A trial of LDE225 for basal cell skin cancer that has spread

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

Cancer type:

Non melanoma skin cancer
Skin cancer




Phase 2

This trial is looking at a drug called LDE225 for basal cell skin cancer that has spread.

Basal cell cancer is the most common type of skin cancer. It is also called basal cell carcinoma or BCC.

Doctors usually treat basal cell skin cancer with surgery. Sometimes they may use radiotherapy instead of, or as well as, surgery.

BCC usually grows slowly, and rarely spreads. But if a BCC does spread or comes back, it may not be possible to remove it with surgery. Or the amount of surgery needed would cause a lot of disfigurement. And if it comes back after radiotherapy, you cannot have radiotherapy again to the same part of the body.

In this trial, researchers are looking at a drug called LDE225. It is for people who have BCC that has spread or come back after treatment and neither surgery nor radiotherapy is a suitable treatment.

Cells normally grow in a very orderly way. Chemical messages or signals tell them when to grow and when to stop. But in cancer cells the normal signalling system changes. So the cells keep on dividing and the cancer grows.

LDE225 is a new drug that blocks a specific protein and stops it sending growth signals. We know from research that the protein it blocks affects the growth of several types of cancer.

The aim of this trial is to see if LDE225 can help people with basal cell cancer.

Who can enter

You may be able to enter this trial if you

  • Have basal cell carcinoma that has spread into surrounding tissue or to another part of your body and you are not going to have surgery, radiotherapy or another treatment to remove it
  • 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 6 months afterwards if there is any chance you or your partner could become pregnant
  • Are at least 18 years old

You cannot enter this trial if you

  • Have had any other type of anti cancer treatment in the last 4 weeks, or have not recovered from the side effects of other treatment
  • Have had an experimental drug as part of another clinical trial in the last 4 weeks
  • Have already had LDE225 or a similar drug
  • Have had major surgery in the last 4 weeks
  • Are taking drugs that affect body substances called cytochrome P enzymes - the trial doctors can advise you about this
  • Have any problems affecting your digestive system Open a glossary item that mean you cannot take or absorb tablets
  • Have a medical condition that affects your muscles, or take drugs that can cause the breakdown of muscle fibres - the trial doctors can advise you about this
  • Have any other medical condition that the trial doctors think could affect your taking part
  • Are pregnant or breastfeeding

Trial design

This is a phase 2 trial. It is an international study that will recruit about 156 people. Everybody taking part will have LDE225 tablets.

The trial is randomised, so the people taking part are put into 1 of 2 treatment groups by a computer. Neither you nor your doctor will be able to decide which group you are in. And neither of you will know which group you are in either. This is called a double blind trial.

People in one group have a low dose of LDE225. People in the other group have a higher dose. There will be twice as many people in the group having the higher dose as in the other group.

You take the tablets everyday for as long as the treatment helps you, or until the study closes. If the treatment is still helping you when the study closes, you may be able to continue having LDE225 in a separate study.

The trial team will ask you to fill out some questionnaires. These will ask about any side effects you have had and about how you have been feeling. This is called a quality of life study. They will also ask you to keep a diary. In this, you record how many tablets you take each day.

The trial team will also ask your permission to take an extra blood sample. They will use this sample to look at the genes in your cells. Researchers want to see if genes can affect how people respond to the trial treatment. They hope that in the future this will help them to work out who is most likely to benefit from LDE225. You do not have to agree to give this sample if you don’t want to, you can still take part in the trial.

Hospital visits

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

  • Physical examination
  • Blood and urine tests
  • Heart trace (ECG Open a glossary item)
  • Chest X-ray (unless you have had a CT scan of your chest in the last 8 weeks)
  • CT scan or MRI scan

You may also have a bone scan.

About 60 people will have extra ECGs before starting treatment and again about 4 months later. This is to learn more about the effect LDE225 has on your heart.

If possible, the trial team will take a sample of tissue from your cancer (a biopsy Open a glossary item) before you start treatment. If you don't have an area of cancer (a lesion) that they can take a biopsy from, they will get a sample that was taken in the past.

A member of the trial team will take some photographs of your skin cancer.

You go to see the trial doctors every week for the first 9 weeks of treatment, then in weeks 11 and 13 and every month after that. The trial team will take more photographs at some of these visits and you have a blood test each time. You have a scan after 5, 9 and 17 weeks of treatment and then once every 8 weeks for the rest of the first year of treatment and once every 12 weeks after that. If possible, the researchers will take another biopsy after your scan and photos in weeks 9 and 17. Depending on your response to treatment, you may have more scans and photos.

When you finish the treatment, you go back to see the trial team within the next 2 weeks. You will have a physical examination, blood tests and an ECG. If it is possible, you will have another biopsy and the trial team will ask you to complete another quality of life questionnaire. You will have photos taken and a scan if you haven't had these done in the last 4 weeks.

If your cancer has got worse, a member of the trial team will contact you every 3 months to see how you are.

If you stop treatment but your cancer has not got worse, the researchers will ask you to continue having scans and skin photographs every 8 weeks for the first year and then every 12 weeks until your cancer does get worse, or you start another treatment, or until the trial has completely finished.

Side effects

As LDE225 is a new drug, there may be side effects we don’t know about yet. Possible side effects that the researchers do know about include

If you have bad side effects, you may need to stop the treatment for a while and then start it again at a lower dose. The trial team will advise you about this.

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 John Lear

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 8395

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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