NCT02435849

Study of Efficacy and Safety of CTL019 in Pediatric ALL Patients

A Phase II, Single Arm, Multicenter Trial to Determine the Efficacy and Safety of CTL019 in Pediatric Patients With Relapsed and Refractory B-cell Acute Lymphoblastic Leukemia

• Status: Completed
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 80 (actual)
• Sponsor: Novartis Pharmaceuticals · Industry
• Sex: All
• Age: 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 CTL019 in pediatric patients with r/r B-cell ALL.

Detailed description

This was a initially a one cohort, open-label, multi-center, phase II study to determine the efficacy and safety of CTL019 in pediatric patients with r/r B-cell ALL. This main cohort completed enrollment. Two new cohorts were added via an amendment, Cohort 1 for high risk B-cell ALL patients at first relapse, and Cohort 2 for feasibility and safety of CTL019 in high risk B-cell ALL in patients that relapsed \<6 months post allo-HSCT. Due to lack of recruitment, Cohort 1 and Cohort 2 halted recruitment. This decision was not related to any safety issue. The study had the following sequential phases: Screening, Pre-Treatment (Cell Product Preparation \& Lymphodepleting Chemotherapy), Treatment and Primary Follow-up, Secondary Follow-up (if applicable) and Survival Follow-up. The total duration of the study is 5 years from CTL019 cell infusion. Efficacy analyses were performed only on the Main Cohort (n=79) who were infused with tisagenlecleucel. However, the data on disposition and demographics presented in this section includes all patients enrolled to the study (98) and all infused patients (80) (Main Cohort + Cohort 1). No patients were enrolled in Cohort 2.

Conditions

• B-cell Acute Lymphoblastic Leukemia

Interventions

Type	Name	Description
BIOLOGICAL	CTL019	Tisagenlecleucel was administered as a single iv infusion. Dose: 2.0 to 5.0x10\^6 tisagenlecleucel per kg body weight (for patients ≤ 50 kg) or 1.0 to 2.5x10\^8 tisagenlecleucel (for patients \>50 kg).

Timeline

• Start date: 2015-04-08
• Primary completion: 2020-01-21
• Completion: 2022-11-17
• First posted: 2015-05-06
• Last updated: 2024-02-13
• Results posted: 2021-11-22

Locations

23 sites across 11 countries: United States, Australia, Austria, Belgium, Canada, France, Germany, Italy, Japan, Norway, Spain

Regulatory

• FDA-regulated drug study