Functional Sucrase Deficiency in Short Bowel Syndrome Patients With Intestinal Failure

Short Gut Syndrome Clinical Trial

Official title:

Functional Sucrase Deficiency in Short Bowel Syndrome Patients With Intestinal Failure

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT04604275
• Other study ID #: 20191253
• Status: Recruiting
• Phase: Phase 2
• Start date: February 2, 2022
• Est. completion date: January 1, 2025

Study information

• Verified date: October 2023
• Source: University of Miami
• Contact: Amanda Fifi, MD
• Phone: 3052433166
• Email: afifi[@]med.miami.edu
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 10
• Est. completion date: January 1, 2025
• Est. primary completion date: September 1, 2024
• Accepts healthy volunteers: No
• Gender: All
• Age group: N/A and older

Eligibility

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

Related Conditions & MeSH terms

• Intestinal Failure
• Short Bowel Syndrome
• Short Gut Syndrome
• Syndrome

Intervention

Drug:
• 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.

Other:
• 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.

Locations

Country	Name	City	State
United States	Jackson Memorial Hospital	Miami	Florida
United States	University of Miami	Miami	Florida

Sponsors (2)

Lead Sponsor	Collaborator
University of Miami	QOL Medical, LLC

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Change in Carbohydrate Malabsorption	Degree of carbohydrate malabsorption will be assessed by decrease in number of stools per day.	baseline, up to 9 weeks
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.	baseline, up to 9 weeks
Primary	Change in Carbohydrate Malabsorption as measured by growth velocity	Carbohydrate malabsorption will be measured by increase in growth velocity in kg/week	baseline, up to 9 weeks
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	baseline, up to 9 weeks
Secondary	Change in digestion	Change in digestion will be measured by change in abdominal distension/girth measured in cm	baseline, up to 9 weeks
Secondary	Change in digestion as measured by amount of emesis	Change in digestion will be assessed by number of emesis per day	baseline, up to 9 weeks
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	baseline, up to 9 weeks