Trial Outcomes & Findings for Functional Sucrase Deficiency in Short Bowel Syndrome Patients With Intestinal Failure (NCT NCT04604275)
NCT ID: NCT04604275
Last Updated: 2025-10-01
Results Overview
Degree of carbohydrate malabsorption will be assessed by decrease in number of stools per day.
TERMINATED
PHASE2
3 participants
baseline, 9 weeks
2025-10-01
Participant Flow
Participant milestones
| Measure |
Sucrase Intervention Followed by Placebo
Participants in this arm will receive sucrase for 4 weeks followed by wash out of 1 week with no drug administered then 4 weeks of placebo.
Sucrase: 1 mL (8,500 I.U.) (one full measuring scoop or 28 drops) per meal or snack for patients up to 15 kg in body weight. 2 mL (17,000 I.U.) for patients over 15kg in body weight. Dosage is 1 or 2 mL (8,500 to 17,000 I.U.) taken orally or by g-tube with each meal or snack diluted in water, milk, or infant formula.
Placebo: 1 mL of placebo per meal or snack for patients up to 15 kg in body weight. 2 mL of placebo per meal of snack for patients above 15kg in body weight. Dosage is 1 or 2 mL of placebo taken orally or by g-tube with each meal or snack diluted in water, milk, or infant formula.
|
Placebo Followed by Sucrase Intervention
Participants in this arm will receive placebo for 4 weeks followed by wash out of 1 week with no drug administered then 4 weeks of sucrase.
Sucrase: 1 mL (8,500 I.U.) (one full measuring scoop or 28 drops) per meal or snack for patients up to 15 kg in body weight. 2 mL (17,000 I.U.) for patients over 15kg in body weight. Dosage is 1 or 2 mL (8,500 to 17,000 I.U.) taken orally or by g-tube with each meal or snack diluted in water, milk, or infant formula.
Placebo: 1 mL of placebo per meal or snack for patients up to 15 kg in body weight. 2 mL of placebo per meal of snack for patients above 15kg in body weight. Dosage is 1 or 2 mL of placebo taken orally or by g-tube with each meal or snack diluted in water, milk, or infant formula.
|
|---|---|---|
|
Intervention 1
STARTED
|
2
|
1
|
|
Intervention 1
COMPLETED
|
2
|
1
|
|
Intervention 1
NOT COMPLETED
|
0
|
0
|
|
Washout-1 Week
STARTED
|
2
|
1
|
|
Washout-1 Week
COMPLETED
|
1
|
1
|
|
Washout-1 Week
NOT COMPLETED
|
1
|
0
|
|
Intervention 2
STARTED
|
1
|
1
|
|
Intervention 2
COMPLETED
|
1
|
1
|
|
Intervention 2
NOT COMPLETED
|
0
|
0
|
Reasons for withdrawal
| Measure |
Sucrase Intervention Followed by Placebo
Participants in this arm will receive sucrase for 4 weeks followed by wash out of 1 week with no drug administered then 4 weeks of placebo.
Sucrase: 1 mL (8,500 I.U.) (one full measuring scoop or 28 drops) per meal or snack for patients up to 15 kg in body weight. 2 mL (17,000 I.U.) for patients over 15kg in body weight. Dosage is 1 or 2 mL (8,500 to 17,000 I.U.) taken orally or by g-tube with each meal or snack diluted in water, milk, or infant formula.
Placebo: 1 mL of placebo per meal or snack for patients up to 15 kg in body weight. 2 mL of placebo per meal of snack for patients above 15kg in body weight. Dosage is 1 or 2 mL of placebo taken orally or by g-tube with each meal or snack diluted in water, milk, or infant formula.
|
Placebo Followed by Sucrase Intervention
Participants in this arm will receive placebo for 4 weeks followed by wash out of 1 week with no drug administered then 4 weeks of sucrase.
Sucrase: 1 mL (8,500 I.U.) (one full measuring scoop or 28 drops) per meal or snack for patients up to 15 kg in body weight. 2 mL (17,000 I.U.) for patients over 15kg in body weight. Dosage is 1 or 2 mL (8,500 to 17,000 I.U.) taken orally or by g-tube with each meal or snack diluted in water, milk, or infant formula.
Placebo: 1 mL of placebo per meal or snack for patients up to 15 kg in body weight. 2 mL of placebo per meal of snack for patients above 15kg in body weight. Dosage is 1 or 2 mL of placebo taken orally or by g-tube with each meal or snack diluted in water, milk, or infant formula.
|
|---|---|---|
|
Washout-1 Week
Withdrawal by Subject
|
1
|
0
|
Baseline Characteristics
Functional Sucrase Deficiency in Short Bowel Syndrome Patients With Intestinal Failure
Baseline characteristics by cohort
| Measure |
Sucrase Intervention Followed by Placebo
n=2 Participants
Participants in this arm will receive sucrase for 4 weeks followed by wash out of 1 week with no drug administered then 4 weeks of placebo.
Sucrase: 1 mL (8,500 I.U.) (one full measuring scoop or 28 drops) per meal or snack for patients up to 15 kg in body weight. 2 mL (17,000 I.U.) for patients over 15kg in body weight. Dosage is 1 or 2 mL (8,500 to 17,000 I.U.) taken orally or by g-tube with each meal or snack diluted in water, milk, or infant formula.
Placebo: 1 mL of placebo per meal or snack for patients up to 15 kg in body weight. 2 mL of placebo per meal of snack for patients above 15kg in body weight. Dosage is 1 or 2 mL of placebo taken orally or by g-tube with each meal or snack diluted in water, milk, or infant formula.
|
Placebo Followed by Sucrase Intervention
n=1 Participants
Participants in this arm will receive placebo for 4 weeks followed by wash out of 1 week with no drug administered then 4 weeks of sucrase.
Sucrase: 1 mL (8,500 I.U.) (one full measuring scoop or 28 drops) per meal or snack for patients up to 15 kg in body weight. 2 mL (17,000 I.U.) for patients over 15kg in body weight. Dosage is 1 or 2 mL (8,500 to 17,000 I.U.) taken orally or by g-tube with each meal or snack diluted in water, milk, or infant formula.
Placebo: 1 mL of placebo per meal or snack for patients up to 15 kg in body weight. 2 mL of placebo per meal of snack for patients above 15kg in body weight. Dosage is 1 or 2 mL of placebo taken orally or by g-tube with each meal or snack diluted in water, milk, or infant formula.
|
Total
n=3 Participants
Total of all reporting groups
|
|---|---|---|---|
|
Age, Categorical
<=18 years
|
2 Participants
n=5 Participants
|
1 Participants
n=7 Participants
|
3 Participants
n=5 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Age, Categorical
>=65 years
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Sex: Female, Male
Female
|
1 Participants
n=5 Participants
|
1 Participants
n=7 Participants
|
2 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
1 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
1 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
2 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
2 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
0 Participants
n=5 Participants
|
1 Participants
n=7 Participants
|
1 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 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
|
0 Participants
n=5 Participants
|
1 Participants
n=7 Participants
|
1 Participants
n=5 Participants
|
|
Race (NIH/OMB)
White
|
2 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
2 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
|
PRIMARY outcome
Timeframe: baseline, 9 weeksPopulation: Due to operational challenges across multiple study sites, the required data for this outcome measure could not be collected or retained. The study involved several coordinators working across dispersed locations, which introduced variability in data handling and documentation practices. Although standardized procedures were in place, inconsistencies emerged in how forms were distributed, tracked, and returned.
Degree of carbohydrate malabsorption will be assessed by decrease in number of stools per day.
Outcome measures
Outcome data not reported
PRIMARY outcome
Timeframe: baseline, 9 weeksPopulation: Due to operational challenges across multiple study sites, the required data for this outcome measure could not be collected or retained. The study involved several coordinators working across dispersed locations, which introduced variability in data handling and documentation practices. Although standardized procedures were in place, inconsistencies emerged in how forms were distributed, tracked, and returned.
Degree of carbohydrate malabsorption will be assessed by change in patient symptomatology by change in score on patient symptom survey. The survey has range from 0-52 with higher score being worse symptoms and lower being better.
Outcome measures
Outcome data not reported
PRIMARY outcome
Timeframe: baseline, 9 weeksPopulation: Due to operational challenges across multiple study sites, the required data for this outcome measure could not be collected or retained. The study involved several coordinators working across dispersed locations, which introduced variability in data handling and documentation practices. Although standardized procedures were in place, inconsistencies emerged in how forms were distributed, tracked, and returned.
Carbohydrate malabsorption will be measured by increase in growth velocity in kg/week
Outcome measures
Outcome data not reported
PRIMARY outcome
Timeframe: baseline, 9 weeksPopulation: Due to operational challenges across multiple study sites, the required data for this outcome measure could not be collected or retained. The study involved several coordinators working across dispersed locations, which introduced variability in data handling and documentation practices. Although standardized procedures were in place, inconsistencies emerged in how forms were distributed, tracked, and returned.
Carbohydrate malabsorption will be measured by ability to advance enteral nutrition in ml/day
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: baseline, 9 weeksPopulation: Due to operational challenges across multiple study sites, the required data for this outcome measure could not be collected or retained. The study involved several coordinators working across dispersed locations, which introduced variability in data handling and documentation practices. Although standardized procedures were in place, inconsistencies emerged in how forms were distributed, tracked, and returned.
Change in digestion will be measured by change in abdominal distension/girth measured in cm
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: baseline, 9 weeksPopulation: Due to operational challenges across multiple study sites, the required data for this outcome measure could not be collected or retained. The study involved several coordinators working across dispersed locations, which introduced variability in data handling and documentation practices. Although standardized procedures were in place, inconsistencies emerged in how forms were distributed, tracked, and returned.
Change in digestion will be assessed by number of emesis per day
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: baseline, 9 weeksPopulation: Due to operational challenges across multiple study sites, the required data for this outcome measure could not be collected or retained. The study involved several coordinators working across dispersed locations, which introduced variability in data handling and documentation practices. Although standardized procedures were in place, inconsistencies emerged in how forms were distributed, tracked, and returned.
Change in digestion will be assessed as a change in stool consistency from liquid (7) to solid(1) using Bristol stool chart
Outcome measures
Outcome data not reported
Adverse Events
Sucrase Intervention Followed by Placebo
Placebo Followed by Sucrase Intervention
Serious adverse events
Adverse event data not reported
Other adverse events
Adverse event data not reported
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place