CD19x22 Chimeric Antigen Receptor T-cell Therapy (CAR T) in Pediatric B-ALL

NCT06559189 | Recruiting Phase 1 DMC FDA Drug

• 2 other identifiers: 22-0998.ccNCI-2024-06242
• Study Type: interventional
• Target: 53
• Locations: 1 country
• Sites: 1

Brief Summary

This study will evaluate the safety and tolerability of administering a novel bispecific CD19/CD22-directed CAR T cell product (CD19x22) for the treatment of relapsed or refractory pediatric B-ALL.

Conditions

B-cell Acute Lymphoblastic Leukemia

Keywords

Pediatric; Relapsed; Refractory; B-cell Acute Lymphoblastic Leukemia; Hematologic diseases

Outcome Measures

Primary Outcomes (1)

• Safety Measured by Dose Limiting Toxicities (DLTs)

  The safety of the administering this bispecific CD19/CD22-directed CAR T cell product will be measured by assessing the DLTs in each disease burden cohort in a Bayesian optimal interval (BOIN) design to determine the MTD

  Infusion date to 28 days post-infusion

Secondary Outcomes (6)

• Overall Response Rate

  28 days post-infusion

• Minimal Residual Disease (MRD) Rate

  Months 1, 2, 3, 6, 9, and 12

• Minimal Residual Disease (MRD) Rate

  Months 1, 3, 6, and 12

• CD19-Relapse

  1 year

• Overall Survival

  1 year

Study Arms (2)

High Disease Burden Cohort

EXPERIMENTAL

≥25% bone marrow lymphoblasts and/or non-CNS extramedullary disease. Dose escalation will proceed independently within each cohort using the Bayesian Optimal Interval (BOIN)design. Dose begins at DL -1(1x10\^5 cells/kg).

Biological: CD19x22 CAR T

Low Disease Burden Cohort

EXPERIMENTAL

\<25% bone marrow lymphoblasts and no non-CNS extramedullary disease. Dose escalation will proceed independently within each cohort using the Bayesian Optimal Interval (BOIN)design. Dose begins at DL1 (3x10\^5 cells/kg).

Biological: CD19x22 CAR T

Interventions

CD19x22 CAR T BIOLOGICAL

The investigational product is an autologous, genetically modified CD19xCD22 CAR T cell product produced by the Gates Biomanufacturing Facility.

High Disease Burden Cohort; Low Disease Burden Cohort

Eligibility Criteria

• Age: 3 Months - 30 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

• Subjects must have a history of B precursor ALL with any of the following conditions:
• Relapsed two or more times.
• Relapsed at any time after allogeneic bone marrow transplant (BMT).
• Relapse or refractory after single antigen targeting CAR T cell therapy.
• i. 90 days must have elapsed post previous CAR infusion prior to apheresis. d. Refractory to standard therapy as determined by the treating physician. e. Patient and/or parents declining BMT options and would prefer CAR T Therapy.
• CD19 and/or CD22 present on last relapsed/refractory disease evaluation.
• Performance score (Lansky or Karnofsky ≥ 50%; or Eastern Cooperative Oncology Group (ECOG) must be ≤2).
• Meets criteria for potential leukapheresis collection or has leukapheresis product previously collected and stored per recommended guidelines.
• Males OR non-pregnant, non-lactating females.
• Aged 3 months to 30 years (inclusive) at time of consent and enrollment.
• Provision of a signed and dated consent form from parent or guardian (patients \< 18), the patient themselves (\> 18), or legally authorized representative (patient \> 18 who lack decision-making capacity) after standard of care (SOC) screening assessments are performed.
• Stated willingness to comply with all study procedures and be available for the duration of the study.
• Willingness to participate in long-term follow-up protocol.

You may not qualify if:

• Active, uncontrolled central nervous system (CNS) leukemia that is progressive despite other therapies or leading to CNS symptoms (including but not limited to: seizures, paresis, aphasia, hemorrhage, dementia, psychosis, or movement disorders) as determined by the treating physician at eligibility, prior to lymphodepleting chemotherapy (LD chemo), and pre- CD19x22 CAR T cell infusion.
• History of allogeneic stem cell transplantation prior to apheresis that meet the following criteria:
• Less than 100 days post-transplant;
• Evidence of active Graft-versus-Host Disease (GvHD) requiring systemic therapy;
• Less than 6 weeks post donor lymphocyte infusion (DLI).
• Active, uncontrolled, life-threatening infection that at the determination of the treating physician would preclude safe apheresis or tolerance of lymphodepleting chemotherapy, cell infusion, or increased risk of cytokine release syndrome.
• Evidence of severe organ dysfunction defined by:
• Baseline oxygen saturation of \< 90% on room air
• Myocardial dysfunction (based on age standards): Ejection fraction ≤ 40% or shortening fraction ≤ 28%, evidence of physiologically significant pericardial effusion as determined by an echocardiogram (ECHO), and clinically significant electrocardiogram (ECG or EKG) findings
• Transaminases \> 10x upper limit of normal (ULN) or bilirubin \> 5x the ULN, unless thought to be related to primary disease
• Estimated Creatinine (Cr) clearance \< 60 mL/min/1.73 m2 (if nuclear medicine GFR or other more specific testing exceeds this level than it can supersede the estimated clearance)
• Subjects of childbearing or child-fathering potential that are not willing to practice birth control from the time of enrollment on this study and for 12 months after receiving the investigational product
• Known HIV infection or active Hepatitis B or Hepatitis C infection.

Sponsors & Collaborators

• University of Colorado, Denver: lead

Study Sites (1)

Children's Hospital Colorado

Aurora, Colorado, 80045, United States

RECRUITING

MeSH Terms

Conditions

Burkitt Lymphoma; Recurrence; Hematologic Diseases

Condition Hierarchy (Ancestors)

Epstein-Barr Virus Infections; Herpesviridae Infections; DNA Virus Infections; Virus Diseases; Infections; Tumor Virus Infections; Lymphoma, B-Cell; Lymphoma, Non-Hodgkin; Lymphoma; Neoplasms by Histologic Type; Neoplasms; Lymphoproliferative Disorders; Lymphatic Diseases; Hemic and Lymphatic Diseases; Immunoproliferative Disorders; Immune System Diseases; Disease Attributes; Pathologic Processes; Pathological Conditions, Signs and Symptoms

Study Officials

• Vanessa Fabrizio, MD

  University of Colorado, Denver

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Vanessa Fabrizio, MD

CONTACT

720-777-6860; BMT@childrenscolorado.org

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: August 15, 2024
• First Posted: August 19, 2024
• Study Start: September 27, 2024
• Primary Completion (Estimated): December 1, 2028
• Study Completion (Estimated): December 1, 2029
• Last Updated: November 28, 2025

Record last verified: 2025-11

Data Sharing

• IPD Sharing: Will not share

Locations

US (1)