Functional Sucrase Deficiency in Short Bowel Syndrome Patients With Intestinal Failure

Phase 2

Terminated

• Conditions: Short Gut Syndrome
• Interventions: Drug: Sucrase; Other: Placebo

• Registration Number: NCT04604275
• Lead Sponsor: University of Miami

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.

Detailed Description

Not available

Study Timeline

• Study First Submitted: 2020-10-22
• Study First Submitted QC: 2020-10-22
• Study First Posted: 2020-10-27
• Study Start: 2022-02-02
• Status Verified: 2024-09
• Primary Completion: 2024-09-01
• Study Completion: 2024-09-01
• Last Update Submitted: 2024-10-16
• Last Update Posted: 2024-10-18

Recruitment & Eligibility

• Status: TERMINATED
• Sex: All
• Target Recruitment: 10

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

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: CROSSOVER

Arm && Interventions

Group	Intervention	Description
Sucrase intervention followed by placebo	Sucrase	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 intervention followed by placebo	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.
Placebo followed by sucrase intervention	Sucrase	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.
Placebo followed by sucrase intervention	Placebo	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.

Primary Outcome Measures

Name	Time	Method
Change in Carbohydrate Malabsorption as measured by enteral nutrition tolerance	baseline, up to 9 weeks	Carbohydrate malabsorption will be measured by ability to advance enteral nutrition in ml/day
Change in Carbohydrate Malabsorption as measured by growth velocity	baseline, up to 9 weeks	Carbohydrate malabsorption will be measured by increase in growth velocity in kg/week
Change in Carbohydrate Malabsorption as measured by patient symptom survey	baseline, up to 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	baseline, up to 9 weeks	Degree of carbohydrate malabsorption will be assessed by decrease in number of stools per day.

Secondary Outcome Measures

Name	Time	Method
Change in digestion as measured by amount of emesis	baseline, up to 9 weeks	Change in digestion will be assessed by number of emesis per day
Change in digestion as measured by stool consistency	baseline, up to 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, up to 9 weeks	Change in digestion will be measured by change in abdominal distension/girth measured in cm

Trial Locations

Locations (2)

Jackson Memorial Hospital; 🇺🇸 Miami, Florida, United States

University of Miami; 🇺🇸 Miami, Florida, United States