NCT03876769

Study of Efficacy and Safety of Tisagenlecleucel in HR B-ALL EOC MRD Positive Patients

A Phase II Trial of Tisagenlecleucel in First-line High-risk (HR) Pediatric and Young Adult Patients With B-cell Acute Lymphoblastic Leukemia (B-ALL) Who Are Minimal Residual Disease (MRD) Positive at the End of Consolidation (EOC) Therapy

• Status: Active Not Recruiting
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 121 (actual)
• Sponsor: Novartis Pharmaceuticals · Industry
• Sex: All
• Age: 1 Year – 25 Years
• Healthy volunteers: Not accepted

Summary

This is a single arm, open-label, multi-center, phase II study to determine the efficacy and safety of tisagenlecleucel in de novo HR pediatric and young adult B-ALL patients who received first-line treatment and are EOC MRD positive. The study will have the following sequential phases: screening, pre-treatment, treatment \& follow-up, and survival. After tisagenlecleucel infusion, patient will have assessments performed more frequently in the first month and then at Day 29, then every 3 months for the first year, every 6 months for the second year, then yearly until the end of the study. Efficacy and safety will be assessed at study visits and as clinically indicated throughout the study. The study is expected to end in approximately 8 years after first patient first treatment (FPFT). A post-study long term follow-up safety will continue under a separate protocol per health authority guidelines.

Conditions

• B-Cell Acute Lymphoblastic Leukemia

Interventions

Type	Name	Description
BIOLOGICAL	CTL019	Based on the subject's weight, one of two possible dose ranges will be prepared for the subject: Subjects ≤ 50 kg: 0.2 to 5.0 x 10(6) CAR-positive viable T cells per kg body weight OR Subjects \> 50 kg: 0.1 to 2.5 x 10(8) CAR-positive viable T cells

Timeline

• Start date: 2019-06-24
• Primary completion: 2025-08-20
• Completion: 2027-10-19
• First posted: 2019-03-15
• Last updated: 2026-04-17

Locations

45 sites across 11 countries: United States, Belgium, Canada, Denmark, France, Italy, Netherlands, Norway, Spain, Sweden, United Kingdom

Regulatory

• FDA-regulated drug study