Open-label Long-term Safety Study of AT1001 (Migalastat Hydrochloride) in Participants With Fabry Disease Who Have Completed a Previous AT1001 Study

• Status: Terminated
• Trial end date: 2012-09-08
• Target enrollment: 0
• Participant gender: All

Summary

Study to evaluate the long-term safety, tolerability, and pharmacodynamics (PD) of migalastat hydrochloride (HCl) (migalastat) in participants with Fabry disease

• Phase: Phase 2
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Amicus Therapeutics

Criteria

Inclusion Criteria:

• Must have completed another Phase 2 study of migalastat in Fabry Disease

• Women of childbearing potential must have had a negative result on their pregnancy test

• Male and female participants agreed to use a reliable method of contraception during study treatment and for 4 weeks after study treatment termination

• Were willing and able to provide written informed consent

Exclusion Criteria:

• Had not completed a Phase 2 study of migalastat in Fabry Disease

• Had a major protocol violation in the preceding migalastat trial and was discontinued

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

• Had been treated with another investigational drug (except migalastat) within 30 days of study start

• Had been treated with Fabrazyme® (agalsidase beta), Replagal™ (agalsidase alfa), Glyset® (miglitol), or Zavesca® (miglustat) within 2 weeks prior to enrollment