Treatment of CD19 Chimeric Antigen Receptor T Cells for Pediatric Patients With CD19-positive B-cell Acute Lymphoblastic Leukemia Who Are Indicated for Hematopoietic Stem Cell Transplantation

This is a phase 2 clinical trial targeting pediatric and adolescent patients diagnosed with CD19-positive B-ALL, considered very high-risk group. The study aims to administer CD19 CAR-T therapy as an alternative to hematopoietic stem cell transplantation in patients eligible for such transplantation. The trial includes patients aged 25 or younger.

Study Overview

• Status: Recruiting
• Conditions: Very High Risk Acute Lymphoblastic Leukemia; CD19 CAR-T Therapy
• Intervention / Treatment: Genetic: SNUH-CD19-CAR-T

• Study Type: Interventional
• Enrollment (Estimated): 50
• Phase: Phase 2

Contacts and Locations

• Study Contact: Name: Hyoung Jin Kang, Professor; Phone Number: 02-2072-3452; Email: kanghj@snu.ac.kr

Study Locations

• Korea, Republic of
  • Seoul, Korea, Republic of
    • Recruiting
    • Seoul National University Children's Hospital
    • Contact: Hyoung Jin Kang

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Child; Adult
• Accepts Healthy Volunteers: No

Description

Inclusion Criteria:

• Subject eligibility criteria include individuals who are under the age of 25 at the time of SNUH-CD19-CAR-T administration. Confirmation of B-cell acute lymphoblastic leukemia diagnosed at the initial assessment by a treating physician for the first diagnosis is required. Immunophenotypic analysis of CD19 expression on leukemia cells must be confirmed through immune profiling at the time of the initial diagnosis of B-cell acute lymphoblastic leukemia. In patients experiencing hematologic relapse of B-cell acute lymphoblastic leukemia, CD19 expression should also be confirmed at the time of relapse confirmation.

Patients must achieve hematologic complete remission defined as less than 5% blasts in the bone marrow after the first-line or second-line chemotherapy (first salvage therapy). Eligible patients for hematopoietic stem cell transplantation, defined by indications for transplantation, include those meeting at least one of the following criteria:

1. Philadelphia chromosome-positive due to t(9;22)(q34;q11) translocation.
2. Hypodiploidy defined by fewer than 44 chromosomes.
3. E2A-HLF gene fusion due to t(17;19) translocation.
4. Detection of minimal residual disease (MRD) positive at 0.01% or higher by next-generation sequencing confirmed after consolidation therapy and maintenance therapy.
5. Failure of the first-line therapy.
6. Cases not meeting any of the above criteria but deemed eligible for hematopoietic stem cell transplantation based on the investigator's judgment.

Exclusion Criteria:

• Patients who have undergone hematopoietic stem cell transplantation.
• Individuals for whom an adequate or sufficient leukapheresis product suitable for the production of SNUH-CD19-CAR-T cannot be obtained or is unavailable.
• Those known to be infected with the human immunodeficiency virus (HIV).
• Presence of uncontrolled active infections, determined by the investigator's assessment. If appropriate treatment has been administered for the infection, and there are no signs of progression at the time of enrollment, it is considered controlled. Persistent fever without other symptoms is not interpreted as progressive infection.
• Women who are pregnant or breastfeeding.
• Individuals deemed clinically inappropriate for participation in the clinical trial based on the investigator's clinical judgment.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Time Frame
Event free survival	above 3 year

Secondary Outcome Measures

Outcome Measure	Time Frame
Overall survival	above 3 year

Collaborators and Investigators

• Sponsor: Seoul National University Hospital

Study record dates

Study Major Dates

• Study Start (Actual): January 19, 2024
• Primary Completion (Estimated): December 31, 2028
• Study Completion (Estimated): December 31, 2029

Study Registration Dates

• First Submitted: January 17, 2024
• First Submitted That Met QC Criteria: February 6, 2024
• First Posted (Actual): February 8, 2024

Study Record Updates

• Last Update Posted (Actual): February 8, 2024
• Last Update Submitted That Met QC Criteria: February 6, 2024
• Last Verified: January 1, 2024

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Immune System Diseases; Neoplasms by Histologic Type; Neoplasms; Lymphoproliferative Disorders; Lymphatic Diseases; Immunoproliferative Disorders; Leukemia; Precursor Cell Lymphoblastic Leukemia-Lymphoma; Leukemia, Lymphoid
• Other Study ID Numbers: SNUH-CART-CD19-SCT

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No
• product manufactured in and exported from the U.S.: No