Short Gut Syndrome
Conditions
Keywords
Intestinal failure
Brief summary
Short gut syndrome with intestinal failure patients may have decreased production of disaccharidases, like sucrase, an enzyme responsible for digesting sugar in foods. This can happen due to loss of bowel length from surgery or from loss of cellular function in the intestines due to use of parenteral nutrition intravenously. Therefore, patients with these conditions may not be able to digest sucrose (sugar) fully. Patients might experience abdominal distension/pain, vomiting and diarrhea when sugar is taken in orally or through the g-tube, which can limit patients' ability to increase oral or g-tube feeds in short gut syndrome patients with intestinal failure. In patients with short gut syndrome and intestinal failure, the administration of exogenous sucrase (enzyme) may improve sucrose (sugar) digestion and thus the ability to tolerate more oral or g-tube feeds.
Interventions
DRUGSucrase
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.
OTHERPlacebo
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.
Sponsors
QOL Medical, LLC
University of Miami
Study design
Allocation
RANDOMIZED
Intervention model
CROSSOVER
Primary purpose
TREATMENT
Masking
QUADRUPLE (Subject, Caregiver, Investigator, Outcomes Assessor)
Eligibility
Sex/Gender
ALL
Healthy volunteers
No
Inclusion criteria
* Short bowel syndrome, of all ages, with dependence on parental support to provide at least 50% of fluid or caloric needs. * Must be on diet containing sucrose. * Must be willing and able to sign informed consent * Adult and Pediatric patients (all ages)
Exclusion criteria
* Current IV antibiotic administration for confirmed bout of bacteremia. * No enteral nutrition * Any condition, disease, illness, or circumstance that in the investigator's opinion puts the subject at any undue risk, prevents completion of the study, or interferes with analysis of the study results
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Change in Carbohydrate Malabsorption | baseline, 9 weeks | Degree of carbohydrate malabsorption will be assessed by decrease in number of stools per day. |
| Change in Carbohydrate Malabsorption as Measured by Patient Symptom Survey | baseline, 9 weeks | 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. |
| Change in Carbohydrate Malabsorption as Measured by Growth Velocity | baseline, 9 weeks | Carbohydrate malabsorption will be measured by increase in growth velocity in kg/week |
| Change in Carbohydrate Malabsorption as Measured by Enteral Nutrition Tolerance | baseline, 9 weeks | Carbohydrate malabsorption will be measured by ability to advance enteral nutrition in ml/day |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Change in Digestion as Measured by Amount of Emesis | baseline, 9 weeks | Change in digestion will be assessed by number of emesis per day |
| Change in Digestion as Measured by Stool Consistency | baseline, 9 weeks | Change in digestion will be assessed as a change in stool consistency from liquid (7) to solid(1) using Bristol stool chart |
| Change in Digestion | baseline, 9 weeks | Change in digestion will be measured by change in abdominal distension/girth measured in cm |
Countries
United States
Participant flow
Participants by arm
| Arm | Count |
|---|---|
| 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. | 2 |
| 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. | 1 |
| Total | 3 |
Withdrawals & dropouts
| Period | Reason | FG000 | FG001 |
|---|---|---|---|
| Washout-1 Week | Withdrawal by Subject | 1 | 0 |
Baseline characteristics
| Characteristic | Sucrase Intervention Followed by Placebo | Placebo Followed by Sucrase Intervention | Total |
|---|---|---|---|
| Age, Categorical <=18 years | 2 Participants | 1 Participants | 3 Participants |
| Age, Categorical >=65 years | 0 Participants | 0 Participants | 0 Participants |
| Age, Categorical Between 18 and 65 years | 0 Participants | 0 Participants | 0 Participants |
| Ethnicity (NIH/OMB) Hispanic or Latino | 2 Participants | 0 Participants | 2 Participants |
| Ethnicity (NIH/OMB) Not Hispanic or Latino | 0 Participants | 1 Participants | 1 Participants |
| Ethnicity (NIH/OMB) Unknown or Not Reported | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) American Indian or Alaska Native | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) Asian | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) Black or African American | 0 Participants | 1 Participants | 1 Participants |
| Race (NIH/OMB) More than one race | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) Native Hawaiian or Other Pacific Islander | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) Unknown or Not Reported | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) White | 2 Participants | 0 Participants | 2 Participants |
| Sex: Female, Male Female | 1 Participants | 1 Participants | 2 Participants |
| Sex: Female, Male Male | 1 Participants | 0 Participants | 1 Participants |
Adverse events
| Event type | EG000 affected / at risk | EG001 affected / at risk |
|---|---|---|
| deaths Total, all-cause mortality | 0 / 2 | 0 / 1 |
| other Total, other adverse events | 0 / 2 | 0 / 1 |
| serious Total, serious adverse events | 0 / 2 | 0 / 1 |
Outcome results
Primary
Change in Carbohydrate Malabsorption
Degree of carbohydrate malabsorption will be assessed by decrease in number of stools per day.
Time frame: baseline, 9 weeks
Population: 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.
Primary
Change in Carbohydrate Malabsorption as Measured by Enteral Nutrition Tolerance
Carbohydrate malabsorption will be measured by ability to advance enteral nutrition in ml/day
Time frame: baseline, 9 weeks
Population: 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.
Primary
Change in Carbohydrate Malabsorption as Measured by Growth Velocity
Carbohydrate malabsorption will be measured by increase in growth velocity in kg/week
Time frame: baseline, 9 weeks
Population: 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.
Primary
Change in Carbohydrate Malabsorption as Measured by Patient Symptom Survey
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.
Time frame: baseline, 9 weeks
Population: 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.
Secondary
Change in Digestion
Change in digestion will be measured by change in abdominal distension/girth measured in cm
Time frame: baseline, 9 weeks
Population: 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.
Secondary
Change in Digestion as Measured by Amount of Emesis
Change in digestion will be assessed by number of emesis per day
Time frame: baseline, 9 weeks
Population: 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.
Secondary
Change in Digestion as Measured by Stool Consistency
Change in digestion will be assessed as a change in stool consistency from liquid (7) to solid(1) using Bristol stool chart
Time frame: baseline, 9 weeks
Population: 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.