CompletedNot ApplicableInterventional

Investigating the use of lenzilumab for acute graft versus host disease (when transplanted stem cells attack the body’s cells)

A stem cell transplant can save lives, but sometimes the donor's cells attack the patient's healthy tissues. This reaction is called acute graft versus host disease (GvHD). Doctors usually treat this with steroids, but steroids do not work well for everyone. This trial is looking at a new targeted medicine called lenzilumab. It blocks a specific protein involved in inflammation. Researchers will test patients' blood to find out their risk level. Those at higher risk will receive either lenzilumab or standard care (which may include a dummy treatment called a placebo) alongside steroids. People at lower risk will simply be monitored to see how they do on standard care alone. The aim of the study is to find out if lenzilumab is safe, reduces symptoms, and improves quality of life for people with high-risk acute GvHD.

At a glance

Status

Completed

Phase

Not Applicable

Sponsor

University of Birmingham

Enrolment target

590

Start

01 Jan 2022

Estimated completion

01 Jan 2025

Graft versus host disease following allogeneic stem cell transplantation

What is this study about?

A stem cell transplant is an important treatment for blood cancers and other serious conditions. However, after receiving cells from a donor, some patients develop a complication known as acute graft versus host disease, or GvHD. This happens when the new immune cells from the donor view the patient's body as unfamiliar and begin to attack healthy organs, such as the skin, gut, or liver.

Steroids are the usual first-line treatment for acute GvHD, but they do not work for every patient. When steroids fail, GvHD can become very difficult to manage. Recently, scientists discovered that a protein called GM-CSF plays a major part in driving this harmful immune reaction. Lenzilumab is a new medicine designed to block this protein and calm down the attack without shutting down the entire immune system.

In this study, researchers want to see whether using a blood test to check a person's risk score can guide better treatment. By identifying who is at higher risk of their GvHD not responding to steroids, doctors hope that adding lenzilumab early on will give patients the best chance of recovery. If successful, this trial could lead to kinder, more effective options for people facing this challenging condition.

Key takeaways

Tests a new medicine called lenzilumab to treat acute graft versus host disease (GvHD).
A special blood test helps doctors decide which study group is right for you.
Open to patients aged 16 and over who have received a donor stem cell transplant.
Eligible treatment participants receive 6 doses through a drip over a period of 14 weeks.
You will continue to receive standard steroid care while taking part.

Who may be eligible?

This trial is open to people aged 16 and over who have had a stem cell transplant from a donor (an allogeneic transplant) and have developed acute GvHD. Participants must need steroid medicine that travels through the whole body, either as tablets or through a drip, and must be able to carry out at least some daily activities.

Patients will have a blood test that checks specific biological markers to measure their risk level. Depending on whether their score is high or low, they may join the group receiving the trial drug or an observational group that only monitors their progress on standard steroid treatment.

You cannot take part if you only need steroid skin creams, if you have already been on high-dose steroids for GvHD for more than four days, or if you are receiving other new treatments for GvHD. People with active hepatitis B, hepatitis C, or HIV are not able to join. Women who are pregnant or breastfeeding cannot take part, and anyone who could become pregnant or father a child must agree to use reliable contraception during the study.

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.

I am aged 16 or over.
I have had a stem cell transplant using cells from a donor.
I have been diagnosed with acute GvHD and need steroid tablets or drips.
I have not been taking steroids for this GvHD flare-up for more than four days.
I am not pregnant or breastfeeding, and I am willing to use effective birth control if applicable.

Answer every question to see your result.

What does participation involve?

If you join the treatment part of this study, you will receive up to 6 doses of the trial medicine (or a placebo) through a drip into a vein. Doses are given over 14 weeks at set intervals: week 0, week 2, week 5, week 8, week 11, and week 14. If your blood test shows you are at lower risk, you may join an observational group where you receive standard care and no trial medicine, but the team will closely follow your health. During study visits, doctors and nurses will check your vital signs, carry out physical examinations, look closely at any GvHD symptoms, take routine blood tests, and ask you to fill in short questionnaires about how you are feeling and your overall quality of life.

Potential risks and benefits

There is no guarantee that taking part will improve your condition, especially as some participants may receive a dummy medicine (placebo) alongside their regular treatment. However, the results could help doctors provide better care for stem cell transplant patients in the future. As with any drug, lenzilumab may cause side effects, including some that doctors do not yet know about. Having blood taken or receiving an intravenous drip can also cause brief pain, mild bruising, or light-headedness. Your hospital team will monitor you closely at every step to keep you safe, and you are free to leave the trial at any time without giving a reason and without it affecting your regular NHS care.

Locations (17)

Gartnavel Royal Hospital
Approximate
Glasgow, Scotland
King's College Hospital
Approximate
London, England
St James's University Hospital
Approximate
Leeds, England
St Mary's Hospital
Approximate
Manchester, England
Freeman Hospital
Approximate
Newcastle upon Tyne, England
John Radcliffe Hospital
Unverified
Oxford, England
Queen Elizabeth Hospital
Unverified
Birmingham, England
The Royal London Hospital
Unverified
London, England
Bristol Royal Infirmary
Unverified
Bristol, England
University College London Hospital
Unverified
London, England
Addenbrookes Hospital
Unverified
Cambridge, England
University Hospital of Wales
Unverified
Cardiff, Wales

Common questions

What is a placebo and will I definitely get the new drug?

A placebo is a dummy treatment that looks identical to the real medicine but contains no active ingredients. In the randomised stage of the trial, participants are chosen by chance (like flipping a coin) to receive either lenzilumab or the placebo, alongside their standard steroid treatment.

Do I have to stop taking my usual medicines?

No, you will continue with your standard steroid treatment and any usual preventive medicines your transplant team has prescribed. However, you cannot start other new experimental treatments for GvHD while in the trial.

How is the trial drug given?

Lenzilumab (or the placebo) is given as a slow drip into a vein (an intravenous infusion) at your hospital appointments.

What happens if my acute GvHD gets worse?

Your medical team will monitor your symptoms very closely. If your condition does not improve or gets worse, your doctors can change your treatment immediately to keep you safe.

Can I leave the study if I change my mind?

Yes. Taking part is completely voluntary. You can choose to withdraw from the trial at any time, and your normal medical care will continue as usual.

How to find out more

Rebecca Collings

RATinG@trials.bham.ac.uk +44 (0)121 371 7868 Official trial record

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