Functional Sucrase Deficiency in Short Bowel Syndrome Patients With Intestinal Failure

Study Overview
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Brief Summary
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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.

Official Title
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Functional Sucrase Deficiency in Short Bowel Syndrome Patients With Intestinal Failure

Conditions
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Short Gut Syndrome

Intervention / Treatment
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* Drug: Sucrase
* Other: Placebo


Participation Criteria
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Eligibility Criteria
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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
Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Allocation: Randomized
Intervention Model: Crossover Assignment
Masking: Quadruple


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: Sucrase intervention followed by placebo<br>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<br>* 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.<br>Other: Placebo<br>* 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.<br>|
| Experimental: Placebo followed by sucrase intervention<br>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<br>* 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.<br>Other: Placebo<br>* 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.<br>|

What is the study measuring?
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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 | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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Intestinal failure