Replagal Enzyme Replacement Therapy for Children With Fabry Disease

Completed · Phase 2

Conditions studied: Fabry Disease

In brief

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)

Key facts

Study ID

NCT00084084

Run by

Shire

People needed

17

Starts

2004-06-10

Expected to finish

2011-06-15

Last updated by the study team

2021-07-30

Who can join

Age: 7 and older, up to 17. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• 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.
• 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.
• 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).

You may not qualify if…

• 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.

Where it is running

• Tucson Access Center of Arizona Kidney Disease Hypertension Center — Tucson, Arizona, United States
• University of Arizona Health Sciences Center — Tucson, Arizona, United States
• Children's Physician Group — Palm Beach Gardens, Florida, United States
• Christus St. Patrick Hospital — Lake Charles, Louisiana, United States
• Clinical Center, National Institutes of Health — Bethesda, Maryland, United States
• Memorial Hospital — Easton, Maryland, United States
• St. Louis Children's Hospital — St Louis, Missouri, United States
• NYU School of Medicine — New York, New York, United States
• Sacred Heart Hospital — Allentown, Pennsylvania, United States
• East Tennessee Children's Hospital — Knoxville, Tennessee, United States
• University of Tennessee, Health Science Center — Memphis, Tennessee, United States
• Institute of Metabolic Diseases — Dallas, Texas, United States
• Office of Michael Cohen — Stafford, Virginia, United States
• The Hospital for Sick Children — Toronto, Ontario, Canada

Full record on ClinicalTrials.gov

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