November 5, 2018
A Study to Assess Efficacy and Safety of RT001 in Subjects With Infantile Neuroaxonal Dystrophy
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 eligible subjects will be treated with RT001 for long-term evaluation of efficacy, safety, tolerability, and pharmacokinetics.
December 13, 2018
Retrotope Announces First Dosing of RT001 in patients with Infantile Neuroaxonal Dystrophy (INAD)
The U.S. Food and Drug Administration (FDA) has granted the company approval to conduct this trial as an open-label Phase 2/3 clinical trial in 15-20 patients. Due to the paucity of publicly available natural history of the progression of the disease, Retrotope is currently also enrolling a prospective natural history study in a matched group of INAD patients who are not participating in the trial. (December 13, 2018)