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

This study is currently recruiting participants. (see Contacts and Locations)
Verified May 2014 by Amicus Therapeutics
Sponsor:
Information provided by (Responsible Party):
Amicus Therapeutics
ClinicalTrials.gov Identifier:
NCT01458119
First received: October 20, 2011
Last updated: May 2, 2014
Last verified: May 2014
  Purpose

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

Study Type: Interventional
Study Design: Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: 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

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 ] [ Designated as safety issue: No ]
    non-serious AEs, serious AEs, and possible suicidality related AEs

  • Withdrawal from treatment due to AEs [ Time Frame: Up to 5 years ] [ Designated as safety issue: No ]
    including non-serious and serious AEs

  • Change from baseline in vital signs [ Time Frame: Up to 5 years ] [ Designated as safety issue: No ]
    blood pressure, heart rate

  • Change from baseline in laboratory parameters [ Time Frame: Up to 5 years ] [ Designated as safety issue: No ]
    blood chemistry, hematology, urinalysis

  • Change from baseline in electrocardiogram (ECG) [ Time Frame: Up to 5 years ] [ Designated as safety issue: No ]
    12-lead ECG

  • Change from baseline in echocardiography (ECHO) [ Time Frame: Up to 5 years ] [ Designated as safety issue: No ]
    ECHO ultrasound


Secondary Outcome Measures:
  • Estimated glomerular filtration rate [ Time Frame: Up to 5 years ] [ Designated as safety issue: No ]
    based on the Modification of Diet in Renal Disease equation

  • Measurement of 24-hour urine [ Time Frame: Up to 5 years ] [ Designated as safety issue: No ]
    protein, albumin, creatinine and globotriaosylceramide (GL-3)

  • Evaluation of left ventricular mass index and ejection fraction [ Time Frame: Up to 5 years ] [ Designated as safety issue: No ]
    as measured by echocardiography

  • Evaluation of leukocyte alpha-galactosidase A (a-Gal A) activity [ Time Frame: Up to 5 years ] [ Designated as safety issue: No ]
    enzyme responsible for breaking down GL-3

  • Evaluation of patient reported assessment of pain [ Time Frame: Up to 5 years ] [ Designated as safety issue: No ]
    as assessed by the Brief Pain Inventory short form

  • Evaluation of patient reported Quality of Life [ Time Frame: Up to 5 years ] [ Designated as safety issue: No ]
    as assessed by the Short Form-36 survey


Estimated Enrollment: 100
Study Start Date: October 2011
Estimated Study Completion Date: July 2016
Estimated Primary Completion Date: July 2016 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: migalstat HCL 150mg
Migalastat is 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 Name: AT1001

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.

  Eligibility

Ages Eligible for Study:   16 Years and older
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

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.
  Contacts and Locations
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, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT01458119

  Show 34 Study Locations
Sponsors and Collaborators
Amicus Therapeutics
Investigators
Study Director: Medical Monitor, Clinical Research Amicus Therapeutics
  More Information

No publications provided

Responsible Party: Amicus Therapeutics
ClinicalTrials.gov Identifier: NCT01458119     History of Changes
Other Study ID Numbers: AT1001-041
Study First Received: October 20, 2011
Last Updated: May 2, 2014
Health Authority: Spain: Agencia Española de Medicamentos y Productos Sanitarios
Belgium: Federal Agency for Medicines and Health Products, FAMHP
United States: Food and Drug Administration
Denmark: Danish Medicines Agency
Brazil: National Health Surveillance Agency
France: Agence Nationale de Sécurité du Médicament et des produits de santé
United Kingdom: Medicines and Healthcare Products Regulatory Agency
Australia: Department of Health and Ageing Therapeutic Goods Administration
Canada: Health Canada
Turkey: Ministry of Health
Italy: Ministry of Health
Austria: Austrian Medicines and Medical Devices Agency
Argentina: Administracion Nacional de Medicamentos, Alimentos y Tecnologia Medica

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

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

ClinicalTrials.gov processed this record on October 29, 2014