Status:
COMPLETED
An Open-Label Clinical Trial of Replagal Enzyme Therapy in Children Ages 7-17 Years With Fabry Disease
Lead Sponsor:
National Institute of Neurological Disorders and Stroke (NINDS)
Conditions:
Fabry Disease
Eligibility:
All Genders
Phase:
PHASE2
Brief Summary
This study will evaluate the safety of multiple biweekly intravenous doses of Replagal over 26 weeks in 25 children with Fabry disease and the way in which that agent can improve the health of this pa...
Detailed Description
The objectives of this clinical trial are to evaluate: 1) the safety of multiple biweekly (i.e. every other week) intravenous (IV) doses of Replagal over 26 weeks in 25 children ages 7-17 years old wi...
Eligibility Criteria
Inclusion
- INCLUSION CRITERIA:
- Male hemizygote with Fabry Disease as documented by clinical evidence and by laboratory evidence of alpha-galactosidase A deficiency.
- Or
- Female heterozygote with Fabry Disease as documented by gene analysis showing a mutation of the alpha-galactosidase A gene. Female patients of child-bearing potential must have a negative pregnancy test at baseline and agree to the use of effective contraception such as oral contraceptive or double barrier method for study entry and while participating in the study.
- 7-17 years of age.
- Adequate general health (as determined by the investigators) to undergo the specified phlebotomy regimen and protocol related procedures.
- The child must assent to participate in the protocol and the parent(s) or legally authorized guardian(s) must have voluntarily signed an Institutional Review Board/Independent Ethics Committee (IRB/IEC) approved informed consent form after all relevant aspects of the study have been explained and discussed with the child and the child's parent(s) or legal guardian(s).
- EXCLUSION CRITERIA:
- Patient has previously participated in a multi-dose clinical study of an investigational therapeutic agent for Fabry Disease.
- Patient and/or the patient's parent(s) or legal guardian(s) are unable to understand the nature, scope, and possible consequences of the study.
- Patient is unable to comply with the protocol, e.g., uncooperative with protocol schedule, refusal to agree to all of the study procedures, inability to return for safety evaluations, or is otherwise unlikely to complete the study, as determined by the investigator or the medical monitor.
Exclusion
Key Trial Info
Start Date :
October 1 2003
Trial Type :
INTERVENTIONAL
End Date :
June 1 2005
Estimated Enrollment :
25 Patients enrolled
Trial Details
Trial ID
NCT00071877
Start Date
October 1 2003
End Date
June 1 2005
Last Update
March 4 2008
Active Locations (1)
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1
National Institute of Neurological Disorders and Stroke (NINDS)
Bethesda, Maryland, United States, 20892