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A Study to Investigate Efficacy and Safety of BCL2 Inhibitor Sonrotoclax as Monotherapy and in Combination With Zanubrutinib in Adults With Waldenström Macroglobulinemia

Phase 2
Recruiting
Conditions
Waldenstrom's Macroglobulinemia Refractory
Waldenstrom's Macroglobulinemia Recurrent
Waldenstrom Macroglobulinemia
Interventions
Registration Number
NCT05952037
Lead Sponsor
BeiGene
Brief Summary

This study will evaluate the safety and efficacy of the BCL2 inhibitor sonrotoclax (BGB-11417) in participants with relapsed/refractory Waldenström's Macroglobulinemia (R/R WM) and in combination with zanubrutinib in adult participants with previously untreated WM.

Detailed Description

This study will test whether sonrotoclax (BGB-11417) can be used to improve outcomes in participants with Waldenström's Macroglobulinemia (WM) both when used alone in those who have not responded well to conventional treatments and when used in combination with zanubrutinib in those who have not yet received treatment. The main goals of the study are to determine how many participants 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 105 participants. All participants will receive sonrotoclax 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.

Recruitment & Eligibility

Status
RECRUITING
Sex
All
Target Recruitment
105
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).
  • For Cohorts 1-3, refractory or relapsed disease 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.
  • For Cohort 4, patients must not have received prior therapy for WM (except for plasmapheresis).
  • 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.

Study & Design

Study Type
INTERVENTIONAL
Study Design
PARALLEL
Arm && Interventions
GroupInterventionDescription
Cohort 1SonrotoclaxParticipants with R/R disease to both Bruton tyrosine kinase (BTK) inhibitor and anti-CD20 antibody-based systemic therapy containing chemotherapy or proteasome inhibitor will receive sonrotoclax at a standard dose, given orally once daily.
Cohort 2SonrotoclaxParticipants with R/R disease to anti-CD20 antibody-based systemic therapy containing chemotherapy or proteasome inhibitor and were intolerant to BTK inhibitor will receive sonrotoclax at a standard dose, given orally once daily.
Cohort 3SonrotoclaxParticipants with R/R disease to a BTK inhibitor treatment and are unsuitable for chemoimmunotherapy will receive sonrotoclax at a standard dose, given orally once daily.
Cohort 4SonrotoclaxParticipants with previously untreated WM will receive sonrotoclax and zanubrutinib combination therapy for a fixed duration.
Cohort 4ZanubrutinibParticipants with previously untreated WM will receive sonrotoclax and zanubrutinib combination therapy for a fixed duration.
Primary Outcome Measures
NameTimeMethod
Cohort 1: Major Response Rate (MRR)Up to approximately 4 years

MRR is defined as the percentage of participants achieving partial response (PR) or better, as assessed by the Independent Review Committee (IRC) per the 11th International Workshop on Waldenström Macroglobulinemia (IWWM-11) WM response criteria.

Secondary Outcome Measures
NameTimeMethod
Cohorts 1, 2, and 3: Time to major response as assessed by the IRCUp to approximately 5 years

Time to major response is defined as the time from start of study treatment to the first documentation of major response.

All Cohorts: Time to major response as assessed by the investigatorUp to approximately 5 years

Time to major response is defined as the time from start of study treatment to the first documentation of major response.

Cohorts 1, 2, and 3: Overall Survival (OS)Up to approximately 5 years

OS is defined as the time from first study drug administration to the date of death due to any cause.

Cohort 4: Time to next treatmentUp to approximately 5 years

Defined as the time from the start of treatment to the start of first subsequent therapy for WM.

Number of participants reporting adverse eventsUp to approximately 5 years

Number of participants with treatment-emergent adverse events (TEAEs) and serious adverse events (SAEs), including laboratory abnormalities, physical examination results, and vital signs.

Cohorts 2 and 3: MRR as assessed by the IRCUp to approximately 5 years

MRR is defined as the percentage of participants achieving PR or better.

All Cohorts: MRR as assessed by the InvestigatorUp to approximately 5 years

MRR is defined as the percentage of participants achieving PR or better.

Cohorts 1, 2, and 3: Duration of Major Response (DoMR) as assessed by the IRCUp to approximately 5 years

DoMR is defined as the time from first determination of major response until first documentation of progression or death, whichever occurs first.

All Cohorts: DoMR as assessed by the InvestigatorUp to approximately 5 years

DoMR is defined as the time from first determination of major response until first documentation of progression or death, whichever occurs first.

Cohorts 1, 2, and 3: Complete Response (CR) + Very Good Partial Response (VGPR) as assessed by the IRCUp to approximately 5 years

CR + VGPR is defined as the percentage of participants who achieve CR or VGPR.

All Cohorts: CR + VGPR as assessed by the InvestigatorUp to approximately 5 years

CR + VGPR is defined as the percentage of participants who achieve CR or VGPR.

Cohorts 1, 2, and 3: Overall Response Rate (ORR) as assessed by the IRCUp to approximately 5 years

ORR is defined as the percentage of participants with minor response (MR) or better.

All cohorts: ORR as assessed by the investigatorUp to approximately 5 years

ORR is defined as the percentage of participants with MR or better.

Cohorts 1, 2, and 3: Duration of Response (DOR) as assessed by the IRCUp to approximately 5 years

DOR is defined as the time from first determination of response until first documentation of progression or death, whichever occurs first.

All Cohorts: DOR as assessed by the investigatorUp to approximately 5 years

DOR is defined as the time from first determination of response until first documentation of progression or death, whichever occurs first.

Cohorts 1, 2, and 3: Progression-Free Survival (PFS)Up to approximately 5 years

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.

All Cohorts: Change from Baseline in Health-Related Quality of Life (HRQoL): NFLymSI-18 Disease-related Symptoms-Physical and Treatment-Related Side Effects SubscalesBaseline and approximately months 7, 13, 19, and 25

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.

Trial Locations

Locations (72)

City of Hope National Medical Center

🇺🇸

Duarte, California, United States

Colorado Blood Cancer Institute

🇺🇸

Denver, Colorado, United States

Medstar Georgetown University Hospital

🇺🇸

Washington, District of Columbia, United States

University of Miami

🇺🇸

Miami, Florida, United States

Northwestern Medicine Cancer Center

🇺🇸

Warrenville, Illinois, United States

Mission Cancer and Blood

🇺🇸

Waukee, Iowa, United States

University of Maryland Greenebaum Comprehensive Cancer Center

🇺🇸

Baltimore, Maryland, United States

Dana Farber Cancer Institute

🇺🇸

Boston, Massachusetts, United States

Mayo Clinic Rochester

🇺🇸

Rochester, Minnesota, United States

Hattiesburg Hematology and Oncology Clinic

🇺🇸

Hattiesburg, Mississippi, United States

Memorial Sloan Kettering Cancer Center Mskcc

🇺🇸

New York, New York, United States

Atrium Health Levine Cancer Institute (Lci)

🇺🇸

Charlotte, North Carolina, United States

Ohio State University Comprehensive Cancer Center

🇺🇸

Columbus, Ohio, United States

Ut Southwestern Medical Center

🇺🇸

Dallas, Texas, United States

Huntsman Cancer Institute

🇺🇸

Salt Lake City, Utah, United States

Virginia Commonwealth University Massey Cancer Center

🇺🇸

Richmond, Virginia, United States

University of Washington

🇺🇸

Seattle, Washington, United States

Concord Repatriation General Hospital

🇦🇺

Concord, New South Wales, Australia

Genesiscare North Shore

🇦🇺

St Leonards, New South Wales, Australia

Princess Alexandra Hospital

🇦🇺

Woolloongabba, Queensland, Australia

Flinders Medical Centre

🇦🇺

Bedford PK, South Australia, Australia

Monash Health

🇦🇺

Clayton, Victoria, Australia

St Vincents Hospital Melbourne

🇦🇺

Fitzroy, Victoria, Australia

Linear Clinical Research

🇦🇺

Nedlands, Western Australia, Australia

Royal Perth Hospital

🇦🇺

Perth, Western Australia, Australia

Cross Cancer Institute

🇨🇦

Edmonton, Alberta, Canada

Lions Gate Hospital Chemotherapy Clinic

🇨🇦

North Vancouver, British Columbia, Canada

Qeii Health Science Center

🇨🇦

Halifax, Nova Scotia, Canada

Princess Margaret Cancer Centre

🇨🇦

Toronto, Ontario, Canada

The First Affiliated Hospital of Xiamen University

🇨🇳

Xiamen, Fujian, China

Guangdong Provincial Peoples Hospital

🇨🇳

Guangzhou, Guangdong, China

Henan Cancer Hospital

🇨🇳

Zhengzhou, Henan, China

Tongji Hospital of Tongji Medical College Huazhong University of Science and Technology

🇨🇳

Wuhan, Hubei, China

Yancheng First Peoples Hospital

🇨🇳

Yancheng, Jiangsu, China

Shengjing Hospital of China Medical Universityshenbei Branch

🇨🇳

Shenyang, Liaoning, China

The Affiliated Hospital of Qingdao University Branch South

🇨🇳

Qingdao, Shandong, China

Affiliated Zhongshan Hospital of Fudan University

🇨🇳

Shanghai, Shanghai, China

The Third Peoples Hospital of Datong

🇨🇳

Datong, Shanxi, China

Institute of Hematology and Hospital of Blood Disease

🇨🇳

Tianjin, Tianjin, China

The First Affiliated Hospital, Zhejiang University School of Medicine

🇨🇳

Hangzhou, Zhejiang, China

The First Hospital of Jiaxing

🇨🇳

Jiaxing, Zhejiang, China

The First Affiliated Hospital of Wenzhou Medical University

🇨🇳

Wenzhou, Zhejiang, China

Centre Hospitalier Universitaire Damiens Hopital Sud

🇫🇷

Amiens Cedex, France

Chu Clermont Ferrand Therapie Cellulaire and Hematolo

🇫🇷

Clermontferrand, France

Institut Paoli Calmettes

🇫🇷

Marseille, France

Hopital de La Pitie Salpetriere

🇫🇷

Paris, France

Chu Hopital Lyon Sud

🇫🇷

PierreBenite, France

Hopital Robert Debre

🇫🇷

Reims, France

General Hospital of Athens Alexandra

🇬🇷

Athens, Greece

Irccs Azienda Ospedaliero Universitaria Bologna

🇮🇹

Bologna, Italy

Azienda Socio Sanitaria Territoriale Degli Spedali Civili Di Brescia

🇮🇹

Brescia, Italy

Istituto Europeo Di Oncologia

🇮🇹

Milano, Italy

Azienda Socio Sanitaria Territoriale Grande Ospedale Metropolitano Niguarda

🇮🇹

Milano, Italy

Irccs Policlinico San Matteo, Universita Degli Studi Di Pavi

🇮🇹

Pavia, Italy

Fondazione Policlinico Universitario Agostino Gemelli

🇮🇹

Roma, Italy

Azienda Sanitaria Universitaria Friuli Centrale Presidio Ospedaliero Universitario Santa Maria Del

🇮🇹

Udine, Italy

Hospital Universitario Vall Dhebron

🇪🇸

Barcelona, Spain

Institut Catala Doncologia

🇪🇸

Barcelona, Spain

Hospital Clinic de Barcelona

🇪🇸

Barcelona, Spain

Hospital Universitario Fundacion Jimenez Diaz

🇪🇸

Madrid, Spain

Hospital Universitario de Salamanca

🇪🇸

Salamanca, Spain

Hospital Universitario Virgen Del Rocio

🇪🇸

Sevilla, Spain

Hospital Universitari Mutua Terrassa

🇪🇸

Terrassa, Spain

University Hospitals Dorset

🇬🇧

Bournemouth, United Kingdom

Beatson West of Scotland Cancer Centre

🇬🇧

Glasgow, United Kingdom

Churchill Hospital Oxford University Hospital Nhs Trust

🇬🇧

Headington, United Kingdom

Nhs Highland

🇬🇧

Inverness, United Kingdom

St Jamess University Hospital

🇬🇧

Leeds, United Kingdom

University College Hospital

🇬🇧

London, United Kingdom

The Christie Nhs Foundation Trust Manchester

🇬🇧

Manchester, United Kingdom

Plymouth Hospitals Nhs Trust

🇬🇧

Plymouth, United Kingdom

Royal Marsden Nhs Foundation Royal Marsden Hospital

🇬🇧

Sutton, United Kingdom

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