CAR-T cell therapy for children with relapsed leukemia 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 is testing a new CAR-T cell therapy to see if it can help children with relapsed leukemia or lymphoma feel better and improve their chances of recovery.

Quick facts

• Phase: NA
• Study type: Interventional
• Enrollment: 18 (estimated)
• Ages: 1 Year to 17 Years
• Sex: All
• Sponsor: Hong Kong Children's Hospital (other)
• Drugs / interventions: CAR T, CAR-T, chimeric antigen receptor, chemotherapy
• Locations: 1 site (Hong Kong)
• Trial ID: NCT06866873 on ClinicalTrials.gov

What this trial studies

This study evaluates the safety and efficacy of CAR-T cell therapy targeting the CD19 antigen in pediatric patients with relapsed or refractory acute lymphoblastic leukemia (ALL) or lymphoma. Participants will receive autologous T cells modified to express a chimeric antigen receptor after undergoing a chemotherapy lymphodepletion regimen. The study aims to validate the safety profile of this treatment and assess the response rates in the targeted patient population.

Who should consider this trial

Good fit: Ideal candidates are children aged 1-17 years with relapsed or refractory ALL or lymphoma who have undergone at least two lines of prior therapy.

Not a fit: Patients whose disease is not CD19 positive or who do not meet the specified organ function criteria may not benefit from this study.

Why it matters

Potential benefit: If successful, this therapy could provide a new treatment option for children with difficult-to-treat leukemia or lymphoma.

How similar studies have performed: Other studies using CAR-T cell therapy for similar conditions have shown promising results, indicating potential for success in this approach.

Eligibility criteria

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.

Where this trial is running

Hong Kong

• Hong Kong Children's Hospital — Hong Kong, Hong Kong (RECRUITING)

Study contacts

• Principal investigator: Pamela Lee, MD — The University of Hong Kong
• Study coordinator: Daniel Cheuk, MBBS

Conditions: Lymphoma, B-Cell, Acute Lymphoblastic Leukemia, Pediatric, CAR-T cell, Pediatric