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)
- Conditions
- Relapsed or Refractory B Cell Non-Hodgkin LymphomaRelapsed or Refractory B Cell Acute Lymphoblastic Leukemia
- Interventions
- Registration Number
- NCT06173518
- Lead Sponsor
- Autolus Limited
- Brief Summary
This is a Phase Ib 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 Ib study to determine the safety and preliminary efficacy of obe-cel administered intravenously in pediatric patients with r/r B ALL and with r/r aggressive mature B NHL. This study is designed to evaluate the safety and preliminary efficacy of obe-cel.
The safety and tolerability of obe-cel in pediatric patients will be continually monitored by the Sponsor. Additionally, the Independent Data Monitoring Committee (IDMC) will review the rolling safety data generated after 6 and 12 treated patients have been monitored for at least 28 days and every 6 months thereafter, and in the event any protocol defined safety stopping criteria are met. If no pre-defined safety events related to obe-cel are met, and the safety data are consistent with what has previously been observed with obe-cel, the IDMC can recommend continuing the study without or with modifications. Based on emerging data, the study may be stopped early due to excessive toxicity, i.e., certain pre-defined obe-cel-related safety events or deaths.
The study will involve consented patients going through the following sequential study periods: screening, leukapheresis, bridging as necessary, lymphodepletion, treatment evaluation, and follow-up.
Recruitment & Eligibility
- Status
- RECRUITING
- Sex
- All
- Target Recruitment
- 30
Not provided
Not provided
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- SINGLE_GROUP
- Arm && Interventions
Group Intervention Description AUTO1 AUTO1 -
- Primary Outcome Measures
Name Time Method 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
- Secondary Outcome Measures
Name Time Method Overall remission rate (ORR) in B ALL patients Up to 24 months Defined as best response of complete remission (CR) or complete remission with incomplete recovery of counts (CRi) per Investigator assessment occurring at any time after obe-cel infusion
Overall response rate (ORR) in B NHL patients Up to 24 months Defined as best response of complete response (CR) or partial response (PR) per Investigator assessment occurring at any time after obe-cel infusion
Detection of CAR T cells (copies/μg genomic deoxyribonucleic acid) measured by droplet digital polymerase chain reaction in the peripheral blood and BM following obe-cel infusion including duration of detectability. Up to 24 months Quantification of depletion of circulating CD19 expressing B cells as determined by flow cytometry in the peripheral blood. Up to 24 months Frequency and duration of hospitalization and/or critical care support to manage obe-cel-related toxicity within 6 months of obe-cel dosing. Up to 6 months Duration of remission (DOR) in B ALL Up to 24 months Duration of response (DOR) in B NHL Up to 24 months Overall survival (OS) in B ALL Up to 24 months Overall survival (OS) in B NHL Up to 24 months Incidence of CD19-negative relapse at any time in B ALL Up to 24 months Incidence of CD19-negative relapse at any time in B NHL Up to 24 months Event-free survival (EFS) in B ALL Up to 24 months Proportion of patients achieving minimal residual disease (MRD)-negative remission in bone marrow (BM) within 3 months of obe-cel dosing in B ALL Up to 24 months Proportion of patients achieving complete remission (CR) within 3 months per Investigator assessment in B ALL Up to 24 months Progression-free survival (PFS) in B NHL Up to 24 months Proportion of patients undergoing stem cell transplantation (SCT) while still in CR/CRi following obe-cel (in B ALL) Up to 24 months Proportion of enrolled patients for whom an obe-cel product can be manufactured and administered. Up to 6 months post-leukapheresis
Trial Locations
- Locations (8)
Hospital Vall d'Hebron
🇪🇸Barcelona, Spain
Hospital Nino Jesus
🇪🇸Madrid, Spain
Children's Hospital of Philadelphia
🇺🇸Philadelphia, Pennsylvania, United States
Royal Manchester Children's Hospital
🇬🇧Manchester, United Kingdom
Methodist Children's Hospital
🇺🇸San Antonio, Texas, United States
Primary Children's Hospital
🇺🇸Salt Lake City, Utah, United States
Great Ormond Street Hospital for Children NHS Foundation Trust
🇬🇧London, United Kingdom
Great North Children's Hospital
🇬🇧Newcastle Upon Tyne, United Kingdom