Molecular profiling of tissue samples from patients who received a Kite-manufactured gene-modified cell therapy and have developed a secondary malignancy of T-cell origin

Study type

• Study topic:
  Disease /health condition

  Human medicinal product
• Study type:
  Non-interventional study
• Scope of the study:
  Feasibility analysis

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

Study drug and medical condition

• Medicinal product name:
  YESCARTA

  TECARTUS
• Study drug International non-proprietary name (INN) or common name:
  AXICABTAGENE CILOLEUCEL

  BREXUCABTAGENE AUTOLEUCEL
• Anatomical Therapeutic Chemical (ATC) code:
  (L01XL03) axicabtagene ciloleucel

  axicabtagene ciloleucel

  (L01XL06) brexucabtagene autoleucel

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

  Mantle cell lymphoma

  Follicular lymphoma

  Acute lymphocytic leukaemia

  Primary mediastinal large B-cell lymphoma

Population studied

• Short description of the study population:
  Patients who have received a Kite-manufactured CAR T-cell therapy (axicabtagene ciloleucel or brexucabtagene autoleucel) and have reported a suspected secondary malignancy of T-cell origin.
• Age groups:
  • 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)

Study design details

• Study design:
  Observational study
• Main study objective:
  To assess potential CAR transgene involvement by performing molecular profiling of tissue samples obtained or are about to be obtained in the course of routine clinical practice from patients who were treated with axicabtagene ciloleucel or brexucabtagene autoleucel and developed a secondary T-cell malignancy.; The molecular profiling will include the following, as applicable:; • The presence of the CAR transgene in the blood and/or tumor and/or bone marrow biopsy, as ; well as the CAR level, if the CAR transgene was detected.; • Presence of RCR.
• Setting:
  This observational study will be conducted in the EEA and UK and will enroll patients on an ongoing basis.
• Outcomes:
  The following laboratory parameters will be used for molecular profiling:; • The presence of the CAR transgene in the blood and/or tumor and/or bone marrow biopsy.; • The CAR level, if the CAR transgene was detected in the blood and/or tumor and/or bone marrow biopsy.; • Presence of RCR.; • Presence of somatic mutations that are common in hematologic malignancies.; • Transgene integration site analysis results.; • Transcriptome/RNA analysis results
• Data analysis plan:
  No statistical analysis is planned. The molecular profiling results will be described for each patient tested.