Study to Evaluate the Efficacy and Safety of BGB-11417 in Participants With Waldenström's Macroglobulinemia

Waldenstrom Macroglobulinemia Clinical Trial

Official title:

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

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT05952037
• Other study ID #: BGB-11417-203
• Secondary ID: U1111-1291-45242
• Status: Recruiting
• Phase: Phase 2
• Start date: September 28, 2023
• Est. completion date: September 2028

Study information

• Verified date: April 2024
• Source: BeiGene
• Contact: Study Director
• Phone: 1-877-828-5568
• Email: clinicaltrials[@]beigene.com
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial Summary

This study will evaluate the safety and efficacy of the BCL2 inhibitor BGB-11417 in participants with relapsed/refractory Waldenström's Macroglobulinemia (R/R WM) in 3 cohorts.

Description:

This study will test whether BGB-11417 can be used to improve outcomes in participants with Waldenström's Macroglobulinemia (WM) who have not responded well to conventional treatments. The main goals of the study are to determine how many participants may no longer have evidence of cancer or have some improvement in the signs and symptoms of cancer after treatment, and to determine what adverse events, or side effects, participants might experience. BCL2 is a key protein involved in cell death, and abnormal levels of BCL2 are associated with many cancers. Blocking the action of BCL2 proteins is a promising approach with potential therapeutic benefits in participants with different types of cancers, including WM. This study will enroll approximately 85 patients. All patients will receive BGB-11417 orally as a tablet. The study will take place at multiple centers worldwide. The overall time to participate in this study is approximately 5 years. Treatments will continue until participants experience worsening disease status, too many side effects, or withdraw consent.

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 85
• Est. completion date: September 2028
• Est. primary completion date: November 2027
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years and older

Eligibility

Inclusion Criteria:

• Clinical and definitive histologic diagnosis of WM.
• Meeting = 1 criterion for treatment according to consensus panel criteria from the 2nd International Workshop on Waldenström's Macroglobulinemia (IWWM).
• Refractory or relapsed disease to the most recent therapy at study entry unless participants had intolerance to the most recent therapy. Refractory disease is defined as not attaining at least a major response, or progressing while on or within 6 months of completing therapy. Relapsed disease is defined as attaining at least a major response to therapy and meeting the criteria for disease progression beyond 6 months after completing therapy.
• Adequate organ function.

Exclusion Criteria:

• Central nervous system (CNS) involvement by WM.
• Transformation to aggressive lymphoma, such as diffuse large B-cell lymphoma.
• History of other malignancies = 2 years before study entry.
• Uncontrolled active systemic infection or recent infection requiring parenteral antimicrobial therapy that was completed = 14 days before the first dose of the study drug. Note: Other protocol defined Inclusion/Exclusion criteria may apply.

Related Conditions & MeSH terms

• Waldenstrom Macroglobulinemia
• Waldenstrom's Macroglobulinemia Recurrent
• Waldenstrom's Macroglobulinemia Refractory

Intervention

Drug:
• BGB-11417
Administered orally as a tablet.

Locations

Country	Name	City	State
Australia	Flinders Medical Centre	Bedford PK	South Australia
Australia	Princess Alexandra Hospital	Brisbane	Queensland
Australia	Monash Health	Clayton	Victoria
Australia	Concord Repatriation General Hospital	Concord	New South Wales
Australia	St Vincents Hospital Melbourne	Fitzroy	Victoria
Australia	Genesiscare North Shore	St Leonards	New South Wales
China	Guangdong Provincial Peoples Hospital	Guangzhou	Guangdong
China	The First Affiliated Hospital, Zhejiang University School of Medicine	Hangzhou	Zhejiang
China	The First Hospital of Jiaxing	Jiaxing	Zhejiang
China	The Affiliated Hospital of Qingdao University Branch South	Qingdao	Shandong
China	Affiliated Zhongshan Hospital of Fudan University	Shanghai	Shanghai
China	Shengjing Hospital Affiliated of China Medical University	Shenyang	Liaoning
China	Institute of Hematology and Hospital of Blood Disease	Tianjin	Tianjin
China	The First Affiliated Hospital of Wenzhou Medical University	Wenzhou	Zhejiang
China	Tongji Hospital of Tongji Medical College Huazhong University of Science and Technology	Wuhan	Hubei
China	Henan Cancer Hospital	Zhengzhou	Henan
France	Chu Clermont Ferrand Therapie Cellulaire and Hematolo	Clermont Ferrand
France	Institut Paoli Calmettes	Marseille
Spain	Hospital Universitario Fundacion Jimenez Diaz	Madrid
Spain	Hospital Universitario Virgen Del Rocio	Sevilla
United Kingdom	Churchill Hospital Oxford University Hospital Nhs Trust	Headington
United States	University of Maryland Greenebaum Comprehensive Cancer Center	Baltimore	Maryland
United States	Dana Farber Cancer Institute	Boston	Massachusetts
United States	Ohio State University Comprehensive Cancer Center	Columbus	Ohio
United States	Colorado Blood Cancer Institute	Denver	Colorado
United States	Mission Cancer and Blood	Des Moines	Iowa
United States	City of Hope National Medical Center	Duarte	California
United States	Hattiesburg Hematology and Oncology Clinic	Hattiesburg	Mississippi
United States	University of Miami	Miami	Florida
United States	Memorial Sloan Kettering Cancer Center Mskcc	New York	New York
United States	Huntsman Cancer Institute	Salt Lake City	Utah

Sponsors (1)

Lead Sponsor	Collaborator
BeiGene

Countries where clinical trial is conducted

United States,  Australia,  China,  France,  Spain,  United Kingdom, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Major Response Rate (MRR) in Cohort 1	MRR is defined as the percentage of participants who achieved complete response (CR), very good partial response (VGPR), or partial response (PR), as assessed by the Independent Review Committee (IRC).	Up to approximately 4 years
Secondary	MRR in Cohorts 1, 2, and 3	MRR is defined as the percentage of participants who achieved complete response (CR), very good partial response (VGPR), or partial response (PR), as assessed by the investigator (Cohorts 1, 2, and 3) and by the IRC (Cohorts 2 and 3).	Up to approximately 5 years
Secondary	Duration of Response (DOR)	DOR is defined as the time from the date that response criteria are first met to the date that progressive disease is objectively documented or death, whichever comes first, as assessed by the IRC and by the investigator.	Up to approximately 5 years
Secondary	CR + VGPR rate	CR + VGPR is defined as the percentage of participants who achieve CR or VGPR, as assessed by the IRC and by the investigator.	Up to approximately 5 years
Secondary	Overall Response Rate (ORR)	ORR is defined as the percentage of participants with minor response (MR) or better, as assessed by the IRC and by the investigator.	Up to approximately 5 years
Secondary	Progression-Free Survival (PFS)	PFS is defined as the time from first dose until first documentation of progression or death, whichever comes first, as assessed by the IRC and by the investigator.	Up to approximately 5 years
Secondary	Time to major response	Time to major response is defined as the time from start of study treatment to the first documentation of major response, as assessed by the IRC and by the investigator.	Up to approximately 5 years
Secondary	Overall Survival (OS)	OS is defined as the time from first study drug administration to the date of death due to any cause.	Up to approximately 5 years
Secondary	Number of participants reporting adverse events	Number of participants with treatment-emergent adverse events (TEAEs) and serious adverse events (SAEs), including laboratory abnormalities, physical examination results, and vital signs.	Up to approximately 5 years
Secondary	Health-Related Quality of Life (HRQoL): NFLymSI-18	HRQoL based on participant-reported outcomes using National Comprehensive Cancer Network/Functional Assessment of Cancer Therapy Lymphoma Cancer Symptom Index - 18 Item (NFLymSI-18) Version 4. The questionnaire contains 18 items, each of which utilizes a Likert scale with 5 possible responses ranging from 0 'Not at all' to 4 'Very much' and is divided into a total score.	Up to approximately 5 years