Functional Sucrase Deficiency in Short Bowel Syndrome Patients With Intestinal Failure

NCT04604275 | Terminated Phase 2 Results FDA Drug

• 1 other identifier: 20191253
• Study Type: interventional
• Target: 3
• Locations: 1 country
• Sites: 2

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.

Conditions

Short Gut Syndrome

Keywords

Intestinal failure

Outcome Measures

Primary Outcomes (4)

• Change in Carbohydrate Malabsorption

  Degree of carbohydrate malabsorption will be assessed by decrease in number of stools per day.

  baseline, 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, 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, 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, 9 weeks

Secondary Outcomes (3)

• Change in Digestion

  baseline, 9 weeks

• Change in Digestion as Measured by Amount of Emesis

  baseline, 9 weeks

• Change in Digestion as Measured by Stool Consistency

  baseline, 9 weeks

Study Arms (2)

Sucrase intervention followed by placebo

EXPERIMENTAL

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; Other: Placebo

Placebo followed by sucrase intervention

EXPERIMENTAL

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; Other: Placebo

Interventions

Sucrase DRUG

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 followed by sucrase intervention; Sucrase intervention followed by placebo

Placebo OTHER

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; Sucrase intervention followed by placebo

Eligibility Criteria

• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• 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)

You may not qualify if:

• 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

Sponsors & Collaborators

• University of Miami: lead
• QOL Medical, LLC: collaborator

Study Sites (2)

Jackson Memorial Hospital

Miami, Florida, 33136, United States

Location

University of Miami

Miami, Florida, 33136, United States

Location

MeSH Terms

Conditions

Short Bowel Syndrome; Intestinal Failure

Interventions

Sucrase

Condition Hierarchy (Ancestors)

Malabsorption Syndromes; Intestinal Diseases; Gastrointestinal Diseases; Digestive System Diseases; Postoperative Complications; Pathologic Processes; Pathological Conditions, Signs and Symptoms

Intervention Hierarchy (Ancestors)

Disaccharidases; Glycoside Hydrolases; Hydrolases; Enzymes; Enzymes and Coenzymes

Limitations and Caveats

The study was terminated due to low accrual. The target population consisted of individuals with a rare condition that is not commonly found in the general population, which significantly limited recruitment feasibility. Despite outreach efforts and engagement with specialized clinical sites, the rarity of the condition and strict eligibility criteria resulted in insufficient enrollment.

Results Point of Contact

• Title: Amanda Fifi
• Organization: University of Miami

amandacfifi@gmail.com

305-243-3166

Study Officials

• Amanda Fifi, MD

  University of Miami

  PRINCIPAL INVESTIGATOR

Publication Agreements

• PI is Sponsor Employee: Yes

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: CROSSOVER
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: October 22, 2020
• First Posted: October 27, 2020
• Study Start: January 31, 2022
• Primary Completion: September 1, 2024
• Study Completion: September 1, 2024
• Last Updated: October 1, 2025
• Results First Posted: October 1, 2025

Record last verified: 2025-09

Data Sharing

• IPD Sharing: Will not share

Locations

US (2)