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

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.

CT.gov Identifier
NCT06904261
EudraCT Identifier
N/A
CTIS:
N/A
Sponsor
Amicus Therapeutics
Collaborator
N/A
Study Contact Information
+1 609 662 2000
Recruiting
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Study Details

Diseases
Fabry Disease
Study Drug
Migalastat hydrochloride
Genes
GLA
Study Dates
Jun 2025 - Dec 2027
Sex
Female & Male
Age
2 - 11 Years
Inclusion and Exclusion Criteria
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 diseaseIf 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:
  • 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

Protocol 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 amenable GLA variants.

Study Locations

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