Phase 2b randomized, double-blind, placebo-controlled crossover study evaluating the efficacy and safety of zagociguat in participants with MELAS (PRIZM)
MELAS is a rare inherited condition that affects mitochondria, the tiny power stations inside our cells. When these power stations struggle, people often face severe exhaustion, muscle weakness, and difficulties with memory and concentration. Currently, treatment options are very limited. This clinical trial, named PRIZM, is testing an experimental medicine called zagociguat. Doctors want to see if zagociguat can safely improve blood flow and help cells work better. Participants will take either zagociguat or a placebo (a dummy tablet that contains no active medicine). Over at least 12 weeks, the research team will track how the treatment affects tiredness, memory, problem-solving, and muscle power through simple tasks and questionnaires. Because this is a crossover trial, participants may take both the active medicine and the placebo at different stages. The main aim is to find out if zagociguat can safely help people with MELAS feel better and do more.
At a glance
What is this study about?
MELAS is a rare genetic condition that affects how the body creates energy. In healthy cells, tiny structures called mitochondria turn food and oxygen into power. In people with MELAS, these power units do not work properly. This lack of energy can lead to serious problems, including stroke-like episodes, muscle weakness, extreme tiredness, and trouble thinking clearly.
This study is testing an investigational drug called zagociguat. Scientists believe this medicine may help improve blood flow and cell communication, helping tissues get the oxygen and support they need even when mitochondria are struggling. The goal is to see whether taking zagociguat tablets daily can ease daily fatigue and boost brain function and physical strength.
To ensure fair results, the trial compares zagociguat against a placebo, which looks identical but has no active ingredient. This is a 'crossover' study, which means participants will take both the real medicine and the dummy tablet at different times. Comparing the results from both periods helps researchers see true differences in how people feel and perform.
If zagociguat proves to be safe and effective, it could become an important new treatment option for people living with MELAS, potentially helping to improve daily stamina, memory, and overall quality of life.
Key takeaways
- Tests a potential new tablet called zagociguat for adults with MELAS.
- Checks if the medicine reduces tiredness and improves brain function and physical strength.
- Compares the active drug against a dummy pill using a crossover design.
- Involves regular clinic visits, memory puzzles, and simple physical checks over 12 weeks or more.
- Participation is voluntary, and safety is monitored closely by doctors throughout.
Who may be eligible?
This trial is open to adults aged 18 and older who have a confirmed medical diagnosis of MELAS. Both men and women are able to join.
To take part, you will need to be well enough to complete regular study visits, answer questions about how you feel, and do basic thinking and memory tasks on a computer or tablet. You also need to be able to try simple physical movements, such as standing up from a chair.
You may not be able to join if you have other severe medical problems that could make the trial unsafe, or if you take certain medicines that cannot be mixed with the study drug. The clinical research team will review your full medical history to make sure taking part is right for you.
Could this study suit you?
Answer these quick questions to see if you may be eligible. This is a guide only — the research team makes the final call.
- I am 18 years of age or older.
- I have a confirmed diagnosis of MELAS.
- I can attend regular hospital clinic visits.
- I am able to complete simple computer puzzles and memory tasks.
- I can manage basic physical checks, such as rising from a chair.
- I am willing to take daily tablets as directed by the study team.
What does participation involve?
Taking part involves taking study tablets once a day by mouth and attending regular check-ups at a study clinic over several months. During visits, doctors will monitor your health, check your blood pressure, and take blood samples. You will be asked to complete questionnaires about your tiredness levels, do short computer-based puzzles to test your memory and reaction speed, and do a quick 30-second sit-to-stand exercise to check your muscle strength. Because this is a crossover trial, you will spend time on both the study medicine and the placebo. Afterwards, there will be follow-up checks to ensure you remain well.
Potential risks and benefits
Locations (2)
- —UnverifiedItaly
- —UnverifiedGermany
Common questions
What is MELAS?
MELAS is a rare genetic condition that damages the power-producing parts of cells (mitochondria), causing tiredness, muscle weakness, and brain-related symptoms.
What is a placebo?
A placebo is a dummy tablet made to look and taste just like the real medicine, but it contains no active ingredients. It helps scientists tell if changes are truly caused by the study drug.
What does a crossover study mean?
It means each person takes both the real medicine and the dummy tablet at different points during the trial, allowing doctors to compare how each individual responds to both.
Does taking part cost anything?
No. The study tablets, clinic visits, and all trial tests are provided free of charge, and travel expenses are usually refunded.
Can I leave the trial if I change my mind?
Yes. Joining is entirely voluntary. You can withdraw at any time, and your usual medical treatment and care will carry on as normal.
How to find out more
Always speak to your GP or specialist before deciding to take part in a study.
Interested in taking part?
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