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Main
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Note: This record shows only 22 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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12 December 2020 |
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Main ID: |
NCT00283959 |
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Date of registration:
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27/01/2006 |
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Prospective Registration:
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Yes |
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Primary sponsor: |
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Public title:
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A 12-Week Safety and Pharmacodynamic Study of AT1001 (Migalastat Hydrochloride) in Participants With Fabry Disease
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Scientific title:
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A Phase 2, Open-Label, Single Dose Level, 12-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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June 27, 2006 |
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Target sample size:
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4 |
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Recruitment status: |
Completed |
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URL:
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https://clinicaltrials.gov/show/NCT00283959 |
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Study type:
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Interventional |
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Study design:
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Allocation: N/A. Intervention model: Single Group Assignment. Primary purpose: Treatment. Masking: None (Open Label).
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Phase:
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Phase 2
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Countries of recruitment
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Australia
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Brazil
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Contacts
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Name:
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Medical Monitor, Clinical Research |
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Address:
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Telephone:
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Email:
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Affiliation:
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Amicus Therapeutics |
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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
- Had a confirmed diagnosis of Fabry disease with a documented missense gene mutation
(individual or familial)
- Had enhanceable enzyme activity based on in vitro tests
- Had documented evidence of cardiac and/or renal dysfunction (for example, abnormal
electrocardiogram (ECG), left ventricular hypertrophy, renal insufficiency) and/or
cerebral tissue dysfunction documented by evidence of stroke and/or peripheral nervous
tissue dysfunction (for example, intolerance to heat/cold, decrease of perspiration)
- Must have been previously untreated by enzyme replacement therapy (ERT) or substrate
depletion for Fabry disease or were able to stop ERT for at least 18 weeks or up to
three months, and be willing to undergo two kidney and three skin biopsies
- Agreed 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
- Were 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 >2 mg per deciliter on Day -2
- Screening 12-lead ECG demonstrating corrected QT interval >450 milliseconds prior to
dosing
- Pacemaker or other contraindication for magnetic resonance imaging (MRI) scanning
- Took any of the following prohibited medications: Fabrazyme® (agalsidase beta),
Replagal™ (agalsidase alfa), Glyset® (miglitol), Zavesca® (miglustat), or any
experimental therapy for any indication
- Participated in a previous clinical trial in the last 30 days
- Any other condition, which, in the opinion of the investigator, would jeopardize the
safety of the participant 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: migalastat HCl
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Primary Outcome(s)
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Number Of Participants Who Experienced Severe Treatment-emergent Adverse Events (TEAEs)
[Time Frame: Day 1 (after dosing) through Week 48 (end of extension period)]
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Secondary Outcome(s)
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a-Galactosidase A (a-Gal A) Activity In Peripheral Blood Mononuclear Cells (PBMC) At Baseline, Week 12, And Week 48
[Time Frame: Baseline, Week 12 (end of treatment period), Week 48 (end of extension period)]
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Secondary ID(s)
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FAB-CL-202 (AA1565521)
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Source(s) of Monetary Support
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Please refer to primary and secondary sponsors
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