A trial of CXD101 for advanced cancer

Cancer type:

All cancer types




Phase 1/2

This trial looked at a drug called CXD101 to treat cancer. It was for people who had a solid tumour Open a glossary item or lymphoma Open a glossary item that had:

  • got worse despite treatment and 
  • there wasn’t a standard treatment  Open a glossary itemavailable

This trial was open for people to join between 2018 and 2019. The team published the results in 2021.

More about this trial

CXD101 is a drug that blocks substances (enzymes Open a glossary item) in the body called histone deacetylases. Cells need these to grow and divide. Blocking them may stop cancer growing. Drugs that block these enzymes are called histone deacetylase inhibitors or HDAC inhibitors.

At the time of this trial, doctors knew from early research that CXD101 may be able to stop cancer cells growing. They weren’t sure how well it would work so they ran this trial to find out more. 

The main aims of this trial were to:

  • find the best dose of CXD101 to give 
  • see if it works for people with advanced cancer Open a glossary item

Summary of results

The team found the best dose of CXD101 to give and that the side effects were manageable. In this very small trial, they also found that CXD101 worked for a few people who had lymphoma. 

Trial design
This was a phase 1/2 trial. A total of 47 people had treatment:

  • 19 people had a solid tumour
  • 28 people had lymphoma 

The trial was in 2 parts. Part 1 looked at the best dose to have. Part 2 tested this dose in more people.

Results for part 1
30 people joined this part of the trial. The team found the best dose of CXD101 to give. The first few people had a low dose of CXD101. If they didn’t have any serious side effects, the next few people had a higher dose. This is called a dose escalation study. 

Results for part 2
17 people joined part 2 of the trial. The team looked at how well treatment worked. 

For those who had a solid tumour, the cancer:

  • didn’t go away or get smaller in anyone 
  • stayed the same in 4 people
  • got worse in 5 people 

For those who had lymphoma, it:

  • went away in 1 person
  • shrunk a bit in 3 people
  • stayed the same in 8 people 
  • got worse in 8 people 

Samples for research 
The team used tissue samples to look for a biomarker Open a glossary item called HR23B. They thought this would help them to work out who CXD101 would work best for. They found this biomarker wasn’t useful to predict this.  

Side effects
Everyone had at least one side effect. Most were mild but some were more severe. 

The most common severe side effects of CXD101 included:

  • a drop in the number of blood cells causing an increased risk of infection, bleeding and bruising, tiredness and breathlessness
  • tiredness (fatigue)

The trial team concluded that CXD101 worked for a few people who had certain types of lymphoma. It didn’t shrink the cancer in anyone who had a solid tumour. Although the cancer stayed the same in some people with a solid tumour. 

The trial team recommend that CXD101 is looked at in combination with other cancer treatments for solid tumours. For example this might include having it with immunotherapy

We have based this summary on information from the research team. The information they sent us has been reviewed by independent specialists (peer reviewed Open a glossary item) and published in a medical journal. The figures we quote above were provided by the trial team who did the research. We have not analysed the data ourselves.

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

Professor Mark Middleton

Supported by

Celleron Therapeutics Ltd
Experimental Cancer Medicine Centre (ECMC)
Oxford University Hospitals NHS Trust

If you have questions about the trial please contact our cancer information nurses

Freephone 0808 800 4040

Last review date

CRUK internal database number:


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

Last reviewed:

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