Status:
COMPLETED
A Study of AT1001 (Migalastat Hydrochloride) in Participants With Fabry Disease
Lead Sponsor:
Amicus Therapeutics
Conditions:
Fabry Disease
Eligibility:
MALE
18-55 years
Phase:
PHASE2
Brief Summary
Study to evaluate the safety, tolerability, pharmacokinetics, and pharmacodynamics of migalastat hydrochloride (HCl) (migalastat) in participants with Fabry disease.
Detailed Description
This was a Phase 2, open-label study in male participants with Fabry disease. All participants who met initial eligibility criteria underwent a 28-day screening period, including a 14-day run-in with ...
Eligibility Criteria
Inclusion
- 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
- 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
Key Trial Info
Start Date :
January 2 2006
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
January 29 2008
Estimated Enrollment :
9 Patients enrolled
Trial Details
Trial ID
NCT00214500
Start Date
January 2 2006
End Date
January 29 2008
Last Update
October 30 2018
Active Locations (5)
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1
Los Angeles, California, United States, 90048
2
Decatur, Georgia, United States, 30033
3
Bethesda, Maryland, United States, 20892
4
New York, New York, United States, 10016