Clinical trial

Functional Sucrase Deficiency in Short Bowel Syndrome Patients With Intestinal Failure

Name

20191253

Description

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.

Trial arms

Trial start

2022-02-02

Estimated PCD

2024-09-01

Trial end

2025-01-01

Status

Recruiting

Phase

Early phase I

Treatment

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.

Arms:

Placebo followed by sucrase intervention, Sucrase intervention followed by placebo

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.

Arms:

Placebo followed by sucrase intervention, Sucrase intervention followed by placebo

Size

Primary endpoint

Change in Carbohydrate Malabsorption

baseline, up to 9 weeks

Change in Carbohydrate Malabsorption as measured by patient symptom survey

baseline, up to 9 weeks

Change in Carbohydrate Malabsorption as measured by growth velocity

baseline, up to 9 weeks

Change in Carbohydrate Malabsorption as measured by enteral nutrition tolerance

baseline, up to 9 weeks

Eligibility criteria

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

Protocol

{'studyType': 'INTERVENTIONAL', 'phases': ['PHASE2'], 'designInfo': {'allocation': 'RANDOMIZED', 'interventionModel': 'CROSSOVER', 'primaryPurpose': 'TREATMENT', 'maskingInfo': {'masking': 'QUADRUPLE', 'whoMasked': ['PARTICIPANT', 'CARE_PROVIDER', 'INVESTIGATOR', 'OUTCOMES_ASSESSOR']}}, 'enrollmentInfo': {'count': 10, 'type': 'ESTIMATED'}}

Updated at

2023-11-01

1 organization

1 product

1 indication

Organization

University of Miami

Product

Sucrase

Indication

Short Bowel Syndrome