PARENT AND PATIENT INFORMATION SHEET
A PHASE I STUDY IN CHILDREN WITH ADVANCED CANCER TO EVALUATE
THE SAFETY AND PHARMACOKINETICS OF SPI-77 WHEN GIVEN
EVERY FOUR WEEKS
The drug cisplatin is currently used for the treatment of children with cancer. However, cisplatin is known to cause kidney damage in some children. SPI-77 is a new anti-cancer drug in which cisplatin is contained in an envelope of lipids or fats called a liposome. We hope that this will make cisplatin safer and reduce the unwanted kidney and hearing side-effects. However, we do not know yet if SPI-77 will help children with cancer. Also we do not know what the side-effects of SPI-77 might be in children, or what the correct dose should be. We are therefore inviting you to take part in a new study of this drug we are undertaking in children who have already received treatment for their cancer, but still have tumour present. We cannot say that SPI-77 will be of benefit to your child but we hope the results may be used to help your child and other patients in the future.
Before your child receives SPI-77 a number of tests will be performed to make sure he/she is suitable for the study. The size of your child's tumour(s) will be measured using x-rays or scans. Tests will include blood tests, urine tests, a heart test (electrocardiogram, ECG) and a hearing test. Your child will also have a complete physical examination and there will be a full review of your child's medical history.
We would wish to give your child an intravenous infusion of SPI-77 which takes at least five hours. During the infusion and afterwards we would take blood samples at specified times for the next 7 days to measure the concentration of SPI-77 in the blood. This is to learn how the drug is handled by the body and is very important in helping us to understand any differences in handling of the drug between children and adults. If your child is old enough we would also wish to collect all their urine for the first 4 days after the first dose of SPI-77, then on some days during the study the first urine sample of the morning. (The days that we need these samples will be explained to you). The samples should be kept in the fridge or cool box until they are brought with you on your next visit to the hospital.
Following SPI-77 infusion we would need to follow what happens very carefully, examining your child and taking more blood tests once a week for another 3 weeks, and asking you to keep a diary recording the general health of your child. It is intended that your child will receive SPI-77 every 4 weeks (this is called a cycle). After your child has completed 2 cycles of SPI-77, or may be sooner, we would wish to repeat some investigations to look at how the tumour is responding to SPI-77. If the treatment is progressing well, we would give further courses. At some point during the treatment we may wish to increase the dose of SPI-77, you will be told when this will happen.
When your child completes or stops the study they will have a final complete evaluation with a physical examination, an ECG, blood and urine tests, a hearing test, and review of any medication changes or medical problems which you may have noted during the previous month. The x-ray or scan investigations to measure the size of your child's tumour will be repeated again. Any side effects continuing at that time will be documented until they have resolved or stabilised.
The side effects of SPI-77 in humans are not known as very few patients have been treated to date. We will therefore be watching your child carefully to see if there are any unwanted effects caused by having SPI-77 in the body for longer than cisplatin would have been present.
It is hoped that because of the fat envelope many of the side effects seen with cisplatin will not occur with SPI-77. However, there is a possibility that some or all of the side effects associated with cisplatin could occur. The hearing and kidney problems of cisplatin have already been mentibned. It?is important that you are aware of the more common side effects of cisplatin which are as follows: nausea and vomiting, a reduction in white blood cell count which can lead to infections, a reduction in platelet count which can lead to bleeding, a reduction in haemoglobin levels which can cause weakness; ringing in the ears or deafness or ear pain, numbness, tingling, weakness or pain in the arms and legs or elsewhere on the body, fatigue, loss of appetite, altered taste or loss of taste, diarrhoea, sores in the mouth and throat, loss of hair which normally grows back after stopping treatment. Sometimes an allergic reaction can occur within a few minutes of starting the drug, which can cause facial flushing, rapid heart beat, low blood pressure and/or difficulty breathing. However, medication will be given before the SPI-77 infusion to reduce the possibility of this occurring.
Transfusions and medications are available if needed to overcome the decreases in blood cells until the bone marrow recovers. If needed antibiotics are available to help treat infections.
Participation in the study is entirely voluntary, and deciding that you do not want your child to take part will not affect his/her treatment in any way. If your child's disease is not responding to treatment, or they experience unacceptable side effects, treatment may be stopped if it is believed that this would be in your child's best interest. You can also ask at any time during the study for the treatment to be stopped. Other treatments for your child's care may be available these will be fully discussed with you.
Whilst your child is participating in this study, you will be kept informed of any new information about the treatment that may affect your willingness for your child to continue. If any changes are needed in the study (e.g. more blood samples) you will be asked for your agreement before this happens.
Participation in the study is entirely confidential. The medical information collected in this study will be anonymous, your child will only be identified by their initials and a number, The data will be collected in files called Case Report Forms (CRFs) and a record of treatment and response to the study medication will be kept in their hospital records . The data will be collected by SEQUUS pharmaceuticals, Inc. and may be submitted to Government Health Authorities.. It is important that all the information collected by SEQUUS Pharmaceuticals is accurate therefore it is necessary for authorised persons from the company and possibly independent auditors and/or the Government Health Authorities to look at your hospital records. This will only be done with your permission and will be performed in the strictest confidence. If information is published about this study, your child will not be identified in any way.
In the event of research-related injury, treatment, and if necessary, compensation, will be made available. This will be governed by the principles of the ABPI (Association of British Pharmaceutical Industry) Guidelines on Clinical Trial Compensation 1991. By signing this consent form you are NOT waiving any of your child's legal rights.
General information regarding this clinical trial is available as part of the Hospital's Patient Information Service but any specific questions about the study should be discussed with Professor Pinkerton or Mrs Dick.
If you require flirther information regarding this study please contact Professor Pinkerton, on extension 3329 or Mrs Gina Dick on bleep 374 at The Royal Marsden NHS Trust, Children's Department, telephone No.0181-642-6011.