A trial of lenzilumab for acute graft versus host disease (RATinG)
Cancer type:
Status:
Phase:
This is a trial of lenzilumab for people with high risk acute graft versus host disease.
There are 2 types of graft versus host disease (GvHD):
- acute GvHD (aGvHD)
- chronic GvHD (cGvHD)
This trial is open to people with acute GvHD who:
- have had a stem cell transplant from another person (
allogeneic transplant 
) and - are to have treatment with
steroids to treat the acute GvHD
You pronounce lenzilumab as lens-ill-lu-mab.
More about this trial
Graft versus host disease (GvHD) is a possible complication after a stem cell transplant from another person. This is called a donor transplant or an allogeneic transplant. It happens when the of the donated stem cells attack your own body cells.
One of the main treatments for GvHD are steroids. Unfortunately steroids don’t work for some people with aGvHD. So researchers are always looking for new treatments for these people.
Lenzilumab is a type of called a monoclonal antibody. It works by targeting a protein called granulocyte-monocyte colony stimulating factor (GM-CSF) and blocking its activity. GM-CSF controls the growth of blood cells and is important in the development of aGvHD.
Doctors think lenzilumab will help people who have high risk GvHD. This means the acute GvHD is more likely to be severe. You have a blood test as part of this trial to see if you have high or low risk acute GvHD.
There are 2 parts to this trial. In the first part of the trial they want to find out if it is safe to give lenzilumab to people with high risk aGvHD. If they find it is, then they will go on to the second part of the trial.
In the second part of the trial there are 2 groups. The:
- treatment group
- observation group
In this part the team want to find out how well lenzilumab works for people with high risk aGvHD. To find this out half the people will have lenzilumab and the other half will have a dummy drug ().
The aims of the trial are to find out:
- whether it is safe to give lenzilumab to people with high risk aGvHD
- how well lenzilumab works for people with high risk aGvHD
Who can enter
The following bullet points are a summary of the entry conditions for this trial. Talk to your doctor or the trial team if you are unsure about any of these. They will be able to advise you.
| There are 2 groups in this trial. The observation group and the treatment group. Apart from the general entry conditions each group has certain entry conditions. |
Who can take part
You may be able to join this trial if all of the following apply. You:
- have had a stem cell transplant from a donor (an allogenic transplant)
- are to have treatment with steroids either taken by mouth such as tablets or as a drip into a vein (infusion) to treat your aGvHD
- are up and about for some of the day and might need help looking after yourself (performance status 0, 1,2 or 3)
- are at least 16 years old
For the treatment group
The following must also apply. You
- have a high risk of developing severe aGVHD. Your doctor will know this.
- are willing to use reliable contraception during treatment and for a time after if there is any chance you or your partner could become pregnant.
For the observation group
You have a low risk of developing severe aGVHD. Your doctor will know this.
Who can’t take part
You cannot join this trial if any of these apply. You:
- are taking other medications for GvHD. This is apart from medications that you were taking before the transplant to prevent GvHD or starting those medications again after the transplant. Your doctor will know about this.
- are only using steroid cream for GvHD
- have had more than 4 days of steroid tablets or through a drip into a vein to treat acute GvHD. Or up to a total dose of 20mg if you are taking steroids for another condition. Your doctor will know about this.
- have HIV
- have active hepatitis B or hepatitis C that needs treatment
- are pregnant or breastfeeding
Trial design
This is a phase 2/3 trial. There are 2 parts to the trial.
Part 1 - open to recruitment
In this part the team need 20 people with high risk acute GvHD (aGvHD). Everyone will have lenzilumab and steroids.
You have lenzilumab as a drip into a vein. You have the first treatment within 7 days of starting steroids for your aGvHD. You have:
- the second treatment 2 weeks later and then
- the remaining 4 treatments every 3 weeks
You have a total of 6 doses. This takes about 14 weeks.
The team want to know if it is safe to give lenzilumab to people with high risk aGvHD.
Part 2 – currently not open to recruitment
There are 2 groups in this part. They are the:
- treatment group
- observation group
Treatment group
The team need 220 people with high risk aGvHD to join.
It is a randomised trial. A computer puts you into a group. Neither you nor your doctor can choose which group you go into. And neither you nor your doctor will know which group you are in. This is a double blinded trial.
Your doctor will be able to find out which group you are in if it is necessary to do so.
You have one of the following:
- lenzilumab
- a dummy drug (placebo)
You have lenzilumab or the placebo as a drip into a vein. You have the first dose within 7 days of starting steroids for your aGvHD. You have:
- the second treatment 2 weeks later and then
- the remaining 4 treatments every 3 weeks
You have a total of 6 doses. This takes about 14 weeks.
Observation group
The team want to recruit as many people as they can to this group. You see the doctor in the clinic as part of the trial. You don’t have any treatment as part of this trial.
Samples for research for everyone
The team take a blood sample when you agree to take part in the trial. This is to look for a substance in the blood () that tells the team if you are a low risk or high risk GvHD.
They then take another blood sample at 4 weeks to look at the same biomarker.
If there is any remaining blood from the samples, the team will ask to store this away for future research. You don’t have to agree to your blood samples being stored. You can still take part in the trial.
Quality of life – people in part 1 and treatment group
You fill in a questionnaire:
- before starting treatment
- during treatment and
- after treatment
The questions ask about:
- your general health and wellbeing
- side effects
- what you can do in your daily life
This is a quality of life questionnaire.
Hospital visits
Everyone sees the doctor for tests before you take part. These tests include:
- a
physical examination - blood tests
- assessment of your GvHD
People in part 1 and treatment group
You see the doctor before each treatment. This is to see how you are and for blood tests.
After treatment you see the doctor for the same tests at:
- 1 month
- 2 months
- 3 months
You see the doctor every year until the end of the trial. This is to see how you are.
Observation group
You then see the doctor for the same tests at:
- 1 month
- 6 months
You see the doctor every year until the end of the trial This is to see how you are.
Side effects
People in part 1 and treatment group
The trial team monitor you during treatment and afterwards. Contact your advice line or tell your doctor or nurse if any side effects are bad or not getting better.
The possible side effects of lenzilumab are:
- a reaction while having the infusion. Symptoms include feeling sick, low blood pressure, pressure on the chest, changes to heart rate, shortness of breath, fever, headache, chills, difficulty breathing and skin changes.
- a drop in the
white blood cells causing an increased risk of infection - infection
- changes to how well your lungs work
We have information about steroids and their side effects.
Your doctor will talk to you about the possible side effects of the treatments and answer questions you might have before you agree to take part.
Location
Recruitment start:
Recruitment end:
How to join a clinical trial
Please note: In order to join a trial you will need to discuss it with your doctor, unless otherwise specified.
Chief Investigator
Professor Adrian Bloor
Supported by
University of Birmingham
IMPACT Partnership
Humanigen Inc
If you have questions about the trial please contact our cancer information nurses
Freephone 0808 800 4040
