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This study evaluates the safety, distribution in the body, and effect of the drug Migalastat HCl in children aged 12 to under 18 with Fabry disease. It enrolled participants who had an amenable GLA gene variant and weighed at least 45 kg. The study was conducted in Phase 3 and has been completed.
The summary above is a plain-language rendering of the official record. The original English title is shown for reference.
Criteria are reproduced from the ClinicalTrials.gov record in the original English. Only the trial team can determine eligibility.
Key Inclusion Criteria * Willing and able to provide written consent or assent (participant and parent/legal guardian, as applicable) * Male or female between 12 and \<18 years of age diagnosed with Fabry disease * Confirmed, amenable GLA variant * Participant weighed at least 45 kg (99 pounds) at screening * Participant had never been treated with ERT or had not received ERT for 14 days prior to screening * Participant had at least 1 complication (such as, laboratory abnormality and/or sign/symptom) of Fabry disease * Participant was able to swallow study medication whole Key Exclusion Criteria * Had moderate or severe renal impairment (estimated glomerular filtration rate (eGFR) \<60 milliliter/minute/1.73 meter squared (m\^2) at screening) * Had advanced kidney disease requiring dialysis or kidney transplantation * History of allergy or sensitivity to study medication (including excipients) or other iminosugars (for example, miglustat, miglitol) * Had received any gene therapy at any time or anticipated starting gene therapy during the study period * Required treatment with Glyset (miglitol) and/or Zavesca (miglustat) within 6 months before screening or throughout the study * Required treatment with Replagal (agalsidase alfa), or Fabrazyme (agalsidase beta) within 14 days before screening or throughout the study * Participant was treated or had been treated with any investigational/experimental drug, biologic or device within 30 days before screening * Any intercurrent illness or condition or concomitant medication use considered to be a contraindication at screening or baseline or that may have precluded the participant from fulfilling the protocol requirements or suggested to the investigator that the potential participant may have had an unacceptable risk by participating in this study * Pregnant or breast-feeding or planned to become pregnant during the study period * Otherwise unsuitable for the study in the opinion of the investigator