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A Study of Ibrutinib (PCI-32765) in Chinese Participants With Relapse or Refractory Waldenstrom's Macroglobulinemia (WM)

Phase 4
Completed
Conditions
Waldenstrom Macroglobulinemia
Interventions
Registration Number
NCT04042376
Lead Sponsor
Janssen Research & Development, LLC
Brief Summary

The purpose of this study is to evaluate the efficacy of ibrutinib based on overall response rate (ORR) (partial response \[PR\] or better) by investigator assessment per the modified Consensus Response Criteria from the Sixth International Workshop on Waldenstrom's Macroglobulinemia (IWWM) (NCCN 2019), in Chinese participants with relapsed or refractory waldenstrom's macroglobulinemia.

Detailed Description

Not available

Recruitment & Eligibility

Status
COMPLETED
Sex
All
Target Recruitment
17
Inclusion Criteria
  • Men and women greater than or equal to (>=) 18 years of age
  • Eastern Cooperative Oncology Group (ECOG) less than or equal to (<=) 2
  • Previously received at least one prior therapy for WM and have had either documented disease progression or had no response to the most recent treatment regimen
  • Centrally confirmed clinicopathological diagnosis of WM
  • Measurable disease defined as serum monoclonal immunoglobulin M (IgM) >0.5 gram per deciliter (g/dL)
  • Symptomatic disease, requiring treatment
  • Hematology and biochemical values within protocol-defined limits
  • Female participants of childbearing potential must have a negative serum pregnancy test at screening and agree to use highly effective methods of contraception while taking study drug. Female participants of childbearing potential should avoid becoming pregnant while taking ibrutinib and for up to 1 month after the last dose of study drug. Male participants must use an effective barrier method of contraception during the study and for 3 months following the last dose of ibrutinib if sexually active with a female of childbearing potential
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Exclusion Criteria
  • Involvement of the central nervous system by WM
  • Evidence of disease transformation
  • Prior exposure to BTK inhibitors
  • Known hypersensitivity reaction to ibrutinib or to the excipients in its formulation
  • Received any WM-related therapy <=30 days prior to first administration of study treatment
  • Received a prior allogeneic hematopoietic stem cell transplant
  • Plasmapheresis <35 days prior to the initiation of study drug, except when at least one serum IgM central assessment was performed during the screening period and was >35 days from the most recent plasmapheresis procedure
  • History of other malignancies, except: (a) malignancy treated with curative intent and with no known active disease present for >=2 years before the first dose of study drug and felt to be at low risk for recurrence by treating physician; (b) adequately treated nonmelanoma skin cancer or lentigo maligna without evidence of disease; (c) adequately treated carcinoma in situ without evidence of disease
  • Vaccinated with live, attenuated vaccines within 4 weeks of first dose of study drug
  • Infection requiring systemic treatment that was completed <=14 days before the first dose of study drug
  • Bleeding disorders or hemophilia
  • Stroke or intracranial hemorrhage within 6 months prior to enrollment
  • Infection with human immunodeficiency virus (HIV) or active infection with hepatitis B or hepatitis C
  • Major surgery within 4 weeks of first dose of study drug
  • Any life-threatening illness, medical condition, or organ system dysfunction that, in the investigator's opinion, could compromise the participant's safety or put the study outcomes at undue risk
  • Currently active, clinically significant hepatic impairment Child-Pugh Class B or C according to the Child Pugh classification
  • Currently active, clinically significant cardiovascular disease
  • Requires or receiving anticoagulation with warfarin or other Vitamin K antagonists
  • Unable to swallow capsules or malabsorption syndrome, disease significantly affecting gastrointestinal function, or resection of the stomach or small bowel, symptomatic inflammatory bowel disease or ulcerative colitis, or partial or complete bowel obstruction
  • Requires treatment with a strong cytochrome P450 (CYP) 3A inhibitor
  • Lactating or pregnant
  • Unable to understand the purpose and risks of the study and to provide a signed and dated informed consent form (ICF) and authorization to use protected health information (in accordance with national and local participant privacy regulations)
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Study & Design

Study Type
INTERVENTIONAL
Study Design
SINGLE_GROUP
Arm && Interventions
GroupInterventionDescription
Ibrutinib 420 milligram (mg)IbrutinibParticipants will receive ibrutinib 420 mg once daily, continuously starting at Day 1 of Week 1 until disease progression or unacceptable toxicity, whichever occurs first.
Primary Outcome Measures
NameTimeMethod
Overall Response Rate (ORR)Up to 4 years

ORR is defined as the percentage of participants who achieve partial response (PR) or better per the modified Consensus Response Criteria from the 6th International Workshop on Waldenstrom Macroglobulinemia (IWWM) (NCCN 2019) as assessed by the investigator.

Secondary Outcome Measures
NameTimeMethod
Overall Survival (OS)Up to 4 years

OS is defined as the time from the date of first dose to the date of the participant's death from any cause.

Trough Plasma Concentration (Ctrough) of IbrutinibDay 1 of Weeks 1, 5 and 9

Ctrough is defined as the observed plasma concentration before dosing or at the end of the dosing interval.

Number of Participants with Adverse Events (AEs) as a Measure of Safety and TolerabilityUp to 4 years

An AE is any untoward medical occurrence in a participant participating in a clinical study that does not necessarily have a causal relationship with the pharmaceutical/biological agent under study.

Percentage of Participants Achieving Clinical Response Rate (CRR)Up to 4 years

CRR is defined as the percentage of participants who achieve minor response (MR) or better according to the modified 4th IWWM (NCCN 2019) criteria as assessed by the investigator.

Progression Free Survival (PFS)Up to 4 years

PFS is defined as duration from the date of first dose to the date of disease progression or death, whichever is first reported, assessed according to the modified 6th IWWM (NCCN 2019) criteria.

Percentage of Participants Achieving Very Good Partial Response (VGPR) or Better ResponseUp to 4 years

VGPR or better rate is defined as the percentage of participants who achieve VGPR or better according to the modified 4th IWWM (NCCN 2019) criteria as assessed by the investigator.

Duration of Response (DOR)Up to 4 years

DOR is defined as duration from the date of initial documentation of a response (PR or better) to the date of first documented evidence of progressive disease or death for responders (PR or better) as assessed by the investigator.

Time to Response (TTR)Up to 4 years

TTR is defined as the time from the date of first dose to the date of initial documentation of a response (PR or better).

Trial Locations

Locations (6)

Wuhan Union Hospital

🇨🇳

Wuhan, China

The Second Affiliated Hospital of Xi'an Jiaotong University

🇨🇳

Xi'an, China

Henan Cancer Hospital

🇨🇳

Zhengzhou, China

The First Hospital of Jilin University

🇨🇳

Changchun, China

Institute of Hematology and Blood Diseases Hospital

🇨🇳

Tianjin, China

First affiliated Hospital of Zhejiang University

🇨🇳

Hangzhou, China

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