Status:
COMPLETED
Replagal Enzyme Replacement Therapy for Children With Fabry Disease
Lead Sponsor:
Shire
Conditions:
Fabry Disease
Eligibility:
All Genders
7-17 years
Phase:
PHASE2
Brief Summary
Primary Objective(s): * To assess the safety of Replagal at a dose of 0.2 mg/kg administered over 40 (+/-10) minutes in children with Fabry disease * To assess the effect of Replagal on heart rate va...
Detailed Description
TKT029 is an open label multi-center study to assess the safety of enzyme replacement therapy with Replagal (agalsidase alfa) in children with Fabry disease, who have completed 6 months of agalsidase ...
Eligibility Criteria
Inclusion
- 1a. For Cohort 1 (both phases):
- \- Patients must have completed all study requirements and assessments for Study TKT023 less than 30 (+/-7) days prior to enrolling in Study TKT029 and must have no safety or medical issues that contraindicate participation.
- OR
- 1b. For Cohort 2:
- The patient is between 7 and 17 years of age at the time of informed consent, inclusive.
- The patient must be ERT-naive.
- The patient is a hemizygous male with Fabry disease as confirmed by a deficiency of alpha-galactosidase A activity measured in serum, leukocytes, or fibroblasts. Male patients who do not already have a documented deficiency of alpha-galactosidase A activity will provide a blood sample during screening for determination of alpha-galactosidase A activity level in their serum.
- OR
- \- The patient is a heterozygous female or hemizygous male with Fabry disease as confirmed by a mutation of the alpha-galactosidase A gene. Patients who do not already have a documented mutation of the alpha-galactosidase A gene will provide a blood sample during screening for genotyping.
- 2\. Adequate general health (as determined by the Investigators) to undergo the specified phlebotomy regimen and protocol-related procedures and no safety or medical contraindications for participation.
- 3\. The minor 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 concent 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
- Patients who meet any of the following criteria are not eligible for this study:
- 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.
Key Trial Info
Start Date :
June 10 2004
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
June 15 2011
Estimated Enrollment :
17 Patients enrolled
Trial Details
Trial ID
NCT00084084
Start Date
June 10 2004
End Date
June 15 2011
Last Update
July 30 2021
Active Locations (14)
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1
Tucson Access Center of Arizona Kidney Disease Hypertension Center
Tucson, Arizona, United States, 85719
2
University of Arizona Health Sciences Center
Tucson, Arizona, United States, 85724
3
Children's Physician Group
Palm Beach Gardens, Florida, United States, 33410
4
Christus St. Patrick Hospital
Lake Charles, Louisiana, United States, 70601