A 24-Week Safety and Pharmacodynamic Study of AT1001 in Patients With Fabry Disease
This study has been completed.
Sponsor:
Amicus Therapeutics
Information provided by:
Amicus Therapeutics
ClinicalTrials.gov Identifier:
NCT00283933
First received: January 27, 2006
Last updated: August 17, 2010
Last verified: August 2010
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Purpose
The purpose of this study is to collect information on the safety of AT1001 (migalastat hydrochloride) and how AT1001 works in patients with Fabry disease.
| Condition | Intervention | Phase |
|---|---|---|
|
Fabry Disease |
Drug: AT1001 (migalastat hydrochloride) |
Phase 2 |
| Study Type: | Interventional |
| Study Design: | Allocation: Non-Randomized Endpoint Classification: Safety/Efficacy Study Intervention Model: Single Group Assignment Masking: Open Label Primary Purpose: Treatment |
| Official Title: | A Phase 2, Open-Label, Single Dose Level, 24-Week Study to Evaluate the 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:
- Safety and tolerability
Secondary Outcome Measures:
- Pharmacodynamic parameters
- Functional parameters (cardiac, renal, CNS)
| Estimated Enrollment: | 8 |
| Study Start Date: | January 2006 |
| Study Completion Date: | March 2008 |
| Primary Completion Date: | March 2008 (Final data collection date for primary outcome measure) |
The purpose of this study is to determine the effect of AT1001 given orally to patients with Fabry disease. Patients will visit the clinic 4 weeks prior to dosing to determine their eligibility for the study, and then return for a second visit for baseline and safety assessments, which will include skin, cardiac, and renal biopsies. Patients will receive oral doses of AT1001 for 24 weeks and will visit the clinic 6 times, once every 4 weeks, for evaluation and tests. A skin biopsy will be repeated after 12 weeks, and then a final set of skin, cardiac, and renal biopsies, and functional assessments will be performed at the end of 24 weeks. Patients may be given the opportunity to enter a study extension phase for an additional 24 weeks, which will require two more clinic visits. All study participants will have a final follow up visit 2 weeks after the end of the study.
Eligibility| Ages Eligible for Study: | 18 Years to 65 Years |
| Genders Eligible for Study: | Male |
| Accepts Healthy Volunteers: | No |
Criteria
Inclusion Criteria:
- Males between 18 and 65 years of age (inclusive)
- Hemizygous for Fabry disease
- Have a confirmed diagnosis of Fabry disease with a documented missense gene mutation (individual or familial)
- Have enhanceable enzyme activity based on in vitro tests
- Have documented evidence of cardiac and/or renal dysfunction (e.g., abnormal ECG, left ventricular hypertrophy, renal insufficiency)
- Must be previously untreated by ERT or substrate depletion for Fabry disease, or if ERT or other specific treatment for Fabry disease was administered, must stop ERT for at least 30 weeks.
- Must be willing to undergo two kidney, two cardiac, and three skin biopsies
- Agree to be sexually abstinent or use a condom with spermicide when engaging in sexual activity during the course of the study and for a period of 30 days following their completion of the study
- Willing and able to sign an informed consent form
Exclusion Criteria:
- History of significant disease other than Fabry disease
- History of organ transplant
- Serum creatinine greater than 176 mmol/dL on Day -2
- Screening 12-lead ECG demonstrating QTc > 450 msec prior to dosing
- Pacemaker or other contraindication for MRI scanning
- Taking a medication prohibited by the protocol or any experimental therapy for any indication
- Participated in a clinical trial in the last 30 days
- Any other condition, which, in the opinion of the investigator, would jeopardize the safety of the patient or impact the validity of the study results
Contacts and Locations
Please refer to this study by its ClinicalTrials.gov identifier: NCT00283933
Locations
| Canada, Quebec | |
| Université de Montréal, Hôpital du Sacré-Coeur de Montréal | |
| Montréal, Quebec, Canada, H4J 1C5 | |
| France | |
| Hôpital Europeen Georges Pompidou | |
| Paris, France | |
| United Kingdom | |
| National Hospital for Neurology & Neurosurgery , Charles Dent Metabolic Unit | |
| London, United Kingdom, WC1N 3BG | |
| The Royal Free Hospital, Lysosomal Storage Disorders Unit, Department of Haematology | |
| London, United Kingdom, NW3 2QG | |
Sponsors and Collaborators
Amicus Therapeutics
Investigators
| Principal Investigator: | Perry Elliot, MD | London Heart Hospital |
More Information
No publications provided by Amicus Therapeutics
Additional publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):
| ClinicalTrials.gov Identifier: | NCT00283933 History of Changes |
| Other Study ID Numbers: | AA1565522 (FAB-CL-203) |
| Study First Received: | January 27, 2006 |
| Last Updated: | August 17, 2010 |
| Health Authority: | United Kingdom: Medicines and Healthcare Products Regulatory Agency France: Afssaps - Agence française de sécurité sanitaire des produits de santé (Saint-Denis) Canada: Health Canada |
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 May 22, 2013