In short
Clinical trials are investigating Zagociguat in people with MELAS, a rare mitochondrial disease that can cause fatigue, thinking problems, and stroke-like episodes. The studies are looking at safety, tolerability, and possible effects on fatigue and cognition in adults who meet the trial criteria.
Key points
- Zagociguat is being studied in Phase 2 clinical trials for MELAS, a rare mitochondrial disease. One study is testing whether it may help with fatigue and thinking skills, while also checking safety. Another study is an open-label extension for people who finished the earlier trial, and it focuses on long-term safety and tolerability. Both studies are authorised and involve adults with MELAS. The main results being measured include fatigue scores, thinking tests, and treatment-emergent adverse events.
Trials overview
Two authorised interventional studies are investigating Zagociguat in people with MELAS, which stands for mitochondrial encephalomyopathy, lactic acidosis, and stroke-like episodes.
The first study, NCT06402123, is a Phase 2 trial designed to evaluate safety and effectiveness in MELAS. The second study, NCT06961344, is an open-label extension study for participants who completed TIS6463-203 and is focused on long-term safety.
Who is being studied
Both trials are focused on people with MELAS, a rare mitochondrial disease that can affect energy use in the body and may cause tiredness and problems with thinking.
The extension study includes participants with MELAS who already finished the earlier TIS6463-203 study, so it follows people who have already taken part in a related trial.
What the trials measure
The main study looks at both efficacy, which means whether the treatment may help, and safety.
For efficacy, the study measures fatigue and cognition during Weeks 9 through 12 using the PROMIS Fatigue MELAS Short Form, the Groton Maze Learning Test, and the International Digit Symbol Substitution Test.
These three results are combined in a global statistical test, which is a planned way to look at several key outcomes together.
For safety, the study measures treatment-emergent adverse events, meaning health problems that begin after the study drug starts.
The extension study also measures treatment-emergent adverse events, with the goal of learning about long-term safety and tolerability.
Trial design and treatment groups
NCT06402123 includes Zagociguat 7.5 mg and 15 mg tablets, along with placebo-to-match tablets for comparison.
A placebo-to-match tablet looks like the study drug but does not contain the active treatment, which helps researchers compare results fairly.
NCT06961344 is an open-label extension study using Zagociguat 15 mg, and open-label means everyone knows the treatment being given.
Trial phases and status
Both studies are Phase 2 trials and are listed as authorised.
Phase 2 studies are used to learn more about whether a treatment may help and to continue checking safety in a specific patient group.
Each study has an enrollment of 44 participants, based on the trial data provided.
What these studies mean for patients
These trials are not general studies of Zagociguat for many diseases; they are focused on MELAS and on outcomes that matter to patients, such as fatigue, thinking ability, and safety.
The main study compares different tablet strengths and placebo-to-match tablets, while the extension study follows participants over a longer time to learn more about ongoing safety.
Because the studies use specific tests and time points, they are designed to measure change in a structured way rather than relying only on general impressions.