A trial looking at RO5503781 for acute myeloid leukaemia

Please note - this trial is no longer recruiting patients. We hope to add results when they are available.

Cancer type:

Acute leukaemia

Acute myeloid leukaemia (AML)

Blood cancers

Leukaemia

Status:

Closed

Phase:

Phase 1

This trial is looking at a new drug called RO5503781 for people with acute myeloid leukaemia (AML).

Doctors can treat acute myeloid leukaemia (AML) with chemotherapy. This works, but doctors are always looking for ways to improve treatment. In this trial they are looking at a drug called RO5503781.

RO5503781 is a type of biological therapy. It is a cancer growth blocker. It stops signals that cancer cells use to divide and grow.

Researchers think that adding RO5503781 to chemotherapy may work better than chemotherapy alone.

The aims of this trial are to

Find the highest safest dose of RO5503781 you can have on its own and with chemotherapy
Learn more about the side effects of RO5503781 on its own and with chemotherapy
See how well the combination of RO5503781 and chemotherapy works for acute myeloid leukaemia

Who can enter

There are a number of parts in this trial. Which part you are able to join will depend on your individual situation. You can ask your doctor about this.

You may be able to join this trial if all of the following apply. You

Have acute myeloid leukaemia (AML)
Are well enough to be up and about for at least half the day (performance status 0, 1 or 2). To join some parts of the trial, you must be well enough to carry out all your normal activities, apart from heavy physical work (performance status of 0 or 1)
Have satisfactory blood test results
Are willing to use reliable contraception during treatment and for 10 days afterwards if there is any chance you or your partner could become pregnant
Are at least 18 years old

You cannot join this trial if any of these apply. You

Have a type of AML called acute promyelocytic leukaemia (APL)
Have AML in your brain or spine
Have had any treatment for your AML in the past 2 weeks
Have had drugs similar to RO5503781 (your doctor can tell you this)
Still have any side effects from earlier treatment unless they are mild (apart from hair loss)
Take other medication that affects body substances called CYP enzymes (your doctor can advise you about this)
Have certain heart problems (the trial team can advise about this)
Have any other medical condition that could affect you taking part in this trial
Are HIV positive and are having combination treatment
Are pregnant or breastfeeding

Trial design

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.

Hospital visits

You see the doctor to have some tests before taking part in this trial. These tests include

A physical examination
3 heart traces (ECG )
Bone marrow test
Blood tests
Urine test
A sample of cells taken from the inside of your cheek
Chest X-ray or CT scan

During treatment you will have the same tests again while having treatment, apart from the sample of cells from inside of your cheek and chest X-ray or CT scan.

After you finish treatment you will see your doctor a month later for a final visit. If your leukaemia disappears (a complete remission Open a glossary item ) you will see your doctor every 2 months until a year from when you started treatment.

Side effects

RO5503781 is a new drug and there may be side effects we don’t know about yet. The side effects reported so far include

Feeling or being sick
Diarrhoea or constipation
Changes to appetite and taste
A drop in blood cells causing an increased risk of infection, bruising and bleeding
Tiredness
Headache
Weakness and loss of strength
Dry mouth
Fever

The most common side effects of cytarabine, idarubicin and daunorubicin are

A drop in blood cells causing an increased risk of infection, bruising and bleeding
Feeling or being sick
Diarrhoea
Mouth ulcers
Hair loss
Changes to the way your liver and heart works
Fever
Skin rash

Your doctor will talk to you about the possible side effects of treatment before you agree to take part in the trial.

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