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History of Changes for Study: NCT01614821
PCI-32765 in Waldenstrom's Macroglobulinemia
Latest version (submitted January 10, 2020) on ClinicalTrials.gov
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Study Record Versions
Version A B Submitted Date Changes
1 June 7, 2012 None (earliest Version on record)
2 July 3, 2012 Recruitment Status, Study Status and Contacts/Locations
3 July 27, 2012 Recruitment Status, Contacts/Locations and Study Status
4 December 19, 2012 Recruitment Status, Study Status and Contacts/Locations
5 April 30, 2013 Recruitment Status, Contacts/Locations, Study Status, Study Identification, Eligibility and Study Design
6 June 13, 2013 Recruitment Status, Contacts/Locations, Study Status and Arms and Interventions
7 June 11, 2014 Study Status
8 December 5, 2014 Study Status
9 May 20, 2015 Study Status
10 November 11, 2015 Study Status
11 June 13, 2016 Study Status
12 October 31, 2016 Study Status
13 March 20, 2017 Study Status
14 September 5, 2017 Study Status
15 May 29, 2018 Study Status
16 October 26, 2018 Recruitment Status, Study Status, Study Design
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Results Submission Events
17 January 10, 2020 Study Status, Outcome Measures, Results and Study Description
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Study NCT01614821
Submitted Date:  June 7, 2012 (v1)

Open or close this module Study Identification
Unique Protocol ID: 12-015
Brief Title: PCI-32765 in Waldenstrom's Macroglobulinemia
Official Title: Phase 2 Study of Bruton's Tyrosine Kinase (Btk) Inhibitor, PCI-32765, in Waldenstrom's Macroglobulinemia
Secondary IDs:
Open or close this module Study Status
Record Verification: June 2012
Overall Status: Recruiting
Study Start: May 2012
Primary Completion: May 2014 [Anticipated]
Study Completion:
First Submitted: May 17, 2012
First Submitted that
Met QC Criteria:
June 7, 2012
First Posted: June 8, 2012 [Estimate]
Last Update Submitted that
Met QC Criteria:
June 7, 2012
Last Update Posted: June 8, 2012 [Estimate]
Open or close this module Sponsor/Collaborators
Sponsor: Dana-Farber Cancer Institute
Responsible Party: Principal Investigator
Investigator: Steven P. Treon, MD, PhD
Official Title: Principal Investigator
Affiliation: Dana-Farber Cancer Institute
Collaborators:
Open or close this module Oversight
U.S. FDA-regulated Drug:
U.S. FDA-regulated Device:
Data Monitoring: Yes
Open or close this module Study Description
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.

Detailed Description:

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.

Open or close this module Conditions
Conditions: Waldenstrom's Macroglobulinemia
Keywords: Relapsed
Refractory
Open or close this module Study Design
Study Type: Interventional
Primary Purpose: Treatment
Study Phase: Phase 2
Interventional Study Model: Single Group Assignment
Number of Arms: 1
Masking: None (Open Label)
Allocation: N/A
Enrollment: 33 [Anticipated]
Open or close this module Arms and Interventions
Arms Assigned Interventions
Experimental: Treatment Arm
PCI-32765
Drug: PCI-32765
Taken orally, once daily in the morning
Open or close this module Outcome Measures
Primary Outcome Measures:
1. 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
Secondary Outcome Measures:
1. 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.
2. 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
Open or close this module Eligibility
Minimum Age: 18 Years
Maximum Age:
Sex: All
Gender Based:
Accepts Healthy Volunteers: No
Criteria:

Inclusion Criteria:

  • Clinicopathological diagnosis of Waldenstrom's Macroglobulinemia
  • Measurable disease
  • Have received at least one prior therapy for WM but no more than 4 prior therapies
  • Disease free of prior malignancies for at least 5 years
  • 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
Open or close this module Contacts/Locations
Central Contact Person: Steven P. Treon, MD, PhD
Telephone: 617-632-2681
Email: streon@partners.org
Study Officials: Steven P Treon, MD PhD
Principal Investigator
Dana-Farber Cancer Institute
Locations: United States, Massachusetts
Brigham and Women's Hospital
[Recruiting]
Boston, Massachusetts, United States, 02215
Contact:Contact: Steven P. Treon, MD, PhD 617-632-2681 streon@partners.org
Contact:Principal Investigator: Steven P. Treon, MD, PhD
Dana-Farber Cancer Institute
[Recruiting]
Boston, Massachusetts, United States, 02215
Contact:Contact: Steven P Treon, MD, PhD 617-632-2681 streon@partners.org
Contact:Contact: Christina Tripsas, MA 617-632-3418 christinak_tripsas@dfci.harvard.edu
Contact:Principal Investigator: Steven P. Treon, MD, PhD
Open or close this module IPDSharing
Plan to Share IPD:
Open or close this module References
Links:
Available IPD/Information:

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