Medical Information Only. Always consult your healthcare provider before enrolling in any clinical trial.
NCT00051935 · ClinicalTrials.gov registry record · Phase 2
A Study of the Safety and Pharmacokinetics of rhGAA in Siblings With Glycogen Storage Disease Type II
A Phase 2 study, sponsored by Genzyme, a Sanofi Company.
- Completed
- Registry status
- Phase 2
- Development phase
- 2
- Enrollment target
NCT00051935: Completed Phase 2 study, sponsored by Genzyme, a Sanofi Company.
NCT00051935 is a Phase 2 study that has completed, run by Genzyme, a Sanofi Company. The registered enrollment target is 2 participants, below the 133-participant average among 30,622 other Phase 2 trials with a reported enrollment target (98% lower). According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT00051935, a Phase 2 study, has completed, sponsored by Genzyme, a Sanofi Company.
- COMPLETED
- Registry status
- Phase 2
- Development phase
- 2 participants
- Enrollment target
Study Summary
GSD-II (also known as Pompe disease) 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 GSD-II, an excessive amount of glycogen accumulates and is stored in various tissues, especially heart and skeletal muscle, which prevents their normal function. This study is being conducted to evaluate the safety, pharmacokinetics, pharmacodynamics and efficacy of recombinant human acid alpha-glucosidase (rhGAA) as a potential enzyme replacement therapy for a pair of siblings with GSD-II. To be eligible for this study, a patient must have a confirmed diagnosis of GSD-II and have a sister or brother who also has a confirmed diagnosis of GSD-II.
Interventions
- DRUG Alglucosidase alfa
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 2 participants |
| Start Date | 2003-01 |
| Est. Completion | 2003-10 |
| Phase | Phase 2 |
What the finished NCT00051935 record still lists
NCT00051935 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 2 participants, a relatively small participant target, below the 133-participant average among 30,622 other Phase 2 trials with a reported enrollment target (98% lower).
The record links to 0 conditions, and to 1 intervention - of which Alglucosidase alfa is the first listed.
NCT00051935 does not publish any study locations in the registry export this page uses.
Frequently Asked Questions
What is clinical trial NCT00051935 about?
NCT00051935 is a clinical study titled "A Study of the Safety and Pharmacokinetics of rhGAA in Siblings With Glycogen Storage Disease Type II". GSD-II (also known as Pompe disease) 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 GSD-II, an e...
What is the current status of trial NCT00051935?
This trial is currently completed. It is a Phase 2 study. The enrollment target is 2 participants. The study started on 2003-01. Estimated completion is 2003-10.
What interventions are being tested in trial NCT00051935?
The interventions under investigation include: Alglucosidase alfa (DRUG).
Who is sponsoring clinical trial NCT00051935?
This trial is sponsored by Genzyme, a Sanofi Company, which has 196 total clinical trials registered on ClinicalTrials.gov.
Learn More About Clinical Trials
How this trial's enrollment target compares
Where NCT00051935's enrollment target sits among peer trials
2 2001st of 2000 the lowest of 2,000 other Phase 2 trials
participants (enrollment target), bucketed by value
Each bar is a band; taller bars hold more other Phase 2 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.
Source ClinicalTrials.gov registry export · 2026-08-08
Nationwide trials with similar profiles
Cross-condition peers matched on enrollment target and registry start date, not the same-condition list above.
Similar registry start date
- NCT05988463 · started 2027-01-01 · Phase 1
Dose-Escalation Study of Artesunate Patients With IPF
- NCT07219706 · started 2026-12-10 · NA
Dietary Fiber and Time Restricted Eating
- NCT02831335 · started 2026-12-01
Technical Development of Multi-Parametric Renocerebral MRI
- NCT05962346 · started 2026-12 · NA
Fetal Endoscopic Tracheal Occlusion for Congenital Diaphragmatic Hernia