Brief Title
Study of ABT-199 (GDC-199) In Patients With Relapsed Or Refractory Waldenström Macroglobulinemia
Official Title
Phase II Study of ABT-199 (GDC-199) In Patients With Relapsed Or Refractory Waldenström Macroglobulinemia
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.
Study Phase
Phase 2
Study Type
Interventional
Primary Outcome
Overall Response Rate
Secondary Outcome
Number of Participants With Treatment-related Adverse Events as Assessed by CTCAE v4.0
Condition
Waldenstrom Macroglobulinemia
Intervention
ABT199
Study Arms / Comparison Groups
ABT199
Description: ABT199 will be administered daily, with 28 consecutive days defined as a treatment cycle for a maximum for 26 cycles
Publications
* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.
Recruitment Information
Recruitment Status
Drug
Estimated Enrollment
33
Start Date
May 9, 2016
Completion Date
February 7, 2022
Primary Completion Date
February 14, 2020
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.
Gender
All
Ages
18 Years - N/A
Accepts Healthy Volunteers
No
Contacts
Jorge J Castillo, MD, ,
Location Countries
United States
Location Countries
United States
Administrative Informations
NCT ID
NCT02677324
Organization ID
15-491
Secondary IDs
A15-751
Responsible Party
Principal Investigator
Study Sponsor
Dana-Farber Cancer Institute
Collaborators
AbbVie
Study Sponsor
Jorge J Castillo, MD, Principal Investigator, Dana-Farber Cancer Institute
Verification Date
April 2022