Trial Outcomes & Findings for The CHARM Trial: Chemotherapy for Ablation and Resolution of Mucinous Pancreatic Cysts (NCT NCT01475331)
NCT ID: NCT01475331
Last Updated: 2018-03-20
Results Overview
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.
Recruitment status
COMPLETED
Study phase
PHASE2
Target enrollment
46 participants
Primary outcome timeframe
6, and 12 months post procedure
Results posted on
2018-03-20
Participant Flow
Participant milestones
| Measure |
Control Group
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(Paclitaxel/ Gemcitabine) 3mg/ml paclitaxel and 19mg/ml gemcitabine.
Ethanol: Cysts will be lavaged for 3-5 minutes with 80% EtOH
Chemotherapy: 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
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(Paclitaxel/ Gemcitabine) 3mg/ml paclitaxel and 19mg/ml gemcitabine.
Normal Saline: Cysts will be lavaged for 3-5 minutes with normal saline
Chemotherapy: 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.
|
|---|---|---|
|
Overall Study
STARTED
|
23
|
23
|
|
Overall Study
COMPLETED
|
18
|
21
|
|
Overall Study
NOT COMPLETED
|
5
|
2
|
Reasons for withdrawal
| Measure |
Control Group
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(Paclitaxel/ Gemcitabine) 3mg/ml paclitaxel and 19mg/ml gemcitabine.
Ethanol: Cysts will be lavaged for 3-5 minutes with 80% EtOH
Chemotherapy: 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
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(Paclitaxel/ Gemcitabine) 3mg/ml paclitaxel and 19mg/ml gemcitabine.
Normal Saline: Cysts will be lavaged for 3-5 minutes with normal saline
Chemotherapy: 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.
|
|---|---|---|
|
Overall Study
Osteophyte obstruction of the proximal
|
1
|
0
|
|
Overall Study
excessive main duct communication
|
1
|
1
|
|
Overall Study
Loss of needle positioning due pt. cough
|
1
|
0
|
|
Overall Study
Sign of malignancy during the procedure
|
2
|
0
|
|
Overall Study
cyst found to be prohibitively small
|
0
|
1
|
Baseline Characteristics
The CHARM Trial: Chemotherapy for Ablation and Resolution of Mucinous Pancreatic Cysts
Baseline characteristics by cohort
| Measure |
Control Group
n=18 Participants
Ethanol: Cysts will be lavaged for 3-5 minutes with 80% EtOH
Chemotherapy: 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
n=21 Participants
Normal Saline: Cysts will be lavaged for 3-5 minutes with normal saline
Chemotherapy: 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.
|
Total
n=39 Participants
Total of all reporting groups
|
|---|---|---|---|
|
Age, Categorical
<=18 years
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
4 Participants
n=5 Participants
|
8 Participants
n=7 Participants
|
12 Participants
n=5 Participants
|
|
Age, Categorical
>=65 years
|
14 Participants
n=5 Participants
|
13 Participants
n=7 Participants
|
27 Participants
n=5 Participants
|
|
Sex: Female, Male
Female
|
13 Participants
n=5 Participants
|
10 Participants
n=7 Participants
|
23 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
5 Participants
n=5 Participants
|
11 Participants
n=7 Participants
|
16 Participants
n=5 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=5 Participants
|
1 Participants
n=7 Participants
|
1 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Asian
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Black or African American
|
2 Participants
n=5 Participants
|
1 Participants
n=7 Participants
|
3 Participants
n=5 Participants
|
|
Race (NIH/OMB)
White
|
16 Participants
n=5 Participants
|
19 Participants
n=7 Participants
|
35 Participants
n=5 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Region of Enrollment
United States
|
18 Participants
n=5 Participants
|
21 Participants
n=7 Participants
|
39 Participants
n=5 Participants
|
PRIMARY outcome
Timeframe: 6, and 12 months post procedurePopulation: Intent to treat population (all participants at least one dose of intervention)
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.
Outcome measures
| Measure |
Control Group
n=18 Participants
Ethanol: Cysts will be lavaged for 3-5 minutes with 80% EtOH
Chemotherapy: 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
n=21 Participants
Normal Saline: Cysts will be lavaged for 3-5 minutes with normal saline
Chemotherapy: 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.
|
|---|---|---|
|
Number of Participants With the Changing in Cyst Volume
Complete Response after 6 mon
|
6 Participants
|
12 Participants
|
|
Number of Participants With the Changing in Cyst Volume
Partial Response after 6 months
|
5 Participants
|
4 Participants
|
|
Number of Participants With the Changing in Cyst Volume
No Response after 6 months
|
6 Participants
|
5 Participants
|
|
Number of Participants With the Changing in Cyst Volume
Complete Response after 12 months
|
11 Participants
|
14 Participants
|
|
Number of Participants With the Changing in Cyst Volume
Partial Response after 12 months
|
4 Participants
|
3 Participants
|
|
Number of Participants With the Changing in Cyst Volume
No Response after 12 months
|
3 Participants
|
4 Participants
|
Adverse Events
Control Group
Serious events: 1 serious events
Other events: 4 other events
Deaths: 0 deaths
Study Group
Serious events: 0 serious events
Other events: 0 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
Control Group
n=18 participants at risk
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(Paclitaxel/ Gemcitabine) 3mg/ml paclitaxel and 19mg/ml gemcitabine.
Ethanol: Cysts will be lavaged for 3-5 minutes with 80% EtOH
Chemotherapy: 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
n=21 participants at risk
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(Paclitaxel/ Gemcitabine) 3mg/ml paclitaxel and 19mg/ml gemcitabine.
Normal Saline: Cysts will be lavaged for 3-5 minutes with normal saline
Chemotherapy: 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.
|
|---|---|---|
|
Gastrointestinal disorders
acute pancreatitis
|
5.6%
1/18 • Number of events 1 • Adverse events were determined and collected through study completion, an average of 1 year.
|
0.00%
0/21 • Adverse events were determined and collected through study completion, an average of 1 year.
|
Other adverse events
| Measure |
Control Group
n=18 participants at risk
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(Paclitaxel/ Gemcitabine) 3mg/ml paclitaxel and 19mg/ml gemcitabine.
Ethanol: Cysts will be lavaged for 3-5 minutes with 80% EtOH
Chemotherapy: 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
n=21 participants at risk
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(Paclitaxel/ Gemcitabine) 3mg/ml paclitaxel and 19mg/ml gemcitabine.
Normal Saline: Cysts will be lavaged for 3-5 minutes with normal saline
Chemotherapy: 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.
|
|---|---|---|
|
Gastrointestinal disorders
abdominal pain
|
22.2%
4/18 • Number of events 4 • Adverse events were determined and collected through study completion, an average of 1 year.
|
0.00%
0/21 • Adverse events were determined and collected through study completion, an average of 1 year.
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place