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NCT00158600 · ClinicalTrials.gov registry record · Phase 3
A Placebo-Controlled Study of Safety and Effectiveness of Myozyme (Alglucosidase Alfa) in Patients With Late-Onset Pompe Disease
A Phase 3 study of Pompe Disease (Late-onset) and Glycogen Storage Disease Type II (GSD-II), sponsored by Genzyme, a Sanofi Company.
- Completed
- Registry status
- Phase 3
- Development phase
- 90
- Enrollment target
- 8
- Study locations
NCT00158600: Completed Phase 3 study of Pompe Disease (Late-onset) and Glycogen Storage Disease Type II (GSD-II), sponsored by Genzyme, a Sanofi Company.
NCT00158600 is a Phase 3 study of Pompe Disease (Late-onset) and Glycogen Storage Disease Type II (GSD-II) that has completed, run by Genzyme, a Sanofi Company. The registered enrollment target is 90 participants, above the 36-participant average among 8 other Pompe Disease (Late-onset) trials with a reported enrollment target (150% higher). The trial reports 8 study locations across 6 states. According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT00158600, a Phase 3 study of Pompe Disease (Late-onset) and Glycogen Storage Disease Type II (GSD-II), has completed, sponsored by Genzyme, a Sanofi Company.
- COMPLETED
- Registry status
- Phase 3
- Development phase
- 90 participants
- Enrollment target
- 8
- Study locations
Study 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.
Primary Outcome
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.
Conditions Studied
Interventions
- DRUG Placebo
- BIOLOGICAL alglucosidase alfa
Study Locations (8)
Other
- Groupe Hospitalier Pitie-Salpetriere - Paris
- Sophia Children's Hospital, Erasmus MC - Rotterdam
- Erasmus Medical Centre Rotterdam - Rotterdam
California
- Tower Hematology Oncology Medical Group - Beverly Hills
District of Columbia
- Children's National Medical Center - Washington D.C.
Missouri
- Washington University Medical Center - St Louis
New York
- Mount Sinai School of Medicine - New York
Pennsylvania
- University of Pittsburgh, Dept. of Neurology - Pittsburgh
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 90 participants |
| Start Date | 2005-09 |
| Est. Completion | 2007-09 |
| Phase | Phase 3 |
What the finished NCT00158600 record still lists
NCT00158600 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 90 participants, a relatively small participant target, above the 36-participant average among 8 other Pompe Disease (Late-onset) trials with a reported enrollment target (150% higher).
The record links to 4 conditions, with Pompe Disease (Late-onset) appearing as the primary indexed condition, and to 2 interventions - of which Placebo is the first listed.
NCT00158600 lists 8 locations in 6 states (Other, California, District of Columbia).
Frequently Asked Questions
What is clinical trial NCT00158600 about?
NCT00158600 is a clinical study titled "A Placebo-Controlled Study of Safety and Effectiveness of Myozyme (Alglucosidase Alfa) in Patients With Late-Onset Pompe Disease". 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 ...
What is the current status of trial NCT00158600?
This trial is currently completed. It is a Phase 3 study. The enrollment target is 90 participants. The study started on 2005-09. Estimated completion is 2007-09.
What conditions does trial NCT00158600 study?
This clinical trial studies the following conditions: Pompe Disease (Late-onset), Glycogen Storage Disease Type II (GSD-II), Acid Maltase Deficiency Disease, Glycogenosis 2.
What interventions are being tested in trial NCT00158600?
The interventions under investigation include: Placebo (DRUG), alglucosidase alfa (BIOLOGICAL).
Who is sponsoring clinical trial NCT00158600?
This trial is sponsored by Genzyme, a Sanofi Company, which has 196 total clinical trials registered on ClinicalTrials.gov.
Where is trial NCT00158600 being conducted?
This trial has 8 study locations across California, District of Columbia, Missouri, New York, Pennsylvania. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
Learn More About Clinical Trials
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Where NCT00158600's enrollment target sits among peer trials
90 2nd of 8 the highest of 8 other Pompe Disease (Late-onset) trials
participants (enrollment target), bucketed by value
Each bar is a band; taller bars hold more other Pompe Disease (Late-onset) trials. The dashed line + filled bar mark this entry. Hover or tap any bar for its full count and share, and where it sits relative to this entry.
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