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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 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.
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.
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 |
Interested in This Trial?
Full Details on ClinicalTrials.gov ↗What the Registry Record Tells You About NCT00158600
The ClinicalTrials.gov registry entry for NCT00158600 describes a study currently listed as completed, categorized as Phase 3. The registered enrollment target is 90 participants, a figure that helps gauge the scale of data the investigators plan to collect, above the 36-participant average among 8 other Pompe Disease (Late-onset) trials with a reported enrollment target (150% higher). The listed sponsor is Genzyme, a Sanofi Company, which has 196 total studies on file at ClinicalTrials.gov.
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 reports 8 study locations spanning 6 distinct geographic areas - top geographies include 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.
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