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A Placebo-Controlled Study of Safety and Effectiveness of Myozyme (Alglucosidase Alfa) in Patients With Late-Onset Pompe Disease
NCT00158600 · View on ClinicalTrials.gov ↗
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 |
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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. It is categorized as Phase 3, which is the standard way researchers label where a study sits along the investigational pathway from early safety work through later efficacy and post-marketing evaluation. The registered enrollment target is 90 participants, a figure that helps gauge the scale of data the investigators plan to collect. The listed sponsor is Genzyme, a Sanofi Company, which has 52 total studies on file at ClinicalTrials.gov, and sponsors are the parties responsible for study design, oversight, and regulatory filings.
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. Interventions can include drugs, devices, procedures, behavioral programs, or observational arms, and each is tracked as a separate registry field so that downstream queries can filter accurately. When a trial lists multiple interventions, it usually reflects a multi-arm design or a comparison protocol rather than a single treatment being tested in isolation. The brief summary published in the registry is the clearest source of protocol intent and should be read before drawing conclusions from any sidebar tags.
Geographic footprint matters for practical reasons: NCT00158600 reports 8 study locations spanning 6 distinct geographic areas — top geographies include Other, California, District of Columbia. A larger site network tends to correlate with broader recruitment capacity, but it does not imply anything about study quality, and site-level enrollment status can diverge from the overall registry status shown above. Every data point on this page comes from the public ClinicalTrials.gov dataset and is reproduced here for reference only; it is not a medical recommendation, an endorsement of the sponsor, or an invitation to enroll. Verify current status, eligibility criteria, and contact details directly at ClinicalTrials.gov, and discuss any participation decision with your own healthcare provider.
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. These conditions were identified from the trial registry and reflect the primary focus areas of the research.
What interventions are being tested in trial NCT00158600?
The interventions under investigation include: Placebo (DRUG), alglucosidase alfa (BIOLOGICAL). Each intervention is being evaluated for safety and efficacy as part of this clinical study.
Who is sponsoring clinical trial NCT00158600?
This trial is sponsored by Genzyme, a Sanofi Company, which has 52 total clinical trials registered on ClinicalTrials.gov. The sponsor is responsible for the study's design, funding, and regulatory compliance.
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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