London: Phase ll Cinical trial of 'omacetaxine' (OM) for CML patients who have not responded to at least 2 TKI 's

Doctors often treat chronic myeloid leukaemia (CML) with biological therapy. The most commonly used drugs are called tyrosine kinase inhibitors (TKIs). But sometimes CML doesn't respond to treatment with a TKI or becomes resistant to it after a time. And some people cannot carry on taking TKIs if they have bad side effects.

OM is a new type of chemotherapy. Researchers think it may help people with CML who have already had treatment with 2 different tyrosine kinase inhibitors.

The aims of this trial are to

* See if OM helps people with CML that did not respond or has become resistant to TKIs, or who could not take TKIs because of side effects
* Learn more about the side effects of OM

Recruitment

Starts 01/01/2007
Ends 31/12/2009

Phase

Phase 2

Who can enter this trial

You can enter this trial if you

* Have CML that has the Philadelphia chromosome and is in either chronic, accelerated or blast phase
* Have already had treatment with at least 2 different tyrosine kinase inhibitors and either your CML did not respond or has become resistant, or you could not carry on having this type of treatment because of side effects
* Finished any other treatment for CML (apart from hydroxyurea) at least 2 weeks ago and have recovered from any side effects
* Have satisfactory blood test results
* Are well enough to take part in the trial
* Are willing to use reliable contraception during this trial and for 6 months after if there is any chance that you or your partner could become pregnant
* Are at least 18 years old

You cannot enter this trial if you

* Are in blast crisis with immature white blood cells that stem from lymphoid stem cells in your blood or bone marrow
* Have any other cancer, apart from non melanoma skin cancer or carcinoma in situ of the cervix
* Have had another experimental drug as part of a clinical trial in the last month
* Have had a heart attack in the last 3 months or have another serious heart problem - the trial doctor will advise you about this
* Have an infection that is not responding to treatment
* Have any other serious medical condition
* Are HIV or HTLV positive
* Are pregnant or breast feeding

Trial design

This is an international trial that will recruit about100 people in different countries. There are 2 parts to the trial. These are called the induction phase and the maintenance phase.

During the induction phase you have OM injections under the skin (subcutaneous) twice each day for 2 weeks. You then have 2 weeks without any treatment. This 4 week period is called a cycle of treatment. You have up to 6 cycles of treatment, so the induction phase could last up to 6 months.

If you have a large number of white blood cells or platelets before you start treatment, you may also have a drug called hydroxyurea during the first 2 cycles of treatment.

If the treatment helps you, the doctors will ask you to take part in the maintenance phase of the trial. You have subcutaneous OM injections twice a day for one week, followed by 3 weeks without treatment. If you don't have any bad side effects, you can continue to have this treatment for as long as it helps you.

The trial team will teach you (or somebody you live with) how to give the injections at home. If this is not possible, a nurse can come to your home twice a day to give you the injections.

Hospital visits

You will see the doctors and have some tests before you start treatment. The tests include

* Physical examination
* A trace of your heart (ECG)
* Chest X-ray
* Blood tests
* Urine test

You will also have a bone marrow test if you have not had one in the last month.

During the induction phase of the trial, you go to hospital once a month to see the doctors. You have a blood test each week, but you may be able to have this at your GP surgery. In the first cycle only, you have another ECG after 2 weeks of treatment.

During the maintenance phase, you continue to go to hospital once a month. In the first 3 cycles of maintenance treatment, you have blood tests every 2 weeks. After that, you have them once a month.

You will have a number of bone marrow tests during treatment and an ECG at least once every 3 months.

Side effects

The most common side effects of OM are

* A drop in the number of white blood cells and platelets, leading to an increased risk of infection and bleeding or bruising problems
* Low blood pressure
* Fast or irregular heart beat

Other possible side effects include

* Diarrhoea
* Sickness
* Fever or chills
* Depression
* Tiredness

As OM is a new drug, there may be other side effects that doctors don't know about yet.

Location of trial

London

Contact details

The Information Nurses
Cancer Research UK
P.O. Box 123
61, Lincoln's Inn Fields
London
WC2A 3PX
Tel:
Email: cancer.info@cancer.org.uk

Chief Investigator

Dr D Marin

Supported by

ChemGenex Pharmaceuticals