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NCT00672022 · ClinicalTrials.gov registry record · Phase 3
Pharmacokinetics, Safety and Tolerability of Zavesca (Miglustat) in Patients With Infantile Onset Gangliosidosis: Single and Steady State Oral Doses
A Phase 3 study, sponsored by Children's National Research Institute.
- Completed
- Registry status
- Phase 3
- Development phase
- 10
- Enrollment target
NCT00672022: Completed Phase 3 study, sponsored by Children's National Research Institute.
NCT00672022 is a Phase 3 study that has completed, run by Children's National Research Institute. The registered enrollment target is 10 participants, below the 772-participant average among 15,282 other Phase 3 trials with a reported enrollment target (99% lower). According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT00672022, a Phase 3 study, has completed, sponsored by Children's National Research Institute.
- COMPLETED
- Registry status
- Phase 3
- Development phase
- 10 participants
- Enrollment target
Study Summary
We want to see if Zavesca (or miglustat) is safe and can be tolerated by patients with acute infantile onset GM2 gangliosidosis - classical Tay-Sachs and infantile onset Sandhoff disease. We know that miglustat inhibits the formation of GM2 ganglioside, the compound that is stored in the brains of children with Tay-Sachs and Sandhoff disease. Since it inhibits the synthesis of ganglioside, miglustat may be able to reduce or delay the onset of clinical symptoms.
Interventions
- DRUG Zavesca (Miglustat)
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 10 participants |
| Start Date | 2004-07 |
| Est. Completion | 2007-08 |
| Phase | Phase 3 |
What the finished NCT00672022 record still lists
NCT00672022 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 10 participants, a relatively small participant target, below the 772-participant average among 15,282 other Phase 3 trials with a reported enrollment target (99% lower).
The record links to 0 conditions, and to 1 intervention - of which Zavesca (Miglustat) is the first listed.
NCT00672022 does not publish any study locations in the registry export this page uses.
Frequently Asked Questions
What is clinical trial NCT00672022 about?
NCT00672022 is a clinical study titled "Pharmacokinetics, Safety and Tolerability of Zavesca (Miglustat) in Patients With Infantile Onset Gangliosidosis: Single and Steady State Oral Doses". We want to see if Zavesca (or miglustat) is safe and can be tolerated by patients with acute infantile onset GM2 gangliosidosis - classical Tay-Sachs and infantile onset Sandhoff disease. We know that miglustat inhibits the formation of GM2 ganglioside, the compound that is stored in the brains of c...
What is the current status of trial NCT00672022?
This trial is currently completed. It is a Phase 3 study. The enrollment target is 10 participants. The study started on 2004-07. Estimated completion is 2007-08.
What interventions are being tested in trial NCT00672022?
The interventions under investigation include: Zavesca (Miglustat) (DRUG).
Who is sponsoring clinical trial NCT00672022?
This trial is sponsored by Children's National Research Institute, which has 92 total clinical trials registered on ClinicalTrials.gov.
Learn More About Clinical Trials
How this trial's enrollment target compares
Where NCT00672022's enrollment target sits among peer trials
10 1989th of 2000 higher than 9 of 2,000 other Phase 3 trials
participants (enrollment target), bucketed by value
Each bar is a band; taller bars hold more other Phase 3 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
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