Open-Label Extension Study of the Long-Term Effects of Migalastat HCL in Patients With Fabry Disease



Status:Active, not recruiting
Conditions:Hematology, Metabolic
Therapuetic Areas:Hematology, Pharmacology / Toxicology
Healthy:No
Age Range:18 - Any
Updated:6/17/2018
Start Date:October 2014
End Date:October 2019

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An Open-Label Extension Study to Evaluate the Long Term Safety and Efficacy of Migalastat Hydrochloride Monotherapy in Subjects With Fabry Disease

This is an open-label extension study intended to provide continued treatment with migalastat
HCl for subjects with Fabry disease who completed treatment of a previous migalastat HCl
monotherapy study.

The study will assess the long-term safety and effectiveness of migalastat HCl in subjects
with Fabry disease who completed migalastat HCl treatment in a previous study.


Inclusion Criteria:

- Subject had completed treatment in a previous study of migalastat HCl

- Age 18 years or older

- Male and female participant agrees to use protocol identified acceptable contraception

- Subject is willing to provide written informed consent and authorization for use and
disclosure of Personal Health Information (PHI)

Exclusion Criteria:

- Subjects last available estimated glomerular filtration rate (eGFR) in the previous
study was <30 mL/min/1.73m2; unless there is measured GFR available within 3 months of
Baseline Visit (Visit 1), which is >30 mL/min/1.73m2

- Subject has undergone, or is scheduled to undergo kidney transplantation or is
currently on dialysis

- Subject has a documented transient ischemic attack, stroke, unstable angina, or
myocardial infarction within the 3 months before Visit 1

- Subject has clinically significant unstable cardiac disease in the opinion of the
investigator (e.g., cardiac disease requiring active management, such as symptomatic
arrhythmia, unstable angina, or NYHA class III or IV congestive heart failure)

- Subject has a history of allergy or sensitivity to AT1001 (including excipients) or
other iminosugars (e.g., miglustat, miglitol)

- Subject requires treatment with Glyset® (miglitol) or Zavesca® (miglustat)

- Subjects with severe or unsuitable concomitant medical condition

- Subjects with clinically significant abnormal laboratory value(s) and clinically
significant electrocardiogram (ECG) findings at baseline
We found this trial at
9
sites
3901 Rainbow Blvd
Kansas City, Kansas 66160
(913) 588-5000
Phone: 913-588-6074
University of Kansas Medical Center The University of Kansas Medical Center serves Kansas through excellence...
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3500 Gaston Avenue
Dallas, Texas 75246
1.800.422.9567
Phone: 214-820-4533
Baylor University Medical Center Baylor University Medical Center in Dallas, TX is ranked nationally in...
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4401 Penn Avenue
Pittsburgh, Pennsylvania 15224
412-692-5325
Phone: 412-692-7746
Children's Hospital of Pittsburgh of UPMC UPMC is one of the leading nonprofit health systems...
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Decatur, Georgia 30033
Phone: 404-778-8565
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Decatur, GA
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Fairfax, Virginia 22031
Phone: 301-451-0900
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Fairfax, VA
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Grand Rapids, Michigan 49525
Phone: 616-954-0600
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Grand Rapids, MI
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New York, New York 10016
Phone: 212-263-8344
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New York, NY
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Pilar,
Phone: 02322482831
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Pilar,
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Portland, Oregon 97239
Phone: 503-494-2604
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Portland, OR
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