The CHARM Trial: Chemotherapy for Ablation and Resolution of Mucinous Pancreatic Cysts
Primary Purpose
Mucinous Pancreatic Cysts
Status
Completed
Phase
Phase 2
Locations
United States
Study Type
Interventional
Intervention
Ethanol
Normal Saline
Chemotherapy
Sponsored by
About this trial
This is an interventional treatment trial for Mucinous Pancreatic Cysts focused on measuring pancreatic cyst, chemotherapy, ethanol, ablation
Eligibility Criteria
Inclusion Criteria:
- Patients age 18 and older of any gender, ethnicity and race
- Voluntary enrollment and ability to give written informed consent
- Capable of safely undergoing endoscopy with deep sedation or general anesthesia
- Patients with previously-detected pancreatic cyst(s) 1-5 cm in diameter, including indeterminate cysts
Exclusion Criteria:
- Pancreatic cyst <1cm or >5cm
- Pancreatic cyst with clear communication with main pancreatic duct
- Clearly benign lesions by clinical and radiographic evaluation (pseudocysts and serous cystadenomas)
- Known or suspected pancreatic cancer or pathologic lymphadenopathy
- Septated cysts with > 5 compartments
- Coagulopathy (international normalized ratio > 1.6, platelets < 30,000)
- Evidence of active pancreatitis or pancreatic infection
- Patients having undergone endoscopic retrograde cholangiopancreatography (ERCP) within 72 hours
- Baseline lab values at the time of consent: white blood cells > 14 or < 2, hematocrit < 30, platelets < 30,000, INR > 1.6, abnormal CA19-9, lipase > 3 times the upper limit of normal, creatinine > 2.5, ALT > 210, total bilirubin > 2.5, positive qualitative beta-hCG.
- Any pre-existing or discovered medical condition that may, at the discretion of the investigator, interfere with the completion of and/or participation in the existing protocol.
- Pregnant, breastfeeding, or incarcerated individuals
Sites / Locations
- Penn State Milton S. Hershey Medical Center
Arms of the Study
Arm 1
Arm 2
Arm Type
Active Comparator
Experimental
Arm Label
Control Group
Study Group
Arm Description
Cyst will be lavaged for 3-5 minutes with Ethanol (alcohol 80%). Following lavage with Ethanol (alcohol 80%), The cyst will be infused with an admixture of chemotherapy drugs (Paclitaxel/ Gemcitabine) 3mg/ml paclitaxel and 19mg/ml gemcitabine.
Cyst will be lavaged for 3-5 minutes with Normal Saline .. Following lavage with Normal Saline, The cyst will be infused with an admixture of chemotherapy drugs (Paclitaxel/ Gemcitabine) 3mg/ml paclitaxel and 19mg/ml gemcitabine.
Outcomes
Primary Outcome Measures
Number of Participants With the Changing in Cyst Volume
The primary outcome of interest will be change in cyst size, as measured on initial, 6, and 12 month CT/MRI, or as determined necessary to evaluate cyst resolution. Cyst size was calculated by measuring x and y diameters and calculating cyst volume using the formula:4/3xpxr3 where r is the average of the cyst radius as measured on the initial, 6-month, and 12-month magnetic resonance imaging or computed tomography. Response was defined according to the same volume percentage reductions as described in previous trials where: complete response is a =>95% reduction in cyst volume, partial response is a 94%-75% reduction, and anon-response is <75% reduction in volume.11 The overall ablation rates in both arms were also compared with historical controls to assess the efficacy of the chemotherapeutic cocktail.
Secondary Outcome Measures
Full Information
NCT ID
NCT01475331
First Posted
November 8, 2011
Last Updated
February 20, 2018
Sponsor
Milton S. Hershey Medical Center
1. Study Identification
Unique Protocol Identification Number
NCT01475331
Brief Title
The CHARM Trial: Chemotherapy for Ablation and Resolution of Mucinous Pancreatic Cysts
Study Type
Interventional
2. Study Status
Record Verification Date
February 2018
Overall Recruitment Status
Completed
Study Start Date
November 2011 (undefined)
Primary Completion Date
January 2017 (Actual)
Study Completion Date
January 2017 (Actual)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Principal Investigator
Name of the Sponsor
Milton S. Hershey Medical Center
4. Oversight
Data Monitoring Committee
Yes
5. Study Description
Brief Summary
The purpose of this study is to evaluate the efficacy of a chemotherapeutic cocktail with or without prior ethanol lavage for ablation of premalignant pancreatic cysts using endoscopic ultrasound-guided fine needle injection (EUS-FNI) for agent delivery.
Detailed Description
While about half of pancreatic cystic lesions have little to no malignant potential, mucinous cystic neoplasms (MCNs) and intraductal papillary mucinous neoplasms (IPMNs) carry a high potential for progression into pancreatic cancer. Therefore, either close radiographic surveillance or surgical resection is generally recommended for these cysts. However, pancreatic surgery is associated with a significant risk of morbidity and mortality and not all patients are good surgical candidates. As an alternative, endoscopic ultrasound-guided fine needle injection(EUS-FNI) has been shown to be moderately effective in ablating cystic lesions.
The goal of this investigation is to improve the efficacy and safety of this procedure when compared with previous versions of the technique. This study is designed to evaluate the efficacy of a chemotherapeutic cocktail with or without prior ethanol lavage for ablation of pancreatic cystic neoplasms using EUS-FNI for agent delivery. This paired, prospective, double-blind, randomized study will include 78 patients referred to the Penn State Hershey Medical Center. Mucinous or indeterminate pancreatic cysts of 1-5cm with less than 5 compartments and without clear communication with the main pancreatic duct will be included. Patients will be randomized in a 1:1 ratio into a control arm or study arm. Patients randomized to the control arm will receive ethanol lavage of the cyst followed by injection of two carefully selected chemotherapeutic agents. Study arm patients will receive a normal saline lavage followed by injection of the same chemotherapy admixture. Patients will be monitored for 2 hours post-procedure, and a follow-up CT will be performed at 6, and 12 months, or as determined necessary to evaluate cyst resolution. Patients may elect to undergo evaluation for surgical resection at any time, regardless of response, in which case a dedicated pathologist will evaluate any surgical specimens.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Mucinous Pancreatic Cysts
Keywords
pancreatic cyst, chemotherapy, ethanol, ablation
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 2
Interventional Study Model
Parallel Assignment
Masking
ParticipantInvestigator
Allocation
Randomized
Enrollment
46 (Actual)
8. Arms, Groups, and Interventions
Arm Title
Control Group
Arm Type
Active Comparator
Arm Description
Cyst will be lavaged for 3-5 minutes with Ethanol (alcohol 80%). Following lavage with Ethanol (alcohol 80%), The cyst will be infused with an admixture of chemotherapy drugs (Paclitaxel/ Gemcitabine) 3mg/ml paclitaxel and 19mg/ml gemcitabine.
Arm Title
Study Group
Arm Type
Experimental
Arm Description
Cyst will be lavaged for 3-5 minutes with Normal Saline .. Following lavage with Normal Saline, The cyst will be infused with an admixture of chemotherapy drugs (Paclitaxel/ Gemcitabine) 3mg/ml paclitaxel and 19mg/ml gemcitabine.
Intervention Type
Drug
Intervention Name(s)
Ethanol
Intervention Description
Cysts will be lavaged for 3-5 minutes with 80% EtOH
Intervention Type
Drug
Intervention Name(s)
Normal Saline
Intervention Description
Cysts will be lavaged for 3-5 minutes with normal saline
Intervention Type
Drug
Intervention Name(s)
Chemotherapy
Intervention Description
Following lavage with either 80% ethanol (control group) or normal saline (study group), cysts will be injected with a cocktail of 3mg/ml paclitaxel and 19mg/ml gemcitabine.
Primary Outcome Measure Information:
Title
Number of Participants With the Changing in Cyst Volume
Description
The primary outcome of interest will be change in cyst size, as measured on initial, 6, and 12 month CT/MRI, or as determined necessary to evaluate cyst resolution. Cyst size was calculated by measuring x and y diameters and calculating cyst volume using the formula:4/3xpxr3 where r is the average of the cyst radius as measured on the initial, 6-month, and 12-month magnetic resonance imaging or computed tomography. Response was defined according to the same volume percentage reductions as described in previous trials where: complete response is a =>95% reduction in cyst volume, partial response is a 94%-75% reduction, and anon-response is <75% reduction in volume.11 The overall ablation rates in both arms were also compared with historical controls to assess the efficacy of the chemotherapeutic cocktail.
Time Frame
6, and 12 months post procedure
10. Eligibility
Sex
All
Minimum Age & Unit of Time
18 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria:
Patients age 18 and older of any gender, ethnicity and race
Voluntary enrollment and ability to give written informed consent
Capable of safely undergoing endoscopy with deep sedation or general anesthesia
Patients with previously-detected pancreatic cyst(s) 1-5 cm in diameter, including indeterminate cysts
Exclusion Criteria:
Pancreatic cyst <1cm or >5cm
Pancreatic cyst with clear communication with main pancreatic duct
Clearly benign lesions by clinical and radiographic evaluation (pseudocysts and serous cystadenomas)
Known or suspected pancreatic cancer or pathologic lymphadenopathy
Septated cysts with > 5 compartments
Coagulopathy (international normalized ratio > 1.6, platelets < 30,000)
Evidence of active pancreatitis or pancreatic infection
Patients having undergone endoscopic retrograde cholangiopancreatography (ERCP) within 72 hours
Baseline lab values at the time of consent: white blood cells > 14 or < 2, hematocrit < 30, platelets < 30,000, INR > 1.6, abnormal CA19-9, lipase > 3 times the upper limit of normal, creatinine > 2.5, ALT > 210, total bilirubin > 2.5, positive qualitative beta-hCG.
Any pre-existing or discovered medical condition that may, at the discretion of the investigator, interfere with the completion of and/or participation in the existing protocol.
Pregnant, breastfeeding, or incarcerated individuals
Facility Information:
Facility Name
Penn State Milton S. Hershey Medical Center
City
Hershey
State/Province
Pennsylvania
ZIP/Postal Code
17033
Country
United States
12. IPD Sharing Statement
Citations:
PubMed Identifier
28802565
Citation
Moyer MT, Sharzehi S, Mathew A, Levenick JM, Headlee BD, Blandford JT, Heisey HD, Birkholz JH, Ancrile BB, Maranki JL, Gusani NJ, McGarrity TJ, Dye CE. The Safety and Efficacy of an Alcohol-Free Pancreatic Cyst Ablation Protocol. Gastroenterology. 2017 Nov;153(5):1295-1303. doi: 10.1053/j.gastro.2017.08.009. Epub 2017 Aug 9.
Results Reference
derived
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The CHARM Trial: Chemotherapy for Ablation and Resolution of Mucinous Pancreatic Cysts
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