 1: Avalglucosidase Alfa 10 mg/kg | Number of Participants With Potentially Clinically Significant Abnormalities in Hematology | Erythrocyte Count: >=6x10^12/L | 0 Participants |
| Group 1: Avalglucosidase Alfa 20 mg/kg | Number of Participants With Potentially Clinically Significant Abnormalities in Hematology | Hemoglobin: Decrease from baseline >=20 g/L | 1 Participants |
| Group 1: Avalglucosidase Alfa 20 mg/kg | Number of Participants With Potentially Clinically Significant Abnormalities in Hematology | Leukocyte Count: <3x10^9/L (Non-Black); <2x10^9/L (Black) | 0 Participants |
| Group 1: Avalglucosidase Alfa 20 mg/kg | Number of Participants With Potentially Clinically Significant Abnormalities in Hematology | Platelet Count: <100x10^9/L | 0 Participants |
| Group 1: Avalglucosidase Alfa 20 mg/kg | Number of Participants With Potentially Clinically Significant Abnormalities in Hematology | Lymphocytes: >4x10^9/L | 0 Participants |
| Group 1: Avalglucosidase Alfa 20 mg/kg | Number of Participants With Potentially Clinically Significant Abnormalities in Hematology | Hemoglobin: <=115 g/L (Male); <=95 g/L (Female) | 1 Participants |
| Group 1: Avalglucosidase Alfa 20 mg/kg | Number of Participants With Potentially Clinically Significant Abnormalities in Hematology | Hemoglobin: >=185 g/L (Male); >=165 g/L (Female) | 0 Participants |
| Group 1: Avalglucosidase Alfa 20 mg/kg | Number of Participants With Potentially Clinically Significant Abnormalities in Hematology | Monocytes: >0.7x10^9/L | 1 Participants |
| Group 1: Avalglucosidase Alfa 20 mg/kg | Number of Participants With Potentially Clinically Significant Abnormalities in Hematology | Neutrophils: <1.5x10^9/L (Non-Black); <1x10^9/L (Black) | 0 Participants |
| Group 1: Avalglucosidase Alfa 20 mg/kg | Number of Participants With Potentially Clinically Significant Abnormalities in Hematology | Erythrocyte Count: >=6x10^12/L | 0 Participants |
| Group 1: Avalglucosidase Alfa 20 mg/kg | Number of Participants With Potentially Clinically Significant Abnormalities in Hematology | Eosinophils: >0.5x10^9/L or >ULN (if ULN >=0.5x10^9/L) | 0 Participants |
| Group 1: Avalglucosidase Alfa 20 mg/kg | Number of Participants With Potentially Clinically Significant Abnormalities in Hematology | Platelet Count: >=700x10^9/L | 0 Participants |
| Group 1: Avalglucosidase Alfa 20 mg/kg | Number of Participants With Potentially Clinically Significant Abnormalities in Hematology | Hematocrit: >=0.55 v/v (Male); >=0.5 v/v (Female) | 0 Participants |
| Group 1: Avalglucosidase Alfa 20 mg/kg | Number of Participants With Potentially Clinically Significant Abnormalities in Hematology | Basophils: >0.1x10^9/L | 2 Participants |
| Group 1: Avalglucosidase Alfa 20 mg/kg | Number of Participants With Potentially Clinically Significant Abnormalities in Hematology | Hematocrit: <=0.37 volume per volume (v/v) (Male); <=0.32 v/v (Female) | 1 Participants |
| Group 1: Avalglucosidase Alfa 20 mg/kg | Number of Participants With Potentially Clinically Significant Abnormalities in Hematology | Leukocyte Count: >=16x10^9/L | 0 Participants |
| Group 2: Avalglucosidase Alfa 5 mg/kg | Number of Participants With Potentially Clinically Significant Abnormalities in Hematology | Erythrocyte Count: >=6x10^12/L | 0 Participants |
| Group 2: Avalglucosidase Alfa 5 mg/kg | Number of Participants With Potentially Clinically Significant Abnormalities in Hematology | Hemoglobin: <=115 g/L (Male); <=95 g/L (Female) | 0 Participants |
| Group 2: Avalglucosidase Alfa 5 mg/kg | Number of Participants With Potentially Clinically Significant Abnormalities in Hematology | Hemoglobin: >=185 g/L (Male); >=165 g/L (Female) | 0 Participants |
| Group 2: Avalglucosidase Alfa 5 mg/kg | Number of Participants With Potentially Clinically Significant Abnormalities in Hematology | Hemoglobin: Decrease from baseline >=20 g/L | 2 Participants |
| Group 2: Avalglucosidase Alfa 5 mg/kg | Number of Participants With Potentially Clinically Significant Abnormalities in Hematology | Hematocrit: <=0.37 volume per volume (v/v) (Male); <=0.32 v/v (Female) | 1 Participants |
| Group 2: Avalglucosidase Alfa 5 mg/kg | Number of Participants With Potentially Clinically Significant Abnormalities in Hematology | Hematocrit: >=0.55 v/v (Male); >=0.5 v/v (Female) | 0 Participants |
| Group 2: Avalglucosidase Alfa 5 mg/kg | Number of Participants With Potentially Clinically Significant Abnormalities in Hematology | Platelet Count: <100x10^9/L | 0 Participants |
| Group 2: Avalglucosidase Alfa 5 mg/kg | Number of Participants With Potentially Clinically Significant Abnormalities in Hematology | Platelet Count: >=700x10^9/L | 0 Participants |
| Group 2: Avalglucosidase Alfa 5 mg/kg | Number of Participants With Potentially Clinically Significant Abnormalities in Hematology | Monocytes: >0.7x10^9/L | 1 Participants |
| Group 2: Avalglucosidase Alfa 5 mg/kg | Number of Participants With Potentially Clinically Significant Abnormalities in Hematology | Basophils: >0.1x10^9/L | 1 Participants |
| Group 2: Avalglucosidase Alfa 5 mg/kg | Number of Participants With Potentially Clinically Significant Abnormalities in Hematology | Leukocyte Count: <3x10^9/L (Non-Black); <2x10^9/L (Black) | 0 Participants |
| Group 2: Avalglucosidase Alfa 5 mg/kg | Number of Participants With Potentially Clinically Significant Abnormalities in Hematology | Leukocyte Count: >=16x10^9/L | 0 Participants |
| Group 2: Avalglucosidase Alfa 5 mg/kg | Number of Participants With Potentially Clinically Significant Abnormalities in Hematology | Neutrophils: <1.5x10^9/L (Non-Black); <1x10^9/L (Black) | 0 Participants |
| Group 2: Avalglucosidase Alfa 5 mg/kg | Number of Participants With Potentially Clinically Significant Abnormalities in Hematology | Lymphocytes: >4x10^9/L | 0 Participants |
| Group 2: Avalglucosidase Alfa 5 mg/kg | Number of Participants With Potentially Clinically Significant Abnormalities in Hematology | Eosinophils: >0.5x10^9/L or >ULN (if ULN >=0.5x10^9/L) | 0 Participants |
| Group 2: Avalglucosidase Alfa 10 mg/kg | Number of Participants With Potentially Clinically Significant Abnormalities in Hematology | Platelet Count: >=700x10^9/L | 0 Participants |
| Group 2: Avalglucosidase Alfa 10 mg/kg | Number of Participants With Potentially Clinically Significant Abnormalities in Hematology | Neutrophils: <1.5x10^9/L (Non-Black); <1x10^9/L (Black) | 0 Participants |
| Group 2: Avalglucosidase Alfa 10 mg/kg | Number of Participants With Potentially Clinically Significant Abnormalities in Hematology | Hemoglobin: <=115 g/L (Male); <=95 g/L (Female) | 0 Participants |
| Group 2: Avalglucosidase Alfa 10 mg/kg | Number of Participants With Potentially Clinically Significant Abnormalities in Hematology | Leukocyte Count: >=16x10^9/L | 0 Participants |
| Group 2: Avalglucosidase Alfa 10 mg/kg | Number of Participants With Potentially Clinically Significant Abnormalities in Hematology | Hemoglobin: Decrease from baseline >=20 g/L | 0 Participants |
| Group 2: Avalglucosidase Alfa 10 mg/kg | Number of Participants With Potentially Clinically Significant Abnormalities in Hematology | Leukocyte Count: <3x10^9/L (Non-Black); <2x10^9/L (Black) | 0 Participants |
| Group 2: Avalglucosidase Alfa 10 mg/kg | Number of Participants With Potentially Clinically Significant Abnormalities in Hematology | Eosinophils: >0.5x10^9/L or >ULN (if ULN >=0.5x10^9/L) | 0 Participants |
| Group 2: Avalglucosidase Alfa 10 mg/kg | Number of Participants With Potentially Clinically Significant Abnormalities in Hematology | Lymphocytes: >4x10^9/L | 0 Participants |
| Group 2: Avalglucosidase Alfa 10 mg/kg | Number of Participants With Potentially Clinically Significant Abnormalities in Hematology | Hematocrit: >=0.55 v/v (Male); >=0.5 v/v (Female) | 1 Participants |
| Group 2: Avalglucosidase Alfa 10 mg/kg | Number of Participants With Potentially Clinically Significant Abnormalities in Hematology | Platelet Count: <100x10^9/L | 0 Participants |
| Group 2: Avalglucosidase Alfa 10 mg/kg | Number of Participants With Potentially Clinically Significant Abnormalities in Hematology | Monocytes: >0.7x10^9/L | 0 Participants |
| Group 2: Avalglucosidase Alfa 10 mg/kg | Number of Participants With Potentially Clinically Significant Abnormalities in Hematology | Hematocrit: <=0.37 volume per volume (v/v) (Male); <=0.32 v/v (Female) | 1 Participants |
| Group 2: Avalglucosidase Alfa 10 mg/kg | Number of Participants With Potentially Clinically Significant Abnormalities in Hematology | Hemoglobin: >=185 g/L (Male); >=165 g/L (Female) | 1 Participants |
| Group 2: Avalglucosidase Alfa 10 mg/kg | Number of Participants With Potentially Clinically Significant Abnormalities in Hematology | Basophils: >0.1x10^9/L | 0 Participants |
| Group 2: Avalglucosidase Alfa 10 mg/kg | Number of Participants With Potentially Clinically Significant Abnormalities in Hematology | Erythrocyte Count: >=6x10^12/L | 1 Participants |
| Group 2: Avalglucosidase Alfa 20 mg/kg | Number of Participants With Potentially Clinically Significant Abnormalities in Hematology | Basophils: >0.1x10^9/L | 0 Participants |
| Group 2: Avalglucosidase Alfa 20 mg/kg | Number of Participants With Potentially Clinically Significant Abnormalities in Hematology | Platelet Count: >=700x10^9/L | 0 Participants |
| Group 2: Avalglucosidase Alfa 20 mg/kg | Number of Participants With Potentially Clinically Significant Abnormalities in Hematology | Hemoglobin: <=115 g/L (Male); <=95 g/L (Female) | 1 Participants |
| Group 2: Avalglucosidase Alfa 20 mg/kg | Number of Participants With Potentially Clinically Significant Abnormalities in Hematology | Leukocyte Count: <3x10^9/L (Non-Black); <2x10^9/L (Black) | 0 Participants |
| Group 2: Avalglucosidase Alfa 20 mg/kg | Number of Participants With Potentially Clinically Significant Abnormalities in Hematology | Platelet Count: <100x10^9/L | 0 Participants |
| Group 2: Avalglucosidase Alfa 20 mg/kg | Number of Participants With Potentially Clinically Significant Abnormalities in Hematology | Hematocrit: >=0.55 v/v (Male); >=0.5 v/v (Female) | 0 Participants |
| Group 2: Avalglucosidase Alfa 20 mg/kg | Number of Participants With Potentially Clinically Significant Abnormalities in Hematology | Leukocyte Count: >=16x10^9/L | 0 Participants |
| Group 2: Avalglucosidase Alfa 20 mg/kg | Number of Participants With Potentially Clinically Significant Abnormalities in Hematology | Hematocrit: <=0.37 volume per volume (v/v) (Male); <=0.32 v/v (Female) | 1 Participants |
| Group 2: Avalglucosidase Alfa 20 mg/kg | Number of Participants With Potentially Clinically Significant Abnormalities in Hematology | Eosinophils: >0.5x10^9/L or >ULN (if ULN >=0.5x10^9/L) | 0 Participants |
| Group 2: Avalglucosidase Alfa 20 mg/kg | Number of Participants With Potentially Clinically Significant Abnormalities in Hematology | Neutrophils: <1.5x10^9/L (Non-Black); <1x10^9/L (Black) | 0 Participants |
| Group 2: Avalglucosidase Alfa 20 mg/kg | Number of Participants With Potentially Clinically Significant Abnormalities in Hematology | Hemoglobin: Decrease from baseline >=20 g/L | 1 Participants |
| Group 2: Avalglucosidase Alfa 20 mg/kg | Number of Participants With Potentially Clinically Significant Abnormalities in Hematology | Hemoglobin: >=185 g/L (Male); >=165 g/L (Female) | 0 Participants |
| Group 2: Avalglucosidase Alfa 20 mg/kg | Number of Participants With Potentially Clinically Significant Abnormalities in Hematology | Lymphocytes: >4x10^9/L | 0 Participants |
| Group 2: Avalglucosidase Alfa 20 mg/kg | Number of Participants With Potentially Clinically Significant Abnormalities in Hematology | Monocytes: >0.7x10^9/L | 0 Participants |
| Group 2: Avalglucosidase Alfa 20 mg/kg | Number of Participants With Potentially Clinically Significant Abnormalities in Hematology | Erythrocyte Count: >=6x10^12/L | 0 Participants |
Primary
Number of Participants With Potentially Clinically Significant Vital Signs Abnormalities
Participants vital signs were examined to determine the abnormalities. Vital signs included heart rate, systolic and diastolic blood pressure.
Time frame: From first dose of IMP up to 4 weeks after the last treatment administration of the IMP, a maximum up to 458 weeks
Population: The safety analysis set included all participants who received at least 1 complete infusion of IMP.
| Arm | Measure | Group | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|---|
| Group 1: Avalglucosidase Alfa 5 mg/kg | Number of Participants With Potentially Clinically Significant Vital Signs Abnormalities | Systolic Blood Pressure: <=95 millimeter of mercury (mmHg) and decrease from baseline >=20 mmHg | 2 Participants |
| Group 1: Avalglucosidase Alfa 5 mg/kg | Number of Participants With Potentially Clinically Significant Vital Signs Abnormalities | Systolic Blood Pressure: >=160 mmHg and increase from baseline >=20 mmHg | 2 Participants |
| Group 1: Avalglucosidase Alfa 5 mg/kg | Number of Participants With Potentially Clinically Significant Vital Signs Abnormalities | Diastolic Blood Pressure: <=45 mmHg and decrease from baseline >=10 mmHg | 2 Participants |
| Group 1: Avalglucosidase Alfa 5 mg/kg | Number of Participants With Potentially Clinically Significant Vital Signs Abnormalities | Diastolic Blood Pressure: >=110 mmHg and increase from baseline >=10 mmHg | 2 Participants |
| Group 1: Avalglucosidase Alfa 5 mg/kg | Number of Participants With Potentially Clinically Significant Vital Signs Abnormalities | Heart Rate (HR): <=50 beats/minute (bpm) and decrease from baseline >=20 bpm | 1 Participants |
| Group 1: Avalglucosidase Alfa 5 mg/kg | Number of Participants With Potentially Clinically Significant Vital Signs Abnormalities | HR: >=120 bpm and increase from baseline >=20 bpm | 2 Participants |
| Group 1: Avalglucosidase Alfa 10 mg/kg | Number of Participants With Potentially Clinically Significant Vital Signs Abnormalities | Systolic Blood Pressure: >=160 mmHg and increase from baseline >=20 mmHg | 0 Participants |
| Group 1: Avalglucosidase Alfa 10 mg/kg | Number of Participants With Potentially Clinically Significant Vital Signs Abnormalities | Diastolic Blood Pressure: >=110 mmHg and increase from baseline >=10 mmHg | 0 Participants |
| Group 1: Avalglucosidase Alfa 10 mg/kg | Number of Participants With Potentially Clinically Significant Vital Signs Abnormalities | HR: >=120 bpm and increase from baseline >=20 bpm | 1 Participants |
| Group 1: Avalglucosidase Alfa 10 mg/kg | Number of Participants With Potentially Clinically Significant Vital Signs Abnormalities | Systolic Blood Pressure: <=95 millimeter of mercury (mmHg) and decrease from baseline >=20 mmHg | 2 Participants |
| Group 1: Avalglucosidase Alfa 10 mg/kg | Number of Participants With Potentially Clinically Significant Vital Signs Abnormalities | Diastolic Blood Pressure: <=45 mmHg and decrease from baseline >=10 mmHg | 0 Participants |
| Group 1: Avalglucosidase Alfa 10 mg/kg | Number of Participants With Potentially Clinically Significant Vital Signs Abnormalities | Heart Rate (HR): <=50 beats/minute (bpm) and decrease from baseline >=20 bpm | 0 Participants |
| Group 1: Avalglucosidase Alfa 20 mg/kg | Number of Participants With Potentially Clinically Significant Vital Signs Abnormalities | HR: >=120 bpm and increase from baseline >=20 bpm | 2 Participants |
| Group 1: Avalglucosidase Alfa 20 mg/kg | Number of Participants With Potentially Clinically Significant Vital Signs Abnormalities | Heart Rate (HR): <=50 beats/minute (bpm) and decrease from baseline >=20 bpm | 1 Participants |
| Group 1: Avalglucosidase Alfa 20 mg/kg | Number of Participants With Potentially Clinically Significant Vital Signs Abnormalities | Diastolic Blood Pressure: >=110 mmHg and increase from baseline >=10 mmHg | 0 Participants |
| Group 1: Avalglucosidase Alfa 20 mg/kg | Number of Participants With Potentially Clinically Significant Vital Signs Abnormalities | Diastolic Blood Pressure: <=45 mmHg and decrease from baseline >=10 mmHg | 0 Participants |
| Group 1: Avalglucosidase Alfa 20 mg/kg | Number of Participants With Potentially Clinically Significant Vital Signs Abnormalities | Systolic Blood Pressure: <=95 millimeter of mercury (mmHg) and decrease from baseline >=20 mmHg | 2 Participants |
| Group 1: Avalglucosidase Alfa 20 mg/kg | Number of Participants With Potentially Clinically Significant Vital Signs Abnormalities | Systolic Blood Pressure: >=160 mmHg and increase from baseline >=20 mmHg | 1 Participants |
| Group 2: Avalglucosidase Alfa 5 mg/kg | Number of Participants With Potentially Clinically Significant Vital Signs Abnormalities | Diastolic Blood Pressure: >=110 mmHg and increase from baseline >=10 mmHg | 0 Participants |
| Group 2: Avalglucosidase Alfa 5 mg/kg | Number of Participants With Potentially Clinically Significant Vital Signs Abnormalities | Systolic Blood Pressure: >=160 mmHg and increase from baseline >=20 mmHg | 2 Participants |
| Group 2: Avalglucosidase Alfa 5 mg/kg | Number of Participants With Potentially Clinically Significant Vital Signs Abnormalities | Diastolic Blood Pressure: <=45 mmHg and decrease from baseline >=10 mmHg | 2 Participants |
| Group 2: Avalglucosidase Alfa 5 mg/kg | Number of Participants With Potentially Clinically Significant Vital Signs Abnormalities | HR: >=120 bpm and increase from baseline >=20 bpm | 2 Participants |
| Group 2: Avalglucosidase Alfa 5 mg/kg | Number of Participants With Potentially Clinically Significant Vital Signs Abnormalities | Heart Rate (HR): <=50 beats/minute (bpm) and decrease from baseline >=20 bpm | 0 Participants |
| Group 2: Avalglucosidase Alfa 5 mg/kg | Number of Participants With Potentially Clinically Significant Vital Signs Abnormalities | Systolic Blood Pressure: <=95 millimeter of mercury (mmHg) and decrease from baseline >=20 mmHg | 2 Participants |
| Group 2: Avalglucosidase Alfa 10 mg/kg | Number of Participants With Potentially Clinically Significant Vital Signs Abnormalities | Systolic Blood Pressure: <=95 millimeter of mercury (mmHg) and decrease from baseline >=20 mmHg | 3 Participants |
| Group 2: Avalglucosidase Alfa 10 mg/kg | Number of Participants With Potentially Clinically Significant Vital Signs Abnormalities | Heart Rate (HR): <=50 beats/minute (bpm) and decrease from baseline >=20 bpm | 1 Participants |
| Group 2: Avalglucosidase Alfa 10 mg/kg | Number of Participants With Potentially Clinically Significant Vital Signs Abnormalities | Systolic Blood Pressure: >=160 mmHg and increase from baseline >=20 mmHg | 0 Participants |
| Group 2: Avalglucosidase Alfa 10 mg/kg | Number of Participants With Potentially Clinically Significant Vital Signs Abnormalities | Diastolic Blood Pressure: <=45 mmHg and decrease from baseline >=10 mmHg | 0 Participants |
| Group 2: Avalglucosidase Alfa 10 mg/kg | Number of Participants With Potentially Clinically Significant Vital Signs Abnormalities | Diastolic Blood Pressure: >=110 mmHg and increase from baseline >=10 mmHg | 0 Participants |
| Group 2: Avalglucosidase Alfa 10 mg/kg | Number of Participants With Potentially Clinically Significant Vital Signs Abnormalities | HR: >=120 bpm and increase from baseline >=20 bpm | 2 Participants |
| Group 2: Avalglucosidase Alfa 20 mg/kg | Number of Participants With Potentially Clinically Significant Vital Signs Abnormalities | Diastolic Blood Pressure: >=110 mmHg and increase from baseline >=10 mmHg | 2 Participants |
| Group 2: Avalglucosidase Alfa 20 mg/kg | Number of Participants With Potentially Clinically Significant Vital Signs Abnormalities | Diastolic Blood Pressure: <=45 mmHg and decrease from baseline >=10 mmHg | 2 Participants |
| Group 2: Avalglucosidase Alfa 20 mg/kg | Number of Participants With Potentially Clinically Significant Vital Signs Abnormalities | Heart Rate (HR): <=50 beats/minute (bpm) and decrease from baseline >=20 bpm | 0 Participants |
| Group 2: Avalglucosidase Alfa 20 mg/kg | Number of Participants With Potentially Clinically Significant Vital Signs Abnormalities | HR: >=120 bpm and increase from baseline >=20 bpm | 4 Participants |
| Group 2: Avalglucosidase Alfa 20 mg/kg | Number of Participants With Potentially Clinically Significant Vital Signs Abnormalities | Systolic Blood Pressure: >=160 mmHg and increase from baseline >=20 mmHg | 3 Participants |
| Group 2: Avalglucosidase Alfa 20 mg/kg | Number of Participants With Potentially Clinically Significant Vital Signs Abnormalities | Systolic Blood Pressure: <=95 millimeter of mercury (mmHg) and decrease from baseline >=20 mmHg | 2 Participants |
Primary
Number of Participants With Treatment-Emergent Adverse Events (TEAEs), Treatment-Emergent Serious Adverse Events (TESAEs), Infusion Associated Reactions (IARs) and Deaths
An adverse event (AE) is any untoward medical occurrence in a participant or clinical investigation participant administered a pharmaceutical product and which does not necessarily have to have a causal relationship with the treatment. An serious AE (SAE) is any untoward medical occurrence that results: death or life-threatening or inpatient hospitalization or prolongation of existing hospitalization or persistent or significant disability or congenital anomaly or medically important event. TEAEs are defined as AEs that develop or worsen during the on-treatment period (that is, from the time of first dose of IMP up to 4 weeks after the last administration of the IMP). Protocol-defined IARs were defined as AEs that occur during either the infusion or the post-infusion observation period (that is, up to 2 hours or longer following the infusion as per the Investigator's discretion) which were deemed to be related or possibly related to the IMP.
Time frame: From first dose of IMP up to 4 weeks after the last treatment administration of the IMP, a maximum up to 458 weeks
Population: The safety analysis set included all participants who received at least 1 complete infusion of IMP.
| Arm | Measure | Group | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|---|
| Group 1: Avalglucosidase Alfa 5 mg/kg | Number of Participants With Treatment-Emergent Adverse Events (TEAEs), Treatment-Emergent Serious Adverse Events (TESAEs), Infusion Associated Reactions (IARs) and Deaths | Protocol-defined IARs | 2 Participants |
| Group 1: Avalglucosidase Alfa 5 mg/kg | Number of Participants With Treatment-Emergent Adverse Events (TEAEs), Treatment-Emergent Serious Adverse Events (TESAEs), Infusion Associated Reactions (IARs) and Deaths | Any TEAEs | 4 Participants |
| Group 1: Avalglucosidase Alfa 5 mg/kg | Number of Participants With Treatment-Emergent Adverse Events (TEAEs), Treatment-Emergent Serious Adverse Events (TESAEs), Infusion Associated Reactions (IARs) and Deaths | TESAEs | 3 Participants |
| Group 1: Avalglucosidase Alfa 5 mg/kg | Number of Participants With Treatment-Emergent Adverse Events (TEAEs), Treatment-Emergent Serious Adverse Events (TESAEs), Infusion Associated Reactions (IARs) and Deaths | Any TEAE leading to death | 0 Participants |
| Group 1: Avalglucosidase Alfa 10 mg/kg | Number of Participants With Treatment-Emergent Adverse Events (TEAEs), Treatment-Emergent Serious Adverse Events (TESAEs), Infusion Associated Reactions (IARs) and Deaths | TESAEs | 1 Participants |
| Group 1: Avalglucosidase Alfa 10 mg/kg | Number of Participants With Treatment-Emergent Adverse Events (TEAEs), Treatment-Emergent Serious Adverse Events (TESAEs), Infusion Associated Reactions (IARs) and Deaths | Any TEAE leading to death | 0 Participants |
| Group 1: Avalglucosidase Alfa 10 mg/kg | Number of Participants With Treatment-Emergent Adverse Events (TEAEs), Treatment-Emergent Serious Adverse Events (TESAEs), Infusion Associated Reactions (IARs) and Deaths | Protocol-defined IARs | 0 Participants |
| Group 1: Avalglucosidase Alfa 10 mg/kg | Number of Participants With Treatment-Emergent Adverse Events (TEAEs), Treatment-Emergent Serious Adverse Events (TESAEs), Infusion Associated Reactions (IARs) and Deaths | Any TEAEs | 3 Participants |
| Group 1: Avalglucosidase Alfa 20 mg/kg | Number of Participants With Treatment-Emergent Adverse Events (TEAEs), Treatment-Emergent Serious Adverse Events (TESAEs), Infusion Associated Reactions (IARs) and Deaths | Any TEAE leading to death | 0 Participants |
| Group 1: Avalglucosidase Alfa 20 mg/kg | Number of Participants With Treatment-Emergent Adverse Events (TEAEs), Treatment-Emergent Serious Adverse Events (TESAEs), Infusion Associated Reactions (IARs) and Deaths | Any TEAEs | 3 Participants |
| Group 1: Avalglucosidase Alfa 20 mg/kg | Number of Participants With Treatment-Emergent Adverse Events (TEAEs), Treatment-Emergent Serious Adverse Events (TESAEs), Infusion Associated Reactions (IARs) and Deaths | Protocol-defined IARs | 2 Participants |
| Group 1: Avalglucosidase Alfa 20 mg/kg | Number of Participants With Treatment-Emergent Adverse Events (TEAEs), Treatment-Emergent Serious Adverse Events (TESAEs), Infusion Associated Reactions (IARs) and Deaths | TESAEs | 1 Participants |
| Group 2: Avalglucosidase Alfa 5 mg/kg | Number of Participants With Treatment-Emergent Adverse Events (TEAEs), Treatment-Emergent Serious Adverse Events (TESAEs), Infusion Associated Reactions (IARs) and Deaths | Any TEAEs | 4 Participants |
| Group 2: Avalglucosidase Alfa 5 mg/kg | Number of Participants With Treatment-Emergent Adverse Events (TEAEs), Treatment-Emergent Serious Adverse Events (TESAEs), Infusion Associated Reactions (IARs) and Deaths | Protocol-defined IARs | 2 Participants |
| Group 2: Avalglucosidase Alfa 5 mg/kg | Number of Participants With Treatment-Emergent Adverse Events (TEAEs), Treatment-Emergent Serious Adverse Events (TESAEs), Infusion Associated Reactions (IARs) and Deaths | Any TEAE leading to death | 0 Participants |
| Group 2: Avalglucosidase Alfa 5 mg/kg | Number of Participants With Treatment-Emergent Adverse Events (TEAEs), Treatment-Emergent Serious Adverse Events (TESAEs), Infusion Associated Reactions (IARs) and Deaths | TESAEs | 1 Participants |
| Group 2: Avalglucosidase Alfa 10 mg/kg | Number of Participants With Treatment-Emergent Adverse Events (TEAEs), Treatment-Emergent Serious Adverse Events (TESAEs), Infusion Associated Reactions (IARs) and Deaths | Protocol-defined IARs | 0 Participants |
| Group 2: Avalglucosidase Alfa 10 mg/kg | Number of Participants With Treatment-Emergent Adverse Events (TEAEs), Treatment-Emergent Serious Adverse Events (TESAEs), Infusion Associated Reactions (IARs) and Deaths | Any TEAEs | 4 Participants |
| Group 2: Avalglucosidase Alfa 10 mg/kg | Number of Participants With Treatment-Emergent Adverse Events (TEAEs), Treatment-Emergent Serious Adverse Events (TESAEs), Infusion Associated Reactions (IARs) and Deaths | Any TEAE leading to death | 0 Participants |
| Group 2: Avalglucosidase Alfa 10 mg/kg | Number of Participants With Treatment-Emergent Adverse Events (TEAEs), Treatment-Emergent Serious Adverse Events (TESAEs), Infusion Associated Reactions (IARs) and Deaths | TESAEs | 1 Participants |
| Group 2: Avalglucosidase Alfa 20 mg/kg | Number of Participants With Treatment-Emergent Adverse Events (TEAEs), Treatment-Emergent Serious Adverse Events (TESAEs), Infusion Associated Reactions (IARs) and Deaths | Any TEAE leading to death | 0 Participants |
| Group 2: Avalglucosidase Alfa 20 mg/kg | Number of Participants With Treatment-Emergent Adverse Events (TEAEs), Treatment-Emergent Serious Adverse Events (TESAEs), Infusion Associated Reactions (IARs) and Deaths | Protocol-defined IARs | 1 Participants |
| Group 2: Avalglucosidase Alfa 20 mg/kg | Number of Participants With Treatment-Emergent Adverse Events (TEAEs), Treatment-Emergent Serious Adverse Events (TESAEs), Infusion Associated Reactions (IARs) and Deaths | Any TEAEs | 6 Participants |
| Group 2: Avalglucosidase Alfa 20 mg/kg | Number of Participants With Treatment-Emergent Adverse Events (TEAEs), Treatment-Emergent Serious Adverse Events (TESAEs), Infusion Associated Reactions (IARs) and Deaths | TESAEs | 2 Participants |
Primary
Terminal Half-Life (t1/2z) of Avalglucosidase Alfa
t1/2z was calculated according to the following equation: t1/2z = 0.693/λz. Where, λz is the slope of the regression line of the terminal phase of the plasma concentration versus time curve. Half-life was calculated by taking the regression of at least 3 points. The non-compartmental PK analysis was performed.
Time frame: Predose (prior to infusion), end of the infusion and at 1, 4, 8, 12, and 24 hours post-dose on Week 312
Population: The PK analysis set included enrolled participants without any critical deviations related to IMP administration, and for whom any PK data were available. As pre-specified, results for this outcome measure are presented combinedly for all dosage groups (5, 10 and 20 mg/kg) under corresponding reporting groups, Group 1: (Avalglucosidase Alfa) and Group 2: (Avalglucosidase Alfa). Overall Number of Participants Analyzed = Number of participants contributed to the analysis for this outcome measure.
| Arm | Measure | Value (GEOMETRIC_MEAN) | Dispersion |
|---|---|---|---|
| Group 1: Avalglucosidase Alfa 5 mg/kg | Terminal Half-Life (t1/2z) of Avalglucosidase Alfa | 1.62 hour | Geometric Coefficient of Variation 9 |
| Group 1: Avalglucosidase Alfa 10 mg/kg | Terminal Half-Life (t1/2z) of Avalglucosidase Alfa | 1.79 hour | Geometric Coefficient of Variation 8 |
Primary
Time Corresponding to the Last Concentration (Tlast) of Avalglucosidase Alfa
Tlast was defined as time corresponding to the last concentration above the limit of quantification, Clast. The non-compartmental PK analysis was performed.
Time frame: Predose (prior to infusion), end of the infusion and at 1, 4, 8, 12, and 24 hours post-dose on Week 312
Population: The PK analysis set included enrolled participants without any critical deviations related to IMP administration, and for whom any PK data were available. As pre-specified, results for this outcome measure are presented combinedly for all dosage groups (5, 10 and 20 mg/kg) under corresponding reporting groups, Group 1: (Avalglucosidase Alfa) and Group 2: (Avalglucosidase Alfa). Overall Number of Participants Analyzed = Number of participants contributed to the analysis for this outcome measure.
| Arm | Measure | Value (MEDIAN) |
|---|---|---|
| Group 1: Avalglucosidase Alfa 5 mg/kg | Time Corresponding to the Last Concentration (Tlast) of Avalglucosidase Alfa | 25.84 hour |
| Group 1: Avalglucosidase Alfa 10 mg/kg | Time Corresponding to the Last Concentration (Tlast) of Avalglucosidase Alfa | 27.6 hour |
Secondary
Change From Baseline in Cross-Sectional Area (CSA) of Skeletal Muscle Magnetic Resonance Imaging (MRI) Up to Week 442
Skeletal muscle MRI performed prior to the muscle needle or open biopsy procedure using both qualitative (T1) and quantitative (T2, dixon) modalities to assess disease severity and detect treatment effects. The T1 weighted axial data was analyzed using the mercuri scale, which determines degree of intact muscle and fatty replacement, providing a qualitative measure of overall disease severity. Trophicity changes were evaluated for 5 muscle groups, including the upper leg muscles (quadriceps, hamstring) and the lower leg muscles (triceps, extensors, fibularis). The measured area of each muscle group, CSA was provided.
Time frame: Baseline (Day 1) and Weeks 104 and 442
Population: The PD analysis set included enrolled participants without any critical deviations related to IMP administration, and for whom any PD data were available. Only participants with data collected at specific time points are reported.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Group 1: Avalglucosidase Alfa 20 mg/kg | Change From Baseline in Cross-Sectional Area (CSA) of Skeletal Muscle Magnetic Resonance Imaging (MRI) Up to Week 442 | Week 104: CSA - Hamstring muscle | 123.0 millimeter square (mm^2) | Standard Deviation 254.7 |
| Group 1: Avalglucosidase Alfa 20 mg/kg | Change From Baseline in Cross-Sectional Area (CSA) of Skeletal Muscle Magnetic Resonance Imaging (MRI) Up to Week 442 | Week 104: CSA - Triceps surae muscle | 283.6 millimeter square (mm^2) | Standard Deviation 123.7 |
| Group 1: Avalglucosidase Alfa 20 mg/kg | Change From Baseline in Cross-Sectional Area (CSA) of Skeletal Muscle Magnetic Resonance Imaging (MRI) Up to Week 442 | Week 104: CSA - Quadriceps muscle | 303.3 millimeter square (mm^2) | Standard Deviation 49.5 |
| Group 1: Avalglucosidase Alfa 20 mg/kg | Change From Baseline in Cross-Sectional Area (CSA) of Skeletal Muscle Magnetic Resonance Imaging (MRI) Up to Week 442 | Week 104: CSA - Fibularis muscle | 57.4 millimeter square (mm^2) | Standard Deviation 3.9 |
| Group 1: Avalglucosidase Alfa 20 mg/kg | Change From Baseline in Cross-Sectional Area (CSA) of Skeletal Muscle Magnetic Resonance Imaging (MRI) Up to Week 442 | Week 104: CSA - Leg extensor muscle | 23.2 millimeter square (mm^2) | Standard Deviation 60.2 |
| Group 2: Avalglucosidase Alfa 5 mg/kg | Change From Baseline in Cross-Sectional Area (CSA) of Skeletal Muscle Magnetic Resonance Imaging (MRI) Up to Week 442 | Week 104: CSA - Hamstring muscle | -135.7 millimeter square (mm^2) | Standard Deviation 138.7 |
| Group 2: Avalglucosidase Alfa 5 mg/kg | Change From Baseline in Cross-Sectional Area (CSA) of Skeletal Muscle Magnetic Resonance Imaging (MRI) Up to Week 442 | Week 104: CSA - Fibularis muscle | -0.9 millimeter square (mm^2) | Standard Deviation 46.9 |
| Group 2: Avalglucosidase Alfa 5 mg/kg | Change From Baseline in Cross-Sectional Area (CSA) of Skeletal Muscle Magnetic Resonance Imaging (MRI) Up to Week 442 | Week 104: CSA - Quadriceps muscle | -27.6 millimeter square (mm^2) | Standard Deviation 45.8 |
| Group 2: Avalglucosidase Alfa 5 mg/kg | Change From Baseline in Cross-Sectional Area (CSA) of Skeletal Muscle Magnetic Resonance Imaging (MRI) Up to Week 442 | Week 104: CSA - Triceps surae muscle | -310.0 millimeter square (mm^2) | Standard Deviation 383.2 |
| Group 2: Avalglucosidase Alfa 5 mg/kg | Change From Baseline in Cross-Sectional Area (CSA) of Skeletal Muscle Magnetic Resonance Imaging (MRI) Up to Week 442 | Week 104: CSA - Leg extensor muscle | 23.1 millimeter square (mm^2) | Standard Deviation 19.1 |
| Group 2: Avalglucosidase Alfa 10 mg/kg | Change From Baseline in Cross-Sectional Area (CSA) of Skeletal Muscle Magnetic Resonance Imaging (MRI) Up to Week 442 | Week 104: CSA - Leg extensor muscle | 107.9 millimeter square (mm^2) | Standard Deviation 145 |
| Group 2: Avalglucosidase Alfa 10 mg/kg | Change From Baseline in Cross-Sectional Area (CSA) of Skeletal Muscle Magnetic Resonance Imaging (MRI) Up to Week 442 | Week 104: CSA - Hamstring muscle | 70.6 millimeter square (mm^2) | Standard Deviation 368.1 |
| Group 2: Avalglucosidase Alfa 10 mg/kg | Change From Baseline in Cross-Sectional Area (CSA) of Skeletal Muscle Magnetic Resonance Imaging (MRI) Up to Week 442 | Week 442: CSA - Hamstring muscle | -461.1 millimeter square (mm^2) | — |
| Group 2: Avalglucosidase Alfa 10 mg/kg | Change From Baseline in Cross-Sectional Area (CSA) of Skeletal Muscle Magnetic Resonance Imaging (MRI) Up to Week 442 | Week 104: CSA - Fibularis muscle | 46.1 millimeter square (mm^2) | Standard Deviation 81.2 |
| Group 2: Avalglucosidase Alfa 10 mg/kg | Change From Baseline in Cross-Sectional Area (CSA) of Skeletal Muscle Magnetic Resonance Imaging (MRI) Up to Week 442 | Week 442: CSA - Leg extensor muscle | -102.7 millimeter square (mm^2) | — |
| Group 2: Avalglucosidase Alfa 10 mg/kg | Change From Baseline in Cross-Sectional Area (CSA) of Skeletal Muscle Magnetic Resonance Imaging (MRI) Up to Week 442 | Week 442: CSA - Quadriceps muscle | -644.7 millimeter square (mm^2) | — |
| Group 2: Avalglucosidase Alfa 10 mg/kg | Change From Baseline in Cross-Sectional Area (CSA) of Skeletal Muscle Magnetic Resonance Imaging (MRI) Up to Week 442 | Week 442: CSA - Triceps surae muscle | -220.9 millimeter square (mm^2) | — |
| Group 2: Avalglucosidase Alfa 10 mg/kg | Change From Baseline in Cross-Sectional Area (CSA) of Skeletal Muscle Magnetic Resonance Imaging (MRI) Up to Week 442 | Week 442: CSA - Fibularis muscle | -41.8 millimeter square (mm^2) | — |
| Group 2: Avalglucosidase Alfa 10 mg/kg | Change From Baseline in Cross-Sectional Area (CSA) of Skeletal Muscle Magnetic Resonance Imaging (MRI) Up to Week 442 | Week 104: CSA - Quadriceps muscle | 134.0 millimeter square (mm^2) | Standard Deviation 569.4 |
| Group 2: Avalglucosidase Alfa 10 mg/kg | Change From Baseline in Cross-Sectional Area (CSA) of Skeletal Muscle Magnetic Resonance Imaging (MRI) Up to Week 442 | Week 104: CSA - Triceps surae muscle | -3.5 millimeter square (mm^2) | Standard Deviation 334.6 |
| Group 2: Avalglucosidase Alfa 20 mg/kg | Change From Baseline in Cross-Sectional Area (CSA) of Skeletal Muscle Magnetic Resonance Imaging (MRI) Up to Week 442 | Week 442: CSA - Triceps surae muscle | -82.3 millimeter square (mm^2) | — |
| Group 2: Avalglucosidase Alfa 20 mg/kg | Change From Baseline in Cross-Sectional Area (CSA) of Skeletal Muscle Magnetic Resonance Imaging (MRI) Up to Week 442 | Week 104: CSA - Fibularis muscle | 26.7 millimeter square (mm^2) | Standard Deviation 40 |
| Group 2: Avalglucosidase Alfa 20 mg/kg | Change From Baseline in Cross-Sectional Area (CSA) of Skeletal Muscle Magnetic Resonance Imaging (MRI) Up to Week 442 | Week 104: CSA - Hamstring muscle | 215.2 millimeter square (mm^2) | Standard Deviation 126.1 |
| Group 2: Avalglucosidase Alfa 20 mg/kg | Change From Baseline in Cross-Sectional Area (CSA) of Skeletal Muscle Magnetic Resonance Imaging (MRI) Up to Week 442 | Week 104: CSA - Leg extensor muscle | 39.1 millimeter square (mm^2) | Standard Deviation 26.6 |
| Group 2: Avalglucosidase Alfa 20 mg/kg | Change From Baseline in Cross-Sectional Area (CSA) of Skeletal Muscle Magnetic Resonance Imaging (MRI) Up to Week 442 | Week 104: CSA - Quadriceps muscle | 193.3 millimeter square (mm^2) | Standard Deviation 103.3 |
| Group 2: Avalglucosidase Alfa 20 mg/kg | Change From Baseline in Cross-Sectional Area (CSA) of Skeletal Muscle Magnetic Resonance Imaging (MRI) Up to Week 442 | Week 104: CSA - Triceps surae muscle | 226.0 millimeter square (mm^2) | Standard Deviation 172.3 |
| Group 2: Avalglucosidase Alfa 20 mg/kg | Change From Baseline in Cross-Sectional Area (CSA) of Skeletal Muscle Magnetic Resonance Imaging (MRI) Up to Week 442 | Week 442: CSA - Fibularis muscle | -55.6 millimeter square (mm^2) | — |
| Group 2: Avalglucosidase Alfa 20 mg/kg | Change From Baseline in Cross-Sectional Area (CSA) of Skeletal Muscle Magnetic Resonance Imaging (MRI) Up to Week 442 | Week 442: CSA - Hamstring muscle | -30.2 millimeter square (mm^2) | — |
| Group 2: Avalglucosidase Alfa 20 mg/kg | Change From Baseline in Cross-Sectional Area (CSA) of Skeletal Muscle Magnetic Resonance Imaging (MRI) Up to Week 442 | Week 442: CSA - Leg extensor muscle | -68.9 millimeter square (mm^2) | — |
| Group 2: Avalglucosidase Alfa 20 mg/kg | Change From Baseline in Cross-Sectional Area (CSA) of Skeletal Muscle Magnetic Resonance Imaging (MRI) Up to Week 442 | Week 442: CSA - Quadriceps muscle | 19.8 millimeter square (mm^2) | — |
Secondary
Change From Baseline in Dixon Fat Fraction of Skeletal Muscle Magnetic Resonance Imaging (MRI) Up to Week 442
Skeletal muscle MRI performed prior to the muscle needle or open biopsy procedure using both qualitative (T1) and quantitative (T2, dixon) modalities to assess disease severity and detect treatment effects. The T1 weighted axial data was analyzed using the mercuri scale, which determines degree of intact muscle and fatty replacement, providing a qualitative measure of overall disease severity. Trophicity changes were evaluated for 5 muscle groups, including the upper leg muscles (quadriceps, hamstring) and the lower leg muscles (triceps, extensors, fibularis). Three-point dixon imaging provided quantification of fat content in muscles \[fat fraction (FF)\].
Time frame: Baseline (Day 1) and Weeks 104 and 442
Population: The PD analysis set included enrolled participants without any critical deviations related to IMP administration, and for whom any PD data were available. Only participants with data collected at specific time points are reported.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Group 1: Avalglucosidase Alfa 20 mg/kg | Change From Baseline in Dixon Fat Fraction of Skeletal Muscle Magnetic Resonance Imaging (MRI) Up to Week 442 | Week 104: Dixon fat fraction - Hamstring muscle | 5.7 percentage (%) | Standard Deviation 3.3 |
| Group 1: Avalglucosidase Alfa 20 mg/kg | Change From Baseline in Dixon Fat Fraction of Skeletal Muscle Magnetic Resonance Imaging (MRI) Up to Week 442 | Week 104: Dixon fat fraction - Triceps surae muscle | 0.4 percentage (%) | Standard Deviation 0.3 |
| Group 1: Avalglucosidase Alfa 20 mg/kg | Change From Baseline in Dixon Fat Fraction of Skeletal Muscle Magnetic Resonance Imaging (MRI) Up to Week 442 | Week 104: Dixon fat fraction - Quadriceps muscle | 2.3 percentage (%) | Standard Deviation 1.4 |
| Group 1: Avalglucosidase Alfa 20 mg/kg | Change From Baseline in Dixon Fat Fraction of Skeletal Muscle Magnetic Resonance Imaging (MRI) Up to Week 442 | Week 104: Dixon fat fraction - Fibularis muscle | 0.9 percentage (%) | Standard Deviation 0.1 |
| Group 1: Avalglucosidase Alfa 20 mg/kg | Change From Baseline in Dixon Fat Fraction of Skeletal Muscle Magnetic Resonance Imaging (MRI) Up to Week 442 | Week 104: Dixon fat fraction - Leg extensor muscle | 0.9 percentage (%) | Standard Deviation 0.1 |
| Group 2: Avalglucosidase Alfa 5 mg/kg | Change From Baseline in Dixon Fat Fraction of Skeletal Muscle Magnetic Resonance Imaging (MRI) Up to Week 442 | Week 104: Dixon fat fraction - Hamstring muscle | -0.2 percentage (%) | Standard Deviation 2.6 |
| Group 2: Avalglucosidase Alfa 5 mg/kg | Change From Baseline in Dixon Fat Fraction of Skeletal Muscle Magnetic Resonance Imaging (MRI) Up to Week 442 | Week 104: Dixon fat fraction - Fibularis muscle | 1.4 percentage (%) | Standard Deviation 2.2 |
| Group 2: Avalglucosidase Alfa 5 mg/kg | Change From Baseline in Dixon Fat Fraction of Skeletal Muscle Magnetic Resonance Imaging (MRI) Up to Week 442 | Week 104: Dixon fat fraction - Quadriceps muscle | -0.5 percentage (%) | Standard Deviation 2.3 |
| Group 2: Avalglucosidase Alfa 5 mg/kg | Change From Baseline in Dixon Fat Fraction of Skeletal Muscle Magnetic Resonance Imaging (MRI) Up to Week 442 | Week 104: Dixon fat fraction - Triceps surae muscle | 1.4 percentage (%) | Standard Deviation 1.7 |
| Group 2: Avalglucosidase Alfa 5 mg/kg | Change From Baseline in Dixon Fat Fraction of Skeletal Muscle Magnetic Resonance Imaging (MRI) Up to Week 442 | Week 104: Dixon fat fraction - Leg extensor muscle | 0.8 percentage (%) | Standard Deviation 2.2 |
| Group 2: Avalglucosidase Alfa 10 mg/kg | Change From Baseline in Dixon Fat Fraction of Skeletal Muscle Magnetic Resonance Imaging (MRI) Up to Week 442 | Week 104: Dixon fat fraction - Leg extensor muscle | -0.3 percentage (%) | — |
| Group 2: Avalglucosidase Alfa 10 mg/kg | Change From Baseline in Dixon Fat Fraction of Skeletal Muscle Magnetic Resonance Imaging (MRI) Up to Week 442 | Week 104: Dixon fat fraction - Hamstring muscle | -0.3 percentage (%) | — |
| Group 2: Avalglucosidase Alfa 10 mg/kg | Change From Baseline in Dixon Fat Fraction of Skeletal Muscle Magnetic Resonance Imaging (MRI) Up to Week 442 | Week 442: Dixon fat fraction - Hamstring muscle | 10.1 percentage (%) | — |
| Group 2: Avalglucosidase Alfa 10 mg/kg | Change From Baseline in Dixon Fat Fraction of Skeletal Muscle Magnetic Resonance Imaging (MRI) Up to Week 442 | Week 104: Dixon fat fraction - Fibularis muscle | -0.7 percentage (%) | — |
| Group 2: Avalglucosidase Alfa 10 mg/kg | Change From Baseline in Dixon Fat Fraction of Skeletal Muscle Magnetic Resonance Imaging (MRI) Up to Week 442 | Week 442: Dixon fat fraction - Leg extensor muscle | 1.6 percentage (%) | — |
| Group 2: Avalglucosidase Alfa 10 mg/kg | Change From Baseline in Dixon Fat Fraction of Skeletal Muscle Magnetic Resonance Imaging (MRI) Up to Week 442 | Week 442: Dixon fat fraction - Quadriceps muscle | 9.7 percentage (%) | — |
| Group 2: Avalglucosidase Alfa 10 mg/kg | Change From Baseline in Dixon Fat Fraction of Skeletal Muscle Magnetic Resonance Imaging (MRI) Up to Week 442 | Week 442: Dixon fat fraction - Triceps surae muscle | 1.8 percentage (%) | — |
| Group 2: Avalglucosidase Alfa 10 mg/kg | Change From Baseline in Dixon Fat Fraction of Skeletal Muscle Magnetic Resonance Imaging (MRI) Up to Week 442 | Week 442: Dixon fat fraction - Fibularis muscle | 2.1 percentage (%) | — |
| Group 2: Avalglucosidase Alfa 10 mg/kg | Change From Baseline in Dixon Fat Fraction of Skeletal Muscle Magnetic Resonance Imaging (MRI) Up to Week 442 | Week 104: Dixon fat fraction - Quadriceps muscle | 0.2 percentage (%) | — |
| Group 2: Avalglucosidase Alfa 10 mg/kg | Change From Baseline in Dixon Fat Fraction of Skeletal Muscle Magnetic Resonance Imaging (MRI) Up to Week 442 | Week 104: Dixon fat fraction - Triceps surae muscle | -0.3 percentage (%) | — |
| Group 2: Avalglucosidase Alfa 20 mg/kg | Change From Baseline in Dixon Fat Fraction of Skeletal Muscle Magnetic Resonance Imaging (MRI) Up to Week 442 | Week 442: Dixon fat fraction - Triceps surae muscle | 0.5 percentage (%) | — |
| Group 2: Avalglucosidase Alfa 20 mg/kg | Change From Baseline in Dixon Fat Fraction of Skeletal Muscle Magnetic Resonance Imaging (MRI) Up to Week 442 | Week 104: Dixon fat fraction - Fibularis muscle | 0.3 percentage (%) | Standard Deviation 1.1 |
| Group 2: Avalglucosidase Alfa 20 mg/kg | Change From Baseline in Dixon Fat Fraction of Skeletal Muscle Magnetic Resonance Imaging (MRI) Up to Week 442 | Week 104: Dixon fat fraction - Hamstring muscle | 3.4 percentage (%) | Standard Deviation 3.9 |
| Group 2: Avalglucosidase Alfa 20 mg/kg | Change From Baseline in Dixon Fat Fraction of Skeletal Muscle Magnetic Resonance Imaging (MRI) Up to Week 442 | Week 104: Dixon fat fraction - Leg extensor muscle | -0.2 percentage (%) | Standard Deviation 1.3 |
| Group 2: Avalglucosidase Alfa 20 mg/kg | Change From Baseline in Dixon Fat Fraction of Skeletal Muscle Magnetic Resonance Imaging (MRI) Up to Week 442 | Week 104: Dixon fat fraction - Quadriceps muscle | 2.4 percentage (%) | Standard Deviation 3.6 |
| Group 2: Avalglucosidase Alfa 20 mg/kg | Change From Baseline in Dixon Fat Fraction of Skeletal Muscle Magnetic Resonance Imaging (MRI) Up to Week 442 | Week 104: Dixon fat fraction - Triceps surae muscle | 0.9 percentage (%) | Standard Deviation 0.5 |
| Group 2: Avalglucosidase Alfa 20 mg/kg | Change From Baseline in Dixon Fat Fraction of Skeletal Muscle Magnetic Resonance Imaging (MRI) Up to Week 442 | Week 442: Dixon fat fraction - Fibularis muscle | 0.8 percentage (%) | — |
| Group 2: Avalglucosidase Alfa 20 mg/kg | Change From Baseline in Dixon Fat Fraction of Skeletal Muscle Magnetic Resonance Imaging (MRI) Up to Week 442 | Week 442: Dixon fat fraction - Hamstring muscle | 0.6 percentage (%) | — |
| Group 2: Avalglucosidase Alfa 20 mg/kg | Change From Baseline in Dixon Fat Fraction of Skeletal Muscle Magnetic Resonance Imaging (MRI) Up to Week 442 | Week 442: Dixon fat fraction - Leg extensor muscle | -0.0 percentage (%) | — |
| Group 2: Avalglucosidase Alfa 20 mg/kg | Change From Baseline in Dixon Fat Fraction of Skeletal Muscle Magnetic Resonance Imaging (MRI) Up to Week 442 | Week 442: Dixon fat fraction - Quadriceps muscle | 0.8 percentage (%) | — |
Secondary
Change From Baseline in Index of Real Muscle Mass (IRMM) of Skeletal Muscle Magnetic Resonance Imaging (MRI) Up to Week 442
Skeletal muscle MRI performed prior to the muscle needle or open biopsy procedure using both qualitative (T1) and quantitative (T2, dixon) modalities to assess disease severity and detect treatment effects. The T1 weighted axial data was analyzed using the mercuri scale, which determines degree of intact muscle and fatty replacement, providing a qualitative measure of overall disease severity. Trophicity changes were evaluated for 5 muscle groups, including the upper leg muscles (quadriceps, hamstring) and the lower leg muscles (triceps, extensors, fibularis). The FF was combined with the CSA measurements trophicity to provide an IRMM in mm\^2 (that is, IRMM = CSA x \[1 - FF\]). A negative change from baseline value in IRMM of skeletal muscle MRI indicates muscle loss (worse outcome) and a positive change from baseline value indicates muscle gain (better outcome).
Time frame: Baseline (Day 1) and Weeks 104 and 442
Population: The PD analysis set included enrolled participants without any critical deviations related to IMP administration, and for whom any PD data were available. Only participants with data collected at specific time points are reported.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Group 1: Avalglucosidase Alfa 20 mg/kg | Change From Baseline in Index of Real Muscle Mass (IRMM) of Skeletal Muscle Magnetic Resonance Imaging (MRI) Up to Week 442 | Week 104: IRMM - Hamstring muscle | -131.6 mm^2 | Standard Deviation 77 |
| Group 1: Avalglucosidase Alfa 20 mg/kg | Change From Baseline in Index of Real Muscle Mass (IRMM) of Skeletal Muscle Magnetic Resonance Imaging (MRI) Up to Week 442 | Week 104: IRMM - Triceps surae muscle | 236.8 mm^2 | Standard Deviation 122.2 |
| Group 1: Avalglucosidase Alfa 20 mg/kg | Change From Baseline in Index of Real Muscle Mass (IRMM) of Skeletal Muscle Magnetic Resonance Imaging (MRI) Up to Week 442 | Week 104: IRMM - Quadriceps muscle | 149.2 mm^2 | Standard Deviation 135.2 |
| Group 1: Avalglucosidase Alfa 20 mg/kg | Change From Baseline in Index of Real Muscle Mass (IRMM) of Skeletal Muscle Magnetic Resonance Imaging (MRI) Up to Week 442 | Week 104: IRMM - Fibularis muscle | 44.8 mm^2 | Standard Deviation 8.3 |
| Group 1: Avalglucosidase Alfa 20 mg/kg | Change From Baseline in Index of Real Muscle Mass (IRMM) of Skeletal Muscle Magnetic Resonance Imaging (MRI) Up to Week 442 | Week 104: IRMM - Leg extensor muscle | 12.4 mm^2 | Standard Deviation 59.9 |
| Group 2: Avalglucosidase Alfa 5 mg/kg | Change From Baseline in Index of Real Muscle Mass (IRMM) of Skeletal Muscle Magnetic Resonance Imaging (MRI) Up to Week 442 | Week 104: IRMM - Hamstring muscle | -100.9 mm^2 | Standard Deviation 150.3 |
| Group 2: Avalglucosidase Alfa 5 mg/kg | Change From Baseline in Index of Real Muscle Mass (IRMM) of Skeletal Muscle Magnetic Resonance Imaging (MRI) Up to Week 442 | Week 104: IRMM - Fibularis muscle | -13.8 mm^2 | Standard Deviation 40.4 |
| Group 2: Avalglucosidase Alfa 5 mg/kg | Change From Baseline in Index of Real Muscle Mass (IRMM) of Skeletal Muscle Magnetic Resonance Imaging (MRI) Up to Week 442 | Week 104: IRMM - Quadriceps muscle | -9.4 mm^2 | Standard Deviation 65.9 |
| Group 2: Avalglucosidase Alfa 5 mg/kg | Change From Baseline in Index of Real Muscle Mass (IRMM) of Skeletal Muscle Magnetic Resonance Imaging (MRI) Up to Week 442 | Week 104: IRMM - Triceps surae muscle | -299.6 mm^2 | Standard Deviation 402.6 |
| Group 2: Avalglucosidase Alfa 5 mg/kg | Change From Baseline in Index of Real Muscle Mass (IRMM) of Skeletal Muscle Magnetic Resonance Imaging (MRI) Up to Week 442 | Week 104: IRMM - Leg extensor muscle | 9.8 mm^2 | Standard Deviation 34.2 |
| Group 2: Avalglucosidase Alfa 10 mg/kg | Change From Baseline in Index of Real Muscle Mass (IRMM) of Skeletal Muscle Magnetic Resonance Imaging (MRI) Up to Week 442 | Week 104: IRMM - Leg extensor muscle | 7.5 mm^2 | — |
| Group 2: Avalglucosidase Alfa 10 mg/kg | Change From Baseline in Index of Real Muscle Mass (IRMM) of Skeletal Muscle Magnetic Resonance Imaging (MRI) Up to Week 442 | Week 104: IRMM - Hamstring muscle | -149.7 mm^2 | — |
| Group 2: Avalglucosidase Alfa 10 mg/kg | Change From Baseline in Index of Real Muscle Mass (IRMM) of Skeletal Muscle Magnetic Resonance Imaging (MRI) Up to Week 442 | Week 442: IRMM - Hamstring muscle | -412.7 mm^2 | — |
| Group 2: Avalglucosidase Alfa 10 mg/kg | Change From Baseline in Index of Real Muscle Mass (IRMM) of Skeletal Muscle Magnetic Resonance Imaging (MRI) Up to Week 442 | Week 104: IRMM - Fibularis muscle | -5.8 mm^2 | — |
| Group 2: Avalglucosidase Alfa 10 mg/kg | Change From Baseline in Index of Real Muscle Mass (IRMM) of Skeletal Muscle Magnetic Resonance Imaging (MRI) Up to Week 442 | Week 442: IRMM - Leg extensor muscle | -109.6 mm^2 | — |
| Group 2: Avalglucosidase Alfa 10 mg/kg | Change From Baseline in Index of Real Muscle Mass (IRMM) of Skeletal Muscle Magnetic Resonance Imaging (MRI) Up to Week 442 | Week 442: IRMM - Quadriceps muscle | -1085.4 mm^2 | — |
| Group 2: Avalglucosidase Alfa 10 mg/kg | Change From Baseline in Index of Real Muscle Mass (IRMM) of Skeletal Muscle Magnetic Resonance Imaging (MRI) Up to Week 442 | Week 442: IRMM - Triceps surae muscle | -288.6 mm^2 | — |
| Group 2: Avalglucosidase Alfa 10 mg/kg | Change From Baseline in Index of Real Muscle Mass (IRMM) of Skeletal Muscle Magnetic Resonance Imaging (MRI) Up to Week 442 | Week 442: IRMM - Fibularis muscle | -53.4 mm^2 | — |
| Group 2: Avalglucosidase Alfa 10 mg/kg | Change From Baseline in Index of Real Muscle Mass (IRMM) of Skeletal Muscle Magnetic Resonance Imaging (MRI) Up to Week 442 | Week 104: IRMM - Quadriceps muscle | -256.0 mm^2 | — |
| Group 2: Avalglucosidase Alfa 10 mg/kg | Change From Baseline in Index of Real Muscle Mass (IRMM) of Skeletal Muscle Magnetic Resonance Imaging (MRI) Up to Week 442 | Week 104: IRMM - Triceps surae muscle | -210.5 mm^2 | — |
| Group 2: Avalglucosidase Alfa 20 mg/kg | Change From Baseline in Index of Real Muscle Mass (IRMM) of Skeletal Muscle Magnetic Resonance Imaging (MRI) Up to Week 442 | Week 442: IRMM - Triceps surae muscle | -85.1 mm^2 | — |
| Group 2: Avalglucosidase Alfa 20 mg/kg | Change From Baseline in Index of Real Muscle Mass (IRMM) of Skeletal Muscle Magnetic Resonance Imaging (MRI) Up to Week 442 | Week 104: IRMM - Fibularis muscle | 21.8 mm^2 | Standard Deviation 41.6 |
| Group 2: Avalglucosidase Alfa 20 mg/kg | Change From Baseline in Index of Real Muscle Mass (IRMM) of Skeletal Muscle Magnetic Resonance Imaging (MRI) Up to Week 442 | Week 104: IRMM - Hamstring muscle | 49.2 mm^2 | Standard Deviation 139 |
| Group 2: Avalglucosidase Alfa 20 mg/kg | Change From Baseline in Index of Real Muscle Mass (IRMM) of Skeletal Muscle Magnetic Resonance Imaging (MRI) Up to Week 442 | Week 104: IRMM - Leg extensor muscle | 34.9 mm^2 | Standard Deviation 33.2 |
| Group 2: Avalglucosidase Alfa 20 mg/kg | Change From Baseline in Index of Real Muscle Mass (IRMM) of Skeletal Muscle Magnetic Resonance Imaging (MRI) Up to Week 442 | Week 104: IRMM - Quadriceps muscle | 120.7 mm^2 | Standard Deviation 166.3 |
| Group 2: Avalglucosidase Alfa 20 mg/kg | Change From Baseline in Index of Real Muscle Mass (IRMM) of Skeletal Muscle Magnetic Resonance Imaging (MRI) Up to Week 442 | Week 104: IRMM - Triceps surae muscle | 181.4 mm^2 | Standard Deviation 143.7 |
| Group 2: Avalglucosidase Alfa 20 mg/kg | Change From Baseline in Index of Real Muscle Mass (IRMM) of Skeletal Muscle Magnetic Resonance Imaging (MRI) Up to Week 442 | Week 442: IRMM - Fibularis muscle | -54.9 mm^2 | — |
| Group 2: Avalglucosidase Alfa 20 mg/kg | Change From Baseline in Index of Real Muscle Mass (IRMM) of Skeletal Muscle Magnetic Resonance Imaging (MRI) Up to Week 442 | Week 442: IRMM - Hamstring muscle | -44.0 mm^2 | — |
| Group 2: Avalglucosidase Alfa 20 mg/kg | Change From Baseline in Index of Real Muscle Mass (IRMM) of Skeletal Muscle Magnetic Resonance Imaging (MRI) Up to Week 442 | Week 442: IRMM - Leg extensor muscle | -64.9 mm^2 | — |
| Group 2: Avalglucosidase Alfa 20 mg/kg | Change From Baseline in Index of Real Muscle Mass (IRMM) of Skeletal Muscle Magnetic Resonance Imaging (MRI) Up to Week 442 | Week 442: IRMM - Quadriceps muscle | -21.5 mm^2 | — |
Secondary
Change From Baseline in Skeletal Muscle Biopsy Up to Week 312
Skeletal muscle needle or open biopsy was performed on the lower extremity (quadriceps) muscle to assess glycogen content. The MRI appearance of the muscle was used to determine the level (axial slice position) that the biopsy procedure should target (avoiding fatty replaced tissue). Glycogen content was measured by histomorphometric analysis or severity grading to determine how effectively avalglucosidase alfa was able to remove glycogen from muscle.
Time frame: Baseline (Day 1) and Weeks 27, 104, 208, 260 and 312
Population: The PD analysis set included enrolled participants without any critical deviations related to IMP administration, and for whom any PD data were available. Only participants with data collected at specific time points are reported.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Group 1: Avalglucosidase Alfa 5 mg/kg | Change From Baseline in Skeletal Muscle Biopsy Up to Week 312 | Left quadriceps muscle: Week 27 | 0.8 percentage of glycogen | — |
| Group 1: Avalglucosidase Alfa 5 mg/kg | Change From Baseline in Skeletal Muscle Biopsy Up to Week 312 | Right quadriceps muscle: Week 27 | -1.7 percentage of glycogen | Standard Deviation 3.3 |
| Group 1: Avalglucosidase Alfa 10 mg/kg | Change From Baseline in Skeletal Muscle Biopsy Up to Week 312 | Right quadriceps muscle: Week 27 | 0.5 percentage of glycogen | Standard Deviation 1.5 |
| Group 1: Avalglucosidase Alfa 20 mg/kg | Change From Baseline in Skeletal Muscle Biopsy Up to Week 312 | Left quadriceps muscle: Week 27 | 0.9 percentage of glycogen | Standard Deviation 0.1 |
| Group 1: Avalglucosidase Alfa 20 mg/kg | Change From Baseline in Skeletal Muscle Biopsy Up to Week 312 | Right quadriceps muscle: Week 27 | -1.1 percentage of glycogen | — |
| Group 2: Avalglucosidase Alfa 5 mg/kg | Change From Baseline in Skeletal Muscle Biopsy Up to Week 312 | Left quadriceps muscle: Week 104 | -4.8 percentage of glycogen | Standard Deviation 9.9 |
| Group 2: Avalglucosidase Alfa 5 mg/kg | Change From Baseline in Skeletal Muscle Biopsy Up to Week 312 | Left quadriceps muscle: Week 260 | -4.3 percentage of glycogen | — |
| Group 2: Avalglucosidase Alfa 5 mg/kg | Change From Baseline in Skeletal Muscle Biopsy Up to Week 312 | Left quadriceps muscle: Week 27 | -3.6 percentage of glycogen | Standard Deviation 6.4 |
| Group 2: Avalglucosidase Alfa 10 mg/kg | Change From Baseline in Skeletal Muscle Biopsy Up to Week 312 | Left quadriceps muscle: Week 27 | 0.5 percentage of glycogen | — |
| Group 2: Avalglucosidase Alfa 10 mg/kg | Change From Baseline in Skeletal Muscle Biopsy Up to Week 312 | Right quadriceps muscle: Week 27 | 2.0 percentage of glycogen | Standard Deviation 1 |
| Group 2: Avalglucosidase Alfa 10 mg/kg | Change From Baseline in Skeletal Muscle Biopsy Up to Week 312 | Right quadriceps muscle: Week 104 | -2.8 percentage of glycogen | — |
| Group 2: Avalglucosidase Alfa 10 mg/kg | Change From Baseline in Skeletal Muscle Biopsy Up to Week 312 | Right quadriceps muscle: Week 208 | -3.8 percentage of glycogen | — |
| Group 2: Avalglucosidase Alfa 10 mg/kg | Change From Baseline in Skeletal Muscle Biopsy Up to Week 312 | Right quadriceps muscle: Week 312 | -5.4 percentage of glycogen | — |
| Group 2: Avalglucosidase Alfa 20 mg/kg | Change From Baseline in Skeletal Muscle Biopsy Up to Week 312 | Left quadriceps muscle: Week 27 | -0.4 percentage of glycogen | Standard Deviation 0.4 |
Secondary
Change From Baseline in T2 of Skeletal Muscle Magnetic Resonance Imaging (MRI) Up to Week 442
Skeletal muscle MRI performed prior to the muscle needle or open biopsy procedure using both qualitative (T1) and quantitative (T2, dixon) modalities to assess disease severity and detect treatment effects. The T1 weighted axial data was analyzed using the mercuri scale, which determines degree of intact muscle and fatty replacement, providing a qualitative measure of overall disease severity. Trophicity changes were evaluated for 5 muscle groups, including the upper leg muscles (quadriceps, hamstring) and the lower leg muscles (triceps, extensors, fibularis). The T2 multi-slice multi-spin echo and B1 mapping provided a quantitative measure of disease activity (edema, inflammation) within muscles.
Time frame: Baseline (Day 1) and Weeks 104 and 442
Population: The PD analysis set included enrolled participants without any critical deviations related to IMP administration, and for whom any PD data were available. Only participants with data collected at specific time points are reported.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Group 1: Avalglucosidase Alfa 5 mg/kg | Change From Baseline in T2 of Skeletal Muscle Magnetic Resonance Imaging (MRI) Up to Week 442 | Week 442: T2 - Fibularis muscle | -4.1 milliseconds (ms) | — |
| Group 1: Avalglucosidase Alfa 5 mg/kg | Change From Baseline in T2 of Skeletal Muscle Magnetic Resonance Imaging (MRI) Up to Week 442 | Week 442: T2 - Quadriceps muscle | -0.5 milliseconds (ms) | — |
| Group 1: Avalglucosidase Alfa 5 mg/kg | Change From Baseline in T2 of Skeletal Muscle Magnetic Resonance Imaging (MRI) Up to Week 442 | Week 442: T2 - Leg extensor muscle | -2.5 milliseconds (ms) | — |
| Group 1: Avalglucosidase Alfa 5 mg/kg | Change From Baseline in T2 of Skeletal Muscle Magnetic Resonance Imaging (MRI) Up to Week 442 | Week 442: T2 - Hamstring muscle | -6.0 milliseconds (ms) | — |
| Group 1: Avalglucosidase Alfa 5 mg/kg | Change From Baseline in T2 of Skeletal Muscle Magnetic Resonance Imaging (MRI) Up to Week 442 | Week 442: T2 - Triceps surae muscle | -3.5 milliseconds (ms) | — |
| Group 1: Avalglucosidase Alfa 20 mg/kg | Change From Baseline in T2 of Skeletal Muscle Magnetic Resonance Imaging (MRI) Up to Week 442 | Week 104: T2 - Fibularis muscle | -0.6 milliseconds (ms) | Standard Deviation 0.6 |
| Group 1: Avalglucosidase Alfa 20 mg/kg | Change From Baseline in T2 of Skeletal Muscle Magnetic Resonance Imaging (MRI) Up to Week 442 | Week 104: T2 - Triceps surae muscle | -0.3 milliseconds (ms) | Standard Deviation 0.3 |
| Group 1: Avalglucosidase Alfa 20 mg/kg | Change From Baseline in T2 of Skeletal Muscle Magnetic Resonance Imaging (MRI) Up to Week 442 | Week 104: T2 - Quadriceps muscle | 0.8 milliseconds (ms) | Standard Deviation 1 |
| Group 1: Avalglucosidase Alfa 20 mg/kg | Change From Baseline in T2 of Skeletal Muscle Magnetic Resonance Imaging (MRI) Up to Week 442 | Week 104: T2 - Leg extensor muscle | -0.8 milliseconds (ms) | Standard Deviation 1.6 |
| Group 1: Avalglucosidase Alfa 20 mg/kg | Change From Baseline in T2 of Skeletal Muscle Magnetic Resonance Imaging (MRI) Up to Week 442 | Week 104: T2 - Hamstring muscle | 0.7 milliseconds (ms) | — |
| Group 2: Avalglucosidase Alfa 5 mg/kg | Change From Baseline in T2 of Skeletal Muscle Magnetic Resonance Imaging (MRI) Up to Week 442 | Week 104: T2 - Fibularis muscle | 0.5 milliseconds (ms) | Standard Deviation 2.4 |
| Group 2: Avalglucosidase Alfa 5 mg/kg | Change From Baseline in T2 of Skeletal Muscle Magnetic Resonance Imaging (MRI) Up to Week 442 | Week 104: T2 - Hamstring muscle | 1.6 milliseconds (ms) | Standard Deviation 3.2 |
| Group 2: Avalglucosidase Alfa 5 mg/kg | Change From Baseline in T2 of Skeletal Muscle Magnetic Resonance Imaging (MRI) Up to Week 442 | Week 104: T2 - Leg extensor muscle | 2.0 milliseconds (ms) | Standard Deviation 2.2 |
| Group 2: Avalglucosidase Alfa 5 mg/kg | Change From Baseline in T2 of Skeletal Muscle Magnetic Resonance Imaging (MRI) Up to Week 442 | Week 104: T2 - Quadriceps muscle | 0.5 milliseconds (ms) | Standard Deviation 2.7 |
| Group 2: Avalglucosidase Alfa 5 mg/kg | Change From Baseline in T2 of Skeletal Muscle Magnetic Resonance Imaging (MRI) Up to Week 442 | Week 104: T2 - Triceps surae muscle | 0.5 milliseconds (ms) | Standard Deviation 1.7 |
| Group 2: Avalglucosidase Alfa 10 mg/kg | Change From Baseline in T2 of Skeletal Muscle Magnetic Resonance Imaging (MRI) Up to Week 442 | Week 442: T2 - Fibularis muscle | 0.7 milliseconds (ms) | — |
| Group 2: Avalglucosidase Alfa 10 mg/kg | Change From Baseline in T2 of Skeletal Muscle Magnetic Resonance Imaging (MRI) Up to Week 442 | Week 104: T2 - Hamstring muscle | -1.4 milliseconds (ms) | — |
| Group 2: Avalglucosidase Alfa 10 mg/kg | Change From Baseline in T2 of Skeletal Muscle Magnetic Resonance Imaging (MRI) Up to Week 442 | Week 442: T2 - Leg extensor muscle | 1.4 milliseconds (ms) | — |
| Group 2: Avalglucosidase Alfa 10 mg/kg | Change From Baseline in T2 of Skeletal Muscle Magnetic Resonance Imaging (MRI) Up to Week 442 | Week 104: T2 - Triceps surae muscle | -0.9 milliseconds (ms) | — |
| Group 2: Avalglucosidase Alfa 10 mg/kg | Change From Baseline in T2 of Skeletal Muscle Magnetic Resonance Imaging (MRI) Up to Week 442 | Week 104: T2 - Leg extensor muscle | 1.7 milliseconds (ms) | — |
| Group 2: Avalglucosidase Alfa 10 mg/kg | Change From Baseline in T2 of Skeletal Muscle Magnetic Resonance Imaging (MRI) Up to Week 442 | Week 442: T2 - Quadriceps muscle | 1.0 milliseconds (ms) | — |
| Group 2: Avalglucosidase Alfa 10 mg/kg | Change From Baseline in T2 of Skeletal Muscle Magnetic Resonance Imaging (MRI) Up to Week 442 | Week 442: T2 - Hamstring muscle | -9.1 milliseconds (ms) | — |
| Group 2: Avalglucosidase Alfa 10 mg/kg | Change From Baseline in T2 of Skeletal Muscle Magnetic Resonance Imaging (MRI) Up to Week 442 | Week 104: T2 - Fibularis muscle | 0.8 milliseconds (ms) | — |
| Group 2: Avalglucosidase Alfa 10 mg/kg | Change From Baseline in T2 of Skeletal Muscle Magnetic Resonance Imaging (MRI) Up to Week 442 | Week 442: T2 - Triceps surae muscle | 1.8 milliseconds (ms) | — |
| Group 2: Avalglucosidase Alfa 10 mg/kg | Change From Baseline in T2 of Skeletal Muscle Magnetic Resonance Imaging (MRI) Up to Week 442 | Week 104: T2 - Quadriceps muscle | -1.3 milliseconds (ms) | — |
| Group 2: Avalglucosidase Alfa 20 mg/kg | Change From Baseline in T2 of Skeletal Muscle Magnetic Resonance Imaging (MRI) Up to Week 442 | Week 442: T2 - Hamstring muscle | 2.2 milliseconds (ms) | — |
| Group 2: Avalglucosidase Alfa 20 mg/kg | Change From Baseline in T2 of Skeletal Muscle Magnetic Resonance Imaging (MRI) Up to Week 442 | Week 104: T2 - Leg extensor muscle | -0.3 milliseconds (ms) | Standard Deviation 0.7 |
| Group 2: Avalglucosidase Alfa 20 mg/kg | Change From Baseline in T2 of Skeletal Muscle Magnetic Resonance Imaging (MRI) Up to Week 442 | Week 442: T2 - Triceps surae muscle | -0.0 milliseconds (ms) | — |
| Group 2: Avalglucosidase Alfa 20 mg/kg | Change From Baseline in T2 of Skeletal Muscle Magnetic Resonance Imaging (MRI) Up to Week 442 | Week 104: T2 - Triceps surae muscle | 0.4 milliseconds (ms) | Standard Deviation 0.6 |
| Group 2: Avalglucosidase Alfa 20 mg/kg | Change From Baseline in T2 of Skeletal Muscle Magnetic Resonance Imaging (MRI) Up to Week 442 | Week 442: T2 - Fibularis muscle | 0.1 milliseconds (ms) | — |
| Group 2: Avalglucosidase Alfa 20 mg/kg | Change From Baseline in T2 of Skeletal Muscle Magnetic Resonance Imaging (MRI) Up to Week 442 | Week 104: T2 - Hamstring muscle | 0.2 milliseconds (ms) | Standard Deviation 0.7 |
| Group 2: Avalglucosidase Alfa 20 mg/kg | Change From Baseline in T2 of Skeletal Muscle Magnetic Resonance Imaging (MRI) Up to Week 442 | Week 104: T2 - Quadriceps muscle | 1.0 milliseconds (ms) | Standard Deviation 0.6 |
| Group 2: Avalglucosidase Alfa 20 mg/kg | Change From Baseline in T2 of Skeletal Muscle Magnetic Resonance Imaging (MRI) Up to Week 442 | Week 442: T2 - Leg extensor muscle | 0.6 milliseconds (ms) | — |
| Group 2: Avalglucosidase Alfa 20 mg/kg | Change From Baseline in T2 of Skeletal Muscle Magnetic Resonance Imaging (MRI) Up to Week 442 | Week 104: T2 - Fibularis muscle | 0.0 milliseconds (ms) | Standard Deviation 0.6 |
| Group 2: Avalglucosidase Alfa 20 mg/kg | Change From Baseline in T2 of Skeletal Muscle Magnetic Resonance Imaging (MRI) Up to Week 442 | Week 442: T2 - Quadriceps muscle | 3.4 milliseconds (ms) | — |
Secondary
Change From Baseline in T2 With B1 of Skeletal Muscle Magnetic Resonance Imaging (MRI) Up to Week 442
Skeletal muscle MRI performed prior to the muscle needle or open biopsy procedure using both qualitative (T1) and quantitative (T2, dixon) modalities to assess disease severity and detect treatment effects. The T1 weighted axial data was analyzed using the mercuri scale, which determines degree of intact muscle and fatty replacement, providing a qualitative measure of overall disease severity. Trophicity changes were evaluated for 5 muscle groups, including the upper leg muscles (quadriceps, hamstring) and the lower leg muscles (triceps, extensors, fibularis). The T2 multi-slice multi-spin echo and B1 mapping provided a quantitative measure of disease activity (edema, inflammation) within muscles.
Time frame: Baseline (Day 1) and Weeks 104 and 442
Population: The PD analysis set included enrolled participants without any critical deviations related to IMP administration, and for whom any PD data were available. Only participants with data collected at specific time points are reported.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Group 1: Avalglucosidase Alfa 5 mg/kg | Change From Baseline in T2 With B1 of Skeletal Muscle Magnetic Resonance Imaging (MRI) Up to Week 442 | Week 442: T2 with B1 - Fibularis muscle | -4.1 milliseconds (ms) | — |
| Group 1: Avalglucosidase Alfa 5 mg/kg | Change From Baseline in T2 With B1 of Skeletal Muscle Magnetic Resonance Imaging (MRI) Up to Week 442 | Week 442: T2 with B1 - Triceps surae muscle | -3.2 milliseconds (ms) | — |
| Group 1: Avalglucosidase Alfa 5 mg/kg | Change From Baseline in T2 With B1 of Skeletal Muscle Magnetic Resonance Imaging (MRI) Up to Week 442 | Week 442: T2 with B1 - Quadriceps muscle | -0.3 milliseconds (ms) | — |
| Group 1: Avalglucosidase Alfa 5 mg/kg | Change From Baseline in T2 With B1 of Skeletal Muscle Magnetic Resonance Imaging (MRI) Up to Week 442 | Week 442: T2 with B1 - Leg extensor muscle | -3.6 milliseconds (ms) | — |
| Group 1: Avalglucosidase Alfa 20 mg/kg | Change From Baseline in T2 With B1 of Skeletal Muscle Magnetic Resonance Imaging (MRI) Up to Week 442 | Week 104: T2 with B1 - Quadriceps muscle | 0.9 milliseconds (ms) | Standard Deviation 0.7 |
| Group 1: Avalglucosidase Alfa 20 mg/kg | Change From Baseline in T2 With B1 of Skeletal Muscle Magnetic Resonance Imaging (MRI) Up to Week 442 | Week 104: T2 with B1 - Hamstring muscle | -0.4 milliseconds (ms) | — |
| Group 1: Avalglucosidase Alfa 20 mg/kg | Change From Baseline in T2 With B1 of Skeletal Muscle Magnetic Resonance Imaging (MRI) Up to Week 442 | Week 104: T2 with B1 - Leg extensor muscle | -0.6 milliseconds (ms) | Standard Deviation 1.9 |
| Group 1: Avalglucosidase Alfa 20 mg/kg | Change From Baseline in T2 With B1 of Skeletal Muscle Magnetic Resonance Imaging (MRI) Up to Week 442 | Week 104: T2 with B1 - Fibularis muscle | -0.5 milliseconds (ms) | Standard Deviation 0.7 |
| Group 1: Avalglucosidase Alfa 20 mg/kg | Change From Baseline in T2 With B1 of Skeletal Muscle Magnetic Resonance Imaging (MRI) Up to Week 442 | Week 104: T2 with B1 - Triceps surae muscle | -0.3 milliseconds (ms) | Standard Deviation 0.4 |
| Group 2: Avalglucosidase Alfa 5 mg/kg | Change From Baseline in T2 With B1 of Skeletal Muscle Magnetic Resonance Imaging (MRI) Up to Week 442 | Week 104: T2 with B1 - Leg extensor muscle | 2.0 milliseconds (ms) | Standard Deviation 2.2 |
| Group 2: Avalglucosidase Alfa 5 mg/kg | Change From Baseline in T2 With B1 of Skeletal Muscle Magnetic Resonance Imaging (MRI) Up to Week 442 | Week 104: T2 with B1 - Triceps surae muscle | 0.1 milliseconds (ms) | Standard Deviation 1.2 |
| Group 2: Avalglucosidase Alfa 5 mg/kg | Change From Baseline in T2 With B1 of Skeletal Muscle Magnetic Resonance Imaging (MRI) Up to Week 442 | Week 104: T2 with B1 - Fibularis muscle | 0.5 milliseconds (ms) | Standard Deviation 2.2 |
| Group 2: Avalglucosidase Alfa 5 mg/kg | Change From Baseline in T2 With B1 of Skeletal Muscle Magnetic Resonance Imaging (MRI) Up to Week 442 | Week 104: T2 with B1 - Hamstring muscle | 2.3 milliseconds (ms) | Standard Deviation 4.6 |
| Group 2: Avalglucosidase Alfa 5 mg/kg | Change From Baseline in T2 With B1 of Skeletal Muscle Magnetic Resonance Imaging (MRI) Up to Week 442 | Week 104: T2 with B1 - Quadriceps muscle | 0.7 milliseconds (ms) | Standard Deviation 2.8 |
| Group 2: Avalglucosidase Alfa 10 mg/kg | Change From Baseline in T2 With B1 of Skeletal Muscle Magnetic Resonance Imaging (MRI) Up to Week 442 | Week 104: T2 with B1 - Fibularis muscle | 1.4 milliseconds (ms) | — |
| Group 2: Avalglucosidase Alfa 10 mg/kg | Change From Baseline in T2 With B1 of Skeletal Muscle Magnetic Resonance Imaging (MRI) Up to Week 442 | Week 104: T2 with B1 - Leg extensor muscle | 0.7 milliseconds (ms) | — |
| Group 2: Avalglucosidase Alfa 10 mg/kg | Change From Baseline in T2 With B1 of Skeletal Muscle Magnetic Resonance Imaging (MRI) Up to Week 442 | Week 104: T2 with B1 - Quadriceps muscle | -1.4 milliseconds (ms) | — |
| Group 2: Avalglucosidase Alfa 10 mg/kg | Change From Baseline in T2 With B1 of Skeletal Muscle Magnetic Resonance Imaging (MRI) Up to Week 442 | Week 104: T2 with B1 - Triceps surae muscle | -1.3 milliseconds (ms) | — |
| Group 2: Avalglucosidase Alfa 10 mg/kg | Change From Baseline in T2 With B1 of Skeletal Muscle Magnetic Resonance Imaging (MRI) Up to Week 442 | Week 104: T2 with B1 - Hamstring muscle | -2.3 milliseconds (ms) | — |
| Group 2: Avalglucosidase Alfa 20 mg/kg | Change From Baseline in T2 With B1 of Skeletal Muscle Magnetic Resonance Imaging (MRI) Up to Week 442 | Week 104: T2 with B1 - Triceps surae muscle | 0.5 milliseconds (ms) | — |
| Group 2: Avalglucosidase Alfa 20 mg/kg | Change From Baseline in T2 With B1 of Skeletal Muscle Magnetic Resonance Imaging (MRI) Up to Week 442 | Week 104: T2 with B1 - Fibularis muscle | 0.3 milliseconds (ms) | — |
| Group 2: Avalglucosidase Alfa 20 mg/kg | Change From Baseline in T2 With B1 of Skeletal Muscle Magnetic Resonance Imaging (MRI) Up to Week 442 | Week 104: T2 with B1 - Leg extensor muscle | -2.1 milliseconds (ms) | — |
| Group 2: Avalglucosidase Alfa 20 mg/kg | Change From Baseline in T2 With B1 of Skeletal Muscle Magnetic Resonance Imaging (MRI) Up to Week 442 | Week 104: T2 with B1 - Quadriceps muscle | 1.6 milliseconds (ms) | Standard Deviation 0.6 |
| Group 2: Avalglucosidase Alfa 20 mg/kg | Change From Baseline in T2 With B1 of Skeletal Muscle Magnetic Resonance Imaging (MRI) Up to Week 442 | Week 104: T2 with B1 - Hamstring muscle | -0.4 milliseconds (ms) | Standard Deviation 1.6 |
Secondary
Change From Baseline in Urinary Glucose Tetrasaccharide (Hex4) Level Up to Week 442
The Hex4, a tetraglucose oligomer, has been shown to be elevated in the urine of participants with Pompe disease. Hence, determination of Hex4 levels may be a means by which the efficacy of treatments were monitored. Urine samples were collected prior to IMP infusion for the assessment of urinary Hex4 concentrations.
Time frame: Baseline (Day 1) and Weeks 1, 3, 5, 7, 9, 11, 13, 15, 17, 19, 21, 23, 25, 27, 52, 78, 104, 130, 156, 182, 208, 234, 260, 286, 312, 338, 364, 390, 416 and 442
Population: The PD analysis set included enrolled participants without any critical deviations related to IMP administration, and for whom any PD data were available.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Group 1: Avalglucosidase Alfa 5 mg/kg | Change From Baseline in Urinary Glucose Tetrasaccharide (Hex4) Level Up to Week 442 | Week 25 | -2.5 mmol per mole | Standard Deviation 3 |
| Group 1: Avalglucosidase Alfa 5 mg/kg | Change From Baseline in Urinary Glucose Tetrasaccharide (Hex4) Level Up to Week 442 | Week 260 | -1.9 mmol per mole | Standard Deviation 0.4 |
| Group 1: Avalglucosidase Alfa 5 mg/kg | Change From Baseline in Urinary Glucose Tetrasaccharide (Hex4) Level Up to Week 442 | Week 156 | -3.6 mmol per mole | Standard Deviation 3.1 |
| Group 1: Avalglucosidase Alfa 5 mg/kg | Change From Baseline in Urinary Glucose Tetrasaccharide (Hex4) Level Up to Week 442 | Week 312 | -3.6 mmol per mole | Standard Deviation 3 |
| Group 1: Avalglucosidase Alfa 5 mg/kg | Change From Baseline in Urinary Glucose Tetrasaccharide (Hex4) Level Up to Week 442 | Week 286 | -7.9 mmol per mole | — |
| Group 1: Avalglucosidase Alfa 5 mg/kg | Change From Baseline in Urinary Glucose Tetrasaccharide (Hex4) Level Up to Week 442 | Week 11 | -0.1 mmol per mole | Standard Deviation 1.9 |
| Group 1: Avalglucosidase Alfa 5 mg/kg | Change From Baseline in Urinary Glucose Tetrasaccharide (Hex4) Level Up to Week 442 | Week 23 | -1.7 mmol per mole | Standard Deviation 1.7 |
| Group 1: Avalglucosidase Alfa 5 mg/kg | Change From Baseline in Urinary Glucose Tetrasaccharide (Hex4) Level Up to Week 442 | Week 13 | -1.3 mmol per mole | Standard Deviation 1.3 |
| Group 1: Avalglucosidase Alfa 5 mg/kg | Change From Baseline in Urinary Glucose Tetrasaccharide (Hex4) Level Up to Week 442 | Week 104 | -3.0 mmol per mole | Standard Deviation 2.9 |
| Group 1: Avalglucosidase Alfa 5 mg/kg | Change From Baseline in Urinary Glucose Tetrasaccharide (Hex4) Level Up to Week 442 | Week 1 | 0.5 mmol per mole | — |
| Group 1: Avalglucosidase Alfa 5 mg/kg | Change From Baseline in Urinary Glucose Tetrasaccharide (Hex4) Level Up to Week 442 | Week 5 | -1.0 mmol per mole | Standard Deviation 0.5 |
| Group 1: Avalglucosidase Alfa 5 mg/kg | Change From Baseline in Urinary Glucose Tetrasaccharide (Hex4) Level Up to Week 442 | Week 182 | -3.5 mmol per mole | Standard Deviation 2.6 |
| Group 1: Avalglucosidase Alfa 5 mg/kg | Change From Baseline in Urinary Glucose Tetrasaccharide (Hex4) Level Up to Week 442 | Week 78 | -1.9 mmol per mole | Standard Deviation 1.3 |
| Group 1: Avalglucosidase Alfa 5 mg/kg | Change From Baseline in Urinary Glucose Tetrasaccharide (Hex4) Level Up to Week 442 | Week 130 | -4.0 mmol per mole | Standard Deviation 3.9 |
| Group 1: Avalglucosidase Alfa 5 mg/kg | Change From Baseline in Urinary Glucose Tetrasaccharide (Hex4) Level Up to Week 442 | Week 21 | -2.3 mmol per mole | Standard Deviation 2.1 |
| Group 1: Avalglucosidase Alfa 5 mg/kg | Change From Baseline in Urinary Glucose Tetrasaccharide (Hex4) Level Up to Week 442 | Week 9 | -0.6 mmol per mole | Standard Deviation 1.7 |
| Group 1: Avalglucosidase Alfa 5 mg/kg | Change From Baseline in Urinary Glucose Tetrasaccharide (Hex4) Level Up to Week 442 | Week 208 | -3.2 mmol per mole | Standard Deviation 3.2 |
| Group 1: Avalglucosidase Alfa 5 mg/kg | Change From Baseline in Urinary Glucose Tetrasaccharide (Hex4) Level Up to Week 442 | Week 390 | -2.6 mmol per mole | — |
| Group 1: Avalglucosidase Alfa 5 mg/kg | Change From Baseline in Urinary Glucose Tetrasaccharide (Hex4) Level Up to Week 442 | Week 364 | -2.9 mmol per mole | — |
| Group 1: Avalglucosidase Alfa 5 mg/kg | Change From Baseline in Urinary Glucose Tetrasaccharide (Hex4) Level Up to Week 442 | Week 3 | -0.6 mmol per mole | Standard Deviation 0.2 |
| Group 1: Avalglucosidase Alfa 5 mg/kg | Change From Baseline in Urinary Glucose Tetrasaccharide (Hex4) Level Up to Week 442 | Week 19 | -2.3 mmol per mole | Standard Deviation 2.4 |
| Group 1: Avalglucosidase Alfa 5 mg/kg | Change From Baseline in Urinary Glucose Tetrasaccharide (Hex4) Level Up to Week 442 | Week 17 | -1.0 mmol per mole | Standard Deviation 0.3 |
| Group 1: Avalglucosidase Alfa 5 mg/kg | Change From Baseline in Urinary Glucose Tetrasaccharide (Hex4) Level Up to Week 442 | Week 27 | -2.6 mmol per mole | — |
| Group 1: Avalglucosidase Alfa 5 mg/kg | Change From Baseline in Urinary Glucose Tetrasaccharide (Hex4) Level Up to Week 442 | Week 7 | -1.2 mmol per mole | Standard Deviation 1.1 |
| Group 1: Avalglucosidase Alfa 5 mg/kg | Change From Baseline in Urinary Glucose Tetrasaccharide (Hex4) Level Up to Week 442 | Week 52 | -1.8 mmol per mole | Standard Deviation 1.6 |
| Group 1: Avalglucosidase Alfa 5 mg/kg | Change From Baseline in Urinary Glucose Tetrasaccharide (Hex4) Level Up to Week 442 | Week 234 | -3.4 mmol per mole | Standard Deviation 3.2 |
| Group 1: Avalglucosidase Alfa 5 mg/kg | Change From Baseline in Urinary Glucose Tetrasaccharide (Hex4) Level Up to Week 442 | Week 442 | -8.5 mmol per mole | — |
| Group 1: Avalglucosidase Alfa 5 mg/kg | Change From Baseline in Urinary Glucose Tetrasaccharide (Hex4) Level Up to Week 442 | Week 15 | -1.4 mmol per mole | Standard Deviation 0.5 |
| Group 1: Avalglucosidase Alfa 10 mg/kg | Change From Baseline in Urinary Glucose Tetrasaccharide (Hex4) Level Up to Week 442 | Week 182 | -11.6 mmol per mole | — |
| Group 1: Avalglucosidase Alfa 10 mg/kg | Change From Baseline in Urinary Glucose Tetrasaccharide (Hex4) Level Up to Week 442 | Week 52 | -4.1 mmol per mole | Standard Deviation 5.2 |
| Group 1: Avalglucosidase Alfa 10 mg/kg | Change From Baseline in Urinary Glucose Tetrasaccharide (Hex4) Level Up to Week 442 | Week 104 | -6.4 mmol per mole | — |
| Group 1: Avalglucosidase Alfa 10 mg/kg | Change From Baseline in Urinary Glucose Tetrasaccharide (Hex4) Level Up to Week 442 | Week 3 | -1.9 mmol per mole | Standard Deviation 1.6 |
| Group 1: Avalglucosidase Alfa 10 mg/kg | Change From Baseline in Urinary Glucose Tetrasaccharide (Hex4) Level Up to Week 442 | Week 5 | -0.2 mmol per mole | Standard Deviation 0.4 |
| Group 1: Avalglucosidase Alfa 10 mg/kg | Change From Baseline in Urinary Glucose Tetrasaccharide (Hex4) Level Up to Week 442 | Week 338 | -0.4 mmol per mole | — |
| Group 1: Avalglucosidase Alfa 10 mg/kg | Change From Baseline in Urinary Glucose Tetrasaccharide (Hex4) Level Up to Week 442 | Week 7 | -2.2 mmol per mole | Standard Deviation 1.7 |
| Group 1: Avalglucosidase Alfa 10 mg/kg | Change From Baseline in Urinary Glucose Tetrasaccharide (Hex4) Level Up to Week 442 | Week 9 | -2.4 mmol per mole | Standard Deviation 3.2 |
| Group 1: Avalglucosidase Alfa 10 mg/kg | Change From Baseline in Urinary Glucose Tetrasaccharide (Hex4) Level Up to Week 442 | Week 11 | -2.7 mmol per mole | Standard Deviation 2.6 |
| Group 1: Avalglucosidase Alfa 10 mg/kg | Change From Baseline in Urinary Glucose Tetrasaccharide (Hex4) Level Up to Week 442 | Week 260 | -10.1 mmol per mole | — |
| Group 1: Avalglucosidase Alfa 10 mg/kg | Change From Baseline in Urinary Glucose Tetrasaccharide (Hex4) Level Up to Week 442 | Week 13 | -5.0 mmol per mole | Standard Deviation 3.4 |
| Group 1: Avalglucosidase Alfa 10 mg/kg | Change From Baseline in Urinary Glucose Tetrasaccharide (Hex4) Level Up to Week 442 | Week 15 | -6.2 mmol per mole | Standard Deviation 3.3 |
| Group 1: Avalglucosidase Alfa 10 mg/kg | Change From Baseline in Urinary Glucose Tetrasaccharide (Hex4) Level Up to Week 442 | Week 156 | -10.4 mmol per mole | — |
| Group 1: Avalglucosidase Alfa 10 mg/kg | Change From Baseline in Urinary Glucose Tetrasaccharide (Hex4) Level Up to Week 442 | Week 234 | -3.8 mmol per mole | — |
| Group 1: Avalglucosidase Alfa 10 mg/kg | Change From Baseline in Urinary Glucose Tetrasaccharide (Hex4) Level Up to Week 442 | Week 17 | -6.7 mmol per mole | Standard Deviation 2.1 |
| Group 1: Avalglucosidase Alfa 10 mg/kg | Change From Baseline in Urinary Glucose Tetrasaccharide (Hex4) Level Up to Week 442 | Week 208 | -6.5 mmol per mole | — |
| Group 1: Avalglucosidase Alfa 10 mg/kg | Change From Baseline in Urinary Glucose Tetrasaccharide (Hex4) Level Up to Week 442 | Week 19 | -6.5 mmol per mole | Standard Deviation 2.5 |
| Group 1: Avalglucosidase Alfa 10 mg/kg | Change From Baseline in Urinary Glucose Tetrasaccharide (Hex4) Level Up to Week 442 | Week 416 | -12.5 mmol per mole | — |
| Group 1: Avalglucosidase Alfa 10 mg/kg | Change From Baseline in Urinary Glucose Tetrasaccharide (Hex4) Level Up to Week 442 | Week 21 | -6.5 mmol per mole | Standard Deviation 2.2 |
| Group 1: Avalglucosidase Alfa 10 mg/kg | Change From Baseline in Urinary Glucose Tetrasaccharide (Hex4) Level Up to Week 442 | Week 25 | -4.5 mmol per mole | Standard Deviation 1 |
| Group 1: Avalglucosidase Alfa 10 mg/kg | Change From Baseline in Urinary Glucose Tetrasaccharide (Hex4) Level Up to Week 442 | Week 23 | -5.3 mmol per mole | Standard Deviation 1.3 |
| Group 1: Avalglucosidase Alfa 10 mg/kg | Change From Baseline in Urinary Glucose Tetrasaccharide (Hex4) Level Up to Week 442 | Week 78 | -3.5 mmol per mole | Standard Deviation 5 |
| Group 1: Avalglucosidase Alfa 10 mg/kg | Change From Baseline in Urinary Glucose Tetrasaccharide (Hex4) Level Up to Week 442 | Week 130 | -9.8 mmol per mole | — |
| Group 1: Avalglucosidase Alfa 20 mg/kg | Change From Baseline in Urinary Glucose Tetrasaccharide (Hex4) Level Up to Week 442 | Week 338 | -4.2 mmol per mole | Standard Deviation 5.5 |
| Group 1: Avalglucosidase Alfa 20 mg/kg | Change From Baseline in Urinary Glucose Tetrasaccharide (Hex4) Level Up to Week 442 | Week 156 | -1.9 mmol per mole | Standard Deviation 3.2 |
| Group 1: Avalglucosidase Alfa 20 mg/kg | Change From Baseline in Urinary Glucose Tetrasaccharide (Hex4) Level Up to Week 442 | Week 208 | -2.6 mmol per mole | Standard Deviation 3.9 |
| Group 1: Avalglucosidase Alfa 20 mg/kg | Change From Baseline in Urinary Glucose Tetrasaccharide (Hex4) Level Up to Week 442 | Week 52 | -2.0 mmol per mole | Standard Deviation 2 |
| Group 1: Avalglucosidase Alfa 20 mg/kg | Change From Baseline in Urinary Glucose Tetrasaccharide (Hex4) Level Up to Week 442 | Week 17 | -1.5 mmol per mole | Standard Deviation 1.2 |
| Group 1: Avalglucosidase Alfa 20 mg/kg | Change From Baseline in Urinary Glucose Tetrasaccharide (Hex4) Level Up to Week 442 | Week 25 | -1.3 mmol per mole | Standard Deviation 2.3 |
| Group 1: Avalglucosidase Alfa 20 mg/kg | Change From Baseline in Urinary Glucose Tetrasaccharide (Hex4) Level Up to Week 442 | Week 19 | -1.6 mmol per mole | Standard Deviation 1 |
| Group 1: Avalglucosidase Alfa 20 mg/kg | Change From Baseline in Urinary Glucose Tetrasaccharide (Hex4) Level Up to Week 442 | Week 23 | -0.7 mmol per mole | Standard Deviation 1.4 |
| Group 1: Avalglucosidase Alfa 20 mg/kg | Change From Baseline in Urinary Glucose Tetrasaccharide (Hex4) Level Up to Week 442 | Week 5 | -1.0 mmol per mole | Standard Deviation 1.1 |
| Group 1: Avalglucosidase Alfa 20 mg/kg | Change From Baseline in Urinary Glucose Tetrasaccharide (Hex4) Level Up to Week 442 | Week 182 | -2.2 mmol per mole | Standard Deviation 2.7 |
| Group 1: Avalglucosidase Alfa 20 mg/kg | Change From Baseline in Urinary Glucose Tetrasaccharide (Hex4) Level Up to Week 442 | Week 260 | -1.7 mmol per mole | Standard Deviation 4 |
| Group 1: Avalglucosidase Alfa 20 mg/kg | Change From Baseline in Urinary Glucose Tetrasaccharide (Hex4) Level Up to Week 442 | Week 3 | -0.9 mmol per mole | Standard Deviation 0.5 |
| Group 1: Avalglucosidase Alfa 20 mg/kg | Change From Baseline in Urinary Glucose Tetrasaccharide (Hex4) Level Up to Week 442 | Week 11 | 0.4 mmol per mole | Standard Deviation 1 |
| Group 1: Avalglucosidase Alfa 20 mg/kg | Change From Baseline in Urinary Glucose Tetrasaccharide (Hex4) Level Up to Week 442 | Week 104 | -2.8 mmol per mole | Standard Deviation 3.4 |
| Group 1: Avalglucosidase Alfa 20 mg/kg | Change From Baseline in Urinary Glucose Tetrasaccharide (Hex4) Level Up to Week 442 | Week 13 | -1.6 mmol per mole | Standard Deviation 1.5 |
| Group 1: Avalglucosidase Alfa 20 mg/kg | Change From Baseline in Urinary Glucose Tetrasaccharide (Hex4) Level Up to Week 442 | Week 416 | -1.9 mmol per mole | Standard Deviation 3.2 |
| Group 1: Avalglucosidase Alfa 20 mg/kg | Change From Baseline in Urinary Glucose Tetrasaccharide (Hex4) Level Up to Week 442 | Week 9 | 0.5 mmol per mole | Standard Deviation 0.7 |
| Group 1: Avalglucosidase Alfa 20 mg/kg | Change From Baseline in Urinary Glucose Tetrasaccharide (Hex4) Level Up to Week 442 | Week 234 | -4.4 mmol per mole | — |
| Group 1: Avalglucosidase Alfa 20 mg/kg | Change From Baseline in Urinary Glucose Tetrasaccharide (Hex4) Level Up to Week 442 | Week 312 | -4.3 mmol per mole | Standard Deviation 3.9 |
| Group 1: Avalglucosidase Alfa 20 mg/kg | Change From Baseline in Urinary Glucose Tetrasaccharide (Hex4) Level Up to Week 442 | Week 21 | -1.8 mmol per mole | Standard Deviation 1.3 |
| Group 1: Avalglucosidase Alfa 20 mg/kg | Change From Baseline in Urinary Glucose Tetrasaccharide (Hex4) Level Up to Week 442 | Week 15 | -1.4 mmol per mole | Standard Deviation 0.5 |
| Group 1: Avalglucosidase Alfa 20 mg/kg | Change From Baseline in Urinary Glucose Tetrasaccharide (Hex4) Level Up to Week 442 | Week 78 | -2.9 mmol per mole | Standard Deviation 3.2 |
| Group 1: Avalglucosidase Alfa 20 mg/kg | Change From Baseline in Urinary Glucose Tetrasaccharide (Hex4) Level Up to Week 442 | Week 130 | -2.4 mmol per mole | Standard Deviation 3.3 |
| Group 1: Avalglucosidase Alfa 20 mg/kg | Change From Baseline in Urinary Glucose Tetrasaccharide (Hex4) Level Up to Week 442 | Week 7 | -0.6 mmol per mole | Standard Deviation 1.3 |
| Group 2: Avalglucosidase Alfa 5 mg/kg | Change From Baseline in Urinary Glucose Tetrasaccharide (Hex4) Level Up to Week 442 | Week 25 | 0.3 mmol per mole | Standard Deviation 3.7 |
| Group 2: Avalglucosidase Alfa 5 mg/kg | Change From Baseline in Urinary Glucose Tetrasaccharide (Hex4) Level Up to Week 442 | Week 17 | -0.0 mmol per mole | Standard Deviation 1.4 |
| Group 2: Avalglucosidase Alfa 5 mg/kg | Change From Baseline in Urinary Glucose Tetrasaccharide (Hex4) Level Up to Week 442 | Week 3 | -0.9 mmol per mole | Standard Deviation 1 |
| Group 2: Avalglucosidase Alfa 5 mg/kg | Change From Baseline in Urinary Glucose Tetrasaccharide (Hex4) Level Up to Week 442 | Week 5 | 0.2 mmol per mole | Standard Deviation 0.7 |
| Group 2: Avalglucosidase Alfa 5 mg/kg | Change From Baseline in Urinary Glucose Tetrasaccharide (Hex4) Level Up to Week 442 | Week 7 | -0.7 mmol per mole | Standard Deviation 0.8 |
| Group 2: Avalglucosidase Alfa 5 mg/kg | Change From Baseline in Urinary Glucose Tetrasaccharide (Hex4) Level Up to Week 442 | Week 9 | 0.0 mmol per mole | Standard Deviation 1.3 |
| Group 2: Avalglucosidase Alfa 5 mg/kg | Change From Baseline in Urinary Glucose Tetrasaccharide (Hex4) Level Up to Week 442 | Week 11 | 0.1 mmol per mole | Standard Deviation 0.8 |
| Group 2: Avalglucosidase Alfa 5 mg/kg | Change From Baseline in Urinary Glucose Tetrasaccharide (Hex4) Level Up to Week 442 | Week 13 | 0.5 mmol per mole | Standard Deviation 0.7 |
| Group 2: Avalglucosidase Alfa 5 mg/kg | Change From Baseline in Urinary Glucose Tetrasaccharide (Hex4) Level Up to Week 442 | Week 15 | 0.6 mmol per mole | Standard Deviation 1.5 |
| Group 2: Avalglucosidase Alfa 5 mg/kg | Change From Baseline in Urinary Glucose Tetrasaccharide (Hex4) Level Up to Week 442 | Week 19 | 0.3 mmol per mole | Standard Deviation 3.1 |
| Group 2: Avalglucosidase Alfa 5 mg/kg | Change From Baseline in Urinary Glucose Tetrasaccharide (Hex4) Level Up to Week 442 | Week 21 | 0.1 mmol per mole | Standard Deviation 2.2 |
| Group 2: Avalglucosidase Alfa 5 mg/kg | Change From Baseline in Urinary Glucose Tetrasaccharide (Hex4) Level Up to Week 442 | Week 23 | 0.7 mmol per mole | Standard Deviation 3.1 |
| Group 2: Avalglucosidase Alfa 5 mg/kg | Change From Baseline in Urinary Glucose Tetrasaccharide (Hex4) Level Up to Week 442 | Week 52 | 0.2 mmol per mole | Standard Deviation 2.7 |
| Group 2: Avalglucosidase Alfa 5 mg/kg | Change From Baseline in Urinary Glucose Tetrasaccharide (Hex4) Level Up to Week 442 | Week 78 | -0.9 mmol per mole | Standard Deviation 2.4 |
| Group 2: Avalglucosidase Alfa 5 mg/kg | Change From Baseline in Urinary Glucose Tetrasaccharide (Hex4) Level Up to Week 442 | Week 104 | 0.8 mmol per mole | Standard Deviation 3.8 |
| Group 2: Avalglucosidase Alfa 5 mg/kg | Change From Baseline in Urinary Glucose Tetrasaccharide (Hex4) Level Up to Week 442 | Week 130 | -1.4 mmol per mole | Standard Deviation 3.9 |
| Group 2: Avalglucosidase Alfa 5 mg/kg | Change From Baseline in Urinary Glucose Tetrasaccharide (Hex4) Level Up to Week 442 | Week 156 | -1.4 mmol per mole | Standard Deviation 3.2 |
| Group 2: Avalglucosidase Alfa 5 mg/kg | Change From Baseline in Urinary Glucose Tetrasaccharide (Hex4) Level Up to Week 442 | Week 182 | -3.1 mmol per mole | Standard Deviation 3.2 |
| Group 2: Avalglucosidase Alfa 5 mg/kg | Change From Baseline in Urinary Glucose Tetrasaccharide (Hex4) Level Up to Week 442 | Week 208 | -3.9 mmol per mole | Standard Deviation 3.5 |
| Group 2: Avalglucosidase Alfa 5 mg/kg | Change From Baseline in Urinary Glucose Tetrasaccharide (Hex4) Level Up to Week 442 | Week 234 | -4.1 mmol per mole | Standard Deviation 2.5 |
| Group 2: Avalglucosidase Alfa 5 mg/kg | Change From Baseline in Urinary Glucose Tetrasaccharide (Hex4) Level Up to Week 442 | Week 260 | -5.9 mmol per mole | Standard Deviation 0.1 |
| Group 2: Avalglucosidase Alfa 5 mg/kg | Change From Baseline in Urinary Glucose Tetrasaccharide (Hex4) Level Up to Week 442 | Week 286 | -3.6 mmol per mole | Standard Deviation 2.2 |
| Group 2: Avalglucosidase Alfa 5 mg/kg | Change From Baseline in Urinary Glucose Tetrasaccharide (Hex4) Level Up to Week 442 | Week 312 | -4.6 mmol per mole | Standard Deviation 1.9 |
| Group 2: Avalglucosidase Alfa 5 mg/kg | Change From Baseline in Urinary Glucose Tetrasaccharide (Hex4) Level Up to Week 442 | Week 338 | -4.9 mmol per mole | — |
| Group 2: Avalglucosidase Alfa 5 mg/kg | Change From Baseline in Urinary Glucose Tetrasaccharide (Hex4) Level Up to Week 442 | Week 390 | -1.3 mmol per mole | — |
| Group 2: Avalglucosidase Alfa 5 mg/kg | Change From Baseline in Urinary Glucose Tetrasaccharide (Hex4) Level Up to Week 442 | Week 416 | -4.7 mmol per mole | Standard Deviation 2.5 |
| Group 2: Avalglucosidase Alfa 10 mg/kg | Change From Baseline in Urinary Glucose Tetrasaccharide (Hex4) Level Up to Week 442 | Week 21 | -0.8 mmol per mole | Standard Deviation 1.7 |
| Group 2: Avalglucosidase Alfa 10 mg/kg | Change From Baseline in Urinary Glucose Tetrasaccharide (Hex4) Level Up to Week 442 | Week 416 | -2.9 mmol per mole | — |
| Group 2: Avalglucosidase Alfa 10 mg/kg | Change From Baseline in Urinary Glucose Tetrasaccharide (Hex4) Level Up to Week 442 | Week 286 | -2.0 mmol per mole | Standard Deviation 0.6 |
| Group 2: Avalglucosidase Alfa 10 mg/kg | Change From Baseline in Urinary Glucose Tetrasaccharide (Hex4) Level Up to Week 442 | Week 15 | -1.0 mmol per mole | Standard Deviation 1.3 |
| Group 2: Avalglucosidase Alfa 10 mg/kg | Change From Baseline in Urinary Glucose Tetrasaccharide (Hex4) Level Up to Week 442 | Week 9 | -0.7 mmol per mole | Standard Deviation 1.3 |
| Group 2: Avalglucosidase Alfa 10 mg/kg | Change From Baseline in Urinary Glucose Tetrasaccharide (Hex4) Level Up to Week 442 | Week 7 | -0.3 mmol per mole | Standard Deviation 1.1 |
| Group 2: Avalglucosidase Alfa 10 mg/kg | Change From Baseline in Urinary Glucose Tetrasaccharide (Hex4) Level Up to Week 442 | Week 19 | -0.8 mmol per mole | Standard Deviation 1.8 |
| Group 2: Avalglucosidase Alfa 10 mg/kg | Change From Baseline in Urinary Glucose Tetrasaccharide (Hex4) Level Up to Week 442 | Week 312 | -1.9 mmol per mole | Standard Deviation 0.7 |
| Group 2: Avalglucosidase Alfa 10 mg/kg | Change From Baseline in Urinary Glucose Tetrasaccharide (Hex4) Level Up to Week 442 | Week 442 | -1.9 mmol per mole | — |
| Group 2: Avalglucosidase Alfa 10 mg/kg | Change From Baseline in Urinary Glucose Tetrasaccharide (Hex4) Level Up to Week 442 | Week 234 | -2.9 mmol per mole | Standard Deviation 1.2 |
| Group 2: Avalglucosidase Alfa 10 mg/kg | Change From Baseline in Urinary Glucose Tetrasaccharide (Hex4) Level Up to Week 442 | Week 5 | -0.5 mmol per mole | Standard Deviation 1.1 |
| Group 2: Avalglucosidase Alfa 10 mg/kg | Change From Baseline in Urinary Glucose Tetrasaccharide (Hex4) Level Up to Week 442 | Week 17 | -0.9 mmol per mole | Standard Deviation 1.3 |
| Group 2: Avalglucosidase Alfa 10 mg/kg | Change From Baseline in Urinary Glucose Tetrasaccharide (Hex4) Level Up to Week 442 | Week 338 | -1.8 mmol per mole | Standard Deviation 0.4 |
| Group 2: Avalglucosidase Alfa 10 mg/kg | Change From Baseline in Urinary Glucose Tetrasaccharide (Hex4) Level Up to Week 442 | Week 13 | -0.7 mmol per mole | Standard Deviation 1.3 |
| Group 2: Avalglucosidase Alfa 10 mg/kg | Change From Baseline in Urinary Glucose Tetrasaccharide (Hex4) Level Up to Week 442 | Week 156 | -2.6 mmol per mole | Standard Deviation 1.8 |
| Group 2: Avalglucosidase Alfa 10 mg/kg | Change From Baseline in Urinary Glucose Tetrasaccharide (Hex4) Level Up to Week 442 | Week 78 | -1.5 mmol per mole | Standard Deviation 1.8 |
| Group 2: Avalglucosidase Alfa 10 mg/kg | Change From Baseline in Urinary Glucose Tetrasaccharide (Hex4) Level Up to Week 442 | Week 52 | -1.2 mmol per mole | Standard Deviation 2 |
| Group 2: Avalglucosidase Alfa 10 mg/kg | Change From Baseline in Urinary Glucose Tetrasaccharide (Hex4) Level Up to Week 442 | Week 11 | -0.5 mmol per mole | Standard Deviation 1.7 |
| Group 2: Avalglucosidase Alfa 10 mg/kg | Change From Baseline in Urinary Glucose Tetrasaccharide (Hex4) Level Up to Week 442 | Week 208 | -1.7 mmol per mole | Standard Deviation 0.6 |
| Group 2: Avalglucosidase Alfa 10 mg/kg | Change From Baseline in Urinary Glucose Tetrasaccharide (Hex4) Level Up to Week 442 | Week 104 | -1.9 mmol per mole | Standard Deviation 1.5 |
| Group 2: Avalglucosidase Alfa 10 mg/kg | Change From Baseline in Urinary Glucose Tetrasaccharide (Hex4) Level Up to Week 442 | Week 260 | -2.9 mmol per mole | Standard Deviation 1.7 |
| Group 2: Avalglucosidase Alfa 10 mg/kg | Change From Baseline in Urinary Glucose Tetrasaccharide (Hex4) Level Up to Week 442 | Week 25 | -0.8 mmol per mole | Standard Deviation 1.9 |
| Group 2: Avalglucosidase Alfa 10 mg/kg | Change From Baseline in Urinary Glucose Tetrasaccharide (Hex4) Level Up to Week 442 | Week 182 | -1.8 mmol per mole | Standard Deviation 0.4 |
| Group 2: Avalglucosidase Alfa 10 mg/kg | Change From Baseline in Urinary Glucose Tetrasaccharide (Hex4) Level Up to Week 442 | Week 23 | -0.9 mmol per mole | Standard Deviation 1.9 |
| Group 2: Avalglucosidase Alfa 10 mg/kg | Change From Baseline in Urinary Glucose Tetrasaccharide (Hex4) Level Up to Week 442 | Week 3 | -0.5 mmol per mole | Standard Deviation 1.1 |
| Group 2: Avalglucosidase Alfa 10 mg/kg | Change From Baseline in Urinary Glucose Tetrasaccharide (Hex4) Level Up to Week 442 | Week 130 | -1.9 mmol per mole | Standard Deviation 1.6 |
| Group 2: Avalglucosidase Alfa 20 mg/kg | Change From Baseline in Urinary Glucose Tetrasaccharide (Hex4) Level Up to Week 442 | Week 23 | -2.9 mmol per mole | Standard Deviation 5.5 |
| Group 2: Avalglucosidase Alfa 20 mg/kg | Change From Baseline in Urinary Glucose Tetrasaccharide (Hex4) Level Up to Week 442 | Week 260 | -5.6 mmol per mole | Standard Deviation 8.8 |
| Group 2: Avalglucosidase Alfa 20 mg/kg | Change From Baseline in Urinary Glucose Tetrasaccharide (Hex4) Level Up to Week 442 | Week 21 | -4.4 mmol per mole | Standard Deviation 5.6 |
| Group 2: Avalglucosidase Alfa 20 mg/kg | Change From Baseline in Urinary Glucose Tetrasaccharide (Hex4) Level Up to Week 442 | Week 19 | -4.0 mmol per mole | Standard Deviation 5.9 |
| Group 2: Avalglucosidase Alfa 20 mg/kg | Change From Baseline in Urinary Glucose Tetrasaccharide (Hex4) Level Up to Week 442 | Week 390 | -10.5 mmol per mole | Standard Deviation 11.7 |
| Group 2: Avalglucosidase Alfa 20 mg/kg | Change From Baseline in Urinary Glucose Tetrasaccharide (Hex4) Level Up to Week 442 | Week 182 | -3.9 mmol per mole | Standard Deviation 6.5 |
| Group 2: Avalglucosidase Alfa 20 mg/kg | Change From Baseline in Urinary Glucose Tetrasaccharide (Hex4) Level Up to Week 442 | Week 17 | -3.4 mmol per mole | Standard Deviation 4.5 |
| Group 2: Avalglucosidase Alfa 20 mg/kg | Change From Baseline in Urinary Glucose Tetrasaccharide (Hex4) Level Up to Week 442 | Week 156 | -4.8 mmol per mole | Standard Deviation 7.9 |
| Group 2: Avalglucosidase Alfa 20 mg/kg | Change From Baseline in Urinary Glucose Tetrasaccharide (Hex4) Level Up to Week 442 | Week 208 | -5.9 mmol per mole | Standard Deviation 9.5 |
| Group 2: Avalglucosidase Alfa 20 mg/kg | Change From Baseline in Urinary Glucose Tetrasaccharide (Hex4) Level Up to Week 442 | Week 15 | -3.4 mmol per mole | Standard Deviation 4.8 |
| Group 2: Avalglucosidase Alfa 20 mg/kg | Change From Baseline in Urinary Glucose Tetrasaccharide (Hex4) Level Up to Week 442 | Week 13 | -2.7 mmol per mole | Standard Deviation 3.6 |
| Group 2: Avalglucosidase Alfa 20 mg/kg | Change From Baseline in Urinary Glucose Tetrasaccharide (Hex4) Level Up to Week 442 | Week 78 | -3.5 mmol per mole | Standard Deviation 5.5 |
| Group 2: Avalglucosidase Alfa 20 mg/kg | Change From Baseline in Urinary Glucose Tetrasaccharide (Hex4) Level Up to Week 442 | Week 234 | -4.9 mmol per mole | Standard Deviation 7.1 |
| Group 2: Avalglucosidase Alfa 20 mg/kg | Change From Baseline in Urinary Glucose Tetrasaccharide (Hex4) Level Up to Week 442 | Week 11 | -2.5 mmol per mole | Standard Deviation 3.3 |
| Group 2: Avalglucosidase Alfa 20 mg/kg | Change From Baseline in Urinary Glucose Tetrasaccharide (Hex4) Level Up to Week 442 | Week 442 | -0.7 mmol per mole | — |
| Group 2: Avalglucosidase Alfa 20 mg/kg | Change From Baseline in Urinary Glucose Tetrasaccharide (Hex4) Level Up to Week 442 | Week 130 | -3.1 mmol per mole | Standard Deviation 3.9 |
| Group 2: Avalglucosidase Alfa 20 mg/kg | Change From Baseline in Urinary Glucose Tetrasaccharide (Hex4) Level Up to Week 442 | Week 9 | -2.2 mmol per mole | Standard Deviation 2.7 |
| Group 2: Avalglucosidase Alfa 20 mg/kg | Change From Baseline in Urinary Glucose Tetrasaccharide (Hex4) Level Up to Week 442 | Week 286 | -0.5 mmol per mole | — |
| Group 2: Avalglucosidase Alfa 20 mg/kg | Change From Baseline in Urinary Glucose Tetrasaccharide (Hex4) Level Up to Week 442 | Week 416 | -0.7 mmol per mole | — |
| Group 2: Avalglucosidase Alfa 20 mg/kg | Change From Baseline in Urinary Glucose Tetrasaccharide (Hex4) Level Up to Week 442 | Week 312 | -2.0 mmol per mole | Standard Deviation 0.2 |
| Group 2: Avalglucosidase Alfa 20 mg/kg | Change From Baseline in Urinary Glucose Tetrasaccharide (Hex4) Level Up to Week 442 | Week 5 | -1.5 mmol per mole | Standard Deviation 2 |
| Group 2: Avalglucosidase Alfa 20 mg/kg | Change From Baseline in Urinary Glucose Tetrasaccharide (Hex4) Level Up to Week 442 | Week 7 | -1.2 mmol per mole | Standard Deviation 2.3 |
| Group 2: Avalglucosidase Alfa 20 mg/kg | Change From Baseline in Urinary Glucose Tetrasaccharide (Hex4) Level Up to Week 442 | Week 52 | -3.4 mmol per mole | Standard Deviation 5.4 |
| Group 2: Avalglucosidase Alfa 20 mg/kg | Change From Baseline in Urinary Glucose Tetrasaccharide (Hex4) Level Up to Week 442 | Week 338 | -5.7 mmol per mole | Standard Deviation 8.2 |
| Group 2: Avalglucosidase Alfa 20 mg/kg | Change From Baseline in Urinary Glucose Tetrasaccharide (Hex4) Level Up to Week 442 | Week 25 | -3.1 mmol per mole | Standard Deviation 5 |
| Group 2: Avalglucosidase Alfa 20 mg/kg | Change From Baseline in Urinary Glucose Tetrasaccharide (Hex4) Level Up to Week 442 | Week 3 | -0.2 mmol per mole | Standard Deviation 1.5 |
| Group 2: Avalglucosidase Alfa 20 mg/kg | Change From Baseline in Urinary Glucose Tetrasaccharide (Hex4) Level Up to Week 442 | Week 104 | -3.6 mmol per mole | Standard Deviation 4.1 |