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NCT00925301 · ClinicalTrials.gov registry record · Phase 3
Study of the Effects of Oral AT1001 (Migalastat Hydrochloride) in Patients With Fabry Disease
A Phase 3 study of Fabry Disease, sponsored by Amicus Therapeutics.
- Completed
- Registry status
- Phase 3
- Development phase
- 67
- Enrollment target
- 20
- Study locations
NCT00925301: Completed Phase 3 study of Fabry Disease, sponsored by Amicus Therapeutics.
NCT00925301 is a Phase 3 study of Fabry Disease that has completed, run by Amicus Therapeutics. The registered enrollment target is 67 participants, below the 541-participant average among 32 other Fabry Disease trials with a reported enrollment target (88% lower). The trial reports 20 study locations across 14 states. According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT00925301, a Phase 3 study of Fabry Disease, has completed, sponsored by Amicus Therapeutics.
- COMPLETED
- Registry status
- Phase 3
- Development phase
- 67 participants
- Enrollment target
- 20
- Study locations
Study Summary
The primary objective of this study was to compare the effect of migalastat (123 milligrams \[mg\] of migalastat \[equivalent to 150 mg of migalastat hydrochloride\]) (migalastat) versus placebo on kidney globotriaosylceramide (GL-3).
Primary Outcome
Renal biopsies were taken at Baseline and Month 6 (Stage 1). The specimens were evaluated using virtual microscopy. The annotation, scoring, and adjudication of the kidney histology assessments were performed by the Clinical Pathology Endpoints Committee. The number of kidney IC GL-3 inclusions was assessed by 3 renal pathologists who were blinded to treatment assignments, participants' data, and biopsy sequence. One pathologist served as annotator and identified the 300 capillaries on up to 8 s
Conditions Studied
Interventions
- DRUG Placebo
- DRUG migalastat hydrochloride
Study Locations (20)
Other
- - Buenos Aires
- - Adelaide
- - Parkville
- - Porto Alegre
- - São Paulo
- - Copenhagen
California
- - Los Angeles
- - San Francisco
Georgia
- - Decatur
Illinois
- - Chicago
Kansas
- - Kansas City
Massachusetts
- - Boston
Michigan
- - Grand Rapids
New York
- - New York
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 67 participants |
| Start Date | 2009-10-23 |
| Est. Completion | 2014-01-29 |
| Phase | Phase 3 |
What the finished NCT00925301 record still lists
NCT00925301 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 67 participants, a relatively small participant target, below the 541-participant average among 32 other Fabry Disease trials with a reported enrollment target (88% lower).
The record links to 1 condition, with Fabry Disease appearing as the primary indexed condition, and to 2 interventions - of which Placebo is the first listed.
NCT00925301 names 20 study sites across 14 states, led by Other, California, Georgia.
Frequently Asked Questions
What is clinical trial NCT00925301 about?
NCT00925301 is a clinical study titled "Study of the Effects of Oral AT1001 (Migalastat Hydrochloride) in Patients With Fabry Disease". The primary objective of this study was to compare the effect of migalastat (123 milligrams \[mg\] of migalastat \[equivalent to 150 mg of migalastat hydrochloride\]) (migalastat) versus placebo on kidney globotriaosylceramide (GL-3).
What is the current status of trial NCT00925301?
This trial is currently completed. It is a Phase 3 study. The enrollment target is 67 participants. The study started on 2009-10-23. Estimated completion is 2014-01-29.
What conditions does trial NCT00925301 study?
This clinical trial studies the following conditions: Fabry Disease.
What interventions are being tested in trial NCT00925301?
The interventions under investigation include: Placebo (DRUG), migalastat hydrochloride (DRUG).
Who is sponsoring clinical trial NCT00925301?
This trial is sponsored by Amicus Therapeutics, which has 34 total clinical trials registered on ClinicalTrials.gov.
Where is trial NCT00925301 being conducted?
This trial has 20 study locations across California, Georgia, Illinois, Kansas, Massachusetts. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
Learn More About Clinical Trials
Similar trials for Fabry Disease
Matched on the same primary condition, ranked to surface studies in the same phase first, then by recruiting status, no relevance scoring or editorial curation.
Where NCT00925301's enrollment target sits among peer trials
67 12th of 32 higher than 21 of 32 other Fabry Disease trials
participants (enrollment target), bucketed by value
Each bar is a band; taller bars hold more other Fabry 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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