Open-Label Phase 3 Long-Term Safety Study of Migalastat

• Status: Terminated
• Trial end date: 2016-02-17
• Target enrollment: 0
• Participant gender: All

Summary

This was a long-term, open-label study of migalastat (123 milligrams [mg] of migalastat [equivalent to 150 mg of migalastat hydrochloride]) (migalastat) in participants with Fabry disease who completed treatment in a previous monotherapy trial with migalastat.

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

Details

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

Criteria

Inclusion Criteria:

• Completed migalastat treatment in a previous Fabry disease protocol

• Both male and female participants were enrolled

• Age 16 years or older

• Male and female participants had to agree to use protocol-identified acceptable contraception

Exclusion Criteria:

• Estimated glomerular filtration rate (eGFR) in the previous study was <30 milliliters/minute/1.73 square meters (mL/min/1.73 m^2) unless there was a measured GFR available within 3 m of the Baseline Visit that was >30 mL/min/1.73 m^2

• Had undergone, or was scheduled to undergo, kidney transplantation or was currently on dialysis

• Pregnant or breast feeding

• Treated with another investigational drug (except migalastat) within 30 days of study start

• Unable to comply with study requirements, or deemed otherwise unsuitable for study entry, in the opinion of the investigator

• Had documented transient ischemic attack, stroke, unstable angina, or myocardial infarction within the 12 m before the Baseline Visit

• Had clinically significant, unstable cardiac disease in the opinion of the investigator

• Had a history of allergy or sensitivity to migalastat (including excipients) or to other iminosugars

• Required treatment with Glyset (miglitol) or Zavesca (miglustat)

• Had any intercurrent illness or condition that may have precluded the participant from fulfilling the protocol requirements

• Had a severe or unsuitable concomitant medical condition

• Had a clinically significant abnormal laboratory value and a clinically significant electrocardiogram finding at the Baseline Visit.