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

  • 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: No

    RDR LOCATION: Georgia

  • TITLE: An Open-label Extension Study Evaluating the Safety of Zagociguat in Participants With MELAS Who Completed TIS6463-203

    SPONSOR: Tisento Therapeutics, Inc.

    INDICATION: MELAS

    PROTOCOL: TIS6463-204

    PHASE: 2

    DESCRIPTION: The goal of this clinical trial is to evaluate the long-term safety and tolerability of zagociguat in patients with MELAS who completed study medication treatment in the lead-in study TIS6463-203. TIS6463-204 is evaluating zagociguat in an open-label extension study at the daily dose of 15mg. The study medication is a once daily oral table and will be provided at the clinic and/or shipped to the participant's home. Study assessments will be conducted during clinic visits which will occur at three months and then at six months. Thereafter, clinic visits will occur every six months.

    STATUS: Active

    RECRUITING PATIENTS: No

    RDR LOCATION: Georgia

Past Trials