A Study of Migalastat in Pediatric Subjects (2 to <12 Yrs) With Fabry Disease and Amenable GLA Variants

Ki vehet részt

• 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

Ki nem vehet részt

• Has received any investigational/experimental drug, biologic, or device within 30 days or 5 half-lives of the investigational product (whichever is longer) before Visit 1 (screening)
• Has received any gene therapy at any time or anticipates starting gene therapy during the study period
• Requires treatment with Glyset (miglitol) or Zavesca (miglustat), within 6 months before Visit 1(screening) or throughout the study
• Has advanced kidney disease requiring dialysis or kidney transplantation
• History of allergy or sensitivity to migalastat (including excipients) or other iminosugars (eg, miglustat, miglitol)
• Has any intercurrent illness or condition at Visit 1 (screening) or Visit 2 (baseline) that may preclude the subject from fulfilling the protocol requirements or suggests to the investigator that the potential subject may have an unacceptable risk by participating in this study
• Has moderate or severe renal impairment (eGFR < 60 mL/min/1.73 m2 at Visit 1 [screening])
• Pregnant or breastfeeding
• Otherwise unsuitable for the study in the opinion of the investigator