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

Status: Recruiting
Location: See location...
Intervention Type: Drug
Study Type: Interventional
Study Phase: Phase 3
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.

Eligibility
Participation Requirements
Sex: All
Minimum Age: 2
Maximum Age: 11
Healthy Volunteers: f
View:

• 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.

Locations
United States
Virginia
Lysosomal and Rare Disorders Research and Treatment Center, Inc.
RECRUITING
Fairfax
Contact Information
Primary
Amicus Therapeutics Patient Advocacy
patientadvocacy@amicusrx.com
609-662-2000
Time Frame
Start Date: 2025-10
Estimated Completion Date: 2028-12
Participants
Target number of participants: 8
Treatments
Experimental: Migalastat HCl 20 mg Dispersible Tablets
Migalastat will be administered every other day (QOD). The initial dose will be based on body weight at baseline.
Related Therapeutic Areas
Sponsors
Leads: Amicus Therapeutics

This content was sourced from clinicaltrials.gov