An Open-Label Clinical Trial of Replagal Enzyme Therapy in Children Ages 7-17 Years With Fabry Disease

• Register: ClinicalTrials.gov
• Last refreshed on: 19 February 2015
• Main ID: NCT00071877
• Date of registration: 03/11/2003
• Prospective Registration: No
• Primary sponsor: National Institute of Neurological Disorders and Stroke (NINDS)
• Public title: An Open-Label Clinical Trial of Replagal Enzyme Therapy in Children Ages 7-17 Years With Fabry Disease
• Scientific title: A Clinical Trial of Replagal Enzyme Replacement Therapy in Children Ages 7 - 17 Years With Fabry Disease
• Date of first enrolment: October 2003
• Target sample size: 25
• Recruitment status: Completed
• URL: http://clinicaltrials.gov/show/NCT00071877
• Study type: Interventional
• Study design: Endpoint Classification: Safety/Efficacy Study, Primary Purpose: Treatment
• Phase: Phase 2

Countries of recruitment

United States

Key inclusion & exclusion criteria

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.



Age minimum: N/A
Age maximum: N/A
Gender: Both

Health Condition(s) or Problem(s) studied

Fabry Disease

Intervention(s)

Drug: Replagal

Secondary ID(s)

04-N-0029

040029

Source(s) of Monetary Support

Please refer to primary and secondary sponsors