Study for Subjects with Multiple Myeloma

This study is testing an experimental infusion called Ciltacabtagene autoleucel (cilta-cel) and the purpose of this study is to find out if it is safe and effective in people with multiple myeloma.

Cilta-cel treatment is a treatment designed to improve the immune response to this cancer (multiple myeloma) by collecting a patient's white blood cells through leukapheresis and genetically modifying the patient’s T-Cells to express a receptor that identifies and eliminates a specific type of cancer cell (BCMA expressing multiple myeloma cells). The cells are returned to the hospital for a one-time infusion into the patient. Cilta-cel is FDA approved in patients that have received at least one prior line of therapy including a proteasome inhibitor and an immunomodulatory agent, and are refractory to Lenalidomide.

Cilta-cel is a type of immunotherapy that is manufactured in a central laboratory using a patient’s own immune cells. The process of manufacturing cilta-cel can take about four weeks from the time the apheresis material is received until it is ready to be infused. Delays can occur if the final product does not meet the predetermined commercial release specifications set forth by the FDA. When the finished cilta-cel product does not meet the criteria it is called cilta-cel out-of-specification (OOS). Once a product is OOS, it is no longer considered commercial product, and not approved for use by the FDA. Once a patient has an OOS product, they have a number of options. If there is back-up apheresis material at the manufacturer from their original blood collection, they can choose to attempt a remanufacture to get a commercial cilta-cel product that is within specification and FDA approved. They can also choose to undergo leukapheresis again to collect a new sample of white blood cells to send to the manufacturer and attempt remanufacturing to get a commercial cilta-cel product that is within specification and FDA approved. They can opt to not take the product and receive a different treatment with their treating physician. Or they can request their OOS product through this expanded access program. Expanded access can be considered for patients with a serious/life-threatening disease or condition, where there is no alternative treatment or where other alternative treatments have been exhausted. In these circumstances, the physician and sponsor will conduct a benefit/risk assessment based on the OOS product results and patient clinical status. If positive, the product may be released through the EAP for use. Each case is also submitted to the FDA for notification or approval. There is no guarantee of patient benefit, but it is released to the patient for therapeutic intent (there is no research component to this access program). The OOS product is given as a single infusion, and not combined with any other anti-myeloma treatment.

Eligibility criteria is based on an individual benefit/risk assessment based on the OOS product and patient clinical status. In addition, as all OOS cilta-cel products begin as commercial CARVYKTI products, the patient population would be those able eligible to receive CARVYKTI based on the USPI (adult patients, so no one under 18). Also, vulnerable patients (as defined by IRB) are excluded per protocol.

This is a partial list of eligibility requirements. To inquire about your eligibility, please call the contact number provided. If you wish to inquire via email, please include the title of the study in your message.