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)
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: AUTO1.
- Who it may be relevant to
- Registry conditions: Relapsed or Refractory B Cell Acute Lymphoblastic Leukemia, Relapsed or Refractory B Cell Non-Hodgkin Lymphoma. Basic parameters: 0 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
- United States, Spain, United Kingdom
- Next step
- Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →
Unsure about the terms? Read our patient guide →
Official title
A Single-Arm, Open-Label, Multicenter, Phase 1b/2 Study Evaluating the Safety and Efficacy of AUTO1 (Obecabtagene Autoleucel [Obe-cel]) in Pediatric Patients With CD19-positive Relapsed/Refractory (R/R) B Cell Acute Lymphoblastic Leukemia (B ALL) or R/R Aggressive Mature B Cell Non-Hodgkin Lymphoma (B NHL).
Overview
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).
Detailed description
This is a single-arm, open-label, multi-center, Phase 1b/2 study to determine the safety and efficacy of obe-cel administered intravenously in pediatric patients \< 18 years old with r/r B ALL and with r/r aggressive mature B NHL.
The safety and tolerability of obe cel in pediatric patients will be continually monitored by the Sponsor. Efficacy endpoints will be determined by an Independent Response Review Committee (IRRC).
The study will involve consented patients going through the following sequential study periods: screening, leukapheresis, bridging as necessary, lymphodepletion, treatment evaluation, and follow-up.
Interventions
- Biological AUTO1
Following lymphodepletion with chemotherapy (cyclophosphamide and fludarabine) patients will be treated with anti-CD19 chimeric antigen receptor (CAR) T cells
Primary outcome measures
- Frequency and severity of adverse events (AEs) and serious adverse events (SAEs) [Time frame: Up to 24 months]
- Incidence and duration of severe hypogammaglobulinemia [Time frame: 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 [Time frame: 3 months]
Secondary outcome measures (10)
- CR per IRRC assessment at any time in B ALL [Time frame: 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 [Time frame: Up to 24 months]
- Minimal residual disease (MRD)-negative ORR per IRRC assessment at any time in B ALL [Time frame: Up to 24 months]
- Event-free survival in B ALL [Time frame: Up to 24 months]
- Overall survival (OS) in B ALL [Time frame: Up to 24 months]
- ORR (CR or partial response [PR]) per Investigator assessment occurring at any time in B NHL [Time frame: Up to 24 months]
- Duration of response in B NHL [Time frame: Up to 24 months]
- Progression-free survival in B NHL [Time frame: Up to 24 months]
- OS in B NHL [Time frame: Up to 24 months]
- Incidence of CD19-negative relapse at any time in B NHL [Time frame: Up to 24 months]
Eligibility criteria
Inclusion criteria
- < 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.
Exclusion criteria
- 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.
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
United States · 3 centers
- Children's Hospital of Philadelphia — Philadelphia
- Methodist Children's Hospital — San Antonio
- Primary Children's Hospital — Salt Lake City
United Kingdom · 3 centers
- Great Ormond Street Hospital for Children NHS Foundation Trust — London
- Royal Manchester Children's Hospital — Manchester
- Great North Children's Hospital — Newcastle upon Tyne
Spain · 2 centers
- Hospital Vall d'Hebron — Barcelona
- Hospital Nino Jesus — Madrid
Publications
- Davis KL, Yao CC, Zimmerman JAO, Rau RE. Immunotherapy in B-Cell Acute Lymphoblastic Leukemia. J Natl Compr Canc Netw. 2025 Dec;23(12):e257067. doi: 10.6004/jnccn.2025.7067. PMID 41671463
Identifiers
NCT: NCT06173518 · AUTO1-PY1 · 2023-506307-26-00