Mitochondrial encephalomyopathy, lactic acidosis, and stroke-like episodes (MELAS) research hub
4 indexed studies · 4 currently recruiting
- recruitingTherapeutic exploratory (Phase II)
Phase 2b randomized, double-blind, placebo-controlled crossover study evaluating the efficacy and safety of zagociguat in participants with MELAS (PRIZM)
This clinical study tests a potential new medicine called zagociguat for adults with MELAS, a rare genetic disorder. Researchers want to see if zagociguat is safe and whether it can improve daily energy, clear thinking, and physical strength compared to a dummy tablet.
Italy · Germany - recruitingTherapeutic exploratory (Phase II)
A Multi-Center, Randomized, Single-Blind, Placebo-Controlled Study to Assess the Efficacy, Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Oral TTI-0102 for Treatment of Patients with Mitochondrial encephalomyopathy, lactic acidosis and strokelike episodes (MELAS).
This study is testing a new medicine, TTI-0102, for people with MELAS. Researchers want to see if it helps improve how well people can move and feel, and if it's safe to take. It's a type of research where some people get the medicine and others get a dummy pill.
France · Netherlands - recruitingTherapeutic exploratory (Phase II)
An open-label extension study evaluating the safety of zagociguat in participants with MELAS who completed TIS6463-203
This study is looking into the long-term safety of a new medicine called zagociguat for people with MELAS, a rare genetic condition. It's for those who have already been in an earlier study of the drug. Researchers will check for any side effects.
Italy · Germany - recruitingPHASE3
KHENERFIN Study: A Trial to Evaluate the Efficacy and Safety of Sonlicromanol in Primary Mitochondrial Diseases
This study, called KHENERFIN, looks at a new medicine called sonlicromanol for people with mitochondrial diseases. It aims to see if the medicine can reduce tiredness and improve physical abilities like balance and strength. Participants will take either the medicine or a dummy pill for a year.
United States · Denmark · France