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Study of Efficacy and Safety of Tisagenlecleucel in HR B-ALL EOC MRD Positive Patients

Trial Status: Active

This is a single arm, open-label, multi-center, phase II study to determine the efficacy and safety of tisagenlecleucel in de novo HR pediatric and young adult B-ALL patients who received first-line treatment and are EOC MRD positive. The study will have the following sequential phases: screening, pre-treatment, treatment & follow-up, and survival. After tisagenlecleucel infusion, efficacy will be assessed at Day 29, then every 3 months for the first year, every 6 months for the second year, then yearly until the end of the study. Safety will be assessed throughout the study. The study is expected to end in approximately 8 years after first patient first treatment (FPFT). A post-study long term follow-up for lentiviral vector safety will continue under a separate protocol per health authority guidelines.

Inclusion Criteria

  • CD19 expressing B-cell Acute Lymphoblastic Leukemia
  • De novo NCI HR B-ALL who received first-line treatment and are MRD ≥ 0.01% at EOC. EOC bone marrow MRD will be collected prior to screening and will be assessed by multi-parameter flow cytometry using central laboratory analysis.
  • Age 1 to 25 years at the time of screening
  • Lansky (age < 16 years) or Karnofsky (age ≥ 16 years) performance status ≥ 60%
  • Adequate organ function during the screening period: A. Renal function based on age/gender B. ALT ≤ 5 times ULN for age C. AST ≤ 5 times ULN for age D. Total bilirubin < 2 mg/dL (for Gilbert's Syndrome subjects total bilirubin < 4 mg/dL) E. Adequate pulmonary function defined as:
  • no or mild dyspnea (≤ Grade 1)
  • oxygen saturation of > 90% on room air F. Adequate cardiac function defined as LVSF ≥ 28% confirmed by echocardiogram or LVEF ≥ 45% confirmed by echocardiogram or MUGA within 6 weeks of screening
  • Prior induction and consolidation chemotherapy allowed: 1st line subjects: ≤ 3 blocks of standard chemotherapy for first-line B-ALL, defined as 4-drug induction, augmented Berlin-Frankfurt-Münster (BFM) consolidation, and interim maintenance with high-dose methotrexate.

Exclusion Criteria

  • M3 marrow at the completion of 1st line induction therapy
  • M2 or M3 marrow or persistent extramedullary disease at the completion of first-line consolidation therapy. Patients with previous CNS disease are eligible if there is no active CNS involvement of leukemia at the time of enrollment.
  • Philadelphia chromosome positive ALL
  • Hypodiploid: less than 44 chromosomes and/or DNA index < 0.81, or other clear evidence of a hypodiploid clone
  • Prior tyrosine kinase inhibitor therapy
  • Subjects with concomitant genetic syndromes associated with bone marrow failure states: such as subjects with Fanconi anemia, Kostmann syndrome, Shwachman syndrome or any other known bone marrow failure syndrome. Subjects with Down syndrome will not be excluded.
  • Subjects with Burkitt's lymphoma/leukemia (i.e. subjects with mature B-ALL, leukemia with B-cell [sIg positive and kappa or lambda restricted positivity] ALL, with FAB L3 morphology and /or a MYC translocation)
  • Has had treatment with any prior anti-CD19 therapy 9. Treatment with any prior gene or engineered T cell therapy


City of Hope Comprehensive Cancer Center
Palo Alto
Stanford Cancer Institute Palo Alto
Status: ACTIVE
San Francisco
UCSF Medical Center-Mount Zion
Status: ACTIVE
Contact: Helen Diller Family Comprehensive Cancer Center
Phone: 877-827-3222


Children's Healthcare of Atlanta - Egleston


Riley Hospital for Children
Contact: Anne Bubnick
Phone: 317-948-0101

New York

New York
Memorial Sloan Kettering Cancer Center
Status: ACTIVE


Case Comprehensive Cancer Center
Nationwide Children's Hospital


Children's Hospital of Philadelphia
Status: ACTIVE
Contact: Laura Motley
Phone: 215-590-2985

South Carolina

Medical University of South Carolina


University of Wisconsin Hospital and Clinics

Trial Phase Phase II

Trial Type Treatment

Lead Organization
Novartis Pharmaceuticals Corporation

  • Primary ID CCTL019G2201J
  • Secondary IDs NCI-2019-02353, 2017-002116-14
  • ID NCT03876769