Safety, Pharmacodynamics, and Efficacy of Migalastat in Pediatric Subjects (Aged >12 Years) With Fabry Disease
Public ClinicalTrials.gov record NCT04049760. Field values are reproduced from the official study page; the official ClinicalTrials.gov record remains the source of truth for eligibility, enrollment, and contact information.
Data is sourced from official ClinicalTrials.gov public API records. Always review the official ClinicalTrials.gov record for the latest information.
Official title
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
Study identification
- NCT ID
- NCT04049760
- Recruitment status
- Completed
- Study type
- Interventional
- Phase
- Phase 3
- Lead sponsor
- Amicus Therapeutics
- Industry
- Enrollment
- 16 participants
Conditions and interventions
Conditions
Interventions
- migalastat HCl 150 mg Drug
Drug
Eligibility (public fields only)
- Age range
- 12 Years to 17 Years
- Sex
- All
- Healthy volunteers
- Healthy volunteers not accepted
This page does not interpret eligibility. Detailed inclusion and exclusion criteria are on the official ClinicalTrials.gov record.
Study timeline
- Start date
- Oct 10, 2019
- Primary completion
- Nov 28, 2024
- Completion
- Nov 28, 2024
- Last update posted
- Feb 4, 2026
2019 – 2024
United States locations
- U.S. sites
- 5
- U.S. states
- 5
- U.S. cities
- 5
| Facility | City | State | ZIP | Site status |
|---|---|---|---|---|
| University of South Florida | Tampa | Florida | 33606 | — |
| The Emory Clinic | Atlanta | Georgia | 30322 | — |
| University of Minnesota Masonic Children's Hospital and Clinics | Minneapolis | Minnesota | 55454 | — |
| Cincinnati Children's Hospital | Cincinnati | Ohio | 45229 | — |
| Lysosomal & Rare Disorders Research & Treatment Center | Fairfax | Virginia | 22030 | — |
Site contact phone numbers, emails, and investigator names are intentionally not displayed here. Open the official ClinicalTrials.gov record for site contact information.
Non-U.S. locations
This page focuses on the U.S. directory. The official record also lists 1 non-U.S. site.
About this trial record page
- What this page shows
- Public field values for ClinicalTrials.gov record NCT04049760, including study identification, conditions, interventions, eligibility (age, sex, healthy volunteer), timeline, and U.S. site list.
- What this page does not do
- No medical advice, eligibility judgments, treatment recommendations, study quality scoring, or AI-generated medical summaries. No site contact phone numbers, emails, or investigator names.
- Where the data comes from
- Sourced from the official ClinicalTrials.gov public API. The official record is the source of truth.
- Last refresh
- Last update posted Feb 4, 2026 · Synced Jul 21, 2026
Related: full search, browse by condition, browse by drug or therapy, browse by sponsor, browse by U.S. city.
Open the official record
The complete protocol, eligibility criteria, and contact information for NCT04049760 live on ClinicalTrials.gov.