A Phase 2a Safety, Tolerability, and Pharmacodynamic Study of OMT-28 in PMD patients with myopathy and/or cardiomyopathy and inflammation (PMD-OPTION)
The PMD-OPTION study is a research trial exploring a new medication called OMT-28 for adults living with Primary Mitochondrial Disease (PMD). This condition often causes muscle weakness (myopathy) and heart issues (cardiomyopathy), along with inflammation. The main goals are to check how safe OMT-28 is, how well people tolerate it, and how it affects the body. Researchers will be especially looking at whether the medicine can reduce levels of a substance in the blood called GDF-15. This study is in an early phase (Phase 2a), which means it's one of the first times this medication has been studied in people with PMD to learn more about its effects. It's an important step in finding new ways to help people with this condition.
At a glance
What is this study about?
This study, known as PMD-OPTION, is designed to investigate a new medication called OMT-28. It's specifically for adults who have Primary Mitochondrial Disease (PMD). PMD is a long-term condition that affects how the body's cells produce energy. This can lead to a variety of symptoms, often including problems with muscles (myopathy), which can cause weakness or tiredness, and issues with the heart (cardiomyopathy). People with PMD can also experience inflammation.
The main purpose of this early-stage study (called Phase 2a) is to find out if OMT-28 is safe for people with PMD and if they can tolerate taking it without too many side effects. We also want to understand how the medicine works in the body and if it has any effect on the disease itself. A key thing we're looking at is whether OMT-28 can lower the level of a specific marker in the blood called GDF-15, which can sometimes be higher in people with certain health conditions.
By taking part, you would be helping researchers learn more about OMT-28 and whether it could potentially become a helpful treatment for people living with Primary Mitochondrial Disease in the future. This kind of research is crucial for developing new medicines that could improve quality of life.
Key takeaways
- Tests a new medicine, OMT-28, for adults with PMD.
- Aims to check safety and how the body handles the medicine.
- Focuses on PMD patients with muscle or heart problems and inflammation.
- Hopes to see if OMT-28 can lower a blood marker called GDF-15.
- Participation helps future research for PMD treatments.
Who may be eligible?
This study is open to adults aged 18 and over. There is no upper age limit, meaning older adults are also welcome to consider participating.
To be considered for this study, you must have a confirmed diagnosis of Primary Mitochondrial Disease (PMD). Additionally, you should be experiencing muscle problems (myopathy) or heart problems (cardiomyopathy), and have signs of inflammation related to your PMD.
Both men and women are invited to take part. There will be other specific medical checks to make sure the study is a safe and suitable option 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.
- Are you 18 years old or older?
- Do you have a diagnosis of Primary Mitochondrial Disease (PMD)?
- Do you experience muscle problems (myopathy) or heart problems (cardiomyopathy) related to your PMD?
- Are you comfortable committing to regular clinic visits for 12 weeks of treatment and some follow-up?
What does participation involve?
If you decide to take part in the PMD-OPTION study, you would be taking the study medication, OMT-28, which comes as a capsule. The study will last for a total of 12 weeks of treatment. During this time, you would have regular visits to the clinic so the study team can monitor your health, check for any side effects, and see how the medication is affecting you. These visits would involve taking blood samples, checking your vital signs (like blood pressure and heart rate), and performing heart checks (like an ECG).
Before you start the medication, there will be a screening period to make sure you meet all the study requirements. After the 12 weeks of treatment, there will also be follow-up visits to ensure your health remains stable. The total duration of your involvement, including screening and follow-up, will be explained in detail by the study team.
Potential risks and benefits
Locations (3)
- —UnverifiedGermany
- —UnverifiedItaly
- —UnverifiedNetherlands
Common questions
What is Primary Mitochondrial Disease (PMD)?
PMD is a condition where the 'powerhouses' of your cells don't work properly, leading to problems with energy production and affecting various body parts, often muscles and the heart.
What is a Phase 2a study?
This is an early stage of research where we learn more about a new medication's safety, side effects, and how it works in a small group of people with the condition.
What is GDF-15?
GDF-15 is a substance in your blood that can be higher in some health conditions, including PMD. The study is looking to see if OMT-28 can lower these levels.
Will I receive the new medicine, OMT-28?
If you are eligible and choose to take part, you would receive the study medication, OMT-28, in capsule form.
How long will I be involved in the study?
The treatment part of the study lasts 12 weeks. Including initial checks and follow-up, the total time will be explained by the study team.
How to find out more
Always speak to your GP or specialist before deciding to take part in a study.
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