A Study of AT1001 (Migalastat Hydrochloride) in Participants With Fabry Disease
Completed · Phase 2
Conditions studied: Fabry Disease
In brief
Study to evaluate the safety, tolerability, pharmacokinetics, and pharmacodynamics of migalastat hydrochloride (HCl) (migalastat) in participants with Fabry disease.
Key facts
- Study ID
- NCT00214500
- Run by
- Amicus Therapeutics
- People needed
- 9
- Starts
- 2006-01-02
- Expected to finish
- 2008-01-29
- Last updated by the study team
- 2018-10-30
Who can join
Age: 18 and older, up to 55. Sex: male. Healthy volunteers: not accepted.
You may qualify if…
- 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
You may not qualify if…
- 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
Where it is running
- Study site — Los Angeles, California, United States
- Study site — Decatur, Georgia, United States
- Study site — Bethesda, Maryland, United States
- Study site — New York, New York, United States
- Study site — Houston, Texas, United States
Full record on ClinicalTrials.gov
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