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NCT03500094 · ClinicalTrials.gov registry record · Phase 3
Safety, Pharmacokinetics, Pharmacodynamics, and Efficacy of Migalastat in Pediatric Subjects (Aged 12 to <18 Years)
A Phase 3 study, sponsored by Amicus Therapeutics.
- Completed
- Registry status
- Phase 3
- Development phase
- 22
- Enrollment target
NCT03500094: Completed Phase 3 study, sponsored by Amicus Therapeutics.
NCT03500094 is a Phase 3 study that has completed, run by Amicus Therapeutics. The registered enrollment target is 22 participants, below the 772-participant average among 15,282 other Phase 3 trials with a reported enrollment target (97% lower). According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT03500094, a Phase 3 study, has completed, sponsored by Amicus Therapeutics.
- COMPLETED
- Registry status
- Phase 3
- Development phase
- 22 participants
- Enrollment target
Study Summary
This was an open-label study to evaluate the safety, pharmacokinetics (PK), pharmacodynamics (PD), and efficacy of migalastat treatment in pediatric participants 12 to \<18 years of age with Fabry disease and amenable gene encoding α-galactosidase A (GLA) variants.
Primary Outcome
TEAEs included adverse events that began on or after the first dose of study drug until 30 days after the last dose. Treatment-related TEAEs were defined as TEAEs that had an investigator-defined relationship to study drug of "Definite," "Probable," or "Possible." A summary of serious and all other non-serious adverse events, regardless of causality, is located in the Reported Adverse Events module.
Interventions
- DRUG Migalastat HCl 150 mg
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 22 participants |
| Start Date | 2018-09-27 |
| Est. Completion | 2021-02-06 |
| Phase | Phase 3 |
What the finished NCT03500094 record still lists
NCT03500094 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 22 participants, a relatively small participant target, below the 772-participant average among 15,282 other Phase 3 trials with a reported enrollment target (97% lower).
The record links to 0 conditions, and to 1 intervention - of which Migalastat HCl 150 mg is the first listed.
NCT03500094 does not publish any study locations in the registry export this page uses.
Frequently Asked Questions
What is clinical trial NCT03500094 about?
NCT03500094 is a clinical study titled "Safety, Pharmacokinetics, Pharmacodynamics, and Efficacy of Migalastat in Pediatric Subjects (Aged 12 to <18 Years)". This was an open-label study to evaluate the safety, pharmacokinetics (PK), pharmacodynamics (PD), and efficacy of migalastat treatment in pediatric participants 12 to \<18 years of age with Fabry disease and amenable gene encoding α-galactosidase A (GLA) variants.
What is the current status of trial NCT03500094?
This trial is currently completed. It is a Phase 3 study. The enrollment target is 22 participants. The study started on 2018-09-27. Estimated completion is 2021-02-06.
What interventions are being tested in trial NCT03500094?
The interventions under investigation include: Migalastat HCl 150 mg (DRUG).
Who is sponsoring clinical trial NCT03500094?
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 NCT03500094's enrollment target sits among peer trials
22 1953rd of 2000 higher than 48 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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