"I am glad that taking part in a trial might help others on their own cancer journey.”
A trial looking at drug called LDK378 for people who have cancer with a change to the ALK gene
Please note - this trial is no longer recruiting patients. We hope to add results when they are available.
This trial is to see if a new drug called LDK378 can stop the growth of cancer cells that have a change (
Cells normally grow in a very orderly way. Chemical messages or signals tell them when to grow and when to stop. But if a gene called ALK is abnormal, the ALK protein can signal to make cells grow abnormally. There is an altered ALK gene in many cancers, such as lung cancer, breast cancer and bowel cancer.
In this trial, researchers are looking at a new drug called LDK378 which blocks the ALK protein and stops it sending growth signals to cancer cells.
We know from research that LDK378 can stop the growth of cancer cells with the altered ALK gene. But this is the first time it has been tested in people. Only people who have cancer with an altered ALK gene can take part.
The aims of the study are to
- Find the highest safe dose of LDK378
- Learn more about what happens to LDK378 in the body
- See if it causes any changes to your cancer
- Learn more about the side effects
Who can enter
You may be able to enter this trial if you
- Have cancer that has a change to the ALK gene – the researchers need a sample of your cancer to test for this
- Are well enough to be up and about for at least half the day (performance status 0, 1 or 2)
- Have satisfactory blood test results
- 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 of age
You cannot enter this trial if you
- Have cancer that has spread to your brain or spinal cord (central nervous system) unless it has been successfully treated with surgery or radiotherapy that finished at least 2 weeks ago, and if you need to take steroids, you have been on a stable dose for at least 2 weeks
- Have had biological therapy, chemotherapy or another experimental drug in the last 2 weeks, or earlier if there is any chance you could still have some of the drug in your body
- Have not recovered from the side effects of other anti cancer drugs (apart from hair loss or changes to your nerves or to your hearing) unless they are very mild
- Have had radiotherapy in the last 3 weeks or have not recovered from the side effects of earlier treatment
- Have had major surgery in the last 2 weeks or have not fully recovered from earlier surgery
- Have sickness or diarrhoea unless it is very mild
- Have any problems with your
digestive systemthat could affect how you absorb tablets
- Have ever had inflammation of your pancreas (pancreatitis) or raised levels of
enzymescalled amylase or lipase
- Have liver disease, including infection with hepatitis B or hepatitis C in the past
- Have a heart condition that is cause for concern – the trial doctors can advise you about this
- Are taking warfarin, or certain other drugs that thin your blood (anticoagulants), a drug called phenytoin, or any other medication that affects an enzyme called CYP3A4 and you cannot stop taking these at least a week before starting the study treatment (your doctor can advise you about this and it is important you don’t stop taking any medication before talking to your doctor)
- Have had another cancer, apart from carcinoma in situ of the cevix, non melanoma skin cancer or any other cancer that was successfully treated with surgery or radiotherapy, and there has been no sign of it coming back for at least 3 years
- Have any other serious medical condition that the trial doctors think could affect your taking part
- Are known to be HIV positive
- Are pregnant or breastfeeding
This phase 1 trial will recruit about 40 people in a number of different countries. You can only join this trial if the researchers can see that the cells in your cancer have an altered ALK gene. They will ask your permission to test a sample of your cancer to see if the ALK gene is altered. You may have had this test already. If not, the researchers will see if there is enough tissue available from when you had surgery or a
Everybody taking part in the trial will have LDK378 capsules once or twice a day. The trial team will tell you exactly how and when to take them.
The first patients taking part will have a low dose of LDK378. If they don’t have any serious side effects, the next patients will have a higher dose. And so on, until the researchers find the highest dose they can give. This is called a dose escalation study. Once they have worked out the highest safe dose, more people will join the trial and have this dose so the researchers can learn more about LDK378 and its side effects. This is called the expansion phase.
If you join the dose escalation study, you start by having a single dose of LDK378. Over the next 3 days, the trial team monitor you closely and take blood samples to see what happens to the drug in your body. This is called
People joining the expansion phase take the capsules everyday from the start.
The researchers call each 3 week period of treatment a cycle of treatment. As long as you don’t have any bad side effects, you can carry on taking LDK378 for as long as it helps you.
The trial team will ask you to fill in a diary at home. In this, you record exactly when you take the capsules and any side effects you have. You must take the diary to each hospital visit.
You will see the trial doctors and have some tests before you start treatment. The tests include
You have your first dose of LDK378 at hospital. As you have a number of blood tests and ECGs before and after taking the drug, you will be at hospital for about 9 hours. People in the dose escalation study go back to see the trial team and have more blood tests on each of the next 2 days.
You go to hospital 5 times during the first cycle of treatment and 3 or 4 times in each of the following cycles. You will have blood tests at most visits. For some visits, the trial team will ask you not eat anything from midnight the day before and not to take the capsules until after you have had a blood test.
During treatment, you have a number of ECGs. You also have a CT or MRI scan every 6 weeks.
When you finish treatment, you see the trial doctors again and may need to have another scan. They will also ask you to have another biopsy, but this is optional.
As LDK378 is a new drug, there may be side effects we don’t know about yet. The possible side effects include
- Inflammation of your bile duct or pancreas
- Damage to your liver or kidneys
- Feeling or being sick
- Inflammation of your lungs
LDK378 could also cause a skin rash if you are exposed to sunlight or other strong light. The trial team will advise you to wear clothing that protects you from the light, including a hat, and to use sunscreen.
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.
Professor Jeff Evans
Experimental Cancer Medicine Centre (ECMC)