Ibrutinib (PCI-32765) in Waldenstrom's Macroglobulinemia

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. Read our disclaimer for details. Identifier: NCT01614821
Recruitment Status : Active, not recruiting
First Posted : June 8, 2012
Last Update Posted : September 6, 2017
Information provided by (Responsible Party):
Steven P. Treon, MD, PhD, Dana-Farber Cancer Institute

May 17, 2012
June 8, 2012
September 6, 2017
May 2012
August 2018   (Final data collection date for primary outcome measure)
Overall Response Rate [ Time Frame: 2 years ]
To assess the overall response rate (> 25% reduction in disease burden), major response rates (> 50% reduction in disease burden), and Very Good Partial Response/Complete Response (VGPR/CR) of PCI-32765 in symptomatic WM patients with relapsed/refractory disease
Same as current
Complete list of historical versions of study NCT01614821 on Archive Site
  • Safety and Tolerability of PCI-32765 [ Time Frame: 2 years ]
    To assess the safety and tolerability of PCI-32765 in symptomatic WM patients with relapsed/refractory disease. Adverse Events determined to be associated with PCI-32765 and subsequent outcomes will constitute the safety profile of PCI-32765 in WM.
  • Determine Progression Free Survival [ Time Frame: 2 years ]
    To determine Progression Free Survival (PFS), and time to next therapy (TTNT) of PCI-32765 in symptomatic WM patients with relapsed/refractory disease
Same as current
Not Provided
Not Provided
Ibrutinib (PCI-32765) in Waldenstrom's Macroglobulinemia
Phase 2 Study of Bruton's Tyrosine Kinase (Btk) Inhibitor, Ibrutinib (PCI-32765), in Waldenstrom's Macroglobulinemia

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.

Patients in this research study will receive up to 26 cycles of treatment. Each treatment cycle lasts 4 weeks. Patients will take PCI-32765 by mouth, once a day in the morning.

During each cycle patients will be asked to visit the clinic for scheduled tests and exams and to receive a supply of PCI-32765 to take at home every day. Patients will visit the clinic on the first day of each of the first 3 cycles, and then just once at the beginning of every three cycles.

During study visits, patients will have a physical exam where they will be asked questions about their general health and specific questions about any problems that they might be having and any medications they may be taking. Patients will have blood tests to see how their disease is responding to the study treatment and how they are tolerating the study drug. Patients may also have CT scans of the chest, abdomen and pelvis as well as a bone marrow aspirate and biopsy. If a patient's disease stays the same or is helped, he/she will continue to get study treatment. If disease worsens, he/she will be taken off study treatment at that time.

After completion of the treatment and as part of standard of care, follow-up tests will include a physical exam, review of symptoms and medications, blood tests, bone marrow aspirate and biopsy, CT scans of the chest, abdomen and pelvis. The investigators would like to continue to monitor progress by following-up every three months for up to two years after completion of the study.

Phase 2
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Waldenstrom's Macroglobulinemia
Drug: PCI-32765
Taken orally, once daily in the morning
Other Name: ibrutinib
Experimental: Treatment Arm
PCI-32765; ibrutinib
Intervention: Drug: PCI-32765
Treon 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 Waldenström's macroglobulinemia. N Engl J Med. 2015 Apr 9;372(15):1430-40. doi: 10.1056/NEJMoa1501548.

*   Includes publications given by the data provider as well as publications identified by Identifier (NCT Number) in Medline.
Active, not recruiting
August 2018
August 2018   (Final data collection date for primary outcome measure)

Inclusion Criteria:

  • 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

Exclusion Criteria:

  • 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
Sexes Eligible for Study: All
18 Years and older   (Adult, Senior)
Contact information is only displayed when the study is recruiting subjects
United States
Not Provided
Not Provided
Steven P. Treon, MD, PhD, Dana-Farber Cancer Institute
Dana-Farber Cancer Institute
Not Provided
Principal Investigator: Steven P Treon, MD PhD Dana-Farber Cancer Institute
Dana-Farber Cancer Institute
September 2017

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP