Trial · NCT00158600
A Placebo-Controlled Study of Safety and Effectiveness of Myozyme (Alglucosidase Alfa) in Patients With Late-Onset Pompe Disease
NCT00158600, Phase 3, Completed, Genzyme, a Sanofi Company; results first posted 2010-07-23
3 sourcestrials-2026-11-12-0.5.0
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ConductScience Library. A Placebo-Controlled Study of Safety and Effectiveness of Myozyme (Alglucosidase Alfa) in Patients With Late-Onset Pompe Disease. Trials, record NCT00158600. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT00158600
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- A Placebo-Controlled Study of Safety and Effectiveness of Myozyme (Alglucosidase Alfa) in Patients With Late-Onset Pompe Disease
- NCT number
- NCT00158600
- Status
- Completed
- Phases
- Phase 3
- Enrollment
- 90 participants, actual
- Lead sponsor
- Genzyme, a Sanofi Company
- Conditions
- Pompe Disease (Late-onset), Glycogen Storage Disease Type II (GSD-II), Acid Maltase Deficiency Disease, Glycogenosis 2
- Interventions
- alglucosidase alfa, Placebo
- First posted
- 2005-09-12
- Last updated
- 2015-04-28
- Other registries' IDs
- eudract:2005-002759-42
- Results first posted
- 2010-07-23
Source ClinicalTrials.gov
Registered outcomes8
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | 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. |
| Primary | 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). |
| Primary | 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%. |
| Primary | 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. |
| Primary | 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. |
| Primary | 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. |
| Secondary | 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. |
| 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 | 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. |
Source ClinicalTrials.gov
Outcomes8
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Summary of Patients Reporting Treatment-Emergent Adverse Events | weeks 0-78 | 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. | participants | NUMBER |
| PRIMARY | Mean Distance Walked as Measured by Six-minute Walk Test (6MWT) at Weeks 0 and 78, and Mean Change From Baseline | weeks 0, 78 | 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. | meters | MEAN |
| PRIMARY | Percent of Predicted Forced Vital Capacity (FVC) | weeks 0, 78 | ITT population. Last observation carried forward. | percent predicted FVC | MEAN |
| SECONDARY | Percent Predicted Proximal Muscle Strength of the Lower Limbs as Measured by Quantitative Muscle Testing (QMT) | weeks 0, 78 | ITT population. Last observation carried forward. | percent predicted QMT | MEAN |
| 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 | weeks 0, 78 | ITT population. Last observation carried forward. | Units on a scale | MEAN |
| PRIMARY | Recombinant Human Acid Alpha-Glucosidase (rhGAA) Pharmacokinetic Parameters: Area Under the Curve (AUC) | weeks 0, 12 and 52 | The subgroup of patients for whom pharmacokinetic samples were obtained was based on those study sites that could accommodate pharmacokinetic sampling needs. | ug*h/mL | MEAN |
| PRIMARY | Recombinant Human Acid Alpha-Glucosidase (rhGAA) Pharmacokinetic Parameters: Mean Maximum Plasma Concentration(Cmax) | weeks 0, 12, 52 | The subgroup of patients for whom pharmacokinetic samples were obtained was based on those study sites that could accommodate pharmacokinetic sampling needs. | ng/mL | MEAN |
| PRIMARY | Recombinant Human Acid Alpha-Glucosidase (rhGAA) Pharmacokinetic Parameters: Mean Time to Maximum Plasma Concentration(Tmax) | weeks 0, 12, 52 | The subgroup of patients for whom pharmacokinetic samples were obtained was based on those study sites that could accommodate pharmacokinetic sampling needs. | hours | MEAN |
Source ClinicalTrials.gov
Results by group48
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Summary of Patients Reporting Treatment-Emergent Adverse Events | Alglucosidase Alfa | Patients with Any AEs | 60 | participants | |
| Summary of Patients Reporting Treatment-Emergent Adverse Events | Placebo | Patients with Any AEs | 30 | participants | |
| Summary of Patients Reporting Treatment-Emergent Adverse Events | Alglucosidase Alfa | Patients with Treatment-Related AEs | 32 | participants | |
| Summary of Patients Reporting Treatment-Emergent Adverse Events | Placebo | Patients with Treatment-Related AEs | 17 | participants | |
| Summary of Patients Reporting Treatment-Emergent Adverse Events | Alglucosidase Alfa | Patients with Infusion-Associated Reactions | 17 | participants | |
| Summary of Patients Reporting Treatment-Emergent Adverse Events | Placebo | Patients with Infusion-Associated Reactions | 7 | participants | |
| Summary of Patients Reporting Treatment-Emergent Adverse Events | Alglucosidase Alfa | Patients with SAEs | 13 | participants | |
| Summary of Patients Reporting Treatment-Emergent Adverse Events | Placebo | Patients with SAEs | 6 | participants | |
| Summary of Patients Reporting Treatment-Emergent Adverse Events | Alglucosidase Alfa | Patients with Severe AEs | 14 | participants | |
| Summary of Patients Reporting Treatment-Emergent Adverse Events | Placebo | Patients with Severe AEs | 10 | participants |
1 to 10 of 48Source ClinicalTrials.gov
See all 48 results by groupParticipant flow6
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | Alglucosidase Alfa | 60 | |
| Overall Study | STARTED | Placebo | 30 | |
| Overall Study | COMPLETED | Alglucosidase Alfa | 55 | |
| Overall Study | COMPLETED | Placebo | 26 | |
| Overall Study | NOT COMPLETED | Alglucosidase Alfa | 5 | |
| Overall Study | NOT COMPLETED | Placebo | 4 |
Source ClinicalTrials.gov
Adverse events1,134
Each adverse event the trial posted, group by group: the participants affected out of those at risk.
| Type | Event | Organ system | Group | Affected | At risk | Events |
|---|---|---|---|---|---|---|
| other | Anaemia | Blood and lymphatic system disorders | Alglucosidase Alfa | 2 | 60 | |
| other | Anaemia | Blood and lymphatic system disorders | Placebo | 0 | 30 | |
| other | Anaemia | Blood and lymphatic system disorders | Overall | 2 | 90 | |
| other | Lymphadenopathy | Blood and lymphatic system disorders | Alglucosidase Alfa | 5 | 60 | |
| other | Lymphadenopathy | Blood and lymphatic system disorders | Placebo | 0 | 30 | |
| other | Lymphadenopathy | Blood and lymphatic system disorders | Overall | 5 | 90 | |
| other | Macrocytosis | Blood and lymphatic system disorders | Alglucosidase Alfa | 0 | 60 | |
| other | Macrocytosis | Blood and lymphatic system disorders | Placebo | 1 | 30 | |
| other | Macrocytosis | Blood and lymphatic system disorders | Overall | 1 | 90 | |
| other | Normochromic normocytic anaemia | Blood and lymphatic system disorders | Alglucosidase Alfa | 1 | 60 |
1 to 10 of 1,134Source ClinicalTrials.gov
See all 1,134 adverse eventsAdverse event totals9
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Alglucosidase Alfa | ||
| Total, all-cause mortality | Placebo | ||
| Total, all-cause mortality | Overall | ||
| Total, other adverse events | Alglucosidase Alfa | 60 | 60 |
| Total, other adverse events | Placebo | 30 | 30 |
| Total, other adverse events | Overall | 90 | 90 |
| Total, serious adverse events | Alglucosidase Alfa | 13 | 60 |
| Total, serious adverse events | Placebo | 6 | 30 |
| Total, serious adverse events | Overall | 19 | 90 |
Source ClinicalTrials.gov
Papers2
The papers the trial lists on ClinicalTrials.gov.
Grants0
The grants NIH RePORTER links to the trial, newest funded first: NIH's, and some of AHRQ's, CDC's, FDA's and VA's. Each shows its title, principal investigators, organization and funder in its newest year.
None in this release.
Trial comparisons0
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
None in this release.
Tests0
The ConductCognition tests this trial's registered outcomes name, as ScienceGraph matched them in trial-tests-2026-11-12-0.2.1.
None in this release.
Data dates
The day each source was copied for this release.
Sources 3
- ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
- ClinicalTrials.govNo license stated
- NIH RePORTER's clinical studies and project filesPublic domain
Each source's terms
- ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
- ClinicalTrials.govnone stated
- NIH RePORTER's clinical studies and project filesPublic domain (NIH RePORTER)
ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.
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