A trial of ribociclib for children and young people with neuroblastoma or a malignant rhabdoid tumour

Cancer type:

Children's cancers

Neuroblastoma

Status:

Results

Phase:

Phase 1

This trial looked at a drug called ribociclib as treatment for neuroblastoma and a rare type of childhood cancer called a malignant rhabdoid tumour.

More about this trial

Ribociclib (LEE011) is a type of cancer growth blocker. It stops signals that cancer cells use to divide and grow. Researchers wanted to find out if it could help children and young people with:

neuroblastoma - a cancer which starts in nerve cells, often in the tummy (abdomen)
malignant rhabdoid tumour (MRT) - a rare type of cancer which can start in the kidneys
other cancers which have specific cell or genetic changes

The children and young people taking part in this trial had already had treatment. But their cancer had not responded to it or had come back after treatment. Their doctors didn’t think other treatments available at the time were likely to help them.

Everyone taking part had ribociclib capsules once a day for 3 weeks, and then a week with no treatment. Each 4 week period is one cycle of treatment. The dose they had depended on which group they were in. They continued having treatment until there were signs their cancer had started to grow or they had serious side effects.

The aim of the trial was to find the best dose of ribociclib for children and young people.

Summary of results

This phase 1 trial showed that ribociclib didn’t cause too many side effects and could help stop cancer growing in some children and young people.

The research team recruited patients in 2013 and 2014, and published the results in 2017.

Results

This trial recruited 32 children and young people between 1 and 20 years old. They all had cancer that had continued to grow despite other treatment:

15 had neuroblastoma
15 had malignant rhabdoid tumour
1 had a type of sarcoma called rhabdomyosarcoma
1 had a type of brain tumour called anaplastic meningioma

The first few people had the lowest dose of ribociclib. They didn’t have too many side effects, so the next few people had a higher dose. And so on, until they found the best dose to give. This is called a dose escalation study.

When the research team looked at how well the treatment worked, they found that the cancer stopped growing in 9 out of the 32 people (28%). The cancer stopped growing for many months in some of these people.

Side effects

Most people who took part had at least 1 side effect. But many were mild or didn’t last long. The most common side effects included:

a drop in white blood cells, red blood cells or blood clotting cells (platelets)
feeling or being sick
extreme tiredness (fatigue)

More people who had the higher doses had side effects, and some of them were more severe.

Conclusion

The research team concluded that they had found the best dose of ribociclib to give without causing too many side effects. They suggest it is looked at alongside other treatments in future trials.

Where this information comes from

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