A Study of CD19 Targeted CAR T Cell Therapy in Pediatric Patients With Relapsed or Refractory B Cell Acute Lymphoblastic Leukemia (B ALL) and Aggressive Mature B-cell Non-Hodgkin Lymphoma (B NHL)

NCT06173518 | Recruiting Phase 1 DMC FDA Drug

• 2 other identifiers: AUTO1-PY12023-506307-26-00
• Study Type: interventional
• Target: 30
• Locations: 3 countries
• Sites: 8

Brief Summary

This is a Phase 1b/2 study to evaluate the safety and efficacy of autologous T cells engineered with a chimeric antigen receptor (CAR) targeting cluster of differentiation (CD)19 in pediatric patients with relapsed or refractory (r/r) B cell acute lymphoblastic leukemia (B ALL) and r/r B cell Non-Hodgkin lymphoma (B NHL).

Conditions

Relapsed or Refractory B Cell Acute Lymphoblastic Leukemia; Relapsed or Refractory B Cell Non-Hodgkin Lymphoma

Keywords

B cell acute lymphoblastic leukemia; B cell Non-Hodgkin lymphoma; Relapsed B cell acute lymphoblastic leukemia; Relapsed B cell Non-Hodgkin lymphoma; Refractory B cell acute lymphoblastic leukemia; Refractory B cell Non-Hodgkin lymphoma; Aggressive mature B cell Non-Hodgkin lymphoma; Pediatric ALL; Pediatric NHL; Obecabtagene autoleucel; CD19-positive CAR T cell; Obe-cel

Outcome Measures

Primary Outcomes (3)

• Frequency and severity of adverse events (AEs) and serious adverse events (SAEs)

  Up to 24 months

• Incidence and duration of severe hypogammaglobulinemia

  Up to 24 months

• Proportion of pediatric participants with r/r B ALL at screening who achieve complete remission (CR) within 3 months of obe-cel infusion per Independent Response Review Committee (IRRC) assessment

  3 months

Secondary Outcomes (10)

• CR per IRRC assessment at any time in B ALL

  Up to 24 months

• Overall remission rate (ORR) (CR + complete remission with incomplete recovery of counts [CRi]) per IRRC assessment at any time in B ALL

  Up to 24 months

• Minimal residual disease (MRD)-negative ORR per IRRC assessment at any time in B ALL

  Up to 24 months

• Event-free survival in B ALL

  Up to 24 months

• Overall survival (OS) in B ALL

  Up to 24 months

Study Arms (1)

AUTO1

EXPERIMENTAL

Biological: AUTO1

Interventions

AUTO1 BIOLOGICAL

Following lymphodepletion with chemotherapy (cyclophosphamide and fludarabine) patients will be treated with anti-CD19 chimeric antigen receptor (CAR) T cells

Also known as: Obecabtagene autoleucel (obe-cel)

AUTO1

Eligibility Criteria

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

You may qualify if:

• \< 18 years old at screening
• ≥ 6 kg body weight at screening
• Pediatric patients with r/r B ALL
• r/r CD19-positive aggressive mature B including the B NHL subtypes: i) diffuse large B cell lymphoma, ii) Burkitt's lymphoma, iii) primary mediastinal large B cell lymphoma, iv) high-grade B cell lymphoma (not otherwise specified).
• Karnofsky (age ≥ 10 years) or Lansky (age \< 10 year) performance status score ≥ 50%.
• In participants with B ALL, local documentation of CD19 expression on leukemic blasts in the BM, peripheral blood, or cerebrospinal fluid or biopsy done no more than 30 days prior to consent.
• Adequate renal, hepatic, pulmonary, and cardiac function.

You may not qualify if:

• Diagnosis of chronic myelogenous leukemia in lymphoid blast crisis.
• History or presence of clinically relevant central nervous system (CNS) pathology unrelated to CNS leukemia.
• Presence of active or uncontrolled fungal, bacterial, viral, or other infection requiring systemic antimicrobials for management.
• Received prior (\< 3 months before obe cel infusion) stem cell transplantation.
• Prior CD19 targeted therapy other than blinatumomab.
• Experienced Grade ≥ 3 neurotoxicity following blinatumomab.

Sponsors & Collaborators

• Autolus Limited: lead

Study Sites (8)

Children's Hospital of Philadelphia

Philadelphia, Pennsylvania, 19104, United States

RECRUITING

Methodist Children's Hospital

San Antonio, Texas, 78229, United States

RECRUITING

Primary Children's Hospital

Salt Lake City, Utah, 84113, United States

RECRUITING

Hospital Vall d'Hebron

Barcelona, Spain

RECRUITING

Hospital Nino Jesus

Madrid, Spain

RECRUITING

Great Ormond Street Hospital for Children NHS Foundation Trust

London, United Kingdom

RECRUITING

Royal Manchester Children's Hospital

Manchester, United Kingdom

RECRUITING

Great North Children's Hospital

Newcastle upon Tyne, United Kingdom

RECRUITING

MeSH Terms

Conditions

Recurrence; Burkitt Lymphoma; Lymphoma, B-Cell; Precursor Cell Lymphoblastic Leukemia-Lymphoma

Condition Hierarchy (Ancestors)

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

Central Study Contacts

Autolus Ltd

CONTACT

+44 (0) 203 911 4385; clinicaltrials@autolus.com

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: December 8, 2023
• First Posted: December 15, 2023
• Study Start: November 16, 2023
• Primary Completion (Estimated): November 1, 2027
• Study Completion (Estimated): November 1, 2027
• Last Updated: March 2, 2026

Record last verified: 2026-02

Locations

US (3); ES (2); GB (3)