Status:
COMPLETED
Safety, Pharmacodynamics, and Efficacy of Migalastat in Pediatric Subjects (Aged >12 Years) With Fabry Disease
Lead Sponsor:
Amicus Therapeutics
Conditions:
Fabry Disease
Eligibility:
All Genders
12-17 years
Phase:
PHASE3
Brief Summary
This is a long-term, Open-label Study to Evaluate the Safety, Pharmacodynamics, and Efficacy of Migalastat in Subjects \> 12 Years of Age With Fabry Disease and Amenable GLA Variants
Eligibility Criteria
Inclusion
- Male or female subjects diagnosed with Fabry disease \> 12 years of age who completed Study AT1001-020
- 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
- If of reproductive potential, both male and female subjects agreed 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
- Subject's last available estimated glomerular filtration rate (eGFR) in the previous study was \< 60 mL/min/1.73 m2
- Subject had advanced kidney disease requiring dialysis or kidney transplantation
- Subject received any investigational/experimental drug, biologic, or device within 30 days before baseline, with the exception of migalastat
- Subject anticipated starting gene therapy during the study period
- Subject had any intercurrent illness or condition at Visit 1 that may have precluded the subject from fulfilling the protocol requirements or suggested to the investigator that the potential subject may have an unacceptable risk by participating in this study
- Subject had a history of allergy or sensitivity to migalastat (including excipients) or other iminosugars (eg, miglustat, miglitol)
- Subject required treatment with Replagal® (agalsidase alfa) or Fabrazyme® (agalsidase beta)
- Subject required treatment with Glyset® (miglitol) or Zavesca® (miglustat)
- Female subject was pregnant or breast-feeding, or was planning to become pregnant during the study period
- In the opinion of the investigator, the subject and/or parent or legally-authorized representative was unlikely or unable to comply with the study requirements
Key Trial Info
Start Date :
October 11 2019
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
November 29 2024
Estimated Enrollment :
16 Patients enrolled
Trial Details
Trial ID
NCT04049760
Start Date
October 11 2019
End Date
November 29 2024
Last Update
June 17 2025
Active Locations (6)
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1
University of South Florida
Tampa, Florida, United States, 33606
2
The Emory Clinic
Atlanta, Georgia, United States, 30322
3
University of Minnesota Masonic Children's Hospital and Clinics
Minneapolis, Minnesota, United States, 55454
4
Cincinnati Children's Hospital
Cincinnati, Ohio, United States, 45229