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 Open a glossary item makes too many blood cells. In PV it is too many red blood cells  Open a glossary itemand in ET it is too many platelets Open a glossary item. These conditions are closely related to leukaemia Open a glossary item.

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

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:

Recruitment end:

How to join a clinical trial

Please note: In order to join a trial you will need to discuss it with your doctor, unless otherwise specified.

Please note - unless we state otherwise in the summary, you need to talk to your doctor about joining a trial.

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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Freephone 0808 800 4040

Last review date

CRUK internal database number:

9306

Please note - unless we state otherwise in the summary, you need to talk to your doctor about joining a trial.

Over 60,000 cancer patients enrolled on clinical trials in the UK last year.

Last reviewed:

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