Replagal Enzyme Replacement Therapy for Children With Fabry Disease (NCT00084084) | Clinical Trial Compass
CompletedPhase 2
Replagal Enzyme Replacement Therapy for Children With Fabry Disease
United States17 participantsStarted 2004-06-10
Plain-language 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 variability in patients 7 to 17 years of age
Secondary Objective(s):
* To determine the pharmacokinetics of Replagal at baseline and after the initiation of enzyme replacement therapy (ERT)
* To determine exploratory measurements of efficacy including renal function (ie, estimated glomerular filtration rate \[eGFR\] and creatinine clearance), clinical outcomes (in Cohorts 1 and 2), and sweating and left ventricular mass index (LVMI) (Cohort 1, Phase 1 only)
Who can participate
Age range7 Years – 17 Years
SexALL
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AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
Inclusion Criteria:
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 C…
What they're measuring
1
Patients Who Experienced At Least One Adverse Event (AE)