A trial of sonidegib for advanced cancer that has got worse despite other treatment

Cancer type:

All cancer types




Phase 1

This trial looked at a drug called sonidegib (also known as LDE225) for solid tumours that have spread to another part of the body. A solid tumour is any type of cancer apart from leukaemia Open a glossary item or lymphoma Open a glossary item.

Sonidegib is a type of biological therapy. It is a cancer growth blocker. It stops the signals that cancer cells use to divide and grow.

This was the first time that sonidegib was given to patients. The people taking part had different types of advanced cancer that had got worse despite having other treatments. The aims of the trial were to

  • Find the best dose of sonidegib to give
  • Learn more about the side effects and what happens to sonidegib in your body
  • See how well it works for advanced cancer

Summary of results

The trial team found the best dose of sonidegib to give. They also found that it helped some people with a type of skin cancer called basal cell cancer (BCC) and some people with a type of brain tumour called medulloblastoma.

103 people with advanced cancer took part in the trial. The first few people had a low dose of sonidegib. As they didn’t have bad side effects, the next few people had a higher dose. And so on, until the research team found the best dose to give. This type of trial is called a dose escalation study.

The trial team looked at how well sonidegib worked to keep the cancer under control. They have the results for 99 people and found that

  • In 1 person with basal cell skin cancer (BCC) the cancer went away completely
  • In 8 people with BCC or medulloblastoma the cancer got a bit smaller
  • In 24 people the cancer stayed the same
  • In 66 people the cancer continued to grow

Sonidegib did have some side effects but they were usually mild to moderate. The side effects included muscle spasms and pain, stomach problems, liver problems, tiredness, changes in taste and hair loss.

The research team concluded that sonidegib was safe to give. They found that it was a useful treatment for some people with BCC and medulloblastoma and they recommend that it is looked at further in other trials.

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

Dr Anne Thomas

Supported by

Experimental Cancer Medicine Centre (ECMC)

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

Freephone 0808 800 4040

Last review date

CRUK internal database number:

Oracle 5890

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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