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Effect on Tumor Perfusion of a Chemotherapy Combining Gemcitabine and Vismodegib Before Surgery in Pancreatic Cancer

Early Phase 1
Conditions
Pancreatic Adenocarcinoma Resectable
Interventions
Procedure: Neoadjuvant chemotherapy
Registration Number
NCT01713218
Lead Sponsor
Jean-Luc Van Laethem
Brief Summary

Pancreatic ductal adenocarcinoma (PDAC) has one of the worst prognoses of all human cancers and is considered as a sanctuary, resistant to most of the drugs used. Identification of new molecular targets involved in its pathogenesis is urgently needed and required both proper and innovative efficacy assessment.

This proof-of-concept trial is studying the "dynamic" tumor response after the administration of a short course (4 weeks) neoadjuvant combination of gemcitabine and a Hedgehog inhibitor (Vismodegib) before surgery in patients with operable pancreatic cancer.

Detailed Description

Pancreatic cancer is characterized by a high stromal density and is a hypoperfused tumor, precluding cytotoxics delivery to the epithelial tumoral compartment. There is thus a rationale for combining chemotherapy and antistromal drugs like Hedgehog inhibitors. Targeting the resectable primary tumor offers an appropriate setting to (1) evaluate and monitor early treatment effects on the tumor, (2) correlate dynamic imaging changes (perfusion and diffusion coefficient) to pre- and post-therapeutic tissue changes, (3) identify specific predictive biomarkers for the drugs used (i.e. gemcitabine transporters and Hedgehog pathway genes and proteins) and (4) assess if this early "dynamic and biomolecular response" can predict treatment benefit and patient outcome.

Recruitment & Eligibility

Status
UNKNOWN
Sex
All
Target Recruitment
21
Inclusion Criteria
  • Histo(cyto)logically proven ductal pancreatic adenocarcinoma
  • Resectable or potentially resectable tumor; resectability assessed during a multidisciplinary meeting with expert surgeon and radiologist
  • First line chemotherapy
  • Age > 18 years
  • WHO performance status (PS) grade 0 or 1;
  • Absolute neutrophil count > 1.5 x 10 9 / L, platelets > 100 x 10 9/ L, creatinine clearance (Cockcroft and Gault formula) > 60 ml/min, haemoglobin level > 10 g/dl (transfusions authorized), bilirubin<1.5 g/dl;
  • Optimal biliary drainage;
  • Women of child-bearing potential (WCBP), defined as a sexually mature woman who has not undergone a hysterectomy or tubal ligation of who has not been naturally postmenopausal for at least 24 consecutive months, must have a negative serum or urine pregnancy test prior to treatment. All WCBP, all sexually active male patients, and all partners of patients must agree to use adequate methods of birth control throughout the study;
  • Signed written informed consent.
Exclusion Criteria
  • Locally advanced non resectable or metastatic pancreatic adenocarcinoma
  • Previous anticancer therapy for the pancreatic adenocarcinoma
  • Biliary obstruction without endoscopic biliary drainage
  • Any contre-indication for surgery
  • Prior malignancy (except non-melanoma skin cancer, and in situ carcinoma of the uterine cervix treated with a curative intent and any other tumor in complete remission with a disease-free interval > 3 years)
  • Uncontrolled congestive heart failure or angina pectoris, myocardial infarction within 1 year prior to study entry, uncontrolled hypertension (systolic pressure > 160 mm or diastolic pressure > 100 mm under well conducted antihypertensive treatment), QT prolongation
  • Major uncontrolled infection
  • Severe hepatic impairment
  • Any medical, psychological, or social condition, which, in the opinion of the investigator, could hamper patient's compliance to the study protocol and/or assessment/interpretation of the data
  • Pregnant or lactating women, or patients of both genders with procreative potential not using adequate contraceptive methods
  • Patients receiving or having received any investigational treatment within 4 weeks prior to study entry, or participating to another clinical study;Subject previously enrolled into this study.

Study & Design

Study Type
INTERVENTIONAL
Study Design
SINGLE_GROUP
Arm && Interventions
GroupInterventionDescription
Gemcitabine+VismodegibgemcitabineNeoadjuvant chemotherapy combining gemcitabine and Vismodegib during 4 weeks before surgery
Gemcitabine+VismodegibVismodegibNeoadjuvant chemotherapy combining gemcitabine and Vismodegib during 4 weeks before surgery
Gemcitabine+VismodegibNeoadjuvant chemotherapyNeoadjuvant chemotherapy combining gemcitabine and Vismodegib during 4 weeks before surgery
Primary Outcome Measures
NameTimeMethod
"Dynamic" tumor response rate as defined by a 20% modification of tumoral perfusion and cellular density parameters.4 weeks (duration of the neoadjuvant chemotherapy).

In order to detect changes in the tumor microenvironment and to monitor treatment efficacy, Dynamic Contrast-Enhanced-Magnetic Resonance Imaging (DCE-MRI) and Diffusion Weighted-Magnetic Resonance Imaging (DW-MRI) constitute tools more and more used. The acquired data can be analyzed using a pharmacokinetic model to obtain quantitative parameters relative to tissue perfusion and vascular permeability (Ktrans, a volume transfer constant of contrast agent between blood plasma and the extravascular extracellular space; Apparent Diffusion Coefficient as a surrogate marker of tissue cellularity). DCE/DW-MRI will be achieved weekly before each neoadjuvant chemotherapy treatment and before surgery. Each patient will be his/her own control by comparing serial imaging results with those of the baseline MRI.

Secondary Outcome Measures
NameTimeMethod
Number of participants with adverse events as assessed by National Cancer Institute (NCI) Common Toxicity Criteria for Adverse Effects (CTCAE) V4.0.End of study follow-up (up to 2 years).

Number of participants with (serious) adverse events will be considered as a measure of safety of the whole therapeutic sequence (gemcitabine + Hedgehog inhibitor+ surgery).

Trial Locations

Locations (2)

Erasme University Hospital (ULB)

🇧🇪

Brussels, Belgium

Antwerp University Hospital (UZA)

🇧🇪

Edegem, Antwerpen, Belgium

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