Status:
COMPLETED
Study of the Effects of Oral AT1001 (Migalastat Hydrochloride) in Patients With Fabry Disease
Lead Sponsor:
Amicus Therapeutics
Conditions:
Fabry Disease
Eligibility:
All Genders
16-74 years
Phase:
PHASE3
Brief Summary
The primary objective of this study was to compare the effect of migalastat (123 milligrams \[mg\] of migalastat \[equivalent to 150 mg of migalastat hydrochloride\]) (migalastat) versus placebo on ki...
Detailed Description
This double-blind, randomized, placebo-controlled study was conducted in 67 participants at 46 sites worldwide. The study consisted of 2 stages and an optional open-label treatment extension phase: S...
Eligibility Criteria
Inclusion
- Male or female between the ages of 16 and 74 diagnosed with Fabry disease.
- Confirmed mutant form of α-galactosidase A shown to be responsive to migalastat in vitro.
- Participant has never been treated with enzyme replacement therapy (ERT) or has not received ERT for 6 consecutive months or longer before the screening visit for the study.
- Urine GL-3 ≥4 times the upper limit of normal at screening.
- Participants taking angiotensin converting enzyme inhibitors or angiotensin receptor blockers must be on a stable dose for a minimum of 4 weeks before the baseline visit.
- Females who can become pregnant and all males agree to be sexually abstinent or use medically accepted methods of birth control during the study and for 30 days after study completion.
- Participant is willing and able to provide written informed consent and assent, if applicable.
Exclusion
- Participant has undergone or is scheduled to undergo kidney transplantation, or is currently on dialysis.
- Estimated glomerular filtration rate \<30 milliliters per minute per 1.73 meters squared (chronic kidney disease Stage 4 or 5) based on the Modification of Diet in Renal Disease equation at screening.
- Pregnant or breast-feeding.
- History of allergy or sensitivity to study medication (including excipients) or other iminosugars (for example, miglustat, miglitol).
- Participant is treated or has been treated with any investigational drug within 30 days of study start.
- Participant is currently treated or has ever been treated with migalastat.
Key Trial Info
Start Date :
October 23 2009
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
January 29 2014
Estimated Enrollment :
67 Patients enrolled
Trial Details
Trial ID
NCT00925301
Start Date
October 23 2009
End Date
January 29 2014
Last Update
October 30 2018
Active Locations (28)
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1
Los Angeles, California, United States, 90048
2
San Francisco, California, United States, 94143
3
Decatur, Georgia, United States, 30033
4
Chicago, Illinois, United States, 60611