Pompe Disease (Late-onset), Glycogen Storage Disease Type II (GSD-II), Acid Maltase Deficiency Disease, Glycogenosis 2
Conditions
Keywords
Glycogen Storage Disease Type II, GSD-II, Pompe Disease
Brief summary
Pompe disease (also known as glycogen storage disease Type II) is caused by a deficiency of a critical enzyme in the body called acid alpha-glucosidase (GAA). Normally, GAA is used by the body's cells to break down glycogen (a stored form of sugar) within specialized structures called lysosomes. In patients with Pompe disease, an excessive amount of glycogen accumulates and is stored in various tissues, especially heart and skeletal muscle, which prevents their normal function. The overall objective is to evaluate the safety, efficacy, and pharmacokinetics (PK) of alglucosidase alfa treatment in patients with late-onset Pompe disease as compared to placebo.
Interventions
BIOLOGICALalglucosidase alfa
IV infusion of 20mg/kg; qow for 78 weeks.
DRUGPlacebo
Placebo Comparator; qow for 78 weeks.
Sponsors
Genzyme, a Sanofi Company
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
QUADRUPLE (Subject, Caregiver, Investigator, Outcomes Assessor)
Eligibility
Sex/Gender
ALL
Age
8 Years to No maximum
Healthy volunteers
No
Inclusion criteria
* Patient must provide signed, informed consent prior to performing any study-related procedures. * Patient must have a diagnosis of Pompe disease based on deficient endogenous GAA activity in cultured skin fibroblasts of less than or equal to 40% of the normal mean of the testing laboratory and 2 confirmed GAA gene mutations; * Patient must be greater than or equal to 8 years of age at the time of enrollment; * Patient must be able to ambulate 40 meters (approximately 130 feet) in 6 minutes on each test performed on two consecutive days (use of assistive devices such as a walker, cane, or crutches, is permitted); * Patient must have an FVC of greater than or equal to 30% and \< 80% predicted in the upright position; * Patient must have a postural drop in FVC (liters) of at least 10% from the upright to the supine position; * Patient must have proximal muscle weakness in the lower limbs based on unilateral QMT of the knee extensors defined as \< 80% of the predicted value based on age, gender and body size * Patient must be able to tolerate pulmonary function testing (PFT) and muscle testing in the supine position; * Patient must have testable muscle in bilateral knee flexors and knee extensors, and testable muscle in bilateral elbow flexors and elbow extensors; * Patient must be able to provide reproducible muscle and pulmonary function test results; * Patient (and patient's legal guardian if patient is \< 18 years of age) must have the ability to comply with the clinical protocol; * A female patient of childbearing potential must have a negative pregnancy test (urine) at Baseline. Note: All female patients of childbearing potential and sexually mature males must use a medically accepted method of contraception throughout the study.
Exclusion criteria
* Patient requires the use of invasive ventilatory support; * Patient requires the use of noninvasive ventilatory support while awake and in an upright position; * Patient has received enzyme replacement therapy with GAA from any source; * Patient has used an investigational product within 30 days prior to study enrollment, or is currently enrolled in another study which involves clinical evaluations, unless prior approval is given by Genzyme; * Patient has a major congenital anomaly, medical condition, serious intercurrent illness, or other extenuating circumstance that, in the opinion of the investigator, may significantly interfere with study compliance, including all prescribed evaluations and follow-up activities;
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Recombinant Human Acid Alpha-Glucosidase (rhGAA) Pharmacokinetic Parameters: Mean Time to Maximum Plasma Concentration(Tmax) | weeks 0, 12, 52 | Time to maximum plasma concentration observed in blood samples taken at the following time points: 0 (before the start of the infusion), 1 and 2 hours after the start of infusion, end of the infusion, and then 0.25, 0.5, 1, 2, 3, 4, 8, 12,and 16 hours after the end of the infusion (with a 5-minute window for time-points after the start of infusion). Pooled figures combine the values for the three timeframes. |
| Recombinant Human Acid Alpha-Glucosidase (rhGAA) Pharmacokinetic Parameters: Area Under the Curve (AUC) | weeks 0, 12 and 52 | Area under the plasma concentration versus time curve from time zero (pre-dose) to 16 hours after the end of infusion. Blood sample time points were 0 (before the start of the infusion), 1 and 2 hours after the start of infusion, end of the infusion, and then 0.25, 0.5, 1, 2, 3, 4, 8, 12,and 16 hours after the end of the infusion (with a 5-minute window for time-points after the start of infusion). Pooled figures combine the values for the three timeframes. |
| Recombinant Human Acid Alpha-Glucosidase (rhGAA) Pharmacokinetic Parameters: Mean Maximum Plasma Concentration(Cmax) | weeks 0, 12, 52 | Maximum plasma concentration observed in blood samples taken at the following time points: 0 (before the start of the infusion), 1 and 2 hours after the start of infusion, end of the infusion, and then 0.25, 0.5, 1, 2, 3, 4, 8, 12,and 16 hours after the end of the infusion (with a 5-minute window for time-points after the start of infusion). Pooled figures combine the values for the three timeframes. |
| Summary of Patients Reporting Treatment-Emergent Adverse Events | weeks 0-78 | Overall safety summary of patients experiencing Adverse Events (AEs), Serious Adverse Events (SAEs), treatment-related AEs, and Infusion Associated Reactions (IARs). Summary is based on Treatment-emergent AEs (TEAEs), defined as AEs that occurred following the initiation of study treatment, i.e., alglucosidase alfa or placebo. |
| Mean Distance Walked as Measured by Six-minute Walk Test (6MWT) at Weeks 0 and 78, and Mean Change From Baseline | weeks 0, 78 | Mean distance walked gives an indication of functional endurance. The greater the distance, the greater the endurance. Mean values of distance walked in a six-minute walk test are offered for baseline, week 78 (or last available observation), and the mean change from baseline (at week 78 or last available post-baseline observation). |
| Percent of Predicted Forced Vital Capacity (FVC) | weeks 0, 78 | Forced vital capacity is a standard pulmonary function test used to quantify respiratory muscle weakness. Forced vital capacity (FVC) is the volume of air that can forcibly be blown out after full inspiration in the upright position, measured in liters. Predicted forced vital capacity is based on a formula using sex, age and height of a person, and is an estimate of healthy lung capacity. Percent of predicted FVC = (observed value)/(predicted value) \* 100%. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Health-related Quality of Life Survey Values Related to Physical Components as Measured by the Medical Outcomes Study (MOS) Short Form-36 Health Survey | weeks 0, 78 | The Medical Outcomes Study Short Form (MOS SF)-36 questionnaire consists of 36 items grouped into 8 domains designed to assess generic health-related quality of life in healthy and ill adult populations. Physical Component Scores (PCS) report the four domains of physical functioning, role-physical, bodily pain, and general health. Higher scores are associated with better quality of life. All questions are scored on a scale from 0 to 100, with 100 representing the highest level of functioning possible. The PCS scores are reported. |
| Percent Predicted Proximal Muscle Strength of the Lower Limbs as Measured by Quantitative Muscle Testing (QMT) | weeks 0, 78 | Quantitative muscle testing (QMT) is a standardized system to measure muscle force production during maximal voluntary isometric contraction. QMT data were collected directly from sensors into laptop computers. Predicted normal values for QMT are based on a formula using sex, age and body mass index of a person, and is an estimate of healthy muscle force. Percent of predicted QMT = (observed value)/(predicted value) \* 100%. The QMT Leg Score is the average of the bilateral means for percent predicted knee flexors and extensors. A value of 100% indicates 'normal' muscle strength. |
Countries
France, Netherlands, United States
Participant flow
Pre-assignment details
One hundred patients screened and 90 enrolled.
Participants by arm
| Arm | Count |
|---|---|
| Alglucosidase Alfa Intravenous (IV) infusions of alglucosidase alfa at 20 milligrams (mg)/kilogram (kg) of body weight every other week (qow) for 78 weeks. | 60 |
| Placebo Intravenous (IV) infusions of placebo every other week (qow) for 78 weeks. | 30 |
| Total | 90 |
Withdrawals & dropouts
| Period | Reason | FG000 | FG001 |
|---|---|---|---|
| Overall Study | Adverse Event | 2 | 1 |
| Overall Study | Death | 1 | 0 |
| Overall Study | unable to commit time to study | 0 | 1 |
| Overall Study | Withdrawal by Subject | 2 | 2 |
Baseline characteristics
| Characteristic | Alglucosidase Alfa | Placebo | Total |
|---|---|---|---|
| Age, Continuous | 45.3 years STANDARD_DEVIATION 12.37 | 42.6 years STANDARD_DEVIATION 11.63 | 44.4 years STANDARD_DEVIATION 12.14 |
| Race/Ethnicity, Customized Asian | 1 participants | 1 participants | 2 participants |
| Race/Ethnicity, Customized Black or African American | 0 participants | 0 participants | 0 participants |
| Race/Ethnicity, Customized Hispanic | 1 participants | 1 participants | 2 participants |
| Race/Ethnicity, Customized Unknown or not reported | 1 participants | 1 participants | 2 participants |
| Race/Ethnicity, Customized White | 57 participants | 27 participants | 84 participants |
| Sex: Female, Male Female | 26 Participants | 19 Participants | 45 Participants |
| Sex: Female, Male Male | 34 Participants | 11 Participants | 45 Participants |
Adverse events
| Event type | EG000 affected / at risk | EG001 affected / at risk | EG002 affected / at risk |
|---|---|---|---|
| deaths Total, all-cause mortality | — / — | — / — | — / — |
| other Total, other adverse events | 60 / 60 | 30 / 30 | 90 / 90 |
| serious Total, serious adverse events | 13 / 60 | 6 / 30 | 19 / 90 |
Outcome results
Primary
Mean Distance Walked as Measured by Six-minute Walk Test (6MWT) at Weeks 0 and 78, and Mean Change From Baseline
Mean distance walked gives an indication of functional endurance. The greater the distance, the greater the endurance. Mean values of distance walked in a six-minute walk test are offered for baseline, week 78 (or last available observation), and the mean change from baseline (at week 78 or last available post-baseline observation).
Time frame: weeks 0, 78
Population: Intent-to-Treat (ITT) population. Last observation carried forward. The last available distance walked for one patient was the Baseline visit; therefore, this patient was excluded from the change from baseline calculation.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Alglucosidase Alfa | Mean Distance Walked as Measured by Six-minute Walk Test (6MWT) at Weeks 0 and 78, and Mean Change From Baseline | Distance Walked at Baseline | 332.20 meters | Standard Deviation 126.69 |
| Alglucosidase Alfa | Mean Distance Walked as Measured by Six-minute Walk Test (6MWT) at Weeks 0 and 78, and Mean Change From Baseline | Distance Walked at Last Available Observation | 357.85 meters | Standard Deviation 141.32 |
| Alglucosidase Alfa | Mean Distance Walked as Measured by Six-minute Walk Test (6MWT) at Weeks 0 and 78, and Mean Change From Baseline | Change at Last Available Observation from Baseline | 26.08 meters | Standard Deviation 64.41 |
| Placebo | Mean Distance Walked as Measured by Six-minute Walk Test (6MWT) at Weeks 0 and 78, and Mean Change From Baseline | Distance Walked at Baseline | 317.93 meters | Standard Deviation 132.29 |
| Placebo | Mean Distance Walked as Measured by Six-minute Walk Test (6MWT) at Weeks 0 and 78, and Mean Change From Baseline | Distance Walked at Last Available Observation | 313.07 meters | Standard Deviation 144.69 |
| Placebo | Mean Distance Walked as Measured by Six-minute Walk Test (6MWT) at Weeks 0 and 78, and Mean Change From Baseline | Change at Last Available Observation from Baseline | -4.87 meters | Standard Deviation 45.24 |
Comparison: The difference between alglucosidase alfa and placebo treatment groups in change in distance walked from baseline to last observation was estimated by ANCOVA after adjusting for baseline value and randomization strata.p-value: 0.034795% CI: [2.07, 54.17]ANCOVA
Primary
Percent of Predicted Forced Vital Capacity (FVC)
Forced vital capacity is a standard pulmonary function test used to quantify respiratory muscle weakness. Forced vital capacity (FVC) is the volume of air that can forcibly be blown out after full inspiration in the upright position, measured in liters. Predicted forced vital capacity is based on a formula using sex, age and height of a person, and is an estimate of healthy lung capacity. Percent of predicted FVC = (observed value)/(predicted value) \* 100%.
Time frame: weeks 0, 78
Population: ITT population. Last observation carried forward.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Alglucosidase Alfa | Percent of Predicted Forced Vital Capacity (FVC) | Change at Week 78 from Baseline | 1.25 percent predicted FVC | Standard Deviation 5.55 |
| Alglucosidase Alfa | Percent of Predicted Forced Vital Capacity (FVC) | Week 78 (or last observation) | 56.71 percent predicted FVC | Standard Deviation 16.3 |
| Alglucosidase Alfa | Percent of Predicted Forced Vital Capacity (FVC) | Baseline (week 0) | 55.43 percent predicted FVC | Standard Deviation 14.44 |
| Placebo | Percent of Predicted Forced Vital Capacity (FVC) | Change at Week 78 from Baseline | -2.30 percent predicted FVC | Standard Deviation 4.33 |
| Placebo | Percent of Predicted Forced Vital Capacity (FVC) | Baseline (week 0) | 53.00 percent predicted FVC | Standard Deviation 15.66 |
| Placebo | Percent of Predicted Forced Vital Capacity (FVC) | Week 78 (or last observation) | 50.70 percent predicted FVC | Standard Deviation 14.88 |
Comparison: The difference between alglucosidase alfa and placebo treatment groups in change in % predicted FVC from baseline to last observation was estimated by ANCOVA after adjusting for baseline value and randomization strata.p-value: 0.005595% CI: [1.03, 5.77]ANCOVA
Primary
Recombinant Human Acid Alpha-Glucosidase (rhGAA) Pharmacokinetic Parameters: Area Under the Curve (AUC)
Area under the plasma concentration versus time curve from time zero (pre-dose) to 16 hours after the end of infusion. Blood sample time points were 0 (before the start of the infusion), 1 and 2 hours after the start of infusion, end of the infusion, and then 0.25, 0.5, 1, 2, 3, 4, 8, 12,and 16 hours after the end of the infusion (with a 5-minute window for time-points after the start of infusion). Pooled figures combine the values for the three timeframes.
Time frame: weeks 0, 12 and 52
Population: The subgroup of patients for whom pharmacokinetic samples were obtained was based on those study sites that could accommodate pharmacokinetic sampling needs.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Alglucosidase Alfa | Recombinant Human Acid Alpha-Glucosidase (rhGAA) Pharmacokinetic Parameters: Area Under the Curve (AUC) | Week 0 | 2672.47 ug*h/mL | Standard Deviation 1139.85 |
| Alglucosidase Alfa | Recombinant Human Acid Alpha-Glucosidase (rhGAA) Pharmacokinetic Parameters: Area Under the Curve (AUC) | Week 12 | 2386.76 ug*h/mL | Standard Deviation 555.09 |
| Alglucosidase Alfa | Recombinant Human Acid Alpha-Glucosidase (rhGAA) Pharmacokinetic Parameters: Area Under the Curve (AUC) | Week 52 | 2699.28 ug*h/mL | Standard Deviation 999.97 |
| Alglucosidase Alfa | Recombinant Human Acid Alpha-Glucosidase (rhGAA) Pharmacokinetic Parameters: Area Under the Curve (AUC) | Pooled | 2586.17 ug*h/mL | Standard Deviation 933.28 |
Primary
Recombinant Human Acid Alpha-Glucosidase (rhGAA) Pharmacokinetic Parameters: Mean Maximum Plasma Concentration(Cmax)
Maximum plasma concentration observed in blood samples taken at the following time points: 0 (before the start of the infusion), 1 and 2 hours after the start of infusion, end of the infusion, and then 0.25, 0.5, 1, 2, 3, 4, 8, 12,and 16 hours after the end of the infusion (with a 5-minute window for time-points after the start of infusion). Pooled figures combine the values for the three timeframes.
Time frame: weeks 0, 12, 52
Population: The subgroup of patients for whom pharmacokinetic samples were obtained was based on those study sites that could accommodate pharmacokinetic sampling needs.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Alglucosidase Alfa | Recombinant Human Acid Alpha-Glucosidase (rhGAA) Pharmacokinetic Parameters: Mean Maximum Plasma Concentration(Cmax) | Week 12 | 349269 ng/mL | Standard Deviation 78620 |
| Alglucosidase Alfa | Recombinant Human Acid Alpha-Glucosidase (rhGAA) Pharmacokinetic Parameters: Mean Maximum Plasma Concentration(Cmax) | Week 0 | 385237 ng/mL | Standard Deviation 105585 |
| Alglucosidase Alfa | Recombinant Human Acid Alpha-Glucosidase (rhGAA) Pharmacokinetic Parameters: Mean Maximum Plasma Concentration(Cmax) | Week 52 | 369744 ng/mL | Standard Deviation 88203 |
| Alglucosidase Alfa | Recombinant Human Acid Alpha-Glucosidase (rhGAA) Pharmacokinetic Parameters: Mean Maximum Plasma Concentration(Cmax) | Pooled | 368083 ng/mL | Standard Deviation 91721 |
Primary
Recombinant Human Acid Alpha-Glucosidase (rhGAA) Pharmacokinetic Parameters: Mean Time to Maximum Plasma Concentration(Tmax)
Time to maximum plasma concentration observed in blood samples taken at the following time points: 0 (before the start of the infusion), 1 and 2 hours after the start of infusion, end of the infusion, and then 0.25, 0.5, 1, 2, 3, 4, 8, 12,and 16 hours after the end of the infusion (with a 5-minute window for time-points after the start of infusion). Pooled figures combine the values for the three timeframes.
Time frame: weeks 0, 12, 52
Population: The subgroup of patients for whom pharmacokinetic samples were obtained was based on those study sites that could accommodate pharmacokinetic sampling needs.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Alglucosidase Alfa | Recombinant Human Acid Alpha-Glucosidase (rhGAA) Pharmacokinetic Parameters: Mean Time to Maximum Plasma Concentration(Tmax) | Week 0 | 3.62 hours | Standard Deviation 0.33 |
| Alglucosidase Alfa | Recombinant Human Acid Alpha-Glucosidase (rhGAA) Pharmacokinetic Parameters: Mean Time to Maximum Plasma Concentration(Tmax) | Week 12 | 3.62 hours | Standard Deviation 0.28 |
| Alglucosidase Alfa | Recombinant Human Acid Alpha-Glucosidase (rhGAA) Pharmacokinetic Parameters: Mean Time to Maximum Plasma Concentration(Tmax) | Week 52 | 3.64 hours | Standard Deviation 0.31 |
| Alglucosidase Alfa | Recombinant Human Acid Alpha-Glucosidase (rhGAA) Pharmacokinetic Parameters: Mean Time to Maximum Plasma Concentration(Tmax) | Pooled | 3.63 hours | Standard Deviation 0.3 |
Primary
Summary of Patients Reporting Treatment-Emergent Adverse Events
Overall safety summary of patients experiencing Adverse Events (AEs), Serious Adverse Events (SAEs), treatment-related AEs, and Infusion Associated Reactions (IARs). Summary is based on Treatment-emergent AEs (TEAEs), defined as AEs that occurred following the initiation of study treatment, i.e., alglucosidase alfa or placebo.
Time frame: weeks 0-78
Population: All patients who received any amount of study treatment comprise the safety population. Patients were considered, for safety analysis, to be in the treatment group of the treatment they actually received.~Missing or invalid safety or resource utilization data were not replaced.
| Arm | Measure | Group | Value (NUMBER) |
|---|---|---|---|
| Alglucosidase Alfa | Summary of Patients Reporting Treatment-Emergent Adverse Events | Patients with Infusion-Associated Reactions | 17 participants |
| Alglucosidase Alfa | Summary of Patients Reporting Treatment-Emergent Adverse Events | Patients with Any AEs | 60 participants |
| Alglucosidase Alfa | Summary of Patients Reporting Treatment-Emergent Adverse Events | Patients with SAEs | 13 participants |
| Alglucosidase Alfa | Summary of Patients Reporting Treatment-Emergent Adverse Events | Patients with Severe AEs | 14 participants |
| Alglucosidase Alfa | Summary of Patients Reporting Treatment-Emergent Adverse Events | Patients who Discontinued Due to AEs (incl death) | 3 participants |
| Alglucosidase Alfa | Summary of Patients Reporting Treatment-Emergent Adverse Events | Patients with Treatment-Related AEs | 32 participants |
| Alglucosidase Alfa | Summary of Patients Reporting Treatment-Emergent Adverse Events | Patients who Died | 1 participants |
| Placebo | Summary of Patients Reporting Treatment-Emergent Adverse Events | Patients who Died | 0 participants |
| Placebo | Summary of Patients Reporting Treatment-Emergent Adverse Events | Patients with Any AEs | 30 participants |
| Placebo | Summary of Patients Reporting Treatment-Emergent Adverse Events | Patients with Treatment-Related AEs | 17 participants |
| Placebo | Summary of Patients Reporting Treatment-Emergent Adverse Events | Patients with Infusion-Associated Reactions | 7 participants |
| Placebo | Summary of Patients Reporting Treatment-Emergent Adverse Events | Patients with Severe AEs | 10 participants |
| Placebo | Summary of Patients Reporting Treatment-Emergent Adverse Events | Patients who Discontinued Due to AEs (incl death) | 1 participants |
| Placebo | Summary of Patients Reporting Treatment-Emergent Adverse Events | Patients with SAEs | 6 participants |
Secondary
Health-related Quality of Life Survey Values Related to Physical Components as Measured by the Medical Outcomes Study (MOS) Short Form-36 Health Survey
The Medical Outcomes Study Short Form (MOS SF)-36 questionnaire consists of 36 items grouped into 8 domains designed to assess generic health-related quality of life in healthy and ill adult populations. Physical Component Scores (PCS) report the four domains of physical functioning, role-physical, bodily pain, and general health. Higher scores are associated with better quality of life. All questions are scored on a scale from 0 to 100, with 100 representing the highest level of functioning possible. The PCS scores are reported.
Time frame: weeks 0, 78
Population: ITT population. Last observation carried forward.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Alglucosidase Alfa | Health-related Quality of Life Survey Values Related to Physical Components as Measured by the Medical Outcomes Study (MOS) Short Form-36 Health Survey | PCS at Baseline (week 0) | 34.33 Units on a scale | Standard Deviation 8.93 |
| Alglucosidase Alfa | Health-related Quality of Life Survey Values Related to Physical Components as Measured by the Medical Outcomes Study (MOS) Short Form-36 Health Survey | PCS at Week 78 (or last available observation) | 35.11 Units on a scale | Standard Deviation 9.84 |
| Placebo | Health-related Quality of Life Survey Values Related to Physical Components as Measured by the Medical Outcomes Study (MOS) Short Form-36 Health Survey | PCS at Baseline (week 0) | 34.91 Units on a scale | Standard Deviation 7.26 |
| Placebo | Health-related Quality of Life Survey Values Related to Physical Components as Measured by the Medical Outcomes Study (MOS) Short Form-36 Health Survey | PCS at Week 78 (or last available observation) | 36.47 Units on a scale | Standard Deviation 9.57 |
Comparison: The difference between alglucosidase alfa and placebo treatment groups in change in PCS from baseline to last observation was estimated by ANCOVA after adjusting for baseline value and randomization strata.p-value: 0.833395% CI: [-3.83, 3.09]ANCOVA
Secondary
Percent Predicted Proximal Muscle Strength of the Lower Limbs as Measured by Quantitative Muscle Testing (QMT)
Quantitative muscle testing (QMT) is a standardized system to measure muscle force production during maximal voluntary isometric contraction. QMT data were collected directly from sensors into laptop computers. Predicted normal values for QMT are based on a formula using sex, age and body mass index of a person, and is an estimate of healthy muscle force. Percent of predicted QMT = (observed value)/(predicted value) \* 100%. The QMT Leg Score is the average of the bilateral means for percent predicted knee flexors and extensors. A value of 100% indicates 'normal' muscle strength.
Time frame: weeks 0, 78
Population: ITT population. Last observation carried forward.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Alglucosidase Alfa | Percent Predicted Proximal Muscle Strength of the Lower Limbs as Measured by Quantitative Muscle Testing (QMT) | Baseline (week 0) | 37.69 percent predicted QMT | Standard Deviation 18.88 |
| Alglucosidase Alfa | Percent Predicted Proximal Muscle Strength of the Lower Limbs as Measured by Quantitative Muscle Testing (QMT) | Week 78 (or last available observation) | 39.05 percent predicted QMT | Standard Deviation 21.83 |
| Alglucosidase Alfa | Percent Predicted Proximal Muscle Strength of the Lower Limbs as Measured by Quantitative Muscle Testing (QMT) | Change at Week 78 from Baseline | 1.22 percent predicted QMT | Standard Deviation 9.88 |
| Placebo | Percent Predicted Proximal Muscle Strength of the Lower Limbs as Measured by Quantitative Muscle Testing (QMT) | Baseline (week 0) | 32.49 percent predicted QMT | Standard Deviation 18.24 |
| Placebo | Percent Predicted Proximal Muscle Strength of the Lower Limbs as Measured by Quantitative Muscle Testing (QMT) | Week 78 (or last available observation) | 30.40 percent predicted QMT | Standard Deviation 20.54 |
| Placebo | Percent Predicted Proximal Muscle Strength of the Lower Limbs as Measured by Quantitative Muscle Testing (QMT) | Change at Week 78 from Baseline | -2.08 percent predicted QMT | Standard Deviation 5.11 |
Comparison: The difference between alglucosidase alfa and placebo treatment groups in change in QMT from baseline to last observation was estimated by ANCOVA after adjusting for baseline value and randomization strata.p-value: 0.109395% CI: [-0.73, 7.08]ANCOVA