Mitochondrial Encephalomyopathy, Lactic Acidosis, and Stroke-like Episodes (MELAS)
MELAS (Mitochondrial Encephalomyopathy, Lactic Acidosis, and Stroke-like Episodes) is a mitochondrial disorder affecting the brain and muscles. Progressive prognosis with variable severity; early onset often leads to greater disability.
Cause: Most often due to MT-TL1 gene mutations in mitochondrial DNA; inherited maternally.
Features: Stroke-like episodes, seizures, muscle weakness, lactic acidosis, hearing loss, headaches, and neurological decline, usually starting in childhood or adolescence.
Diagnosis: Based on symptoms, high lactic acid, brain MRI, muscle biopsy, and confirmed by genetic testing.
Treatment: No cure; managed with supplements (e.g. L-arginine, CoQ10), seizure medications, and supportive care.
Active Trials
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TITLE: Phase 2b Randomized, Double-blind, Placebo-controlled Crossover Study Evaluating the Efficacy and Safety of Zagociguat in Participants with MELAS (PRIZM)
SPONSOR: Tisento Therapeutics, Inc.
INDICATION: MELAS
PROTOCOL: TIS6463-203
PHASE: 2b
DESCRIPTION: The goal of this clinical trial is to learn about the effectiveness of zagociguat in patients with MELAS. 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 either to receive placebo in period 1 followed by active drug in period 2 OR to receive active drug in period 1 followed by placebo in period 2. Study medication is a once daily oral tablet and will be provided at the clinic and/or shipped to the participant's home. Clinic visits will occur at screening and Week 1 and Week 12 of each treatment period. Visits at Week 4 and Week 8 of both crossover periods will either be in clinic or optionally at the participant'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.
STATUS: Active
RECRUITING PATIENTS: Yes
RDR LOCATION: Georgia