A trial looking at treosulfan to treat Ewing's sarcoma that has come back after treatment (OTIS)

Cancer type:

Children's cancers
Ewing sarcoma




Phase 2

This trial looked at treosulfan chemotherapy for children, young people and adults with Ewing’s sarcoma who already had treatment. The trial was supported by Cancer Research UK.

This trial was for both adults and children. We use the term 'you' in this summary, but if you are a parent, we are referring to your child.

More about this trial

Ewing’s sarcoma is a type of cancer that usually develops in, or near, the bones. It mainly affects teenagers, young adults and children.

Doctors usually treat Ewing’s sarcoma with chemotherapy. But if the tumour does not completely disappear, or continues to grow during chemotherapy, there are not many other treatments that doctors can use. So they are always looking for new ways to help people in this situation. In this trial, they looked at a drug called treosulfan.

Treosulfan is a chemotherapy drug similar to one that was already used to treat people with Ewing’s sarcoma. We know from research that treosulfan can help people with other types of cancer. The researchers thought that treosulfan may help people who had already had treatment for Ewing’s sarcoma.

The aims of this trial were to find out

• What effect treosulfan has on Ewing’s sarcoma
• What the side effects are

Summary of results

The trial team found that the dose of treosulfan they used didn’t help people with Ewing’s sarcoma.

This trial recruited 21 people and the team were able to look at the results of 20 people. They found that

  • In 1 person, the sarcoma didn’t get any worse or any better (doctors call this stable disease)
  • In 13 people, the sarcoma continued to grow
  • 6 people stopped treatment because they became more sick. It was believed that this was due to the sarcoma continuing to grow, but these people were too unwell to have a scan Open a glossary item to confirm this.

The trial team looked at the average length of time that people were living without any sign of their cancer getting worse. They found this was just under 2 months. They also looked at the average length of time people lived overall and found this was just over 6 months.

The most common bad side effect of treosulfan was a drop in blood cells causing an increased risk of infection, bruising and bleeding.

The trial team concluded that this dose of treosulfan didn’t work for people who had already had treatment for Ewing’s sarcoma.

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.

Recruitment start:

Recruitment end:

How to join a clinical trial

Please note: In order to join a trial you will need to discuss it with your doctor, unless otherwise specified.

Please note - unless we state otherwise in the summary, you need to talk to your doctor about joining a trial.

Chief Investigator

Dr Maria Michelagnoli

Supported by

Cancer Research UK
Experimental Cancer Medicine Centre (ECMC)
NIHR Clinical Research Network: Cancer
University College London (UCL)
The Adam Dealey Foundation

Other information

This is Cancer Research UK trial number CRUKE/09/013.

If you have questions about the trial please contact our cancer information nurses

Freephone 0808 800 4040

Last review date

CRUK internal database number:

Oracle 3874

Please note - unless we state otherwise in the summary, you need to talk to your doctor about joining a trial.

Last reviewed:

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