Phase II Study of Phosphatidylinositol-3-kinase (PI3K) Inhibitor Idelalisib (GS-1101) in Waldenström Macroglobulinemia
试验速览
- 阶段
- 2 期
- 状态
- 终止
- 入组人数
- 5
- 试验地点
- 1
- 主要终点
- Overall Response Rate (ORR)
研究概览
简要总结
This research study is evaluating a drug called idelalisib (formerly known as GS-1101 or CAL-101) as a possible treatment for Waldenstrom's Macroglobulinemia (WM).
详细描述
This research study is a Phase II clinical trial. Phase II clinical trials test the effectiveness of an investigational drug, idelalisib, to learn whether idelalisib works in treating a specific cancer. "Investigational" means that idelalisib is still being studied and that research doctors are trying to find out more about it-such as the safest dose to use, the side effects it may cause, and if idelalisib is effective for treating different types of cancer. Idelalisib has already been approved in the US by the FDA to treat patients with relapsed chronic lymphocytic leukemia, follicular lymphoma and small lymphocytic lymphoma.
Idelalisib is a newly discovered drug that is being developed as an anti-cancer agent. This drug has been used in laboratory experiments and other research studies in B-cell malignancies and information from those other research studies suggests that idelalisib may help to target the tumor cells in B-cell malignancies, including WM. B cells are a type of white blood cell responsible for making antibodies.
In this research study, the investigators are testing the safety and efficacy of idelalisib as a treatment option for relapsed or refractory Waldenstrom's Macroglobulinemia.
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 18 Years 至 —（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Participants must meet the following criteria on screening examination to be eligible to participate in the study:
- •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 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) < 2.5 X institutional upper limit of normal
- •Creatinine ≤ 2 mg/dL
- •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:
- •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
排除标准
- •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 idelalisib
- •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; alter the absorption, distribution, metabolism or excretion of Idelalisib; or impair the assessment of study results
- •Grade > 2 toxicity (other than alopecia) continuing from prior anti-cancer therapy
- •Known central nervous system 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 capsules
- •History of non-compliance to medical regimens
- •Unwilling or unable to comply with the protocol
研究组 & 干预措施
GS-1101
After the screening procedures confirms eligibility to participate in the research study. Treatment will be administered on an outpatient basis.
-- Idelalisib (GS-11-01) orally, predetermined dose twice daily per cycle for up to 6 cycles. After this initial 6 month period, for Cycles 7 and beyond, Idelalisib will be administered once a day until disease progression.
干预措施: GS-1101 (Drug)
结局指标
主要结局
Overall Response Rate (ORR)
时间窗: Participants were followed for the duration of therapy, a median of one cycle, for up to 3 cycles.
ORR measured by decrease in serum IgM level by at least 25% from baseline.
次要结局
- Percentage of Participants With Adverse Events(Participants were followed for the duration of therapy, a median of one cycle, for up to 3 cycles.)
- Rate of Complete Response (CR)(Participants were followed for the duration of therapy, a median of one cycle, for up to 3 cycles.)
- Rate of Very Good Partial Response (VGPR)(Participants were followed for the duration of therapy, a median of one cycle, for up to 3 cycles.)
- Rate of Partial Response (PR)(Participants were followed for the duration of therapy, a median of one cycle, for up to 3 cycles.)
- Rate of Minimal Response(Participants were followed for the duration of therapy, a median of one cycle, for up to 3 cycles.)
- Rate of Stable Disease(Participants were followed for the duration of therapy, a median of one cycle, for up to 3 cycles.)
- Rate of Progressive Disease(Participants were followed for the duration of therapy, a median of one cycle, for up to 3 cycles.)
研究者
Jorge J. Castillo, MD
Principal Investigator
Dana-Farber Cancer Institute