Short Bowel Syndrome
Conditions
Keywords
pancreatic enzymes
Brief summary
Patients with short bowel syndrome have a high mortality rate that is mainly attributed to complications from central lines and long-term intravenous (IV) nutrition. There are few medical therapies to date that improve gut absorption in patients with short bowel syndrome. The primary objective of this study is to evaluate if absorption from the GI tract improves in subjects with short bowel syndrome following therapy with pancreatic enzymes.
Detailed description
This is an interventional study of subjects with short bowel syndrome to determine if enteral absorption improves following therapy with pancreatic enzymes. The study will assess enteral absorption and nutritional status at baseline through a series of stool tests, blood tests, and anthropometric measurements. Following approximately ten days of therapy with pancreatic enzymes, the study will reassess enteral absorption.
Interventions
All subjects will take pancreatic enzymes at a dose appropriate for their weight. Pancreatic enzymes are used to help digest fat and other nutrients.
Sponsors
AbbVie
Children's Hospital of Philadelphia
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
4 Years to 65 Years
Healthy volunteers
No
Inclusion criteria
* history of a small bowel resection with subsequent dependence on parenteral nutrition for at least three months * age 4 years to 65 years * usual state of health for the past two weeks with no medication changes * able to participate in a study for about four weeks with four study visits * able to take pancreatic enzyme medication orally
Exclusion criteria
* significant disease other than short bowel syndrome affecting the gastrointestinal tract that impacts absorption or digestions * motility disorder * medications that directly alter fat absorption * cholestatic liver disease defined as a serum conjugated bilirubin greater than 1.0 mg/dL, chronic renal failure, gout, or hyperuricemia * history of a pork allergy * women who are pregnant or lactating * history of fibrosing colonopathy Those subjects who are eligible for the malabsorption blood test (MBT) test will be excluded if they have a history of a soy or safflower oil allergy.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Change in Coefficient of Fat Absorption | Up to 10 days | Coefficient of fat absorption (CFA) measures the amount of fat excreted in the stool compared to how much fat was consumed over the course of 72 hours. This is a measure of fat absorption. CFA was measured at baseline off of pancreatic enzymes and then again while on ten days of pancreatic enzyme supplementation. The change between CFA values at each timepoint was the primary outcome. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Change in the Coefficient of Nitrogen Absorption | Up to 10 days | Coefficient of nitrogen absorption (CNA) measures the amount of nitrogen excreted in the stool compared to how much nitrogen was consumed in a 72 hour period. This is a measure of protein absorption. CNA was measured at baseline off of pancreatic enzymes and then again while on ten days of pancreatic enzyme supplementation. The change between CNA values at each timepoint was a study outcome. |
Other
| Measure | Time frame | Description |
|---|---|---|
| Change in Malabsorption Blood Test | Up to 10 days | This test will only be performed on subjects 18 years or older. This an isotope test. Subjects consume a high fat shake with two labeled fats. They have blood tests measured at baseline and then every 1 hour for 8 hours to check for serum levels of labeled fats. This helps determine how well the labeled fats are being absorbed by the intestine. |
Countries
United States
Participant flow
Participants by arm
| Arm | Count |
|---|---|
| Pancreatic Enzymes All subjects in this study will have exposure to therapy with pancreatic enzymes for a period of about ten days.
Pancreatic Enzyme: All subjects will take pancreatic enzymes at a dose appropriate for their weight. Pancreatic enzymes are used to help digest fat and other nutrients. | 15 |
| Total | 15 |
Withdrawals & dropouts
| Period | Reason | FG000 |
|---|---|---|
| Overall Study | Withdrawal by Subject | 1 |
Baseline characteristics
| Characteristic | Pancreatic Enzymes |
|---|---|
| Age, Categorical <=18 years | 8 Participants |
| Age, Categorical >=65 years | 2 Participants |
| Age, Categorical Between 18 and 65 years | 5 Participants |
| Race (NIH/OMB) American Indian or Alaska Native | 0 Participants |
| Race (NIH/OMB) Asian | 0 Participants |
| Race (NIH/OMB) Black or African American | 3 Participants |
| Race (NIH/OMB) More than one race | 0 Participants |
| Race (NIH/OMB) Native Hawaiian or Other Pacific Islander | 0 Participants |
| Race (NIH/OMB) Unknown or Not Reported | 2 Participants |
| Race (NIH/OMB) White | 10 Participants |
| Sex: Female, Male Female | 8 Participants |
| Sex: Female, Male Male | 7 Participants |
Adverse events
| Event type | EG000 affected / at risk |
|---|---|
| deaths Total, all-cause mortality | 0 / 15 |
| other Total, other adverse events | 8 / 15 |
| serious Total, serious adverse events | 0 / 15 |
Outcome results
Primary
Change in Coefficient of Fat Absorption
Coefficient of fat absorption (CFA) measures the amount of fat excreted in the stool compared to how much fat was consumed over the course of 72 hours. This is a measure of fat absorption. CFA was measured at baseline off of pancreatic enzymes and then again while on ten days of pancreatic enzyme supplementation. The change between CFA values at each timepoint was the primary outcome.
Time frame: Up to 10 days
Population: 3 subjects were not included in the analysis. Two of the subjects did not accurately complete dietary records in order to assess fat intake. One subject submitted such a low stool volume that it was assessed as unlikely to represent a full and accurate stool collection.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Pancreatic Enzymes | Change in Coefficient of Fat Absorption | 2.3 percentage of fat absorbed | Standard Deviation 13.6 |
Secondary
Change in the Coefficient of Nitrogen Absorption
Coefficient of nitrogen absorption (CNA) measures the amount of nitrogen excreted in the stool compared to how much nitrogen was consumed in a 72 hour period. This is a measure of protein absorption. CNA was measured at baseline off of pancreatic enzymes and then again while on ten days of pancreatic enzyme supplementation. The change between CNA values at each timepoint was a study outcome.
Time frame: Up to 10 days
Population: 3 subjects were not included in the final analysis. Two subjects did not accurately complete a three day dietary record in order to assess nitrogen intake. 1 subject submitted such a low volume of stool that it was assessed as an inaccurate and incomplete stool collection.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Pancreatic Enzymes | Change in the Coefficient of Nitrogen Absorption | 6.9 percentage of nitrogen absorbed | Standard Deviation 15.9 |
Other Pre-specified
Change in Malabsorption Blood Test
This test will only be performed on subjects 18 years or older. This an isotope test. Subjects consume a high fat shake with two labeled fats. They have blood tests measured at baseline and then every 1 hour for 8 hours to check for serum levels of labeled fats. This helps determine how well the labeled fats are being absorbed by the intestine.
Time frame: Up to 10 days