Non Hodgkin Lymphoma Clinical Trial
Phase II Open Label Trial to Determine Safety & Efficacy of Tisagenlecleucel in Pediatric Non-Hodgkin Lymphoma Patients
The purpose of the study is to assess the efficacy and safety of tisagenlecleucel in children and adolescents with relapsed/refractory B-cell non-Hodgkin lymphoma (r/r B-NHL). For pediatric patients who have r/r B-NHL, survival rates are dismal, only ~20-50% subjects are alive at 2 years with overall response rate (ORR) of 20-30% after conventional salvage chemotherapy.
This study is part of an agreed Pediatric Investigation Plan (PIP). The single-arm study design includes r/r B-cell NHL subject population with poor prognosis, lack of approved effective therapies in this setting. Subject population will include aggressive subtypes of B-cell NHL and will be allowed to receive "bridging therapy" of investigator's choice After assessment of eligibility, subjects qualifying for the study will be enrolled and are allowed to start lymphodepleting chemotherapy as recommended in protocol after which a single dose of tisagenlecleucel product will be infused. The efficacy of tisagenlecleucel will be evaluated through the primary endpoint of Overall Response Rate (ORR) which includes complete response (CR) and partial response (PR) as determined by local assessment. Safety assessments will be conducted through the study completion.
Histologically confirmed pediatric 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. Patients with Burkitt leukemia who don't meet radiological criteria must have bone marrow involvement of >25% by local assessment of bone marrow aspirate and/or biopsy.
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:
Absolute neutrophil count (ANC) >1000/mm3
Hemoglobin ≥8.0 g/dl
Adequate organ function defined as:
a serum creatinine (sCR) based on gender/age as follows: Maximum Serum Creatinine (mg/dL) Age Male Female
1 to <2 years 0.6 0.6 2 to <6 years 0.8 0.8 6 to <10 years 1.0 1.0 10 to <13 years 1.2 1.2 13 to <16 years 1.5 1.4
≥16 years 1.7 1.4
Aspartate aminotransferase (AST) and alanine aminotransferase (ALT) ≤5 times the upper limit of normal (ULN) for age
Total bilirubin <2 mg/dL (for Gilbert's Syndrome patients total bilirubin <4 mg/dL)
Adequate pulmonary function
i. Oxygen saturation of >91% on room air ii. No or mild dyspnea (≤Grade 1)
Must have a leukapheresis material of non-mobilized cells accepted for manufacturing.
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 (e.g., blood cultures, PCR for DNA/RNA, etc.)
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.
Other protocol-defined inclusion/exclusion criteria may apply.
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There are 26 Locations for this study
Los Angeles California, 90027, United States
San Francisco California, 94143, United States
Baltimore Maryland, 21231, United States
Boston Massachusetts, 02215, United States
New York New York, 10065, United States
Cincinnati Ohio, 45229, United States
Philadelphia Pennsylvania, 19104, United States
Dallas Texas, 75235, United States
Randwick New South Wales, 2031, Australia
Parkville Victoria, 3052, Australia
Wien , A 109, Austria
Toronto Ontario, M5G 1, Canada
Copenhagen , 2100, Denmark
Helsinki , 00029, Finland
Paris Cedex , 75019, France
Villejuif Cedex , 94800, France
Muenster , 48149, Germany
Monza MB, 20900, Italy
Roma RM, 00165, Italy
Sakyo Ku Kyoto, 606 8, Japan
Setagaya-ku Tokyo, 157-8, Japan
Utrecht CS, 3584, Netherlands
Oslo , 0424, Norway
Barcelona Catalunya, 08035, Spain
Madrid , 28046, Spain
London , WC1N , United Kingdom
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