MedPath

VTX-2337 and Pegylated Liposomal Doxorubicin (PLD) in Patients With Recurrent or Persistent Epithelial Ovarian, Fallopian Tube or Primary Peritoneal Cancer

Phase 2
Completed
Conditions
Epithelial Ovarian Cancer
Fallopian Tube Cancer
Primary Peritoneal Cancer
Interventions
Registration Number
NCT01666444
Lead Sponsor
Celgene
Brief Summary

The purpose of this study is to compare the overall survival of patients treated with VTX-2337 + pegylated liposomal doxorubicin (PLD) versus those treated with PLD alone in women with recurrent or persistent, epithelial ovarian, fallopian tube or primary peritoneal cancer.

VTX-2337, a small molecule agonist of Toll-like Receptor 8 (TLR8), activates multiple components of the innate immune system and is being developed as a novel therapeutic agent for use in oncology. Experimental data obtained in an animal model of ovarian cancer supports the combination of VTX-2337 with PLD. In this model, the combination of VTX-2337 and PLD resulted in a significant reduction in tumor growth compared to either agent alone and an increase in the number of T lymphocytes infiltrating the tumor. The combination of PLD and VTX-2337 has been tested in a small number of women with ovarian cancer in a Phase 1b study and appears to be generally well-tolerated.

Detailed Description

OBJECTIVES

Primary Objectives:

* To compare the overall survival (OS) of patients treated with VTX-2337 + PLD versus those treated with PLD alone in women with recurrent or persistent, epithelial ovarian, fallopian tube or primary peritoneal cancer.

* To compare the progression-free survival (PFS) between the two treatment groups using Immune-Related Response Evaluation Criteria in Solid Tumors (irRECIST).

Secondary Objectives:

* To compare the progression-free survival (PFS) between the two treatment groups using Response Evaluation Criteria In Solid Tumors (RECIST 1.1).

* To compare the nature, frequency and severity of drug-related adverse events (AEs) between the two treatment groups.

Exploratory Objectives:

* To compare the best overall response rate (ORR) and duration of response (based on the probability of being in response function \[PBRF\]) between the two treatment groups using irRECIST and RECIST 1.1.

* To compare the disease control rate (DCR) between the two treatment groups using irRECIST and RECIST 1.1.

* To assess the impact of immune status and response on the clinical effects (OS, PFS, DCR, ORR, PBRF, AEs) of study treatment.

* To assess the effect of TLR8 polymorphisms and BRCA1/BRCA2 mutations on the clinical effects (OS, PFS, DCR, ORR, PBRF, AEs) of study treatment.

* To assess the effect of immune cell subsets, as measured by immunohistochemistry and micro RNA in primary tumor tissue (e.g. immune score), on the clinical effects (OS, PFS, DCR, ORR, PBRF, AEs) of study treatment.

* To assess whether the presence of autoantibodies to tumor-derived proteins are predictive of the clinical effects (OS, PFS, DCR, ORR, PBRF, AEs) of study treatment.

OUTLINE:

This is Phase 2 multicenter clinical study to evaluate the efficacy and safety of the combination of VTX-2337 + PLD compared to PLD + Placebo.

The dosing schedule will be the same for both treatment arms, and will be based on a 28-day cycle. The starting dose schedule is PLD on Day 1 plus VTX-2337 or placebo on Day 3, Day 10, and Day 17 for the first 4 cycles. Starting with cycle 5, the dose regimen will be PLD on Day 1 plus VTX-2337 or placebo on Day 3.

Blood samples are collected periodically during cycle 1 for pharmacodynamics, pharmacogenomics, and other research studies.

Patients will receive therapy until disease progression based on Immune-Related RECIST or until adverse effects prohibit further therapy. Following treatment completion, all patients will be followed with physical exams and histories every three months for the first two years, and then every six months for the next three years, and then

Recruitment & Eligibility

Status
COMPLETED
Sex
Female
Target Recruitment
297
Inclusion Criteria
  1. Patients must have recurrent or persistent epithelial ovarian, fallopian tube, or primary peritoneal carcinoma.

  2. Patients with the following histologic cell types are eligible: serous adenocarcinoma, endometrioid adenocarcinoma, mucinous adenocarcinoma, undifferentiated carcinoma, clear cell adenocarcinoma, mixed epithelial adenocarcinoma, transitional cell carcinoma, malignant Brenner's tumor or adenocarcinoma not otherwise specified.

  3. Patient must have measurable disease as defined by RECIST 1.1.

  4. Patients must have received treatment with a platinum-based chemotherapeutic regimen for management of primary disease containing carboplatin, cisplatin or another organoplatinum compound. This initial treatment may have included intraperitoneal therapy, consolidation, non-cytotoxic agents or extended therapy administered after surgical or non-surgical assessment.

    Patients are allowed to receive, but are not required to receive, one additional cytotoxic regimen for management of recurrent or persistent disease.

    Patients are allowed to have received, but are not required to have received, biologic/targeted therapy (e.g., bevacizumab and/or PARP inhibitor) as part of their primary treatment regimen or for management of recurrent or persistent disease.

  5. Patients must have platinum-resistant disease, defined as having a platinum-free interval (PFI) of < 12 months after first- or second-line platinum-based chemotherapy, or having disease progression while receiving second-line platinum-based chemotherapy.

  6. Patients must have adequate bone marrow, renal, hepatic, and neurologic functions as defined by the following:

    • Bone marrow function: absolute neutrophil count (ANC) ≥ 1,500/mm3. This ANC cannot have been induced or supported by granulocyte colony stimulating factors. Platelets ≥ 100,000/mm3. Hemoglobin ≥ 9 g/dL.
    • Renal function: creatinine ≤ 1.5 x institutional upper limit normal (ULN).
    • Hepatic function: bilirubin < 1.2 mg/dL, SGOT (AST) and SGPT (ALT) ≤ 3.0 x ULN and alkaline phosphatase ≤ 2.5 x ULN.
  7. Patients must have recovered from effects of recent surgery, radiotherapy or chemotherapy:

    • Patients should be free of active infection requiring parenteral antibiotics.
    • Any hormonal therapy directed at the malignant tumor must be discontinued at least one week prior to registration. Continuation of hormone replacement therapy is permitted.
    • Any other prior therapy directed at the malignant tumor, including chemotherapy, biologic/targeted agents and immunologic agents, must be discontinued at least three weeks prior to registration.
    • Any prior radiation therapy must be completed at least four weeks prior to registration.
  8. Patients must have a GOG performance status of 0 or 1.

  9. Patients of childbearing potential must have a negative pregnancy test prior to the study entry and be practicing an effective form of contraception. If applicable, patients must discontinue breastfeeding prior to study entry.

  10. Patients must meet the entry requirements and undergo the baseline procedures.

  11. Patients must have signed an IRB-approved informed consent form and authorization permitting release of personal health information.

Exclusion Criteria
  1. Patients who have had treatment with VTX-2337, doxorubicin, PLD, or any other anthracycline.
  2. Patients who have received an investigational agent < 30 days prior to registration.
  3. Patients who have received oral or parenteral corticosteroids < 2 weeks prior to registration or who require ongoing systemic immunosuppressive therapy for any reason.
  4. Patients with active autoimmune disease. "Active" refers to any condition currently requiring therapy. Examples of autoimmune disease include systemic lupus erythematosus, multiple sclerosis, inflammatory bowel disease and rheumatoid arthritis.
  5. Patients with a history of other invasive malignancies, with the exception of non-melanoma skin cancer, are excluded if there is any evidence of the other malignancy being present within the last three years.
  6. Patients who have received prior radiotherapy OTHER THAN for the treatment of ovarian, fallopian tube or primary peritoneal cancer within the last three years are excluded.
  7. Patients who have received prior chemotherapy OTHER THAN for the treatment of ovarian, fallopian tube or primary peritoneal cancer within the last three years are excluded.
  8. Patients with history or evidence upon physical examination of CNS disease, including primary brain tumor, seizures not controlled with standard medical therapy, any brain metastases, or history of cerebrovascular accident (CVA, stroke), transient ischemic attack (TIA) or subarachnoid hemorrhage within six months of the first date of treatment on this study.
  9. Patients with clinically significant cardiovascular disease.
  10. Patients who are pregnant or nursing.
  11. Patients under the age of 18.
  12. Patients with clinical symptoms or signs of gastrointestinal obstruction and/or who require parenteral hydration or nutrition.

Study & Design

Study Type
INTERVENTIONAL
Study Design
PARALLEL
Arm && Interventions
GroupInterventionDescription
PLD 40 mg/m2 plus placeboPlaceboThe dosing schedule will be based on a 28-day cycle. The starting dose schedule is PLD on Day 1 plus placebo on Day 3, Day 10, and Day 17 for the first 4 cycles. Starting with cycle 5, the dose regimen will be PLD on Day 1 plus placebo on Day 3 only.
PLD 40 mg/m2 plus VTX-2337VTX-2337The dosing schedule will be be based on a 28-day cycle. The starting dose schedule is PLD on Day 1 plus VTX-2337 on Day 3, Day 10, and Day 17 for the first 4 cycles. Starting with cycle 5, the dose regimen will be PLD on Day 1 plus VTX-2337 on Day 3 only, without additional doses of VTX-2337 on Days 10 and Day 17.
PLD 40 mg/m2 plus placebopegylated liposomal doxorubicin (PLD)The dosing schedule will be based on a 28-day cycle. The starting dose schedule is PLD on Day 1 plus placebo on Day 3, Day 10, and Day 17 for the first 4 cycles. Starting with cycle 5, the dose regimen will be PLD on Day 1 plus placebo on Day 3 only.
PLD 40 mg/m2 plus VTX-2337pegylated liposomal doxorubicin (PLD)The dosing schedule will be be based on a 28-day cycle. The starting dose schedule is PLD on Day 1 plus VTX-2337 on Day 3, Day 10, and Day 17 for the first 4 cycles. Starting with cycle 5, the dose regimen will be PLD on Day 1 plus VTX-2337 on Day 3 only, without additional doses of VTX-2337 on Days 10 and Day 17.
Primary Outcome Measures
NameTimeMethod
Overall SurvivalSurvival is measured from date of enrollment and randomization on the study until death from any cause, or if alive at last contact, date of last contact.

Comparison of duration of survival between the 2 treatment groups

Secondary Outcome Measures
NameTimeMethod
Progression-free Survival (PFS)Progression-free survival is measured from enrollment and randomization on the study until first indication of progression based on irRECIST criteria or death from any cause, or if progression-free at last contact, the date of last disease assessment.

Comparison of PFS between the 2 treatment groups

Frequency and Severity of Adverse Events (AEs)Assessed during each cycle of therapy and within 30 days after the last cycle of therapy

An AE is any untoward medical occurrence associated with the use of a drug in humans, whether or not considered drug-related. An AE can be unfavorable and unintended sign, symptom, or disease which is temporally associated with the use of investigational product (IP), whether or not considered related to the IP. A serious AE = an AE occurring at any dose that: • Results in death • Is life- threatening • Requires or prolongs existing inpatient hospitalization • Results in persistent or significant disability/incapacity • Is a congenital anomaly/birth defect; • Constitutes an important medical event. The Investigator assessed the relationship of each AE to IP and graded the severity according to the National Cancer Institute Common Terminology Criteria for Adverse Events, Version 4.0: Grade (GR) 1 = Mild; asymptomatic or mild symptoms; GR 2 = Moderate (minimal, local or noninvasive intervention indicated); GR 3 = Severe or medically significant; GR 4 = Life-threatening; GR 5 = Death

Trial Locations

Locations (127)

Ohio State University Medical Center

🇺🇸

Columbus, Ohio, United States

Aspirus Regional Cancer Center

🇺🇸

Wausau, Wisconsin, United States

Johns Hopkins Medical Institution

🇺🇸

Baltimore, Maryland, United States

Marshfield Clinic

🇺🇸

Marshfield, Wisconsin, United States

St. Dominic-Jackson Memorial Hospital

🇺🇸

Jackson, Mississippi, United States

University of Mississippi Medical Center

🇺🇸

Jackson, Mississippi, United States

Munson Medical Center

🇺🇸

Traverse City, Michigan, United States

Reed City Hospital - Spectrum Health

🇺🇸

Reed City, Michigan, United States

Mercy Health Partners - Mercy Campus

🇺🇸

Muskegon, Michigan, United States

Cooper University Hospital

🇺🇸

Camden, New Jersey, United States

North Shore University Hospital

🇺🇸

Manhasset, New York, United States

Dartmouth-Hitchcock Medical Center

🇺🇸

Lebanon, New Hampshire, United States

Hackensack University Medical Center

🇺🇸

Hackensack, New Jersey, United States

Gynecologic Oncology of Central New York - SUNY Upstate

🇺🇸

Syracuse, New York, United States

Women's Cancer Center at Kettering Medical Center

🇺🇸

Kettering, Ohio, United States

Bon Secours St. Francis Hospital

🇺🇸

Greenville, South Carolina, United States

Green Bay Oncology at St. Vincent's Hospital

🇺🇸

Green Bay, Wisconsin, United States

University Hospitals of Cleveland

🇺🇸

Cleveland, Ohio, United States

Northwestern University - Robert H. Lurie Comprehensive Cancer Center

🇺🇸

Chicago, Illinois, United States

MD Anderson Cancer Center

🇺🇸

Houston, Texas, United States

The Methodist Hospital

🇺🇸

Houston, Texas, United States

Pacific Gynecology Specialists

🇺🇸

Seattle, Washington, United States

Seattle Cancer Care Alliance

🇺🇸

Seattle, Washington, United States

Kaiser Permanente Medical Center

🇺🇸

Walnut Creek, California, United States

Hartford Hospital

🇺🇸

Hartford, Connecticut, United States

The Hospital of Central Connecticut

🇺🇸

New Britain, Connecticut, United States

Northeast Georgia Medical Center

🇺🇸

Gainesville, Georgia, United States

Greater Baltimore Medical Center

🇺🇸

Baltimore, Maryland, United States

Lahey Hospital & Medical Center

🇺🇸

Burlington, Massachusetts, United States

Bronson Battle Creek

🇺🇸

Battle Creek, Michigan, United States

Grand Rapids Clinical Oncology

🇺🇸

Grand Rapids, Michigan, United States

Gynecologic Oncology of West Michigan

🇺🇸

Grand Rapids, Michigan, United States

West Michigan Cancer Center

🇺🇸

Kalamazoo, Michigan, United States

Summa Health System

🇺🇸

Akron, Ohio, United States

University of Washington Medical Center

🇺🇸

Seattle, Washington, United States

Bay Area Medical Center

🇺🇸

Marinette, Wisconsin, United States

Indiana University Medical Center

🇺🇸

Indianapolis, Indiana, United States

St. Vincent Gynecologic Oncology

🇺🇸

Indianapolis, Indiana, United States

Women's Cancer Care Center of Nevada

🇺🇸

Las Vegas, Nevada, United States

Providence Saint Joseph Medical Center

🇺🇸

Burbank, California, United States

Long Beach Memorial Medical Center

🇺🇸

Long Beach, California, United States

Stanford University School of Medicine

🇺🇸

Stanford, California, United States

Northside Hospital

🇺🇸

Atlanta, Georgia, United States

Central Georgia Gynecologic Oncology

🇺🇸

Macon, Georgia, United States

Georgia Regents University

🇺🇸

Augusta, Georgia, United States

Memorial Health University Medical Center

🇺🇸

Savannah, Georgia, United States

St. Joseph's - Candler Gynecologic Oncology

🇺🇸

Savannah, Georgia, United States

Rush University Medical Center

🇺🇸

Chicago, Illinois, United States

Sudarshan K. Sharma, MD, LTD

🇺🇸

Hinsdale, Illinois, United States

Carle Cancer Center

🇺🇸

Urbana, Illinois, United States

University of Iowa Hospitals and Clinics

🇺🇸

Iowa City, Iowa, United States

McFarland Clinic

🇺🇸

Ames, Iowa, United States

Ellis Fischel Cancer Center - University of Missouri

🇺🇸

Columbia, Missouri, United States

Memorial Medical Center

🇺🇸

Las Cruces, New Mexico, United States

Women's Cancer Care Associates

🇺🇸

Albany, New York, United States

Roswell Park Cancer Institute

🇺🇸

Buffalo, New York, United States

SUNY Downstate Medical Center

🇺🇸

Brooklyn, New York, United States

NYU Langone Medical Center - Cancer Institute

🇺🇸

New York, New York, United States

Long Island Jewish Medical Center

🇺🇸

New Hyde Park, New York, United States

Monter Cancer Center

🇺🇸

Lake Success, New York, United States

Columbia University Medical Center

🇺🇸

New York, New York, United States

Memorial Sloan Kettering Cancer Center

🇺🇸

New York, New York, United States

Hope Women's Cancer Center

🇺🇸

Asheville, North Carolina, United States

Carolinas Medical Center / Levine Cancer Institute

🇺🇸

Charlotte, North Carolina, United States

Alamance Regional Cancer Center

🇺🇸

Burlington, North Carolina, United States

Carolinas Medical Center - Northeast

🇺🇸

Concord, North Carolina, United States

University of Cincinnati

🇺🇸

Cincinnati, Ohio, United States

Fairview Hospital Moll Pavilion Cancer Center

🇺🇸

Cleveland, Ohio, United States

Cleveland Clinic Foundation

🇺🇸

Cleveland, Ohio, United States

Tulsa Cancer Institute

🇺🇸

Tulsa, Oklahoma, United States

Hillcrest Hospital - Cleveland Clinic

🇺🇸

Mayfield Heights, Ohio, United States

Lake University Seidman Cancer Center

🇺🇸

Mentor, Ohio, United States

Abington Memorial Hospital; Hanjani Institute for Gynecologic Oncology

🇺🇸

Abington, Pennsylvania, United States

University of Pennsylvania Medical Center

🇺🇸

Philadelphia, Pennsylvania, United States

Hillman Cancer Center - University of Pittsburgh

🇺🇸

Pittsburgh, Pennsylvania, United States

Geisinger Medical Center

🇺🇸

Danville, Pennsylvania, United States

Western Pennsylvania Hospital

🇺🇸

Pittsburgh, Pennsylvania, United States

Reading Hospital (McGlinn Family Regional Cancer Center)

🇺🇸

West Reading, Pennsylvania, United States

Women and Infants Hospital of Rhode Island

🇺🇸

Providence, Rhode Island, United States

UT Southwestern Medical Center

🇺🇸

Dallas, Texas, United States

University of Texas Medical Branch

🇺🇸

Galveston, Texas, United States

Carilion Clinic Gynecological Oncology

🇺🇸

Roanoke, Virginia, United States

Mid Atlantic Pelvic Surgery Associates

🇺🇸

Annandale, Virginia, United States

Northwest Hospital - UW Medicine

🇺🇸

Seattle, Washington, United States

Women's Cancer Care of Seattle

🇺🇸

Seattle, Washington, United States

Abbott Northwestern Hospital

🇺🇸

Minneapolis, Minnesota, United States

Huntsman Cancer Institute, University of Utah

🇺🇸

Salt Lake City, Utah, United States

Minnesota Oncology Coon Rapids Clinic

🇺🇸

Coon Rapids, Minnesota, United States

Fairview Southdale Hospital

🇺🇸

Edina, Minnesota, United States

Woodbury Clinic - CornerStone Medical Specialty Centre

🇺🇸

Woodbury, Minnesota, United States

Minnesota Oncology Hematology - St. Paul Cancer Center

🇺🇸

Saint Paul, Minnesota, United States

Metro Minnesota Clinical Oncology Program

🇺🇸

Saint Louis Park, Minnesota, United States

Park Nicollet Frauenshuh Cancer Center

🇺🇸

Saint Louis Park, Minnesota, United States

Winthrop P. Rockefeller Cancer Institute - University of Arkansas

🇺🇸

Little Rock, Arkansas, United States

Sinai Hospital of Baltimore

🇺🇸

Baltimore, Maryland, United States

University of Maryland Medical Center

🇺🇸

Baltimore, Maryland, United States

St. Francis Hospital and Medical Center

🇺🇸

Hartford, Connecticut, United States

Spectrum Health at Butterworth Campus

🇺🇸

Grand Rapids, Michigan, United States

Avera Cancer Institute

🇺🇸

Sioux Falls, South Dakota, United States

St Vincent Hospital

🇺🇸

Green Bay, Wisconsin, United States

University of Massachusetts Memorial Healthcare

🇺🇸

Worcester, Massachusetts, United States

Maine Medical Partners Women's Health

🇺🇸

Scarborough, Maine, United States

Holy Family Memorial Medical Center

🇺🇸

Manitowoc, Wisconsin, United States

Women's Cancer Associates

🇺🇸

Saint Petersburg, Florida, United States

Saint Mary's Health Care

🇺🇸

Grand Rapids, Michigan, United States

Gibbs Cancer Center

🇺🇸

Spartanburg, South Carolina, United States

Fox Chase Cancer Center

🇺🇸

Philadelphia, Pennsylvania, United States

St. Joseph's Hospital and Medical Center

🇺🇸

Phoenix, Arizona, United States

Karmanos Cancer Institute - Wayne State University

🇺🇸

Detroit, Michigan, United States

Henry Ford Health System

🇺🇸

Detroit, Michigan, United States

Peggy and Charles Stephenson Cancer Center

🇺🇸

Oklahoma City, Oklahoma, United States

University of Colorado Cancer Center

🇺🇸

Aurora, Colorado, United States

Aurora St. Luke's Medical Center Gynecologic Oncology

🇺🇸

Milwaukee, Wisconsin, United States

Medical College of Wisconsin

🇺🇸

Milwaukee, Wisconsin, United States

Sutter Cancer Center

🇺🇸

Sacramento, California, United States

St. Joseph Mercy Hospital

🇺🇸

Ann Arbor, Michigan, United States

MD Anderson Cancer Center - Orlando

🇺🇸

Orlando, Florida, United States

Wake Forest University Health Science

🇺🇸

Winston-Salem, North Carolina, United States

Yale - New Haven Hospital

🇺🇸

New Haven, Connecticut, United States

Medical University of South Carolina

🇺🇸

Charleston, South Carolina, United States

Virginia Gynecology Oncology

🇺🇸

Richmond, Virginia, United States

Green Bay Oncology at St. Mary's Hospital

🇺🇸

Green Bay, Wisconsin, United States

Kapiolani Medical Center for Women and Children

🇺🇸

Honolulu, Hawaii, United States

University of Wisconsin-Madison

🇺🇸

Madison, Wisconsin, United States

Southwest Gynecologic Oncology Associates

🇺🇸

Albuquerque, New Mexico, United States

University of New Mexico Cancer Center

🇺🇸

Albuquerque, New Mexico, United States

University of Kansas Medical Center

🇺🇸

Westwood, Kansas, United States

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