Functional Sucrase Deficiency in Short Bowel Syndrome Patients With Intestinal Failure

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ClinicalTrials.gov Identifier: NCT04604275

Recruitment Status : Recruiting

First Posted : October 27, 2020

Last Update Posted : February 7, 2022

See Contacts and Locations

Sponsor:

Collaborator:

QOL Medical, LLC

Information provided by (Responsible Party):

Amanda Fifi, University of Miami

Study Description

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.

Condition or disease	Intervention/treatment	Phase
Short Gut Syndrome	Drug: Sucrase Other: Placebo	Phase 2

Study Design

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Study Type :	Interventional (Clinical Trial)
Estimated Enrollment :	10 participants
Allocation:	Randomized
Intervention Model:	Crossover Assignment
Masking:	Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose:	Treatment
Official Title:	Functional Sucrase Deficiency in Short Bowel Syndrome Patients With Intestinal Failure
Actual Study Start Date :	February 2, 2022
Estimated Primary Completion Date :	November 1, 2022
Estimated Study Completion Date :	January 1, 2023

Resource links provided by the National Library of Medicine

Genetic and Rare Diseases Information Center resources: Short Bowel Syndrome

U.S. FDA Resources

Arms and Interventions

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.

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

Other: Placebo

Outcome Measures

Primary Outcome Measures :

Change in Carbohydrate Malabsorption [ Time Frame: baseline, up to 9 weeks ]
Degree of carbohydrate malabsorption will be assessed by decrease in number of stools per day.
Change in Carbohydrate Malabsorption as measured by patient symptom survey [ Time Frame: baseline, up to 9 weeks ]
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.
Change in Carbohydrate Malabsorption as measured by growth velocity [ Time Frame: baseline, up to 9 weeks ]
Carbohydrate malabsorption will be measured by increase in growth velocity in kg/week
Change in Carbohydrate Malabsorption as measured by enteral nutrition tolerance [ Time Frame: baseline, up to 9 weeks ]
Carbohydrate malabsorption will be measured by ability to advance enteral nutrition in ml/day

Secondary Outcome Measures :

Change in digestion [ Time Frame: baseline, up to 9 weeks ]
Change in digestion will be measured by change in abdominal distension/girth measured in cm
Change in digestion as measured by amount of emesis [ Time Frame: baseline, up to 9 weeks ]
Change in digestion will be assessed by number of emesis per day
Change in digestion as measured by stool consistency [ Time Frame: baseline, up to 9 weeks ]
Change in digestion will be assessed as a change in stool consistency from liquid (7) to solid(1) using Bristol stool chart

Eligibility Criteria

Information from the National Library of Medicine

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Ages Eligible for Study:	Child, Adult, Older Adult
Sexes Eligible for Study:	All
Accepts Healthy Volunteers:	No

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

Contacts and Locations

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04604275

Contacts

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Contact: Amanda Fifi, MD	3052433166	afifi@med.miami.edu
Contact: Cara Axelrod, RD	3052433166	cxa630@med.miami.edu

Locations

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

Sponsors and Collaborators

University of Miami

QOL Medical, LLC

Investigators

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Principal Investigator:	Amanda Fifi, MD	University of Miami

More Information

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Responsible Party:	Amanda Fifi, Assistant Professor of Pediatrics, University of Miami
ClinicalTrials.gov Identifier:	NCT04604275
Other Study ID Numbers:	20191253
First Posted:	October 27, 2020 Key Record Dates
Last Update Posted:	February 7, 2022
Last Verified:	February 2022
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD:	No

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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

Keywords provided by Amanda Fifi, University of Miami:


Intestinal failure

Additional relevant MeSH terms:

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Short Bowel Syndrome Syndrome Disease Pathologic Processes Malabsorption Syndromes	Intestinal Diseases Gastrointestinal Diseases Digestive System Diseases Postoperative Complications

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