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

Study type

• Study topic:
  Human medicinal product
• 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, non-interventional post-authorization safety study, registry

Study drug and medical condition

• Name of medicine:
  CARVYKTI
• Study drug International non-proprietary name (INN) or common name:
  CILTACABTAGENE AUTOLEUCEL
• Anatomical Therapeutic Chemical (ATC) code:
  200000030588

  ciltacabtagene autoleucel
• Additional medical condition(s):
  Relapsed/refractory multiple myeloma

Population studied

• Age groups:
  Adults (18 to < 65 years)

  Adults (18 to < 46 years)

  Adults (46 to < 65 years)

  Elderly (≥ 65 years)

  Adults (65 to < 75 years)

  Adults (75 to < 85 years)

  Adults (85 years and over)
• Estimated number of subjects:
  300

Study design details

• Study design:
  This is a non-interventional PASS to describe the safety profile of cilta-cel in the treatment of adult patients with multiple myeloma, primarily in the European Union (EU) region, with the option to expand to other regions/countries.
• Main study objective:
  This study aims to document the short- and long-term safety of adult patients with multiple myeloma receiving ciltacabtagene autoleucel in the post-authorization setting per the health authority approved product information in the respective country/region.
• Outcomes:
  Primary outcomes: Safety will be measured through the rate of selected adverse events associated with the administration of cilta-cel regardless of causality and seriousness. Selected adverse events include but are not limited to subsequent malignancies, neurotoxicity, hematologic disorders, hypogammaglobulinemia, clinically significant infections, cytokine release syndrome, graft-versus-host disease and others.; ; Secondary outcomes: The effectiveness of cilta-cel measured through the following parameters: Overall survival (OS), Progression-free survival (PFS), Duration of response (DOR), Overall response rate (ORR), 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. All documented adverse events (AEs) will be analyzed. All AEs regardless of causality to cilta-cel beginning from product administration on Day 0 until Day 100 following CAR-T infusion will be analyzed. Thereafter, only nonserious AEs related to cilta-cel (with some exceptions) and all SAEs regardless of causality up to End of Study 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. 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.