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

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

ClinicalTrials.gov Identifier: NCT03610724

Novartis Reference Number: CCTL019C2202

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All compounds are either investigational or being studied for (a) new use(s). Efficacy and safety have not been established. There is no guarantee that they will become commercially available for the use(s) under investigation. 

Study Description

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.

Condition 
Non-Hodgkin Lymphoma
Phase 
Phase 2
Overall status 
Recruiting
Enrollment count 
35 participants
Start date 
Feb 15, 2019
Completion date 
Feb 15, 2023
Gender 
All
Age(s)
25 Years and older (Child, Adult)

Interventions

Biological
Tisagenlecleucel
Single intravenous infusion

Eligibility Criteria

Inclusion Criteria:

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.

Exclusion Criteria:

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.

Study Locations

United States
Childrens Hospital Los Angeles
Recruiting
Los Angeles, 90027
California
United States
UCSF Medical Center
Recruiting
San Francisco, 94143
California
United States
Children s National Health System
Recruiting
Washington, 20010
District of Columbia
United States
Childrens Healthcare of Atlanta At Scottish Rite
Recruiting
Atlanta, 30342
Georgia
United States
Johns Hopkins Oncology Center ORA
Recruiting
Baltimore, 21231
Maryland
United States
Dana Farber Cancer Institute Dept.of DFCI
Recruiting
Boston, 02215
Massachusetts
United States
University of Michigan
Recruiting
Ann Arbor, 48109-2800
Michigan
United States
Memorial Sloan Kettering Cancer Center MSKCC (8)
Recruiting
New York, 10065
New York
United States
Cincinnati Children s Hospital Medical Center
Recruiting
Cincinnati, 45229-3039
Ohio
United States
Oregon Health and Science University
Recruiting
Portland, 97239
Oregon
United States
The Childrens Hospital of Philadelphia Drug Shipment
Recruiting
Philadelphia, 19104
Pennsylvania
United States
Monroe Carell Jr Childrens Hospital at Vanderbilt
Recruiting
Nashville, 37232
Tennessee
United States
University of Texas Southwestern Medical Center
Recruiting
Dallas, 75235
Texas
United States
University of Wisconsin Hospital and Clinics
Recruiting
Madison, 53792
Wisconsin
United States
Australia
Novartis Investigative Site
Recruiting
Randwick, 2031
New South Wales
Australia
Novartis Investigative Site
Recruiting
Parkville, 3052
Victoria
Australia
Austria
Novartis Investigative Site
Recruiting
Wien, A 1090
-
Austria
Canada
Novartis Investigative Site
Recruiting
Toronto, M5G 1X8
Ontario
Canada
Denmark
Novartis Investigative Site
Recruiting
Copenhagen, 2100
-
Denmark
Finland
Novartis Investigative Site
Recruiting
Helsinki, 00029
-
Finland
France
Novartis Investigative Site
Recruiting
Nantes Cedex 01, 44093
-
France
Novartis Investigative Site
Recruiting
Paris Cedex, 75019
-
France
Novartis Investigative Site
Recruiting
Villejuif Cedex, 94800
-
France
Italy
Novartis Investigative Site
Recruiting
Roma, 00165
ITA
Italy
Novartis Investigative Site
Recruiting
Monza, 20900
MB
Italy
Novartis Investigative Site
Recruiting
Torino, 10126
TO
Italy
Japan
Kyoto University Hospital
Recruiting
Sakyo Ku, 606 8507
Kyoto
Japan
Netherlands
Prinses Maxima Centrum voor Kinderoncologie
Recruiting
Utrecht, 3584
CS
Netherlands
Norway
Novartis Investigative Site
Recruiting
Oslo, 0424
-
Norway
Spain
Novartis Investigative Site
Recruiting
Barcelona, 08035
Barcelona/ Cataluny/Espanya
Spain
Novartis Investigative Site
Recruiting
Madrid, 28046
-
Spain
United Kingdom
Novartis Investigative Site
Recruiting
London, WC1N 1EH
-
United Kingdom

Contacts

Name: 
Novartis Pharmaceuticals
Phone: 
1-888-669-6682
Name: 
Novartis Pharmaceuticals
Phone: 
+41613241111
Email: 

Have a question?

Call 1-999-669-6682 or email [email protected]