This research study is studying a targeted therapy as a possible treatment for relapsed or refractory Waldenstrom's Macroglobulinemia (WM). This study is using the study intervention ABT-199.
This research study is a Phase II clinical trial. ABT-199 is a pill that blocks BCL-2, a protein that is important for the survival of WM cells. The purpose of this research study is to evaluate how well the study drug works and the safety of ABT-199 as a single agent in participants with WM that has come back or has shown no response to previous treatment. The FDA (the U.S. Food and Drug Administration) has not approved ABT-199 as a treatment for any disease.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
33
Oral BCL-2 antagonist
City of Hope National Medical Center
Duarte, California, United States
Stanford University
Palo Alto, California, United States
Dana Farber Cancer Institute
Boston, Massachusetts, United States
Weill Cornell Medical College
New York, New York, United States
Overall Response Rate
Overall Response Rate= Minor response (\>25%-50% reduction in serum IgM from baseline) + Partial Response (\>50-90% reduction in serum IgM from baseline) + Very Good Partial Response (\>90% reduction in serum IgM from baseline) + Complete Response (resolution of all symptoms, normalization of serum IgM with disappearance of IgM paraprotein, resolution of any adenopathy or splenomegaly).
Time frame: 2 years
Number of Participants With Treatment-related Adverse Events as Assessed by CTCAE v4.0
Number of participants who experienced an adverse event while on ABT-199
Time frame: 2 years
Number of Participants With Complete Response
A complete response is defined as having resolution of WM related symptoms, normalization of serum IgM levels with complete disappearance of IgM paraprotein by immunofixation, and resolution of any adenopathy or splenomegaly.
Time frame: 2 years
Number of Participants With Very Good Partial Response
Very Good Partial Response (VGPR): is defined as ≥90% reduction in serum IgM levels, or normalization of serum IgM levels.
Time frame: 2 years
Number of Participants With Partial Response
Partial response (PR) is defined as achieving a ≥50% reduction in serum IgM levels.
Time frame: 2 years
Number of Participants With Minor Response
Minor Response (MR): A minor response (MR) is defined 25-49% reduction in serum IgM levels.
Time frame: 2 years
Number of Participants With Stable Disease
This platform is for informational purposes only and does not constitute medical advice. Always consult a qualified healthcare professional.
Stable disease is defined as having \<25% increase in serum IgM levels and \<25% reduction in serum IgM levels
Time frame: 2 years
Progression Free Survival
Amount of time following ABT-199 administration until \>25% increase in serum IgM
Time frame: 4 years
Overall Response Rate Among CXCR4 Mutated Participants
Overall Response Rate for participants who tested positive for a CXCR4 mutation= Minor response (\>25%-50% reduction in serum IgM from baseline) + Partial Response (\>50-90% reduction in serum IgM from baseline) + Very Good Partial Response (\>90% reduction in serum IgM from baseline) + Complete Response (resolution of all symptoms, normalization of serum IgM with disappearance of IgM paraprotein, resolution of any adenopathy or splenomegaly).
Time frame: 2 years
Overall Response Rate Among Participants Without CXCR4 Mutations
Overall Response Rate in participants who tested negative for a CXCR4 mutation= Minor response (\>25%-50% reduction in serum IgM from baseline) + Partial Response (\>50-90% reduction in serum IgM from baseline) + Very Good Partial Response (\>90% reduction in serum IgM from baseline) + Complete Response (resolution of all symptoms, normalization of serum IgM with disappearance of IgM paraprotein, resolution of any adenopathy or splenomegaly).
Time frame: 2 years