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NCT00433147 · ClinicalTrials.gov registry record · Phase 2
A Study of AT2101 (Afegostat Tartrate) in Adult Patients With Type 1 Gaucher Disease Currently Receiving Enzyme Replacement Therapy
A Phase 2 study of Gaucher Disease and Gaucher Disease, Type 1, sponsored by Amicus Therapeutics.
- Completed
- Registry status
- Phase 2
- Development phase
- 30
- Enrollment target
- 10
- Study locations
NCT00433147: Completed Phase 2 study of Gaucher Disease and Gaucher Disease, Type 1, sponsored by Amicus Therapeutics.
NCT00433147 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 30 participants, below the 1,056-participant average among 13 other Gaucher Disease trials with a reported enrollment target (97% lower). The trial reports 10 study locations across 9 states. According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT00433147, 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
- 30 participants
- Enrollment target
- 10
- Study locations
Study Summary
This study was conducted to test the safety and tolerability of afegostat tartrate in participants with type 1 Gaucher disease already receiving enzyme replacement therapy.
Primary Outcome
TEAEs were defined as any adverse event (AE) with a start date on or after administration of the study drug (on Day 1). A severe AE was 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 7 days after the last dose of study drug (Day 35) is presented. A summary of serious and all other non-serious AEs regardless of causality is located in the Reported Adverse Events module.
Conditions Studied
Interventions
- DRUG Afegostat tartrate
Study Locations (10)
Pennsylvania
- - Philadelphia
- - Pittsburgh
California
- - San Francisco
Florida
- - Coral Springs
Georgia
- - Decatur
Iowa
- - Iowa City
New York
- - New York
Ohio
- - Cincinnati
Oregon
- - Portland
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 30 participants |
| Start Date | 2007-03-23 |
| Est. Completion | 2008-02-19 |
| Phase | Phase 2 |
What the finished NCT00433147 record still lists
NCT00433147 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 30 participants, a relatively small participant target, below the 1,056-participant average among 13 other Gaucher Disease trials with a reported enrollment target (97% 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.
NCT00433147 lists 10 locations in 9 states (Pennsylvania, California, Florida).
Frequently Asked Questions
What is clinical trial NCT00433147 about?
NCT00433147 is a clinical study titled "A Study of AT2101 (Afegostat Tartrate) in Adult Patients With Type 1 Gaucher Disease Currently Receiving Enzyme Replacement Therapy". This study was conducted to test the safety and tolerability of afegostat tartrate in participants with type 1 Gaucher disease already receiving enzyme replacement therapy.
What is the current status of trial NCT00433147?
This trial is currently completed. It is a Phase 2 study. The enrollment target is 30 participants. The study started on 2007-03-23. Estimated completion is 2008-02-19.
What conditions does trial NCT00433147 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 NCT00433147?
The interventions under investigation include: Afegostat tartrate (DRUG).
Who is sponsoring clinical trial NCT00433147?
This trial is sponsored by Amicus Therapeutics, which has 34 total clinical trials registered on ClinicalTrials.gov.
Where is trial NCT00433147 being conducted?
This trial has 10 study locations across California, Florida, Georgia, Iowa, New York. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
Learn More About Clinical Trials
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Where NCT00433147's enrollment target sits among peer trials
30 8th of 13 higher than 5 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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