Tirabrutinib for Central Nervous System Lymphoma

Recruiting at 1 trial location

Overseen By International Clinical Trial Support Desk

Age: 18+

Sex: Any

Trial Phase: Phase 1

Sponsor: Ono Pharmaceutical Co. Ltd

Must not be taking: CYP3A4 inducers, St. John's Wort

Disqualifiers: Liver transplant, Stroke, Infections, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Compensation: Varies

Number of Visits: 73

Duration: 1 day

32 Participants Needed

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests how the drug tirabrutinib behaves in individuals with varying levels of liver function. It focuses on those with central nervous system lymphoma, a cancer affecting the brain and spinal cord. The study includes participants with mild, moderate, or severe liver issues, along with healthy individuals for comparison. Suitable candidates have stable liver conditions, have not undergone a liver transplant, and can swallow pills. As a Phase 1 trial, the research aims to understand how the treatment works in people, offering participants the opportunity to be among the first to receive this new treatment.

Will I have to stop taking my current medications?

Yes, you will need to stop taking any medications, including prescription, over-the-counter, and herbal supplements, at least 14 days before starting the trial.

Is there any evidence suggesting that tirabrutinib is likely to be safe for humans?

Research has shown that tirabrutinib has been safe in past studies. For example, one study found that side effects were usually mild or treatable. Another study highlighted tirabrutinib's long-term benefits and good safety record. While some side effects might occur, they are generally not serious and can be managed. Prospective participants should discuss potential risks with a healthcare provider before joining a clinical trial. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising?

Tirabrutinib is unique because it targets the Bruton’s tyrosine kinase (BTK), which plays a crucial role in the growth and survival of certain cancer cells, including those in central nervous system lymphoma. Unlike traditional chemotherapy, which can affect a wide range of rapidly dividing cells, Tirabrutinib offers a more targeted approach, potentially reducing side effects. Researchers are excited about this treatment because its targeted action may lead to better outcomes for patients with varying liver function, offering tailored options where existing treatments might fall short.

What evidence suggests that tirabrutinib might be an effective treatment for central nervous system lymphoma?

Research shows that tirabrutinib yields promising results for treating central nervous system lymphoma. In earlier studies, about two-thirds of patients saw their cancer respond to the treatment. Nearly half of these patients experienced a complete or almost complete reduction in their cancer symptoms. The treatment's side effects are generally manageable, meaning they are usually tolerable. These findings suggest tirabrutinib could be an effective option for this condition. ^{1 2 4 5 6}

Who Is on the Research Team?

Project Leader

Principal Investigator

Ono Pharmaceutical Co. Ltd

Are You a Good Fit for This Trial?

This trial is for individuals with varying degrees of liver function, from mild to severe impairment, as well as healthy participants. It's designed to understand how a drug called Tirabrutinib behaves in the body in these different groups.

Inclusion Criteria

BMI between 18 and 40 kg/m2, inclusive, at Screening

Estimated glomerular filtration rate (eGFR) of 45 mL/minute/1.73 m2 or higher calculated using the Chronic Kidney Disease Epidemiology Collaboration (CKD-EPI) equation at Screening

I can swallow pills.

Exclusion Criteria

History of Stevens-Johnson syndrome or toxic epidermal necrolysis

Positive results at Screening for human immunodeficiency virus (HIV)

History of hypersensitivity or anaphylaxis to tirabrutinib

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

1-2 weeks

Treatment

Participants receive a single dose of Tirabrutinib 80 mg administered orally on Day 1

1 day

1 visit (in-person)

Follow-up

Participants are monitored for safety and effectiveness after treatment

72 hours

Multiple assessments over 72 hours

What Are the Treatments Tested in This Trial?

Interventions

• Tirabrutinib

Trial Overview The study focuses on Tirabrutinib, a medication potentially used for Central Nervous System Lymphoma. Researchers will compare how this drug moves through and affects people with different levels of liver health.

How Is the Trial Designed?

Group I: Tirabrutinib (Severe Hepatic Impairment) Experimental Treatment 1 Intervention

Participants received a single dose of Tirabrutinib 80 mg administered orally on Day 1.

Group II: Tirabrutinib (Normal Hepatic Function) Experimental Treatment 1 Intervention

Participants received a single dose of Tirabrutinib 80 mg administered orally on Day 1.

Group III: Tirabrutinib (Moderate Hepatic Impairment) Experimental Treatment 1 Intervention

Participants received a single dose of Tirabrutinib 80 mg administered orally on Day 1.

Group IV: Tirabrutinib (Mild Hepatic Impairment) Experimental Treatment 1 Intervention

Participants received a single dose of Tirabrutinib 80 mg administered orally on Day 1.

Who Is Running the Clinical Trial?

Ono Pharmaceutical Co. Ltd

Lead Sponsor

Trials

182

Recruited

97,100+

Shinji Takai

Ono Pharmaceutical Co. Ltd

Chief Medical Officer since 2023

MD from an unspecified institution

Gyo Sagara

Ono Pharmaceutical Co. Ltd

Chief Executive Officer since 2024

PhD in Pharmaceutical Sciences from Kyoto University

Citations

1. ascopubs.org ascopubs.org/doi/10.1200/JCO.2025.43.16_suppl.2019

Tirabrutinib for the treatment of relapsed or refractory ... Conclusions: With an ORR of 66.7%, CR/CRu rate of 43.8%, median DOR of 9.3 mo, and a manageable safety profile, the PROSPECT trial supports ...

2. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC11059299/

Three-year follow-up analysis of phase 1/2 study on ... The results demonstrated the long-term clinical benefit of tirabrutinib, with deep and durable response in a subset of patients and acceptable safety profile.

3. ashpublications.org ashpublications.org/blood/article/144/Supplement%201/572/530380/Interim-Analysis-Data-from-Multi-Center

Interim Analysis Data from Multi-Center Retrospective ... Conclusion: These data demonstrate the effectiveness and safety of tirabrutinib in a large number of patients in clinical practice, and the ...

4. ono-pharma.com ono-pharma.com/en/news/20250528.html

ono pharmaceutical co., ltd. Exploratory endpoints included median overall survival, which was not reached, and median progression-free survival, which was 6.0 months.2

5. onclive.com onclive.com/view/tirabrutinib-for-the-treatment-of-relapsed-or-refractory-primary-central-nervous-system-lymphoma-efficacy-and-safety-from-the-phase-ii-prospect-study

Tirabrutinib for the Treatment of Relapsed or Refractory ... Tirabrutinib monotherapy yields a 66.7% overall response rate and a manageable safety profile in patients with relapsed or refractory primary central nervous ...

6. clinicaltrials.gov clinicaltrials.gov/study/NCT04947319

Study of Tirabrutinib (ONO-4059) in Patients With Primary ... This study will evaluate the efficacy, safety, and pharmacokinetics of tirabrutinib monotherapy in patients with relapsed or refractory PCNSL (Part A),