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NCT00455195 · ClinicalTrials.gov registry record · Phase 4
Late-Onset Treatment Study Extension Protocol
A Phase 4 study of Pompe Disease (Late-onset) and Glycogen Storage Disease Type II (GSD-II), sponsored by Genzyme, a Sanofi Company.
- Completed
- Registry status
- Phase 4
- Development phase
- 81
- Enrollment target
- 20
- Study locations
NCT00455195: Completed Phase 4 study of Pompe Disease (Late-onset) and Glycogen Storage Disease Type II (GSD-II), sponsored by Genzyme, a Sanofi Company.
NCT00455195 is a Phase 4 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 81 participants, above the 37-participant average among 8 other Pompe Disease (Late-onset) trials with a reported enrollment target (119% higher). The trial reports 20 study locations across 16 states. According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT00455195, a Phase 4 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 4
- Development phase
- 81 participants
- Enrollment target
- 20
- 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 objective of this extension study is to assess the long-term safety and efficacy of alglucosidase alfa treatment in patients with Late-Onset Pompe Disease who were previously treated under the placebo-controlled, double-blind study AGLU02704 (NCT00158600).
Primary Outcome
The numbers of participants who experienced 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 with alglucosidase alfa. Participants with long-term exposure to alglucosidase alfa (those from the Alglucosidase Alfa/Alglucosidase Alfa treatment group) are included. Time frames are stated from the start of th
Conditions Studied
Interventions
- BIOLOGICAL alglucosidase alfa
Study Locations (20)
Florida
- - Coral Springs
- - Gainesville
- - Sarasota
California
- - Beverly Hills
- - San Diego
Ohio
- - Cincinnati
- - Toledo
Alaska
- - Anchorage
Arizona
- - Phoenix
District of Columbia
- - Washington D.C.
Illinois
- - Chicago
Kansas
- - Kansas City
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 81 participants |
| Start Date | 2007-03 |
| Est. Completion | 2008-11 |
| Phase | Phase 4 |
What the finished NCT00455195 record still lists
NCT00455195 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 81 participants, a relatively small participant target, above the 37-participant average among 8 other Pompe Disease (Late-onset) trials with a reported enrollment target (119% higher).
The record links to 4 conditions, with Pompe Disease (Late-onset) appearing as the primary indexed condition, and to 1 intervention - of which alglucosidase alfa is the first listed.
NCT00455195 names 20 study sites across 16 states, led by Florida, California, Ohio.
Frequently Asked Questions
What is clinical trial NCT00455195 about?
NCT00455195 is a clinical study titled "Late-Onset Treatment Study Extension Protocol". 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 NCT00455195?
This trial is currently completed. It is a Phase 4 study. The enrollment target is 81 participants. The study started on 2007-03. Estimated completion is 2008-11.
What conditions does trial NCT00455195 study?
This clinical trial studies the following conditions: Pompe Disease (Late-onset), Glycogen Storage Disease Type II (GSD-II), Glycogenesis Type II, Acid Maltase Deficiency (AMD).
What interventions are being tested in trial NCT00455195?
The interventions under investigation include: alglucosidase alfa (BIOLOGICAL).
Who is sponsoring clinical trial NCT00455195?
This trial is sponsored by Genzyme, a Sanofi Company, which has 196 total clinical trials registered on ClinicalTrials.gov.
Where is trial NCT00455195 being conducted?
This trial has 20 study locations across Alaska, Arizona, California, District of Columbia, Florida. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
Learn More About Clinical Trials
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Where NCT00455195's enrollment target sits among peer trials
81 3rd of 8 higher than 6 of 8 other Pompe Disease (Late-onset) trials
participants (enrollment target), bucketed by value
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