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Protocol No. UW18084

Principal Investigator Capitini, Christian

Phase II (Cancer Control)

Age Group Both

Scope National

Sponsor Type Industry

Title A Phase II, Single Arm, Multicenter Open Label Trial to Determine the Safety and Efficacy of Tisagenlecleucel in Pediatric Patients with Relapsed or Refractory Mature B-cell Non-Hodgkin Lymphoma (NHL) (BIANCA), C2202

Objective The purpose of this study is to test an experimental approach called modified T-cell therapy for r/r B-cell NHL. In this study, T cells (a type of white blood cell) will be removed from your child’s blood, modified in order to target the lymphoma, and then returned back to your child. The T cells will be changed in a way that can allow them to identify and possibly kill your child’s NHL tumor cells that display on their surface a protein named CD19, including the tumor’s B cells (another type of white blood cells) and normal antibody producing B cells. The modification of the T cells is done by a process called gene transfer which results in a genetic modification to the T cells taken from your child’s body. The modified T cells are called tisagenlecleucel cells. If you agree to let your child join this study, your child will receive one dose of tisagenlecleucel cells administered by intravenous infusion

Treatment The investigational treatment is a single intravenous (IV) tisagenlecleucel infusion with a proposed dose range of 0.2 to 5 x 106 CAR-positive viable T cells per kg body weight (subjects less than or equal to 50 kg) or 0.1 to 2.5 x 108 CAR-positive viable T cells (subjects greater than 50 kg)

Description Phase II, single arm, multicenter open label trial to determine the safety and efficacy of tisagenlecleucel in pediatric patients with relapsed or refractory mature B-cell non-Hodgkin lymphoma (NHL)

Key Eligibility
  • Histologically confirmed (local evaluation) mature B-cell non-Hodgkin lymphoma (B-cell NHL) including the following subtypes; Burkitt Lymphoma (BL), Diffuse Large B-Cell Lymphoma (DLBCL), Primary Mediastinal B-Cell Lymphoma (PMBCL), Gray Zone lymphoma (GZL), and Follicular Lymphoma (FL). Note: Patients with bone marrow involvement of >25% lymphoma cells by bone marrow biopsy/aspirate evaluation, will be excluded. Patients with B-cell NHL associated with Nijmegen breakage syndrome will be allowed
  • Patients less than 18 years of age and weighing at least 6 kg at the time of screening
  • Patients who have relapsed after one or more prior therapies (can include allogeneic and autologous hematopoietic stem cell transplant) or are primary refractory (have not achieved a CR or PR after the first line of therapy)
  • Measurable disease by radiological criteria in all patients at the time of screening
  • Karnofsky (age greater than or equal to 16 years) or Lansky (age less than 16 years) performance status greater than or equal to 60
  • Adequate bone marrow, renal, hepatic and pulmonary function as defined by the protocol
  • Must have a leukapheresis material of non-mobilized cells accepted for manufacturing
    EXCLUSION
  • Prior gene therapy or engineered T cell therapy or prior treatment with any anti-CD19 therapy
  • Allogeneic hematopoietic stem cell transplant (HSCT) less than 3 months prior to screening and less than or equal to 4 months prior to infusion
  • Presence of grade 2 to 4 acute or extensive chronic graft-versus-host disease (GVHD) in patients who received prior allogeneic HSCT
  • Active, uncontrolled infection despite treatment at screening
  • Presence of active or prior hepatitis B or C as indicated by serology
  • Human Immunodeficiency Virus (HIV) positive test
  • Active neurological autoimmune or inflammatory disorders (eg: Guillain-Barre syndrome, Amyotrophic Lateral Sclerosis)
  • Active CNS involvement by malignancy. Note: Patients with history of CNS disease that have been effectively treated will be eligible
  • Patients with B-cell NHL in the context of post-transplant lymphoproliferative disorders (PTLD) associated lymphomas
  • Patients with concomitant genetic syndromes associated with bone marrow failure status such as patients with Fanconi anemia, Kostmann syndrome, Shwachman syndrome or any other known bone marrow failure syndrome. Note: Patients with Down syndrome will not be excluded
  • Intolerance to the excipients of the tisagenlecleucel cell product
  • Cardiac or cardiac repolarization abnormality as defined by the protocol
  • Can not be pregnant or breastfeeding
  • Male and Female of childbearing potential must agree to adequate birth control as defined by the protocol

  • Applicable Disease Sites Hematologic cancers, other; Lymphoma

    Status Open

    Participating Institutions UW Hospital and Clinics