NCT04094311

Study of Out of Specification for Tisagenlecleucel

A Phase IIIb Study of the Safety and Efficacy of Tisagenlecleucel Out of Specification for Commercial Release in Patients Who Are Consistent With the Label Indication

• Status: Recruiting
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 200 (estimated)
• Sponsor: Novartis Pharmaceuticals · Industry
• Sex: All
• Age: 0 Years – 100 Years
• Healthy volunteers: Not accepted

Summary

This study will evaluate the safety of tisagenlecleucel that is out of specification( OOS) for release as commercial product. Specifically, this study will evaluate the safety of CTL019 in the patients treated within the approved label by Japan Health Authority in Part 2. Only for Part 1, in addition to safety, key efficacy of CTL019 will also be evaluated.

Detailed description

This is a single-arm, open-label, multicenter, interventional Phase IIIb study in pediatric/young adult patients with relapsed/refractory (r/r) B-cell acute lymphoblastic leukemia (pALL) and adult patients with r/r large B-cell lymphoma (LBCL) including diffuse large B-cell lymphoma (DLBCL) not otherwise specified, high-grade B cell lymphoma, and DLBCL arising from follicular lymphoma for Part 1 and and r/r ALL and r/r non-Hodgkin's lymphomas (NHL) for Part 2 Patients whose final manufactured tisagenlecleucel patient-specific batch does not meet the approved local commercial release specifications are eligible for inclusion. Each case will be individually assessed and approved by the Novartis manufacturing facility and the Novartis global medical team (including Patient Safety). Following a single infusion of CTL019, the patient will be followed for 3 months for Part 1, and 1 day for Part 2.

Conditions

• B-cell Acute Lymphoblastic Leukemia
• Diffuse Large B-cell Lymphoma

Interventions

Type	Name	Description
BIOLOGICAL	CTL019	A single intravenous (i.v.) infusion of CAR-positive viable T cells.

Timeline

• Start date: 2019-11-21
• Primary completion: 2027-03-31
• Completion: 2027-03-31
• First posted: 2019-09-18
• Last updated: 2026-04-02

Locations

53 sites across 2 countries: Canada, Japan

Regulatory

• FDA-regulated drug study