A Study to Assess Efficacy and Safety of RT001 in Subjects With Infantile Neuroaxonal Dystrophy
NCT ID: NCT03570931
Last Updated: 2021-10-14
Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
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ACTIVE_NOT_RECRUITING
PHASE2/PHASE3
19 participants
INTERVENTIONAL
2018-11-05
2022-06-30
Brief Summary
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Detailed Description
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Conditions
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Study Design
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NA
SINGLE_GROUP
TREATMENT
NONE
Study Groups
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RT001
RT001, oral, 3.84 g/day
RT001
RT001 is encapsulated di-deutero synthetic homologue of linoleic acid ethyl ester. Each capsule contains 960 mg of RT001.
Interventions
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RT001
RT001 is encapsulated di-deutero synthetic homologue of linoleic acid ethyl ester. Each capsule contains 960 mg of RT001.
Other Intervention Names
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Eligibility Criteria
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Inclusion Criteria
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
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
18 Months
10 Years
ALL
No
Sponsors
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Biojiva LLC
INDUSTRY
Responsible Party
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Principal Investigators
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Peter Milner, MD
Role: STUDY_DIRECTOR
Chief Medical Officer
Locations
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University of California San Francisco, Benioff Children's Hospital
San Francisco, California, United States
Jacobs Levy Genomics and Research Program
Morristown, New Jersey, United States
Countries
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Other Identifiers
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RT001-008
Identifier Type: -
Identifier Source: org_study_id