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Inability to recruit further patients for study
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| Name | Class |
|---|---|
| QOL Medical, LLC | INDUSTRY |
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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.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| 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. |
|
| 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. |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| 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. |
| Measure | Description | Time Frame |
|---|---|---|
| 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 |
| Measure | Description | Time Frame |
|---|---|---|
| Change in Digestion | Change in digestion will be measured by change in abdominal distension/girth measured in cm | baseline, 9 weeks |
| Change in Digestion as Measured by Amount of Emesis | Change in digestion will be assessed by number of emesis per day |
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Inclusion Criteria:
Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| Amanda Fifi, MD | University of Miami | Principal Investigator |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Jackson Memorial Hospital | Miami | Florida | 33136 | United States | ||
| University of Miami |
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| ID | Title | Description |
|---|---|---|
| FG000 | 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. 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. 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. |
| FG001 | 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. 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. 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. |
| Title | Milestones | Reasons Not Completed | ||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Intervention 1 |
| |||||||||||||
| Washout-1 Week |
| |||||||||||||
| Intervention 2 |
|
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| ID | Title | Description |
|---|---|---|
| BG000 | 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. 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. 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. |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes |
|---|---|---|---|---|---|---|---|---|---|
| Age, Categorical | Count of Participants |
| Type | Title | Description | Population Description | Reporting Status | Anticipated Posting Date | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Time Frame | Units Analyzed | Denominator Units Selected | Arm/Group Information | Denominators | Classes | Analyses | |||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Primary | Change in Carbohydrate Malabsorption | Degree of carbohydrate malabsorption will be assessed by decrease in number of stools per day. | Due to operational challenges across multiple study sites, the required data for this outcome measure could not be collected or retained. The study involved several coordinators working across dispersed locations, which introduced variability in data handling and documentation practices. Although standardized procedures were in place, inconsistencies emerged in how forms were distributed, tracked, and returned. | Posted | baseline, 9 weeks |
|
9 weeks
Adverse Events are being reported per arm. There are only 2 arms for this study.
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| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | 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. 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. 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. |
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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.
| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Amanda Fifi | University of Miami | 305-243-3166 | amandacfifi@gmail.com |
| Type | Includes Protocol | Includes SAP | Includes ICF | Document Label | Document Date | Document Uploaded Date | Document File Name |
|---|---|---|---|---|---|---|---|
| Prot | Yes | No | No | Study Protocol | Aug 27, 2020 | Jun 12, 2025 | Prot_000.pdf |
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| ID | Term |
|---|---|
| D012778 | Short Bowel Syndrome |
| D000090124 | Intestinal Failure |
| ID | Term |
|---|---|
| D008286 | Malabsorption Syndromes |
| D007410 | Intestinal Diseases |
| D005767 | Gastrointestinal Diseases |
| D004066 | Digestive System Diseases |
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| ID | Term |
|---|---|
| D013393 | Sucrase |
| ID | Term |
|---|---|
| D004186 | Disaccharidases |
| D006026 | Glycoside Hydrolases |
| D006867 | Hydrolases |
| D004798 | Enzymes |
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|
| 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. |
|
| baseline, 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, 9 weeks |
| Miami |
| Florida |
| 33136 |
| United States |
| NOT COMPLETED |
|
|
| NOT COMPLETED |
|
| BG001 | 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. 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. 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. |
| BG002 | Total | Total of all reporting groups |
| Participants |
|
| Sex: Female, Male | Count of Participants | Participants |
|
| Ethnicity (NIH/OMB) | Count of Participants | Participants |
|
| Race (NIH/OMB) | Count of Participants | Participants |
|
| OG001 | 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. 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. 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 | 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. | Due to operational challenges across multiple study sites, the required data for this outcome measure could not be collected or retained. The study involved several coordinators working across dispersed locations, which introduced variability in data handling and documentation practices. Although standardized procedures were in place, inconsistencies emerged in how forms were distributed, tracked, and returned. | Posted | baseline, 9 weeks |
|
|
| Primary | Change in Carbohydrate Malabsorption as Measured by Growth Velocity | Carbohydrate malabsorption will be measured by increase in growth velocity in kg/week | Due to operational challenges across multiple study sites, the required data for this outcome measure could not be collected or retained. The study involved several coordinators working across dispersed locations, which introduced variability in data handling and documentation practices. Although standardized procedures were in place, inconsistencies emerged in how forms were distributed, tracked, and returned. | Posted | baseline, 9 weeks |
|
|
| Primary | Change in Carbohydrate Malabsorption as Measured by Enteral Nutrition Tolerance | Carbohydrate malabsorption will be measured by ability to advance enteral nutrition in ml/day | Due to operational challenges across multiple study sites, the required data for this outcome measure could not be collected or retained. The study involved several coordinators working across dispersed locations, which introduced variability in data handling and documentation practices. Although standardized procedures were in place, inconsistencies emerged in how forms were distributed, tracked, and returned. | Posted | baseline, 9 weeks |
|
|
| Secondary | Change in Digestion | Change in digestion will be measured by change in abdominal distension/girth measured in cm | Due to operational challenges across multiple study sites, the required data for this outcome measure could not be collected or retained. The study involved several coordinators working across dispersed locations, which introduced variability in data handling and documentation practices. Although standardized procedures were in place, inconsistencies emerged in how forms were distributed, tracked, and returned. | Posted | baseline, 9 weeks |
|
|
| Secondary | Change in Digestion as Measured by Amount of Emesis | Change in digestion will be assessed by number of emesis per day | Due to operational challenges across multiple study sites, the required data for this outcome measure could not be collected or retained. The study involved several coordinators working across dispersed locations, which introduced variability in data handling and documentation practices. Although standardized procedures were in place, inconsistencies emerged in how forms were distributed, tracked, and returned. | Posted | baseline, 9 weeks |
|
|
| Secondary | 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 | Due to operational challenges across multiple study sites, the required data for this outcome measure could not be collected or retained. The study involved several coordinators working across dispersed locations, which introduced variability in data handling and documentation practices. Although standardized procedures were in place, inconsistencies emerged in how forms were distributed, tracked, and returned. | Posted | baseline, 9 weeks |
|
|
| 0 |
| 2 |
| 0 |
| 2 |
| 0 |
| 2 |
| EG001 | 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. 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. 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. | 0 | 1 | 0 | 1 | 0 | 1 |
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| D011183 | Postoperative Complications |
| D010335 | Pathologic Processes |
| D013568 | Pathological Conditions, Signs and Symptoms |
| D045762 |
| Enzymes and Coenzymes |