CD-19 CAR-T Cell for Pediatric ALL or Lymphoma

Safety and Feasibility Study of CD19 Chimeric Antigen Receptor (CAR) T Cells in Children with Relapsed or Refractory CD19 Positive Acute Lymphoblastic Leukemia or Lymphoma

This study seeks to examine the efficacy and safety of the administration of autologous T cells that have been modified through the introduction of a chimeric antigen receptor (CAR) targeting the B cell surface antigen CD19 following administration of chemotherapy lymphodepletion regimen in children with relapsed or refractory acute lymphoblastic leukemia (ALL) or lymphoma. The overall goal of this study is to validate the safety profile of administration CD19-CAR T cells and describe the response rate in children with relapsed/refractory ALL or lymphoma.

Study Overview

• Status: Recruiting
• Conditions: Lymphoma, B-Cell; Acute Lymphoblastic Leukemia, Pediatric
• Intervention / Treatment: Biological: CAR-T

• Study Type: Interventional
• Enrollment (Estimated): 18
• Phase: Not Applicable

Contacts and Locations

• Study Contact: Name: Daniel Cheuk, MBBS; Phone Number: 852-35136049; Email: cheukkld@ha.org.hk

Study Locations

• Hong Kong
  • Hong Kong, Hong Kong
    • Recruiting
    • Hong Kong Children's Hospital
    • Contact: Daniel Cheuk, MBBS; Phone Number: 852-35136049; Email: cheukkld@ha.org.hk

Participation Criteria

Eligibility Criteria

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

Description

Inclusion Criteria:

• Subjects must have relapsed or refractory ALL or lymphoma treated with at least two lines of therapy. Disease must have either progressed after the last regimen or presented failure to achieve partial or complete remission with the last regimen.
• The patient's disease must be CD19 positive, either by immunohistochemistry or flow cytometry analysis on the last biopsy available.
• Age 1-17 years.
• Performance status: Subjects > 10 years of age: Karnofsky ≥ 50%; Subjects ≤ 10 years of age: Lansky scale ≥ 50%.

• Normal organ function.

  • Total bilirubin ≤ 3 times upper limit of normal
  • AST (SGOT) ≤ 5 times upper limit of normal
  • ALT (SGPT) ≤ 5 times upper limit of normal
  • Serum Creatinine ≤ 2 times upper limit of normal
  • Subjects must have the following hematologic function parameters: Hemoglobin (Hb) level > 8 g/dL; Absolute Lymphocyte Count > 0.1x10^9/L; Platelet > 50x10^9/L
• Prior therapy wash-out. At least 2 weeks or 5 half lives, whichever is shorter, must have elapsed since any prior systemic therapy at the time the subject is planned for leukapheresis.
• Subjects' parent or legal guardian must have the ability to understand and the willingness to sign a written informed consent document.

Exclusion Criteria:

• Autologous transplant within 6 weeks of planned CAR T cell infusion.
• Recipient of CAR-T cell therapy outside of this protocol.
• Active central nervous system (CNS) or meningeal involvement by tumor.
• History of additional active malignancy other than non-melanoma skin cancer, carcinoma in situ (e.g. cervix, bladder, breast).
• Active human immunodeficiency virus (HIV) infection.
• Subjects with uncontrolled intercurrent illness including, but not limited to ongoing or active infection, symptomatic congestive heart failure, unstable angina pectoris, cardiac arrhythmia, pulmonary abnormalities or psychiatric illness/social situations that would limit compliance with study requirements.
• Pregnant or breastfeeding women.
• Evidence of myelodysplasia or cytogenetic abnormality indicative of myelodysplasia on any bone marrow biopsy prior to initiation of therapy.
• Serologic status reflecting active hepatitis B or C infection.

Study Plan

How is the study designed?

Design Details

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

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: CAR-T cell therapy; CAR-T cell infusion intravenously once	Biological: CAR-T; CAR-T cell (CHXCART01) infusion intravenously once at a dose of 0.2-2 million cells/kg recipient body weight

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Complete response rate (CRR) for ALL and Overall response rate (ORR) for lymphoma	Complete response for ALL was defined as leukemic cells <5% in bone marrow. Overall response for lymphoma was defined as complete response plus partial response defined by Lugano criteria.	1 month for subjects with ALL, and 3 months for subjects with lymphoma
Incidence and severity of adverse events	Severity of adverse events are graded according to CTCAEv5.0.	through study completion, an average of 6 months

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Frequency of minimal residual disease for ALL	Measurable residual disease in bone marrow by flow cytometry or PCR methods.	1 month
Overall survival	survival as estimated by Kaplan-Meier method. Death from any cause is considered as event for analysis.	1 year
Event-free survival	Event-free survival as estimated by Kaplan-Meier method. Events are death from any cause, or relapse or progression of underlying disease.	1 year
Proportion of products successfully manufactured	Success defined as meeting product release criteria with at least 0.2 million cells/kg recipient body weight	at the time of CAR-T cell infusion

Collaborators and Investigators

• Sponsor: Hong Kong Children's Hospital
• Investigators: Principal Investigator: Pamela Lee, MD, The University of Hong Kong

Study record dates

Study Major Dates

• Study Start (Actual): May 1, 2024
• Primary Completion (Estimated): December 31, 2037
• Study Completion (Estimated): December 31, 2037

Study Registration Dates

• First Submitted: March 3, 2025
• First Submitted That Met QC Criteria: March 4, 2025
• First Posted (Actual): March 25, 2025

Study Record Updates

• Last Update Posted (Actual): March 25, 2025
• Last Update Submitted That Met QC Criteria: March 4, 2025
• Last Verified: March 1, 2025

More Information

Terms related to this study

• Keywords: Pediatric; CAR-T cell
• Additional Relevant MeSH Terms: Neoplasms; Immune System Diseases; Neoplasms by Histologic Type; Hematologic Diseases; Lymphatic Diseases; Lymphoproliferative Disorders; Immunoproliferative Disorders; Lymphoma, Non-Hodgkin; Leukemia; Lymphoma; Lymphoma, B-Cell; Leukemia, Lymphoid; Precursor Cell Lymphoblastic Leukemia-Lymphoma
• Other Study ID Numbers: HKCH-REC-2021-032

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