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

Who can enter

This trial is open to children and adults up to the age of 50 years.

You can enter this trial if

• You have advanced Ewing’s sarcoma or peripheral neuroectodermal tumour (PNET) that has continued to grow during treatment (is ‘refractory’)
• Your tumour can be measured on a scan
• You have had at least 1 course of chemotherapy
• Your tumour has continued to grow in the last 8 weeks
• You are well enough to be up and about for at least half the day and look after yourself (performance status 0, 1, 2) but if you have damage to your spine, you are in bed or a chair for half the day and are otherwise well or if younger than 16 years, are mainly in bed and at least able to take part in quiet activities (Lansky score greater than 30)
• You have satisfactory blood test results
• You are willing to use reliable contraception if there is a chance you or your partner could become pregnant
• Are able to swallow capsules

You cannot enter this trial if you

• Are newly diagnosed with Ewing’s sarcoma
• Are able to have your tumour completely removed with surgery
• Are having another treatment for your cancer (you may join the trial if you are having radiotherapy to relieve symptoms of cancer that has spread to other parts of the body)
• Are taking a drug as a part of another clinical trial
• Are taking drugs to help you grow blood cells (growth factors)
• Are pregnant or breastfeeding

Trial design

This is a phase 2 trial. It will recruit about 25 people in the UK. Everyone taking part will have treosulfan.

Treosulfan is a capsule. You take 2 to 3 capsules, 3 times a day for 1 week and then have 3 weeks of no treosulfan. Every 4 week period is called a cycle of treatment.

You have a diary card to write down the dates and times you take your capsules. You can also use this diary card to write down any side effects you may have from the treatment.

You continue to have treosulfan as long as it is helping you and you have no serious side effects.

Hospital visits

You will see the doctor and have some tests before starting treatment. These tests include

• Physical examination
• Blood tests
• X-ray or CT scan of the chest
• MRI scan (if needed)
• Bone scan
• Pregnancy test (if appropriate)

Before each cycle of treatment you have a physical examination and blood test. During treatment you also have a weekly blood test done at your local hospital.

Every 8 to 12 weeks you have a CT scan or MRI scan.

One month after treatment finishes, you see the doctors for a physical examination and blood tests. Then you see them every 8 to 12 weeks until your cancer starts to grow again.

Side effects

The side effects of treosulfan can include

• A drop in blood cells causing an increased risk of infection, bruising or bleeding problems
• Rash or itchy skin
• Tiredness (fatigue)
• Feeling, or being, sick (nausea)
• Hair loss

You can find more about treosulfan on CancerHelp UK.