Mucinous Pancreatic CystsPhase 2NCT01475331
The CHARM Trial: Chemotherapy for Ablation and Resolution of Mucinous Pancreatic Cysts
A Phase 2 study testing Ethanol, Normal Saline and Chemotherapy in people with Mucinous Pancreatic Cysts, run by Milton S. Hershey Medical Center at 1 site in United States.
Trial 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. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Mucinous Pancreatic Cysts
- What is being tested?
- Ethanol, Normal Saline and Chemotherapy
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 46 participants
- Where
- 1 siteUnited States
- Study dates
- Nov 2011to Jan 2017
- Phase
- Phase 2
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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
Who cannot join
- 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)
Show all 11Show fewer
- 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
Trial locations and contacts
1 site in 1 country.
United States1 site
Penn State Milton S. Hershey Medical Center
Hershey, Pennsylvania, 17033
About this study
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.
Read the 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.
- Conditions
- Mucinous Pancreatic Cysts
- Being tested
- Ethanol, Normal Saline, Chemotherapy
- Keywords
- pancreatic cyst, chemotherapy, ethanol, ablation
- Medical subject headings
- Pancreatic Cyst
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
EthanolDrug
Cysts will be lavaged for 3-5 minutes with 80% EtOH
Normal SalineDrug
Cysts will be lavaged for 3-5 minutes with normal saline
ChemotherapyDrug
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.
| Study group | Role | What they receive |
|---|---|---|
| Control Group | Active comparator | 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. Receives: Drug: Ethanol; Drug: Chemotherapy |
| Study Group | Experimental | 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. Receives: Drug: Normal Saline; Drug: Chemotherapy |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: 6, and 12 months post procedure
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted21 Nov 2011 (estimated)
- Study startNov 2011
- Primary completionJan 2017
- Study completionJan 2017
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Mucinous Pancreatic CystsStudies of the same condition, open and under way.
- More trials run by Milton S. Hershey Medical CenterThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Double blind
- Who is masked
- Participant, Investigator
- Enrolment
- 46 participants (actual)
Sponsor and investigators
- Lead sponsor
- Milton S. Hershey Medical Center
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Matthew T. Moyer, Milton S. Hershey Medical Center
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01475331
- Sponsor's protocol ID
- IRB 33751
- Status last verified
- Feb 2018
- First submitted
- 8 Nov 2011
- Last updated
- 20 Mar 2018
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
Publications
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.
PubMed 28802565
Study at a glance
From the registry record, last updated by the sponsor on 20 Mar 2018. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- The CHARM Trial: Chemotherapy for Ablation and Resolution of Mucinous Pancreatic Cysts
- ClinicalTrials.gov ID
- NCT01475331
- Acronym / sponsor ID
- IRB 33751
- Conditions
- Mucinous Pancreatic Cysts
- Keywords
- pancreatic cyst, chemotherapy, ethanol, ablation
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 46 participants (actual)
- Lead sponsor
- Milton S. Hershey Medical CenterUniversity, hospital or other
- Study start
- Nov 2011
- Primary completion
- Jan 2017 (actual)
- Study completion
- Jan 2017 (actual)
- First posted
- 21 Nov 2011 (estimated)
- Results
- Posted 20 Mar 2018
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Double blind (participant, investigator)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Control Group | Active comparator | Drug: Ethanol, Drug: ChemotherapyCyst 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. |
| Study Group | Experimental | Drug: Normal Saline, Drug: ChemotherapyCyst 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. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (4 listed)
- 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
Key exclusion criteria (11 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| EthanolCysts will be lavaged for 3-5 minutes with 80% EtOH | Drug | Control Group |
| Normal SalineCysts will be lavaged for 3-5 minutes with normal saline | Drug | Study Group |
| ChemotherapyFollowing 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. | Drug | Control Group, Study Group |
Primary outcome
Number of Participants With the Changing in Cyst Volume
Time frame: 6, and 12 months post procedure
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.
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 20 Mar 2018.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Derived from this study: PubMed 28802565Moyer 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.
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Pancreatic Cyst
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Ethanol, Normal Saline and Chemotherapy in people with Mucinous Pancreatic Cysts, run by Milton S. Hershey Medical Center at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 4 inclusion and 11 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 20 Mar 2018. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 20 Mar 2018). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Number of Participants With the Changing in Cyst Volume (time frame: 6, and 12 months post procedure). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT01475331, which also lists 1 linked publication.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 20 Mar 2018.