A Study of AT1001 (Migalastat Hydrochloride) in Participants With Fabry Disease

• Status: Completed
• Trial end date: 2008-01-29
• Target enrollment: 0
• Participant gender: Male

Summary

Study to evaluate the safety, tolerability, pharmacokinetics, and pharmacodynamics of migalastat hydrochloride (HCl) (migalastat) in participants with Fabry disease.

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

Details

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

Criteria

Inclusion Criteria:

• Males between 18 and 55 years of age (inclusive)

• Hemizygous for Fabry disease

• Had a confirmed diagnosis of Fabry disease with a documented missense gene mutation (individual or familial)

• Had enhanceable enzyme activity

• In the judgment of the investigator, were either able to safely suspend ERT throughout the study, or be ERT naive

• Agreed to be sexually abstinent or use a condom with spermicide when engaging in sexual activity during the course of the study and for a period of 30 days following completion of the study

• Were willing and able to sign an informed consent form

Exclusion Criteria:

• History of significant disease other than Fabry disease (for example, end-stage renal disease; Class III or IV heart disease [per the New York Heart Association classification]; current diagnosis of cancer, except for basal cell carcinoma of the skin; diabetes [unless hemoglobin A1c ≤8]; or neurological disease that would have impaired the participant's ability to participate in the study)

• History of organ transplant

• Serum creatinine >2 mg per deciliter on Day -2

• Screening 12-lead electrocardiogram demonstrating corrected QT interval >450 milliseconds prior to dosing

• Taking a medication prohibited by the protocol: Fabrazyme® (agalsidase beta), Replagal™ (agalsidase alfa), Glyset® (miglitol), Zavesca® (miglustat), or any experimental therapy for any indication

• Participated in a previous clinical trial in the last 30 days

• Any other condition, which, in the opinion of the investigator, would jeopardize the safety of the participant or impact the validity of the study results