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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
Sponsor:
Collaborator:
QOL Medical, LLC
Information provided by (Responsible Party):
Amanda Fifi, University of Miami

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

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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


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.




Primary Outcome Measures :
  1. 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.

  2. 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.


  3. 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

  4. 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 :
  1. 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

  2. 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

  3. 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



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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

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
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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