Trial Outcomes & Findings for Efficacy, Safety and Tolerability of Enteric-Coated Cholestyramine Capsules for Adult Short Bowel Syndrome (NCT NCT04046328)
NCT ID: NCT04046328
Last Updated: 2022-07-19
Results Overview
Change in the weekly frequency of bowel movements measured between baseline and the second week of treatment. Baseline is defined as the second week of screening for treatment period 1 and second week of washout for treatment period 2.
Recruitment status
TERMINATED
Study phase
PHASE2
Target enrollment
13 participants
Primary outcome timeframe
Baseline and Week 2 of treatment (Days 8 to 14, and Days 36 to 42)
Results posted on
2022-07-19
Participant Flow
This was a multiple center, randomized, double-blind, double dummy, 2-period, 2-sequence cross-over study design planned to evaluate 18 patients at three study centers. Due to early study termination only 13 patients were randomized and received treatment with ECC.
Participant milestones
| Measure |
"Low" Dose ECC Regimen, Then "High" Dose ECC Regimen
"Low" Dose: ECC at the 1.7 g daily dose, administered BID as 2 capsules of ECC, plus 3 capsules of placebo, at least 30 minutes before breakfast and 2 capsules of ECC, plus 3 capsules of placebo at least 30 minutes before evening meal.
"High" Dose: ECC at the 4.25 g daily dose, administered BID as 5 capsules of ECC at least 30 minutes before breakfast and 5 capsules of ECC at least 30 minutes before evening meal.
Enteric-Coated Cholestyramine (ECC) Capsule: Enteric-Coated Delayed Release Cholestyramine Capsules
Placebo: Enteric-Coated Delayed Release Placebo Capsules
|
"High" Dose ECC Regimen, Then "Low" Dose ECC Regimen
"High" Dose: ECC at the 4.25 g daily dose, administered BID as 5 capsules of ECC at least 30 minutes before breakfast and 5 capsules of ECC at least 30 minutes before evening meal.
"Low" Dose: ECC at the 1.7 g daily dose, administered BID as 2 capsules of ECC, plus 3 capsules of placebo, at least 30 minutes before breakfast and 2 capsules of ECC, plus 3 capsules of placebo at least 30 minutes before evening meal.
Enteric-Coated Cholestyramine (ECC) Capsule: Enteric-Coated Delayed Release Cholestyramine Capsules
Placebo: Enteric-Coated Delayed Release Placebo Capsules
|
|---|---|---|
|
First Intervention (14 Days)
STARTED
|
6
|
7
|
|
First Intervention (14 Days)
COMPLETED
|
6
|
7
|
|
First Intervention (14 Days)
NOT COMPLETED
|
0
|
0
|
|
Washout (14 Days)
STARTED
|
6
|
7
|
|
Washout (14 Days)
COMPLETED
|
6
|
7
|
|
Washout (14 Days)
NOT COMPLETED
|
0
|
0
|
|
Second Intervention (14 Days)
STARTED
|
6
|
7
|
|
Second Intervention (14 Days)
COMPLETED
|
6
|
7
|
|
Second Intervention (14 Days)
NOT COMPLETED
|
0
|
0
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
Efficacy, Safety and Tolerability of Enteric-Coated Cholestyramine Capsules for Adult Short Bowel Syndrome
Baseline characteristics by cohort
| Measure |
Intent-to-Treat (ITT Population)
n=13 Participants
Demographics and baseline characteristics were assessed for the overall ITT Population
|
|---|---|
|
Age, Continuous
|
54.9 years
STANDARD_DEVIATION 15.98 • n=5 Participants
|
|
Sex: Female, Male
Female
|
6 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
7 Participants
n=5 Participants
|
|
Race/Ethnicity, Customized
Asian
|
0 Participants
n=5 Participants
|
|
Race/Ethnicity, Customized
Black
|
0 Participants
n=5 Participants
|
|
Race/Ethnicity, Customized
Caucasian
|
13 Participants
n=5 Participants
|
|
Race/Ethnicity, Customized
Other
|
0 Participants
n=5 Participants
|
|
Region of Enrollment
Poland
|
13 participants
n=5 Participants
|
|
Body Mass Index (BMI)
|
24.1 kg/m^2
STANDARD_DEVIATION 3.84 • n=5 Participants
|
PRIMARY outcome
Timeframe: Baseline and Week 2 of treatment (Days 8 to 14, and Days 36 to 42)Change in the weekly frequency of bowel movements measured between baseline and the second week of treatment. Baseline is defined as the second week of screening for treatment period 1 and second week of washout for treatment period 2.
Outcome measures
| Measure |
"Low" Dose ECC Regimen
n=13 Participants
ECC at the 1.7 g daily dose, administered BID as 2 capsules of ECC, plus 3 capsules of placebo, at least 30 minutes before breakfast and 2 capsules of ECC, plus 3 capsules of placebo at least 30 minutes before evening meal.
Enteric-Coated Cholestyramine (ECC) Capsule: Enteric-Coated Delayed Release Cholestyramine Capsules
Placebo: Enteric-Coated Delayed Release Placebo Capsules
|
"High" Dose ECC Regimen
n=13 Participants
ECC at the 4.25 g daily dose, administered BID as 5 capsules of ECC at least 30 minutes before breakfast and 5 capsules of ECC at least 30 minutes before evening meal.
Enteric-Coated Cholestyramine (ECC) Capsule: Enteric-Coated Delayed Release Cholestyramine Capsules
|
|---|---|---|
|
Change in the Weekly Frequency of Bowel Movements Measured Between Baseline and the Second Week of Treatment
|
-11.5 Weekly bowel movements
Standard Deviation 11.38
|
-13.4 Weekly bowel movements
Standard Deviation 9.73
|
SECONDARY outcome
Timeframe: Days 1 to 14 and Days 29 to 42Outcome measures
| Measure |
"Low" Dose ECC Regimen
n=13 Participants
ECC at the 1.7 g daily dose, administered BID as 2 capsules of ECC, plus 3 capsules of placebo, at least 30 minutes before breakfast and 2 capsules of ECC, plus 3 capsules of placebo at least 30 minutes before evening meal.
Enteric-Coated Cholestyramine (ECC) Capsule: Enteric-Coated Delayed Release Cholestyramine Capsules
Placebo: Enteric-Coated Delayed Release Placebo Capsules
|
"High" Dose ECC Regimen
n=13 Participants
ECC at the 4.25 g daily dose, administered BID as 5 capsules of ECC at least 30 minutes before breakfast and 5 capsules of ECC at least 30 minutes before evening meal.
Enteric-Coated Cholestyramine (ECC) Capsule: Enteric-Coated Delayed Release Cholestyramine Capsules
|
|---|---|---|
|
Total Number of Bowel Movements for the Whole 2-week Treatment Period
|
52.5 Bowel movements
Standard Deviation 20.12
|
55.1 Bowel movements
Standard Deviation 25.18
|
SECONDARY outcome
Timeframe: Days 8 to 14, and Days 36 to 42The BSFS classifies the form of human feces into seven categories (Type 1 to Type 7) based on stool shape and consistency. Types or scores of 1 and 2 indicate constipation, with 3 and 4 being the ideal stools as they are easy to defecate while not containing excess liquid, 5 tending towards diarrhea, and 6 and 7 indicate diarrhea.
Outcome measures
| Measure |
"Low" Dose ECC Regimen
n=13 Participants
ECC at the 1.7 g daily dose, administered BID as 2 capsules of ECC, plus 3 capsules of placebo, at least 30 minutes before breakfast and 2 capsules of ECC, plus 3 capsules of placebo at least 30 minutes before evening meal.
Enteric-Coated Cholestyramine (ECC) Capsule: Enteric-Coated Delayed Release Cholestyramine Capsules
Placebo: Enteric-Coated Delayed Release Placebo Capsules
|
"High" Dose ECC Regimen
n=13 Participants
ECC at the 4.25 g daily dose, administered BID as 5 capsules of ECC at least 30 minutes before breakfast and 5 capsules of ECC at least 30 minutes before evening meal.
Enteric-Coated Cholestyramine (ECC) Capsule: Enteric-Coated Delayed Release Cholestyramine Capsules
|
|---|---|---|
|
Mean Daily Stool Form Score According to the BSFS (Bristol Stool Form Scale), Measured During the Second Week of Treatment
|
3.6 Score on BSFS scale
Standard Deviation 1.42
|
3.7 Score on BSFS scale
Standard Deviation 1.76
|
SECONDARY outcome
Timeframe: Days 8 to 14, and Days 36 to 42Outcome measures
| Measure |
"Low" Dose ECC Regimen
n=7 Participants
ECC at the 1.7 g daily dose, administered BID as 2 capsules of ECC, plus 3 capsules of placebo, at least 30 minutes before breakfast and 2 capsules of ECC, plus 3 capsules of placebo at least 30 minutes before evening meal.
Enteric-Coated Cholestyramine (ECC) Capsule: Enteric-Coated Delayed Release Cholestyramine Capsules
Placebo: Enteric-Coated Delayed Release Placebo Capsules
|
"High" Dose ECC Regimen
n=7 Participants
ECC at the 4.25 g daily dose, administered BID as 5 capsules of ECC at least 30 minutes before breakfast and 5 capsules of ECC at least 30 minutes before evening meal.
Enteric-Coated Cholestyramine (ECC) Capsule: Enteric-Coated Delayed Release Cholestyramine Capsules
|
|---|---|---|
|
Mean Daily Dose of Loperamide in mg, if Used, During the Second Week of Treatment
|
72.9 mg
Standard Deviation 43.05
|
68.9 mg
Standard Deviation 53.19
|
Adverse Events
"Low" Dose ECC Regimen
Serious events: 0 serious events
Other events: 3 other events
Deaths: 0 deaths
"High" Dose ECC Regimen
Serious events: 0 serious events
Other events: 4 other events
Deaths: 0 deaths
Serious adverse events
Adverse event data not reported
Other adverse events
| Measure |
"Low" Dose ECC Regimen
n=13 participants at risk
ECC at the 1.7 g daily dose, administered BID as 2 capsules of ECC, plus 3 capsules of placebo, at least 30 minutes before breakfast and 2 capsules of ECC, plus 3 capsules of placebo at least 30 minutes before evening meal.
Enteric-Coated Cholestyramine (ECC) Capsule: Enteric-Coated Delayed Release Cholestyramine Capsules
Placebo: Enteric-Coated Delayed Release Placebo Capsules
|
"High" Dose ECC Regimen
n=13 participants at risk
ECC at the 4.25 g daily dose, administered BID as 5 capsules of ECC at least 30 minutes before breakfast and 5 capsules of ECC at least 30 minutes before evening meal.
Enteric-Coated Cholestyramine (ECC) Capsule: Enteric-Coated Delayed Release Cholestyramine Capsules
|
|---|---|---|
|
Gastrointestinal disorders
Abdominal pain upper
|
7.7%
1/13 • From patient randomization to the end of study visit, approximately 44 days
|
7.7%
1/13 • From patient randomization to the end of study visit, approximately 44 days
|
|
General disorders
Oedema peripheral
|
7.7%
1/13 • From patient randomization to the end of study visit, approximately 44 days
|
7.7%
1/13 • From patient randomization to the end of study visit, approximately 44 days
|
|
Infections and infestations
Pulpitis dental
|
7.7%
1/13 • From patient randomization to the end of study visit, approximately 44 days
|
7.7%
1/13 • From patient randomization to the end of study visit, approximately 44 days
|
|
Investigations
C-reactive protein increased
|
0.00%
0/13 • From patient randomization to the end of study visit, approximately 44 days
|
7.7%
1/13 • From patient randomization to the end of study visit, approximately 44 days
|
|
Metabolism and nutrition disorders
Hypokalaemia
|
7.7%
1/13 • From patient randomization to the end of study visit, approximately 44 days
|
0.00%
0/13 • From patient randomization to the end of study visit, approximately 44 days
|
|
Metabolism and nutrition disorders
Hypomagnesaemia
|
0.00%
0/13 • From patient randomization to the end of study visit, approximately 44 days
|
7.7%
1/13 • From patient randomization to the end of study visit, approximately 44 days
|
|
Metabolism and nutrition disorders
Decreased appetite
|
0.00%
0/13 • From patient randomization to the end of study visit, approximately 44 days
|
7.7%
1/13 • From patient randomization to the end of study visit, approximately 44 days
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee Publication of the study results by the Investigator, the Center and by any member of the Investigational Team shall require a prior express approval of Pharmascience in writing.
- Publication restrictions are in place
Restriction type: OTHER