Experimental Treatments for Friedreich's Ataxia

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.

Investigational therapies currently in Phase 1 development for Friedreich’s ataxia include CTI-1601 and methylprednisolone. To read more about each specific therapy, click the Learn More button.

If an investigational therapy passes the safety testing in a Phase 1 trial, it will move on to Phase 2 trials. These studies put more emphasis on the effectiveness of a therapy and whether it produces the desired effect in the target patient population while also continuing to monitor and evaluate safety.

Investigational therapies currently in Phase 2 development for Friedreich’s ataxia include epicatechin, etravirine, nicotinamide, leriglitazone, and resveratrol. To read more about each specific therapy, click the Learn More button.

Pending positive results in a Phase 2 trial, an investigational therapy may move on to Phase 3 trials, which will continue to evaluate its effectiveness in a larger portion of the target population. These studies typically compare the effects of the experimental therapy against either a placebo or the current standard treatment. Phase 3 results are often what determines whether or not a regulatory agency will approve a potential therapy.

Investigational therapies currently in Phase 3 development for Friedreich’s ataxia include omaveloxolone and RT001. To read more about each specific therapy, click the Learn More button.

Evaluation of these therapies has been discontinued, typically due to either safety concerns or ineffectiveness.