NCT06904261View on ClinicalTrials.gov

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

PHASE3RecruitingINTERVENTIONAL
Enrollment

8

Participants

Timeline

Start Date

October 31, 2025

Primary Completion Date

December 31, 2027

Study Completion Date

December 31, 2028

Conditions
Fabry Disease
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.

Trial Locations (7)

3000

NOT_YET_RECRUITING

Universitair Ziekenhuis (UZ) Leuven, Leuven

15224

NOT_YET_RECRUITING

UPMC Children's Hospital of Pittsburgh, Pittsburgh

22030

RECRUITING

Lysosomal and Rare Disorders Research and Treatment Center, Inc., Fairfax

28046

NOT_YET_RECRUITING

Hospital Universitario de la Paz, Madrid

30322

NOT_YET_RECRUITING

Emory Genetics, Atlanta

WC1N 3JH

NOT_YET_RECRUITING

Great Ormond Street Hospital for Children NHS Foundation Trust, London

M13 9WL

NOT_YET_RECRUITING

Manchester University NHS Foundation Trust, Manchester

Sponsors
All Listed Sponsors
lead

Amicus Therapeutics

INDUSTRY

NCT06904261 - A Study of Migalastat in Pediatric Subjects (2 to <12 Yrs) With Fabry Disease and Amenable GLA Variants | Biotech Hunter | Biotech Hunter