A study looking at pembrolizumab for children and teenagers with a solid tumour or lymphoma (KEYNOTE 051)

Cancer type:

Blood cancers
Children's cancers
Lymphoma
Melanoma
Skin cancer

Status:

Open

Phase:

Phase 1/2

This study is looking at a drug called pembrolizumab for children and teenagers who have advanced melanoma or certain other types of solid tumour or lymphoma.

This study is for children who are at least 6 months old but haven’t yet reached their 18th birthday. We use the term ‘you’ in this summary, but if you are a parent, we are referring to your child.

More about this trial

Pembrolizumab is a type of biological therapy called a monoclonal antibody. It helps the immune system to kill cancer cells. It is already used as a treatment for adults with advanced melanoma.

Researchers want to see if pembrolizumab will be a useful treatment for children and teenagers in this situation. They also want to see if it will help children or teenagers who have a solid tumour or a lymphoma that has continued to grow or has come back despite other treatment, or that has spread elsewhere in the body (advanced cancer).

The aims of this study are to

  • Find the highest safe dose of pembrolizumab to give that does not cause serious side effects
  • Find out what happens to the drug in your body
  • Learn more about the side effects
  • See how well pembrolizumab works as a treatment for children and teenagers with advanced melanoma or certain solid tumours or lymphoma

Who can enter

You may be able to join this study if all of the following apply.

  • You have advanced melanoma, or a solid tumour Open a glossary item including brain tumours or lymphoma that has continued to grow or has come back despite treatment, or that has spread elsewhere in your body. If you have a cancer other than melanoma, it must produce a protein called PD-L1 (your doctor will test for this)
  • Your cancer can be measured using a scan
  • If you are between 6 months and 16 years old, you must be up and around even if you are not playing as energetically as normal and spend less time than usual playing (Lansky Play Scale of 70 or more)
  • If you are 17 years old, you must be able to care for yourself even if you are not able to carry on with all your normal activities or do active work (Karnofsky score of 70 or more)
  • You have satisfactory blood test results
  • You are at least 6 months old but haven’t yet reached your 18th birthday
  • You are willing to use reliable contraception during the time of the study and for up to 4 months afterwards if you are sexually active and there is any chance you or your partner could become pregnant

You cannot join this study if any of these apply. You

  • Are already taking part in a clinical trial or research study, or have had an experimental treatment in the last 4 weeks
  • Have already had an anti PD-L1 or anti PD-L2 drug such as ipilimumab, unless you have had this as a treatment for melanoma
  • Are having treatment that affects your immune system, though certain steroid therapy may be allowed (your doctor can advise you about this)
  • Have had treatment with an anti cancer  monoclonal antibody in the last 4 weeks
  • Have not recovered from any side effects due to having had a monoclonal antibody in the past
  • Have had chemotherapy, other cancer drugs or radiotherapy in the last 2 weeks
  • Have had a stem cell transplant Open a glossary itemusing donor stem cells in the last 5 years
  • Have not recovered from any major surgery
  • Have had a live vaccine in the last 30 days
  • Have another type of cancer that needs treatment except for successfully treated squamous cell skin cancer or carcinoma in situ of the cervix
  • Have cancer that has spread to your brain, including the brain stem or spinal cord and is causing symptoms (you can take part if cancer spread to your brain was treated at least 4 weeks ago and is not causing symptoms)
  • Have a problem with your immune system Open a glossary item making you more likely to get infections
  • Have an autoimmune disease Open a glossary item for which you have had treatment that reaches your whole body (systemic treatment Open a glossary item) such as drugs that suppress the immune system in the last 2 years (some other types of drugs are allowed and your doctor can advise you about this)
  • Have a lung condition called pneumonitis or you have had pneumonitis in the past and needed treatment with steroids
  • Have an infection which needs treatment
  • Have HIV or active hepatitis B or C
  • Have any other medical or mental health problems that the study team think could prevent you taking part
  • Are pregnant or breastfeeding

Trial design

This is a phase1/2 study. The researchers need about 310 children and teenagers to join. The first part of the study (phase1) will find the highest safe dose of pembrolizumab. During this part of the study, the researchers will also look at what happens to the drug in your body, learn more about the side effects and see how well pembrolizumab works as a treatment.

So if you are in the first part of the study how much pembrolizumab you have and how often you have it may change. The study doctors will discuss this with you.

In the second part of the study (phase 2) the researchers will know the best dose of pembrolizumab and how often it should be given. But they still want to look at the side effects of the drug and how useful it is as a treatment.

In the second part of the study the researchers may include children and teenagers who have a cancer that is PD-L1 negative. Your doctor can advise you if this applies to you.

Everybody taking part will have pembrolizumab. You have it through a drip into a vein every 2 or 3 weeks. It takes around 30 minutes each time. You have treatment for up to 2 years.

If your cancer gets worse or you have very bad side effects, the treatment will be stopped. If this happens, your doctors will discuss other treatment options with you.

If you agree to take part in this study, the researchers will ask for a sample of tumour tissue from a biopsy Open a glossary item you have had in the past. If this is not available then you will need to have a tissue sample taken. This will be used to test if your tumour has specific biomarkers. A biomarker is a substance that can be used to measure how the cancer is developing or how a treatment is working.

Hospital visits

You see the study team and have some tests before you start treatment. The tests include

• Physical examination
• Blood tests
• Heart trace (ECG Open a glossary item)
• CT scan or an  MRI scan

You may also have

 PET scan
• Bone scan
• MIBG scan

This will depend on the type of cancer you have. The study team will let you know if you need these.

You go to hospital once every 2 or 3 weeks to have pembrolizumab. At each visit, you have a physical examination and blood tests.

You have a CT or MRI scan every 8 weeks. You may also have a PET scan, MIBG scan or bone scan. You always have the same type of scans as this is what the doctors will use to see how well the treatment is working.

When you finish having pembrolizumab, the study team will see you about 1 month later. You then have a follow up appointment every 8 weeks. At these appointments you will have a physical examination and blood tests.

The study team will see you until either your cancer gets worse or you start a new treatment. If this happens, the study team will then contact you by phone every 3 months until the end of the study.

Side effects

The most common side effects of pembrolizumab in adults are

We have information about the side effects of pembrolizumab.

Your doctor will talk to you about the possible side effects before you agree to take part in this trial.

Location

London

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 Lynley Marshall

Supported by

Merck, Sharp & Dohme Limited (MSD)

Questions about cancer? Contact our information nurses

Freephone 0808 800 4040

Last review date

CRUK internal database number:

12843

Please note - unless we state otherwise in the summary, you need to talk to your doctor about joining a trial.

Over 60,000 cancer patients enrolled on clinical trials in the UK last year.

Last reviewed:

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