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

This study is ongoing, but not recruiting participants.
Sponsor:
ClinicalTrials.gov Identifier:
NCT02194985
First Posted: July 21, 2014
Last Update Posted: October 2, 2017
The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
Information provided by (Responsible Party):
Amicus Therapeutics
  Purpose

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.


Condition Intervention Phase
Fabry Disease Drug: migalastat HCl 150 mg Phase 3

Access to an investigational treatment associated with this study is available outside the clinical trial.   More info ...

Study Type: Interventional
Study Design: Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: An Open-Label Extension Study to Evaluate the Long Term Safety and Efficacy of Migalastat Hydrochloride Monotherapy in Subjects With Fabry Disease

Resource links provided by NLM:


Further study details as provided by Amicus Therapeutics:

Primary Outcome Measures:
  • Incidence of adverse events (AEs) [ Time Frame: up to 5 years ]
    non-serious AEs, serious AEs., possible suicidality related AEs

  • Withdrawal due to AEs [ Time Frame: up to 5 years ]
    including non-serious and serious AEs

  • Vital signs and body weight - change in baseline in vital signs and body weight [ Time Frame: up to 5 years ]
    blood pressure and heart rate

  • Change from baseline in laboratory parameters [ Time Frame: up to 5 years ]
    hematology, chemistry, and urinalysis

  • Change from baseline in electrocardiogram (ECG) [ Time Frame: up to 5 years ]
    12-lead ECG

  • Change from baseline in echocardiography [ Time Frame: up to 5 years ]
    echocardiography ultrasound


Secondary Outcome Measures:
  • Estimated glomerular filtration rate (eGFR) [ Time Frame: up to 5 years ]
    based on the Modification of Diet in Renal Disease (MDRD) equation and CKD-EPI equation

  • Measurement of left ventricular mass (LVM) and left ventricular mass index (LVMi) [ Time Frame: up to 5 years ]
    as measured by echocardiography

  • Ejection fraction [ Time Frame: up to 5 years ]
    as measured by echocardiography

  • Fractional shortening [ Time Frame: up to 5 years ]
    as assessed by echocardiography

  • Measurement of LV internal dimension (LVIDd and LVIDs) and wall thickness [ Time Frame: up to 5 years ]
    as assessed by echocardiography

  • Measurement of white blood cell (WBC) α-Gal A activity [ Time Frame: up to 5 years ]
    enzyme responsible for breaking down GL-3

  • Measurement of subject reported Quality of Life [ Time Frame: up to 5 years ]
    as assessed by the Short Form - 36 Survey (SF-36)

  • Measurement of 24-hour urine protein [ Time Frame: up to 5 years ]
  • Plasma lyso-Gb3 [ Time Frame: up to 5 years ]
    biomarker shown to be elevated in Fabry subjects


Enrollment: 76
Study Start Date: October 2014
Estimated Study Completion Date: October 2019
Estimated Primary Completion Date: October 2019 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: migalastat HCl 150 mg
Migalastat HCl is a capsule provided in 14-day supply blister packs. Migalastat HCl is taken every other day by mouth. An inactive reminder capsule is taken or a punch-out reminder is used on days between migalastat HCl.
Drug: migalastat HCl 150 mg
Capsule provided in 14-day supply blister packs. Migalastat HCl is taken every other day by mouth. An inactive reminder capsule is taken or a punch-out reminder is used on the days between migalastat HCl.
Other Name: AT1001

  Eligibility

Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.


Ages Eligible for Study:   18 Years and older   (Adult, Senior)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

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
  Contacts and Locations
Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02194985


  Hide Study Locations
Locations
United States, Georgia
Emory University
Decatur, Georgia, United States, 30033
United States, Kansas
University of Kansas Medical Center
Kansas City, Kansas, United States, 66160
United States, Michigan
Infusion Associates N.E.
Grand Rapids, Michigan, United States, 49525
United States, New York
NYU School of Medicine
New York, New York, United States, 10016
United States, Oregon
Oregon Health and Sciences University Hospital
Portland, Oregon, United States, 97239
United States, Pennsylvania
Children's Hospital of Pittsburgh of UPMC
Pittsburgh, Pennsylvania, United States, 15224
United States, Texas
Baylor University Medical Center
Dallas, Texas, United States, 75226
United States, Virginia
O & O Alpan, LLC
Fairfax, Virginia, United States, 22030
Argentina
Hospital Universitario Austral
Pilar, Argentina, B1629ODT
Australia, Victoria
Royal Melbourne Hospital
Parkville, Victoria, Australia, 3050
Australia
Women's and Children's Hospital
North Adelaide, Australia, 5006
Austria
Medizinische Universitat Wien
Wien, Austria, 1090
Belgium
Antwerpen Koningin Paola Kinderziekenhuis
Edegem, Belgium, 2650
Brazil
Hospital de Clinicas de Porto Alegre
Porto Alegre, Brazil, 90035-001
Canada, Quebec
Hospital du Sacre-Coeur de Montreal
Montreal, Quebec, Canada, H4J 1C5
Denmark
Rigshospitalet
Copenhagen, Denmark, 2100
Egypt
Kasr El Ainy Hospital
Cairo, Egypt, 11451
France
Hopital Raymond Poincare
Garches, France, 92380
Hôpital Claude Huriez, CHRU Lille
Lille, France, 59037
Italy
Azienda Ospedaliera Universitaria Careggi
Firenze, Italy, 50134
Policlinico Universitario A. Gemelli
Roma, Italy, 00168
Japan
Osaka University Hospital
Suita, Osaka, Japan, 565-0871, 27
Niigata University Medical & Dental Hospital
Niigata, Japan, 951-8520, 15
Osaka City University Hospital
Osaka-shi, Japan, 545-8586, 27
Jikei University Hospital
Tokyo, Japan, 105-8471-13
Spain
Fundacion Puigvert
Barcelona, Spain, 08025
Turkey
Gazi University Medical Faculty Gazi
Ankara, Turkey, 06500
United Kingdom
Royal Free Hospital
London, United Kingdom, NW3 2QG
Charles Dent Metabolic Unit
London, United Kingdom, WC1N3BG
Sponsors and Collaborators
Amicus Therapeutics
Investigators
Study Director: Medical Monitor, Clinical Research, MD Amicus Therapeutics, Inc.
  More Information

Responsible Party: Amicus Therapeutics
ClinicalTrials.gov Identifier: NCT02194985     History of Changes
Other Study ID Numbers: AT1001-042
First Submitted: July 17, 2014
First Posted: July 21, 2014
Last Update Posted: October 2, 2017
Last Verified: November 2016

Keywords provided by Amicus Therapeutics:
Fabry disease
Lysosomal storage disease
migalastat HCl
AT1001

Additional relevant MeSH terms:
Fabry Disease
Sphingolipidoses
Lysosomal Storage Diseases, Nervous System
Brain Diseases, Metabolic, Inborn
Brain Diseases, Metabolic
Brain Diseases
Central Nervous System Diseases
Nervous System Diseases
Cerebral Small Vessel Diseases
Cerebrovascular Disorders
Vascular Diseases
Cardiovascular Diseases
Genetic Diseases, X-Linked
Genetic Diseases, Inborn
Metabolism, Inborn Errors
Lipidoses
Lipid Metabolism, Inborn Errors
Lysosomal Storage Diseases
Metabolic Diseases
Lipid Metabolism Disorders
1-Deoxynojirimycin
Antiviral Agents
Anti-Infective Agents
Enzyme Inhibitors
Molecular Mechanisms of Pharmacological Action