An Open-Label, Multicenter Phase 2 Study to Evaluate the Efficacy and Safety of BCL2 Inhibitor BGB-11417 in Patients With Relapsed/Refractory Waldenström’s Macroglobulinemia
Recipient organisationNIHR Biomedical Research Centre at the Royal Marsden and the ICRSource-published name: NIHR Royal Marsden Biomedical Research Centre
NIHR supportRecorded as supported by this research centre
PeriodMar 2025 — Jan 2029
In plain English
AI plain-English summary
A new drug, BGB-11417, is being tested in 85 patients whose Waldenström’s Macroglobulinemia has stopped responding to existing treatments. Waldenström’s Macroglobulinemia is a rare, slow-growing blood cancer that causes the body to produce too much of a large protein, thickening the blood and leading to fatigue, bleeding problems, and nerve damage. When the cancer returns or resists standard therapies, patients have few options. This study is the first to test BGB-11417—a drug that blocks the BCL2 protein, which cancer cells rely on to avoid dying—specifically in this disease. If the drug proves safe and effective, it could offer a new treatment line for patients who have run out of options. The study also tracks quality of life through patient questionnaires, ensuring that any benefit is not overshadowed by side effects. Because the drug is taken as a daily pill, it could simplify treatment compared to intravenous chemotherapy. The results will determine whether larger trials are warranted, potentially changing the standard of care for this hard-to-treat cancer.
View original technical description
This is an open-label study, which means that the participants, study doctor, and the research team know that the participants are receiving study medication and know participants are receiving which doses of study medication. This is a multicentre and Phase 2 study of BGB-11417.This study will look at the safety and effectiveness of an investigational anticancer medication currently known as BGB-11417 in the treatment of Waldenström’s Macroglobulinemia (WM). The study will also assess if BGB11417 is safe and well tolerated by participants and how BGB-11417 may interact and/or stay in participant’s blood after they take it. The study will evaluate how BGB-11417 impact quality of life by answers collected by participant reported outcomes questionnaires.The study is composed of a screening period (up to 28 days), a treatment period, and a follow-up period. After the screening period, eligible patients will receive BGB-11417 at 320 mg once daily after an 8-day ramp-up period. Participant will begin study treatment with at a low dose and study doctor will gradually increase the dose over 8 days (ramp-up scheme) until Cycle 1 Day 1. Each treatment cycle is 28 days in duration with Cycle 1 Day 1 starting on the day participant receive the first target dose of 320 mg. After treatment discontinuation, patients will have an End of Treatment (EOT) Visit followed by a Safety Follow-up Visit.The research study is conducted and funded by BeiGene, Ltd and approximately 85 patients will be enrolled in this global study.
Plain English summaries and category classifications on this site are generated by AI and may not perfectly reflect the original research.
Is something wrong? Let us know