AuthorisedTherapeutic exploratory (Phase II)Interventional

An open-label extension study evaluating the safety of zagociguat in participants with MELAS who completed TIS6463-203

This research is an 'extension study' for people with a rare genetic condition called MELAS (Mitochondrial Encephalomyopathy, Lactic Acidosis, and Stroke-like Episodes). MELAS can affect the brain, muscles, and other parts of the body. The study aims to look closely at the long-term safety of a medicine called zagociguat, which participants have already started taking in an earlier study. Researchers will frequently check for any side effects and monitor how participants are doing overall. They will also measure various health markers to see how the body reacts to the medicine over time. This helps to build a more complete picture of the medicine's safety profile.

At a glance

Status

Authorised

Phase

Therapeutic exploratory (Phase II)

Sponsor

Tisento Therapeutics Inc.

Enrolment target

Start

16 Apr 2025

Mitochondrial encephalomyopathy lactic acidosis and stroke-like episodes (MELAS)

What is this study about?

This study is an ongoing part of research into a medicine called zagociguat for people living with a rare condition called MELAS. MELAS is a genetic disorder that mainly affects the brain and muscles, but can also impact other organs. It can lead to stroke-like episodes, muscle weakness, and other health issues. This particular study is called an 'open-label extension study,' which means participants who completed a previous study on zagociguat will continue to receive the medicine, and both they and the doctors will know what treatment is being given.

The main goal of this study is to carefully monitor the safety of zagociguat over a longer period. Researchers will be looking for any side effects the medicine might cause. They will also be tracking many different health measures, like blood pressure, heart health, bone density, and various substances in the blood that can show how the body is functioning. This extensive monitoring helps doctors understand the medicine better and ensures that any potential risks are identified.

By continuing to study zagociguat in this way, researchers hope to gather more information about its effects when used long-term. This information is crucial for understanding if the medicine could eventually become a treatment option for MELAS. It helps build a comprehensive safety profile, which is a key step in developing new therapies for conditions like MELAS.

Key takeaways

This study is for people with MELAS who have already taken zagociguat in a previous trial.
The main goal is to carefully monitor the long-term safety of zagociguat.
Researchers will check for side effects and how the medicine affects overall health.
It's an 'open-label' study, meaning everyone knows you are receiving the medicine.
Participation involves regular clinic visits for health checks and assessments.

Who may be eligible?

To join this study, you must be at least 18 years old. Both men and women can take part. A key requirement is that you must have previously completed an earlier study looking at zagociguat (study TIS6463-203). This study is a continuation for those participants.

The research team will have specific medical criteria to decide if this study is right for you. This often includes reviewing your overall health, current medications, and the specific details of your MELAS condition. They'll also check if you meet all the safety requirements for taking this medicine over a longer period.

It's important to discuss your complete medical history with the study doctors. They will ensure you meet all the necessary qualifications to safely participate.

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.

Are you 18 years old or older?
Have you been diagnosed with MELAS?
Did you complete the previous zagociguat study (TIS6463-203)?
Are you able to attend regular clinic appointments for monitoring?

Answer every question to see your result.

What does participation involve?

If you decide to participate in this study, you would continue to take the study medicine, zagociguat, as prescribed. You would have regular visits to the clinic, where doctors and nurses will monitor your health very closely. These visits will involve various checks, such as measuring your blood pressure and heart rate (vital signs), taking blood samples for lab tests, and possibly urine samples. You might also have heart recordings (ECGs) and scans to check your bone density (DEXA scans).

Additionally, you will be asked questions about any new health problems or symptoms you experience, and how you are feeling generally. There will be specific questionnaires, like those asking about any thoughts of self-harm (C-SSRS) or about your disease symptoms (NMDAS, CGI-C). All these assessments are done to understand how your body is reacting to the medicine and to track any changes in your MELAS condition. The total length of your participation will depend on the study design, but generally, extension studies involve ongoing follow-up.

Potential risks and benefits

Participating in this study might offer the potential benefit of continued access to a new medicine for MELAS. However, as with any medicine, there are potential risks, including side effects from zagociguat. The study team will carefully monitor you for any adverse effects, which will be the primary focus of this research. All medical procedures also carry some risks, such as discomfort from blood draws. You have the right to withdraw from the study at any time, for any reason, without it affecting your medical care.

Locations (2)

Some site locations are approximate. We're improving this — please verify with the trial team before travelling.

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Unverified
Italy
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Unverified
Germany

Common questions

What is MELAS?

MELAS is a rare genetic condition that primarily affects the brain and muscles. It can lead to various problems, including stroke-like episodes, muscle weakness, and other health issues.

What is an 'extension study'?

An extension study means that people who completed an earlier study for a medicine can continue taking it. It helps doctors gather more information about the medicine's long-term safety and effects.

Is zagociguat a new medicine?

Yes, zagociguat is a new medicine being investigated for MELAS. This study will help us understand its effects better.

What does 'open-label' mean?

Open-label means that both you and your study doctors will know that you are taking the study medicine, zagociguat.

What are 'side effects'?

Side effects are any unwanted or unexpected reactions to a medicine. This study is specifically designed to carefully track and record all potential side effects.