Open Label Long-term Safety Study of AT1001 in Patients With Fabry Disease Who Have Completed a Previous AT1001 Study

This study has been completed.
Sponsor:
Information provided by (Responsible Party):
Amicus Therapeutics
ClinicalTrials.gov Identifier:
NCT00526071
First received: September 5, 2007
Last updated: December 19, 2013
Last verified: December 2013
  Purpose

This is a long term open label study of AT1001 in patients with Fabry disease previously enrolled in a Phase 2 AT1001 trial.


Condition Intervention Phase
Fabry Disease
Drug: AT1001
Phase 2

Study Type: Interventional
Study Design: Allocation: Non-Randomized
Endpoint Classification: Safety Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
Official Title: Open-label Extension Study to Evaluate the Long-term Safety, Tolerability and Pharmacodynamics of AT1001 in Patients With Fabry Disease

Resource links provided by NLM:


Further study details as provided by Amicus Therapeutics:

Primary Outcome Measures:
  • Long term safety and outcomes measures assessed at each visit. [ Time Frame: Long term ] [ Designated as safety issue: Yes ]

Enrollment: 23
Study Start Date: September 2007
Study Completion Date: October 2012
Primary Completion Date: October 2012 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: 1
AT1001 dose group 1
Drug: AT1001
AT1001

Detailed Description:

This is a long term open label study of AT1001 in patients with Fabry disease previously enrolled in a Phase 2 AT1001 trial. Patients will return to the study center approximately every 3 months for safety evaluations including physical exam, blood and urine tests.

  Eligibility

Ages Eligible for Study:   18 Years to 65 Years
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  1. Must have completed another Phase 2 trial of AT1001 in Fabry Disease
  2. Women of childbearing potential must have negative pregnancy result
  3. Male and Female subjects agree to use reliable method of contraception during treatment and four weeks after treatment has completed.
  4. Subject is willing and able to provide written informed consent

Exclusion Criteria:

  1. Subject did not complete Phase 2 trial of AT1001 in Fabry Disease
  2. Has undergone or is scheduled to undergo kidney transplantation or is currently on dialysis
  3. Has been treated with another investigational drug (except AT1001) within 30 days of study start
  Contacts and Locations
Please refer to this study by its ClinicalTrials.gov identifier: NCT00526071

Locations
United States, Georgia
Emory University
Decatur, Georgia, United States, 30033
United States, New York
New York University
New York, New York, United States, 10016
United States, Texas
Baylor Research Institute
Dallas, Texas, United States, 75226
Australia, Victoria
Royal Melbourne Hospital
Parkville, Victoria, Australia, 3050
Brazil
Hospital De Clinicas
Porto Alegre, Brazil, 90035-003
France
Hopital Europeen Georges Pompidou
Paris, France, 75015
United Kingdom
The Royal Free Hospital
London, United Kingdom, WC1N 3BG
Hope Hospital Salford
Manchester, United Kingdom, M6 8HD
Sponsors and Collaborators
Amicus Therapeutics
Investigators
Study Director: Medical Director Amicus Therapeutics
  More Information

No publications provided

Responsible Party: Amicus Therapeutics
ClinicalTrials.gov Identifier: NCT00526071     History of Changes
Other Study ID Numbers: FAB-CL-205
Study First Received: September 5, 2007
Last Updated: December 19, 2013
Health Authority: United States: Food and Drug Administration
Australia: Department of Health and Ageing Therapeutic Goods Administration
Brazil: Ministry of Health
France: Afssaps - Agence française de sécurité sanitaire des produits de santé (Saint-Denis)
United Kingdom: Medicines and Healthcare Products Regulatory Agency

Keywords provided by Amicus Therapeutics:
Fabry Disease
Lysosomal Storage Disorder

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 17, 2014