Real-World Evidence of Effectiveness and Safety of Tirabrutinib in Patients with Relapsed or Refractory Primary Central Nervous System Lymphoma in Taiwan: a Nationwide Study

Not yet recruiting

• Conditions: Primary Central Nervous System Lymphoma (PCNSL)

• Registration Number: NCT06714370
• Lead Sponsor: Ono Pharmaceutical Co. Ltd

Brief Summary

The goal of this observational study is to describe the real-world effectiveness and safety of tirabrutinib among relapsed or refractory PCNSL patients in Taiwan.

Detailed Description

Not available

Study Timeline

• Status Verified: 2024-11
• Study First Submitted: 2024-11-05
• Study First Submitted QC: 2024-11-28
• Last Update Submitted: 2024-11-28
• Study Start: 2024-12
• Study First Posted: 2024-12-03
• Last Update Posted: 2024-12-03
• Primary Completion: 2026-12
• Study Completion: 2026-12

Recruitment & Eligibility

• Status: NOT_YET_RECRUITING
• Sex: All
• Target Recruitment: 35

Inclusion Criteria

1. Patient is ≥ 18 years of age.
2. r/r PCNSL patient, who has newly started NHI public reimbursement tirabrutinib from 01 June 2024 to 30 June 2025.
3. Have provided voluntary written consent, directly from the subject, or through his/her legal representative for a patient who has died or lacks the capacity to give informed consent

Note: The NHI reimbursement criteria are listed below for reference purposes only. Subject enrollment depends on whether reimbursement has been received.

1. Histopathologically confirmed large B-cell PCNSL.
2. Patients with relapsed or refractory PCNSL previously treated at least 2 cycles of HD-MTX.
3. Exclude HIV infection
4. Exclude Burkitt lymphoma
5. Exclude patients using chemotherapy or monoclonal antibodies at the initiation of tirabrutinib treatment.

Exclusion Criteria

Not provided

Study & Design

• Study Type: OBSERVATIONAL
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
Objective response rate (ORR)	At least 9 months for each subject	objective response rate is defined as the proportion of subjects who respond either Complete response (CR), Partial response (PR), or unconfirmed Complete response (CRu) to therapy, according to International PCNSL Collaborative Group (IPCG) criteria.

Secondary Outcome Measures

Name	Time	Method
Overall survival (OS)	At least 9 months for each subject	overall survival is defined as time from newly started tirabrutinib treatment to death.
Duration of response (DOR)	At least 9 months for each subject	Duration of response is defined as the time from onset of response\* to disease progression or death, wichever occurs earlier.; \*for patients who achieve complete response (CR), partial response (PR), or unconfirmed complete response (CRu), according to IPCG criteria.
Time-to-treatment response (TTR)	At least 9 months for each subject	Time-to-treatment response was defined as time from newly started tirabrutinib treatment to the first objective tumor response observed for patients who achieved a complete response (CR), partial response (PR), or unconfirmed complete response (CRu), according to IPCG criteria.
Adverse events of special interest (AESIs)	At least 9 months for each subject	Drug-related adverse events for treatment interruption, dose reduction, and discontinuation of tirabrutinib: febrile neutropenia (grade≥3), thrombocytopenia with hemorrhage (grade 3), neutropenia (grade 4), thrombocytopenia (grade 4), interstitial lung disease (grade≥2), skin disorder (grade≥2), hematotoxicity (grade≥3, other than events described above) and nonhematological toxicity (other than interstitial lung disease and skin disorder, grade≥3) (the grading will be according to CTCAE v5.0.) important identified risks: infection, severe skin disorder, bone marrow depression, hypersensitivity, interstitial lung disease (ILD), hepatic dysfunction, hemorrhage, arrhythmia.
Clinical laboratory test	At least 9 months for each subject	Clinical laboratory tests (hematology, blood biochemistry, urinalysis, etc.) will be summarized with dexcriptive statistics. The change from baseline will be presented also, if possible.
Treatment-emergent adverse events (TEAEs)	At least 9 months for each subject	in this study, an AE is defined as any unfavorable or unintended injury/disease or sign (including an abnormal laboratory finding) in a subject administered tirabrutinib, regardless of causality. AE also includes suspected transmission of infectious agents by tirabrutinib. TEAE is defined as an AE that began after the start of tirabrutinib treatment. Concurrent disease at the start of tirabrutinib treatment that has worsened during tirabrutinib treatment for the subject is also included. worsening of the primary disease is not an TEAE. all severity of AEs will be graded according to CTCAE v5.0. All AE that are not considered serious AE are not-serious AE.
Progression-free survival (PFS)	At least 9 months for each subject	Progression-free survival is defined as time from newly started tirabrutinib treatment to either first evidence of progression disease (PD), according to IPCG criteria, or death.