The purpose of this Managed Access Program (MAP), which is an Expanded Access, is to allow treatment with tisagenlecleucel (CTL019) for eligible patients diagnosed with large B-cell lymphomas who meet all of the following criteria: are 1) consistent with the approved prescribing information, 2) unable to receive commercially manufactured product due to failure of the incoming apheresis material to meet acceptance specifications or final outgoing product to meet the commercial release specifications or other specification within the prescribing information, and 3) where no overwhelming safety concerns has been identified for manufacture and release of the out of specification product. The requesting treating physician submitted a request toNovartis to access tisagenlecleucel that does not meet commercial specifications, which was reviewed and approved by the medical team experienced with tisagenlecleucel and the indication.
Adult patients who were, per the treating physician assessment, eligible for treatment with tisagenlecleucel per the approved prescribing information (PI).
Patients with relapsed or refractory (r/r) large B-cell lymphoma after two or more lines of systemic therapy including diffuse large B-cell lymphoma (DLBCL) not otherwise specified, high grade B-cell lymphoma and DLBCL arising from follicular lymphoma.
Has a patient specific batch of tisagenlecleucel allocated, however, either the incoming apheresis material is out of specification due to failure to meet acceptance specifications or the final manufactured product is out of specification due to failure to meet the commercial release specifications or other specification within the prescribing information.
Repeat leukapheresis is not clinically appropriate per the treating physician assessment
Product can be commercially manufactured per the specification of the country in which treatment will occur.
Active Hepatitis B, active or latent Hepatitis C, and HIV positivity
Uncontrolled active infection or inflammation
You will be asked to come to the doctor’s office/clinic/study site at least 3 times in order to make sure you are eligible to receive the tisagenlecleucel cells, and to prepare you for the experimental treatments.
You will have some tests that will help your doctor decide if you can receive tisagenlecleucel. The tests may include but are not limited to the following:
- Physical Examination (a “check-up”), including and measurement of your height, weight, temperature, blood pressure and heart rate (pulse)
- Pulmonary reserve assessment to determine the additional volume of air that your lungs can inhale and exhale when breathing
- An electrical tracing of your heartbeat (electrocardiogram or ECG), and a sound wave test of the heart (echocardiogram or ECHO) or a radioisotope study of your heart (MUGA)
- Blood tests for routine lab testing to check the status of your health and to see if you have HIV or hepatitis
- Blood tests that will look at the genetic make-up of your disease and the current status of your disease
- If you are female you will have a blood test to see if you are pregnant
- A urine sample will be obtained for routine analysis
- If not performed recently, a bone marrow biopsy and aspirate will be performed to look at the current status of your disease.
- If indicated and not performed recently, a spinal tap (lumbar puncture) will be performed to evaluate for the presence of leukemic blasts in your brain and spine.
- Imaging tests such as PET scan, CT scan or MRI (pictures of the body taken using x-ray or magnetic energy) may be performed. If indicated, you will undergo a biopsy of your lymph node(s) or other tissues (take a small piece of lymph node with a needle or by surgery) to look for potential disease.
- You may undergo a rapid influenza diagnostic test within 10 days prior to your planned tisagenlecleucel infusion. If you test positive for influenza, you may be prescribed a full course of treatment. You must complete the full course of treatment prior to receiving tisagenlecleucel.
- The total amount of blood that may be collected from you during this visit will be approximately 2 tablespoons. Some of these tests will only be repeated if there is a significant change in your health from the initial assessment.
If you meet the entry criteria of the MAP, T cells from your body will need to be collected in order to make the tisagenlecleucel cells.
After receiving tisagenlecleucel, it will be important for you to remain within 2 hours of your treatment site for at least 4 weeks following infusion so that your doctor can closely monitor your condition and reaction to tisagenlecleucel.
If you and your doctor decide that you are well enough, you may be able to leave the clinic site and remain outpatient within 2 hours of the treatment site. However, you will need to return to your doctor frequently at regular intervals as per your doctor’s recommendations so that your condition and safety can be closely monitored. It is very important that you come to all scheduled visits and that you follow your doctor’s instructions.