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

Recruiting now · Phase 3

Conditions studied: Fabry Disease

In brief

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 facts

Study ID: NCT06904261
Run by: Amicus Therapeutics
People needed: 8
Starts: 2026-01-08
Expected to finish: 2028-12-01
Last updated by the study team: 2026-06-25

Who can join

Age: 2 and older, up to 11. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

Male or female subjects, diagnosed with Fabry disease who are between ages 2 and < 12 years at randomization (subjects aged 11 years must have birthdays > 30 days after randomization)
Subject's parent or legally authorized representative is willing and able to provide written informed consent and authorization for use and disclosure of personal health information or research-related health information, and subject provides assent, if applicable.
Subject has a GLA variant documented in his/her medical record that is amenable to migalastat prior to Visit 2.
Subject has not received ERT (eg, Replagal® [agalsidase alfa] or Fabrazyme® [agalsidase beta]) for at least 14 days prior to Baseline visit.
Subject has at least 1 documented complication (ie, historical or current laboratory abnormality or sign/symptom) of Fabry disease
If of reproductive potential, both male and female subjects agree to use a medically accepted method of contraception throughout the duration of the study and for up to 30 days after their last dose of migalastat.

You may not qualify if…

Has moderate or severe renal impairment (eGFR < 60 mL/min/1.73 m2 at Visit 1 [screening]).
Has advanced kidney disease requiring dialysis or kidney transplantation.
History of allergy or sensitivity to migalastat (including excipients) or other iminosugars (eg, miglustat, miglitol).
Has received any investigational/experimental drug, biologic, or device within 30 days or 5 half-lives of the investigational product (whichever is longer) before Visit 1 (screening).
Has received any gene therapy at any time or anticipates starting gene therapy during the study period.
Requires treatment with Glyset (miglitol) or Zavesca (miglustat), within 6 months before Visit 1(screening) or throughout the study.
Has any intercurrent illness or condition at Visit 1 (screening) or Visit 2 (baseline) that may preclude the subject from fulfilling the protocol requirements or suggests to the investigator that the potential subject may have an unacceptable risk by participating in this study.
Pregnant or breastfeeding
Otherwise unsuitable for the study in the opinion of the investigator

Where it is running

Manchester University NHS Foundation Trust — Manchester, United Kingdom (enrolling)
University of Minnesota Masonic Children's Hospital — Minneapolis, Minnesota, United States (enrolling)
Atrium Health Levine Children's Hospital — Charlotte, North Carolina, United States (enrolling)
Cincinnati Children's Hospital Medical Center — Cincinnati, Ohio, United States (enrolling)
Hospital Universitario de la Paz — Madrid, Madrid, Spain (enrolling)
Emory Genetics — Atlanta, Georgia, United States (enrolling)
Lysosomal and Rare Disorders Research and Treatment Center, Inc. — Fairfax, Virginia, United States (enrolling)
Universitair Ziekenhuis (UZ) Leuven — Leuven, Vlaams-Brabant, Belgium (enrolling)
Universitäetsklinikum Müenster (UKM) Klinik für Kinder- und Jugendmedizin - Allgemeine Paediatrie — Münster, North Rhine-Westphalia, Germany (enrolling)
UPMC Children's Hospital of Pittsburgh — Pittsburgh, Pennsylvania, United States
Great Ormond Street Hospital for Children NHS Foundation Trust — London, United Kingdom

Full record on ClinicalTrials.gov

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