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NCT06904261 · ClinicalTrials.gov registry record · Phase 3
A Study of Migalastat in Pediatric Subjects (2 to <12 Yrs) With Fabry Disease and Amenable GLA Variants
A Phase 3 study of Fabry Disease, sponsored by Amicus Therapeutics.
- Recruiting
- Registry status
- Phase 3
- Development phase
- 8
- Enrollment target
- 9
- Study locations
NCT06904261 is a Phase 3 study of Fabry Disease that is actively recruiting participants, run by Amicus Therapeutics. The registered enrollment target is 8 participants, below the 543-participant average among 32 other Fabry Disease trials with a reported enrollment target (99% lower). The trial reports 9 study locations across 8 states.
The verdict
NCT06904261, a Phase 3 study of Fabry Disease, is actively recruiting participants, sponsored by Amicus Therapeutics.
- RECRUITING
- Registry status
- Phase 3
- Development phase
- 8 participants
- Enrollment target
- 9
- Study locations
Study Summary
An open-label study to evaluate the safety, pharmacokinetics (PK), pharmacodynamics (PD), and efficacy of migalastat treatment in pediatric subjects 2 to \< 12 years of age with Fabry disease and with amenable GLA variants.
Conditions Studied
Interventions
- DRUG Migalastat HCl 20 mg
Study Locations (9)
Other
- Great Ormond Street Hospital for Children NHS Foundation Trust - London
- Manchester University NHS Foundation Trust - Manchester
Georgia
- Emory Genetics - Atlanta
Ohio
- Cincinnati Children's Hospital Medical Center - Cincinnati
Pennsylvania
- UPMC Children's Hospital of Pittsburgh - Pittsburgh
Virginia
- Lysosomal and Rare Disorders Research and Treatment Center, Inc. - Fairfax
Vlaams-Brabant
- Universitair Ziekenhuis (UZ) Leuven - Leuven
North Rhine-Westphalia
- Universitäetsklinikum Müenster (UKM) Klinik für Kinder- und Jugendmedizin - Allgemeine Paediatrie - Münster
Madrid
- Hospital Universitario de la Paz - Madrid
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 8 participants |
| Start Date | 2026-01-08 |
| Est. Completion | 2028-12 |
| Phase | Phase 3 |
Interested in This Trial?
Full Details on ClinicalTrials.gov ↗What the Registry Record Tells You About NCT06904261
The ClinicalTrials.gov registry entry for NCT06904261 describes a study currently listed as recruiting, categorized as Phase 3. The registered enrollment target is 8 participants, a figure that helps gauge the scale of data the investigators plan to collect, below the 543-participant average among 32 other Fabry Disease trials with a reported enrollment target (99% lower). The listed sponsor is Amicus Therapeutics, which has 34 total studies on file at ClinicalTrials.gov.
The record links to 1 condition, with Fabry Disease appearing as the primary indexed condition, and to 1 intervention - of which Migalastat HCl 20 mg is the first listed.
NCT06904261 reports 9 study locations spanning 8 distinct geographic areas - top geographies include Other, Georgia, Ohio.
Frequently Asked Questions
What is clinical trial NCT06904261 about?
NCT06904261 is a clinical study titled "A Study of Migalastat in Pediatric Subjects (2 to <12 Yrs) With Fabry Disease and Amenable GLA Variants". An open-label study to evaluate the safety, pharmacokinetics (PK), pharmacodynamics (PD), and efficacy of migalastat treatment in pediatric subjects 2 to \< 12 years of age with Fabry disease and with amenable GLA variants.
What is the current status of trial NCT06904261?
This trial is currently recruiting. It is a Phase 3 study. The enrollment target is 8 participants. The study started on 2026-01-08. Estimated completion is 2028-12.
What conditions does trial NCT06904261 study?
This clinical trial studies the following conditions: Fabry Disease.
What interventions are being tested in trial NCT06904261?
The interventions under investigation include: Migalastat HCl 20 mg (DRUG).
Who is sponsoring clinical trial NCT06904261?
This trial is sponsored by Amicus Therapeutics, which has 34 total clinical trials registered on ClinicalTrials.gov.
Where is trial NCT06904261 being conducted?
This trial has 9 study locations across Georgia, Ohio, Pennsylvania, Virginia, Vlaams-Brabant. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
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Read our methodology - how this data is sourced, computed, and verified.
Related
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