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Clinical Trials in the USA / NCT06904261
Recruiting Phase 3

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

NCT06904261 · tracked via the Priya Life Science USA tracker
Sponsor
Amicus Therapeutics
Phase
Phase 3
Started
2026-01-08
Last updated
2026-09-01

Condition(s) studied

Fabry Disease

Investigational drug(s) / intervention(s)

Migalastat HCl 20 mg

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

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.

Eligibility

Sex
ALL
Min age
2 Years
Max age
11 Years
Healthy volunteers
No
Inclusion Criteria * 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. Exclusion Criteria * 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

Primary outcome measure(s)

Trial sites (12)

FacilityCityRegionStatus
Emory Genetics Atlanta Georgia Recruiting
University of Minnesota Masonic Children's Hospital Minneapolis Minnesota Recruiting
Atrium Health Levine Children's Hospital Charlotte North Carolina Recruiting
Cincinnati Children's Hospital Medical Center Cincinnati Ohio Recruiting
UPMC Children's Hospital of Pittsburgh Pittsburgh Pennsylvania Recruiting
Lysosomal and Rare Disorders Research and Treatment Center, Inc. Fairfax Virginia Recruiting
Universitair Ziekenhuis (UZ) Leuven Leuven Vlaams-Brabant Recruiting
Universitäetsklinikum Müenster (UKM) Klinik für Kinder- und Jugendmedizin - Allgemeine Paediatrie Münster North Rhine-Westphalia Recruiting
Kitasato University Hospital Sagamihara Kanagawa Not Yet Recruiting
Hospital Universitario de la Paz Madrid Madrid Recruiting
Great Ormond Street Hospital for Children NHS Foundation Trust London United Kingdom Not Yet Recruiting
Manchester University NHS Foundation Trust Manchester United Kingdom Recruiting

More Amicus Therapeutics trials in the USA

Other trials for the same condition

Official registry record

This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.

View NCT06904261 on ClinicalTrials.gov ↗ ← All trials in the USA