Ongoing, recruitingTherapeutic exploratory (Phase II)Interventional

A phase IIa, open label, single centre study to assess the safety, tolerability, pharmacokinetics, pharmacodynamics and preliminary efficacy of orally dosed MBF-015 in Huntington's disease patients

This study is a small, early test (Phase IIa) of a new medicine, MBF-015, for people with Huntington's disease. The main goal is to find out if the medicine is safe and if people can tolerate it over 28 days. Researchers will monitor for any side effects and changes in important body signs like blood pressure and heart rate. They will also look at how the body processes the medicine and if it shows any early signs of helping with Huntington's symptoms, such as movement, thinking, and daily activities. This study will involve taking the medicine for 28 days, followed by a check-up at day 43.

At a glance

Status

Ongoing, recruiting

Phase

Therapeutic exploratory (Phase II)

Sponsor

Medibiofarma S.L.

Enrolment target

Start

11 Sep 2023

Huntington's disease

What is this study about?

This study is an important step in exploring a new possible treatment, MBF-015, for Huntington's disease. Huntington's disease is a condition that affects the brain and can cause uncontrolled movements, problems with thinking, and changes in mood. Currently, there's no cure for Huntington's, so new treatments are really needed.

This particular study is a 'Phase IIa' trial. This means it's still quite early, and the main focus is on making sure the new medicine is safe and doesn't cause too many problems for people. The researchers will be looking very closely at any side effects and how well people tolerate taking the medicine over 28 days. They will also be studying how the medicine moves through the body, how quickly it's used up, and how much of it gets to where it needs to go – this is called pharmacokinetics.

Beyond safety, the study will also gently explore if MBF-015 might have any positive effects on the disease. They'll measure changes in things like movement, how well people can think and remember, and their overall ability to manage daily tasks. They will also look at certain markers in the blood and spinal fluid that can give clues about how the brain is doing. It's important to remember that because this is an early study, any improvements would just be preliminary, and much more research would be needed.

Key takeaways

This is an early study (Phase IIa) testing a new medicine, MBF-015, for Huntington's disease.
The main focus is to check if MBF-015 is safe and well-tolerated over 28 days.
Researchers will also look for early signs of how the medicine works in the body and if it helps symptoms.
Participation involves taking the medicine for 28 days and follow-up visits until around Day 43.
Both men and women aged 18 and older with Huntington's disease may be eligible.

Who may be eligible?

To join this study, you must be at least 18 years old. Both men and women are welcome to participate. The study is specifically for people who have been diagnosed with Huntington's disease.

There might be other health conditions or medications that would prevent someone from joining, as these could affect the study results or your safety. These details would be discussed fully by the study doctors after reviewing your medical history.

The research team will carefully check if this study is suitable for you based on your medical history and current health. It's really important that the study is the right fit for your safety and to make sure the research provides clear, accurate information.

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 Huntington's disease?
Are you able to take medicine by mouth (swallow tablets or capsules)?
Are you able to attend regular clinic visits for about 6 weeks?
Do you have a good understanding of what the study involves?

Answer every question to see your result.

What does participation involve?

If you decide to take part, you would be given the study medicine, MBF-015, to take by mouth for 28 days. During this time, and for a short period afterwards, you would have several visits to the study clinic. These visits would involve various checks to see how you are doing.

Assessments would include regular checks of your vital signs (like blood pressure and heart rate), physical examinations, and blood tests. You might also have ECGs (heart tracings) and tests to assess your movement, thinking skills (like memory and attention), and mood. Some advanced tests might include looking at your spinal fluid. The total time you'd be involved in the study, including the follow-up, would be about 43 days from when you start taking the medicine.

Potential risks and benefits

Taking part in a study like this might offer a potential benefit: you could receive a new medicine that isn't yet available and might help with your Huntington's disease. However, because this is an early-stage study, any benefits are not guaranteed and might be small or temporary. The main goal is to understand safety. Potential risks include experiencing side effects from the medicine, which could range from mild to more serious. There's also the time commitment involved with clinic visits and tests. You always have the right to withdraw from the study at any time, for any reason, without it affecting your usual medical care.

Locations (1)

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

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Unverified
Spain

Common questions

What is MBF-015?

MBF-015 is a new medicine being tested as a potential treatment for Huntington's disease. It's not yet approved and is in early stages of research.

How long will I take the medicine?

You would take the study medicine, MBF-015, for 28 days.

What happens after I stop taking the medicine?

After you finish taking the medicine, you will have a follow-up visit around Day 43 to check on your health and any effects from the medicine.

Why do they take so many measurements?

Researchers take many measurements to carefully check the medicine's safety, how it works in your body, and if it has any early effects on your Huntington's symptoms.

Can I stop participating if I want to?

Yes, you can choose to stop participating in the study at any time, for any reason, without needing to explain why.