A trial of vosaroxin with cytarabine for acute myeloid leukaemia (VALOR)

Cancer type:

Acute leukaemia

Acute myeloid leukaemia (AML)

Blood cancers

Leukaemia

Status:

Results

Phase:

Phase 3

This trial was for people whose acute myeloid leukaemia had not responded to treatment or had come back after the first treatment.

More about this trial

Chemotherapy is one of the usual first treatments for acute myeloid leukaemia (AML). But sometimes, the treatment doesn’t work or the leukaemia comes back. When this trial was done, doctors weren’t sure of the best way to treat this. So researchers were looking at new treatments.

In this trial, they looked at a new chemotherapy drug called vosaroxin. They wanted to see how well it worked alongside a standard chemotherapy drug for AML called cytarabine.

The trial compared the following treatments:

cytarabine and vosaroxin
cytarabine and a dummy drug (placebo )

The aims of the trial were to:

find out if vosaroxin alongside cytarabine is a useful treatment for AML that has come back or stopped responding to treatment
learn more about the side effects

Summary of results

The trial team found that on average, there was no benefit to adding vosaroxin to cytarabine. It did not increase the length of time that people lived after treatment (overall survival). However there was some suggestion that it might help people over 60 years old.

711 people with AML took part.

Everyone was put into 1 of 2 groups at random Open a glossary item .

356 had vosaroxin and cytarabine
355 had cytarabine and a dummy drug

They looked at how long people lived for after treatment. On average this was:

7.5 months in the people who had vosaroxin and cytarabine
6.1 months in the people who had vosaroxin and the dummy drug

Although these numbers look different, this difference wasn’t significant Open a glossary item when tested mathematically

The researchers did some more analysis on the results of AML subgroups. These groups were based on;

people’s age
where they lived
the type of leukaemia they had
any differences in how the leukaemia had responded to the first treatment

The researchers found in people over the age of 60, overall survival was:

7.1 months in people who had vosaroxin and cytarabine
5 months in people who had cytarabine and the dummy drug

The researchers say this suggests that adding vosaroxin to cytarabine might help people aged 60 years or older.

They also looked at how well treatment worked. They looked at whose leukaemia went away completely. This is called complete remission. This happened in:

107 people who had vosaroxin and cytarabine
58 cytarabine and the dummy drug

Even though more people had remission with the combination, the number of people who went on to have a stem cell transplant Open a glossary item was similar in each group. This might be because more people whose AML has come back or stopped responding to treatment are having stem cell transplants than in the past.

People who had vosaroxin had more serious side effects that included:

a drop in the number of white blood cells
high temperatures caused by a drop in the number of white blood cells (neutropenic fever)
sore mouth and lips (stomatitis)
infections including pneumonia
a drop in the level of potassium in the blood

But the trial team say the side effects were manageable and the increased side effects did not outweigh the benefit of having treatment.

The trial team concluded that there was no difference in overall survival. Although, having vosaroxin and cytarabine might be a useful treatment for people aged 60 years or older.

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.