MOXIe Clinical Study
MOXIe is a Phase 2 clinical study evaluating the safety and effectiveness of omaveloxolone (an oral investigational drug) for the treatment of Friedreich’s ataxia (FA).
The study has two different parts. Part 1 enrollment completed in February 2017 and the data were shared publicly in June of 2017. Part 1 was a dose ranging study to evaluate the safety and effectiveness of omaveloxolone. The purpose of a dose ranging study is to determine which dose(s) is better tolerated or most effective. MOXIe Part 1 evaluated doses ranging from 2.5 mg to 300 mg. Participants were assigned to receive omaveloxolone or placebo for 12 weeks. Part 1 enrolled a total of 69 patients, and the key observations included the following:
- Omaveloxolone significantly improved mFARS (modified FA Rating Scale) from baseline across all doses
- In omaveloxolone-treated patients, mFARS was improved at Week 4 and further improved by Week 12
- Omaveloxolone at 160 mg dose showed large mFARS improvements as early as Week 4
- Omaveloxolone was well-tolerated and adverse events were generally mild in severity
Part 2 of MOXIe opened for screening in July of 2017. All participants in Part 2 will be randomly assigned (like by the flip of a coin) to either 150 mg of omaveloxolone or placebo (an inactive capsule). Data collected from the group of patients assigned to omaveloxolone will be compared to the data collected from patients assigned to placebo, in order to help researchers better understand how patients are responding to the study drug. You may not directly benefit from participating in this study, but you and other participants may make an important contribution to advancing the understanding and treatment of FA.
Patients who complete Part 1 or Part 2 may be eligible to continue in an extension portion of the study where all patients receive the active drug, omaveloxolone. At the discretion of your doctor, you may participate in the extension portion of the study until the drug is commercially available or the study is stopped.
MOXIe (Part 2) is now open for enrollment. Up to 100 participants will be enrolled in MOXIe Part 2 at approximately 20 research centers around the world. Participation may last up to 12 months, and will include a screening visit, 8 study visits, 4 scheduled phone calls, and a final follow-up visit at the end of the study.
The investigational drug, study-related procedures, and doctor visits will be provided at no cost. If you travel to the site for your study visits, travel expenses will be reimbursed, and compensation for study-related time may be provided.
MOXIe (Part 2) Study Overview at a Glance