A trial looking at ruxolitinib to treat polycythaemia vera and essential thrombocythaemia (MAJIC)

Cancer type:

Blood cancers

Polycythaemia

Thrombocythaemia

Status:

Results

Phase:

Phase 2

This trial looked at a drug called ruxolitinib to treat high risk polycythaemia vera (PV) and high risk essential thrombocythaemia (ET).

Polycythaemia vera and essential thrombocythaemia are myeloproliferative disorders Open a glossary item . These are conditions in which the bone marrow makes too many blood cells. In PV it is too many red blood cells and in ET it is too many platelets . These conditions are closely related to leukaemia .

This trial closed in 2016. These results published in 2017 are for essential thrombocythemia. The trial team will publish the results of polycythaemia vera at a later date. When these results are published we will update this summary.

More about this trial

Doctors treat these conditions with hydroxycarbamide. Unfortunately some people can’t take hydroxycarbamide and sometimes the disease stops responding to it. So researchers are looking for other treatments.

Ruxolitinib is a targeted cancer drug. It is a cancer growth blocker. It stops signals that cancer cells use to divide and grow.

We knew from research that ruxolitinib might help people with PV and ET.

The aims of this trial were to find out:

how well ruxolitinib worked for people with polycythaemia vera and essential thrombocythaemia who couldn’t have hydroxycarbamide
how safe it was

Summary of results

The team found that ruxolitinib isn’t any better than other treatment that is available for people with essential thrombocythaemia who can’t have hydroxycarbamide.

This was a phase 2 trial. 110 people with essential thrombocythaemia took part. It was a randomised trial. The people taking part were put into 1 of 2 treatment groups. And you couldn’t choose which group you were in.

58 people had ruxolitinib
52 people had best available treatment

MAJIC

Best Available Treatment (BAT)

The doctor chose the best available treatment. It was either a single drug or a combination. The most common ones used at least once included:

Changing and discontinuing treatment

After 2 years of follow up 30 people in the BAT group had changed their treatment at least once.

In total 45 people had stopped their treatment:

35 in the ruxolitinib group
10 in the BAT group

The main reasons included their disease had:

stopped responding to the treatment
changed (transformed) into acute myeloid leukaemia or myelofibrosis

Response to treatment

The trial team looked at the number of people whose cancer had completely gone (a complete response). They found that:

27 (46.5%) people in the ruxolitinib group
23 (44.2%) people in the BAT group

They also looked at the number of people whose cancer had got a bit better (a partial response). They found that:

27 people in the ruxolitinib group
27 people in the BAT group

For those who had ruxolitinib their essential thrombocythaemia responded significantly quicker to treatment than those who had BAT.

After 1 year of treatment the team looked at the number of people in each group who were alive and had no sign of their essential thrombocythemia getting worse. They found that it was similar in each group.

After 2 years of follow up the researchers looked at how many people in each group:

whose essential thrombocythemia had changed (transformation)
who had blood clots and bleeding episodes

Transformation

Essential thrombocythaemia can change (transform) into myelofibrosis and acute myeloid leukaemia (AML).

The number of people whose essential thrombocythemia had changed to myelofibrosis was:

8 in the ruxolitinib group
5 in the BAT group

Only 1 person’s essential thrombocythaemia in the ruxolitinib group had changed to AML.

Blood clots and bleeding episodes

Symptoms of essential thrombocythaemia are mostly caused by blood clots forming and bleeding problems.

The number of people who had blood clots were:

10 in the ruxolitinib group
3 in the BAT group

The number who had bleeding episodes were:

1 in the ruxolitinib group
5 in the BAT group

Side effects

The most common severe side effects in each group included:

a drop in blood cells
low amount of sodium in the blood
changes to the amount of minerals in the blood such as potassium, magnesium and phosphate

Quality of life

Overall during the 1st year the symptoms people had in each group weren’t different. But the symptoms of people in the ruxolitinib group were controlled more quickly than in the BAT group.

Compared to people in the BAT group, those in the ruxolitinib group reported:

better concentration
lower levels of anxiety and depression
a higher ability to do their daily activities

Conclusion

Ruxolitinib improved some of the symptoms of essential thrombocythaemia. But the trial team concluded that it didn’t work any better than best available treatment for essential thrombocythaemia.

All trial results help doctors and researchers understand more about a condition and the best way to treat them.

We have based this summary on information from the research team. 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 who did the research. We have not analysed the data ourselves.

Recruitment start:

01/08/2012

Recruitment end:

31/07/2016

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Chief Investigator

Prof Claire Harrison

Supported by

Bloodwise
Cambridge Blood and Stem Cell Biobank
Experimental Cancer Medicine Centre (ECMC)
Guy's and St Thomas' NHS Foundation Trust
Mayo Clinic
NIHR Clinical Research Network: Cancer
Novartis
University of Birmingham

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