Safety, Pharmacodynamics, and Efficacy of Migalastat in Pediatric Subjects (Aged >12 Years) With … (NCT04049760) | Clinical Trial Compass
CompletedPhase 3
Safety, Pharmacodynamics, and Efficacy of Migalastat in Pediatric Subjects (Aged >12 Years) With Fabry Disease
United States16 participantsStarted 2019-10-11
Plain-language 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
Who can participate
Age range12 Years – 17 Years
SexALL
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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)…
What they're measuring
1
Incidence of Treatment-emergent Adverse Events (TEAEs), Serious Adverse Events (SAEs), and AEs (Adverse Events) Leading to Discontinuation of Study Drug