acute lymphoblastic leukemia. Lymphoblastic (diffuse) lymphoma
Conditions
Interventions
Intervention group: single-arm, This study is a single-arm trial evaluating autologous CAR-T cell therapy in pediatric patients with acute lymphoblastic leukemia (ALL). The intervention is conducted a
Sponsors
Tehran University of Medical Sciences
Eligibility
Sex/Gender
All
Age
3 Years to 18 Years
Inclusion criteria
Inclusion criteria: Confirmed diagnosis of acute lymphoblastic leukemia (ALL) of either T-cell or B-cell lineage, verified by immunophenotyping or molecular methods. Age between 3 and 18 years Relapsed disease after hematopoietic stem cell transplantation (HSCT) or eligible for transplantation but lacking a suitable donor. Performance status = 60% (Karnofsky or Lansky scale, as appropriate) Adequate organ function, defined as:Serum creatinine and renal function within age-appropriate normal limits,ALT/AST = 2.5 × upper limit of normal (ULN),Left ventricular ejection fraction (LVEF) = 45%. No evidence of uncontrolled active infection At least 4 weeks since the last chemotherapy or radiotherapy session Written informed consent obtained from parents or legal guardians Ability and willingness of the patient and family to comply with study procedures and follow-up visits
Exclusion criteria
Exclusion criteria: Presence of uncontrolled active infection, including sepsis, systemic fungal, or severe viral infection Severe organ dysfunction, defined as:Cardiac failure with LVEF 2 × upper normal limit (age-adjusted),Hepatic failure with AST/ALT > 5 × upper normal limit or total bilirubin > 2 × upper normal limit Active central nervous system (CNS3) leukemia involvement at screening Active autoimmune disease or requirement for long-term corticosteroid or immunosuppressive therapy. Active chronic viral infections, including HIV, hepatitis B, or hepatitis C (positive screening tests) Prior exposure to cell or gene therapy, including previous CAR-T cell therapy Pregnancy or breastfeeding in female patients of childbearing potential Inability or unwillingness of the patient or legal guardians to provide written informed consent or comply with study requirements. Any medical or psychiatric condition that, in the investigator’s judgment, may compromise patient safety or interfere with study outcomes.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Safety and Tolerability. Timepoint: 3 month after the injection. Method of measurement: Recording clinical symptoms and blood tests.;Preliminary Therapeutic Response. Timepoint: 4 weeks after the injection (day 28). Method of measurement: The first assessment of therapeutic response, including Complete Remission (CR) or Partial Remission (PR), will be performed according to international ALL response criteria. | — |
Secondary
| Measure | Time frame |
|---|---|
| Progression-Free Survival, PFS. Timepoint: 3 month after the injection. Method of measurement: Weekly or biweekly clinical and laboratory assessments to monitor disease status and initial response.;Long-term evaluation of safety and delayed complications. Timepoint: 12 month after the injection. Method of measurement: Monthly evaluations for late-onset adverse events, immune dysregulation, or organ toxicities. | — |
Countries
Iran (Islamic Republic of)
Contacts
Public ContactDr Javad Verdi
Tehran University of Medical Sciences
Outcome results
None listed