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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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19 February 2015 |
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Main ID: |
NCT00074971 |
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Date of registration:
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24/12/2003 |
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Prospective Registration:
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No |
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Primary sponsor: |
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Public title:
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A Study of the Safety and Efficacy of Fabrazyme in Patients With Fabry Disease
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Scientific title:
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A Multi-center, Open-Label Extension Study of the Safety and Efficacy of Recombinant Human a-Galactosidase A (r-haGAL) Replacement in Patients With Fabry Disease |
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Date of first enrolment:
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October 1999 |
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Target sample size:
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58 |
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Recruitment status: |
Completed |
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URL:
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http://clinicaltrials.gov/show/NCT00074971 |
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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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Phase:
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Phase 3
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Countries of recruitment
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France
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Netherlands
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Puerto Rico
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United Kingdom
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United States
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Contacts
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Name:
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Medical Monitor |
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Address:
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Telephone:
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Email:
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Affiliation:
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Genzyme Coorporation |
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Key inclusion & exclusion criteria
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Inclusion Criteria:
- Patients must have successfully completed the previous double-blind study
(AGAL-1-002-98)
- Patients must provide written informed consent prior to study participation
- Female patients must have a negative pregnancy test prior to each dosing and use a
medically accepted method of contraception throughout the study
Exclusion criteria:
- Patient has undergone kidney transplant or is currently on dialysis
- Patient is pregnant or lactating
- Patient is unwilling to comply with the requirements of the protocol
- Patient has a clinically significant organic disease (with the exception of symptoms
related to Fabry disease), including clinically significant cardiovascular, hepatic,
pulmonary, neurologic, or renal disease, or other medical condition, serious
intercurrent illness, or extenuating circumstances that, in the opinion of the
investigator, would preclude participation in the study
Age minimum:
16 Years
Age maximum:
N/A
Gender:
Both
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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: Fabrazyme (agalsidase beta)
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Primary Outcome(s)
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Safety and efficacy
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Morphologic assessment of GL-3 inclusions in the capillary endothelium (vasculature) of the kidney
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Secondary Outcome(s)
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Autonomic status
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Changes in McGill Pain Questionnaire
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Glomerular filtration
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Physician's assessment of Fabry Symptoms and pain medication
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SF-36 Health Survey
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Functional assessment of urinary protein excretion Ophthalmic changes
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Secondary ID(s)
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AGAL-005-99
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Source(s) of Monetary Support
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Please refer to primary and secondary sponsors
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Results
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Results available:
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Date Posted:
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Date Completed:
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URL:
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