Status:
COMPLETED
Open-Label Extension Study of the Long-Term Effects of Migalastat HCL in Patients With Fabry Disease
Lead Sponsor:
Amicus Therapeutics
Conditions:
Fabry Disease
Eligibility:
All Genders
18+ years
Phase:
PHASE3
Brief Summary
This is an open-label extension study intended to provide continued treatment with migalastat hydrochloride (HCl) for participants with Fabry disease who completed treatment of a previous migalastat H...
Eligibility Criteria
Inclusion
- Participant had completed treatment in a previous study of migalastat HCl given as a monotherapy
- Male and female participant agreed to use protocol-identified acceptable contraception
- Participant was willing to provide written informed consent and authorization for use and disclosure of Personal Health Information (PHI)
Exclusion
- Participant's last available estimated glomerular filtration rate (eGFR) in the previous study was \<30 milliliter (mL)/minute (min)/1.73 meters squared (m\^2); unless there was measured GFR available within 3 months of Baseline Visit, which was \>30 mL/min/1.73 m\^2
- Participant had undergone, or was scheduled to undergo kidney transplantation or was currently on dialysis
- Participant had a documented transient ischemic attack, stroke, unstable angina, or myocardial infarction within the 3 months before Baseline Visit
- Participant had clinically significant unstable cardiac disease in the opinion of the investigator (for example, cardiac disease requiring active management, such as symptomatic arrhythmia, unstable angina, or New York Heart Association class III or IV congestive heart failure)
- Participant had a history of allergy or sensitivity to AT1001 (including excipients) or other iminosugars (for example, miglustat, miglitol)
- Participant required treatment with Glyset® (miglitol) or Zavesca® (miglustat)
- Participants with severe or unsuitable concomitant medical condition
- Participants with clinically significant abnormal laboratory value(s) and/or clinically significant electrocardiogram (ECG) findings
Key Trial Info
Start Date :
March 14 2015
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
October 23 2019
Estimated Enrollment :
84 Patients enrolled
Trial Details
Trial ID
NCT02194985
Start Date
March 14 2015
End Date
October 23 2019
Last Update
December 21 2020
Active Locations (28)
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1
Clinical Study Site
Atlanta, Georgia, United States, 30322
2
Clinical Study Site
Kansas City, Kansas, United States, 66160
3
Clinical Study Site
Grand Rapids, Michigan, United States, 49525
4
Clinical Study Site
New York, New York, United States, 10016