An Open-label Extension Safety Study of MELAS Patients Who Completed TIS6463-203 (PRIZM)

Running, not enrolling · Phase 2

Conditions studied: Mitochondrial Encephalopathy, Lactic Acidosis and Stroke-Like Episodes (MELAS Syndrome)

In brief

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.

Key facts

Study ID

NCT06961344

Run by

Tisento Therapeutics

People needed

44

Starts

2025-08-18

Expected to finish

2028-07-01

Last updated by the study team

2026-07-07

Who can join

Age: any. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• Signed an Institutional Review Board (IRB)/Independent Ethics Committee (IEC)-approved informed consent form (ICF).
• Completed TIS6463-203 treatment through the Period 2 Week 12 Visit.
• Agrees to follow lifestyle restrictions.
• Other criteria per the protocol.

You may not qualify if…

• Any medical condition or clinical finding that, per investigator judgement, would preclude safe study participation and/or completion of all trial requirements.

Where it is running

• UC San Diego - Altman Clinical and Translational Research Institute — La Jolla, California, United States
• Children's Hospital of Colorado — Aurora, Colorado, United States
• Rare Disease Research — Atlanta, Georgia, United States
• Massachusetts General Hospital — Boston, Massachusetts, United States
• Mount Sinai - Ichan School of Medicine — New York, New York, United States
• Akron Children's Hospital — Akron, Ohio, United States
• Children's Hospital of Philadelphia — Philadelphia, Pennsylvania, United States
• Baylor College of Medicine — Houston, Texas, United States
• UT Health Houston — Houston, Texas, United States
• Neuroscience Research Australia — Sydney, New South Wales, Australia
• Royal Melbourne Hospital — Melbourne, Victoria, Australia
• LMU Klinikum, Friedrich-Baur-Institute, Department of Neurology — Munich, Germany
• Neurologic Institute Carlo Besta of Milan — Milan, Italy
• Fondazione Policlinico Universitario Agostino Gemelli — Rome, Italy
• University College London Hospital. The National Hospital for Neurology and Neurosurgery, Centre for Neuromuscular Diseases — London, United Kingdom
• The Newcastle upon Tyne NHS Foundation Trust — Newcastle upon Tyne, United Kingdom

Full record on ClinicalTrials.gov

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