This was a single-arm, multicenter Phase 2 study in Chinese participants with relapsed or refractory Waldenström's macroglobulinemia who exhibited one or more of the criteria for requiring treatment based on consensus guidelines from the Seventh International Workshop on Waldenström's Macroglobulinemia (IWWM). The study comprised an initial screening phase (up to 28 days), a single-arm treatment phase, and a follow-up phase.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
44
Oral administration using 80 mg capsules
Cancer Hospital Chinese Academy of Medical Sciences
Beijing, Beijing Municipality, China
Peking Union Medical College Hospital
Beijing, Beijing Municipality, China
Guangdong Provincial Peoples Hospital
Guangzhou, Guangdong, China
Major Response Rate (MRR) as Assessed by the Independent Review Committee
MRR is defined as the percentage of participants who achieved complete response (CR) + very good partial response (VGPR) + partial response (PR), as assessed by an independent review committee according to modified Owen's criteria
Time frame: Up to approximately 1 year and 9 months
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 an independent review committee using modified Owen's criteria
Time frame: Up to approximately 1 year and 9 months
PFS: Event-free Rate
Progression/death event-free rates were estimated by Kaplan-Meier method with 95% confidence intervals (CIs) estimated using Greenwood's formula
Time frame: Up to approximately 1 year and 9 months
Overall Response Rate (ORR)
ORR is defined as the percentage of participants with a minor, partial, very good partial, and complete response, as assessed by an independent review committee using modified Owen's criteria
Time frame: Up to approximately 1 year and 9 months
Duration of Major Response (DOMR)
DOMR is defined as the time from the date that the major response criteria are first met to the date that progressive disease (PD) is objectively documented or death, whichever comes first, as assessed by an independent review committee using modified Owen's criteria
Time frame: Up to approximately 1 year and 9 months
DOMR: Event-free Rate
DOMR event-free rates were estimated by Kaplan-Meier method with 95% CIs estimated using Greenwood's formula
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Henan Cancer Hospital
Zhengzhou, Henan, China
Tongji Hospital of Tongji Medical College Huazhong University of Science and Technology
Wuhan, Hubei, China
Jiangsu Province Hospital
Nanjing, Jiangsu, China
The First Affiliated Hospital of Soochow University Branch Shizi
Suzhou, Jiangsu, China
Rui Jin Hospital Shanghai Jiao Tong University School of Medicine
Shanghai, Shanghai Municipality, China
West China Hospital, Sichuan University
Chengdu, Sichuan, China
The First Affiliated Hospital, Zhejiang University School of Medicine
Hangzhou, Zhejiang, China
Time frame: Up to approximately 1 year and 9 months
Number of Participants With Resolution of Treatment-precipitating Symptoms
Number of participants with resolution of treatment-precipitating symptoms, defined as any resolution (from "Yes" at baseline to "No" at any postbaseline point onwards during study) of the indications for initiation of therapy in WM signs and symptoms evaluation.
Time frame: Up to approximately 1 year and 9 months
Number of Participants With an Anti-lymphoma Effect
Number of participants with an anti-lymphoma effect, defined as any reduction during the course of study in bone marrow involvement by lymphoplasmacytoid lymphocytes and/or size of lymphadenopathy and/or splenomegaly by computed tomography scan as assessed by an independent review committee; lymphadenopathy is defined as any node with longest diameter (LDi) \> 1.5 cm and splenomegaly is defined as vertical spleen length \> 13 cm
Time frame: Up to approximately 1 year and 9 months
Number of Participants With Adverse Events
Number of participants with treatment-emergent adverse events (TEAEs), grade 3 or higher TEAEs, serious adverse events (SAEs), treatment-related adverse events (AEs), adverse events of special interest, and TEAEs leading to study drug discontinuation, dose reduction and treatment interruption
Time frame: Up to approximately 3 years and 5 months