A trial of rigosertib for myelodysplastic syndrome (INSPIRE)

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

Cancer type:

Blood cancers

Myelodysplastic syndrome (MDS)

Status:

Closed

Phase:

Phase 3

This trial is looking at rigosertib for people who have already had treatment for their myelodysplastic syndrome (MDS).

Myelodysplastic syndrome (MDS) causes the bone marrow to produce blood cells that aren’t mature and don’t function properly. This causes a drop in the normal blood cells.

More about this trial

Treatment for MDS can include chemotherapy such as azacitidine and a stem cell transplant. Unfortunately sometimes these treatments do not work. And the MDS continues to get worse or comes back after treatment.

Researchers want to see if rigosertib can help people in this situation. They will compare rigosertib with the doctor’s choice of standard treatment.

The aims of this trial are to find:

how well rigosertib works for people with MDS that has continued to get worse during treatment or came back afterwards
how rigosertib affects quality of life
how safe it is
what happens to rigosertib in the body

Who can enter

The following bullet points list the entry conditions for this trial. Talk to your doctor or the trial team if you are unsure about any of these. They will be able to advise you.

You may be able to join this trial if all of the following apply. You have myelodysplastic syndrome (MDS) and you are in one of the following situations:

Your MDS didn’t get any better after treatment with azacitidine or decitabine
Your MDS continued to get worse while having azacitidine or decitabine
Your MDS came back after having responded to azacitidine or decitabine
You are unable to have azacitidine or decitabine
You have MDS with the 5 q syndrome and it did not respond to, or got worse, while taking lenalidomide

And all of the following apply:

You have between 5% and 30% blast cells in your bone marrow
Your blood test results show you have a low red blood cell count, low platelets or a low neutrophil count
All your other blood results are satisfactory
Your last dose of azacitidine or decitabine was within the past 6 months
Your total time of any MDS treatment was 9 months or less,and, or you had a total of 9 cycles of treatment in 12 months or less
You had an allogeneic stem cell transplant that was not successful or you were not able to have a transplant or did not want to have one
You are well enough to be up and about for at least half the day (performance status 0, 1 or 2)
You are between 18 and 81 years old
You are willing to use reliable contraception during treatment and for a month afterwards if there is any possibility you or your partner could become pregnant

You cannot join this trial if any of these apply.

Cancer related

You have been diagnosed with acute myeloid leukaemia (AML) and the blast cells in your bone marrow or blood was or is greater than 30%
You are having an allogeneic stem cell transplant
You are able to have chemotherapy to get rid of the blast cells in your bone marrow (a remission)
You have had treatment for MDS in the past 4 weeks apart from treatment such as growth factors to help control the symptoms
You are currently having treatment for MDS including as part of another clinical trial
You have had previous treatment with rigosertib
You have had another cancer in the past year apart from non melanoma skin cancer or in situ carcinoma of the cervix or breast

Medical conditions

You have an infection that isn’t responding to treatment
You have HIV that isn't controlled
You have or had hepatitis B and there are antibodies to it in your blood
You have or had hepatitis C and the virus is present in your blood
You have not fully recovered from any major surgery
You are to have major surgery in the 3 weeks before been randomised for this trial
You have high blood pressure that isn’t controlled by medication
You have had fits (seizures) in the 3 months before been randomised and they are new
You have fits that are not controlled by medication
You are having steroids unless it is for an ongoing (chronic) condition and the dose is 20mg a day or less
You have any other medical or mental health condition that the trial team think could affect you taking part

Other

You are pregnant or breastfeeding

Trial design

This is a phase 3 trial. The researchers need 225 people to join.

It is a randomised trial. People taking part are put into 1 of 2 treatment groups by a computer. Neither you or your doctor can choose which group you are in. The 2 groups are:

rigosertib
doctor’s choice of treatment

You have a 2 out of 3 chance of being in the rigosertib group.

INSPIRE Trial Diagram

Before starting rigosertib you have a central line or portacath put in if you don’t already have one.

You have a pump attached to the central line that delivers the rigosertib continuously. You have it over 3 days.

At the start you have it every 2 weeks for 16 weeks. Afterwards it is every 4 weeks.

You continue having rigosertib as long as it is helping and the side effects aren’t too bad.

If you are not having rigosertib your doctor will talk to you about what other treatment might be best for you. This could include more chemotherapy or supportive treatment such as blood transfusions.

Samples

The researchers will ask for samples of your bone marrow and blood. These are taken when you have a bone marrow test and blood tests done as part of your routine care.

These samples are used to find out more about MDS. They will also use the blood samples to find what happens to rigosertib in the body.

You don’t have to agree to give these samples to the researchers. You can still take part in the trial.

Quality of life

You fill in a questionnaire before you start treatment, then every 4 weeks during treatment and at the end of treatment. The questions will ask about any symptoms you have and how you are feeling. This is a quality of life study.

Hospital visits

You see the doctor to have some tests before taking part. These tests include:

a physical examination
blood tests
urine test
heart trace (ECG)
bone marrow test if needed

During treatment you see the doctor regularly. You have:

blood tests every week
a physical examination every 2 weeks
a urine test every 4 weeks

You have a bone marrow test every 2 months up to 6 months and then a year after starting treatment.

At the end of treatment you see the doctor for a physical examination and blood tests.

After treatment the researchers will phone every month to see how you are and ask if you have started any more treatment. If you prefer not to be phoned they can contact your family doctor (GP).

Side effects

Rigosertib might have side effects we don’t know about. The most common side effects reported include:

a drop in blood cells causing shortness of breath, an increased risk of infection, bruising and bleeding
diarrhoea or constipation
tiredness
feeling or being sick
feeling of fullness and tightness in the stomach (bloating)
tummy (abdominal) pain
difficulty sleeping or falling asleep
headache
dizziness
difficulty or pain passing urine
blood in the urine
high temperature (fever)
a change to the way your liver works
a decrease in the amount of potassium and sodium on the blood
a decrease in the amount of the protein albumin in the blood causing general swelling
swelling of the arms and legs
loss of appetite and changes to taste