Aim of the trial:
To see if new combinations of drugs are better at preventing graft vs host disease (GvHD) following an allogeneic stem cell transplant than current standard treatments.
Why is the trial important?
GvHD is a very common side effect of having a stem cell transplant that can affect patients at different times in their recovery. It’s caused when the donor’s immune cells recognise the patient’s own cells as different and start to attack them. It can often be successfully managed using a combination of drugs, steroids and other treatments which control the immune system.
However, some patients experience GvHD which doesn’t respond to standard treatments so there is a great need to develop new alternatives. This includes treatments that prevent the onset of GvHD altogether.
Many treatments used to control GvHD work by controlling the effects of the immune system, which means they stop your donor’s cells attacking your own cells. White blood cells called T cells are believed to be the main cell responsible for doing this, so new drugs that stop T cells working properly are also being developed to treat GvHD.
Who can take part?
Adult patients who are due to undergo an allogeneic stem cell transplant and have one of the following types of cancer:
- Acute Myeloid Leukaemia (AML)
- Acute lymphoblastic leukaemia (ALL)
- Chronic myelomonocytic leukaemia (CMML)
- Myelodysplastic syndromes (MDS)
- Non-Hodgkin lymphoma (NHL)
- Hodgkin lymphoma (HL)
- Multiple myeloma (MM)
- Chronic lymphocytic leukaemia (CLL)
- Chronic myeloid leukaemia (CML)
What will happen to me?
Following their stem cell transplant, patients will be randomly placed into one of three different groups. Each group will be given treatments to prevent GvHD developing. This will either be the current NHS standard, or one of two new treatment combinations being tested to see if they are more effective. The three treatments being tested are:
- Two chemotherapy drugs called ‘cyclophosphamide’ and ‘sirolimus’ (rapamycin) that are both used to control your immune system.
- Cyclophosphamide and a calcineurin inhibitor, which is a used to stop T cells functioning.
- Thymoglobulin, the standard treatment used to reduce the number of T cells in the blood.
How will we know if the trial is successful?
The number of patients who do not relapse and remain GvHD free one year after transplant will be used to decide if the new treatments are more effective.
The trial will also consider other factors including how many patients require a donor lymphocyte infusion (DLI) and how many develop infections, plus quality of life measures to determine if the new treatments give significant benefits to patients compared to the standard treatment.
Stage: Phase 2
Study lead: Professor Ronjon Chakraverty
Recruiting: 400 patients over four years
Time: Five years, patients will be followed up for a minimum of one year
Information published: 10/05/21
Next review due: 03/09/23