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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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12 December 2020 |
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Main ID: |
NCT00568698 |
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Date of registration:
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04/12/2007 |
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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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A Pilot Therapeutic Trial Using Hydroxyurea in Type I Spinal Muscular Atrophy Patients
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Scientific title:
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A Pilot Therapeutic Trial Using Hydroxyurea in Type I Spinal Muscular Atrophy Patients |
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Date of first enrolment:
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January 2004 |
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Target sample size:
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29 |
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Recruitment status: |
Completed |
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URL:
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https://clinicaltrials.gov/show/NCT00568698 |
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Study type:
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Interventional |
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Study design:
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Allocation: Randomized. Intervention model: Single Group Assignment. Primary purpose: Treatment. Masking: Double.
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Phase:
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Phase 1/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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Dr Ching H. Wang |
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Address:
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Telephone:
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Email:
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Affiliation:
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Stanford University |
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Key inclusion & exclusion criteria
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Inclusion Criteria:1. Laboratory confirmation of a homozygous deletion or mutation of the
SMN1 gene 2. Clinical Diagnosis of Type I SMA (never achieved independent sitting) 3. Onset
of disease before the age of 6 months 4. Enrollment in study within 6 months of diagnosis
Exclusion Criteria:1. Known hematological disorders, such as chronic anemia (defined as
platelet count less than 100,000/mm^3) in two contiguous measures in two weeks 2. Severe
systemic disorders such as congenital heart disease, other major birth defects involving
internal organs, or severe birth asphyxia 3. Participation in SMA clinical trials for other
experimental drugs 4. Requiring continuous respiratory support before the initiation of HU
treatment
Age minimum:
N/A
Age maximum:
2 Years
Gender:
All
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Health Condition(s) or Problem(s) studied
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Muscular Atrophy, Spinal
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Intervention(s)
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Drug: Hydroxyurea
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Drug: Placebo to match hydroxyurea
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Primary Outcome(s)
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Safety: Frequency of Adverse Events/Lab Abnormalities
[Time Frame: Up to 8 years, 1 month]
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Efficacy: Length of Survival (LOS) and Age of Ventilator Dependence (AVD)
[Time Frame: Up to 8 years, 1 month]
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Secondary Outcome(s)
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Motor Unit Number Estimation (MUNE)
[Time Frame: Up to 8 years, 1 month]
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Biomarker Assays: SMN Protein and SMN mRNA
[Time Frame: Up to 8 years, 1 month]
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Secondary ID(s)
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SU-11012007-783
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78811
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Source(s) of Monetary Support
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Please refer to primary and secondary sponsors
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