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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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18 October 2021 |
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Main ID: |
NCT03570931 |
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Date of registration:
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11/06/2018 |
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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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A Study to Assess Efficacy and Safety of RT001 in Subjects With Infantile Neuroaxonal Dystrophy
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Scientific title:
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A Prospective Open-label Study to Assess Efficacy and Safety of RT001 in Subjects With Infantile Neuroaxonal Dystrophy |
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Date of first enrolment:
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November 5, 2018 |
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Target sample size:
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19 |
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Recruitment status: |
Active, not recruiting |
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URL:
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https://clinicaltrials.gov/show/NCT03570931 |
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Study type:
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Interventional |
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Study design:
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Allocation: N/A. Intervention model: Single Group Assignment. Primary purpose: Treatment. Masking: None (Open Label).
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Phase:
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Phase 2/Phase 3
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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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Peter Milner, 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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Chief Medical Officer |
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Key inclusion & exclusion criteria
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Inclusion Criteria:
1. Male or female 18 months to 10 years of age
2. Medical history consistent with the symptoms of classic INAD (onset of symptoms
between the ages of 6 months and 3 years)
3. Homozygous for PLA2G6 deficiency (variant alleles may be mixed heterozygotes)
4. Must have impairment in at least 2 of the assessed categories at baseline
5. Signed informed consent form (ICF) prior to entry into the study
6. Able to provide the necessary blood samples
Exclusion Criteria:
1. Received treatment with other experimental therapies within the last 30 days prior to
the first dose
2. Requiring mechanical ventilation, other than positive air pressure support primarily
for mitigation of sleep apnea.
3. Have a life expectancy of less than one year
4. Diagnosis of atypical NAD (ANAD)
5. Unwilling or unable to comply with the requirements of this protocol, including the
presence of any condition (physical, mental, or social) that is likely to affect the
subject's ability to return for visits as scheduled
Age minimum:
18 Months
Age maximum:
10 Years
Gender:
All
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Health Condition(s) or Problem(s) studied
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Infantile Neuroaxonal Dystrophy
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Intervention(s)
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Drug: RT001
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Primary Outcome(s)
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Modified Ashworth Spasticity Scale
[Time Frame: 12 months]
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Secondary Outcome(s)
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INAD Progression Composite
[Time Frame: 12 months]
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Progression Free Survival Time
[Time Frame: All available data]
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Secondary ID(s)
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RT001-008
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Source(s) of Monetary Support
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Please refer to primary and secondary sponsors
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Results
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Results available:
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Date Posted:
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Date Completed:
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URL:
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