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Main
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Note: This record shows only 22 elements of the WHO Trial Registration Data Set. To view changes that have been made to the source record, or for additional information about this trial, click on the URL below to go to the source record in the primary register. |
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Register:
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ClinicalTrials.gov |
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Last refreshed on:
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19 October 2017 |
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Main ID: |
NCT00084084 |
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Date of registration:
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05/06/2004 |
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Prospective Registration:
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No |
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Primary sponsor: |
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Public title:
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Replagal Enzyme Replacement Therapy for Children With Fabry Disease
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Scientific title:
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An Open Label Clinical Trial of Replagal Enzyme Replacement Therapy In Children With Fabry Disease Who Have Completed Study TKT023 or Who Are Naive to Enzyme Replacement Therapy |
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Date of first enrolment:
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June 2004 |
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Target sample size:
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17 |
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Recruitment status: |
Completed |
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URL:
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https://clinicaltrials.gov/show/NCT00084084 |
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Study type:
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Interventional |
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Study design:
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Phase:
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Phase 2
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Countries of recruitment
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Canada
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United States
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Contacts
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Name:
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Ray Pais, MD, FAAP |
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Address:
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Affiliation:
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East Tennessee Children's Hospital |
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Name:
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Gregory M. Pastores, MD |
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Address:
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Affiliation:
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New York University School of Medicine |
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Name:
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Lynda Bideau, MD |
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Address:
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Affiliation:
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Children's Physicians Group |
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Name:
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Brian J. Corden, MD, PhD |
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Address:
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Email:
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Affiliation:
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Memorial hospital |
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Name:
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Alison Whelan, MD |
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Address:
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Affiliation:
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St. Louis Children's Hospital |
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Name:
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Rick A. Martin, MD |
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Address:
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Affiliation:
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St. Louis Children's Hospital |
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Name:
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Karen L. Johnson, MD, MPH |
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Address:
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Affiliation:
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University of Tennessee Health Science Center |
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Name:
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Yang-Tze Yoko Broussard, MD |
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Address:
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Affiliation:
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Christus St. Patrick Hospital |
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Name:
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Raphael Schiffman, MD |
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Address:
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Affiliation:
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Institute of Metabolic Disease, Baylor Research Institute |
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Name:
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Michael E. Cohen, MD |
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Address:
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Email:
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Affiliation:
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Office of Michael Cohen |
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Name:
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Joe T. Clarke, MD, PhD |
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Address:
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Email:
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Affiliation:
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The Hospital for Sick Children |
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Name:
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Victoria Castaneda, MD |
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Address:
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Email:
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Affiliation:
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East Tennessee Children's Hospital |
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Name:
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Tyler Reimschisel, MD |
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Address:
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Email:
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Affiliation:
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St. Louis Children's Hospital |
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Name:
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Y. Howard Lien, MD, PhD |
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Address:
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Affiliation:
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Tuscon Access Center of Arizona Kidney Disease Hypertension Center |
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Name:
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Li-Wen Lai, PhD |
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Address:
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Affiliation:
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University of Arizona Health Sciences Center |
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Name:
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Tanya J. Lehky, MD |
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Address:
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Email:
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Affiliation:
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Clinical Center, National Institutes of Health |
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Name:
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Leslie F. Carroll, MD |
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Address:
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Email:
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Affiliation:
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Sacred Heart Hospital |
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Name:
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Manju Thomas, MD |
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Address:
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Email:
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Affiliation:
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Sacred Heart Hospital |
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Key inclusion & exclusion criteria
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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 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 Criteria:
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.
Age minimum:
7 Years
Age maximum:
17 Years
Gender:
All
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Health Condition(s) or Problem(s) studied
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Fabry Disease
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Intervention(s)
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Drug: Agalsidase alfa
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Primary Outcome(s)
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Patients Who Experienced At Least One Adverse Event (AE)
[Time Frame: 362 weeks]
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Secondary Outcome(s)
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Pharmacokinetics - Maximum Observed Serum Concentration (Cmax)
[Time Frame: 341 weeks]
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Pharmacokinetics - Area Under the Serum Concentration-Time Curve (AUC0-8)
[Time Frame: 341 weeks]
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Source(s) of Monetary Support
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Please refer to primary and secondary sponsors
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