A Study to Assess Efficacy and Safety of RT001 in Subjects With Infantile Neuroaxonal Dystrophy

• Status: Active, not recruiting
• Trial end date: 2022-06-30
• Target enrollment: 0
• Participant gender: All

Summary

The purpose of this study is to evaluate the efficacy and safety of RT001 in patients with Infantile Neuroaxonal Dystrophy (INAD).

• Phase: Phase 2/Phase 3
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Retrotope, Inc.

Criteria

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