NCT05734495

Pirtobrutinib and Venetoclax in Waldenström Macroglobulinemia

A Phase II Study Evaluating Venetoclax and Pirtobrutinib in Previously Treated Waldenström Macroglobulinemia

• Status: Recruiting
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 42 (estimated)
• Sponsor: Dana-Farber Cancer Institute · Academic / Other
• Sex: All
• Age: 18 Years
• Healthy volunteers: Not accepted

Summary

This study is being done to examine the safety and effectiveness of pirtobrutinib combined with venetoclax as a possible treatment for participants with Waldenström Macroglobulinemia (WM). The names of the study drugs involved in this study are: * Pirtobrutinib (a Noncovalent Bruton Tyrosine Kinase (BTK) inhibitor) * Venetoclax (a BCL2 inhibitor)

Detailed description

This is a single-arm, open-label, Phase II study to evaluate the safety and efficacy of venetoclax combined with pirtobrutinib (VEN-P) in participants with symptomatic Waldenström Macroglobulinemia (WM) with previously treated disease. Pirtobrutinib blocks a type of protein called Bruton Tyrosine Kinase (BTK) that helps cells live and grow. Venetoclax blocks BCL-2, a protein essential for WM cells' survival. The U.S. Food and Drug Administration (FDA) has not approved pirtobrutinib for Waldenström Macroglobulinemia (WM), but it has been approved for other uses. The FDA has not approved venetoclax for Waldenström Macroglobulinemia (WM), but it has been approved for other uses. The FDA has not approved the combination of pirtobrutinib and venetoclax as a treatment for any disease. Study procedures include screening for eligibility, treatment visits, CT scans, blood tests, and bone marrow aspirates and biopsies. Participants will receive study treatment for up to 2 years and will be followed for up to 4 years or until they start a new therapy. It is expected that about 42 people will take part in this research study. Eli Lilly supports this research study by providing study drug pirtobrutinib and funding.

Conditions

• Waldenstrom Macroglobulinemia

Interventions

Type	Name	Description
DRUG	Pirtobrutinib	Noncovalent Bruton Tyrosine Kinase (BTK) inhibitor, tablet taken orally.
DRUG	Venetoclax	Small-molecule B-cell lymphoma-2 (Bcl-2) family inhibitor, tablet taken orally.

Timeline

• Start date: 2023-05-02
• Primary completion: 2029-01-25
• Completion: 2033-01-25
• First posted: 2023-02-21
• Last updated: 2025-05-11

Locations

2 sites across 1 country: United States

Regulatory

• FDA-regulated drug study