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

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ClinicalTrials.gov Identifier: NCT00526071
Recruitment Status : Completed
First Posted : September 6, 2007
Last Update Posted : January 27, 2014
Sponsor:
Information provided by (Responsible Party):
Amicus Therapeutics

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

Condition or disease Intervention/treatment Phase
Fabry Disease Drug: AT1001 Phase 2

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.

Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 23 participants
Allocation: Non-Randomized
Intervention Model: Single Group Assignment
Masking: None (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
Study Start Date : September 2007
Primary Completion Date : October 2012
Study Completion Date : October 2012


Arm Intervention/treatment
Experimental: 1
AT1001 dose group 1
Drug: AT1001
AT1001



Primary Outcome Measures :
  1. Long term safety and outcomes measures assessed at each visit. [ Time Frame: Long term ]


Information from the National Library of Medicine

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Ages Eligible for Study:   18 Years to 65 Years   (Adult)
Sexes Eligible for Study:   All
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

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): 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

Responsible Party: Amicus Therapeutics
ClinicalTrials.gov Identifier: NCT00526071     History of Changes
Other Study ID Numbers: FAB-CL-205
First Posted: September 6, 2007    Key Record Dates
Last Update Posted: January 27, 2014
Last Verified: December 2013

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
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