Treatments in Phase 1 Trials for FA
Overview
Phase 1 clinical trials are the first in-human studies in the drug development process. The goal of these studies is to test the initial safety of an experimental therapy in a small group of either healthy volunteers or patients. They are also intended to find the optimal dose of the study drug, that is, the one that is most effective with the fewest side effects. Below are some experimental therapies currently in Phase 1 trials for Friedreich’s ataxia.
CTI-1601
CTI-1601 is an investigational therapy designed to deliver the frataxin protein to the mitochondria of Friedreich’s ataxia patients. Dosing was completed in a Phase 1 trial in December 2020, and top-line data are expected in the second quarter of 2021.
Methylprednisolone
Methylprednisolone is a steroid used to reduce inflammation, and may be able to help ease the symptoms of Friedreich’s ataxia. A Phase 1 trial was completed in 2019, but it did not meet its primary objective, with results showing no clear benefit for FA patients. No information is available yet on further studies.
