Patient information brochure

Transcription

1 Clinical Development and Medical Affairs Patient information brochure 1. European Medicines Agency. Nilotinib Summary of Product Characteristics. March Available from: jsp?curl=pages/medicines/human/medicines/000798/human_med_ jsp&mid=wc0b01ac058001d124 [Last accessed April 2013] Novartis Pharma AG, CH-4002, Basel, Switzerland, Novartis 2013, 04/13, G-CML

2 Thank you......for your interest in the ENESTPath study. ENESTPath is a clinical trial for patients with chronic myeloid leukaemia (CML) who are currently being treated with imatinib and who have had a good, but not optimal response to treatment. The ENESTPath study is looking at two things. One is whether changing treatment from imatinib to nilotinib, a more recent treatment for CML and one which appears to give patients a deeper response, will result in a better response to treatment for people with CML. The second stage of the study will tell us whether it may be safe for people with CML who have achieved and sustained a deep response with nilotinib to eventually stop treatment. This is called the treatment free remission phase of the study. What is a clinical trial? A clinical trial is a type of medical research that compares one way of treating patients with another. Most trials compare a new drug or medical approach with a standard drug that is already available or with a sugar pill (a placebo). Who conducts clinical trials? Every clinical trial is led by a principal investigator, who is a medical doctor. Clinical trials also have a research team that may include other doctors, nurses and other healthcare professionals. Clinical trials can be sponsored, or funded, by pharmaceutical companies (as is the case for ENESTPath), academic medical centres, voluntary groups, and other organisations, in addition to government agencies. Doctors, healthcare providers, and other individuals can also sponsor clinical research. How will the ENESTPath study be run? The diagram below shows how the study is designed. There are two stages to the study. People with a good response to nilotinib treatment in the first stage will continue to the second stage, where we will investigate if it is safe for people to eventually stop treatment altogether. First stage Second stage This brochure and the consent form have been developed by Novartis, the sponsor of the study, to provide you with the information you need to make a decision on whether you would like to participate. We hope that most of the questions you may have will be answered in this brochure and by reading the consent form, but if you have any additional questions, or would like to discuss the study further, please contact [INSERT LOCAL INVESTIGATOR NAME, AFFILIATION AND CONTACT DETAILS]. ALL patients: 2 years of treatment with Nilotinib Check if you have reached MR4.0 YES You will be chosen at random to enter one of these treatment arms arm 1 arm 2 1 year of treatment with Nilotinib 3 years of no treatment Check if you have reached MR4.0 YES 2 years of no treatment NO NO Exit from study with monitoring by your doctor until 5 years from when you first entered Novartis Oncology Clinical Development and Medical Affairs 4

3 Patient information brochure The way we will decide how good your response to treatment has been is based on the level of BCR- ABL gene expression in your body the abnormal protein that causes leukaemia cells to grow. If this has been reduced to very low levels you have achieved what is known as molecular response 4.0, which is abbreviated to MR4.0. If you maintain this low level for a sustained period of time then you will be eligible for the second stage of the study. What does it mean to reach MMR and MR4.0? To reach a major molecular response (MMR) or MR4.0, the level of BCR-ABL and therefore the number of leukaemia cells in your blood has to be reduced to very low levels. This example uses a typical sample of 100,000 white blood cells to show you how low: How long is the ENESTPath study? The study is scheduled to last for approximately 5 years. Who can enter the ENESTPath study? To ensure that the people who join the ENESTPath study get the most benefit, and to ensure their safety, there are strict criteria around who is able to enter the study. Your doctor will help to decide whether you are able to take part; however the main criteria we are looking for are outlined below: You must have been diagnosed with chronic phase Philadelphia chromosome positive CML (Ph+ CP CML) been treated with imatinib since you were diagnosed, and for a minimum of 2 years 80,000 NORMAL WHITE BLOOD CELLS 99,980 NORMAL WHITE BLOOD CELLS 99,998 NORMAL WHITE BLOOD CELLS responded to treatment so that your levels of the BCR-ABL protein are low enough to be in major molecular response (see diagram in the What does it mean to reach MMR and MR4.0? section). If your levels are not low enough, you may need to have a bone marrow assessment to examine your response to treatment You must not have 20,000 leukemia CELLS 20 leukemia CELLS 2 leukemia CELLS responded so well to imatinib that you have already achieved very low levels of BCR-ABL (MR4.0 or better) had any previous treatment for CML other than with imatinib, apart from chemotherapy DIAGNOSIS MMR MR4.0 Novartis is responsible for providing the study drugs and will pay for any medical tests and procedures that are performed for the purpose of the study. What is nilotinib and how does it work? Nilotinib is a medicine for CML that you take orally as a capsule. In CML, blood cells make an abnormal protein called BCR-ABL. The BCR-ABL protein causes leukaemia cells to grow and it interferes with the body s ability to produce white blood cells, an essential part of your body s immune system. Nilotinib blocks BCR-ABL activity and hence the growth of leukaemia cells, therefore reducing their number in the blood. any other severe and/or uncontrolled health problem that your doctor thinks could put you at risk or mean you are not able to take part in the study a history of any other type of cancer in the last 5 years that spread to other parts of your body or has not been fully cured recently had surgery that you are still recovering from treatment with any other drugs that are being investigated in clinical trials in the last 4 weeks any problems with your stomach or digestion that mean you are not able to absorb the study drug properly Nilotinib has been approved by health authorities in the USA and Europe since 2007 for the treatment of CML. 5 Novartis Oncology Clinical Development and Medical Affairs 6

4 Patient information brochure What treatments will I receive in the ENESTPath study? The only treatment you will receive in this study is nilotinib. This drug produces more effective inhibition of the BCR-ABL protein than imatinib, and there are some other important differences between nilotinib and imatinib you should be aware of. How will I take the medication 1? Each capsule of Nilotinib contains 150mg of the drug capsules to be taken orally in the morning Two capsules to be taken orally in the evening Will nilotinib affect other medications I am taking 1? There are some drugs which interact with nilotinib and should either be used with caution or not used at all. If you are taking any medications then your doctor will advise you of potential problems and discuss your treatment options with you. Approximately between doses The dosing regimen for Nilotinib is more complicated than you will be used to with Imatinib Unlike Imatinib, Nilotinib must be taken on an empty stomach and should not be taken with food No food should be eaten for 2 hours before you take Nilotinib No food or drink other than plain water should be taken for at least an hour after you have taken Nilotinib Capsules should be swallowed whole with a full glass of plain water and should not be chewed If you experienced any side effects with imatinib it does not mean you will experience side effects with nilotinib, and vice versa if you did not experience any side effects with imatinib you may experience side effects with nilotinib in this study. More detailed information on the side effects you may experience with nilotinib are available in the consent form and should be discussed with your doctor. If you experience any side effects with nilotinib treatment, you should tell your doctor immediately. 7 Novartis Oncology Clinical Development and Medical Affairs 8

5 Patient information brochure Why should I join the ENESTPath study? As with all studies, there is no guarantee that the treatment in this study will work for you. However there is evidence that people with CML have a better response to treatment with nilotinib than with imatinib, and if you do respond well to nilotinib treatment you may be able to stop your treatment during the treatment-free remission phase. During this stage of the study there is a small chance that your CML may progress, however this has not been the case in other similar studies and the primary objective of ENESTPath is to prove that this is not the case. Close monitoring during this phase will ensure that any new occurrence of CML leukaemia cells will be detected and treated very quickly. The information on treatment-free remission will also contribute to new information about better medical care, benefiting CML patients in the future. Are there any risks if I join the ENESTPath study? As with all treatments, there can be a risk associated with possible side effects of nilotinib treatment and with the standard medical tests carried out as part of the study at each visit. Information on the possible side effects you may experience with nilotinib is available in the consent form and should be discussed with your doctor. If your doctor thinks you may not be responding well to treatment at certain points in the study, for example if your level of BCR-ABL protein is less than major molecular response at screening or you lose major molecular response during the study (see diagram in the What does it mean to reach MMR and MR4.0? section), they may need to take a sample of tissue from inside of your bone (a bone marrow assessment) to look at your response to treatment. This will only occur in exceptional circumstances and is not routine. How often will I need to see my doctor? If you are able to participate in the study, you will see your doctor again within 4 weeks to receive your first dose of nilotinib. At this time your doctor will conduct a physical examination and check your vital signs, as well as take some blood and do some non-invasive tests that look at the function of your heart known as an electrocardiogram (ECG) and echocardiogram. We want to perform these tests as a precaution and will compare the results to another ECG test after 3 months to check the function of your heart has stayed the same. You may have to have additional ECGs to monitor you during the study if your doctor thinks they are needed. For the first 2 years of the study you will be asked to visit your doctor every 3 months for a physical examination, to take some blood and monitor your response to treatment as well as determining if you are experiencing any side effects. If you are eligible to enter the second stage of the study, you will be chosen to either carry on taking nilotinib for another year before stopping treatment, or to stop taking it straight away. If you continue taking nilotinib for another year, you will still be asked to see your doctor every 3 months. When you stop treatment you will be asked to see your doctor every month for the first 6 months, every other month for the following 6 months and then every 3 months for the rest of the study (which will be either 2 or 3 years depending on when you stopped nilotinib). If you do not enter the second stage of the study, your doctor will run some tests and decide if you should continue on nilotinib treatment or if you should change to a different drug. After this, your doctor will contact you every 3 months until the end of the study 5 years from when you first entered to ask you questions about your disease and its treatment. How will I be protected against any risks in the ENESTPath study? Your safety is a key priority for us in this study, and for this reason your doctor will explain all of the potential risks of treatment with nilotinib and ask you to visit regularly to monitor your response to the treatment and any side effects. If you experience any side effects then your doctor will discuss the best way of managing these with you. If you experience any side effects with nilotinib, it is very important that you tell your doctor immediately. 9 Novartis Oncology Clinical Development and Medical Affairs 10

6 INSERT TITLE HERE What if I don t respond to my treatment or stop responding? Effectively treating CML is a key priority for us in this study, so all patients are closely monitored for their response to treatment. Unfortunately for some people who reach treatment-free remission, leukaemia cells will start to grow again. If at 3 visits in a row the level of BCR-ABL is less than the MR4.0 level, or if at one visit someone has lost major molecular response (see diagram in the What does it mean to reach MMR and MR4.0? section) your doctor will ask you to start taking nilotinib again. Based on people in similar studies, the number of leukaemia cells quickly reduced to very low levels when nilotinib was restarted. If at any time your BCR-ABL level is found to be less than major molecular response (see diagram in the What does it mean to reach MMR and MR4.0? section), a test will need to be carried out to check the stem cells in your bone marrow to ensure that nilotinib is the right treatment for your disease. According to the test results, your doctor will advise if you should continue on nilotinib treatment or change to a different drug. Am I able to become pregnant during the ENESTPath study? The effects of nilotinib on pregnancy and unborn babies are not known and nilotinib treatment may have harmful side effects for pregnant women. 1 Women participating in this study should not try to become pregnant during the study period or for up to 3 months after the last day they took nilotinib. How can I be sure the ENESTPath study will be carried out correctly and who will monitor this? As with all studies, information about study participants will be kept strictly confidential and according to the relevant laws and regulations. As with all clinical trials, the ENESTPath study has been reviewed and approved by an independent ethics committee. Novartis and local doctors running the trial are responsible for the conduct of the study. Participation in this study is entirely voluntary for patients, and if you wish to do so you are able to withdraw from the study at any point and without any reason. 11 Novartis Oncology Clinical Development and Medical Affairs 12