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A Study of Migalastat in Pediatric Subjects (2 to <12 Yrs) With Fabry Disease and Amenable GLA Variants

NCT06904261 · Status: RECRUITING · Phase: PHASE3 · Type: INTERVENTIONAL · Enrollment: 8

Last updated 2026-05-15

No results posted yet for this 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

Interventions

DRUG

Migalastat HCl 20 mg

Migalastat will be supplied as 20-mg dispersible tablets. Migalastat 20-mg dispersible tablets contain 16 mg migalastat free base.

Sponsors & Collaborators

  • Amicus Therapeutics

    lead INDUSTRY

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
2 Years
Max Age
11 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2026-01-08
Primary Completion
2027-12-31
Completion
2028-12-31
FDA Drug
Yes

Countries

  • United States
  • Belgium
  • Germany
  • Spain
  • United Kingdom

Study Locations

More Related Trials

Entities

Diseases

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT06904261 on ClinicalTrials.gov