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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 · Autolus Limited
In plain English
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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).
About this study
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.
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.
Study design
Enrollment target: 30 participants
Allocation: na
Masking: none
Age groups: child, adult
Timeline
Starts: 2023-11-16
Estimated completion: 2027-11
Last updated: 2026-03-02
Interventions
Biological: AUTO1
Primary outcomes
- • 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)
Sponsor
Autolus Limited · industry
Contacts & investigators
ContactAutolus Ltd · contact · clinicaltrials@autolus.com · +44 (0) 203 911 4385
All locations (8)
Children's Hospital of PhiladelphiaRecruiting
Philadelphia, Pennsylvania, United States
Methodist Children's HospitalRecruiting
San Antonio, Texas, United States
Primary Children's HospitalRecruiting
Salt Lake City, Utah, United States
Hospital Vall d'HebronRecruiting
Barcelona, Spain
Hospital Nino JesusRecruiting
Madrid, Spain
Great Ormond Street Hospital for Children NHS Foundation TrustRecruiting
London, United Kingdom
Royal Manchester Children's HospitalRecruiting
Manchester, United Kingdom
Great North Children's HospitalRecruiting
Newcastle upon Tyne, United Kingdom