A trial looking at erlotinib (Tarceva) for children with brain tumours (NAG 2005 09)

Cancer type:

Brain (and spinal cord) tumours

Children's cancers

Status:

Results

Phase:

Phase 1

This trial looked at erlotinib (also called Tarceva and OSI - 774) for children and young adults with brain tumours.

This trial is for children from 1 year and young people up to the age of 21. We use the term ‘children’, but we are referring to children and young adults.

Doctors often use surgery in combination with radiotherapy or chemotherapy, or both, to treat brain tumours.

When this trial started, erlotinib was a new drug that had mainly been tested in trials for adult cancers. This trial looked at erlotinib for two groups of children with brain tumours

Children with a malignant brain tumour that had continued to grow or had come back after treatment
Children who had been recently diagnosed with a type of malignant brain tumour called brain stem glioma

The aims of this trial were to find out

The best dose of erlotinib to treat children with brain tumours in the future
More about the side effects of erlotinib

Summary of results

The research team found that erlotinib was safe and were able to find the best dose to give.

This was a phase 1 trial and it recruited 51 children into 2 groups.

The first group was children who had a brain tumour that had continued to grow despite treatment. There were 30 children in this group and they all had erlotinib. They continued to take erlotinib every day unless they had severe side effects or there were signs that their cancer had continued to grow.

The second group was children who had recently been diagnosed with a type of brain tumour called a brain stem glioma. There were 21 children in this group, and they all had erlotinib and radiotherapy. They had radiotherapy once a day (Monday to Friday) for 6 weeks. They took erlotinib tablets every day unless they had severe side effects or their cancer started to grow.

The first few children having erlotinib in each group had the lowest dose. The next few had a higher dose, and so on until they found the best dose to use. This is called a dose escalation trial.

There were 4 dose levels. For both groups the research team decided that the 3rd dose level they tested was the best dose to use.

The most common side effects were

Rash or acne
Dry skin
Diarrhoea
Bleeding within the brain tumour

The research team concluded that they had found the best dose to give children and young people, and that erlotinib was safe to use for this group of patients. They suggest that it is looked at further in other trials, possibly in combination with different treatments.

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.