Open Label Long-term Safety Study of AT1001 in Patients With Fabry Disease Who Have Completed a Previous AT1001 Study
This study is ongoing, but not recruiting participants.
Sponsor:
Amicus Therapeutics
Collaborator:
GlaxoSmithKline
Information provided by (Responsible Party):
Amicus Therapeutics
ClinicalTrials.gov Identifier:
NCT00526071
First received: September 5, 2007
Last updated: August 29, 2011
Last verified: August 2011
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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:
Genetics Home Reference related topics:
Chanarin-Dorfman syndrome
cholesteryl ester storage disease
Fabry disease
Farber lipogranulomatosis
Schindler disease
succinic semialdehyde dehydrogenase deficiency
U.S. FDA Resources
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 ]
| Estimated Enrollment: | 27 |
| Study Start Date: | September 2007 |
| Estimated Study Completion Date: | December 2012 |
| Estimated Primary Completion Date: | December 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:
- Must have completed another Phase 2 trial of AT1001 in Fabry Disease
- Women of childbearing potential must have negative pregnancy result
- Male and Female subjects agree to use reliable method of contraception during treatment and four weeks after treatment has completed.
- Subject is willing and able to provide written informed consent
Exclusion Criteria:
- Subject did not complete Phase 2 trial of AT1001 in Fabry Disease
- Has undergone or is scheduled to undergo kidney transplantation or is currently on dialysis
- 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
GlaxoSmithKline
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: | August 29, 2011 |
| 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 June 18, 2013