Trial Outcomes & Findings for Use of Pancreatic Enzymes in Short Bowel Syndrome (NCT NCT03097029)

NCT ID: NCT03097029

Last Updated: 2020-03-20

Results Overview

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.

• Recruitment status: COMPLETED
• Study phase: PHASE2
• Target enrollment: 16 participants
• Primary outcome timeframe: Up to 10 days
• Results posted on: 2020-03-20

Participant Flow

Participant milestones

Measure	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.
Overall Study STARTED	15
Overall Study COMPLETED	14
Overall Study NOT COMPLETED	1

Reasons for withdrawal

Measure	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.
Overall Study Withdrawal by Subject	1

Baseline Characteristics

Use of Pancreatic Enzymes in Short Bowel Syndrome

Baseline characteristics by cohort

Measure	Pancreatic Enzymes n=15 Participants 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.
Age, Categorical <=18 years	8 Participants n=5 Participants
Age, Categorical Between 18 and 65 years	5 Participants n=5 Participants
Age, Categorical >=65 years	2 Participants n=5 Participants
Sex: Female, Male Female	8 Participants n=5 Participants
Sex: Female, Male Male	7 Participants n=5 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=5 Participants
Race (NIH/OMB) Asian	0 Participants n=5 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=5 Participants
Race (NIH/OMB) Black or African American	3 Participants n=5 Participants
Race (NIH/OMB) White	10 Participants n=5 Participants
Race (NIH/OMB) More than one race	0 Participants n=5 Participants
Race (NIH/OMB) Unknown or Not Reported	2 Participants n=5 Participants

PRIMARY outcome

Timeframe: 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.

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.

Outcome measures

Measure	Pancreatic Enzymes n=11 Participants 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.
Change in Coefficient of Fat Absorption	2.3 percentage of fat absorbed Standard Deviation 13.6

SECONDARY outcome

Timeframe: 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.

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.

Outcome measures

Measure	Pancreatic Enzymes n=11 Participants 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.
Change in the Coefficient of Nitrogen Absorption	6.9 percentage of nitrogen absorbed Standard Deviation 15.9

OTHER_PRE_SPECIFIED outcome

Timeframe: 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.

Outcome measures

Outcome data not reported

Adverse Events

Pancreatic Enzymes

Serious events: 0 serious events

Other events: 8 other events

Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Measure	Pancreatic Enzymes n=15 participants at risk 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.
Gastrointestinal disorders Diarrhea	6.7% 1/15 • Adverse event data is collected on a daily basis for four weeks, from the time of enrollment (Visit 0), to the final follow-up call (Visit 3). The final study visit was a follow-up telephone call after the subjects were off of the study intervention, pancreatic enzymes. The purpose of this phone call was to assess for adverse events and adherence to the recommended pancreatic enzyme study dose.
Gastrointestinal disorders Constipation	20.0% 3/15 • Adverse event data is collected on a daily basis for four weeks, from the time of enrollment (Visit 0), to the final follow-up call (Visit 3). The final study visit was a follow-up telephone call after the subjects were off of the study intervention, pancreatic enzymes. The purpose of this phone call was to assess for adverse events and adherence to the recommended pancreatic enzyme study dose.
Gastrointestinal disorders Abdominal Pain	13.3% 2/15 • Adverse event data is collected on a daily basis for four weeks, from the time of enrollment (Visit 0), to the final follow-up call (Visit 3). The final study visit was a follow-up telephone call after the subjects were off of the study intervention, pancreatic enzymes. The purpose of this phone call was to assess for adverse events and adherence to the recommended pancreatic enzyme study dose.
Gastrointestinal disorders Decreased appetite	6.7% 1/15 • Adverse event data is collected on a daily basis for four weeks, from the time of enrollment (Visit 0), to the final follow-up call (Visit 3). The final study visit was a follow-up telephone call after the subjects were off of the study intervention, pancreatic enzymes. The purpose of this phone call was to assess for adverse events and adherence to the recommended pancreatic enzyme study dose.
General disorders Increased flatus	6.7% 1/15 • Adverse event data is collected on a daily basis for four weeks, from the time of enrollment (Visit 0), to the final follow-up call (Visit 3). The final study visit was a follow-up telephone call after the subjects were off of the study intervention, pancreatic enzymes. The purpose of this phone call was to assess for adverse events and adherence to the recommended pancreatic enzyme study dose.
Renal and urinary disorders Urinary frequency	6.7% 1/15 • Adverse event data is collected on a daily basis for four weeks, from the time of enrollment (Visit 0), to the final follow-up call (Visit 3). The final study visit was a follow-up telephone call after the subjects were off of the study intervention, pancreatic enzymes. The purpose of this phone call was to assess for adverse events and adherence to the recommended pancreatic enzyme study dose.
Musculoskeletal and connective tissue disorders Back pain	6.7% 1/15 • Adverse event data is collected on a daily basis for four weeks, from the time of enrollment (Visit 0), to the final follow-up call (Visit 3). The final study visit was a follow-up telephone call after the subjects were off of the study intervention, pancreatic enzymes. The purpose of this phone call was to assess for adverse events and adherence to the recommended pancreatic enzyme study dose.

Additional Information

Dr. Nina Sainath

Children's Hospital of Philadelphia

Phone: 267.426.1736

Email: sainathn@email.chop.edu

Results disclosure agreements

• Principal investigator is a sponsor employee
• Publication restrictions are in place