Study of Ibrutinib in Patients With Symptomatic, Previously Untreated Waldenstrom's Macroglobulinemia, and Impact on Tumor Genomic Evolution Using Whole Genome Sequencing
|Study Design:||Endpoint Classification: Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
|Official Title:||Phase II Study of Ibrutinib in Patients With Symptomatic, Previously Untreated Waldenstrom's Macroglobulinemia, and Impact on Tumor Genomic Evolution Using Whole Genome Sequencing|
- Major Response Rate [ Time Frame: 2 years ] [ Designated as safety issue: No ]To asses the proportion of participants with a PR (50% reduction or more in serum IgM) or better.
- Best Overall Response Rate [ Time Frame: 2 years ] [ Designated as safety issue: No ]To asses the proportion of participants with an MR (25% reduction or more in serum IgM) or better.
- Safety and tolerability of ibrutinib assessed by Adverse events considered to be related to the study drug will be recorded. [ Time Frame: 2 years ] [ Designated as safety issue: No ]
- Response rates by genetic variants [ Time Frame: 2 years ] [ Designated as safety issue: No ]Identifying genomic variants that are determinants of response and response duration
- Duration of response [ Time Frame: 2 years ] [ Designated as safety issue: No ]
|Study Start Date:||November 2015|
|Estimated Study Completion Date:||February 2023|
|Estimated Primary Completion Date:||February 2019 (Final data collection date for primary outcome measure)|
This is single arm, open label, Phase II, single center study designed to evaluate the safety and efficacy of ibrutinib in previously untreated WM patients. Treatment will be administered in 4-week cycles, and participants will receive treatment for up to 48 cycles. Treatment will be comprised of ibrutinib at 420 mg per day by oral administration.
This research study is a Phase II clinical trial. Phase II clinical trials test the safety and effectiveness of an investigational intervention to learn whether the intervention works in treating a specific disease. "Investigational" means that the intervention is being studied.
The FDA (the U.S. Food and Drug Administration) has approved ibrutinib as a form of treatment for the patient specific disease.
Ibrutinib has been under investigation in research studies in participants with recurrent B-cell lymphoma, chronic lymphocytic leukemia (CLL), small lymphocytic lymphoma (SLL), mantle cell lymphoma (MCL), diffuse large B-cell lymphoma (DLBCL), and prolymphocytic leukemia, and WM. In a study of ibrutinib in relapsed/refractory WM patients, response rates were high and the treatment was well tolerated.
The prior studies suggest that ibrutinib may be a useful treatment strategy for untreated WM patients. This study will test the safety and efficacy of ibrutinib as an option for untreated WM patients. The study will also conduct genomic sequencing of malignant WM cells before the start of treatment, and 6, 12, 24, 36 and 48 months afterwards. Genomic sequencing is the analysis of the entire DNA structure from tumor and normal cells. The purpose of this sequencing is to study which genetic changes effect how ibrutinib works. The results of these studies could also help in better understanding the course of WM disease, and be applicable to the development of other effective drug treatments.
Please refer to this study by its ClinicalTrials.gov identifier: NCT02604511
|Contact: Steven P Treon, MD, PhD||617-632-2681|
|United States, Massachusetts|
|Dana-Farber Cancer Institute||Not yet recruiting|
|Boston, Massachusetts, United States, 02215|
|Contact: Steven P Treon, MD, PhD 617-632-2681|
|Principal Investigator: Steven P. Treon, MD, MA, PhD|
|Massacusetts General Hospital||Not yet recruiting|
|Boston, Massachusetts, United States, 02114|
|Contact: Elizabeth O'Donnell, MD 617-724-4000|
|Principal Investigator: Elizabeth O'Donnell, MD|
|Principal Investigator:||Steven P Treon, MD, PhD||Dana-Farber Cancer Institute|