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Main
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Note: This record shows only the 20 elements of the WHO Trial Registration Data Set. To view changes that have been made to the source record, or for additional information about this trial, click on the URL below to go to the source record in the primary register. |
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Register:
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ClinicalTrials.gov |
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Last refreshed on:
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17 October 2012 |
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Main ID: |
NCT00283933 |
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Date of registration:
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27/01/2006 |
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Primary sponsor: |
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Public title:
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A 24-Week Safety and Pharmacodynamic Study of AT1001 in Patients With Fabry Disease
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Scientific title:
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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 |
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Date of first enrolment:
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January 2006 |
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Target sample size:
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8 |
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Recruitment status: |
Completed |
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URL:
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http://clinicaltrials.gov/show/NCT00283933 |
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Study type:
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Interventional |
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Study design:
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Allocation: Non-Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment
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Countries of recruitment
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Canada
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France
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United Kingdom
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Contacts
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Name:
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Perry Elliot, MD |
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Address:
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Telephone:
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Email:
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Affiliation:
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London Heart Hospital |
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Key inclusion & exclusion criteria
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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
Age minimum:
18 Years
Age maximum:
65 Years
Gender:
Male
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Health Condition(s) or Problem(s) studied
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Fabry Disease
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Intervention(s)
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Drug: AT1001 (migalastat hydrochloride)
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Primary Outcome(s)
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Safety and tolerability
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Secondary Outcome(s)
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Functional parameters (cardiac, renal, CNS)
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Pharmacodynamic parameters
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Secondary ID(s)
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AA1565522 (FAB-CL-203)
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Source(s) of Monetary Support
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Please refer to primary and secondary sponsors
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