Study of Efficacy and Safety of Reinfusion of Tisagenlecleucel in Pediatric and Young Adult Patients With Acute Lymphoblastic Leukemia (ALL)

Novartis

Status and phase: Terminated; Phase 2

Conditions: Acute Lymphoblastic Leukemia

Treatments: Biological: Tisagenlecleucel

Study type: Interventional

Funder types: Industry

Identifiers

NCT04225676

CCTL019BUS03

Details and patient eligibility

About

This was a multi-center Phase II study investigating the efficacy and safety of reinfusion of tisagenlecleucel in pediatric and young adult patients with acute lymphoblastic leukemia (ALL) who were treated with tisagenlecleucel and experience B cell recovery.

Full description

This trial was a phase II, open label, multi-center trial to determine the efficacy and safety of tisagenlecleucel re-infusion in pediatric and adolescent young adult (AYA) patients with acute lymphoblastic leukemia (ALL) experiencing loss of B cell aplasia. Loss of B-cell aplasia is defined as: peripheral blood (PB) absolute B lymphocyte count ≥ 50/µL, OR PB B lymphocyte ≥ 10% of the total lymphocytes. B-cell aplasia is defined as PB absolute B lymphocyte count <50/µL.

The study had the following phases for all patients: Screening, Treatment and Follow-up. The total duration of the study was about 12 months. After tisagenlecleucel re-infusion, efficacy was assessed at months 1, 3, 6, and End of Study at which time blood samples were obtained.

The study stopped early due to slow enrollment into the trial. The rate of enrollment made the trial no longer feasible to continue.

The patients were able to voluntarily withdraw from the study for any reason, at any time. Patients who received commercial tisagenlecleucel had to be followed for up to 15 years post-infusion. Patients could have been followed under the Center for International Blood and Marrow Transplant Research (CIBMTR) cellular therapy registry if consented for participation. For patients who do not provide consent for participation in the Center for International Blood and Marrow Transplant Research (CIBMTR) registry, adverse events were to be reported for 15 years or until the patient enrolls in the registry.

Enrollment: 5 patients

Sex: All

Ages: 2 to 25 years old

Volunteers: No Healthy Volunteers

Inclusion criteria

• Signed informed consent must be obtained prior to participation in the study
• Must have an additional dose of unexpired, commercial tisagenlecleucel available and prescribed by a physician in the course of medical practice
• Age up to and including 25 years
• Patients must have CD-19+ Leukemia
• Patients who were previously treated with tisagenlecleucel and present with evidence of B-cell recovery as defined by: Peripheral blood (PB) absolute B lymphocyte count ≥ 50/µL, OR PB B lymphocyte ≥ 10% of the total lymphocytes

Exclusion criteria

• Prior gene therapy other than tisagenlecleucel
• Prior adoptive T cell therapy other than tisagenlecleucel
• Active CNS involvement by malignancy
• Active or latent hepatitis B or active hepatitis C, or any uncontrolled infection at screening
• HIV positive test within 8 weeks of screening

Trial design

Primary purpose: Treatment

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

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5 participants in 1 patient group

Tisagenlecleucel

Experimental group

Description:

Tisagenlecleucel Cell Dispersion for Infusion given once during the study. The approved dose range for tisagenlecleucel is: 0.2 to 5.0×106 CAR positive viable T cells / kg for patients' ≤ 50 kg body weight or 0.1 to 2.5×108 CAR-positive viable T cells for patients \> 50 kg body weight.

Treatment:

Biological: Tisagenlecleucel