Ibrutinib (PCI-32765) in Waldenstrom's Macroglobulinemia
Phase 2 Study of Bruton's Tyrosine Kinase (Btk) Inhibitor, Ibrutinib (PCI-32765), in Waldenstrom's Macroglobulinemia
1 other identifier
interventional
63
1 country
4
Brief Summary
This research study is a Phase II clinical trial. Phase II clinical trials test the effectiveness of an investigational drug, PCI-32765, to learn whether PCI-32765 works in treating a specific cancer. "Investigational" means that PCI-32765 is still being studied and that research doctors are trying to find out more about it-such as the safest dose to use, the side effects it may cause, and if PCI-32765 is effective for treating different types of cancer. It also means that the FDA has not yet approved PCI-32765 for use in patients, including people with Waldenstrom's Macroglobulinemia. PCI-32765 is a newly discovered drug that is being developed as an anti-cancer agent. PCI-32765 is a Bruton's tyrosine kinase (Btk) inhibitor drug which interrupts B cell receptor (BCR) signaling in lymphomas by selectively and irreversibly binding to the Btk protein, which then results in malignant cell death. This drug has been used in laboratory experiments and other research studies in B-cell malignancies and information from those other research studies suggests that PCI-32765 may be a treatment strategy for B-cell malignancies, including Waldenstrom's Macroglobulinemia. In this research study, the investigators are testing the safety and efficacy of PCI-32765 as a treatment option for relapsed or refractory Waldenstrom's Macroglobulinemia.
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at P50-P75 for phase_2
Started May 2012
Longer than P75 for phase_2
4 active sites
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
Click on a node to explore related trials.
Study Timeline
Key milestones and dates
Study Start
First participant enrolled
May 1, 2012
CompletedFirst Submitted
Initial submission to the registry
May 17, 2012
CompletedFirst Posted
Study publicly available on registry
June 8, 2012
CompletedPrimary Completion
Last participant's last visit for primary outcome
September 9, 2016
CompletedStudy Completion
Last participant's last visit for all outcomes
September 15, 2018
CompletedResults Posted
Study results publicly available
January 21, 2020
CompletedJanuary 21, 2020
January 1, 2020
4.4 years
May 17, 2012
November 13, 2019
January 10, 2020
Conditions
Keywords
Outcome Measures
Primary Outcomes (1)
Overall Response Rate
To assess the overall response rate (\>25% reduction in serum IgM from baseline).
4 years
Secondary Outcomes (5)
Safety and Tolerability of PCI-32765
4 years
Determine Progression Free Survival
6 years
To Determine Time to Next Therapy (TTNT) of PCI-32765 in Symptomatic WM Patients With Relapsed/Refractory Disease
6 years
Major Response Rates
4 years
Very Good Partial Response Rate
4 years
Study Arms (1)
Treatment Arm
EXPERIMENTALPCI-32765; ibrutinib
Interventions
Eligibility Criteria
You may qualify if:
- Clinicopathological diagnosis of Waldenstrom's Macroglobulinemia
- Measurable disease
- Have received at least one prior therapy for WM therapies
- Disease free of prior malignancies
- Able to adhere to study visit schedule and other protocol requirement
You may not qualify if:
- Pregnant or breastfeeding
- Any other serious medical condition
- Concurrent use of other anti-cancer agents or treatments
- Prior exposure to PCI-32765
- Known CNS lymphoma
- Significant cardiovascular disease
- Any disease affecting gastrointestinal function
Contact the study team to confirm eligibility.
Sponsors & Collaborators
Study Sites (4)
Stanford University
Palo Alto, California, 94305, United States
Brigham and Women's Hospital
Boston, Massachusetts, 02215, United States
Dana-Farber Cancer Institute
Boston, Massachusetts, 02215, United States
Memorial Sloan-Kettering Cancer Center
New York, New York, 10065, United States
Related Publications (3)
Guijosa A, Ramirez-Gamero A, Sarosiek S, Branagan AR, von Keudell G, Treon SP, Castillo JJ. Ibrutinib plus rituximab vs ibrutinib monotherapy in patients with Waldenstrom macroglobulinemia: a pooled analysis. Blood Adv. 2025 Sep 23;9(18):4705-4715. doi: 10.1182/bloodadvances.2025016536.
PMID: 40674744DERIVEDTreon SP, Meid K, Gustine J, Yang G, Xu L, Liu X, Patterson CJ, Hunter ZR, Branagan AR, Laubach JP, Ghobrial IM, Palomba ML, Advani R, Castillo JJ. Long-Term Follow-Up of Ibrutinib Monotherapy in Symptomatic, Previously Treated Patients With Waldenstrom Macroglobulinemia. J Clin Oncol. 2021 Feb 20;39(6):565-575. doi: 10.1200/JCO.20.00555. Epub 2020 Sep 15.
PMID: 32931398DERIVEDTreon SP, Tripsas CK, Meid K, Warren D, Varma G, Green R, Argyropoulos KV, Yang G, Cao Y, Xu L, Patterson CJ, Rodig S, Zehnder JL, Aster JC, Harris NL, Kanan S, Ghobrial I, Castillo JJ, Laubach JP, Hunter ZR, Salman Z, Li J, Cheng M, Clow F, Graef T, Palomba ML, Advani RH. Ibrutinib in previously treated Waldenstrom's macroglobulinemia. N Engl J Med. 2015 Apr 9;372(15):1430-40. doi: 10.1056/NEJMoa1501548.
PMID: 25853747DERIVED
MeSH Terms
Conditions
Interventions
Condition Hierarchy (Ancestors)
Results Point of Contact
- Title
- Dr. Steven Treon
- Organization
- Dana-Farber Cancer Institute
Study Officials
- PRINCIPAL INVESTIGATOR
Steven P Treon, MD PhD
Dana-Farber Cancer Institute
Publication Agreements
- PI is Sponsor Employee
- No
- Restrictive Agreement
- No
Study Design
- Study Type
- interventional
- Phase
- phase 2
- Allocation
- NA
- Masking
- NONE
- Purpose
- TREATMENT
- Intervention Model
- SINGLE GROUP
- Sponsor Type
- OTHER
- Responsible Party
- PRINCIPAL INVESTIGATOR
- PI Title
- Principal Investigator
Study Record Dates
First Submitted
May 17, 2012
First Posted
June 8, 2012
Study Start
May 1, 2012
Primary Completion
September 9, 2016
Study Completion
September 15, 2018
Last Updated
January 21, 2020
Results First Posted
January 21, 2020
Record last verified: 2020-01