Migalastat in Young Children with Fabry Disease

Who can take part

• 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. • For subjects without a known GLA variant, an amenable GLA genotyping result from the local laboratory must be received 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, including but not limited to: a. corneal whorls b. neuropathic pain and/or acroparesthesia and/or acute crises persisting or recurring at least twice over the previous 3 months or longer, or requiring management with analgesia c. Fabry disease-related gastrointestinal signs and symptoms (eg, diarrhea, abdominal pain) persisting or recurring at least twice over the previous 3 months or longer d. hypohidrosis (present for at least 3 months) e. left ventricular mass index (LVMi) above the normal range for age and sex f. rhythm and/or conduction disturbances, for example: − episode of tachycardia or bradycardia, − arrhythmia, or; − abnormal PR, QRS, or QT interval g. reduced estimated glomerular filtration rate (eGFR) (using the Schwartz formula) for age and sex, or hyperfiltration (> 135 mL/min/1.73 m2) h. proteinuria or albuminuria in a spot urine (early morning preferable) or as determined by the investigator i. elevated plasma globotriaosylsphingosine (lyso-Gb3) j. hearing impairment and/or tinnitus k. transient ischemic attack/stroke
• 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

Who cannot take part

• Subject 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)
• Subject has received any gene therapy at any time or anticipates starting gene therapy during the study period
• Subject requires treatment with Glyset® (miglitol) or Zavesca® (miglustat), within 6 months before Visit 1 (screening) or throughout the study
• Subject has advanced kidney disease requiring dialysis or kidney transplantation
• Subject has a history of allergy or sensitivity to migalastat (including excipients) or other iminosugars (eg, miglustat, miglitol)
• Subject 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
• Subject has moderate or severe renal impairment (eGFR < 60 mL/min/1.73 m2 at Visit 1 [screening])
• Female subject is pregnant or breastfeeding or is planning to become pregnant during the study period
• In the opinion of the investigator, the subject and/or parent or legally-authorized representative is unlikely or unable to comply with the study requirements