A trial looking at a new drug called pegylated recombinant human arginase for children and young adults with cancer (PARC)

Please note - this trial is no longer recruiting patients. We hope to add results when they are available.

Cancer type:

Acute leukaemia

Acute lymphoblastic leukaemia (ALL)

Acute myeloid leukaemia (AML)

Brain (and spinal cord) tumours

Children's cancers

Leukaemia

Neuroblastoma

Sarcoma

Secondary cancers

Status:

Closed

Phase:

Phase 1/2

This trial is for children and young adults with cancer that has come back or continued to grow despite treatment.

It is for children and young adults with one of the following cancers:

a type of leukaemia called acute lymphoblastic leukaemia (ALL) or acute myeloid leukaemia (AML)
neuroblastoma
sarcoma
a type of brain tumour called glioma that usually grows quickly and is likely to spread (high grade glioma)

Everyone taking part needs to be older than 12 months but less than 25 years old. We use the term ‘you’ in this summary, but if you are a parent, we are referring to your child.

Cancer Research UK supports this trial.

More about this trial

Chemotherapy is a common treatment for children and young adults with cancer. It uses anti cancer (cytotoxic) drugs to destroy cancer cells. But chemotherapy doesn’t always work. So doctors are looking for new ways to help children and young adults with a cancer that continues to grow or comes back after treatment. This is called refractory or relapsed cancer.

A protein (an amino acid) called arginine is important for cells to grow and survive. Normal, healthy cells can make arginine using a protein (an enzyme) that is often missing in cancer cells. If the amount of arginine available is reduced, it starves the cancer cells and stops them from growing.

Healthy cells are able to survive much better than cancer cells when there is less arginine available. This is partly because they can make it. So, by reducing the amount of arginine available the cancer might stop growing.

In this trial, doctors are looking at a drug called pegylated recombinant human arginase (BCT-100), which reduces the amount of arginine available.

This trial is in 2 parts. In the 1st part, doctors want to find the highest dose of BCT-100 that you can safely have. Once doctors find the best dose, part 2 will open. In the 2nd part of this trial, everyone has the best dose of BCT-100 found during part 1.

Please note – doctors are currently looking for people to join the 2nd part of this trial.

The main aims of this trial are:

learn about the side effects of BCT-100
find out the highest safest dose of BCT-100
find out how well BCT-100 works as a treatment for children and young adults with cancer

Who can enter

The following bullet points list the entry conditions for this trial. Talk to your doctor or the trial team if you are unsure about any of these. They will be able to advise you.

Who can take part

You may be able to join this trial if you have 1 of the following cancers:

As well as one of the above, all of the following must apply:

you are at least 12 months old and less than 25 years old
your cancer has come back or continued to grow despite treatment
your cancer can be measured using a scan or a bone marrow test
you have satisfactory blood tests results
if there is any possibility that you or your partner could become pregnant you are willing to use reliable contraception during treatment and for a year afterwards

Who can’t take part

You cannot join this trial if any of these apply. You:

have had treatment with a drug that decreases the amount of arginine or any other similar drug
have severe side effects from previous anti cancer treatments
have an infection that isn’t controlled
are pregnant or breastfeeding

Trial design

This is an international phase 1/2 trial. Doctors hope that up to 64 children and young adults will take.

This trial is in 2 parts. Doctors are currently looking for people to join part 2.

Part 1

The first few people taking part have a low dose of pegylated recombinant human arginase (BCT-100). If they don’t have any serious side effects, the next few people have a higher dose of BCT-100. And so on, until doctors find the highest dose of BCT-100 that you can safely have.

Once doctors find the best dose of BCT-100, part 2 opens.

Part 2

Everyone has the highest safest dose of BCT-100 found during part 1. Doctors want to find out whether BCT-100 helps children and young adults with refractory or relapsed cancer.

How do I have BCT-100?

Everyone has BCT-100 as a drip into a vein (intravenously) every week. It takes about an hour each time you have it.

You can have up to 24 treatments with BCT-100, taking around 6 months in total. After 6 months, you might be able to continue to have BCT-100 for as long as it is helping you and the side effects aren’t too bad. Your doctor can tell you more about this.

Blood tests and tissue samples
You have extra blood tests as part of this trial. Doctors want to:

find out what happens to BCT-100 in your body
look at the levels of arginine in your body

You have the extra blood tests before the start of treatment and at set times during the trial.

Doctors might ask you to give extra tissue samples if you are having tests such as lumbar puncture that are part of your normal care. They may also ask for a sample of tissue from a biopsy that you had in the past.

Hospital visits

You see a doctor and have some tests before taking part in this trial. The tests may include:

a physical examination
blood tests
urine test
a bone marrow test
a lumbar puncture
a CT scan, MRI scan or MIBG scan

During treatment, you see the trial doctor every week. You have blood tests and a physical examination each time you see them.

You have a test to find out how well the treatment is working every 8 weeks. It can be:

a bone marrow test
lumbar puncture
a scan such as CT scan, MRI scan or MIBG scan

Your doctor can tell you which test you will have. It depends on your cancer type.

You have treatment for up to 24 weeks (about 6 months). After 24 weeks, you may be able to continue to have BCT-100. Your doctor can tell you more about this.

When you finish treatment, you see the trial team and have more tests such as blood tests and scans. You then continue to see your doctor as part of your normal care.

Side effects

Pegylated recombinant human arginase (BCT-100) is a new treatment and there might be side effects we don’t know about yet. The trial team will monitor you during the time you have treatment. You have a phone number to call them if you are worried about anything.

Adults with liver cancer have already had BCT-100. But this is the first time that children have had BCT-100.

The most common side effects of BCT-100 reported so far are: