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

Cancer type:

Non melanoma skin cancer

Skin cancer

Status:

Results

Phase:

Phase 2

This trial looked at sonidegib (LDE225) for basal cell skin cancer that had spread.
Basal cell cancer is the most common type of skin cancer. It is also called basal cell carcinoma or BCC.

More about this trial

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 looked at a drug called sonidegib. It was for people who had BCC that had spread or came back after treatment. And neither surgery nor radiotherapy were 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.

Sonidegib is a targeted cancer drug Open a glossary item . It blocks a specific protein that sends growth signals to cancer cells. We knew from research that the protein it blocks affects the growth of several types of cancer.

The aim of this trial was to see if sonidegib helped people with basal cell cancer.

Summary of results

The trial team found that sonidegib might be offered as a treatment choice for people with basal cell skin cancer that has spread.

About this trial
This was an international phase 2 trial.

It was a randomised trial. Everyone who took part was put into 1 of 2 treatment groups. Neither they or their doctor chose which group they were in. And neither knew which group they were in.

230 people took part. Everyone had sonidegib. There were 2 different doses, 200mg and 800mg. 2 out of every 3 people were in the 800mg group.

79 people had 200mg
151 people had 800mg

BOLT Diagram 1

Results
The average follow up was just under 14 months (13.9).

Researchers wanted to find out how many people had some response to treatment. They looked at whether:

there was no sign of the cancer (complete response)
the cancer had got smaller (partial response)
the cancer had stayed the same (stable disease)

Looking at the results the team also grouped the people according to whether their cancer had:

only spread into the surrounding tissue (locally advanced)
spread to another part of the body (metastatic)

To find out if the cancer responded to treatment everyone had an MRI scan.

For the people who had locally advanced basal cell cancer the researchers also used a photograph of the cancer to match with the MRI scan.

Results for the 200mg group
Of the 79 people in the 200mg group, 66 people had locally advanced cancer. Of these 66 people the total number of people whose cancer had responded to cancer was 60 (91%). For:

2 people (3%) there was no sign of their cancer
29 people (44%) their cancer had shrunk
29 people (44%) their cancer had stayed the same

The number of people in this group whose cancer had spread to another part of their body (metastatic) was 13 people. They found that for a total of 12 people (92%) their cancer had responded to treatment. The results were:

no one (0%) had no sign of their cancer
for 2 people (15%) the cancer had shrunk
for 10 people (77%) the cancer had stayed the same

Results for the 800mg group
Of the 151 people in the 800mg group, 128 people had locally advanced cancer. Of these 128 people the total number of people whose cancer had responded to treatment was 100 people (78%). The results were:

no one (0%) had no sign of their cancer
for 45 people (35%) the cancer had shrunk
for 55 people (43%) the cancer had stayed the same

For 23 people in this group their cancer had spread to another part of the body (metastatic). Of these 23 people, 19 people (83%) had responded to treatment. The results were:

no one (0%) had no sign of their cancer
for 4 people (17%) the cancer had shrunk
for 15 people (66%) the cancer had stayed the same

Overall results
The team also looked at the overall number of people in each group whose cancer had responded to treatment.

Of the 79 people who had 200mg of sonidegib, they were able to look at the results of 55. Of these 55 people, for 20 people (36%) their cancer had responded to treatment.

Of the 151 people who had 800mg of sonidegib, they were able to look at the results of 116 people. Of these 116 people, for 39 people (34%) their cancer had responded to treatment.

BOLT Graph 1

Quality of life
For most people in both groups their quality of life stayed the same or improved.

For people in the 200mg group, 2 parts of their quality of life got worse. This was tiredness and weight loss.

For people in the 800mg group, there were 4 parts where their quality of life got worse. These were:

physical performance (how fit and well they were)
socialising and social wellbeing
tiredness
weight loss

Side effects
In general people in the 200mg group had less side effects than those in the 800mg group.

The most common side reported included:

muscle spasms
hair loss
changes in taste
being sick
a raised level of creatinine in the blood
tiredness
weight loss
diarrhoea
loss of appetite
muscle and joint pain
headache

Conclusion
The trial team say that it is difficult to treat basal cell skin cancer that has spread.

They conclude that 200mg of sonidegib could be a promising treatment for this cancer because of the overall number of people whose cancer had been controlled. And that their side effects were less than those who had 800mg.

Where this information comes from
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.