Clinical trial NCT04225676: Study of Efficacy and Safety of Reinfusion of Tisagenlecleucel in Pediatric and Young Adult Patients With Acute Lymphoblastic Leukemia (ALL)
Official title: 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 Experiencing Loss of B Cell Aplasia
Study type: INTERVENTIONAL
Overall status: TERMINATED
Phase: PHASE2
Conditions: Acute Lymphoblastic Leukemia
Keywords: CTL019
Kymriah
Tisagenlecleucel
B-Cell Acute Lymphoblastic Leukemia
Leukemia
ALL
Pediatric
Young Adult
Reinfusion
Lead sponsor: Novartis
Sponsor class: INDUSTRY
First posted: 2020-01-13
Enrollment: 5
Sex: ALL
Minimum age (years): 2
Maximum age (years): 25
Standard ages: CHILD
ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief summary: 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.

Detailed 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.

Eligibility criteria: 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

Primary outcomes:
measure: Percentage of Patients Who Establish B Cell Aplasia Within 9 Months of Reinfusion
timeFrame: Post-reinfusion up to 9 months (Day 1 is excluded)
Secondary outcomes:
measure: Complete Response (CR) or Complete Response With Incomplete Blood Count Recovery (CRi) by Day
timeFrame: Post-reinfusion up to 9 months
measure: Participants With an Event
timeFrame: Reinfusion up to 9 months
measure: Overall Survival (OS)
timeFrame: Reinfusion up to 9 months
Other outcomes:
Locations:
city: Los Angeles
country: United States
facility: Childrens Hospital Los Angeles Divisionof Hematology/Oncology
state: California
city: Chicago
country: United States
facility: Ann and Robert H Lurie Childrens Hospital of Chicago
state: Illinois
city: Kansas City
country: United States
facility: Children s Mercy Hospital
state: Missouri
city: Dallas
country: United States
facility: UT Southwestern Medical Center
state: Texas
Study officials:
affiliation: Novartis Pharmaceuticals
name: Novartis Pharmaceuticals
role: STUDY_DIRECTOR
References: