MOXle Study: Evaluating the drug RTA 408 in the treatment of Friedreich’s Ataxia.
A clinical trial for a novel medication to treat Friedreich’s Ataxia was announced this past week. Reata Pharmaceuticals, a privately held biopharmaceutical company based out of Texas, announced the beginning of enrollments for a phase 2 and 3 clinical study titled “MOXle – RTA 408 Capsules in Patients with Friedreich’s Ataxia.” The study aims to further evaluate the safety, efficacy and pharmacodynamics of the treatment medication. A randomized, double blind, study to evaluate the maximum tolerated dose will be part one of the study. Part two will consist of splinting participants into two groups and giving two different dose levels of RTA 408. The second part will also be a randomized, placebo-controlled, double blinded study. This will be a multi-center study in which 52 patients are being sought.
Two clinical interests of the study will be peak workload during exercise as well as the modified Friedreich’s ataxia rating scale (FARS). The latter is a measurement tool which monitors proficiency of patients during activities. Additional biochemical endpoints will also be evaluated. Treatment with the study medication or placebo will be given to patient’s once daily for twelve weeks.
Nrf2 or Nuclear Factor 2, is a protein that regulates the expression of antioxidant proteins that protect against oxidative damage. Friedreich’s Ataxia is caused by defects in the gene for frataxin which is involved in the regulation of iron levels in the mitochondria. In pre-clinical studies, lower expressions of Nrf2 were correlated with frataxin deficiency and lower mitochondrial function. It is believed that RTA 408 can activate the Nrf2 pathways and improve mitochondrial function. Taken from animal models, RTA 408 showed the ability to improve overall cellular metabolism.
The study is expected to achieve final outcome measures by the spring of 2015 and study completion closer to June 2016.