A trial of panobinostat with azacitidine for acute myeloid leukaemia, chronic myelomonocytic leukaemia and myelodysplastic syndromes

Please note - this trial is no longer recruiting patients. We hope to add results when they are available.

Cancer type:

Acute leukaemia
Acute myeloid leukaemia (AML)
Blood cancers
Myelodysplastic syndrome (MDS)




Phase 1/2

This trial is looking a drug called panobinostat alongside the chemotherapy drug azacitidine, for certain types of leukaemia Open a glossary item and myelodysplastic syndromes. It is for people who can’t have a stem cell transplant.

Leukaemia is cancer of the white blood cells.  Myelodysplastic syndrome (MDS) is a group of conditions that affect the bone marrow Open a glossary item. MDS can sometimes turn into a type of leukaemia.

The people taking part in this trial have 1 of the following

Doctors can treat leukaemia or high risk MDS with chemotherapy.  Azacitidine (also known as Vidaza) is a drug they may use.

Everybody taking part in this trial has azacitidine. Some people also have a drug called panobinostat (also known as LBH589). Panobinostat blocks enzymes Open a glossary item called deacetylases (pronounced dee-as-et-isle-azes). Cells need these enzymes to grow and divide. Blocking them may stop leukaemia cells growing.

The aim of the trial is to see if the combination of azacitidine and panobinostat helps people with high risk MDS, chronic myelomonocytic leukaemia or acute myeloid leukaemia, more than azacitidine alone.

Who can enter

You may be able to enter this trial if you have 1 of the following

And, as well as the above, you

  • Have satisfactory blood test results
  • Are well enough to be up and about for at least half the day (performance status 0, 1 or 2)
  • Are willing to use reliable contraception during the trial and for 3 months afterwards if there is any chance you or your partner could become pregnant

You cannot enter this trial if you

  • Are going to have a stem cell transplant
  • Have MDS or AML that was caused by having another type of treatment
  • Have AML that has not responded to another type of treatment or has come back after having treatment
  • Have had other treatments (apart from a drug called hydroxyurea) in the last 4 weeks
  • Are having another experimental treatment
  • Have symptoms that your doctors think may be caused by having leukaemia cells in your brain or spinal cord
  • Have already had azacitidine
  • Have had another drug that blocks deacetylases
  • Have had a heart attack in the last 6 months, have certain other heart problems or need to take other medication that can affect your heart – the study doctors can advise you about this
  • Have problems with your digestive system Open a glossary item that affect how you swallow or absorb tablets
  • Have symptoms caused by bleeding inside your body
  • Have another serious medical condition
  • Are HIV, hepatitis B or hepatitis C positive
  • Are pregnant or breastfeeding

If your doctors think you may be very sensitive to azacitidine or to a drug called mannitol, you will not be able to take part in this trial.

Trial design

This phase 1/2 trial is in 2 parts. In the 1st part of the trial, researchers found the highest safe dose of panobinostat that you can have at the same time as azacitidine.

The 2nd part of the trial is randomised. The people taking part are put into treatment groups by a computer. Neither you nor your doctor can decide which group you are in.

Half the people taking part have azacitidine and the highest safe dose of panobinostat that was found in part 1. The other half have azacitidine alone.

Panobinostat with azacitidine for aml cmml and mds trial diagram

Everybody has 4 week cycles of treatment. You have azacitidine as an injection under your skin (subcutaneous injection) once a day for the 1st week of each treatment cycle (you don’t have any azacitidine for the next 3 weeks).

If you are in the group having panobinostat, you take this as capsules that you swallow. You take them on 7 days in each cycle of treatment. The trial team will tell you when to take the capsules.

As long as you don’t have bad side effects, you can carry on having the treatment for as long as it helps you.

Hospital visits

You see the trial team and have some tests before you start treatment. The tests include

  • Physical examination
  • Blood tests
  • Bone marrow test
  • Urine tests
  • Heart trace (ECG Open a glossary item)
  • Heart ultrasound (echocardiogram Open a glossary item)

In the 1st cycle of treatment, you go to hospital

  • Every day in the 1st week
  • 3 times in the 2nd week
  • Twice in the 3rd week

In each of the following treatment cycles, you go to hospital

  • Every day in the 1st week
  • Once in the 2nd week
  • Twice in the 3rd week

When you finish the trial treatment, you go back to see the trial team 4 weeks later. You then see them every 3 months for a year and every 6 months after that.

Side effects

As panobinostat is a new drug, there may be side effects we don’t know about yet. The most common known side effects include

The trial doctor will give you more information about other possible side effects before you agree to join the trial. It is possible that side effects may be worse when you have panobinostat alongside azacitidine.

We have more information about the side effects of azacitidine in our cancer drugs section.

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.

Please note - unless we state otherwise in the summary, you need to talk to your doctor about joining a trial.

Chief Investigator

Dr Jamie Cavenagh

Supported by

Experimental Cancer Medicine Centre (ECMC)
National Institute for Health Research Cancer Research Network (NCRN)

Questions about cancer? Contact our information nurses

Freephone 0808 800 4040

Last review date

CRUK internal database number:

Oracle 9435

Please note - unless we state otherwise in the summary, you need to talk to your doctor about joining a trial.

Wendy took part in a new trial studying the possible side effect of hearing loss

A picture of Wendy

"I was delighted to take part in a clinical trial as it has the potential to really help others in the future.”

Last reviewed:

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