A trial looking at a PARP-1 inhibitor for advanced cancer, particularly in people with a BRCA gene mutation

Cancer type:

All cancer types




Phase 1

This trial was looking at a new treatment called AZD2281 for advanced solid tumours. More than a third of the people who took part in this trial had a solid tumour Open a glossary item with a BRCA gene fault.

PARP-1 is an enzyme Open a glossary item that helps damaged cells to repair themselves. PARP stands for “poly ADP-ribose polymerase” and is found in some cancer cells.

Doctors hope that if they can stop PARP-1 working, the cancer cells will not be able to repair themselves and will die. This type of treatment is called a ‘PARP-1 inhibitor’. The PARP-1 inhibitor in this trial is called AZD2281. It was formerly known as KU-0059436 and is also called olaparib.

The main aims of this trial were to find out

  • The best dose of AZD2281 to give
  • What happens to AZD2281 in the body
  • What the side effects are
  • How well AZD2281 worked

Summary of results

The trial team found that AZD2281 can have an effect on cancer in people with a BRCA gene fault, without causing too many side effects.

The trial recruited 60 people. 22 of them were known to have a BRCA gene fault.

The first few people had a low dose of AZD 2281. As they didn’t have too many bad side effects, the next few people had a higher dose and so on until the researchers worked out the best dose to give. This is called a dose escalation study.

The side effects that people did have included

Side effects were no worse in the group of people with the BRCA gene fault.

The researchers learnt more abut how the drug worked in the body and found that it had an effect on cancer in people with the BRCA gene fault. The cancer got smaller in nearly half the people with the gene fault. But cancers did not respond to AZD2281 in the other people taking part in this trial.

The researchers found a safe dose of AZD2281 to give that did not cause too many side effects. And they found that this drug had some effect on cancers in people with a BRCA gene fault.

We have based this summary on information from the team who ran the trial. 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. 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

Prof Johann de Bono

Supported by

Breakthrough Breast Cancer
Experimental Cancer Medicine Centre (ECMC)
KuDOS Pharmaceuticals Ltd

Questions about cancer? Contact our information nurses

Freephone 0808 800 4040

Last review date

CRUK internal database number:

Oracle - 615

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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