A Multi-Center, Randomized, Single-Blind, Placebo-Controlled Study to Assess the Efficacy, Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Oral TTI-0102 for Treatment of Patients with Mitochondrial encephalomyopathy, lactic acidosis and strokelike episodes (MELAS).
This research study is looking into a new medicine called TTI-0102 for a rare condition called MELAS (Mitochondrial encephalomyopathy, lactic acidosis and stroke-like episodes). The main goal is to find out if this new medicine can help people with MELAS walk better and improve their overall health. We also want to check if the medicine is safe and how well people tolerate it. Participants will either receive the new medicine or a dummy pill (placebo) without knowing which one they are taking. We'll be closely monitoring their health, including physical abilities, tiredness, and quality of life over a period of 24 weeks. This study hopes to find a new way to help those living with MELAS.
At a glance
What is this study about?
This study is like a thorough test drive for a new medicine called TTI-0102. It's being developed for people who have a rare condition called MELAS. MELAS is a health problem that affects the body's energy-producing factories, called mitochondria. This can lead to various issues, including muscle weakness, headaches, and sometimes stroke-like episodes.
The main aim of this study is to see if TTI-0102 can make a real difference in how people with MELAS feel and function in their daily lives. Specifically, we'll be checking if the medicine helps improve how far people can walk in a set amount of time. Researchers also want to understand if the medicine is safe to use and if it causes any unwanted side effects. We're also trying to understand how the medicine works in the body.
To make sure the results are fair and accurate, this study is set up so that some people will receive the new medicine, TTI-0102, and others will receive a 'dummy' pill, called a placebo. The dummy pill looks just like the real medicine but contains no active ingredients. Neither the participants nor the doctors will know who is getting which, to avoid any bias. This helps us truly see if any changes are due to the medicine itself.
Key takeaways
- This study is testing a new medicine (TTI-0102) for MELAS.
- It aims to see if the medicine improves walking ability and overall well-being.
- Some participants will receive the new medicine, others a dummy pill.
- The study will last about 24 weeks and involves regular clinic visits.
- Safety and potential side effects will be closely monitored.
- Participation is voluntary, and you can withdraw at any time.
Who may be eligible?
This study is looking for adults who have been diagnosed with MELAS. You need to be at least 18 years old to take part. There isn't an upper age limit mentioned, so older adults could also be eligible. Both men and women are welcome to participate in this research.
There will be specific health checks and tests to make sure that taking part in the study is right for you and that you meet all the requirements. For example, the study team will need to confirm your MELAS diagnosis and that you don't have other health conditions that might affect the study results or your safety.
- Are you 18 years old or older?
- Have you been diagnosed with MELAS?
- Are you able to attend regular clinic visits for about 6 months?
- Are you willing to potentially receive either the new medicine or a dummy pill?
- Are you generally in a stable medical condition (some other health issues might exclude you, which the doctors will check)?
This is a guide only — the research team will confirm whether you can take part.
What does participation involve?
If you decide to join this study, you might receive the new medicine TTI-0102 or a dummy pill (placebo). You won't know which one you're getting. Your participation will last for about 24 weeks, which is roughly six months. During this time, you'll have several visits to the study clinic. At these visits, doctors and nurses will check on your health, ask about any changes you've noticed, and perform various tests.
These tests will include blood samples, physical examinations, and checking your vital signs like blood pressure and heart rate. You’ll also do a 12-minute walking test to see how far you can walk. You'll also be asked to fill out questionnaires about how you're feeling, your tiredness levels, and your general quality of life. An ECG (heart tracing) will be done, and any seizure activity will be monitored. You'll have regular check-ups at Weeks 4, 8, 12, 16, 20, and a final check at Week 24.
Potential risks and benefits
Locations (2)
- —France
- —Netherlands
Common questions
What is MELAS?
MELAS is quite a rare condition that affects the body's energy production in cells, leading to problems with muscles, the brain, and sometimes other organs.
What is TTI-0102?
TTI-0102 is the name of the new medicine being tested in this study. It's aiming to help improve symptoms of MELAS.
What is a 'dummy pill' or placebo?
A dummy pill (placebo) looks exactly like the real medicine but contains no active ingredients. It's used to compare against the new medicine to see if it's truly effective.
How long will I be in the study?
If you join, your participation in the study will last for about 24 weeks, which is roughly six months.
Will I know if I'm getting the real medicine?
No, during the study, neither you nor the study doctors will know whether you are receiving TTI-0102 or the dummy pill. This helps keep the study fair.
How to find out more
Always speak to your GP or specialist before deciding to take part in a study.
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