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

Infantile Neuroaxonal Dystrophy Clinical Trial

Official title: A Prospective Open-label Study to Assess Efficacy and Safety of RT001 in Subjects With Infantile Neuroaxonal Dystrophy

Status Active, not recruiting

Clinical Trial Summary

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

Clinical Trial 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. ;

Related Conditions & MeSH terms

• Infantile Neuroaxonal Dystrophy

• NCT number: NCT03570931
• Study type: Interventional
• Source: Retrotope, Inc.
• Status: Active, not recruiting
• Phase: Phase 2/Phase 3
• Start date: November 5, 2018
• Completion date: June 30, 2022