Safety, Pharmacokinetics, Pharmacodynamics, and Efficacy of Migalastat in Pediatric Subjects (Aged 12 to <18 Years)
Public ClinicalTrials.gov record NCT03500094. 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
An Open-label Study of the Safety, Pharmacokinetics, Pharmacodynamics, and Efficacy of 12 Month Treatment With Migalastat in Pediatric Subjects (Aged 12 to <18 Years) With Fabry Disease and Amenable GLA Variants
Brief summary
Reproduced verbatim from the official ClinicalTrials.gov record. Not medical advice.
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.
Study identification
- NCT ID
- NCT03500094
- Recruitment status
- Completed
- Study type
- Interventional
- Phase
- Phase 3
- Enrollment
- 22 participants
Conditions and interventions
Conditions
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
- Sep 27, 2018 (Actual)
- Primary completion
- Feb 2, 2021 (Actual)
- Completion
- Feb 6, 2021 (Actual)
- Last update posted
- Nov 30, 2021
2018 – 2021
United States locations
- U.S. sites
- 7
- U.S. states
- 7
- U.S. cities
- 7
| Facility | City | State | ZIP | Site status |
|---|---|---|---|---|
| Clinical Study Site | Tampa | Florida | 33606 | — |
| Clinical Study Site | Atlanta | Georgia | 30322 | — |
| Clinical Study Site | Minneapolis | Minnesota | 55454 | — |
| Clinical Study Site | Columbia | Missouri | 65212 | — |
| Clinical Study Site | Cincinnati | Ohio | 45229 | — |
| Clinical Study Site | Pittsburgh | Pennsylvania | 15224 | — |
| Clinical Study Site | 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 NCT03500094, 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 Nov 30, 2021 · Synced Oct 18, 2026
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Open the official record
The complete public registry record, listed eligibility criteria, and available contact information for NCT03500094 live on ClinicalTrials.gov.