Active not recruitingPHASE2INTERVENTIONAL

Trial of Efficacy and Safety of MC0518 Versus Best Available Therapy in Participants With Steroid-Refractory Acute Graft Versus Host Disease

This research study is for children and young people who have a condition called 'steroid-refractory acute graft-versus-host disease' (SR-aGvHD). This condition can happen after a bone marrow transplant when the new immune cells attack the patient's body. 'Steroid-refractory' means that usual steroid medicines haven't worked well enough. The study wants to compare a new medicine, MC0518, with the treatments doctors normally use to see which is more effective at improving the condition. Researchers will check how well patients respond to the treatments 28 days after they start. This will help doctors understand if MC0518 could be a better option for these patients.

At a glance

Status

Active not recruiting

Phase

PHASE2

Sponsor

medac GmbH

Enrolment target

Start

13 Nov 2023

Estimated completion

01 Jun 2031

Steroid-refractory Acute Graft-versus-host Disease

What is this study about?

This study is designed to help children and young people who have a serious complication after a bone marrow or stem cell transplant. This complication is called 'acute graft-versus-host disease,' or aGvHD for short. It happens when the new healthy cells from the donor start to see the patient's body as foreign and attack it. It can affect different parts of the body, like the skin, liver, or gut.

Usually, doctors first try to treat aGvHD with steroid medications. However, sometimes these steroids don't work well enough, or the disease gets worse. When this happens, it's called 'steroid-refractory acute graft-versus-host disease' (SR-aGvHD). This is a very challenging situation for patients and their families.

That's where this study comes in. Researchers are testing a new medicine called MC0518. They want to compare how well MC0518 works against the standard treatments doctors usually give in this situation. By doing this, they hope to find out if MC0518 could be a safer and more effective way to help children and young people who are struggling with SR-aGvHD.

Key takeaways

This study is testing a new medicine (MC0518) for children and young people.
It's for a serious condition called acute graft-versus-host disease (aGvHD) that hasn't responded to steroids.
The new medicine is compared to standard treatments to see which works best.
The main goal is to see if the condition improves by Day 28 after starting treatment.
Participation involves regular checks and potentially new medicines that might help.
You can stop taking part at any time without affecting your normal care.

Who may be eligible?

This study is looking for children and young people who are between 28 days old and 17 years old, and weigh at least 3.2 kilograms (about 7 pounds). They must have had a bone marrow or stem cell transplant in the past, either for a serious blood condition, a problem with their immune system, some genetic conditions, or certain cancers.

Crucially, participants must have been diagnosed with aGvHD that is considered 'Grade II to IV', meaning it's moderately to severely affecting them. The main requirement for joining the study is that their aGvHD has not responded well to standard steroid treatment. This means their condition either got worse, didn't improve enough, or came back, even after receiving steroids as prescribed. The study team will look closely at their medical records to confirm this.

There are also some important safety rules. For example, if a female participant could become pregnant, or a male participant could father a child, they would need to agree to use effective birth control throughout the study to prevent pregnancy. This is to protect any potential unborn babies from exposure to the study medicine, as its effects on pregnancy are not yet known.

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 between 28 days and 17 years old?
Have you had a bone marrow or stem cell transplant?
Do you have acute graft-versus-host disease (aGvHD)?
Has your aGvHD not improved with steroid treatment?
Do you (or your parents/guardians) understand the need for contraception rules during the study, if applicable?

Answer every question to see your result.

What does participation involve?

If you decide to take part in this study, you would first have a `Screening Visit` to check if you meet all the requirements. If you do, you would then be randomly assigned to receive either the new medicine, MC0518, or the best available standard treatment. You wouldn't be able to choose which one you receive, and neither would your doctor – it's like flipping a coin to make sure the study is fair.

You would receive treatment and have several appointments and checks, especially in the first 28 days, to see how your body is responding to the medicine. The doctors and nurses would regularly check your health, take blood tests, and ask how you are feeling. The main goal is to see if your aGvHD improves by day 28. The total length of your participation would depend on how long you need treatment and follow-up, but the most important early assessment happens at Day 28.

Potential risks and benefits

Participating in any research study has potential benefits and potential risks. A potential benefit of this study is that you might receive a new treatment, MC0518, which could be more effective than current standard treatments for severe aGvHD that isn't responding to steroids. However, as with any medication, MC0518 could have side effects, and these would be carefully explained to you by the study team. You would also receive treatments that are already in use, which also have known risks and benefits. It's important to remember that you are free to leave the study at any time, for any reason, without it affecting your usual medical care.

Locations (36)

CHU de Bordeaux - Hopital des Enfants
Verified postcode
Bordeaux, France
CHU Grenoble Alpes - Hopital Couple Enfant (HCE)
Verified postcode
La Tronche, France
Centre Hospitalier Universitaire de Lille CHU Lille - Hopital Jeanne de Flandre HJF
Verified postcode
Lille, France
Institut d'Hematologie et d'Oncologie Pediatrique (IHOPe)
Verified postcode
Lyon, France
CHU de Marseille-Hopital de la Timone
Verified postcode
Marseille, France
Centre Hospitalier Regional Universitaire (CHRU) Montpellier - hopital Arnaud de Villeneuve
Verified postcode
Montpellier, France
CHU de Nantes - Hopital Mere Enfant
Verified postcode
Nantes, France
Hopital Robert Debre
Verified postcode
Paris, France
CHU de Rouen - Hopital Charles Nicolle
Verified postcode
Rouen, France
CHRU de Strasbourg - Hopital de Hautepierre
Verified postcode
Strasbourg, France
CHRU Nancy, Hopitaux de Brabois
Verified postcode
Vandœuvre-lès-Nancy, France
Uniklinik RWTH Aachen, Klinik fur Kinder- und Jugendmedizin
Verified postcode
Aachen, Germany

Common questions

What is aGvHD?

aGvHD stands for 'acute graft-versus-host disease.' It's a complication that can happen after a bone marrow or stem cell transplant when the new cells attack the patient's body.

What does 'steroid-refractory' mean?

It means that the usual steroid medicines, which are often the first treatment for aGvHD, haven't worked well enough to control the condition.

Will I definitely get the new medicine?

You have an equal chance of receiving either the new medicine (MC0518) or the best standard treatment. The choice is made randomly, like drawing lots.

How long will I be in the study?

The study will closely check your progress, especially for the first 28 days, to see how your condition responds. The overall duration you're involved depends on your treatment and follow-up needs, but the main goal is to assess improvement by Day 28.

Can I stop participating in the study?

Yes, you can choose to leave the study at any time, for any reason. Your decision will not affect your standard medical care.