- ICH GCP
- US Clinical Trials Registry
- Clinical Trial NCT02439138
Study of Phosphatidylinositol-3-kinase (PI3K) Inhibitor Idelalisib (GS-1101) in Waldenström Macroglobulinemia
Phase II Study of Phosphatidylinositol-3-kinase (PI3K) Inhibitor Idelalisib (GS-1101) in Waldenström Macroglobulinemia
Study Overview
Detailed Description
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.
Study Type
Enrollment (Actual)
Phase
- Phase 2
Contacts and Locations
Study Locations
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Massachusetts
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Boston, Massachusetts, United States, 02215
- Dana Farber Cancer Institute
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Participation Criteria
Eligibility Criteria
Ages Eligible for Study
Accepts Healthy Volunteers
Genders Eligible for Study
Description
Inclusion Criteria:
- 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:
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 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
Study Plan
How is the study designed?
Design Details
- Primary Purpose: Treatment
- Allocation: N/A
- Interventional Model: Single Group Assignment
- Masking: None (Open Label)
Arms and Interventions
Participant Group / Arm |
Intervention / Treatment |
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Experimental: 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. |
Oral twice daily for 6 months followed by once daily until disease progression or unacceptable toxicity.
Other Names:
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What is the study measuring?
Primary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
---|---|---|
Overall Response Rate (ORR)
Time Frame: Participants were followed for the duration of therapy, a median of one cycle, for up to 3 cycles.
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ORR measured by decrease in serum IgM level by at least 25% from baseline.
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Participants were followed for the duration of therapy, a median of one cycle, for up to 3 cycles.
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Secondary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
---|---|---|
Percentage of Participants With Adverse Events
Time Frame: Participants were followed for the duration of therapy, a median of one cycle, for up to 3 cycles.
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Assess the safety and tolerability of idelalisib
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Participants were followed for the duration of therapy, a median of one cycle, for up to 3 cycles.
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Rate of Complete Response (CR)
Time Frame: Participants were followed for the duration of therapy, a median of one cycle, for up to 3 cycles.
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CR measured by decrease in serum IgM levels to normal range, disappearnace of monoclonal protein by immunofixation, no evidence of bone marrow involvement, and resolution of any extramedullary disease by CT scan.
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Participants were followed for the duration of therapy, a median of one cycle, for up to 3 cycles.
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Rate of Very Good Partial Response (VGPR)
Time Frame: Participants were followed for the duration of therapy, a median of one cycle, for up to 3 cycles.
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VGPR measured by decrease in serum IgM levels of at least 90% from baseline.
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Participants were followed for the duration of therapy, a median of one cycle, for up to 3 cycles.
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Rate of Partial Response (PR)
Time Frame: Participants were followed for the duration of therapy, a median of one cycle, for up to 3 cycles.
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PR measured by decrease in serum IgM levels of between 25% and 50% from baseline.
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Participants were followed for the duration of therapy, a median of one cycle, for up to 3 cycles.
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Rate of Minimal Response
Time Frame: Participants were followed for the duration of therapy, a median of one cycle, for up to 3 cycles.
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Minimal response measured by decrease in serum IgM levels of between 25% and 50%.
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Participants were followed for the duration of therapy, a median of one cycle, for up to 3 cycles.
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Rate of Stable Disease
Time Frame: Participants were followed for the duration of therapy, a median of one cycle, for up to 3 cycles.
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Stable disease measured by serum IgM levels <25% reduced from baseline.
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Participants were followed for the duration of therapy, a median of one cycle, for up to 3 cycles.
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Rate of Progressive Disease
Time Frame: Participants were followed for the duration of therapy, a median of one cycle, for up to 3 cycles.
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Progressive disease measured by an 25% increase in serum IgM level with an absolute increase of at least 500mg/dL from the lowest attained IgM on therapy.
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Participants were followed for the duration of therapy, a median of one cycle, for up to 3 cycles.
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Collaborators and Investigators
Sponsor
Collaborators
Study record dates
Study Major Dates
Study Start
Primary Completion (Actual)
Study Completion (Actual)
Study Registration Dates
First Submitted
First Submitted That Met QC Criteria
First Posted (Estimate)
Study Record Updates
Last Update Posted (Estimate)
Last Update Submitted That Met QC Criteria
Last Verified
More Information
Terms related to this study
Keywords
Additional Relevant MeSH Terms
- Cardiovascular Diseases
- Vascular Diseases
- Immune System Diseases
- Neoplasms by Histologic Type
- Neoplasms
- Lymphoproliferative Disorders
- Lymphatic Diseases
- Immunoproliferative Disorders
- Hematologic Diseases
- Hemorrhagic Disorders
- Hemostatic Disorders
- Paraproteinemias
- Blood Protein Disorders
- Neoplasms, Plasma Cell
- Waldenstrom Macroglobulinemia
- Molecular Mechanisms of Pharmacological Action
- Enzyme Inhibitors
- Antineoplastic Agents
- Idelalisib
Other Study ID Numbers
- 15-040
- ISR IN-US-313-1609 (Other Grant/Funding Number: Gilead Sciences, Inc.)
Plan for Individual participant data (IPD)
Plan to Share Individual Participant Data (IPD)?
This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.
Clinical Trials on Waldenstrom's Macroglobulinemia
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Weill Medical College of Cornell UniversityMayo Clinic; Janssen Scientific Affairs, LLCTerminatedWaldenstrom Macroglobulinemia | Waldenstrom's Macroglobulinemia Recurrent | Waldenstrom's Macroglobulinemia Refractory | Waldenstrom's Disease | Waldenström; Hypergammaglobulinemia | Waldenstrom's Macroglobulinemia of Lymph Nodes | Waldenstrom's Macroglobulinaemia, Without Mention of RemissionUnited States
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BeiGeneRecruitingWaldenstrom Macroglobulinemia | Waldenstrom's Macroglobulinemia Recurrent | Waldenstrom's Macroglobulinemia RefractoryUnited States, Australia, France, China, Spain, United Kingdom
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Beijing InnoCare Pharma Tech Co., Ltd.CompletedWaldenstrom's Macroglobulinemia Recurrent | Waldenstrom's Macroglobulinemia RefractoryChina
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Dana-Farber Cancer InstituteBristol-Myers SquibbTerminatedWaldenstrom's MacroglobulinemiaUnited States
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Fondazione Italiana Linfomi ONLUSCompletedWaldenstrom's MacroglobulinemiaItaly
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Dana-Farber Cancer InstituteMillennium Pharmaceuticals, Inc.CompletedWaldenstrom's MacroglobulinemiaUnited States
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Dana-Farber Cancer InstituteBeth Israel Deaconess Medical Center; Millennium Pharmaceuticals, Inc.CompletedWaldenstrom's MacroglobulinemiaUnited States
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Dana-Farber Cancer InstituteNovartis; Millennium Pharmaceuticals, Inc.TerminatedWaldenstrom's MacroglobulinemiaUnited States
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Idera Pharmaceuticals, Inc.TerminatedWaldenstrom's MacroglobulinemiaUnited States
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Dana-Farber Cancer InstituteAmgenCompletedWaldenstrom's MacroglobulinemiaUnited States
Clinical Trials on GS-1101
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Gilead SciencesTerminatedMantle Cell Lymphoma | Chronic Lymphocytic Leukemia | Diffuse Large B-cell Lymphoma | Indolent Non-Hodgkin's LymphomaUnited States
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John Mark SloanGilead SciencesTerminated
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Gilead SciencesActive, not recruitingB-cell MalignanciesUnited States, United Kingdom, France
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Gilead SciencesWithdrawn
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Gilead SciencesCompleted
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Gilead SciencesCompletedFollicular Lymphoma | Marginal Zone Lymphoma | Small Lymphocytic Lymphoma | Indolent Non-Hodgkin's LymphomaUnited States
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Gilead SciencesTerminatedLymphoma, Non-Hodgkin | Chronic Lymphocytic LeukemiaUnited States
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Gilead SciencesGerman CLL Study GroupCompleted
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Gilead SciencesCompletedFollicular Lymphoma | Marginal Zone Lymphoma | Chronic Lymphocytic Leukemia | Small Lymphocytic Lymphoma | Indolent Non-Hodgkin Lymphoma | Lymphoplasmacytic Lymphoma (With or Without Waldenstrom Macroglobulinemia)Japan
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Gilead SciencesCompletedHodgkin LymphomaUnited States