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Zagociguat

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.

Table of Contents

Trials overview

Two authorised interventional studies are investigating Zagociguat in people with MELAS, which stands for mitochondrial encephalomyopathy, lactic acidosis, and stroke-like episodes.[1][2]

The first study, NCT06402123, is a Phase 2 trial designed to evaluate safety and effectiveness in MELAS.[1] The second study, NCT06961344, is an open-label extension study for participants who completed TIS6463-203 and is focused on long-term safety.[2]

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.[1][2]

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.[2]

What the trials measure

The main study looks at both efficacy, which means whether the treatment may help, and safety.[1]

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.[1]

These three results are combined in a global statistical test, which is a planned way to look at several key outcomes together.[1]

For safety, the study measures treatment-emergent adverse events, meaning health problems that begin after the study drug starts.[1]

The extension study also measures treatment-emergent adverse events, with the goal of learning about long-term safety and tolerability.[2]

Trial design and treatment groups

NCT06402123 includes Zagociguat 7.5 mg and 15 mg tablets, along with placebo-to-match tablets for comparison.[1]

A placebo-to-match tablet looks like the study drug but does not contain the active treatment, which helps researchers compare results fairly.[1]

NCT06961344 is an open-label extension study using Zagociguat 15 mg, and open-label means everyone knows the treatment being given.[2]

Trial phases and status

Both studies are Phase 2 trials and are listed as authorised.[1][2]

Phase 2 studies are used to learn more about whether a treatment may help and to continue checking safety in a specific patient group.[1][2]

Each study has an enrollment of 44 participants, based on the trial data provided.[1][2]

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.[1][2]

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.[1][2]

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.[1]

Trial ID Phase Condition studied Status Enrollment
NCT06402123 Phase 2 MELAS Authorised 44
NCT06961344 Phase 2 MELAS Authorised 44

FAQ

  • What condition are the Zagociguat trials studying? The trials are studying MELAS, which stands for mitochondrial encephalomyopathy, lactic acidosis, and stroke-like episodes. This is a rare disease that affects the body’s energy-making parts in cells.
  • What are the main goals of these trials? One trial is checking whether Zagociguat may help with fatigue and thinking skills in MELAS. Both trials also measure safety, including treatment-emergent adverse events, which means health problems that start after the study drug is given.
  • What phase are the Zagociguat trials in? Both listed studies are Phase 2 trials. Phase 2 studies look more closely at whether a treatment may work and continue to monitor safety.
  • Who can take part in these trials? The studies are for participants with MELAS. One study includes people with MELAS, and the open-label extension study includes people with MELAS who finished the earlier TIS6463-203 study.
  • How is Zagociguat being studied in these trials? In the main study, participants receive Zagociguat or placebo-to-match tablets, which look the same but do not contain the study drug. In the extension study, participants receive Zagociguat in an open-label design, which means everyone knows they are taking the study drug.

Ongoing Clinical Trials on Zagociguat

  • Long-term safety study of zagociguat tablets in patients with MELAS syndrome (Mitochondrial encephalomyopathy, lactic acidosis, and stroke-like episodes) who completed previous trial

    Not yet recruiting

    1 1
    Investigated diseases:
    Investigated drugs:
    Germany Italy

  • Study on the Safety and Effectiveness of Zagociguat for Treating MELAS in Patients

    Not recruiting

    1
    Investigated diseases:
    Investigated drugs:
    Germany Italy

Glossary

  • MELAS: A rare condition called mitochondrial encephalomyopathy, lactic acidosis, and stroke-like episodes. It can affect muscles, the brain, and energy use in the body.
  • Mitochondrial disease: A disease that affects mitochondria, the parts of cells that help make energy.
  • Phase 2: A trial stage that looks more closely at whether a treatment may help and continues to check safety.
  • Interventional study: A study where participants are given a treatment so researchers can measure its effects.
  • Placebo-to-match: A tablet that looks like the study drug but does not contain the active treatment. It helps compare results fairly.
  • Open-label extension study: A follow-up study where participants keep taking the study treatment and both the participant and study team know what is being given.
  • Safety: How well a treatment is tolerated and whether it causes unwanted health problems.
  • Tolerability: How manageable a treatment is for participants, including how well they can take it over time.
  • Fatigue: A strong feeling of tiredness or low energy.
  • Cognition: Thinking skills such as memory, attention, and processing speed.
  • Primary outcome: The main result a trial is designed to measure.
  • Treatment-emergent adverse events: Health problems that appear or get worse after the study treatment starts.

References