Safety and Efficacy of CAR T Cell Therapy in Patients with R/r B-ALL
For patients and families
In plain language
An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.
- What is being studied
- The protocol lists: anti-CD19 CAR T cell therapy.
- Who it may be relevant to
- Registry conditions: Relapse/Refractory B-cell Acute Lymphoblastic Leukemia. Basic parameters: 2 years — 18 years · All.
- What needs checking
- Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
- Where it takes place
- Iran
- Next step
- Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →
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Official title
A Phase I/II Single Arm Study, Safety and Efficacy Assessment of the CD19 CAR T Cell on Pediatric Patients with Relapsing or Refractory B Cell Acute Lymphoblastic Leukemia (r/r B-ALL)
Overview
The goal of this clinical trial is to evaluate the safety and efficacy of CD19 CAR-T cells in pediatric patients of all genders, aged 2 to 18 years, with relapsing or refractory B cell acute lymphoblastic leukemia (r/r B-ALL). The main questions it aims to answer are as following: 1. What is the percentage of patients with overall remission rate (ORR) of complete response (CR) or complete remission with incomplete blood count recovery (CRi)? 2. What is the rate of Event-free survival at first month and 2-3 months after intervention? 3. What is the rate of Overall survival at first month and at 3 months after the intervention?
Detailed description
B-cell acute lymphoblastic leukemia (B-ALL), as the most common type of pediatric tumor, is identified by unregulated cell proliferation of immature lymphoid cells that can infiltrate the bone marrow and blood. Also, relapse and refractory B-ALL (R/R B-ALL) is the main reason of global mortality due to the constraints of combination chemotherapy.
Over the past few years, substantial advancements have been made in treatment of ALL, specifically in the R/R context. Chimeric antigen receptor T (CAR-T) cells are a type of cancer immunotherapy treatment that function through modification of patient T cells to express CAR antigen on their surface. CAR-T cells aimed at CD19 have demonstrated promising activity in treatment of r/r B-ALL. In this study we aim to evaluate safety and efficacy of Anti-CD19 CAR T cell therapy in children with R/R B-ALL.
Interventions
- Biological anti-CD19 CAR T cell therapy
Anti-CD19 CAR-T cell therapy for R/R B-ALL pediatric patients. For patients 50 kg and less: 0.2 to 5 in ten to the power of six live CAR+ T cells per kilogram of body weight/ For patients over 50 kg: 0.1 to 2.5 in ten to the power of eight live CAR+ T cells (without considering weight).
Primary outcome measures
- Percentage of patients with overall remission rate (ORR) of complete response (CR) or complete remission with incomplete blood count recovery (CRi) [Time frame: First month and 2-3 months after intervention]
- Overall survival [Time frame: First month and 3 months after intervention]
- Incidence of cytokine release syndrome: grade 3 and 4 [Time frame: First month and 3 months after intervention]
- Incidence of Immune effector cell-associated neurotoxicity syndrome (ICANS): grade 3 and 4 [Time frame: First month and 3 months after intervention]
- Event-free survival [Time frame: First month and 2-3 months after intervention]
Secondary outcome measures (9)
- Percentage of patients with overall remission rate (ORR) of complete response (CR) or complete remission with incomplete blood count recovery (CRi) [Time frame: 6 months and 12 months after intervention]
- Investigation of Minimal residual disease in patient [Time frame: First month and 2-3 months after intervention]
- Incidence of cytokine release syndrome: grade 3 and 4 [Time frame: 6 months and 12 months after intervention]
- Incidence of Immune effector cell-associated neurotoxicity syndrome (ICANS): grade 3 and 4 [Time frame: 6 months and 12 months after intervention]
- Incidence of tumor lysis syndrome (TLS) [Time frame: Months 1, 3, 6, and 12 after the intervention]
- Incidence of leukopenia [Time frame: Months 1, 3, 6, and 12 after the intervention]
- Incidence of infection [Time frame: Months 1, 3, 6, and 12 after the intervention]
- Event-free survival [Time frame: 6 months and 12 months after intervention]
- Overall survival [Time frame: 6 months and 12 months after intervention]
Eligibility criteria
Inclusion criteria
- Ages 2 to 18 years with relapsed or refractory CD19+ B-ALL
- Presence of disease in the bone marrow
- Able to tolerate the apheresis process
- Life expectancy \> 12 weeks
- Lansky or Karnofsky score \> 50%
- At least 7 days passed since the last chemotherapy and the last treatment with corticosteroids
- Informed consent
- Having potential donor for stem cell transplantation
Exclusion criteria
- Presence of active malignancy other than the disease under study
- Chloroma and leukemic infiltration on MRI or significant neurological symptoms
- Any CNS disorder
- Presence of active GVHD
- Radiation therapy within last 14 days
- History of Anti-CD19 or Anti-CD20 therapy
- Donor lymphocyte injection or other cell therapy methods within the last 30 days
- Presence of severe active infection
- Organ dysfunction
Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.
Healthy volunteers: No
Study design
- Allocation
- N/A
- Model
- Single group
- Masking
- Open label
- Primary purpose
- Treatment
Study locations
Iran · 1 center
- Pediatric cell and gene therapy research center, Children medical center — Tehran
Identifiers
NCT: NCT06635330 · IR.NREC.1403.003