Actively Recruiting
An Open-label Study of the Safety, Pharmacokinetics, Pharmacodynamics, and Efficacy of 12 Month Treatment With Migalastat in Pediatric Subjects (Aged 2 to < 12 Years) With Fabry Disease and Amenable GLA Variants
Led by Amicus Therapeutics · Updated on 2026-05-15
8
Participants Needed
11
Research Sites
52 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
Researchers are evaluating the safety, pharmacokinetics, pharmacodynamics, and effectiveness of migalastat treatment in children aged 2 to less than 12 years with Fabry disease who have specific GLA gene variants that respond to this treatment. This Phase 3b, open-label, multicenter study includes pediatric subjects either new to enzyme replacement therapy (ERT) or who have stopped ERT at least 14 days before starting the study. The study consists of two consecutive treatment stages totaling 12 months of migalastat given every other day, with the initial dose based on body weight. Stage 1 lasts about 3 months, followed immediately by Stage 2 of 9 months. Participants are randomly assigned to one of three pharmacokinetic sampling groups, with blood samples collected at specified times during the study to measure migalastat levels. Participants will undergo regular safety monitoring, including tracking treatment-emergent adverse events, and blood tests to measure migalastat concentration and disease-related markers. Additional assessments include kidney function tests, heart measurements, symptom questionnaires, and quality of life surveys conducted at various times throughout the 12-month treatment and a 30-day follow-up after treatment ends. The study extends beyond the initial treatment period with an open-label extension phase for ongoing evaluation.
CONDITIONS
Brief Title
A Study of Migalastat in Pediatric Subjects (2 to <12 Yrs) With Fabry Disease and Amenable GLA Variants
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Male or female subjects diagnosed with Fabry disease aged 2 to less than 12 years at randomization (subjects aged 11 must have birthdays more than 30 days after randomization)
- Parent or legal representative able to provide written informed consent and subject provides assent if applicable
- Documented GLA variant amenable to migalastat prior to Visit 2
- No enzyme replacement therapy for at least 14 days before Baseline visit
- At least one documented complication of Fabry disease (laboratory abnormality or sign/symptom)
- If of reproductive potential, agree to use medically accepted contraception during the study and for 30 days after last dose
You will not qualify if you...
- Moderate or severe kidney impairment (eGFR less than 60 mL/min/1.73 m2 at screening)
- Advanced kidney disease requiring dialysis or kidney transplant
- Allergy or sensitivity to migalastat or similar iminosugars
- Use of any investigational drug, biologic, or device within 30 days or 5 half-lives before screening
- Prior or planned gene therapy during the study period
- Use of Glyset (miglitol) or Zavesca (miglustat) within 6 months before screening or during the study
- Any illness or condition at screening or baseline that increases risk or prevents following protocol
- Pregnancy or breastfeeding
- Otherwise unsuitable for the study as judged by the investigator
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Your Study Journey
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
1 visit (in-person)
Duration - 12 months
Participants receive migalastat treatment every other day based on body weight for 12 months.
Approximately monthly visits during the first month with PK sampling on Days 15 to 30, and visits at Months 3, 6, and 12 for assessments
Duration - 30 days
Participants who discontinue treatment enter a 30-day safety follow-up period without treatment.
1 visit (in-person)
Trial Site Locations
Total: 11 locations
1
Emory Genetics
Atlanta, Georgia, United States, 30322
Actively Recruiting
2
University of Minnesota Masonic Children's Hospital
Minneapolis, Minnesota, United States, 55455
Actively Recruiting
3
Atrium Health Levine Children's Hospital
Charlotte, North Carolina, United States, 28203
Actively Recruiting
4
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, United States, 45229
Actively Recruiting
5
UPMC Children's Hospital of Pittsburgh
Pittsburgh, Pennsylvania, United States, 15224
Not Yet Recruiting
6
Lysosomal and Rare Disorders Research and Treatment Center, Inc.
Fairfax, Virginia, United States, 22030
Actively Recruiting
7
Universitair Ziekenhuis (UZ) Leuven
Leuven, Vlaams-Brabant, Belgium, 3000
Not Yet Recruiting
8
Universitäetsklinikum Müenster (UKM) Klinik für Kinder- und Jugendmedizin - Allgemeine Paediatrie
Münster, North Rhine-Westphalia, Germany, 48149
Actively Recruiting
9
Hospital Universitario de la Paz
Madrid, Madrid, Spain, 28046
Actively Recruiting
10
Great Ormond Street Hospital for Children NHS Foundation Trust
London, United Kingdom, WC1N 3JH
Not Yet Recruiting
11
Manchester University NHS Foundation Trust
Manchester, United Kingdom, M13 9WL
Actively Recruiting
Research Team
A
Amicus Therapeutics Patient Advocacy
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NA
Model
SINGLE_GROUP
Primary Purpose
TREATMENT
Number of Arms
1
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