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NCT04049760 · ClinicalTrials.gov registry record · Phase 3
Safety, Pharmacodynamics, and Efficacy of Migalastat in Pediatric Subjects (Aged >12 Years) With Fabry Disease
A Phase 3 study, sponsored by Amicus Therapeutics.
- Completed
- Registry status
- Phase 3
- Development phase
- 16
- Enrollment target
NCT04049760: Completed Phase 3 study, sponsored by Amicus Therapeutics.
NCT04049760 is a Phase 3 study that has completed, run by Amicus Therapeutics. The registered enrollment target is 16 participants, below the 772-participant average among 15,282 other Phase 3 trials with a reported enrollment target (98% lower). According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT04049760, a Phase 3 study, has completed, sponsored by Amicus Therapeutics.
- COMPLETED
- Registry status
- Phase 3
- Development phase
- 16 participants
- Enrollment target
Study Summary
This is a long-term, Open-label Study to Evaluate the Safety, Pharmacodynamics, and Efficacy of Migalastat in Subjects \> 12 Years of Age With Fabry Disease and Amenable GLA Variants
Primary Outcome
Number of subjects with TEAE, SAE, and AE leading to discontinuation during the study period
Interventions
- DRUG migalastat HCl 150 mg
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 16 participants |
| Start Date | 2019-10-11 |
| Est. Completion | 2024-11-29 |
| Phase | Phase 3 |
What the finished NCT04049760 record still lists
NCT04049760 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 16 participants, a relatively small participant target, below the 772-participant average among 15,282 other Phase 3 trials with a reported enrollment target (98% lower).
The record links to 0 conditions, and to 1 intervention - of which migalastat HCl 150 mg is the first listed.
NCT04049760 does not publish any study locations in the registry export this page uses.
Frequently Asked Questions
What is clinical trial NCT04049760 about?
NCT04049760 is a clinical study titled "Safety, Pharmacodynamics, and Efficacy of Migalastat in Pediatric Subjects (Aged >12 Years) With Fabry Disease". This is a long-term, Open-label Study to Evaluate the Safety, Pharmacodynamics, and Efficacy of Migalastat in Subjects \> 12 Years of Age With Fabry Disease and Amenable GLA Variants
What is the current status of trial NCT04049760?
This trial is currently completed. It is a Phase 3 study. The enrollment target is 16 participants. The study started on 2019-10-11. Estimated completion is 2024-11-29.
What interventions are being tested in trial NCT04049760?
The interventions under investigation include: migalastat HCl 150 mg (DRUG).
Who is sponsoring clinical trial NCT04049760?
This trial is sponsored by Amicus Therapeutics, which has 34 total clinical trials registered on ClinicalTrials.gov.
Learn More About Clinical Trials
How this trial's enrollment target compares
Where NCT04049760's enrollment target sits among peer trials
16 1974th of 2000 higher than 24 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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