An Open-label Extension Safety Study of MELAS Patients Who Completed TIS6463-203 (PRIZM)
This research study is checking the long-term safety of a medicine called zagociguat for people living with a condition called MELAS syndrome. MELAS is a serious illness that affects the brain, muscles, and other parts of the body. This study is specifically for patients who have already taken part in a previous study (TIS6463-203) involving the same medicine. Participants will take a 15mg tablet of zagociguat by mouth once a day. They will have regular check-ups at a clinic, starting three months after they begin, then at six months, and then every six months after that. The main goal is to see if the medicine is safe and how well people tolerate it over a longer period.
At a glance
What is this study about?
This study is about a condition called MELAS syndrome. MELAS stands for Mitochondrial Encephalopathy, Lactic Acidosis, and Stroke-like Episodes. It's a complex and often serious illness that affects different parts of the body, particularly the brain and muscles. It's caused by problems with mitochondria, which are like the powerhouses of our cells.
The main aim of this particular study is to understand the long-term safety of a medicine called zagociguat. This medicine is currently being explored as a possible treatment for MELAS. Because MELAS is a very serious condition, finding effective and safe treatments is incredibly important for improving the lives of those affected.
This study is a continuation for people who have already been involved in an earlier study (called TIS6463-203) testing zagociguat. By continuing to monitor these participants, doctors can gather more information about how the medicine affects people over a longer period, helping to make sure it's as safe as possible.
Key takeaways
- This study is for long-term safety of zagociguat in MELAS.
- Only for those who completed the previous TIS6463-203 study.
- Involves taking a pill once a day.
- Regular health check-ups at the clinic are required.
- Aims to gather more safety information for this new medicine.
Who may be eligible?
To join this study, you must have already taken part in a previous study called TIS6463-203 and completed all the treatment visits up to 12 weeks. You also need to agree to follow certain lifestyle instructions that the study team will provide.
There are some reasons why you might not be able to join. If a doctor on the study team believes that taking part would not be safe for you, or if you have any medical conditions that would make it difficult for you to complete all the study tasks, then you would not be able to participate.
Anyone of any age or gender can be considered for this study, as long as they meet the other requirements.
- Have I completed the earlier TIS6463-203 study up to the 12-week mark?
- Am I willing to follow specific rules about my lifestyle during the study?
- Do I have any other medical conditions that might make participating unsafe or difficult?
- Am I comfortable with regular clinic visits and taking daily medication?
This is a guide only — the research team will confirm whether you can take part.
What does participation involve?
If you join this study, you will continue to take zagociguat as a 15mg tablet, once a day by mouth. The medicine will be provided to you, either at the clinic or possibly shipped directly to your home. On the day before you start the medicine, you'll have a screening appointment where you'll sign a consent form and have some checks to make sure you're suitable for the study. You'll then start taking the medicine at home.
You'll have regular clinic visits to check on your health and how you're feeling. The first visit will be after three months, then another after six months. After that, your clinic visits will happen every six months. If you need to stop taking the medicine at any point, you'll have a follow-up visit about four weeks later to check how you're doing.
Potential risks and benefits
Locations (16)
- UC San Diego - Altman Clinical and Translational Research InstituteLa Jolla, United States
- Children's Hospital of ColoradoAurora, United States
- Rare Disease ResearchAtlanta, United States
- Massachusetts General HospitalBoston, United States
- Mount Sinai - Ichan School of MedicineNew York, United States
- Akron Children's HospitalAkron, United States
- Children's Hospital of PhiladelphiaPhiladelphia, United States
- Baylor College of MedicineHouston, United States
- UT Health HoustonHouston, United States
- Neuroscience Research AustraliaSydney, Australia
- Royal Melbourne HospitalMelbourne, Australia
- LMU Klinikum, Friedrich-Baur-Institute, Department of NeurologyMunich, Germany
+4 more sites — see the official record for the full list.
Common questions
What is MELAS syndrome?
MELAS is a rare and serious inherited condition that affects many parts of the body, especially the brain and muscles, due to problems with energy production in cells.
What is zagociguat?
Zagociguat is a medicine being tested to see if it can help people with MELAS syndrome. This study is looking at its long-term safety.
How often do I take the medicine?
You will take one 15mg tablet of zagociguat by mouth once a day.
How often will I have clinic visits?
You'll have visits at 3 months, then 6 months, and then every 6 months after that.
Can I stop participating if I want to?
Yes, you have the right to withdraw from the study at any time, for any reason, without it affecting your medical care.
How to find out more
Always speak to your GP or specialist before deciding to take part in a study.
Interested in taking part?
Discussion
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