Safety, Pharmacodynamics, and Efficacy of Migalastat in Pediatric Subjects (Aged >12 Years) With Fabry Disease
Completed · Phase 3
Conditions studied: Fabry Disease
In brief
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
Key facts
- Study ID
- NCT04049760
- Run by
- Amicus Therapeutics
- People needed
- 16
- Starts
- 2019-10-11
- Expected to finish
- 2024-11-29
- Last updated by the study team
- 2026-02-05
Who can join
Age: 12 and older, up to 17. Sex: any. Healthy volunteers: not accepted.
You may qualify if…
- 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
You may not qualify if…
- 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
Where it is running
- University of South Florida — Tampa, Florida, United States
- The Emory Clinic — Atlanta, Georgia, United States
- University of Minnesota Masonic Children's Hospital and Clinics — Minneapolis, Minnesota, United States
- Cincinnati Children's Hospital — Cincinnati, Ohio, United States
- Lysosomal & Rare Disorders Research & Treatment Center — Fairfax, Virginia, United States
- Royal Free London NHS Foundation Trust — London, United Kingdom
Full record on ClinicalTrials.gov
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