A trial looking at RO5503781 for acute myeloid leukaemia

This is an international phase 1 trial. The researchers need 143 people to join. This trial is in 4 parts.

In part 1, the researchers want to find the highest safe dose of RO5503781. The first few people taking part will have a low dose. If they don’t have any serious side effects, the next few people will have a higher dose. And so on, until they find the best dose to give. This is called a dose escalation study.

In part 2, the researchers want to find the best dose of RO5503781 to give alongside the chemotherapy drug cytarabine. Once they find this, some people will have RO5503781 only and others will have RO5503781 and cytarabine.

The first few people taking part will have a low dose. If they don’t have any serious side effects, the next few people will have a higher dose. And so on, until they find the best dose to give.

In part 3, everybody will have RO5503781 and cytarabine. Some people will also have idarubicin and some will have daunorubicin. Your doctor will decide which of these 2 drugs you have.

In part 4, everybody will have RO5503781 and cytarabine. A different formulation of RO5503781 will be used in this part of the study only.

RO5503781 is a tablet. You take it for 5 days every 4 weeks. Each 4 week period is called a cycle of treatment. Your doctor will tell you how many tablets to take.

Depending on which part of the trial you join, there may be days when you take RO5503781 at the hospital and not at home. Your doctor will tell you if this applies to you and when to do so.

You have cytarabine and idarubicin or daunorubicin as injections into a vein. If you have cytarabine, you have it each day for 6 or 7 days at the beginning of each cycle of treatment. If you have idarubicin or daunorubicin you have it on 3 days in each cycle of treatment.

The trial team will give you a diary to keep at home. In this you write down when you take your RO5503781 tablets.

You have at least 2 cycles of treatment. You may then continue treatment if it is helping you and the side effects aren’t too bad.

If you agree to take part in this study, the researchers will ask for extra blood samples and tissue samples from inside your mouth. They will take the tissue samples from inside your mouth using a cotton tipped swab and gently rub the inside of your cheek to collect some of the surface cells.

This is to find out what happens to RO5503781 in the body. On the days you give extra blood samples, you need to stay at the hospital for about 12 hours. Your doctor will tell you when this will happen and how often. You must agree to this to take part in this trial.