"I was delighted to take part in a clinical trial as it has the potential to really help others in the future.”
A trial of panobinostat with azacitidine for acute myeloid leukaemia, chronic myelomonocytic leukaemia and myelodysplastic syndromes
This trial looked at a drug called panobinostat alongside the chemotherapy drug azacitidine, for certain types of leukaemia and myelodysplastic syndromes. It was for people who couldn’t have a stem cell transplant.
More about this trial
Leukaemia is cancer of the white blood cells. Myelodysplastic syndrome (MDS) is a group of conditions that affect the bone marrow. MDS can sometimes turn into a type of leukaemia.
The people taking part in this trial had 1 of the following:
- a type of leukaemia called acute myeloid leukaemia (AML)
- myelodysplastic syndrome (MDS) that had a medium to high risk of turning into leukaemia
- chronic myelomonocytic leukaemia (CMML)
Doctors can treat leukaemia or high risk MDS with chemotherapy. Azacitidine is a drug they may use. But doctors want to improve treatment. In this trial, they looked at adding a drug called panobinostat to azacitidine.
Panobinostat blocks enzymes called deacetylases. Cells need these enzymes to grow and divide. Blocking them may stop leukaemia cells growing.
Everybody taking part had azacitidine. And some people also had panobinostat (also known as LBH589).
The aims of the trial were to:
- see how well treatment worked
- learn more about the side effects
Summary of results
The trial team found that adding panobinostat to azacitidine didn’t work better than azacitidine alone. And the side effects were worse.
The researchers published the results in 2017. This phase 1/2 trial took place in the UK. 113 people took part in total.
The trial was in 2 parts. In the 1st part of the trial, researchers found the highest safe dose of panobinostat alongside azacitidine. 31 people joined this part.
In the 2nd part of the trial, people were put into treatment groups at random. 82 people joined and:
- 40 had panobinostat and azacitidine
- 42 had azacitidine
The trial team looked at how well treatment worked. To do this they looked at:
- how long before the cancer got worse
- how many people were alive at 1 year (overall survival)
They didn’t find a difference between the 2 groups. So, having panobinostat alongside azacitidine didn’t work better than azacitidine alone.
People who had panobinostat and azacitidine had worse side effects. The most common were a drop in the number of blood cells causing:
- bleeding and bruising
- an increased risk of infection
- tiredness and breathlessness
2 people who had panobinostat and azacitidine died because of a serious side effect. The causes were:
- a complication of a serious infection (septic shock)
- a major bleed
The trial team concluded that panobinostat and azacitidine didn’t work better than azacitidine alone.
This trial has increased knowledge about what works and what doesn’t for acute myeloid leukaemia, chronic myelomonocytic leukaemia and myelodysplastic syndromes.
How to join a clinical trial
Dr Jamie Cavenagh
Experimental Cancer Medicine Centre (ECMC)
NIHR Clinical Research Network: Cancer