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Open-Label Phase 3 Long-Term Safety Study of Migalastat (AT1001-041)

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. Identifier: NCT01458119
Recruitment Status : Completed
First Posted : October 24, 2011
Last Update Posted : January 16, 2018
Information provided by (Responsible Party):
Amicus Therapeutics

Brief Summary:
This is a long term, open label study of migalastat HCl (AT1001) in subjects with Fabry disease who completed treatment in a previous monotherapy trial with migalastat hydrochloride (HCl).

Condition or disease Intervention/treatment Phase
Fabry Disease Drug: migalastat HCl 150mg Phase 3

Detailed Description:
AT1001-041 is an open-label, non-comparative, multicenter, long term extension study for subjects with Fabry disease who have completed treatment in a previous trial of migalastat HCl (also known as AT1001) given as monotherapy. This is an extension study designed to evaluate the long-term safety and efficacy of migalastat HCl for the treatment of Fabry disease. Study visits will occur every 6 months to evaluate safety and assess efficacy. Visit evaluations will include physical examination, clinical laboratory parameters, adverse events and patient reported outcomes.

Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 85 participants
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: An Open-Label Extension Study to Evaluate the LongTerm Safety and Efficacy of Migalastat Hydrochloride Monotherapy in Subjects With Fabry Disease
Study Start Date : October 2011
Actual Primary Completion Date : February 2016
Actual Study Completion Date : February 2016

Resource links provided by the National Library of Medicine

Arm Intervention/treatment
Experimental: migalastat HCl 150 mg
Migalastat HCl s provided in 14-day supply blister packs. Migalastat HCl is taken every other day by mouth. An inactive reminder capsule is taken taken on the days between migalastat HCl.
Drug: migalastat HCl 150mg
Provided in 14-day supply blister packs. Migalastat HCl is taken every other day by mouth. An inactive reminder capsule is taken taken on the days between migalastat HCl.
Other Names:
  • AT1001
  • Galafold

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

  2. Withdrawal from treatment due to AEs [ Time Frame: Up to 5 years ]
    including non-serious and serious AEs

  3. Change from baseline in vital signs [ Time Frame: Up to 5 years ]
    blood pressure, heart rate

  4. Change from baseline in laboratory parameters [ Time Frame: Up to 5 years ]
    blood chemistry, hematology, urinalysis

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

  6. Change from baseline in echocardiography (ECHO) [ Time Frame: Up to 5 years ]
    ECHO ultrasound

Secondary Outcome Measures :
  1. Estimated glomerular filtration rate [ Time Frame: Up to 5 years ]
    based on the Modification of Diet in Renal Disease equation

  2. Measurement of 24-hour urine [ Time Frame: Up to 5 years ]
    protein, albumin, creatinine and globotriaosylceramide (GL-3)

  3. Evaluation of left ventricular mass index and ejection fraction [ Time Frame: Up to 5 years ]
    as measured by echocardiography

  4. Evaluation of leukocyte alpha-galactosidase A (a-Gal A) activity [ Time Frame: Up to 5 years ]
    enzyme responsible for breaking down GL-3

  5. Evaluation of patient reported assessment of pain [ Time Frame: Up to 5 years ]
    as assessed by the Brief Pain Inventory short form

  6. Evaluation of patient reported Quality of Life [ Time Frame: Up to 5 years ]
    as assessed by the Short Form-36 survey

Information from the National Library of Medicine

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Ages Eligible for Study:   16 Years and older   (Child, Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

Inclusion Criteria:

  • Completed migalastat HCl treatment in a previous Fabry disease protocol
  • Both: both female and male participants are being studied
  • Ages 16 years or older
  • Males and females must agree to use protocol identified acceptable contraception

Exclusion Criteria:

  • Estimated glomerular filtration rate 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
  • Has undergone, or is scheduled to undergo kidney transplantation or is currently on dialysis
  • Pregnancy or breast feeding
  • Treated with another investigational drug (except migalastat HCl) 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
  • Has documented transient ischemic attack, stroke, unstable angina, or myocardial infarction within the 12 months before Visit 1
  • Has clinically significant unstable cardiac disease in the opinion of the investigator
  • Has history of allergy or sensitivity to AT1001 (including excipients) or other iminosugars
  • Requires treatment with Glyset, (miglitol), or Zavesca (miglustat)
  • Has any intercurrent illness or condition that may preclude the subject from fulfilling the protocol requirements
  • Patients with severe or unsuitable concomitant medical condition
  • Patients with clinically significant abnormal laboratory value(s) and clinically significant electrocardiogram (ECG) findings at baseline.

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 identifier (NCT number): NCT01458119

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Sponsors and Collaborators
Amicus Therapeutics
Study Director: Medical Monitor, Clinical Research Amicus Therapeutics

Publications automatically indexed to this study by Identifier (NCT Number):
Responsible Party: Amicus Therapeutics Identifier: NCT01458119     History of Changes
Other Study ID Numbers: AT1001-041
First Posted: October 24, 2011    Key Record Dates
Last Update Posted: January 16, 2018
Last Verified: January 2018

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

Additional relevant MeSH terms:
Fabry Disease
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
Lipid Metabolism, Inborn Errors
Lysosomal Storage Diseases
Metabolic Diseases
Lipid Metabolism Disorders
Antiviral Agents
Anti-Infective Agents
Enzyme Inhibitors
Molecular Mechanisms of Pharmacological Action