Phase II Open Label Trial to Determine Safety & Efficacy of Tisagenlecleucel in Pediatric Non-Hodgkin Lymphoma Patients

Completed · Phase 2

Conditions studied: Non-Hodgkin Lymphoma

In brief

The purpose of the study was to assess the efficacy and safety of tisagenlecleucel in pediatric, adolescent and young adult patients with relapsed/refractory B-cell non-Hodgkin lymphoma (r/r B-NHL) including Burkitt Lymphoma and Burkitt Leukemia. For pediatric patients who have r/r B-NHL including Burkitt Lymphoma and Burkitt Leukemia, 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.

Key facts

Study ID

NCT03610724

Run by

Novartis Pharmaceuticals

People needed

34

Starts

2019-02-15

Expected to finish

2023-04-26

Last updated by the study team

2024-06-20

Who can join

Age: any, up to 25. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• 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
• Platelets ≥50000//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.

You may not qualify if…

• 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.

Where it is running

• Childrens Hospital Los Angeles — Los Angeles, California, United States
• UCSF Medical Center — San Francisco, California, United States
• Johns Hopkins Oncology Center ORA — Baltimore, Maryland, United States
• Dana Farber Cancer Institute Dept.of DFCI — Boston, Massachusetts, United States
• Memorial Sloan Kettering Cancer Center MSKCC (8) — New York, New York, United States
• Cinn Children Hosp Medical Center — Cincinnati, Ohio, United States
• The Childrens Hospital of Philadelphia Drug Shipment — Philadelphia, Pennsylvania, United States
• University of Texas Southwestern Medical Center . — Dallas, Texas, United States
• Novartis Investigative Site — Randwick, New South Wales, Australia
• Novartis Investigative Site — Parkville, Victoria, Australia
• Novartis Investigative Site — Vienna, Austria
• Novartis Investigative Site — Toronto, Ontario, Canada
• Novartis Investigative Site — Copenhagen, Denmark
• Novartis Investigative Site — Helsinki, Finland
• Novartis Investigative Site — Paris, France
• Novartis Investigative Site — Villejuif, France
• Novartis Investigative Site — Münster, Germany
• Novartis Investigative Site — Monza, MB, Italy
• Novartis Investigative Site — Roma, RM, Italy
• Kyoto University Hospital — Sakyō-ku, Kyoto, Japan
• Novartis Investigative Site — Setagaya-ku, Tokyo, Japan
• Prinses Maxima Centrum voor Kinderoncologie — Utrecht, CS, Netherlands
• Novartis Investigative Site — Oslo, Norway
• Novartis Investigative Site — Barcelona, Catalonia, Spain
• Novartis Investigative Site — Madrid, Spain

Full record on ClinicalTrials.gov

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