Safety, Pharmacodynamics, and Efficacy of Migalastat in Pediatric Subjects (Aged >12 Years) With Fabry Disease

Amicus Therapeutics

Status and phase

Completed

Phase 3

Conditions

Fabry Disease

Treatments

Drug: migalastat HCl 150 mg

Study type

Interventional

Funder types

Industry

Identifiers

NCT04049760

2019-000222-21 (EudraCT Number)

AT1001-036

Details and patient eligibility

About

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

Enrollment

16 patients

Sex

All

Ages

12 to 17 years old

Volunteers

No Healthy Volunteers

Inclusion criteria

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 criteria

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