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Phase 2 Study of Bruton's Tyrosine Kinase (Btk) Inhibitor, Ibrutinib (PCI-32765), in Waldenstrom's Macroglobulinemia

Phase 2
18 Years
Open (Enrolling)
Waldenstrom's Macroglobulinemia

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Trial Information

Phase 2 Study of Bruton's Tyrosine Kinase (Btk) Inhibitor, Ibrutinib (PCI-32765), in 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.

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

Type of Study:


Study Design:

Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment

Outcome Measure:

Overall Response Rate

Outcome Description:

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

Outcome Time Frame:

2 years

Safety Issue:


Principal Investigator

Steven P Treon, MD PhD

Investigator Role:

Principal Investigator

Investigator Affiliation:

Dana-Farber Cancer Institute


United States: Food and Drug Administration

Study ID:




Start Date:

May 2012

Completion Date:

Related Keywords:

  • Waldenstrom's Macroglobulinemia
  • Relapsed
  • Refractory
  • Waldenstrom Macroglobulinemia



Memorial Sloan-Kettering Cancer Center New York, New York  10021
Stanford University Stanford, California  94305
Dana-Farber Cancer Institute Boston, Massachusetts  02115
Brigham and Women's Hospital Boston, Massachusetts  02115