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

Phase 1

Recruiting

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

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 CD19 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 AUTO1 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 AUTO1.

The safety and tolerability of AUTO1 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 in the event any protocol defined safety stopping criteria are met. If no pre-defined safety events related to AUTO1 are met, and the safety data are consistent with what has previously been observed with AUTO1, 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 AUTO1-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

Inclusion Criteria

Not provided

Exclusion Criteria

Not provided

Arm && Interventions

Group	Intervention	Description
AUTO1	AUTO1	-

Primary Outcome Measures

Name	Time	Method
Incidence of severe hypogammaglobulinemia	Up to 24 months
Frequency and severity of adverse events (AE) and serious adverse events (SAE)	Up to 24 months
Duration of severe hypogammaglobulinemia	Up to 24 months

Secondary Outcome Measures

Name	Time	Method
Duration of remission (DOR) 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 NHL	Up to 24 months
Proportion of patients achieving minimal residual disease (MRD)-negative remission in bone marrow (BM) within 3 months of AUTO1 dosing in B ALL	Up to 24 months
Overall survival (OS) in B ALL	Up to 24 months
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 AUTO1 infusion
Proportion of patients achieving complete remission (CR) within 3 months per Investigator assessment in B ALL	Up to 24 months
Duration of response (DOR) in B NHL	Up to 24 months
Incidence of CD19-negative relapse at any time in B ALL	Up to 24 months
Progression-free survival (PFS) in B NHL	Up to 24 months
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 AUTO1 infusion
Event-free survival (EFS) in B ALL	Up to 24 months

Locations (6): Methodist Children's Hospital
🇺🇸
San Antonio, Texas, United States
Hospital Vall d'Hebron
🇪🇸
Barcelona, Spain
Great North Children's Hospital
🇬🇧
Newcastle Upon Tyne, United Kingdom
Great Ormond Street Hospital for Children NHS Foundation Trust
🇬🇧
London, United Kingdom
Primary Children's Hospital
🇺🇸
Salt Lake City, Utah, United States
Hospital Nino Jesus
🇪🇸
Madrid, Spain