Safety, Pharmacokinetics, Pharmacodynamics, and Efficacy of Migalastat in Pediatric Subjects (Aged 12 to <18 Years)

• Status: Completed
• Trial end date: 2021-02-02
• Target enrollment: 0
• Participant gender: All

Summary

This is an open-label study to evaluate the safety, pharmacokinetics, pharmacodynamics, and efficacy of migalastat treatment in pediatric subjects 12 to <18 years of age with Fabry disease and amenable GLA variants.

• Phase: Phase 3
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Amicus Therapeutics
• Treatments: 1-Deoxynojirimycin

Criteria

Inclusion Criteria

• Willing and able to provide written consent or assent (subject and parent/legal guardian, as applicable)

• Male or female between 12 and <18 years of age diagnosed with Fabry disease

• Confirmed GLA variant that has shown to be responsive to AT1001 in vivo

• Subject weighs at least 45 kg (99 pounds) at screening

• Subject has never been treated with ERT or has not received ERT for 14 days prior to screening

• Subject has at least one complication (ie, laboratory abnormality and/or sign/symptom) of Fabry disease

• Subject is able to swallow study medication whole

• Women/girls who can become pregnant and all males agree to be sexually abstinent or use medically accepted methods of birth control during the study and for 30 days after study completion

Exclusion Criteria

• Has moderate or severe renal impairment (eGFR <60 ml/min/1.73 m2 at screening)

• Has advanced kidney disease requiring dialysis or kidney transplantation

• History of allergy or sensitivity to study medication (including excipients) or other iminosugars (eg, miglustat, miglitol)

• Has received any gene therapy at any time or anticipates starting gene therapy during the study period

• Requires treatment with Glyset (miglitol), Zavesca (miglustat) within 6 months before screening or throughout the study

• Requires treatment with Replagal (agalsidase alfa), or Fabrazyme (agalsidase beta) within 14 days before screening or throughout the study

• Subject is treated or has 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 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

• Pregnant or breast-feeding

• Otherwise unsuitable for the study in the opinion of the investigator