A Study to Assess Efficacy and Safety of RT001 in Subjects With Infantile Neuroaxonal Dystrophy
A Prospective Open-label Study to Assess Efficacy and Safety of RT001 in Subjects With Infantile Neuroaxonal Dystrophy
1 other identifier
interventional
19
1 country
2
Brief Summary
The purpose of this study is to evaluate the efficacy and safety of RT001 in patients with Infantile Neuroaxonal Dystrophy (INAD).
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at below P25 for phase_2
Started Nov 2018
Typical duration for phase_2
2 active sites
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
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Study Timeline
Key milestones and dates
First Submitted
Initial submission to the registry
June 11, 2018
CompletedFirst Posted
Study publicly available on registry
June 27, 2018
CompletedStudy Start
First participant enrolled
November 5, 2018
CompletedPrimary Completion
Last participant's last visit for primary outcome
August 9, 2020
CompletedStudy Completion
Last participant's last visit for all outcomes
June 30, 2022
CompletedOctober 14, 2021
October 1, 2021
1.8 years
June 11, 2018
October 6, 2021
Conditions
Keywords
Outcome Measures
Primary Outcomes (1)
Modified Ashworth Spasticity Scale
Change from baseline in the Modified Ashworth spasticity scale.
12 months
Secondary Outcomes (2)
INAD Progression Composite
12 months
Progression Free Survival Time
All available data
Other Outcomes (5)
Modified Infantile Neuroaxonal Dystrophy Rating Scale (mINAD-RS24)
12 months
Modified Parental Rating Scale (mPRS22)
12 months
Original Infantile Neuroaxonal Dystrophy Rating Scale (INAD-RS40)
12 months
- +2 more other outcomes
Study Arms (1)
RT001
EXPERIMENTALRT001, oral, 3.84 g/day
Interventions
RT001 is encapsulated di-deutero synthetic homologue of linoleic acid ethyl ester. Each capsule contains 960 mg of RT001.
Eligibility Criteria
You may qualify if:
- Male or female 18 months to 10 years of age
- Medical history consistent with the symptoms of classic INAD (onset of symptoms between the ages of 6 months and 3 years)
- Homozygous for PLA2G6 deficiency (variant alleles may be mixed heterozygotes)
- Must have impairment in at least 2 of the assessed categories at baseline
- Signed informed consent form (ICF) prior to entry into the study
- Able to provide the necessary blood samples
You may not qualify if:
- Received treatment with other experimental therapies within the last 30 days prior to the first dose
- Requiring mechanical ventilation, other than positive air pressure support primarily for mitigation of sleep apnea.
- Have a life expectancy of less than one year
- Diagnosis of atypical NAD (ANAD)
- 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
Contact the study team to confirm eligibility.
Sponsors & Collaborators
- Biojiva LLClead
Study Sites (2)
University of California San Francisco, Benioff Children's Hospital
San Francisco, California, 94158, United States
Jacobs Levy Genomics and Research Program
Morristown, New Jersey, 07960, United States
MeSH Terms
Conditions
Interventions
Condition Hierarchy (Ancestors)
Study Officials
- STUDY DIRECTOR
Peter Milner, MD
Chief Medical Officer
Study Design
- Study Type
- interventional
- Phase
- phase 2
- Allocation
- NA
- Masking
- NONE
- Masking Details
- Open Label
- Purpose
- TREATMENT
- Intervention Model
- SINGLE GROUP
- Sponsor Type
- INDUSTRY
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
June 11, 2018
First Posted
June 27, 2018
Study Start
November 5, 2018
Primary Completion
August 9, 2020
Study Completion
June 30, 2022
Last Updated
October 14, 2021
Record last verified: 2021-10