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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: Recruiting Phase 3 study of Fabry Disease, sponsored by Amicus Therapeutics.

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. According to ClinicalTrials.gov, the official US trial registry.

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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

Fabry Disease

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

FieldValue
Enrollment Target 8 participants
Start Date 2026-01-08
Est. Completion 2028-12
Phase Phase 3
Amicus Therapeutics

34 total trials

What NCT06904261 shows while recruiting

NCT06904261 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 8 participants, a relatively small participant target, below the 543-participant average among 32 other Fabry Disease trials with a reported enrollment target (99% lower).

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 lists 9 locations in 8 states (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.

Learn More About Clinical Trials

Similar trials for Fabry Disease

Matched on the same primary condition, ranked to surface studies in the same phase first, then by recruiting status, no relevance scoring or editorial curation.

Where NCT06904261's enrollment target sits among peer trials

8 32nd of 32 higher than 1 of 32 other Fabry Disease trials

participants (enrollment target), bucketed by value

Each bar is a band; taller bars hold more other Fabry Disease 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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Cross-condition peers matched on enrollment target and registry start date, not the same-condition list above.

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More recruiting context for NCT06904261

Source: ClinicalTrials.gov NCT06904261, the US trial registry maintained by the National Library of Medicine. NCT06904261 (small enrollment · multi site footprint · recruiting) retrieved and formatted by PlainTrial, see methodology.