A Post-authorization Safety Study to Evaluate the Safety of Multiple Myeloma Patients Treated with Ciltacabtagene Autoleucel

Study type

• Study type:
  Non-interventional study
• Scope of the study:
  Assessment of risk minimisation measure implementation or effectiveness

  Effectiveness study (incl. comparative)

  Safety study (incl. comparative)

Non-interventional study

• Non-interventional study design:
  Other
• Non-interventional study design, other:
  Prospective, secondary data analysis of databases

Study drug and medical condition

• Name of medicine:
  CARVYKTI
• Medical condition to be studied:
  Plasma cell myeloma refractory

Population studied

• Age groups:
  Adults (18 to < 46 years)

  Adults (46 to < 65 years)

  Adults (65 to < 75 years)

  Adults (75 to < 85 years)

  Adults (85 years and over)
• Estimated number of subjects:
  1500

Study design details

• Main study objective:
  The primary objective of the study is to evaluate the short- and long-term safety including the risk of subsequent malignancy, of cilta-cel in adult patients with multiple myeloma. Long-term data on replication-competent lentivirus (RCL) in patients who develop subsequent malignancies will also be collected, where allowed per local regulations in the context of a non-interventional study.
• Outcomes:
  Primary outcomes of the study are the short- and long-term safety including the subsequent malignancy and replication-competent lentivirus (RCL) in patients who develop subsequent malignancies. The secondary outcomes include: 1) Overall response rate (ORR) 2)Duration of response (DOR) 3)Progression-free survival 4) Overall survival (OS) 5)Cilta-cel's effect on myeloma-related comorbidities (amyloidosis and POEMS syndrome, if present)
• Data analysis plan:
  Analysis set will include all patients who meet the selection criteria. Patient demographics, medical and disease history, current disease status and any previous therapies for multiple myeloma will be descriptively summarized at baseline. Only selected toxicities will be collected in the registry and reported in this study. All documented AEs will be analyzed. All AEs regardless of causality to cilta-cel will be analyzed. The verbatim terms used to identify AEs will be coded per MedDRA. For each AE, the percentage of patients who experience at least 1 occurrence of the given event will be summarized. Where appropriate, additional summaries, listings may be provided. Additionally, cumulative incidence estimates, or rates of AEs reported in person years may be used. The survival analysis will be performed for time-to-event variables (i.e. PFS, OS). Responses to cilta-cel will be summarized with count and percentage.