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

This study is currently recruiting participants.
Verified February 2014 by Amicus Therapeutics
Sponsor:
Information provided by (Responsible Party):
Amicus Therapeutics
ClinicalTrials.gov Identifier:
NCT01458119
First received: October 20, 2011
Last updated: February 6, 2014
Last verified: February 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
Please refer to this study by its ClinicalTrials.gov identifier: NCT01458119

  Show 28 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: February 6, 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
Sphingolipidoses
Lysosomal Storage Diseases, Nervous System
Brain Diseases, Metabolic, Inborn
Brain Diseases, Metabolic
Brain Diseases
Central Nervous System Diseases
Nervous System Diseases
Genetic Diseases, X-Linked
Genetic Diseases, Inborn
Metabolism, Inborn Errors
Lipidoses
Lipid Metabolism, Inborn Errors
Lysosomal Storage Diseases
Metabolic Diseases
Lipid Metabolism Disorders

ClinicalTrials.gov processed this record on April 16, 2014