SJCAR19: CAR T-Cell Therapy for Children and Young Adults with Acute Lymphoblastic Leukemia

A Phase I/II Study Evaluating CD19-Specific CAR Engineered Autologous T-Cells in Pediatric and Young Adult Patients with Relapsed or Refractory CD19+ Acute Lymphoblastic Leukemia

Categories:

Leukemia / Lymphoma

Phase I/II

Diseases Treated:

Acute lymphoblastic leukemia

Eligibility Overview:

 

 

  1. Brief Summary

    This is a new Phase I/II immunotherapy clinical trial designed to evaluate the use of CD19-specific chimeric antigen receptor (CAR) engineered autologous T cells in children and young adults with relapsed or refractory CD19+ acute lymphoblastic leukemia (ALL).

    Although cure rates for pediatric ALL have steadily improved and are now nearing 90%, patients with relapsed or refractory disease do much poorer. Treatment options are typically limited for these patients, and therapy relies primarily on hematopoietic cell transplantation. Significant improvement in cure rates requires the development of treatments that bypass cellular mechanisms of drug resistance and have high therapeutic indexes.

    St. Jude has developed a second-generation lentiviral CD19-specific CAR construct containing a 4-1BB.ζ domain. The vector for the study is produced at the St. Jude Good Manufacturing Practices (GMP) facility using a stable producer cell line. When autologous T cells are engineered with this CAR vector, the final cellular product is referred to as SJCAR19.

    While pre-clinical, translational evaluation of this CAR vector has proved promising, this trial is the first clinical study evaluating the safety and efficacy of this CAR construct for the treatment of this disease.

    The protocol includes three parts: autologous apheresis, SJCAR19 manufacturing, and treatment. Most patients will receive a lymphodepleting chemotherapy regimen, followed by a single infusion of the CAR T cells.  

    Primary Objectives

    • To determine the maximum tolerated dose (MTD) and characterize the safety profile and dose-limiting toxicities (DLTs) of treatment with SJCAR19 in these patients (Phase I)
    • To evaluate the complete response rates of SJCAR19 in these patients (Phase II)

    Eligibility Criteria

    Inclusion criteria include:

        Autologous apheresis:

    • Children and young adults up to 21 years old
    • Refractory or relapsed CD19+ ALL
    • Estimated life expectancy of at least 12 weeks

        Manufacturing SJCAR19:

    • Children and young adults up to 21 years old
    • Refractory or relapsed CD19+ ALL
    • Meets criteria to undergo autologous apheresis or has previously undergone autologous apheresis
    • Estimated life expectancy of at least 12 weeks

        Treatment with SJCAR19:

    • Children and young adults up to 21 years old
    • Refractory or relapsed CD19+ ALL
    • Estimated life expectancy of at least 8 weeks
    • At least 3 months since allogeneic hematopoietic transplant, if any

    Exclusion Criteria include:

    • Known primary immunodeficiency
    • History of hypersensitivity reactions to murine protein-containing products

    Study Sites

    St. Jude Children’s Research Hospital, Memphis, Tennessee

  2. About this study

    This clinical trial will test a new treatment for acute lymphoblastic leukemia (ALL) that has relapsed (come back after treatment) or refractory (did not respond to treatment). This new treatment is called SJCAR19. SJCAR19 is a type of chimeric antigen receptor (CAR) T-cell therapy, also known as CAR T-cell therapy or CAR-T therapy. CAR-T therapy takes T cells, a type of white blood cell, and improves their ability to recognize and kill cancer cells.

    In this study, doctors will collect T cells from your body and send them to a manufacturing facility on the St. Jude campus. There, researchers will turn the T cells into CAR-T cells to help them better recognize and kill cancer cells. Finally, doctors will infuse these modified T cells, called SJCAR19, back into your blood.   In some cases, the doctors may give you chemotherapy before receiving the SJCAR19 cells.

    Purpose of this clinical trial

    The main purpose of this study is to find out the largest and safest dose of SJCAR19 you can take. Doctors also want to learn about the good and bad effects of SJCAR19, and how well it treats your cancer.

    Eligibility overview

    • Children and young adults up to 21 years old
    • Refractory or relapsed CD19+ ALL
  3. SJCAR19 Quick View
    Sponsors St. Jude Children’s Research Hospital
    ClinicalTrials.gov identifier NCT03573700
    Trial Start Date June 2018
    Estimated Enrollment 35
    Study Type Interventional
    Study Phase Phase I/II
    Conditions Acute lymphoblastic leukemia
    Ages Children and young adults up to 21 years old
    Principal investigators Aimee Talleur, MD
    Stephen Gottschalk, MD
    Study Sites St. Jude Children’s Research Hospital
    For a consultation or to discuss SJCAR19 St. Jude Physician/Patient Referral Office
    1-888-226-4343
    referralinfo@stjude.org

St. Jude Children’s Research Hospital
262 Danny Thomas Place
Memphis, TN 38105  USA
Voice: 1-888-226-4343 or 901-595-4055
24-Hour Emergency Access Pager: 1-800-349-4334
Email: referralinfo@stjude.org

The above information is intended to provide only a basic description about a research protocol that may be currently active at St. Jude. The details made available here may not be the most up-to-date information on protocols used by St. Jude. To receive full details about a protocol and its status and or use at St. Jude, a physician must contact St. Jude directly.