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Study of huCART19 for Very High-Risk (VHR) Subsets of Pediatric B-ALL

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details. Identifier: NCT03792633
Recruitment Status : Recruiting
First Posted : January 3, 2019
Last Update Posted : June 24, 2021
Children's Hospital of Philadelphia
Information provided by (Responsible Party):
University of Pennsylvania

Brief Summary:
This is a phase 2 study to evaluate humanized CD19 redirected autologous T cells (or huCART19 cells) with CD19 expressing relapsed and refractory B-cell acute lymphoblastic leukemia. This study is targeting pediatric and young adult patients aged 1-29 years with CD19+ B cell malignancies in newly diagnosed B-ALL patients predicted to have an exceedingly poor outcome with conventional chemotherapy, in high-risk first relapse, or and in second or greater relapse in this phase 2 trial. In addition, a second cohort will test the efficacy of huCART19 in patients with poor response to prior B cell directed engineered cell therapy.

Condition or disease Intervention/treatment Phase
Acute Lymphoid Leukemia Biological: huCART19 Phase 2

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 63 participants
Allocation: Non-Randomized
Intervention Model: Parallel Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: Phase 2 Study of Humanized CD19-directed Chimeric Antigen Receptor-modified T Cells (huCART19) for Very High-Risk Subsets of B Cell Acute Lymphoblastic Leukemia (B-ALL)
Actual Study Start Date : January 18, 2019
Estimated Primary Completion Date : August 2021
Estimated Study Completion Date : August 2022

Arm Intervention/treatment
Experimental: Newly Diagnosed VHR B-ALL or High-Risk Relapse of B Biological: huCART19
huCART19 infusion
Other Name: huCTL019

Experimental: Poor Response to Prior B Cell Directed Engineered cell therapy Biological: huCART19
huCART19 infusion
Other Name: huCTL019

Primary Outcome Measures :
  1. 1-year Event-Free Survival in patients with newly diagnosed VHR B-ALL or high-risk relapse of B-ALL [ Time Frame: 1 year ]
  2. 1-year Event-Free Survival in patients with poor response to prior B cell directed engineered cell therapy [ Time Frame: 1 year ]

Information from the National Library of Medicine

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Ages Eligible for Study:   1 Year to 29 Years   (Child, Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

Inclusion Criteria:

Relapsed or refractory B-cell ALL:

  • Cohort A: Patients with newly diagnosed VHR B-ALL or high-risk relapse of B-ALL who meet one of the following criteria:

    • Newly diagnosed NCI HR B-ALL with induction failure: M3 marrow (>25% blasts) at end of induction OR
    • First marrow relapse of B-ALL at < 36 months from diagnosis OR
    • 2nd or greater relapse OR
    • Any relapse after allogeneic hematopoietic stem cell transplantation (HSCT) and ≥ 4 months from stem cell transplant (SCT) at enrollment OR
    • Refractory disease defined as having not achieved a minimal residual disease (MRD)_-negative and/or cerebral spinal fluid (CSF)-negative complete response (CR) after ≥ 2 chemotherapy regimens/cycles of frontline therapy or 1 cycle of reinduction therapy for patients in first relapse OR
    • Ineligible for allogeneic stem cell transplant
  • Cohort B: Patients previously treated with B cell directed engineered cell therapy who meet one of the following criteria:

    • partial response or no response to prior cell therapy
    • CD19+ relapse after prior cell therapy
    • demonstrated early (≤6 months from infusion) B cell recovery suggesting loss of engineered cells
  • Patients with prior or current history of CNS3 disease will be eligible if central nervous system (CNS) disease is responsive to therapy
  • Documentation of CD19 tumor expression in bone marrow, peripheral blood, CSF, or tumor tissue by flow cytometry at relapse
  • Adequate organ function
  • Age 1-29 years
  • Adequate performance status

Exclusion Criteria:

  • Active hepatitis B or active hepatitis C.
  • HIV Infection.
  • Active acute or chronic graft-versus-host disease (GVHD) requiring systemic therapy.
  • Concurrent use of systemic steroids at the time of cell infusion or cell collection, or a condition, in the treating physician's opinion, that is likely to require steroid therapy during collection or after infusion. Steroids for disease treatment at times other than cell collection or at the time of infusion are permitted. Use of physiologic replacement hydrocortisone or inhaled steroids is permitted as well.
  • CNS3 disease that is progressive on therapy, or with CNS parenchymal lesions that might increase the risk of CNS toxicity.
  • Pregnant or nursing (lactating) women.
  • Uncontrolled active infection.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): NCT03792633

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Contact: Mia Benson-Smith 267-426-0762
Contact: Claire White

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United States, Pennsylvania
Children's Hospital of Pennsylvania Recruiting
Philadelphia, Pennsylvania, United States, 19104
Contact: Shannon         
Sponsors and Collaborators
University of Pennsylvania
Children's Hospital of Philadelphia
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Principal Investigator: Shannon Maude, MD, PhD Children's Hospital of Philadelphia
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Responsible Party: University of Pennsylvania Identifier: NCT03792633    
Other Study ID Numbers: 831916
18CT014 ( Other Identifier: CHOP )
First Posted: January 3, 2019    Key Record Dates
Last Update Posted: June 24, 2021
Last Verified: June 2021

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Additional relevant MeSH terms:
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Leukemia, Lymphoid
Precursor Cell Lymphoblastic Leukemia-Lymphoma
Neoplasms by Histologic Type
Lymphoproliferative Disorders
Lymphatic Diseases
Immunoproliferative Disorders
Immune System Diseases