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

Part of paid clinical trials in Atlanta, Georgia.

Sponsor
Amicus Therapeutics
Study ID
NCT06904261
Phase
PHASE3
Status
Recruiting
Apply to this trial

Conditions

Eligibility Criteria

Sex
ALL
Age
2 Years - 11 Years
Healthy Volunteers
Not accepted

Interventions

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

Study Details

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.

Key Dates

Start date
Jan 8, 2026
Status verified
Jun 2025
Primary completion
Dec 31, 2027
Completion
Dec 31, 2028

Study Design

Enrollment
8 participants (estimated)
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT

Arms

  • Experimental: Migalastat HCl 20 mg Dispersible Tablets
    Migalastat will be administered every other day (QOD). The initial dose will be based on body weight at baseline.

Primary Outcome Measure

Safety: Incidence of TEAEs, SAEs, and AEs leading to discontinuation of study drug [ Time Frame: Day 1 (after dosing) through Month 12 and follow-up (30 days after last dose) ]

Central Contacts

Locations (6)

FacilityCityStateZIPSite coordinators
Emory GeneticsAtlantaGeorgia30322-
University of Minnesota Masonic Children's HospitalMinneapolisMinnesota55455-
Atrium Health Levine Children's HospitalCharlotteNorth Carolina28203-
Cincinnati Children's Hospital Medical CenterCincinnatiOhio45229-
UPMC Children's Hospital of PittsburghPittsburghPennsylvania15224-
Lysosomal and Rare Disorders Research and Treatment Center, Inc.FairfaxVirginia22030-

Find similar trials in Atlanta, GA

By research site
Emory Genetics· Atlanta, GAUniversity of Minnesota Masonic Children's Hospital· Minneapolis, MNAtrium Health Levine Children's Hospital· Charlotte, NCCincinnati Children's Hospital Medical Center· Cincinnati, OHUPMC Children's Hospital of Pittsburgh· Pittsburgh, PALysosomal and Rare Disorders Research and Treatment Center, Inc.· Fairfax, VA

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