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Short Bowel Syndrome and Teduglutide Versus Placebo

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ClinicalTrials.gov Identifier: NCT02099084
Recruitment Status : Completed
First Posted : March 28, 2014
Results First Posted : February 22, 2016
Last Update Posted : February 22, 2016
Sponsor:
Information provided by (Responsible Party):
Michael Camilleri, Mayo Clinic

Study Type Interventional
Study Design Allocation: Randomized;   Intervention Model: Crossover Assignment;   Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor);   Primary Purpose: Treatment
Condition Short Bowel Syndrome
Interventions Drug: Teduglutide
Drug: Placebo
Enrollment 8
Recruitment Details Subjects were recruited from the Mayo Clinic Home Parenteral Nutrition program in Rochester, Minnesota.
Pre-assignment Details  
Arm/Group Title Teduglutide First, Then Placebo Placebo First, Then Teduglutide
Hide Arm/Group Description Teduglutide 0.05 mg/kg/d administered subcutaneously for 7 days, followed by a 14-day washout period, and placebo administered subcutaneously for 7 days. Placebo administered subcutaneously for 7 days, followed by a 14-day washout period, and Teduglutide 0.05 mg/kg/d administered subcutaneously for 7 days.
Period Title: First Intervention
Started 4 4
Completed 4 4
Not Completed 0 0
Period Title: Washout Period of 14 Days
Started 4 4
Completed 4 4
Not Completed 0 0
Period Title: Second Intervention
Started 4 4
Completed 4 4
Not Completed 0 0
Arm/Group Title Entire Study Population
Hide Arm/Group Description Includes groups randomized to receive placebo first and Teduglutide first.
Overall Number of Baseline Participants 8
Hide Baseline Analysis Population Description
[Not Specified]
Age, Continuous  
Mean (Standard Deviation)
Unit of measure:  Years
Number Analyzed 8 participants
54  (13)
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 8 participants
Female
4
  50.0%
Male
4
  50.0%
Region of Enrollment  
Measure Type: Number
Unit of measure:  Participants
United States Number Analyzed 8 participants
8
1.Primary Outcome
Title Gastric Emptying Half-Time (T1/2)
Hide Description The time for half of the ingested solids or liquids to leave the stomach.
Time Frame approximately 2 hours after radiolabeled meal is ingested
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Teduglutide Placebo
Hide Arm/Group Description:
Teduglutide 0.05 mg/kg/d administered subcutaneously for 7 days in either first intervention period or second intervention period
Placebo administered subcutaneously daily for 7 days in either first intervention period or second intervention period
Overall Number of Participants Analyzed 8 8
Mean (Standard Deviation)
Unit of Measure: minutes
113  (16) 106  (20)
Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Teduglutide, Placebo
Comments Level of significance = .05
Type of Statistical Test Superiority or Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.74
Comments [Not Specified]
Method t-test, 2 sided
Comments [Not Specified]
2.Primary Outcome
Title Overall Gut Transit
Hide Description Given the variable extent of the residual length of the small intestine and colon, the proportion emptied from the body at 6 hours was assessed as an overall estimate of the whole gut transit. The 6-hour values for intra-abdominal counts were then compared with the 100% reference values of counts (at time zero, which is immediately after ingestion of the radiolabeled meal) to determine the percentage of isotope retained in the abdomen. 100% minus the percentage of retained isotope reflected the amount emptied from the GI tract.
Time Frame baseline, approximately 6 hours after ingestion of radiolabeled meal
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Teduglutide Placebo
Hide Arm/Group Description:
Teduglutide 0.05 mg/kg/d administered subcutaneously for 7 days in either first intervention period or second intervention period
Placebo administered subcutaneously daily for 7 days in either first intervention period or second intervention period
Overall Number of Participants Analyzed 8 8
Mean (Standard Deviation)
Unit of Measure: Percentage of isotope emptied
53.4  (15) 62.4  (15.2)
Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Teduglutide, Placebo
Comments Level of significance = .05
Type of Statistical Test Superiority or Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.075
Comments [Not Specified]
Method t-test, 2 sided
Comments [Not Specified]
3.Secondary Outcome
Title Change in Small Intestinal and Colonic Permeability as Measured by Urinary Excretion of Mannitol
Hide Description Permeability is measured through differential excretion of urine saccharides. A sugar solution (200 mg of mannitol and 1 g lactulose in 30 mL of water) was administered with the radiolabeled test meal at visits 1 and 2. Urine was collected during 0-2 and 2-8 hours. A baseline urine sample was also collected prior to ingestion of the sugars. Chemical analysis was preformed with high-speed liquid chromatography tandem mass spectrometry.
Time Frame baseline, approximately 2 hours and 8 hours after ingestion of radiolabeled meal
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Teduglutide Placebo
Hide Arm/Group Description:
Teduglutide 0.05 mg/kg/d administered subcutaneously for 7 days in either first intervention period or second intervention period
Placebo administered subcutaneously daily for 7 days in either first intervention period or second intervention period
Overall Number of Participants Analyzed 8 8
Mean (Standard Error)
Unit of Measure: mg
0 - 2 hours 16.2  (3.6) 11.3  (2.2)
0 - 8 hours 48.8  (8.9) 32.7  (5.9)
Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Teduglutide, Placebo
Comments Comparison between the groups for change between 0- and 2-hours. Level of significance = .05
Type of Statistical Test Superiority or Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.20
Comments [Not Specified]
Method t-test, 2 sided
Comments [Not Specified]
Hide Statistical Analysis 2
Statistical Analysis Overview Comparison Group Selection Teduglutide, Placebo
Comments Comparison between the groups for change between 0- and 8-hours. Level of significance = .05
Type of Statistical Test Superiority or Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.17
Comments [Not Specified]
Method t-test, 2 sided
Comments [Not Specified]
4.Secondary Outcome
Title Change in Small Intestinal and Colonic Permeability as Measured by Urinary Excretion of Lactulose at 2 Hours
Hide Description Permeability is measured through differential excretion of urine saccharides. A sugar solution (200 mg of mannitol and 1 g lactulose in 30 mL of water) was administered with the radiolabeled test meal at visits 1 and 2. Urine was collected during 0-2 and 2-8 hours. A baseline urine sample was also collected prior to ingestion of the sugars. Chemical analysis was preformed with high-speed liquid chromatography tandem mass spectrometry.
Time Frame baseline, approximately 2 hours after ingestion of radiolabeled meal
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Teduglutide Placebo
Hide Arm/Group Description:
Teduglutide 0.05 mg/kg/d administered subcutaneously for 7 days in either first intervention period or second intervention period
Placebo administered subcutaneously daily for 7 days in either first intervention period or second intervention period
Overall Number of Participants Analyzed 8 8
Mean (Standard Deviation)
Unit of Measure: mg
0.42  (0.14) 0.38  (0.14)
5.Secondary Outcome
Title Change in Small Intestinal and Colonic Permeability as Measured by Lactulose/Mannitol Ratio at 2 Hours
Hide Description Permeability is measured through differential excretion of urine saccharides. A sugar solution (200 mg of mannitol and 1 g lactulose in 30 mL of water) was administered with the radiolabeled test meal at visits 1 and 2. Urine was collected during 0-2 and 2-8 hours. A baseline urine sample was also collected prior to ingestion of the sugars. Chemical analysis was preformed with high-speed liquid chromatography tandem mass spectrometry.
Time Frame baseline, approximately 2 hours after ingestion of radiolabeled meal
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Teduglutide Placebo
Hide Arm/Group Description:
Teduglutide 0.05 mg/kg/d administered subcutaneously for 7 days in either first intervention period or second intervention period
Placebo administered subcutaneously daily for 7 days in either first intervention period or second intervention period
Overall Number of Participants Analyzed 8 8
Mean (Standard Deviation)
Unit of Measure: ratio
0.024  (0.005) 0.021  (0.005)
6.Other Pre-specified Outcome
Title Stool Weight at 8 Hours
Hide Description After an overnight fast, subjects received a single dose of placebo or Teduglutide 1 hour before breakfast, then consumed a radiolabeled meal. After 8 hours a stool collection was taken.
Time Frame approximately 8 hours after ingestion of radiolabeled meal
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Teduglutide Placebo
Hide Arm/Group Description:
Teduglutide 0.05 mg/kg/d administered subcutaneously for 7 days in either first intervention period or second intervention period
Placebo administered subcutaneously daily for 7 days in either first intervention period or second intervention period
Overall Number of Participants Analyzed 8 8
Mean (Standard Deviation)
Unit of Measure: g
77  (18) 106  (43)
Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Teduglutide, Placebo
Comments Level of significance = .05
Type of Statistical Test Superiority or Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.42
Comments [Not Specified]
Method t-test, 2 sided
Comments [Not Specified]
7.Other Pre-specified Outcome
Title Urine Volume at 8 Hours
Hide Description After an overnight fast, subjects received a single dose of placebo or Teduglutide 1 hour before breakfast, then consumed a radiolabeled meal. Urine was collected twice: from the start of the ingestion of the meal to 2 hours, and 2-8 hours. The total volume of urine collected was the sum of these two collections.
Time Frame Start of the ingestion of the radiolabeled meal until 8 hours after the meal
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Teduglutide Placebo
Hide Arm/Group Description:
Teduglutide 0.05 mg/kg/d administered subcutaneously for 7 days in either first intervention period or second intervention period
Placebo administered subcutaneously daily for 7 days in either first intervention period or second intervention period
Overall Number of Participants Analyzed 8 8
Mean (Standard Deviation)
Unit of Measure: mL
408.9  (52.2) 365.7  (57.3)
Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Teduglutide, Placebo
Comments Level of significance = .05
Type of Statistical Test Superiority or Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.34
Comments [Not Specified]
Method t-test, 2 sided
Comments [Not Specified]
Time Frame Adverse event data were collected for approximately 21 days.
Adverse Event Reporting Description Adverse events were recorded in the daily stool diary. Participants were contacted on day 2 and day 5 of each phase of the study medications. Participants also met with study staff on visits 1-3.
 
Arm/Group Title Teduglutide Placebo
Hide Arm/Group Description Teduglutide 0.05 mg/kg/d administered subcutaneously for 7 days in either first intervention period or second intervention period Placebo administered subcutaneously daily for 7 days in either first intervention period or second intervention period
All-Cause Mortality
Teduglutide Placebo
Affected / at Risk (%) Affected / at Risk (%)
Total   --/--      --/--    
Hide Serious Adverse Events
Teduglutide Placebo
Affected / at Risk (%) # Events Affected / at Risk (%) # Events
Total   0/8 (0.00%)      0/8 (0.00%)    
Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 0%
Teduglutide Placebo
Affected / at Risk (%) # Events Affected / at Risk (%) # Events
Total   2/8 (25.00%)      1/8 (12.50%)    
Gastrointestinal disorders     
Moderate abdominal discomfort   0/8 (0.00%)  0 1/8 (12.50%)  2
Nausea   1/8 (12.50%)  1 0/8 (0.00%)  0
Increase in size of stoma   2/8 (25.00%)  2 0/8 (0.00%)  0
General disorders     
Headache   1/8 (12.50%)  1 0/8 (0.00%)  0
Indicates events were collected by systematic assessment
Certain Agreements
All Principal Investigators ARE employed by the organization sponsoring the study.
Results Point of Contact
Layout table for Results Point of Contact information
Name/Title: Dr. Michael Camilleri
Organization: Mayo Clinic
Phone: 507-284-6218
EMail: camilleri.michael@mayo.edu
Layout table for additonal information
Responsible Party: Michael Camilleri, Mayo Clinic
ClinicalTrials.gov Identifier: NCT02099084    
Other Study ID Numbers: 13-004866
UL1TR000135 ( U.S. NIH Grant/Contract )
First Submitted: March 25, 2014
First Posted: March 28, 2014
Results First Submitted: January 25, 2016
Results First Posted: February 22, 2016
Last Update Posted: February 22, 2016