Migalastat for children aged 2 to <12 with Fabry disease and amenable GLA variants

Is this trial a fit for you?

A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.

What this trial studies

This Phase 3b, open-label, uncontrolled, multicenter study will treat children aged 2 to <12 with Fabry disease and amenable GLA variants with oral migalastat 20 mg for 12 months divided into an approximately 3-month Stage 1 and a 9-month Stage 2 with no break between stages. Subjects must be ERT-naïve or have stopped enzyme replacement therapy at least 14 days before baseline. Participants are randomized 1:1:1 into three pharmacokinetic (PK) sampling groups with intensive PK sampling between Days 15–30 and at Month 6 and trough PK samples at Months 6 and 12. The study tracks safety, pharmacokinetics, pharmacodynamics, and efficacy measures over the treatment period, with a 30-day safety follow-up after any discontinuation.

Why it matters

Who can join: the full rules

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

Where it's running

• Emory Genetics
  Atlanta, Georgia, United States Recruiting
• University of Minnesota Masonic Children's Hospital
  Minneapolis, Minnesota, United States Recruiting
• Atrium Health Levine Children's Hospital
  Charlotte, North Carolina, United States Recruiting
• Cincinnati Children's Hospital Medical Center
  Cincinnati, Ohio, United States Recruiting
• UPMC Children's Hospital of Pittsburgh
  Pittsburgh, Pennsylvania, United States Recruiting
• Lysosomal and Rare Disorders Research and Treatment Center, Inc.
  Fairfax, Virginia, United States Recruiting

Study contacts

• Study coordinator: Amicus Therapeutics Patient Advocacy
• Email: patientadvocacy@amicusrx.com
• Phone: 609-662-2000

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How to take part

1. Review the eligibility criteria above with your treating physician.
2. Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
3. Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.