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Pilot Study With CY, Pembrolizumab, GVAX, and IMC-CS4 (LY3022855) in Patients With Borderline Resectable Adenocarcinoma of the Pancreas

Early Phase 1
Completed
Conditions
Pancreatic Cancer
Interventions
Drug: Cyclophosphamide
Drug: GVAX Pancreas Vaccine (GVAX)
Drug: Pembrolizumab
Drug: IMC-CS4
Registration Number
NCT03153410
Lead Sponsor
Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins
Brief Summary

The purpose of this study is to evaluate whether combining cyclophosphamide (CY), pembrolizumab, GVAX and IMC-CS4 is effective and safe in patients with borderline resectable pancreatic cancer.

Detailed Description

This was a single center, open label, pilot study to evaluate the safety and intratumoral immune response of neoadjuvant/adjuvant triplet immunotherapy: CY/GVAX, Pembrolizumab, and IMC-CS4 in patients with resectable or borderline resectable Pancreatic ductal adenocarcinoma.

Patients received the combination of CY/GVAX with pembrolizumab and IMC-CS4 following completion of standard neoadjuvant chemotherapy (with or without radiation). Immunotherapy agents were given prior to and after surgical resection.

Patients receive neoadjuvant immunotherapy consisting of cyclophosphamide and pembrolizumab on day 1, IMC-CS4 on days 1, 8, and 15, and GVAX on day 2 of Cycles 1 and 2 (3 weeks/cycle). Patients will then undergo pancreaticoduodenectomy 2-3 weeks later, followed by standard of care adjuvant chemotherapy at the discretion of the primary oncologist. Patients may then receive 4 cycles (3 weeks/cycle) of adjuvant immunotherapy (same regimen as Cycles 1 and 2). Disease free patients may receive an additional 12 booster cycles (3 weeks/cycle) of immunotherapy consisting of pembrolizumab on day 1 of all 12 booster cycles, as well as cyclophosphamide on day 1 and GVAX on day 2 of the 6th and 12th booster cycle.

Recruitment & Eligibility

Status
COMPLETED
Sex
All
Target Recruitment
12
Inclusion Criteria
  • Have histologically or cytologically proven adenocarcinoma of the pancreas.
  • Patient's acceptance to have a core biopsy.
  • Presence of at least one measurable lesion.
  • Must not have metastatic disease.
  • Patients must have measurable disease, defined as at least one lesion that can be accurately measured in at least one dimension (longest diameter to be recorded) as >20 mm with conventional techniques or as >10 mm with spiral CT scan. See Section 10.1.2 for the evaluation of measurable disease.
  • Must have received last dose of stereotactic body radiotherapy no longer than 28 days prior to enrollment.
  • Must have received last dose of chemotherapy at least 14 days or longer prior to entry into the study.
  • Age >18 years.
  • ECOG performance status 0-1.
  • Patient's blood, kidney and liver function must within normal limits
  • Must use an acceptable form of birth control while on study.
  • Ability to understand and the willingness to sign a written informed consent document.
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Exclusion Criteria
  • Currently is participating or has participated in a study using any investigational therapy within the past 28 days or is currently using an investigational device.
  • Major surgery 28 days prior to study entry excluding minor procedures (dental work, skin biopsy, etc.), celiac plexus block, and biliary stent placement.
  • Used any systemic steroids, immunosuppressant medications and anti-neoplastic treatment in the past 14 days.
  • Prior treatment with immunotherapy agents (including, but not limited to: IL-2, interferon, anti-PD-1, anti-PD-L1, anti-PD-L2, anti-CD137, anti-OX-40, anti-CD40, or anti-CTLA-4 antibodies).
  • Used any growth factors including, but not limited to, granulocyte-colony stimulating factor (G-CSF), GM-CSF, erythropoietin, within 14 days of study drug administration. Use of such agents while on study is also prohibited.
  • Received any prophylactic vaccine within 14 days of first dose of study drug or received a live vaccine within 30 days of study treatment.
  • Currently have or have history of certain study-specified heart, liver, kidney, lung, neurological, psychological, immune or other medical conditions.
  • History of any autoimmune disease: inflammatory bowel disease, (including ulcerative colitis and Crohn's Disease), rheumatoid arthritis, systemic progressive sclerosis (scleroderma), systemic lupus erythematosus (SLE) autoimmune vasculitis (e.g., Wegener's Granulomatosis), CNS or motor neuropathy considered to be of autoimmune origin (e.g., Guillian-Barre Syndrome, Myasthenia Gravis, Multiple Sclerosis). Patients with thyroid disease will be allowed.
  • Has history of (non-infectious) pneumonitis that required steroids, history or evidence of interstitial lung disease or active, non-infectious pneumonitis.
  • Has a pulse oximetry < 92% on room air.
  • Evidence of ascites on imaging.
  • Requires the use of home oxygen.
  • Have known history of infection with HIV, hepatitis B, or hepatitis C.
  • Have been diagnosed with another cancer in the past 5 years (except for superficial bladder cancer, non-melanoma skin cancers, or a low grade prostate cancer not requiring therapy)
  • History of severe hypersensitivity reaction to any monoclonal antibody.
  • Known or suspected hypersensitivity to GM-CSF, hetastarch, corn, dimethyl sulfoxide, fetal bovine serum, trypsin (porcine origin), yeast or any other component of GVAX pancreas vaccine.
  • Pregnant or breastfeeding women.
  • Positive pregnancy test during the study.
  • Women sexually active with a fertile man and of childbearing potential unwilling or unable to use an acceptable method to avoid pregnancy for the entire study and for up to 120 days after the last dose of study drug.
  • Unwilling or unable to follow the study schedule for any reason.
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Study & Design

Study Type
INTERVENTIONAL
Study Design
SINGLE_GROUP
Arm && Interventions
GroupInterventionDescription
Cyclophosphamide, GVAX, Pembrolizumab and IMC-CS4Cyclophosphamide-
Cyclophosphamide, GVAX, Pembrolizumab and IMC-CS4GVAX Pancreas Vaccine (GVAX)-
Cyclophosphamide, GVAX, Pembrolizumab and IMC-CS4Pembrolizumab-
Cyclophosphamide, GVAX, Pembrolizumab and IMC-CS4IMC-CS4-
Primary Outcome Measures
NameTimeMethod
Number of Patients With a Treatment-related Immunologic Effect8 weeks

Treatment-related immunologic effect is defined as an 80% or greater increase in the number of CD8+ T cells (and at least 1.8 times the baseline median absolute deviation) in surgically resected tumor tissue in comparison to the baseline biopsy in subjects that received at least 1 dose of neoadjuvant combination immunotherapy and underwent a R0, R1, or R2 surgical resection.

Safety of the Combination of GVAX Pancreas Vaccine (With CY), Pembrolizumab, and a Macrophage Targeting Agent (CSF1R Inhibitor IMC-CS4) in Patients With Resectable or Borderline Resectable Pancreatic Cancer (BRPC) Prior to and Following Surgery25 months

Number of subjects that experienced a grade 3 or higher study drug related adverse event

Secondary Outcome Measures
NameTimeMethod
Overall Survival (OS)44 months

OS will be measured from date of first dose until death or end of follow-up (OS will be censored on the date the subject was last known to be alive for subjects without documentation of death at the time of analysis). Estimation based on the Kaplan-Meier curve.

Immune-related Objective Response Rate (irORR)Up to 10 weeks from baseline irRC read

Immune-related Objective Response Rate (irORR) is defined as the number of patients achieving a complete response (irCR) or partial response (irPR) based on immune related response criteria (irRC) following the study immunotherapy prior to surgical resection. Per irRC criteria, Complete Response (irCR) is the disappearance of all target lesions, Partial Response (irPR) is a decrease in tumor burden by 50% or greater.

Pathologic Response Rate8 weeks

Pathologic response as determined by surgical margins status at the time of surgery and response to neoadjuvant treatment per surgical specimen assessment (participants with \<10% residual viable tumor is reported).

Disease Free Survival (DFS)37 months

DFS is defined as time from fist dose of study drug to the date of progression or death. Subjects were censored at the date of last scan if alive at the time of analyses or if the subject's date of death was greater than 3 months after last scan. Estimation based on the Kaplan-Meier curve.

Surgical Resectability Rate8 weeks

Surgical resectability after neoadjuvant therapy as determined by the number of patients able to undergo surgical resection.

Trial Locations

Locations (1)

Sidney Kimmel Comprehensive Cancer Center

🇺🇸

Baltimore, Maryland, United States

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