Mitochondrial Encephalopathy, Lactic Acidosis and Stroke-Like Episodes (MELAS Syndrome) Clinical Trial
Official title:
Phase 2b Randomized, Double-blind, Placebo-controlled Crossover Study Evaluating the Efficacy and Safety of Zagociguat in Participants With MELAS (PRIZM)
PRIZM is a Phase 2b randomized, double-blind, placebo-controlled, 3-treatment, 2-period, crossover study evaluating the efficacy and safety of oral zagociguat 15 and 30 mg vs. placebo when administered daily for 12 weeks in participants with genetically and phenotypically defined MELAS.
The goal of this clinical trial is to learn about the effectiveness of zagociguat in patients with MELAS. The main questions it aims to answer are: - Does zagociguat improve fatigue in patients with MELAS? - Does zagociguat improve cognitive performance in patients with MELAS? - What is the safety and tolerability profile of zagociguat? The PRIZM study is evaluating 2 dose levels of zagociguat in a crossover design consisting of two 12-week treatment periods separated by a 4-week washout. Patients will be screened and if eligible, randomly assigned to either receive placebo in period 1 followed by active drug in period 2. Study medication is a once daily oral tablet and will be provided at the clinic and/or shipped to the patient's home. Clinic visits will occur at screening and Week 1 and Week 12 of each crossover period. Visits at Week 4 and Week 8 of both crossover periods will either be in clinic or optionally at the patient's home. Study assessments will be conducted weekly on a phone app and a separate tablet and additional assessments will be conducted during visits. Patients who complete the study will be eligible for an open label extension study. ;