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NCT00446550 · ClinicalTrials.gov registry record · Phase 2
A Study of Oral AT2101 (Afegostat Tartrate) in Treatment-naive Patients With Gaucher Disease
A Phase 2 study of Gaucher Disease and Gaucher Disease, Type 1, sponsored by Amicus Therapeutics.
- Completed
- Registry status
- Phase 2
- Development phase
- 19
- Enrollment target
- 11
- Study locations
NCT00446550: Completed Phase 2 study of Gaucher Disease and Gaucher Disease, Type 1, sponsored by Amicus Therapeutics.
NCT00446550 is a Phase 2 study of Gaucher Disease and Gaucher Disease, Type 1 that has completed, run by Amicus Therapeutics. The registered enrollment target is 19 participants, below the 1,057-participant average among 13 other Gaucher Disease trials with a reported enrollment target (98% lower). The trial reports 11 study locations across 8 states. According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT00446550, a Phase 2 study of Gaucher Disease and Gaucher Disease, Type 1, has completed, sponsored by Amicus Therapeutics.
- COMPLETED
- Registry status
- Phase 2
- Development phase
- 19 participants
- Enrollment target
- 11
- Study locations
Study Summary
This study evaluated the safety and tolerability of afegostat tartrate in participants with type 1 Gaucher disease who were not receiving enzyme replacement therapy (ERT) or substrate reduction therapy (SRT).
Primary Outcome
A TEAE was defined as any adverse event (AE) with start date on or after administration of study drug or pre-existing conditions that worsened on or after the start of the first study drug administration (on Day 1). A severe AE defined as an AE that was incapacitating and required medical intervention. The number of participants who experienced 1 or more severe TEAEs after dosing on Day 1 through the end of follow-up (Day 183) is presented. A summary of serious and all other non-serious AEs rega
Conditions Studied
Interventions
- DRUG afegostat tartrate
Study Locations (11)
Other
- - Haifa
- - Tel Aviv
- - Johannesburg
- - London
California
- - Beverly Hills
Florida
- - Coral Springs
Georgia
- - Decatur
Iowa
- - Iowa City
Kansas
- - Kansas City
Massachusetts
- - Boston
Ohio
- - Cincinnati
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 19 participants |
| Start Date | 2008-06-11 |
| Est. Completion | 2009-08-20 |
| Phase | Phase 2 |
What the finished NCT00446550 record still lists
NCT00446550 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 19 participants, a relatively small participant target, below the 1,057-participant average among 13 other Gaucher Disease trials with a reported enrollment target (98% lower).
The record links to 3 conditions, with Gaucher Disease appearing as the primary indexed condition, and to 1 intervention - of which afegostat tartrate is the first listed.
NCT00446550 lists 11 locations in 8 states (Other, California, Florida).
Frequently Asked Questions
What is clinical trial NCT00446550 about?
NCT00446550 is a clinical study titled "A Study of Oral AT2101 (Afegostat Tartrate) in Treatment-naive Patients With Gaucher Disease". This study evaluated the safety and tolerability of afegostat tartrate in participants with type 1 Gaucher disease who were not receiving enzyme replacement therapy (ERT) or substrate reduction therapy (SRT).
What is the current status of trial NCT00446550?
This trial is currently completed. It is a Phase 2 study. The enrollment target is 19 participants. The study started on 2008-06-11. Estimated completion is 2009-08-20.
What conditions does trial NCT00446550 study?
This clinical trial studies the following conditions: Gaucher Disease, Gaucher Disease, Type 1, Type 1 Gaucher Disease.
What interventions are being tested in trial NCT00446550?
The interventions under investigation include: afegostat tartrate (DRUG).
Who is sponsoring clinical trial NCT00446550?
This trial is sponsored by Amicus Therapeutics, which has 34 total clinical trials registered on ClinicalTrials.gov.
Where is trial NCT00446550 being conducted?
This trial has 11 study locations across California, Florida, Georgia, Iowa, Kansas. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
Learn More About Clinical Trials
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Where NCT00446550's enrollment target sits among peer trials
19 12th of 13 higher than 2 of 13 other Gaucher Disease trials
participants (enrollment target), bucketed by value
Each bar is a band; taller bars hold more other Gaucher Disease 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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