Functional Sucrase Deficiency in Short Bowel Syndrome Patients With Intestinal Failure

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.

Study Overview

• Status: Recruiting
• Conditions: Short Gut Syndrome
• Intervention / Treatment: Drug: Sucrase; Other: Placebo

• Study Type: Interventional
• Enrollment (Estimated): 10
• Phase: Phase 2

Contacts and Locations

• Study Contact: Name: Amanda Fifi, MD; Phone Number: 3052433166; Email: afifi@med.miami.edu
• Study Contact Backup: Name: Cara Axelrod, RD; Phone Number: 3052433166; Email: cxa630@med.miami.edu

Study Locations

• United States
  • Florida
    • Miami, Florida, United States, 33136
      • Recruiting
      • Jackson Memorial Hospital
      • Contact: Amanda Fifi, MD; Phone Number: 305-243-3166; Email: afifi@med.miami.edu
      • Contact: Cara Axelrod, MD; Phone Number: 3052433166; Email: cxa630@med.miami.edu
      • Principal Investigator: AMANDA FIFI, MD
    • Miami, Florida, United States, 33136
      • Not yet recruiting
      • University of Miami
      • Contact: Amanda Fifi, MD; Phone Number: 305-243-3166; Email: afifi@med.miami.edu
      • Principal Investigator: AMANDA FIFI, MD
      • Contact: Cara Axelrod, RD; Phone Number: FIFI 3052433166; Email: amandacfifi@gmail.com

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Child; Adult; Older Adult
• Accepts Healthy Volunteers: No

Description

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 Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Randomized
• Interventional Model: Crossover Assignment
• Masking: Quadruple

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: 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.	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.
Experimental: 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.	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.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Change in Carbohydrate Malabsorption	Degree of carbohydrate malabsorption will be assessed by decrease in number of stools per day.	baseline, up to 9 weeks
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
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
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 Outcome Measures

Outcome Measure	Measure Description	Time Frame
Change in digestion	Change in digestion will be measured by change in abdominal distension/girth measured in cm	baseline, up to 9 weeks
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
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

Collaborators and Investigators

• Sponsor: University of Miami
• Collaborators: QOL Medical, LLC
• Investigators: Principal Investigator: Amanda Fifi, MD, University of Miami

Study record dates

Study Major Dates

• Study Start (Actual): February 2, 2022
• Primary Completion (Estimated): September 1, 2024
• Study Completion (Estimated): January 1, 2025

Study Registration Dates

• First Submitted: October 22, 2020
• First Submitted That Met QC Criteria: October 22, 2020
• First Posted (Actual): October 27, 2020

Study Record Updates

• Last Update Posted (Actual): November 1, 2023
• Last Update Submitted That Met QC Criteria: October 31, 2023
• Last Verified: October 1, 2023

More Information

Terms related to this study

• Keywords: Intestinal failure
• Additional Relevant MeSH Terms: Digestive System Diseases; Pathologic Processes; Postoperative Complications; Disease; Gastrointestinal Diseases; Intestinal Diseases; Malabsorption Syndromes; Syndrome; Short Bowel Syndrome; Intestinal Failure
• Other Study ID Numbers: 20191253

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No
• product manufactured in and exported from the U.S.: No