Trials / Active Not Recruiting
Active Not RecruitingNCT03570931
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
- Status
- Active Not Recruiting
- Phase
- Phase 2 / Phase 3
- Study type
- Interventional
- Enrollment
- 19 (actual)
- Sponsor
- Biojiva LLC · Industry
- Sex
- All
- Age
- 18 Months – 10 Years
- Healthy volunteers
- Not accepted
Summary
The purpose of this study is to evaluate the efficacy and safety of RT001 in patients with Infantile Neuroaxonal Dystrophy (INAD).
Detailed description
This is a single arm open-label study with a structured observation of INAD patients treated with RT001. Enrolled subjects will undergo observation and testing to determine the effect of RT001 treatment. Fifteen to twenty eligible subjects will be treated with RT001 for long-term evaluation of efficacy, safety, tolerability, and pharmacokinetics.
Conditions
Interventions
| Type | Name | Description |
|---|---|---|
| DRUG | RT001 | RT001 is encapsulated di-deutero synthetic homologue of linoleic acid ethyl ester. Each capsule contains 960 mg of RT001. |
Timeline
- Start date
- 2018-11-05
- Primary completion
- 2020-08-09
- Completion
- 2022-06-30
- First posted
- 2018-06-27
- Last updated
- 2021-10-14
Locations
2 sites across 1 country: United States
Regulatory
- FDA-regulated drug study
Source: ClinicalTrials.gov record NCT03570931. Inclusion in this directory is not an endorsement.