Clinical trial NCT06635330: Safety and Efficacy of CAR T Cell Therapy in Patients with R/r B-ALL
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)
Study type: INTERVENTIONAL
Overall status: RECRUITING
Phase: PHASE1
PHASE2
Conditions: Relapse/Refractory B-cell Acute Lymphoblastic Leukemia
Lead sponsor: Kara Yakhteh Tajhiz Azma Company
Sponsor class: NETWORK
First posted: 2024-10-10
Enrollment: 5
Sex: ALL
Minimum age (years): 2
Maximum age (years): 18
Standard ages: CHILD
ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief summary: 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.

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 \&gt; 12 weeks
* Lansky or Karnofsky score \&gt; 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

Primary outcomes:
measure: Percentage of patients with overall remission rate (ORR) of complete response (CR) or complete remission with incomplete blood count recovery (CRi)
timeFrame: First month and 2-3 months after intervention
measure: Overall survival
timeFrame: First month and 3 months after intervention
measure: Incidence of cytokine release syndrome: grade 3 and 4
timeFrame: First month and 3 months after intervention
measure: Incidence of Immune effector cell-associated neurotoxicity syndrome (ICANS): grade 3 and 4
timeFrame: First month and 3 months after intervention
measure: Event-free survival
timeFrame: First month and 2-3 months after intervention
Secondary outcomes:
measure: Percentage of patients with overall remission rate (ORR) of complete response (CR) or complete remission with incomplete blood count recovery (CRi)
timeFrame: 6 months and 12 months after intervention
measure: Investigation of Minimal residual disease in patient
timeFrame: First month and 2-3 months after intervention
measure: Incidence of cytokine release syndrome: grade 3 and 4
timeFrame: 6 months and 12 months after intervention
measure: Incidence of Immune effector cell-associated neurotoxicity syndrome (ICANS): grade 3 and 4
timeFrame: 6 months and 12 months after intervention
measure: Incidence of tumor lysis syndrome (TLS)
timeFrame: Months 1, 3, 6, and 12 after the intervention
measure: Incidence of leukopenia
timeFrame: Months 1, 3, 6, and 12 after the intervention
measure: Incidence of infection
timeFrame: Months 1, 3, 6, and 12 after the intervention
measure: Event-free survival
timeFrame: 6 months and 12 months after intervention
measure: Overall survival
timeFrame: 6 months and 12 months after intervention
Other outcomes:
Locations:
city: Tehran
country: Iran, Islamic Republic of
facility: Pediatric cell and gene therapy research center, Children medical center
Study officials:
References: