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

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.

Recruitment

Start 30/08/2011

End 31/10/2012

Phase

Phase 2

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 or radiotherapy
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
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 2 weeks
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 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 120 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. You take the tablets everyday for as long as the treatment helps you.

The trial team will ask you to fill out a questionnaire after 1 week and 4 weeks of treatment, and then once a month. The questionnaire 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)
Chest X-ray (unless you have had a CT scan of your chest in the last 8 weeks)
CT scan or MRI scan

A member of the trial team will take some photographs of your skin cancer. And they will get a sample of your cancer that was removed when you had surgery. If a sample is not available, and your cancer is in an accessible part of your body, they will take a sample (a biopsy) before you start treatment.

Depending on where your cancer has spread to, the researchers may ask to take some other biopsies before you start taking LDE225, and then again in weeks 9 and 17 of treatment. If your cancer disappears during treatment, they will take the later biopsies from the area where the cancer was.

You go to see the trial doctors every 2 weeks for the first 3 months of treatment and then every month after that. The trial team will take more photographs at some of these visits. You have a blood test each time and an ECG every 4 weeks. 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.

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. 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 until the trial has completely finished.