AI Cure is a research tool showing open data. Not medical advice. Discuss with your doctor. Terms

← Trials
🧪

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

RecruitingPhase 3Ages 2 Years - 11 YearsAllAmicus Therapeutics
💬

In plain words

What the trial is, and whether it could suit you

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.

Sign in to get the eligibility rules as simple questions to take to your doctor.

📋 Eligibility rules (as published)
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
💊

What it tests

  • Migalastat HCl 20 mg
📍

Where

12 sites
Emory Genetics
Atlanta, United States
University of Minnesota Masonic Children's Hospital
Minneapolis, United States
Atrium Health Levine Children's Hospital
Charlotte, United States
Cincinnati Children's Hospital Medical Center
Cincinnati, United States
UPMC Children's Hospital of Pittsburgh
Pittsburgh, United States
Lysosomal and Rare Disorders Research and Treatment Center, Inc.
Fairfax, United States
Universitair Ziekenhuis (UZ) Leuven
Leuven, Belgium
Universitäetsklinikum Müenster (UKM) Klinik für Kinder- und Jugendmedizin - Allgemeine Paediatrie
Münster, Germany

…and 4 more on ClinicalTrials.gov.

🤝 Taking part is your choice, and you can leave at any time. Only the trial team can confirm you are eligible — always decide together with your own doctor.
🛡️

AI Cure shows public evidence (Open Targets, ClinicalTrials.gov, PubMed, openFDA, DepMap, AlphaFold) with its source, and AI summaries that cite it — not medical advice and not a cure. Treatment decisions belong with your doctor. Research ideas are shared only with approved researchers and labs, who can test them. In an emergency call your local emergency number (112 in India and Europe, 911 in the US). AI Cure Terms of Use · Privacy