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).
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.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
30
Following lymphodepletion with chemotherapy (cyclophosphamide and fludarabine) patients will be treated with anti-CD19 chimeric antigen receptor (CAR) T cells
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, United States
RECRUITINGMethodist Children's Hospital
San Antonio, Texas, United States
RECRUITINGPrimary Children's Hospital
Salt Lake City, Utah, United States
RECRUITINGHospital Vall d'Hebron
Barcelona, Spain
RECRUITINGHospital Nino Jesus
Madrid, Spain
RECRUITINGGreat Ormond Street Hospital for Children NHS Foundation Trust
London, United Kingdom
RECRUITINGRoyal Manchester Children's Hospital
Manchester, United Kingdom
RECRUITINGGreat North Children's Hospital
Newcastle upon Tyne, United Kingdom
RECRUITINGFrequency 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
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
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