Phase 2
N=33
Study of ABT-199 (GDC-199) In Patients With Relapsed Or Refractory Waldenström Macroglobulinemia
Waldenstrom Macroglobulinemia
Bottom Line
View on ClinicalTrials.gov: NCT02677324 ↗Enrolled (actual)
33
Serious AEs
39.4%
Results posted
May 2021
Primary outcome: Primary: Overall Response Rate — 26 Participants
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 2
- Interventions
- ABT199 (Drug)
- Age
- Adult, Older Adult · 18+ yrs
- Sex
- All
- Sponsor
- Dana-Farber Cancer Institute
- Primary completion
- Feb 2020
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Overall Response Rate |
26 | — |
| SECONDARY Number of Participants With Treatment-related Adverse Events as Assessed by CTCAE v4.0 |
33 | — |
| SECONDARY Number of Participants With Complete Response |
— | — |
| SECONDARY Number of Participants With Very Good Partial Response |
6 | — |
| SECONDARY Number of Participants With Partial Response |
19 | — |
| SECONDARY Number of Participants With Minor Response |
1 | — |
| SECONDARY Number of Participants With Stable Disease |
2 | — |
| SECONDARY Progression Free Survival |
31.5 | — |
| SECONDARY Overall Response Rate Among CXCR4 Mutated Participants |
14 | — |
| SECONDARY Overall Response Rate Among Participants Without CXCR4 Mutations |
13 | — |
Summary
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.
Eligibility Criteria
Inclusion Criteria
- Clinicopathological diagnosis of Waldenstrom's Macroglobulinemia and meeting criteria for treatment using consensus panel criteria from the Second International Workshop on Waldenstrom's macroglobulinemia (Owen 2003; Kyle 2003).
- Measurable disease, defined as presence of serum immunoglobulin M (IgM) with a minimum IgM level of > 2 times the upper limit of normal of each institution is required.
- Have received at least one prior therapy for WM.
- Age ≥ 18 years.
- ECOG performance status 1,000/mm3
- Platelets > 50,000/mm3
- Hemoglobin > 8 g/dL
- Total bilirubin ≤ 1.5 mg/dL or 2 toxicity (other than alopecia) continuing from prior anti-cancer therapy.
- Known CNS lymphoma.
- Significant cardiovascular disease such as uncontrolled or symptomatic arrhythmias, congestive heart failure, or myocardial infarction within 6 months of screening.
- New York Heart Association classification III or IV heart failure.
- Malabsorption syndrome, disease significantly affecting gastrointestinal function, or resection of the stomach or small bowel, ulcerative colitis, symptomatic inflammatory bowel disease, or partial or complete bowel obstruction.
- Known history of Human Immunodeficiency Virus (HIV), Hepatitis B Virus (HBV), and/or Hepatitis C Virus (HCV) infection.
- Lactating or pregnant women.
- Inability to swallow tablets.
- History of non-compliance to medical regimens.
- Unwilling or unable to comply with the protocol.
Data sourced from ClinicalTrials.gov (NCT02677324). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.