A Study of Migalastat in Children with Fabry Disease
This study is looking at a medication called migalastat (20 mg dispersible tablets) for children aged 2 to less than 12 years old who have Fabry disease. Fabry disease is a rare genetic condition. The study aims to understand how safe migalastat is, how the body processes it (pharmacokinetics or PK), and how well it works. Researchers will be looking for any side effects (adverse events or AEs) and how much migalastat is in the blood over time. To join, children must have Fabry disease and specific genetic changes (amenable GLA variants). The study is currently recruiting 8 participants.
- Study design
- This is an open-label study, meaning everyone knows what treatment is being given. It will include 8 participants and has two treatment stages followed by an open-label extension.
- What's involved
- Participants will receive migalastat treatment for about 12 months, with blood samples taken at specific times during the first month, at Month 6, and at Month 12.
- Compensation
- Not stated in the trial record.
- Follow-up
- Participants will have a 30-day safety follow-up period after their last dose of migalastat.
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NCT06904261
A Study of Migalastat in Pediatric Subjects (2 to <12 Yrs) With Fabry Disease and Amenable GLA Variants
Recruiting
PHASE3Ages 2–11InterventionalTreatmentAmicus Therapeutics
~8 participants
Updated 2026-06-25 on ClinicalTrials.gov
What's tested:Migalastat HCl 20 mg
At a glance
Recruiting sites
9 of 11 listed sites are recruiting right now
RecruitingSuspended, closed, or not yet open
What they're measuring
Safety: Incidence of TEAEs, SAEs, and AEs leading to discontinuation of study drug
Measured over Day 1 (after dosing) through Month 12 and follow-up (30 days after last dose)
+3 more outcomes measured
Conditions
Where it's being run
11 sites across 10 statesUnited Kingdom2
Georgia1
Minnesota1
North Carolina1
Ohio1
Pennsylvania1
Virginia1
Vlaams-Brabant1
Who to contact
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Do you actually qualify for this trial?
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Eligibility criteria
Inclusion
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
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 this trial measures
- Safety: Incidence of TEAEs, SAEs, and AEs leading to discontinuation of study drugDay 1 (after dosing) through Month 12 and follow-up (30 days after last dose)
- Pharmacokinetics (PK): Maximum Observed Plasma Concentration (Cmax) of Migalastat0 to 12 hours postdose during the first month of study and trough samples at Months 6 and 12
- Pharmacokinetics (PK): Minimum Observed Plasma Concentration (Cmin) of Migalastat0 to 12 hours postdose during the first month of study and trough samples at Months 6 and 12
- Pharmacokinetics (PK): Area Under The Plasma Concentration-time Curve Over The Dosing Interval (AUCtau) of Migalastat0 to 12 hours postdose during the first month of study and trough samples at Months 6 and 12