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Ongoing, recruitingTherapeutic confirmatory (Phase III)Interventional

EvAluation of the efficacy of MaaT013 as salvage theRapy in acute GVHD patiEntS with gastrointestinal involvement, refractory to ruxolitinib; a multi-center open-label phase III trial.

This study is called 'EvAluation of the efficacy of MaaT013 as salvage theRapy in acute GVHD patiEntS with gastrointestinal involvement, refractory to ruxolitinib'. In simpler terms, it's testing a new medicine, MaaT013, for a condition called acute graft-versus-host disease (aGVHD) that affects the stomach and bowels. This particular study is for people whose aGVHD hasn't responded to, or they couldn't tolerate, a common treatment called ruxolitinib. It's a phase III trial, which means it's a later stage study designed to confirm how well the treatment works. Doctors will be checking to see if MaaT013 helps improve the aGVHD symptoms in the gut, and they'll also look at how safe it is for patients.

At a glance

Status
Ongoing, recruiting
Phase
Therapeutic confirmatory (Phase III)
Sponsor
MaaT PHARMA
Enrolment target
73
Start
18 Jul 2024

What is this study about?

You might be reading this because you or someone you know is dealing with acute graft-versus-host disease, often shortened to aGVHD. This serious condition can sometimes happen after a stem cell or bone marrow transplant, where the donated immune cells see the patient's body as 'foreign' and start to attack it. Our study focuses on aGVHD specifically when it affects the digestive system, like the stomach and intestines, which can cause symptoms like tummy pain, sickness, or diarrhoea.

For many patients, a medicine called ruxolitinib is used to treat aGVHD. However, for some, this treatment doesn't work well enough, or they might have side effects that make it difficult to continue. That's where this new study comes in. We are looking at a treatment called MaaT013, which is a new type of therapy. This study aims to find out if MaaT013 can be a helpful 'rescue' treatment for those who haven't improved with ruxolitinib.

This is a 'Phase III' study, which means it's a very important step. We're carefully examining how well MaaT013 works to ease the gut-related aGVHD symptoms and how safe it is for patients. Doctors will closely monitor participants to understand if this new treatment can make a real difference in their health and quality of life.

Key takeaways

  • This study is testing a new treatment called MaaT013 for gut-related aGVHD.
  • It's for people whose aGVHD hasn't responded to previous treatment with ruxolitinib.
  • The main goal is to see how well MaaT013 improves gut symptoms after 28 days.
  • Safety is also a key focus, with close monitoring of participants.
  • You would have regular hospital visits for check-ups and discussions.
  • Participation involves receiving MaaT013 and being closely monitored by doctors.

Who may be eligible?

This study is for adults generally aged 18 and older. Both men and women can take part.

The main requirement is that you have acute graft-versus-host disease (aGVHD) which is affecting your digestive system (your gut). This means you would be experiencing symptoms like stomach pain, sickness, or diarrhoea because of your aGVHD.

Crucially, your aGVHD must also not have improved, or you've had problems with, a standard treatment called ruxolitinib. This study is specifically for people who need other treatment options because ruxolitinib hasn't worked for them.

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.

  1. Are you 18 years old or older?
  2. Do you have acute graft-versus-host disease (aGVHD) affecting your gut?
  3. Has your aGVHD not improved with ruxolitinib, or could you not tolerate it?
  4. Are you willing to attend regular hospital visits for assessments?
  5. Are you comfortable with receiving the study medication MaaT013?
Answer every question to see your result.

What does participation involve?

If you decide to take part in this study, you will receive the study medicine called MaaT013. This won't be a random choice; everyone in this study will get MaaT013 because we are specifically testing this particular treatment. You'll have regular visits to your hospital or clinic. These visits will involve blood tests, physical check-ups, and discussions about how you're feeling and any symptoms you might be experiencing. Doctors will keep a close eye on your aGVHD symptoms, especially those in your gut. They will also track any changes in your overall health and how you tolerate the study medicine. The main period of the study will focus on assessing your response to MaaT013 after about 28 days, and then again at 56 days and three months. There will also be longer-term follow-up to understand the lasting effects of the treatment and your health over time. The total duration of your involvement could extend for several months, with different follow-up schedules.

Potential risks and benefits

Taking part in any medical study has potential benefits and risks. You might benefit from receiving a new treatment that could help improve your acute GVHD symptoms, especially if other treatments haven't worked for you. This study also helps us learn more about this condition and potential new ways to treat it, which could help future patients. However, there's always a chance that the new medicine may not work for you, or you might experience side effects. The study team will explain all known side effects and monitor you closely for any new ones. You are free to withdraw from the study at any time, for any reason, without it affecting your usual medical care.

Locations (6)

Some site locations are approximate. We're improving this — please verify with the trial team before travelling.
  • Unverified
    Germany
  • Unverified
    Belgium
  • Unverified
    Italy
  • Unverified
    France
  • Unverified
    Austria
  • Unverified
    Spain

Common questions

What is aGVHD?

Acute graft-versus-host disease (aGVHD) is a complication that can happen after a stem cell or bone marrow transplant, where the new immune cells attack your body.

What does 'gastrointestinal involvement' mean?

This means your aGVHD is affecting your digestive system, such as your stomach or intestines, causing symptoms like sickness or diarrhoea.

What is ruxolitinib?

Ruxolitinib is a common medicine used to treat aGVHD. This study is for people who haven't improved with it.

What is MaaT013?

MaaT013 is the new treatment being tested in this study. It's a type of therapy aiming to help with gut-related aGVHD.

Will I definitely get MaaT013 if I join?

Yes, in this study, all participants will receive the MaaT013 treatment. It's not a study where some people get a dummy treatment.

How to find out more

Always speak to your GP or specialist before deciding to take part in a study.

Discussion

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