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Antioxidant
RT001 for Infantile Neuroaxonal Dystrophy
Phase 2 & 3
Waitlist Available
Research Sponsored by Retrotope, Inc.
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial Must have
Signed informed consent form (ICF) prior to entry into the study
Homozygous for PLA2G6 deficiency (variant alleles may be mixed heterozygotes)
Timeline
Screening 3 weeks
Treatment Varies
Follow Up 12 months
Awards & highlights
Study Summary
This study is evaluating whether a drug called RT001 can improve symptoms in people with Infantile Neuroaxonal Dystrophy.
Eligible Conditions
- Infantile Neuroaxonal Dystrophy
Eligibility Criteria
Inclusion Criteria
You may be eligible if you check “Yes” for the criteria belowTimeline
Screening ~ 3 weeks3 visits
Treatment ~ Varies
Follow Up ~ 12 months
Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~12 months
Treatment Details
Study Objectives
Outcome measures can provide a clearer picture of what you can expect from a treatment.Primary outcome measures
Modified Ashworth Spasticity Scale
Secondary outcome measures
INAD Progression Composite
Progression Free Survival Time
Other outcome measures
Incidence of Treatment-Emergent Adverse Events
Modified Infantile Neuroaxonal Dystrophy Rating Scale (mINAD-RS24)
Modified Parental Rating Scale (mPRS22)
+2 moreSide effects data
From 2021 Phase 3 trial • 65 Patients • NCT0410250121%
Falls
12%
nausea
12%
headache
9%
diarrhea
3%
non-cardiac chest pain
100%
80%
60%
40%
20%
0%
Study treatment Arm
RT001
Placebo
Trial Design
1Treatment groups
Experimental Treatment
Group I: RT001Experimental Treatment1 Intervention
RT001, oral, 3.84 g/day
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
Linoleic acid
FDA approved
Find a Location
Who is running the clinical trial?
Retrotope, Inc.Lead Sponsor
6 Previous Clinical Trials
220 Total Patients Enrolled
Peter Milner, MDStudy DirectorChief Medical Officer
3 Previous Clinical Trials
121 Total Patients Enrolled
Frequently Asked Questions
These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.
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