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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details. Identifier: NCT04042376
Recruitment Status : Recruiting
First Posted : August 1, 2019
Last Update Posted : December 11, 2020
Information provided by (Responsible Party):
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.

Condition or disease Intervention/treatment Phase
Waldenstrom Macroglobulinemia Drug: Ibrutinib Phase 4

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 17 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: A Single Arm, Multicenter, Phase 4 Study of the Bruton's Tyrosine Kinase (BTK) Inhibitor, Ibrutinib (PCI-32765) in Chinese Subjects With Relapse or Refractory Waldenström's Macroglobulinemia
Actual Study Start Date : December 18, 2019
Estimated Primary Completion Date : December 31, 2023
Estimated Study Completion Date : March 29, 2024

Arm Intervention/treatment
Experimental: Ibrutinib 420 milligram (mg)
Participants will receive ibrutinib 420 mg once daily, continuously starting at Day 1 of Week 1 until disease progression or unacceptable toxicity, whichever occurs first.
Drug: Ibrutinib
Ibrutinib will be administered orally, once daily, at a dose of 420 mg (140 mg*3 capsules taken together at one time).
Other Names:
  • JNJ-54179060
  • PCI-32765

Primary Outcome Measures :
  1. Overall Response Rate (ORR) [ Time Frame: 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 :
  1. Percentage of Participants Achieving Clinical Response Rate (CRR) [ Time Frame: 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.

  2. Percentage of Participants Achieving Very Good Partial Response (VGPR) or Better Response [ Time Frame: Up 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.

  3. Duration of Response (DOR) [ Time Frame: 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.

  4. Time to Response (TTR) [ Time Frame: 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).

  5. Progression Free Survival (PFS) [ Time Frame: 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.

  6. Overall Survival (OS) [ Time Frame: 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.

  7. Trough Plasma Concentration (Ctrough) of Ibrutinib [ Time Frame: Day 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.

  8. Number of Participants with Adverse Events (AEs) as a Measure of Safety and Tolerability [ Time Frame: Up 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.

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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

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

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)

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): NCT04042376

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Contact: Study Contact 844-434-4210

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The First Hospital of Jilin University Recruiting
Changchun, China, 130021
Peking Union Medical College Hospital Withdrawn
Dongcheng, China, 100032
Fujian Meidical University Union Hospital Withdrawn
Fuzhou, China, 350001
Guangdong Provincial People's Hospital Withdrawn
Guangzhou, China, 510100
First affiliated Hospital of Zhejiang University Recruiting
Hangzhou, China, 310003
The People's Hospital of Jiangsu Province Withdrawn
Nanjing, China, 210029
Institute of Hematology & Blood Diseases Hospital Recruiting
Tianjin, China, 300320
Wuhan Union Hospital Recruiting
Wuhan, China, 430022
The Second Affiliated Hospital of Xi'an Jiaotong University Recruiting
Xi'an, China, 710004
Henan Cancer Hospital Recruiting
Zhengzhou, China, 450008
Sponsors and Collaborators
Janssen Research & Development, LLC
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Study Director: Janssen Research & Development, LLC Clinical Trial Janssen Research & Development, LLC
Additional Information:
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Responsible Party: Janssen Research & Development, LLC Identifier: NCT04042376    
Other Study ID Numbers: CR108654
54179060WAL4001 ( Other Identifier: Janssen Research & Development, LLC )
First Posted: August 1, 2019    Key Record Dates
Last Update Posted: December 11, 2020
Last Verified: December 2020
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: Yes
Plan Description:

The data sharing policy of the Janssen Pharmaceutical Companies of Johnson & Johnson is available at

As noted on this site, requests for access to the study data can be submitted through Yale Open Data Access (YODA) Project site at


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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Product Manufactured in and Exported from the U.S.: Yes
Additional relevant MeSH terms:
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Waldenstrom Macroglobulinemia
Neoplasms, Plasma Cell
Neoplasms by Histologic Type
Hemostatic Disorders
Vascular Diseases
Cardiovascular Diseases
Blood Protein Disorders
Hematologic Diseases
Hemorrhagic Disorders
Lymphoproliferative Disorders
Lymphatic Diseases
Immunoproliferative Disorders
Immune System Diseases