A Phase II, single arm, multicenter open label trial to determine the safety and efficacy of tisagenlecleucel in children and adolescents with non-Hodgkin lymphoma (NHL) (BIANCA)
- Conditions
- B-cell non-Hodgkin lymphoma (B-cell NHL) including the following subtypesBurkitt Lymphoma and Burkitt Leukemia (L3 B ALL) (BL), Diffuse Large B-Cell Lymphoma (DLBCL), Primary Mediastinal B-Cell Lymphoma (PMBCL), Gray Zone lymphoma (GZL), and Follicular Lymphoma (FL).
- Registration Number
- JPRN-jRCT1080224705
- Lead Sponsor
- ovartis Pharma. K.K.
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- completed
- Sex
- All
- Target Recruitment
- 2
Patients eligible for inclusion in this study must meet all of the following criteria:
-Histologically confirmed (local evaluation) mature B-cell non-Hodgkin lymphoma (B-cell NHL) including the following subtypes; Burkitt lymphoma/Burkitt leukemia (BL), diffuse large B-cell lymphoma (DLBCL), primary mediastinal B-cell lymphoma (PMBCL), gray zone lymphoma (GZL), and follicular lymphoma (FL) Note: Patients with B-cell NHL associated with Nijmegen breakage syndrome will be allowed.
-Patients =<25 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 >=16 years) or Lansky (age <16 years) performance status >=60.
-Adequate bone marrow reserve without transfusions (transfusion >2 weeks prior to laboratory assessment is allowed) defined as:
a. Absolute neutrophil count (ANC) >1000/mm3
b. Platelets >=50000/mm3
c. Hemoglobin >=8.0 g/dl
-Adequate organ function:
a. a serum creatinine (sCR) based on gender/age as follows:
Maximum Serum Creatinine (mg/dL)
Age: 1 to <2 years; Male: 0.6, Female: 0.6
Age: 2 to <6 years; Male: 0.8, Female: 0.8
Age: 6 to <10 years; Male:1.0, Female:1.0
Age:10 to <13 years; Male: 1.2, Female:1.2
Age:13 to <16 years; Male: 1.5, Female:1.4
Age: >=16 years; Male: 1.7, Female: 1.4
b. Aspartate aminotransferase (AST) and alanine aminotransferase (ALT) =<5 times the upper limit of normal (ULN) for age
c. Total bilirubin <2 mg/dL (for Gilbert Syndrome patients total bilirubin <4 mg/dL)
d. Adequate pulmonary function
i. Oxygen saturation of >91% on room air
ii. No or mild dyspnea (=9. Must have a leukapheresis material of non-mobilized cells accepted for manufacturing.
Patients meeting any of the following criteria are not eligible for inclusion in this study.
-Prior gene therapy or engineered T cell therapy
-Prior treatment with any anti-CD19 therapy
-Allogeneic hematopoietic stem cell transplant (HSCT) <3 months prior to screening and =<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.
-Prior diagnosis of malignancy other than study indication, and not disease free for 5 years
-Clinically significant active infection confirmed by clinical evidence, imaging, or positive laboratory tests
-Presence of active hepatitis B or C as indicated by serology.
-Human Immunodeficiency Virus (HIV) positive test.
-Active neurological autoimmune or inflammatory disorders not related to B cell NHL (eg: Guillain-Barre syndrome, Amyotrophic Lateral Sclerosis)
-Active central nervous system (CNS) involvement by malignancy.
-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.
-Cardiac disorder defined as:
a. Cardiac or cardiac repolarization abnormality, including any of the following:
i. History of myocardial infarction (MI), angina pectoris, or coronary artery bypass graft (CABG) within 6 months prior to starting study treatment
ii. Clinically significant cardiac arrhythmias (eg: ventricular tachycardia), complete left bundle branch block, high-grade atrioventricular (AV) block (eg: bifascicular block, Mobitz type II and third degree AV block)
b. Left ventricular ejection fraction (LVEF) <45% as determined by echocardiography (ECHO) or magnetic resonance angiogram (MRA) or multigated acquisition (MUGA)
c. New York Heart Association (NYHA) functional class III or IV (Chavey et al 2001)
Study & Design
- Study Type
- Interventional
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method
- Secondary Outcome Measures
Name Time Method