Medical Information Only. Always consult your healthcare provider before enrolling in any clinical trial.
NCT00357786 · ClinicalTrials.gov registry record · Phase 1
An Open-Label Maintenance Study of the Enzyme Replacement Therapy Replagal in Patients With Fabry Disease
A Phase 1 study, sponsored by National Institute of Neurological Disorders and Stroke (NINDS).
- Completed
- Registry status
- Phase 1
- Development phase
- 3
- Enrollment target
NCT00357786: Completed Phase 1 study, sponsored by National Institute of Neurological Disorders and Stroke (NINDS).
NCT00357786 is a Phase 1 study that has completed, run by National Institute of Neurological Disorders and Stroke (NINDS). The registered enrollment target is 3 participants, below the 60-participant average among 29,502 other Phase 1 trials with a reported enrollment target (95% lower). According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT00357786, a Phase 1 study, has completed, sponsored by National Institute of Neurological Disorders and Stroke (NINDS).
- COMPLETED
- Registry status
- Phase 1
- Development phase
- 3 participants
- Enrollment target
Study Summary
This study will continue to evaluate the safety of using intravenous doses of Replagal for two patients with Fabry disease. Fabry disease is a genetic disorder inherited as an X-linked recessive trait. It causes a deficiency in the enzyme alpha galactosidase, which normally breaks down a lipid, or fatty substance called ceramidetrihexoside, a building block in all cells of the body. The deficiency in breaking down the lipid eventually causes that lipid to accumulate and injure cells. Vascular, renal, and neurological problems are the results. It is not known exactly how lipid accumulation brings about such problems, studies of another lipid storage disorder. Two patients 7 to 17 years of age who have Fabry disease and have been receiving intravenous infusions of Replagal at a dose of 0.2 mg/kg of body weight every 2 weeks may be eligible for this study. Participants will undergo the following tests and procedures: * Physical examination. * Neurological examination. * Medical and medication history. * Vital signs. * Assessment of height and weight. * Blood tests to determine complete blood count and chemistries. * Electrocardiogram. * Doppler blood flow study. Participants will go through a baseline evaluation, over a period of about 1 day. They will receive an intravenous infusion of Replagal every other week, at the dose of 0.2 mg/kg of body weight. Vital signs will be measured before the infusion and immediately and after and 1 hour afterward. There will be careful monitoring for allergic reactions and side effects. The infusion time takes approximately 40 minutes. This study will last at least 1 year, or until the sponsor doing the investigating or the drug manufacturer decides to withdraw support of the study.
Interventions
- DRUG Replagal
- DRUG Replagal agalsidase alfa
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 3 participants |
| Start Date | 2003-10 |
| Phase | Phase 1 |
What the finished NCT00357786 record still lists
NCT00357786 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 3 participants, a relatively small participant target, below the 60-participant average among 29,502 other Phase 1 trials with a reported enrollment target (95% lower).
The record links to 0 conditions, and to 2 interventions - of which Replagal is the first listed.
NCT00357786 does not publish any study locations in the registry export this page uses.
Frequently Asked Questions
What is clinical trial NCT00357786 about?
NCT00357786 is a clinical study titled "An Open-Label Maintenance Study of the Enzyme Replacement Therapy Replagal in Patients With Fabry Disease". This study will continue to evaluate the safety of using intravenous doses of Replagal for two patients with Fabry disease. Fabry disease is a genetic disorder inherited as an X-linked recessive trait. It causes a deficiency in the enzyme alpha galactosidase, which normally breaks down a lipid, or f...
What is the current status of trial NCT00357786?
This trial is currently completed. It is a Phase 1 study. The enrollment target is 3 participants. The study started on 2003-10.
What interventions are being tested in trial NCT00357786?
The interventions under investigation include: Replagal (DRUG), Replagal agalsidase alfa (DRUG).
Who is sponsoring clinical trial NCT00357786?
This trial is sponsored by National Institute of Neurological Disorders and Stroke (NINDS), which has 567 total clinical trials registered on ClinicalTrials.gov.
Learn More About Clinical Trials
How this trial's enrollment target compares
Where NCT00357786's enrollment target sits among peer trials
3 1999th of 2000 higher than 1 of 2,000 other Phase 1 trials
participants (enrollment target), bucketed by value
Each bar is a band; taller bars hold more other Phase 1 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