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)
Relapsed or Refractory B Cell Acute Lymphoblastic LeukemiaRelapsed or Refractory B Cell Non-Hodgkin Lymphoma
Investigational drug(s) / intervention(s)
AUTO1
AUTO1: Following lymphodepletion with chemotherapy (cyclophosphamide and fludarabine) patients will be treated with anti-CD19 chimeric antigen receptor (CAR) T cells
Study summary
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).
Eligibility
Sex
ALL
Min age
0 Years
Max age
18 Years
Healthy volunteers
No
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.
Primary outcome measure(s)
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
Trial sites (9)
Facility
City
Region
Status
The Mount Sinai Hospital
New York
New York
Recruiting
Children's Hospital of Philadelphia
Philadelphia
Pennsylvania
Recruiting
Methodist Children's Hospital
San Antonio
Texas
Recruiting
Primary Children's Hospital
Salt Lake City
Utah
Recruiting
Hospital Vall d'Hebron
Barcelona
Spain
Recruiting
Hospital Nino Jesus
Madrid
Spain
Recruiting
Great Ormond Street Hospital for Children NHS Foundation Trust
This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.
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