A trial looking at clofarabine for children and teenagers with acute lymphoblastic leukaemia (BIOV 111)

Cancer type:

Acute leukaemia

Acute lymphoblastic leukaemia (ALL)

Blood cancers

Children's cancers

Leukaemia

Status:

Results

Phase:

Phase 2

This trial looked at clofarabine for children and teenagers with acute lymphoblastic leukaemia (ALL) that had not responded to treatment, or had come back after treatment.

This trial was for children and young people up to and including the age of 21. We use the term ‘you’ in this summary, but of course if you are a parent, we are referring to your child.

Doctors often treat ALL with chemotherapy and possibly a bone marrow or stem cell transplant. This often works very well, and these days about 8 out of 10 children with ALL (80%) are cured.

But unfortunately either the treatment doesn’t work (the ALL is ‘refractory’), or the leukaemia comes back again (relapses) in the other 2 out of 10 children (20%). If this happens, ALL can be very difficult to treat. The drugs that are currently available for this situation often don’t work very well.

Clofarabine is a chemotherapy drug. It is similar to other drugs called fludarabine and cladribine, which are used to treat different types of leukaemia. Doctors hoped that clofarabine would be useful for treating children with ALL that was refractory or had relapsed.

The aim of this trial was to find out how well clofarabine worked for children with relapsed or refractory ALL.

Summary of results

The trial team found that clofarabine did help children whose ALL hadn’t responded to treatment or had come back afterwards.

This was a phase 2 trial. It recruited 74 children. Everyone taking part had clofarabine.

The trial team were able to look at the results of 65 of the 74 children who took part. They looked at how well the children responded after at least 1 course of treatment with clofarabine. 17 children had no sign of ALL in their blood tests (a complete remission Open a glossary item ). That is about 1 in 4 (26%).

The main side effects were

A drop in white blood cells causing an increased risk of infection
Temporary changes to the way the liver worked
Possible changes to the way the kidneys worked
Sore, red hands and feet (palmar plantar syndrome)
Bone pain

The trial team concluded that clofarabine was safe and worked well for children with ALL that hadn’t responded to treatment or had come back afterwards.

We have based this summary on information from the team who ran the trial. 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. We have not analysed the data ourselves.