Safety and Efficacy of CAR T Cell Therapy in Patients with R/r B-ALL

October 8, 2024 updated by: Kara Yakhteh Tajhiz Azma Company

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)

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?

Study Overview

Status

Recruiting

Conditions

Intervention / Treatment

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.

Study Type

Interventional

Enrollment (Estimated)

5

Phase

  • Phase 2
  • Phase 1

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Locations

  • Iran, Islamic Republic of
      • Tehran, Iran, Islamic Republic of, 1419733151
        • Recruiting
        • Pediatric cell and gene therapy research center, Children medical center
        • Contact:

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

  • Child
  • Adult

Accepts Healthy Volunteers

No

Description

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

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: N/A
  • Interventional Model: Single Group Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: Anti-CD19 CAR-T treatment group
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).

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Time Frame
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
First month and 2-3 months after intervention
Overall survival
Time Frame: First month and 3 months after intervention
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
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
First month and 3 months after intervention
Event-free survival
Time Frame: First month and 2-3 months after intervention
First month and 2-3 months after intervention

Secondary Outcome Measures

Outcome Measure
Time Frame
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
6 months and 12 months after intervention
Investigation of Minimal residual disease in patient
Time Frame: First month and 2-3 months after intervention
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
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
6 months and 12 months after intervention
Incidence of tumor lysis syndrome (TLS)
Time Frame: Months 1, 3, 6, and 12 after the intervention
Months 1, 3, 6, and 12 after the intervention
Incidence of leukopenia
Time Frame: Months 1, 3, 6, and 12 after the intervention
Months 1, 3, 6, and 12 after the intervention
Incidence of infection
Time Frame: Months 1, 3, 6, and 12 after the intervention
Months 1, 3, 6, and 12 after the intervention
Event-free survival
Time Frame: 6 months and 12 months after intervention
6 months and 12 months after intervention
Overall survival
Time Frame: 6 months and 12 months after intervention
6 months and 12 months after intervention

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Kara Yakhteh Tajhiz Azma Company

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

May 20, 2024

Primary Completion (Estimated)

September 22, 2026

Study Completion (Estimated)

September 22, 2027

Study Registration Dates

First Submitted

October 1, 2024

First Submitted That Met QC Criteria

October 8, 2024

First Posted (Actual)

October 10, 2024

Study Record Updates

Last Update Posted (Actual)

October 10, 2024

Last Update Submitted That Met QC Criteria

October 8, 2024

Last Verified

October 1, 2024

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

  • IR.NREC.1403.003

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

No

Studies a U.S. FDA-regulated device product

No

product manufactured in and exported from the U.S.

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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