PI: Dr. Marcus Kilpatrick, Education
Sponsor: Retrotope, Inc.
The RT001-002 protocol is a two center study planned for 18 patients with FA who are ambulatory (with or without an assistive device). The primary endpoints are to evaluate the safety and tolerability of two dose levels of RT001 when administered orally to patients with FA for 28 consecutive days; to determine the PK profile of RT001 at both dose levels following a single and multiple oral administration; and to determine the dose for future studies. The secondary endpoints are to evaluate the effects of RT001 on disease state endpoints using the Friedreich's Ataxia Rating Scale neurological sub-score and the Timed 25-Foot Walk (T25FW) performance measurement relevant to ataxia.