CAR-T cell therapy for children with MRD-positive B-ALL

Clinical Study on the Safety and Efficacy of CD19/CD22 CAR-T Cell Therapy in MRD-Positive B-lineage Acute Lymphoblastic Leukemia in Children.

This study is testing a new CAR-T cell therapy for children with a type of leukemia who still have leftover cancer cells after their first treatment, to see if it can help them get better.

Quick facts

• Phase: Phase 1
• Study type: Interventional
• Enrollment: 10 (estimated)
• Ages: 1 Year to 18 Years
• Sex: All
• Sponsor: Guangzhou Women and Children's Medical Center (Academic / other)
• Drugs / interventions: chemotherapy, CAR-T, CAR T, CART, cyclophosphamide, fludarabine
• Locations: 1 site (Guangzhou, Guangdong)
• Trial ID: NCT06752785 on ClinicalTrials.gov

What this trial studies

This clinical trial evaluates the safety and efficacy of CD19/CD22 dual-target CAR-T cell therapy in children with minimal residual disease (MRD) positive B-lineage acute lymphoblastic leukemia (B-ALL) after induction remission. The study involves preparing autologous CAR-T cells, administering them after lymphocyte depletion, and continuing chemotherapy post-infusion. The trial aims to improve outcomes for children who have not achieved complete remission after initial treatment. It is conducted at a single center in Guangzhou, China.

Who should consider this trial

Good fit: Ideal candidates are Chinese children aged 1-18 years with MRD-positive B-ALL after induction therapy.

Not a fit: Patients who do not express CD19/CD22 or have severe organ dysfunction may not benefit from this study.

Why it matters

Potential benefit: If successful, this therapy could significantly improve the prognosis for children with MRD-positive B-ALL.

How similar studies have performed: Previous studies have shown promising results with CAR-T therapies for similar conditions, indicating potential for success in this approach.

Eligibility criteria

Inclusion Criteria:

1. Parents or legal guardians fully understand, are informed of this study and sign the informed consent form (ICF); are willing to follow and can complete all test procedures.
2. Chinese children aged 1-18 years old at the time of screening, regardless of gender, with a body weight ≥ 10 kg.
3. Bone marrow examination confirms that MRD is still positive on the 46th day after induction remission.
4. Tumor cells in the bone marrow (BM) or peripheral blood (PB) express CD19/CD22 within 3 months before screening.
5. Good organ function, which needs to meet the following criteria: (1)ALT ≤ 5 times the upper limit of normal value (ULN); (2)total bilirubin ≤ 2 times ULN (Gilbert's syndrome ≤ 3 times ULN); (3)without \> grade 1 dyspnea when not inhaling oxygen, and oxygen saturation \> 95%; (4)left ventricular ejection fraction (LVEF) ≥ 50%; (5)serum creatinine ≤ 1.5 times ULN.
6. Karnofsky score (≥ 16 years old) ≥ 70 or Lansky (\< 16 years old) score ≥ 50.
7. Expected survival period of at least 12 weeks.
8. Have sufficient venous access (for apheresis or venous blood sampling), and have no other contraindications for blood cell separation.

Exclusion Criteria:

1. Have genetic diseases, except Down syndrome.
2. Have a history of other malignancies or have other malignancies simultaneously.
3. Meet any of the following conditions: (1)hepatitis B surface antigen (HBsAg) positive or HBV DNA quantification higher than the upper limit of normal value; (2)hepatitis C antibody (HCV Ab) positive and HCV RNA quantification higher than the upper limit of normal value; (3)human immunodeficiency virus antibody (HIV-Ab) positive; (4)Treponema pallidum antibody (TP-Ab) positive; (5)EBV DNA higher than the upper limit of normal value; (6)cytomegalovirus DNA higher than the upper limit of normal value.
4. Have or are suspected to have uncontrolled or require intravenous drug treatment for fungal, bacterial, viral or other infections.
5. Long-acting G-CSF is prohibited within 21 days before screening, and short-acting G-CSF is prohibited within 7 days before screening.
6. Have active central nervous system leukemia.
7. Are allergic to albumin and aminoglycoside antibiotics.
8. Have undergone organ transplantation (except hematopoietic stem cell transplantation).
9. Have participated in other interventional clinical studies within 3 months before screening (received active test drug treatment), or intend to participate in another clinical trial or receive anti-tumor treatment other than that specified in the protocol during the entire study period.
10. Cannot tolerate chemotherapy and cytokine storm due to impaired function of important organs.
11. Other situations that the investigator deems not suitable for participating in this clinical trial.

Where this trial is running

Guangzhou, Guangdong

• Guangzhou Medical University Affiliated Women and Children's Medical Center — Guangzhou, Guangdong, China (Recruiting)

Study contacts

• Study coordinator: Hua Jiang, phd

Conditions Acute Lymphoblastic Leukemia; B-ALL, CAR-T