NCT02906371

Study of the Tocilizumab Optimization Timing for CART19 Associated Cytokine Release Syndrome

A Two Cohort Pilot Study of the Tocilizumab Optimization Timing for CART19 Associated Cytokine Release Syndrome (CRS) Management in Pediatric Patients With CD19 Expressing Relapsed/Refractory B-cell Acute Lymphoblastic Leukemia (ALL)

• Status: Completed
• Phase: Phase 1
• Study type: Interventional
• Enrollment: 80 (actual)
• Sponsor: University of Pennsylvania · Academic / Other
• Sex: All
• Age: 1 Year – 24 Years
• Healthy volunteers: Not accepted

Summary

This is a two cohort, open-label, pilot study to describe the efficacy of administration timing of tocilizumab on CART19 (CTL019) associated cytokine release syndrome safety events in pediatric patients with CD19 expressing relapsed and refractory B-cell acute lymphoblastic leukemia with high versus low pre-infusion tumor burden following redirected autologous T cells transduced with the anti-CD19 lentiviral vector (CART19/CTL019).

Detailed description

The duration of active protocol intervention is approximately 12-15 months from the screening visit. The protocol will require approximately 12-18 months to complete enrollment.Approximately 39 enrolled patients to reach at least 35 infused patients, with the ultimate goal of 15 patients in the high tumor burden cohort (Cohort A). Inclusion criteria are designed to include pediatric patients aged 1-24 years with CD19 expressing relapsed/refractory B-cell acute lymphoblastic leukemia (ALL).Tocilizumab will be given once to high disease burden patients, and then the patients will be managed for CRS as per the standard algorithm (including subsequent tocilizumab, if needed). Two cohorts are defined based upon pre-infusion high versus low tumor burden; with the high tumor burden cohort (high risk of severe CRS) to receive earlier administration of tocilizumab for CRS management and the low tumor burden cohort (low risk of severe CRS) to receive standard timing of tocilizumab for CRS

Conditions

• Lymphoblastic Leukemia, Acute, Childhood

Interventions

Type	Name	Description
DRUG	Tocilizumab	Patients with ≥ 40% blasts in the bone marrow at pre-infusion will be enrolled in the early tocilizumab cohort and will follow early CRS treatment algorithm.
DRUG	Tocilizumab	Patients with ˂ 40% blasts in the bone marrow at pre-infusion (\~ Day -5 to -1) will follow the standard CRS Rx algorithm
BIOLOGICAL	CART 19	CART-19 cells transduced with a lentiviral vector to express either anti-CD19ζ scFv TCRζ:41BB, administered by i.v. injection using an intra-patient dose escalation approach: 10% on day 0, 30% on day 1 with a total dose goal of \~1.5 x107 - 5 x109 (\~3x105 - 1x108/kg) T cells.

Timeline

• Start date: 2016-08-01
• Primary completion: 2020-03-11
• Completion: 2021-06-30
• First posted: 2016-09-20
• Last updated: 2021-07-02

Locations

1 site across 1 country: United States

Regulatory

• FDA-regulated drug study