A trial looking at nilotinib to treat acral and mucosal melanoma skin cancer that has spread (NICAM)
Cancer type:
Status:
Phase:
This trial looked at nilotinib for 
that had spread to the surrounding tissues, or to another part of the body.
It was for two rare types of melanoma:
- acral melanoma
- mucosal melanoma
The trial was supported by Cancer Research UK. It was open for people to join between 2009 and 2014. The team published the results in 2024.
More about this trial
Acral melanoma most commonly develops on the palms of the hands or soles of the feet. It is sometimes called lentiginous melanoma.
Mucosal melanoma starts in the moist tissue (mucosa) which lines the eyes, mouth, nose, food pipe, anus, vulva and vagina.
Doctors can use chemotherapy to relieve symptoms of advanced melanoma. They wanted to find out if nilotinib could be a useful treatment for people with acral or mucosal melanoma.
Nilotinib is a type of targeted cancer treatment called a tyrosine kinase inhibitor (TKI). It helps block the signals that melanoma cells need to divide and grow.
Research had shown that nilotinib works better for people with a change () in a gene called KIT. So the team looked at each person’s melanoma cells to see if they had a KIT mutation. Those who did could have treatment as part of this trial.
The main aims of this trial were to find out if:
- nilotinib is a useful treatment for advanced acral and mucosal melanoma
- more about the side effects
- whether the test they used to look for KIT mutations was accurate
Summary of results
This trial showed that nilotinib might be a useful treatment for advanced acral or mucosal melanoma.
Results
A total of 219 people registered for the trial and had a test to look for a KIT gene change (mutation). Tests showed that 39 people (18%) did have a KIT gene mutation. And 29 of these people went on to have nilotinib.
Of the 29 people who had treatment:
- 23 had mucosal melanoma
- 6 had acral melanoma
Everyone took nilotinib tablets twice a day, for as long as it was helping.
How well treatment worked
The team looked at how long it was before the cancer started to grow again in half the people taking part. This is called the median time. They found it was 3.7 months. It was lower for people who had acral melanoma compared to those who had mucosal melanoma.
They also looked at how long it was until half the people taking part had died. This is called the median survival. They found it was 7.7 months. This was also lower for people with acral melanoma than it was for those with mucosal melanoma.
They then looked at how many people were living, a year after joining the trial. They found it was just under half (44%).
These results are similar to other treatments that have been looked at for these rare melanomas. They can be difficult to treat, because used for skin melanomas don’t always work as well for mucosal or acral melanoma.
Side effects
Most people taking part had at least 1 side effect from treatment. Some of these were mild or didn’t last long. The most common side effects were:
- extreme tiredness (fatigue)
- feeling sick
- constipation
18 people had a more severe side effect. The most common of these were:
- extreme tiredness (fatigue)
- tummy (abdominal) pain
- feeling or being sick
We have more information about the side effects of nilotinib in our Cancer drugs section.
How well the gene test worked
The trial team looked for KIT gene changes in the melanoma cells of everyone who registered for the trial. They used cell samples taken during .
They also used a test called ddPCR to look for KIT gene changes in 18 people who took part. This stands for droplet digital polymerase chain reaction. It’s a way of looking for genetic mutations in blood samples () rather than tissue samples. This is called a liquid biopsy.
They found that the ddPCR test worked well and correctly found the gene changes.
Conclusion
The trial team concluded that nilotinib could be a useful treatment for people with advanced acral or mucosal melanoma. They suggest further trials are done to find out more.
They also concluded that the ddPCR blood test was an accurate way to find genetic changes.
More detailed information
There is more information about this research in the reference below.
Please note, the information we link to here is not in plain English. It has been written for healthcare professionals and researchers.
Nilotinib in KIT-driven advanced melanoma: Results from the phase II single-arm NICAM trial
J Larkin and others
Cell Reports Medicine, 2024. Volume 5, issue 3, reference 101435.
Where this information comes from
We have based this summary on the information in the article above. This has been reviewed by independent specialists () and published in a medical journal. We have not analysed the data ourselves. As far as we are aware, the link we list above is active and the article is free and available to view.
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.
Chief Investigator
Professor James Larkin
Supported by
Cancer Research UK
Experimental Cancer Medicine Centre (ECMC)
Institute of Cancer Research (ICR)
NIHR Clinical Research Network: Cancer
Novartis
The Royal Marsden NHS Foundation Trust
Other information
This is Cancer Research UK trial number CRUK/09/028.
If you have questions about the trial please contact our cancer information nurses
Freephone 0808 800 4040
