Status:
TERMINATED
Open-Label Phase 3 Long-Term Safety Study of Migalastat
Lead Sponsor:
Amicus Therapeutics
Conditions:
Fabry Disease
Eligibility:
All Genders
16+ years
Phase:
PHASE3
Brief Summary
This was a long-term, open-label study of migalastat (123 milligrams \[mg\] of migalastat \[equivalent to 150 mg of migalastat hydrochloride\]) (migalastat) in participants with Fabry disease who comp...
Detailed Description
Study AT1001-041 was an open-label, noncomparative, multicenter, long-term extension study for participants with Fabry disease who completed treatment in one of three previous trials of migalastat (AT...
Eligibility Criteria
Inclusion
- Completed migalastat treatment in a previous Fabry disease protocol
- Both male and female participants were enrolled
- Age 16 years or older
- Male and female participants had to agree to use protocol-identified acceptable contraception
Exclusion
- Estimated glomerular filtration rate (eGFR) in the previous study was \<30 milliliters/minute/1.73 square meters (mL/min/1.73 m\^2) unless there was a measured GFR available within 3 m of the Baseline Visit that was \>30 mL/min/1.73 m\^2
- Had undergone, or was scheduled to undergo, kidney transplantation or was currently on dialysis
- Pregnant or breast feeding
- Treated with another investigational drug (except migalastat) within 30 days of study start
- Unable to comply with study requirements, or deemed otherwise unsuitable for study entry, in the opinion of the investigator
- Had documented transient ischemic attack, stroke, unstable angina, or myocardial infarction within the 12 m before the Baseline Visit
- Had clinically significant, unstable cardiac disease in the opinion of the investigator
- Had a history of allergy or sensitivity to migalastat (including excipients) or to other iminosugars
- Required treatment with Glyset (miglitol) or Zavesca (miglustat)
- Had any intercurrent illness or condition that may have precluded the participant from fulfilling the protocol requirements
- Had a severe or unsuitable concomitant medical condition
- Had a clinically significant abnormal laboratory value and a clinically significant electrocardiogram finding at the Baseline Visit.
Key Trial Info
Start Date :
October 14 2011
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
February 17 2016
Estimated Enrollment :
85 Patients enrolled
Trial Details
Trial ID
NCT01458119
Start Date
October 14 2011
End Date
February 17 2016
Last Update
October 2 2018
Active Locations (25)
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1
Atlanta, Georgia, United States, 30322
2
Chicago, Illinois, United States, 60611
3
Kansas City, Kansas, United States, 66160
4
Boston, Massachusetts, United States, 02114