A study looking at the drug palifermin in people having chemotherapy and stem cell transplant for myeloma (20050219)

Who can enter

You can enter this trial if you

• Have myeloma
• Are going to have high dose chemotherapy and stem cell transplant using your own blood stem cells (autologous stem cell transplant)
• Have satisfactory lung function tests
• Have satisfactory blood tests
• Are well enough to take part (performance status 0, 1 or 2)
• Are willing to use reliable contraception for at least 30 days after your transplant 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

• Have had any cancer except carcinoma in situ of the cervix, non melanoma skin cancer or another cancer that has been removed with surgery and have been clear for at least 3 years
• Have already had a stem cell transplant using your own stem cells (autologous) or someone else’s (allogeneic)
• Have already had treatment with palifermin or a similar drug - you can check this with your doctor
• Have taken part in any other clinical trial within a month of being accepted for this study
• Are known to be allergic to any of the drugs in the study, including drugs that come from the bacteria E coli
• Have kidneys that do not work properly, and have regular treatment to clean your blood (dialysis)
• Already have painful sores in your mouth
• Are very overweight (Body mass index (BMI) greater than 35)
• Have hepatitis B, hepatitis C or HIV
• Are pregnant or breastfeeding
• Are not willing to use reliable contraception
• Are over 70 years old
• Have already taken part in this study

Trial design

This is a phase 3 study. It is running in approximately 40 centres across Europe. It will recruit 275 people into 3 groups. This is a randomised study. The people taking part are put into treatment groups by a computer. Neither you nor your doctor will be able to decide or know which group you are in. Researchers call this a ‘double blind’ study.

Two groups will have a dummy drug (placebo) during the study. This is to help researchers see whether any benefits or side effects come from palifermin or not. Researchers call this a ‘placebo controlled study’. Everyone will have 6 doses of their study drug (palifermin or placebo). Four out of 5 people (80%) will have palifermin in this study. One out of 5 people (20%) will have the placebo.

If you are in group 1, you will have palifermin before and after your chemotherapy and transplant. If you are in group 2, you will have palifermin before your chemotherapy and a dummy drug (placebo) after your transplant. If you are in group 3, you will have a dummy drug before and after your chemotherapy and transplant.

You will have either palifermin or the placebo, or both, through a drip into a vein, over about 30 seconds.

You will have a daily dose of your particular study drug (palifermin or placebo) for 3 days before your chemotherapy. You will then have another daily dose of your study drug for 3 days after your transplant. You will also have regular assessments, including questionnaires and blood tests to monitor your progress on your study drug.

Researchers also want to find out if palifermin causes cloudiness in the lens of the eye (cataracts), or makes them worse. You can take part in this section if you can see well, and have at least one healthy, or only very slightly clouded lens. You cannot take part if you have any condition that may affect your sight or make it difficult to examine your eye, are waiting for cataract surgery, or have already had it.

You will have eye examinations to see if you can take part in this section of the study. If you take part in the cataract mini study, you will have eye examinations during your study appointments.

Hospital visits

Before the study, you will see the doctor and have some tests. These tests include

• Lung function test
• Physical examination including heart rate, blood pressure and temperature
• Blood tests
• Eye examination
• A questionnaire about your health
• Pregnancy test (if appropriate)
• Mouth check (oral cavity assessment)

It may be possible to have your first 3 doses of study drug as an outpatient. You can discuss this with your study team. When you come to hospital to prepare for your chemotherapy and transplant, you will continue your study treatment as an inpatient. You will also have daily assessments until you leave hospital. These assessments include

• A quality of life questionnaire
• Mouth check (oral cavity assessment)
• Temperature
• Blood tests
• Reporting any side effects you may have

If you still have a sore mouth when you leave hospital, you will see the study team 3 times a week until your mouth is better. At these visits, you will complete a quality of life questionnaire and a mouth check. After the study, you will see the study team one month after your transplant and then at

• 3 months
• 6 months
• 12 months

At these visits, you will have some or all of the following checks

• Quality of life questionnaire
• Mouth check (at one month visit)
• Blood test (at one month visit)
• Eye examination

You will then see the study team once a year for up to 10 years after your transplant, to tell them about your health.

Throughout the study and after, you will also remain under the care of your specialist cancer team.

Side effects

Common side effects of palifermin include

• Rash, itching, reddening and swelling of the skin of your face, neck, hands and upper body
• Tingling and a ‘thickening’ feeling of your mouth and lips
• Tongue colour changes
• Taste changes
• General pain in your body, and one or more joints
• Shortness of breath
• Flushed skin
• Headache
• Swelling around the eyes
• Sensitive skin

It is possible that you may have a mild allergic reaction to palifermin. The nurses will watch you closely, and if you do have a reaction, they will give you a drug to stop it straight away.

You can find out more about melphalan chemotherapy on CancerHelp UK.