Long-term Follow up Local Registry Study of Kymriah in South Korea

Not yet recruiting

• Conditions: B-Cell Acute Lymphoblastic Leukemia, Diffuse Large B-Cell Lymphoma, Follicular Lymphoma

• Registration Number: NCT06785818
• Lead Sponsor: Novartis Pharmaceuticals

Brief Summary

This study is multicenter, primary data collection, non-interventional registry study to assess long-term safety, secondary malignancy risk, and effectiveness of tisagenlecleucel in patients with B-cell malignancies in a routine clinical practice setting in Korea.

Detailed Description

This study will inform on long-term real-world safety and effectiveness of tisagenlecleucel. The primary objective is to evaluate the long-term safety and the risk of secondary malignancies in patients with B lymphocyte malignancies treated with tisagenlecleucel in a real-world setting. The main secondary objective is to evaluate the longterm effectiveness of tisagenlecleucel.

All participants enrolled in this study will be followed up for 15 years from the time of Kymriah® infusion.

Study Timeline

• Study First Submitted: 2025-01-15
• Study First Submitted QC: 2025-01-15
• Study First Posted: 2025-01-21
• Status Verified: 2025-03
• Last Update Submitted: 2025-03-04
• Last Update Posted: 2025-03-05
• Study Start: 2025-04-10
• Primary Completion: 2039-12-30
• Study Completion: 2039-12-30

Recruitment & Eligibility

• Status: NOT_YET_RECRUITING
• Sex: All
• Target Recruitment: 500

Inclusion Criteria

1. Patients who receive tisagenlecleucel infusion in the commercial setting, treated under a managed access program or other pathway, e.g., when product was manufactured for the commercial setting but turned out to be out of specification (OOS).
2. Consented to data collection.

Exclusion Criteria

1. Patients who are enrolled or will be enrolled in the Novartis long term follow-up protocol CCTL019A2205B.

Study & Design

• Study Type: OBSERVATIONAL
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
The type and frequency of AEs, ADRs, SAEs, SADRs, UAEs, UADRs, USAEs, USADRs, AESI	Up to 15 years post-infusion	The type and frequency of adverse event (AE)/adverse drug reaction (ADR)/ serious adverse event (SAE)/serious adverse drug reaction (SADR)/ unexpected adverse event (UAE)/unexpected adverse drug reaction (UADR)/ unexpected serious adverse event (USAE)/unexpected serious adverse drug reaction (USADR)/ adverse event of special interest (AESI)
Identify participants for chimeric antigen receptor (CAR) transgene detection and/or CAR surface expression (if applicable).	Up to 15 years post-infusion	When applicable, identify patients for CAR transgene detection and/or CAR surface expression by quantitative polymerase chain reaction (q-PCR), in-situ hybridization, flow cytometry and/or immunohistochemistry (IHC), whichever testing is appropriate, in relevant samples (blood, bone marrow, etc.)
Identify presence of replication competent lentivirus (RCL) in blood or tissues	Up to 15 years post-infusion	Replication competent lentiviruses (RCL) are virus particles capable of infecting cells and replicating to produce additional infectious particles.

Secondary Outcome Measures

Name	Time	Method
B-Cell Acute Lymphoblastic Leukemia - Overall response rate (ORR)	Up to 15 years post-infusion	The overall response rate (ORR) is defined as the percentage of subjects with a best overall disease response of complete response (CR) or Complete remission with incomplete blood count recovery (CRi). For ALL (Acute Lymphoblastic Leukemia), complete remission is defined as: less than 5% blasts in marrow, less than 1% blasts in blood and no EM disease.
B-Cell Acute Lymphoblastic Leukemia - Duration of response (DOR)	Up to 15 years post-infusion	Duration of Response (DoR) is defined as the time from first documented evidence of response (the first response prior to confirmation) until time of documented disease progression or death due to any cause, whichever was first.
B-Cell Acute Lymphoblastic Leukemia - Relapse-free survival (RFS)	Up to 15 years post-infusion	RFS is defined as the time from the date of first dose of the study treatment to the date of the first documented disease recurrence or death due to any cause whichever comes first.
B-Cell Acute Lymphoblastic Leukemia - Event-free survival (EFS)	Up to 15 years post-infusion	Event free survival is the time from the date of start of treatment to the earliest of the following: * Death from any cause after remission; * Relapse; * Treatment failure (failure to achieve remission, including death without remission)
B-Cell Acute Lymphoblastic Leukemia - Proportion of patients with minimal residual disease (MRD) negative status in bone marrow who achieve a best overall response (BOR) of CR or CRi	Up to 15 years post-infusion	MRD is a term used to describe a very small number of cancer cells that remain in the body during or after treatment. MRD is defined as positive by immunophenotype if 1/1000 cells is positive.
Diffuse Large B-Cell Lymphoma - Overall response rate (ORR)	Up to 15 years post-infusion	The overall response rate (ORR) is defined as the percentage of subjects with a best overall disease response of complete response (CR) or Partial response (PR)
Diffuse Large B-Cell Lymphoma - DOR	Up to 15 years post-infusion	Duration of Response (DoR) is defined as the time from first documented evidence of response (the first response prior to confirmation) until time of documented disease progression or death due to any cause, whichever was first.
Diffuse Large B-Cell Lymphoma - RFS	Up to 15 years post-infusion	RFS is defined as the time from the date of first dose of the study treatment to the date of the first documented disease recurrence or death due to any cause whichever comes first.
Diffuse Large B-Cell Lymphoma - Progression-free survival (PFS)	Up to 15 years post-infusion	Progression-free survival is defined as the time from the date of start of treatment to the date of the first documented progression or death due to any cause whichever comes first.
Diffuse Large B-Cell Lymphoma - Overall survival (OS)	Up to 15 years post-infusion	Overall survival is the time from date of start of treatment to the date of death due to any reason.
Follicular Lymphoma - ORR	Up to 15 years post-infusion	The overall response rate (ORR) is defined as the percentage of subjects with a best overall disease response of complete response (CR) or Partial response (PR)
Follicular Lymphoma -Complete response rate (CRR)	Up to 15 years post-infusion	Complete response rate is the proportion of patients with a complete disease response, which is defined as the best disease response recorded from date of start of treatment until progressive disease or start of new anticancer therapy, whichever comes first.
Follicular Lymphoma - DOR	Up to 15 years post-infusion	Duration of Response (DoR) is defined as the time from first documented evidence of response (the first response prior to confirmation) until time of documented disease progression or death due to any cause, whichever was first.
Follicular Lymphoma - RFS	Up to 15 years post-infusion	RFS is defined as the time from the date of first dose of the study treatment to the date of the first documented disease recurrence or death due to any cause whichever comes first.
Follicular Lymphoma - PFS	Up to 15 years post-infusion	Progression-free survival is defined as the time from the date of start of treatment to the date of the first documented progression or death due to any cause whichever comes first.
Follicular Lymphoma - Overall survival (OS)	Up to 15 years post-infusion	Overall survival is the time from date of start of treatment to the date of death due to any reason.
Frequency and rate of pregnancy outcomes	Up to 15 years post-infusion	pregnancy outcomes: * Live birth, at term (presence or absence of congenital abnormality); * Live birth, premature (presence or absence of congenital abnormality); * Intrauterine fetal death; * Spontaneous abortion; * Elective abortion; * Unknown