Pompe Disease, Glycogen Storage Disease Type II (GSD II)
Conditions
Brief summary
* The primary objective of this study was to characterize the pharmacokinetics (PK) of alglucosidase alfa manufactured at the 4000 L scale in participants who had a confirmed diagnosis of Pompe disease. * A secondary objective of this study was to evaluate and explore the relationship between anti-recombinant human acid alpha-glucosidase antibody titers and the PK of alglucosidase alfa.
Detailed description
The total study duration per participant was 4 to 8 weeks that consisted of a screening period (from 2 days to 4 weeks), treatment visit (1 day), and a follow up call (greater than or equal to 30 days). Two participants enrolled prior to protocol amendment 2 (dated 17 December 2015), which changed the study to single-dose, and were treated for 26 weeks, with a 4-week follow up.
Interventions
BIOLOGICALalglucosidase alfa
Intravenous (IV) infusion of 20mg/kg body weight every other week (qow)
Sponsors
Genzyme, a Sanofi Company
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
OTHER
Masking
NONE
Eligibility
Sex/Gender
ALL
Healthy volunteers
No
Inclusion criteria
A participant was to meet all of the following criteria to be eligible for this study: * The participant and/or the participant's parent/legal guardian was willing and able to provide signed informed consent. * The participant had a confirmed acid alpha-glucosidase (GAA) enzyme deficiency from skin, blood, or muscle tissue and/or 2 confirmed GAA gene mutations. * Infant and toddler Pompe disease participants could be included in the study only under condition (minimal body weight) that the trial-related blood loss (including any losses in the maneuver) would not exceed 3 percent (%) of the total blood volume during a period of 4 weeks and would not exceed 1 % at any single time. * The participant, if female and of childbearing potential, must have had a negative pregnancy test (urine beta-human chorionic gonadotropin) at screening. Note: All female participants of childbearing potential and sexually mature males must have agreed to use a medically accepted method of contraception throughout the study. * For participants previously treated with alglucosidase alfa the participant had received alglucosidase alfa for at least 6 months.
Exclusion criteria
A participant who met any of the following criteria was excluded from this study: * The participant was participating in another clinical study using an investigational product. * The participant, in the opinion of the Investigator, was unable to adhere to the requirements of the study. 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 |
|---|---|---|
| Pharmacokinetics: Volume of Distribution at Steady State (Vss) of Alglucosidase Alfa | Pre-dose and at 1, 2, 4, 8, 12, and 24 hours Post-dose on Day 1 | Volume of distribution (Vd) is defined as the theoretical volume in which the total amount of drug would need to be uniformly distributed to produce the desired plasma concentration of a drug. Vss is the apparent volume of distribution at steady-state. |
| Pharmacokinetics (PK): Maximum Observed Plasma Concentration (Cmax) of Alglucosidase Alfa | Pre-dose and at 1, 2, 4, 8, 12, and 24 hours Post-dose on Day 1 | Cmax was defined as maximum observed plasma concentration. |
| Pharmacokinetics: Time to Reach Maximum Observed Plasma Concentration (Tmax) of Alglucosidase Alfa | Pre-dose and at 1, 2, 4, 8, 12, and 24 hours Post-dose on Day 1 | Tmax was defined as time to reach maximum observed plasma concentration. |
| Pharmacokinetics: Area Under the Plasma Concentration-Time Curve (AUC) of Alglucosidase Alfa | Pre-dose and at 1, 2, 4, 8, 12, and 24 hours Post-dose on Day 1 | AUC was defined as area under the plasma concentration-time curve from time 0 to 24 hours post-dose. |
| Pharmacokinetics: Area Under the Concentration-time Curve From Time 0 to the Time of the Last Quantifiable Concentration (AUC0-last) of Alglucosidase Alfa | Pre-dose and at 1, 2, 4, 8, 12, and 24 hours Post-dose on Day 1 | AUC0-last was defined as area under the concentration-time curve from time 0 to the time of the last quantifiable concentration. |
| Pharmacokinetics: Terminal Elimination Half-life (T1/2) of Alglucosidase Alfa | Pre-dose and at 1, 2, 4, 8, 12, and 24 hours Post-dose on Day 1 | T1/2 was defined as the time taken by drug to reduce to half of its initial plasma concentration. |
| Pharmacokinetics: Total Systemic Clearance (CL) of Alglucosidase Alfa | Pre-dose and at 1, 2, 4, 8, 12, and 24 hours Post-dose on Day 1 | CL of a drug is a measure of the rate at which a drug is metabolized or eliminated by normal biological processes. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Pharmacokinetics: Maximum Observed Plasma Concentration of Alglucosidase Alfa in Anti-Recombinant Human Acid Alpha-Glucosidase Antibody Positive and Negative Participants | Pre-dose and at 1, 2, 4, 8, 12, and 24 hours Post-dose on Day 1 | Cmax was defined as maximum observed plasma concentration. |
| Pharmacokinetics: Time to Reach Maximum Observed Plasma Concentration in Anti-rhGAA Antibody Positive and Negative Participants | Pre-dose and at 1, 2, 4, 8, 12, and 24 hours Post-dose on Day 1 | Tmax was defined as time to reach maximum observed plasma concentration. |
| Pharmacokinetics: Terminal Elimination Half-life of Alglucosidase Alfa in Anti-rhGAA Antibody Positive and Negative Participants | Pre-dose and at 1, 2, 4, 8, 12, and 24 hours Post-dose on Day 1 | T1/2 was defined as the time taken by drug to reduce to half of its initial plasma concentration. |
| Pharmacokinetics: Area Under the Concentration-time Curve From Time 0 to the Time of the Last Quantifiable Concentration of Alglucosidase Alfa in Anti-rhGAA Antibody Positive and Negative Participants | Pre-dose and at 1, 2, 4, 8, 12, and 24 hours Post-dose on Day 1 | AUC0-last was defined as area under the plasma concentration-time curve from time 0 to the time of the last quantifiable concentration. |
| Pharmacokinetics: Area Under the Concentration-time Curve From Time 0 and Extrapolated to Infinite Time (AUC0-inf) in Anti-rhGAA Antibody Positive and Negative Participants | Pre-dose and at 1, 2, 4, 8, 12, and 24 hours Post-dose on Day 1 | AUC0-inf was defined as area under the concentration-time curve from time 0 extrapolated to infinite time. |
| Pharmacokinetics: Total Systemic Clearance in Anti-rhGAA Antibody Positive and Negative Participants | Pre-dose and at 1, 2, 4, 8, 12, and 24 hours Post-dose on Day 1 | CL of a drug is a measure of the rate at which a drug is metabolized or eliminated by normal biological processes. |
| Pharmacokinetics: Volume of Distribution in Anti-rhGAA Antibody Positive and Negative Participants | Pre-dose and at 1, 2, 4, 8, 12, and 24 hours Post-dose on Day 1 | Vd is defined as the theoretical volume in which the total amount of drug would need to be uniformly distributed to produce the desired plasma concentration of a drug. Vss is the apparent volume of distribution at steady-state. |
Countries
Bulgaria, India, Russia, Ukraine, United Kingdom, United States
Participant flow
Recruitment details
The study was conducted at 12 sites in 6 countries. A total of 27 participants were screened between 03-Nov-2014 and 23-Sep-2020, of whom 6 participants were screen failures and 21 participants were enrolled in the study.
Pre-assignment details
Out of 21 participants, 1 participant signed the informed consent, but due to health status did not continue in the study to the treatment visit.
Participants by arm
| Arm | Count |
|---|---|
| Alglucosidase Alfa: <18 Years Participants with \<18 years of age received IV infusion of Alglucosidase alfa 20 mg/kg body weight on Day 1. Infusion was administered at an initial rate of approximately 1 mg/kg/hr with allowed rate increased of 2 mg/kg/hr every 30 minutes, if there were no signs of IARs, until a maximum rate of approximately 7 mg/kg/hr was reached. | 10 |
| Alglucosidase Alfa: >=18 Years Participants with \>=18 years of age received IV infusion of Alglucosidase alfa 20 mg/kg body weight on Day 1. Infusion was administered at an initial rate of approximately 1 mg/kg/hr with allowed rate increased of 2 mg/kg/hr every 30 minutes, if there were no signs of IARs, until a maximum rate of approximately 7 mg/kg/hr was reached. | 10 |
| Total | 20 |
Withdrawals & dropouts
| Period | Reason | FG000 | FG001 |
|---|---|---|---|
| Overall Study | Withdrawal by Subject | 0 | 1 |
Baseline characteristics
| Characteristic | Total | Alglucosidase Alfa: <18 Years | Alglucosidase Alfa: >=18 Years |
|---|---|---|---|
| Age, Continuous | 23.4 years STANDARD_DEVIATION 20.86 | 5.1 years STANDARD_DEVIATION 3.95 | 41.8 years STANDARD_DEVIATION 12.44 |
| Anti-Recombinant Human Acid Alpha-Glucosidase (rhGAA) Immunoglobulin G (IgG) Antibody Status Missing | 1 Participants | 1 Participants | 0 Participants |
| Anti-Recombinant Human Acid Alpha-Glucosidase (rhGAA) Immunoglobulin G (IgG) Antibody Status Negative | 12 Participants | 6 Participants | 6 Participants |
| Anti-Recombinant Human Acid Alpha-Glucosidase (rhGAA) Immunoglobulin G (IgG) Antibody Status Positive | 7 Participants | 3 Participants | 4 Participants |
| Ethnicity (NIH/OMB) Hispanic or Latino | 2 Participants | 1 Participants | 1 Participants |
| Ethnicity (NIH/OMB) Not Hispanic or Latino | 14 Participants | 6 Participants | 8 Participants |
| Ethnicity (NIH/OMB) Unknown or Not Reported | 4 Participants | 3 Participants | 1 Participants |
| Race (NIH/OMB) American Indian or Alaska Native | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) Asian | 4 Participants | 3 Participants | 1 Participants |
| Race (NIH/OMB) Black or African American | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) More than one race | 2 Participants | 2 Participants | 0 Participants |
| Race (NIH/OMB) Native Hawaiian or Other Pacific Islander | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) Unknown or Not Reported | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) White | 14 Participants | 5 Participants | 9 Participants |
| Sex: Female, Male Female | 7 Participants | 5 Participants | 2 Participants |
| Sex: Female, Male Male | 13 Participants | 5 Participants | 8 Participants |
Adverse events
| Event type | EG000 affected / at risk | EG001 affected / at risk |
|---|---|---|
| deaths Total, all-cause mortality | 0 / 10 | 0 / 10 |
| other Total, other adverse events | 4 / 10 | 3 / 10 |
| serious Total, serious adverse events | 1 / 10 | 0 / 10 |
Outcome results
Primary
Pharmacokinetics: Area Under the Concentration-time Curve From Time 0 to the Time of the Last Quantifiable Concentration (AUC0-last) of Alglucosidase Alfa
AUC0-last was defined as area under the concentration-time curve from time 0 to the time of the last quantifiable concentration.
Time frame: Pre-dose and at 1, 2, 4, 8, 12, and 24 hours Post-dose on Day 1
Population: Analysis was performed on all participants who received any amount of alglucosidase alfa.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Alglucosidase Alfa: <18 Years | Pharmacokinetics: Area Under the Concentration-time Curve From Time 0 to the Time of the Last Quantifiable Concentration (AUC0-last) of Alglucosidase Alfa | 1040000 hours per nanograms per milliliter | Standard Deviation 590000 |
| Alglucosidase Alfa: >=18 Years | Pharmacokinetics: Area Under the Concentration-time Curve From Time 0 to the Time of the Last Quantifiable Concentration (AUC0-last) of Alglucosidase Alfa | 1840000 hours per nanograms per milliliter | Standard Deviation 901000 |
Primary
Pharmacokinetics: Area Under the Plasma Concentration-Time Curve (AUC) of Alglucosidase Alfa
AUC was defined as area under the plasma concentration-time curve from time 0 to 24 hours post-dose.
Time frame: Pre-dose and at 1, 2, 4, 8, 12, and 24 hours Post-dose on Day 1
Population: Analysis was performed on all participants who received any amount of alglucosidase alfa.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Alglucosidase Alfa: <18 Years | Pharmacokinetics: Area Under the Plasma Concentration-Time Curve (AUC) of Alglucosidase Alfa | 1110000 hours per nanograms per milliliter | Standard Deviation 753000 |
| Alglucosidase Alfa: >=18 Years | Pharmacokinetics: Area Under the Plasma Concentration-Time Curve (AUC) of Alglucosidase Alfa | 1890000 hours per nanograms per milliliter | Standard Deviation 969000 |
Primary
Pharmacokinetics (PK): Maximum Observed Plasma Concentration (Cmax) of Alglucosidase Alfa
Cmax was defined as maximum observed plasma concentration.
Time frame: Pre-dose and at 1, 2, 4, 8, 12, and 24 hours Post-dose on Day 1
Population: Analysis was performed on all participants who received any amount of alglucosidase alfa.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Alglucosidase Alfa: <18 Years | Pharmacokinetics (PK): Maximum Observed Plasma Concentration (Cmax) of Alglucosidase Alfa | 204000 nanograms per milliliter (ng/mL) | Standard Deviation 94600 |
| Alglucosidase Alfa: >=18 Years | Pharmacokinetics (PK): Maximum Observed Plasma Concentration (Cmax) of Alglucosidase Alfa | 307000 nanograms per milliliter (ng/mL) | Standard Deviation 143000 |
Primary
Pharmacokinetics: Terminal Elimination Half-life (T1/2) of Alglucosidase Alfa
T1/2 was defined as the time taken by drug to reduce to half of its initial plasma concentration.
Time frame: Pre-dose and at 1, 2, 4, 8, 12, and 24 hours Post-dose on Day 1
Population: Analysis was performed on all participants who received any amount of alglucosidase alfa.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Alglucosidase Alfa: <18 Years | Pharmacokinetics: Terminal Elimination Half-life (T1/2) of Alglucosidase Alfa | 5.43 hours | Standard Deviation 3.82 |
| Alglucosidase Alfa: >=18 Years | Pharmacokinetics: Terminal Elimination Half-life (T1/2) of Alglucosidase Alfa | 3.84 hours | Standard Deviation 0.801 |
Primary
Pharmacokinetics: Time to Reach Maximum Observed Plasma Concentration (Tmax) of Alglucosidase Alfa
Tmax was defined as time to reach maximum observed plasma concentration.
Time frame: Pre-dose and at 1, 2, 4, 8, 12, and 24 hours Post-dose on Day 1
Population: Analysis was performed on all participants who received any amount of alglucosidase alfa.
| Arm | Measure | Value (MEDIAN) |
|---|---|---|
| Alglucosidase Alfa: <18 Years | Pharmacokinetics: Time to Reach Maximum Observed Plasma Concentration (Tmax) of Alglucosidase Alfa | 4.23 hours |
| Alglucosidase Alfa: >=18 Years | Pharmacokinetics: Time to Reach Maximum Observed Plasma Concentration (Tmax) of Alglucosidase Alfa | 3.85 hours |
Primary
Pharmacokinetics: Total Systemic Clearance (CL) of Alglucosidase Alfa
CL of a drug is a measure of the rate at which a drug is metabolized or eliminated by normal biological processes.
Time frame: Pre-dose and at 1, 2, 4, 8, 12, and 24 hours Post-dose on Day 1
Population: Analysis was performed on all participants who received any amount of alglucosidase alfa.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Alglucosidase Alfa: <18 Years | Pharmacokinetics: Total Systemic Clearance (CL) of Alglucosidase Alfa | 0.529 Liters per hour | Standard Deviation 0.613 |
| Alglucosidase Alfa: >=18 Years | Pharmacokinetics: Total Systemic Clearance (CL) of Alglucosidase Alfa | 1.26 Liters per hour | Standard Deviation 1.24 |
Primary
Pharmacokinetics: Volume of Distribution at Steady State (Vss) of Alglucosidase Alfa
Volume of distribution (Vd) is defined as the theoretical volume in which the total amount of drug would need to be uniformly distributed to produce the desired plasma concentration of a drug. Vss is the apparent volume of distribution at steady-state.
Time frame: Pre-dose and at 1, 2, 4, 8, 12, and 24 hours Post-dose on Day 1
Population: Analysis was performed on all participants who received any amount of alglucosidase alfa.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Alglucosidase Alfa: <18 Years | Pharmacokinetics: Volume of Distribution at Steady State (Vss) of Alglucosidase Alfa | 2.35 Liters | Standard Deviation 2.02 |
| Alglucosidase Alfa: >=18 Years | Pharmacokinetics: Volume of Distribution at Steady State (Vss) of Alglucosidase Alfa | 5.59 Liters | Standard Deviation 3.72 |
Secondary
Pharmacokinetics: Area Under the Concentration-time Curve From Time 0 and Extrapolated to Infinite Time (AUC0-inf) in Anti-rhGAA Antibody Positive and Negative Participants
AUC0-inf was defined as area under the concentration-time curve from time 0 extrapolated to infinite time.
Time frame: Pre-dose and at 1, 2, 4, 8, 12, and 24 hours Post-dose on Day 1
Population: Analysis was performed on FAS that included all participants who received any amount of alglucosidase alfa. Here, overall number of participants analyzed = participants with available data for this outcome measure.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Alglucosidase Alfa: <18 Years | Pharmacokinetics: Area Under the Concentration-time Curve From Time 0 and Extrapolated to Infinite Time (AUC0-inf) in Anti-rhGAA Antibody Positive and Negative Participants | 1450000 hours per nanograms per milliliter | Standard Deviation 941000 |
| Alglucosidase Alfa: >=18 Years | Pharmacokinetics: Area Under the Concentration-time Curve From Time 0 and Extrapolated to Infinite Time (AUC0-inf) in Anti-rhGAA Antibody Positive and Negative Participants | 1700000 hours per nanograms per milliliter | Standard Deviation 985000 |
Secondary
Pharmacokinetics: Area Under the Concentration-time Curve From Time 0 to the Time of the Last Quantifiable Concentration of Alglucosidase Alfa in Anti-rhGAA Antibody Positive and Negative Participants
AUC0-last was defined as area under the plasma concentration-time curve from time 0 to the time of the last quantifiable concentration.
Time frame: Pre-dose and at 1, 2, 4, 8, 12, and 24 hours Post-dose on Day 1
Population: Analysis was performed on FAS that included all participants who received any amount of alglucosidase alfa. Here, overall number of participants analyzed = participants with available data for this outcome measure.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Alglucosidase Alfa: <18 Years | Pharmacokinetics: Area Under the Concentration-time Curve From Time 0 to the Time of the Last Quantifiable Concentration of Alglucosidase Alfa in Anti-rhGAA Antibody Positive and Negative Participants | 1410000 hours per nanograms per milliliter | Standard Deviation 865000 |
| Alglucosidase Alfa: >=18 Years | Pharmacokinetics: Area Under the Concentration-time Curve From Time 0 to the Time of the Last Quantifiable Concentration of Alglucosidase Alfa in Anti-rhGAA Antibody Positive and Negative Participants | 1610000 hours per nanograms per milliliter | Standard Deviation 86800 |
Secondary
Pharmacokinetics: Maximum Observed Plasma Concentration of Alglucosidase Alfa in Anti-Recombinant Human Acid Alpha-Glucosidase Antibody Positive and Negative Participants
Cmax was defined as maximum observed plasma concentration.
Time frame: Pre-dose and at 1, 2, 4, 8, 12, and 24 hours Post-dose on Day 1
Population: Analysis was performed on FAS that included all participants who received any amount of alglucosidase alfa. Here, overall number of participants analyzed = participants with available data for this outcome measure.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Alglucosidase Alfa: <18 Years | Pharmacokinetics: Maximum Observed Plasma Concentration of Alglucosidase Alfa in Anti-Recombinant Human Acid Alpha-Glucosidase Antibody Positive and Negative Participants | 256000 ng/mL | Standard Deviation 121000 |
| Alglucosidase Alfa: >=18 Years | Pharmacokinetics: Maximum Observed Plasma Concentration of Alglucosidase Alfa in Anti-Recombinant Human Acid Alpha-Glucosidase Antibody Positive and Negative Participants | 262000 ng/mL | Standard Deviation 160000 |
Secondary
Pharmacokinetics: Terminal Elimination Half-life of Alglucosidase Alfa in Anti-rhGAA Antibody Positive and Negative Participants
T1/2 was defined as the time taken by drug to reduce to half of its initial plasma concentration.
Time frame: Pre-dose and at 1, 2, 4, 8, 12, and 24 hours Post-dose on Day 1
Population: Analysis was performed on FAS that included all participants who received any amount of alglucosidase alfa. Here, overall number of participants analyzed = participants with available data for this outcome measure.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Alglucosidase Alfa: <18 Years | Pharmacokinetics: Terminal Elimination Half-life of Alglucosidase Alfa in Anti-rhGAA Antibody Positive and Negative Participants | 4.68 hours | Standard Deviation 2.95 |
| Alglucosidase Alfa: >=18 Years | Pharmacokinetics: Terminal Elimination Half-life of Alglucosidase Alfa in Anti-rhGAA Antibody Positive and Negative Participants | 4.79 hours | Standard Deviation 2.93 |
Secondary
Pharmacokinetics: Time to Reach Maximum Observed Plasma Concentration in Anti-rhGAA Antibody Positive and Negative Participants
Tmax was defined as time to reach maximum observed plasma concentration.
Time frame: Pre-dose and at 1, 2, 4, 8, 12, and 24 hours Post-dose on Day 1
Population: Analysis was performed on FAS that included all participants who received any amount of alglucosidase alfa. Here, overall number of participants analyzed = participants with available data for this outcome measure.
| Arm | Measure | Value (MEDIAN) |
|---|---|---|
| Alglucosidase Alfa: <18 Years | Pharmacokinetics: Time to Reach Maximum Observed Plasma Concentration in Anti-rhGAA Antibody Positive and Negative Participants | 3.94 hours |
| Alglucosidase Alfa: >=18 Years | Pharmacokinetics: Time to Reach Maximum Observed Plasma Concentration in Anti-rhGAA Antibody Positive and Negative Participants | 4.33 hours |
Secondary
Pharmacokinetics: Total Systemic Clearance in Anti-rhGAA Antibody Positive and Negative Participants
CL of a drug is a measure of the rate at which a drug is metabolized or eliminated by normal biological processes.
Time frame: Pre-dose and at 1, 2, 4, 8, 12, and 24 hours Post-dose on Day 1
Population: Analysis was performed on FAS that included all participants who received any amount of alglucosidase alfa. Here, overall number of participants analyzed = participants with available data for this outcome measure.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Alglucosidase Alfa: <18 Years | Pharmacokinetics: Total Systemic Clearance in Anti-rhGAA Antibody Positive and Negative Participants | 0.765 Liters per hour | Standard Deviation 0.518 |
| Alglucosidase Alfa: >=18 Years | Pharmacokinetics: Total Systemic Clearance in Anti-rhGAA Antibody Positive and Negative Participants | 1.21 Liters per hour | Standard Deviation 1.6 |
Secondary
Pharmacokinetics: Volume of Distribution in Anti-rhGAA Antibody Positive and Negative Participants
Vd is defined as the theoretical volume in which the total amount of drug would need to be uniformly distributed to produce the desired plasma concentration of a drug. Vss is the apparent volume of distribution at steady-state.
Time frame: Pre-dose and at 1, 2, 4, 8, 12, and 24 hours Post-dose on Day 1
Population: Analysis was performed on FAS that included all participants who received any amount of alglucosidase alfa. Here, overall number of participants analyzed = participants with available data for this outcome measure.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Alglucosidase Alfa: <18 Years | Pharmacokinetics: Volume of Distribution in Anti-rhGAA Antibody Positive and Negative Participants | 5.82 Liters | Standard Deviation 6.49 |
| Alglucosidase Alfa: >=18 Years | Pharmacokinetics: Volume of Distribution in Anti-rhGAA Antibody Positive and Negative Participants | 7.26 Liters | Standard Deviation 9.16 |