Status:
RECRUITING
A Study of Migalastat in Pediatric Subjects (2 to <12 Yrs) With Fabry Disease and Amenable GLA Variants
Lead Sponsor:
Amicus Therapeutics
Conditions:
Fabry Disease
Eligibility:
All Genders
2-11 years
Phase:
PHASE3
Brief Summary
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...
Detailed Description
This is a Phase 3b, 2-stage, open-label, uncontrolled, multicenter study to evaluate the safety, PK, PD, and efficacy of 12 months of migalastat treatment in pediatric subjects 2 to \< 12 years of age...
Eligibility Criteria
Inclusion
- 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
Exclusion
Key Trial Info
Start Date :
December 1 2025
Trial Type :
INTERVENTIONAL
Allocation :
ESTIMATED
End Date :
December 1 2028
Estimated Enrollment :
8 Patients enrolled
Trial Details
Trial ID
NCT06904261
Start Date
December 1 2025
End Date
December 1 2028
Last Update
December 4 2025
Active Locations (9)
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1
Emory Genetics
Atlanta, Georgia, United States, 30322
2
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, United States, 45229
3
UPMC Children's Hospital of Pittsburgh
Pittsburgh, Pennsylvania, United States, 15224
4
Lysosomal and Rare Disorders Research and Treatment Center, Inc.
Fairfax, Virginia, United States, 22030