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 
) 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.