Registry study to assess the long-term safety of patients with B lymphocyte malignancies treated with tisagenlecleucel

Study type

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

  Safety study (incl. comparative)

Non-interventional study

• Non-interventional study design:
  Other
• Non-interventional study design, other:
  Non-interventional post-authorization safety study (PASS)

Study drug and medical condition

• Name of medicine:
  KYMRIAH
• Study drug International non-proprietary name (INN) or common name:
  TISAGENLECLEUCEL
• Anatomical Therapeutic Chemical (ATC) code:
  (L01XL04) tisagenlecleucel

  tisagenlecleucel
• Medical condition to be studied:
  Diffuse large B-cell lymphoma

  Acute lymphocytic leukaemia

  Follicular lymphoma

Population studied

• Short description of the study population:
  In cohort 1: a 5-year enrollment period is planned to enroll approximately 2,500 patients with either r/r pediatric/young adult B-cell ALL (at least 1,000 patients) or with r/r large B-cell lymphoma (at least 1,500 patients). ; In cohort 2: a 3-year enrollment is planned to enroll approximately 300 patients with r/r follicular lymphoma.
• Age groups:
  • Paediatric Population (< 18 years)

    • Infants and toddlers (28 days – 23 months)
    • Children (2 to < 12 years)
    • Adolescents (12 to < 18 years)
  • Adult and elderly population (≥18 years)

    • 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)
• Special population of interest:
  Hepatic impaired

  Immunocompromised

  Pregnant women

  Renal impaired
• Estimated number of subjects:
  2800

Study design details

• Main study objective:
  The primary objective is to evaluate the long-term safety and the risk of secondary malignancies in patients with B lymphocyte malignancies treated with tisagenlecleucel in a real-world setting as measured by type and frequency of AEs.
• Outcomes:
  The type and frequency of AEs (including secondary malignancies). ; The identification of patients with secondary malignancies for detection of CAR transgene and/ or CAR surface expression and presence of replication-competent lentivirus. ; 1) Evaluate the long-term effectiveness of tisagenlecleucel by approved indication ; 2) Evaluate any pregnancy occurring in women of child-bearing potential or female partners of males after infusion with tisagenlecleucel
• Data analysis plan:
  Safety data will be summarized and listed by approved indication in interim reports up until the end of the study. ; The final Clinical Study Report will be prepared including all planned effectiveness and safety analyses at the end of the study.