NCT02677324 · REGISTRY CONDITION SEARCH

Study of ABT-199 (GDC-199) In Patients With Relapsed Or Refractory Waldenström Macroglobulinemia

Found through the registry condition index, not selected as a treatment recommendation. This discovery collection was restricted to studies with posted results.

Phase: PHASE2
Status at capture: COMPLETED
Registry last update: 2022-05-11

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.

Open the original ClinicalTrials.gov record → · Download preserved record

What did the study report?

Outcomes, safety and baseline populations are separate source sections. Quality-of-life measures appear under their original outcome titles.

flow

Participant flow

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baseline

Baseline characteristics

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outcome · POSTED

Overall Response Rate

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outcome · POSTED

Number of Participants With Treatment-related Adverse Events as Assessed by CTCAE v4.0

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outcome · POSTED

Number of Participants With Complete Response

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outcome · POSTED

Number of Participants With Very Good Partial Response

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outcome · POSTED

Number of Participants With Partial Response

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outcome · POSTED

Number of Participants With Minor Response

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outcome · POSTED

Number of Participants With Stable Disease

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outcome · POSTED

Progression Free Survival

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outcome · POSTED

Overall Response Rate Among CXCR4 Mutated Participants

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outcome · POSTED

Overall Response Rate Among Participants Without CXCR4 Mutations

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adverse events

Adverse events

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more info

Limitations, agreements, and source contact

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Who could take part

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 \<2 (see Appendix A). * Participants must have normal organ and marrow function (growth factors cannot be given prophylactically to establish eligibility) as defined below: * Absolute neutrophil count \> 1,000/mm3 * Platelets \> 50,000/mm3 * Hemoglobin \> 8 g/dL * Total bilirubin ≤ 1.5 mg/dL or \< 2 mg/dL if attributable to hepatic infiltration by neoplastic disease * AST (SGOT) and ALT (SGPT) \< 2.5X the institutional upper limit of normal * Creatinine clearance ≥50 ml/min * Not on any active therapy for other malignancies with the exception of topical therapies for basal cell or squamous cell cancers of the skin. * Females of childbearing potential (FCBP) must agree to use two reliable forms of contraception simultaneously or have or will have complete abstinence from heterosexual intercourse during the following time periods related to this study: 1) while participating in the study; and 2) for at least 28 days after discontinuation from the study. Men must agree to use a latex condom during sexual contact with a FCBP even if they have had a successful vasectomy. FCBP must be referred to a qualified provider of contraceptive methods if needed. * Able to adhere to the study visit schedule and other protocol requirements. * Ability to understand and the willingness to sign a written informed consent document. Exclusion Criteria: * Any serious medical condition, laboratory abnormality, uncontrolled intercurrent illness, or psychiatric illness/social condition that would prevent the participant from signing the informed consent form. * Concurrent use of any other anti-cancer agents or treatments or any other study agents. * Prior exposure to ABT-199 or BCL2 inhibitors. * Prior or ongoing clinically significant illness, medical condition, surgical history, physical finding, ECG finding, or laboratory abnormality that, in the investigator's opinion, could affect the safety of the patient, including symptomatic hyperviscosity; alter the absorption, distribution, metabolism or excretion of ABT-199; or impair the assessment of study results. * Grade \> 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.
healthy Volunteers: false
minimum Age: 18 Years
sex: ALL
std Ages: ADULT
OLDER_ADULT

Treatment arms and interventions

arm Groups

description
ABT199 will be administered daily, with 28 consecutive days defined as a treatment cycle for a maximum for 26 cycles
intervention Names
Drug: ABT199
label
ABT199
type
EXPERIMENTAL

interventions

arm Group Labels
ABT199
description
Oral BCL-2 antagonist
name
ABT199
other Names
Venetoclax
type
DRUG

Study design

allocation

intervention Model

SINGLE_GROUP

masking Info

masking: NONE

primary Purpose

TREATMENT

Enrollment

count: 33
type: ACTUAL

Registered outcome plans (not posted results)

primary Outcomes

description
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).
measure
Overall Response Rate
time Frame
2 years

secondary Outcomes

description
Number of participants who experienced an adverse event while on ABT-199
measure
Number of Participants With Treatment-related Adverse Events as Assessed by CTCAE v4.0
time Frame
2 years
description
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.
measure
Number of Participants With Complete Response
time Frame
2 years
description
Very Good Partial Response (VGPR): is defined as ≥90% reduction in serum IgM levels, or normalization of serum IgM levels.
measure
Number of Participants With Very Good Partial Response
time Frame
2 years
description
Partial response (PR) is defined as achieving a ≥50% reduction in serum IgM levels.
measure
Number of Participants With Partial Response
time Frame
2 years
description
Minor Response (MR): A minor response (MR) is defined 25-49% reduction in serum IgM levels.
measure
Number of Participants With Minor Response
time Frame
2 years
description
Stable disease is defined as having \<25% increase in serum IgM levels and \<25% reduction in serum IgM levels
measure
Number of Participants With Stable Disease
time Frame
2 years
description
Amount of time following ABT-199 administration until \>25% increase in serum IgM
measure
Progression Free Survival
time Frame
4 years
description
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).
measure
Overall Response Rate Among CXCR4 Mutated Participants
time Frame
2 years
description
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).
measure
Overall Response Rate Among Participants Without CXCR4 Mutations
time Frame
2 years

Full study description

brief 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.
detailed Description: 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.

Source references

references

citation
Castillo JJ, Guijosa A, Allan JN, Siddiqi T, Advani RH, Flynn CA, Meid K, Budano N, Nguyen J, Ramirez-Gamero A, Tsakmaklis N, Hunter ZR, Patterson CJ, Treon SP, Sarosiek S. Long-term follow-up of venetoclax monotherapy in previously treated patients with Waldenstrom macroglobulinemia. Blood Adv. 2025 Oct 14;9(19):4842-4847. doi: 10.1182/bloodadvances.2025016890.
pmid
40674749
type
DERIVED
citation
Castillo JJ, Allan JN, Siddiqi T, Advani RH, Meid K, Leventoff C, White TP, Flynn CA, Sarosiek S, Branagan AR, Demos MG, Guerrera ML, Kofides A, Liu X, Munshi M, Tsakmaklis N, Xu L, Yang G, Patterson CJ, Hunter ZR, Davids MS, Furman RR, Treon SP. Venetoclax in Previously Treated Waldenstrom Macroglobulinemia. J Clin Oncol. 2022 Jan 1;40(1):63-71. doi: 10.1200/JCO.21.01194. Epub 2021 Nov 18.
pmid
34793256
type
DERIVED

Source notices and limitations

Discovery and provenance

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Preserved source evidence · Not medical advice