Study of Tirabrutinib vs Rituximab/Temozolomide for PCNSL
Research type
Research Study
Full title
A Phase 3, Multi-regional, Open-label, Randomized Study of Tirabrutinib vs Rituximab and Temozolomide in Participants with Relapsed/Refractory Primary Central Nervous System Lymphoma
IRAS ID
1013300
Contact name
Kathryn Paunicka
Contact email
Sponsor organisation
Ono Pharmaceutical Co., Ltd.
Eudract number
2025-523389-26
Clinicaltrials.gov Identifier
Research summary
Tirabrutinib (also called ONO-4059) is an investigational drug, which means that it has not been approved by the United States (US) Food and Drug Administration (FDA) or some other regulatory agencies. Tirabrutinib is approved in Japan, South Korea, and Taiwan to treat relapsed or refractory (R/R) primary central nervous system lymphoma (PCNSL). PCNSL is a rare cancer that occurs within tissues of the brain, usually without cancerous cell growth in other parts of the body. In people with R/R PCNSL, their condition has not responded to, or has got worse on, their previous treatment. In the US and Europe, there are currently no drug therapies specifically approved for the treatment of PCNSL. Researchers are doing this clinical trial to learn more about how well tirabrutinib works against cancer and how safe it is compared to the combination of rituximab and temozolomide in participants with R/R PCNSL. Rituximab and temozolomide are treatments that are already approved by the US FDA and other regulatory agencies for the treatment of other types of cancers, but not for PCNSL. The combination of rituximab and temozolomide has been assessed in PCNSL in small studies, and researchers think they may be a possible treatment option for PCNSL. Participants will receive either tirabrutinib or a combination of rituximab and temozolomide. Tirabrutinib is made as a tablet that is taken orally (by mouth) on an empty stomach. Participants will receive rituximab through a needle into a vein (IV infusion) and temozolomide as a capsule that is taken orally. Researchers will use a computer program to randomly assign the treatment each participant will receive. Participants assigned to tirabrutinib will take treatment daily in 4-week treatment blocks, called cycles. Participants assigned to rituximab and temozolomide will receive rituximab once a month and temozolomide daily for 5 days for a total of up to 6 cycles.
REC name
South West - Central Bristol Research Ethics Committee
REC reference
26/SW/0026
Date of REC Opinion
2 Apr 2026
REC opinion
Further Information Favourable Opinion