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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 below

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~12 months
This trial's timeline: 3 weeks for screening, Varies for treatment, and 12 months for reporting.

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 more

Side effects data

From 2021 Phase 3 trial • 65 Patients • NCT04102501
21%
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.
~3 spots leftby Mar 2025