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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: |
NCT01363492 |
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Date of registration:
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31/03/2011 |
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Prospective Registration:
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Yes |
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Primary sponsor: |
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Public title:
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Safety Study of Replagal® Therapy in 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 Are Naive to Enzyme Replacement Therapy |
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Date of first enrolment:
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May 2011 |
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Target sample size:
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15 |
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Recruitment status: |
Completed |
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URL:
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https://clinicaltrials.gov/show/NCT01363492 |
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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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United States
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Contacts
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Name:
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Suma P. Shankar, MD, PhD |
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Address:
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Telephone:
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Email:
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Affiliation:
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Emory Division of Medical Genetics |
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Name:
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Ozlem Goker-Alpan, MD |
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Address:
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Telephone:
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Email:
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Affiliation:
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O & O Alpan LLC |
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Name:
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Raphael Schiffmann, MD |
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Address:
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Telephone:
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Email:
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Affiliation:
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Baylor Health Care System |
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Name:
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Nicola Longo, MD, PhD |
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Address:
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Telephone:
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Email:
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Affiliation:
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University of Utah Hospital |
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Name:
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Marie T. McDonald, MD |
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Address:
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Telephone:
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Email:
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Affiliation:
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Duke University |
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Key inclusion & exclusion criteria
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Inclusion Criteria:
Patients must meet all of the following criteria to be enrolled in this study.
1. All patients must be diagnosed with Fabry disease by the following criteria:
- Male Patients: The patient is a hemizygous male with Fabry disease as confirmed
by a deficiency of alfa-galactosidase A activity measured in serum, leukocytes,
or fibroblasts or has a confirmed mutation of the alfa-galactosidase-A gene.
- Female Patients: The patient is a heterozygous female with Fabry disease as
confirmed by a mutation of the alfa-galactosidase A gene.
Note: If the diagnosis of Fabry disease is previously documented in the patient's
medical record, screening tests do not need to be repeated.
2. The patient is 7 to <18 years of age
3. The patient is ERT-naïve
4. 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
5. The minor child must assent to participate in the protocol and the parent(s) or
legally authorized representative(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 legally authorized representative(s)
Exclusion Criteria:
Patients who meet any of the following criteria will be excluded from the study.
1. Patient and/or the patient's parent(s) or legally authorized representative(s) are
unable to understand the nature, scope, and possible consequences of the study
2. Patient is unable to comply with the protocol, eg, uncooperative with protocol
schedule, refusal to agree to all of the study procedures, inability to return for
evaluations, or is otherwise unlikely to complete the study, as determined by the
Investigator or the medical monitor.
3. Otherwise unsuitable for the study, in the opinion of the Investigator.
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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Biological: Replagal (agalsidase alfa)
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Primary Outcome(s)
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Change From Baseline in Heart Rate Variability Parameter rMSSD
[Time Frame: Baseline to week 55]
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Change From Baseline in Heart Rate Variability Parameter SDNN
[Time Frame: Baseline to week 55]
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Development of IgG Anti-Agalsidase Alfa Antibody
[Time Frame: Baseline to Week 55]
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Change From Baseline in Heart Rate Variability Parameter pNN50
[Time Frame: Baseline to week 55]
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Number of Treatment Emergent Adverse Event (TEAE)
[Time Frame: Baseline to week 55]
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Number of Serious Adverse Event (SAE)
[Time Frame: Baseline to week 55]
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Secondary Outcome(s)
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Change From Baseline in MFS
[Time Frame: Baseline to week 55]
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Change From Baseline in Plasma Gb3
[Time Frame: Baseline to week 55]
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Change From Baseline in Urine Gb3
[Time Frame: Baseline to week 55]
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Change From Baseline in LVMI
[Time Frame: Baseline to week 55]
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Secondary ID(s)
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HGT-REP-084
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Source(s) of Monetary Support
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Please refer to primary and secondary sponsors
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