Actively Recruiting
A Study of Migalastat in Pediatric Subjects (2 to <12 Yrs) With Fabry Disease and Amenable GLA Variants
Led by Amicus Therapeutics · Updated on 2026-04-24
8
Participants Needed
11
Research Sites
151 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
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.
CONDITIONS
Official 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
- Parent or legal guardian willing and able to provide written informed consent, and subject provides assent if applicable
- Documented GLA variant in medical record that is 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)
- For subjects of reproductive potential, agreement to use medically accepted contraception during the study and for 30 days after last dose
You will not qualify if you...
- Moderate or severe renal impairment (eGFR less than 60 mL/min/1.73 m2 at screening)
- Advanced kidney disease requiring dialysis or kidney transplantation
- History of allergy or sensitivity to migalastat or other iminosugars
- Received investigational drug, biologic, or device within 30 days or 5 half-lives before screening
- Received gene therapy previously or plans to start gene therapy during the study
- Requires treatment with Glyset (miglitol) or Zavesca (miglustat) within 6 months before screening or during the study
- Any illness or condition at screening or baseline that may prevent fulfilling study requirements or poses unacceptable risk
- Pregnant or breastfeeding
- Otherwise unsuitable for the study as determined by the investigator
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
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
Not Yet 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
CONTACT
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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