Non-interventional, post-authorization safety study (PASS) of patients treated with commercially available liso-cel (lisocabtagene maraleucel) for large B-cell lymphomas (JCAR017-BCM-005)

Study type

• Study topic:
  Human medicinal product
• Study type:
  Non-interventional study
• Scope of the study:
  Effectiveness study (incl. comparative)

  Safety study (incl. comparative)
• Data collection methods:
  Secondary use of data

Non-interventional study

• Non-interventional study design:
  Cohort

Study drug and medical condition

• Medicinal product name:
  BREYANZI
• Study drug International non-proprietary name (INN) or common name:
  LISOCABTAGENE MARALEUCEL
• Anatomical Therapeutic Chemical (ATC) code:
  (L01XL08) lisocabtagene maraleucel

  lisocabtagene maraleucel
• Medical condition to be studied:
  Follicular lymphoma
• Additional medical condition(s):
  relapsed/refractory diffuse large B-cell lymphoma,primary mediastinal B-cell lymphoma

Population studied

• Short description of the study population:
  All patients who meet the following inclusion criterion will be selected from the registries: ;  Patient must have been treated with at least 1 infusion of liso-cel in the postmarketing setting for an approved indication according to the EMA SmPC, including large B-cell lymphomas (DLBCL, HGBCL, PMBCL, and FL3B), FL and MCL indications. Patients treated with OOS product will also be eligible.; ; Patients who meet the following exclusion criterion will not be eligible for selection from the registries:;  Patients known to be participating in investigational studies at the time of liso-cel infusion.
• 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:
  750

Study design details

• Study design:
  This study is designed as a noninterventional registry-based cohort study that is based on secondary use of data from existing independent registries of patients with lymphoma who were treated with liso-cel therapy in the postmarketing setting, which includes patients treated with OOS product.
• Main study objective:
  The main objective is to the incidence and severity of selected adverse drug reactions (ADRs), as outlined in the Summary of Product Characteristics (SmPC), in patients treated with liso-cel in the postmarketing setting and to monitor for potential clinically important adverse events (AEs) that have not yet been identified as part of the liso-cel safety profile.
• Setting:
  This noninterventional cohort study will be based on secondary use of data that are collected from existing independent registries, such as, but not limited to, the EBMT and the CIBMTR. Both registries use electronic Registry Case Report Forms (CRFs) onto which data may be entered directly by the treating centers.
• Outcomes:
  Secondary malignancies Cytokine release syndrome (CRS) all grades Neurotoxicities all grades Prolonged cytopenias Pregnancy outcome Other AEs considered related to liso-cel treatment (Grade ≥ 3, where applicable), Overall response rate (ORR) Complete response rate (CRR) Duration of response (DoR) Progression-free survival (PFS) Overall survival (OS) Time to next treatment (TTNT)
• Data analysis plan:
  Results will be analyzed and reported descriptively and no formal hypothesis testing is intended. Summary statistics will consist of the number and percentage of patients in each category for discrete variables, whereas for continuous variables the sample size, mean, median, standard deviation, minimum, and maximum will be given. For the primary safety endpoints, incidence proportions and incidence rates will be calculated with the appropriate time periods and methods, analyses will be carried out both with and without accounting for competing risks. For the secondary effectiveness endpoints, Kaplan-Meier estimates and curves will be generated.