Working...
ClinicalTrials.gov
ClinicalTrials.gov Menu

Short Bowel Syndrome Research Study for Children Up To 17 Years of Age on Parenteral Nutrition

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
ClinicalTrials.gov Identifier: NCT02682381
Recruitment Status : Completed
First Posted : February 15, 2016
Results First Posted : October 16, 2018
Last Update Posted : May 16, 2019
Sponsor:
Information provided by (Responsible Party):
Shire

Study Type Interventional
Study Design Allocation: Randomized;   Intervention Model: Single Group Assignment;   Masking: Triple (Participant, Care Provider, Investigator);   Primary Purpose: Treatment
Condition Short Bowel Syndrome
Interventions Drug: Teduglutide 0.05mg/kg
Drug: Teduglutide 0.025 mg/kg
Other: Standard of Care
Enrollment 59
Recruitment Details Study was conducted at 27 study centers in the United States, Belgium, Canada, the United Kingdom, Finland, Germany and Italy between 03 June 2016 (first participant first visit) and 18 August 2017 (last participant last visit).
Pre-assignment Details Overall, 59 participants were enrolled; 50 in the teduglutide treatment arm (24 participants in the 0.025 milligram per kilogram per day (mg/kg/day) dose group and 26 participants in the 0.05 mg/kg/day dose group)and 9 in the standard of care arm.
Arm/Group Title 0.025 mg/kg/Day Teduglutide 0.05 mg/kg/Day Teduglutide Standard of Care
Hide Arm/Group Description Participants received 0.025 milligram per kilogram per day (mg/kg/day) of teduglutide subcutaneously for 24 weeks along with standard medical therapy. Participants received 0.05 mg/kg/day of teduglutide subcutaneously for 24 weeks along with standard medical therapy. Participants received standard medical therapy.
Period Title: Overall Study
Started 24 26 9
Completed 24 26 9
Not Completed 0 0 0
Arm/Group Title 0.025 mg/kg/Day Teduglutide 0.05 mg/kg/Day Teduglutide Standard of Care Total
Hide Arm/Group Description Participants received 0.025 mg/kg/day of teduglutide subcutaneously for 24 weeks along with standard medical therapy. Participants received 0.05 mg/kg/day of teduglutide subcutaneously for 24 weeks along with standard medical therapy. Participants received standard medical therapy. Total of all reporting groups
Overall Number of Baseline Participants 24 26 9 59
Hide Baseline Analysis Population Description
Intention-to-treat (ITT) population included all enrolled participants.
Age, Continuous  
Mean (Standard Deviation)
Unit of measure:  Years
Number Analyzed 24 participants 26 participants 9 participants 59 participants
6.6  (3.61) 6.2  (3.67) 5.7  (4.72) 6.3  (3.76)
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 24 participants 26 participants 9 participants 59 participants
Female
8
  33.3%
7
  26.9%
3
  33.3%
18
  30.5%
Male
16
  66.7%
19
  73.1%
6
  66.7%
41
  69.5%
Ethnicity (NIH/OMB)  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 24 participants 26 participants 9 participants 59 participants
Hispanic or Latino
5
  20.8%
5
  19.2%
4
  44.4%
14
  23.7%
Not Hispanic or Latino
16
  66.7%
20
  76.9%
2
  22.2%
38
  64.4%
Unknown or Not Reported
3
  12.5%
1
   3.8%
3
  33.3%
7
  11.9%
Race/Ethnicity, Customized   [1] 
Measure Type: Count of Participants
Unit of measure:  Participants
Race Customized Number Analyzed 24 participants 26 participants 9 participants 59 participants
White
16
  66.7%
21
  80.8%
2
  22.2%
39
  66.1%
Black or African American
3
  12.5%
3
  11.5%
1
  11.1%
7
  11.9%
Asian
1
   4.2%
1
   3.8%
1
  11.1%
3
   5.1%
American Indian or Alaska Native
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
Native Hawaiian or Other Pacific Islander
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
Other
1
   4.2%
0
   0.0%
2
  22.2%
3
   5.1%
Not allowed based on local regulations
3
  12.5%
1
   3.8%
3
  33.3%
7
  11.9%
[1]
Measure Description: Customized race population was reported here.
1.Primary Outcome
Title Number of Participants Who Achieved at Least a 20 Percent (%) Reduction in Weight-Normalized Average Daily Parenteral Nutrition Intravenous (PN/IV) Volume at Week 24
Hide Description Reduction in weight-normalized PN/IV volume was performed using both participant diary and investigator prescribed data. Number of participants who achieved at least a 20% reduction in weight-normalized PN/IV volume between the baseline and week 24/EOT visit were reported.
Time Frame Baseline through Week 24
Hide Outcome Measure Data
Hide Analysis Population Description
ITT population included all enrolled participants.
Arm/Group Title 0.025 mg/kg/Day Teduglutide 0.05 mg/kg/Day Teduglutide Standard of Care
Hide Arm/Group Description:
Participants received 0.025 mg/kg/day of teduglutide subcutaneously for 24 weeks along with standard medical therapy.
Participants received 0.05 mg/kg/day of teduglutide subcutaneously for 24 weeks along with standard medical therapy.
Participants received standard medical therapy.
Overall Number of Participants Analyzed 24 26 9
Measure Type: Count of Participants
Unit of Measure: Participants
Participant Diary
13
  54.2%
18
  69.2%
1
  11.1%
Investigator Prescribed
13
  54.2%
18
  69.2%
2
  22.2%
2.Secondary Outcome
Title Number of Participants With Treatment-emergent Adverse Events (TEAEs)
Hide Description An adverse event (AE) was defined as any untoward medical occurrence in a clinical investigation participant administered a pharmaceutical product and that did not necessarily have a causal relationship with this treatment. TEAEs were defined as AEs that started or worsened on or after the date of first dose for treatment groups and those that started or worsened on or after the baseline visit for standard of care group.
Time Frame From start of study treatment up to 28 weeks
Hide Outcome Measure Data
Hide Analysis Population Description
Safety analysis population included all participants who received at least 1 dose of teduglutide and had undergone at least 1 post-baseline safety assessment in teduglutide treatment group or participants who had undergone at least 1 post-baseline safety assessment in standard of care treatment group.
Arm/Group Title 0.025 mg/kg/Day Teduglutide 0.05 mg/kg/Day Teduglutide Standard of Care
Hide Arm/Group Description:
Participants received 0.025 mg/kg/day of teduglutide subcutaneously for 24 weeks along with standard medical therapy.
Participants received 0.05 mg/kg/day of teduglutide subcutaneously for 24 weeks along with standard medical therapy.
Participants received standard medical therapy.
Overall Number of Participants Analyzed 24 26 9
Measure Type: Count of Participants
Unit of Measure: Participants
24
 100.0%
25
  96.2%
9
 100.0%
3.Secondary Outcome
Title Number of Participants Who Were Completely Weaned Off Parenteral Nutrition Intravenous (PN/IV) Support at Week 24
Hide Description A participant was considered to have achieved independence from PN/IV support (completely weaned off PN/IV) if the investigator prescribed no PN/IV at EOT and there was no use of PN/IV recorded in the participant diary during the week prior to EOT.
Time Frame Week 24
Hide Outcome Measure Data
Hide Analysis Population Description
ITT population included all enrolled participants.
Arm/Group Title 0.025 mg/kg/Day Teduglutide 0.05 mg/kg/Day Teduglutide Standard of Care
Hide Arm/Group Description:
Participants received 0.025 mg/kg/day of teduglutide subcutaneously for 24 weeks along with standard medical therapy.
Participants received 0.05 mg/kg/day of teduglutide subcutaneously for 24 weeks along with standard medical therapy.
Participants received standard medical therapy.
Overall Number of Participants Analyzed 24 26 9
Measure Type: Count of Participants
Unit of Measure: Participants
2
   8.3%
3
  11.5%
0
   0.0%
4.Secondary Outcome
Title Change From Baseline in Parenteral Nutrition Intravenous (PN/IV) Volume at Week 24
Hide Description Change in PN/IV volume was reported based on the participant diary and the investigator prescribed data.
Time Frame Baseline, Week 24
Hide Outcome Measure Data
Hide Analysis Population Description
ITT population included all enrolled participants.
Arm/Group Title 0.025 mg/kg/Day Teduglutide 0.05 mg/kg/Day Teduglutide Standard of Care
Hide Arm/Group Description:
Participants received 0.025 mg/kg/day of teduglutide subcutaneously for 24 weeks along with standard medical therapy.
Participants received 0.05 mg/kg/day of teduglutide subcutaneously for 24 weeks along with standard medical therapy.
Participants received standard medical therapy.
Overall Number of Participants Analyzed 24 26 9
Mean (Standard Deviation)
Unit of Measure: Milliliters per kilogram per day
Participant Diary Number Analyzed 20 participants 25 participants 9 participants
-16.16  (10.52) -23.30  (17.50) -6.03  (4.55)
Investigator Prescribed Number Analyzed 24 participants 26 participants 9 participants
-11.28  (15.51) -22.13  (17.92) -5.84  (9.80)
5.Secondary Outcome
Title Change From Baseline in Parenteral Nutrition Intravenous (PN/IV) Caloric Intake at Week 24
Hide Description Change in PN/IV caloric intake was reported based on the participant diary and the investigator prescribed data.
Time Frame Baseline, Week 24
Hide Outcome Measure Data
Hide Analysis Population Description
ITT population included all enrolled participants.
Arm/Group Title 0.025 mg/kg/Day Teduglutide 0.05 mg/kg/Day Teduglutide Standard of Care
Hide Arm/Group Description:
Participants received 0.025 mg/kg/day of teduglutide subcutaneously for 24 weeks along with standard medical therapy.
Participants received 0.05 mg/kg/day of teduglutide subcutaneously for 24 weeks along with standard medical therapy.
Participants received standard medical therapy.
Overall Number of Participants Analyzed 24 26 9
Mean (Standard Deviation)
Unit of Measure: Kilocalories per kilogram per day
Participant Diary Number Analyzed 20 participants 25 participants 9 participants
-14.92  (8.29) -18.99  (14.28) -0.46  (4.95)
Investigator Prescribed Number Analyzed 24 participants 25 participants 9 participants
-11.76  (10.46) -18.51  (13.22) -0.27  (2.73)
6.Secondary Outcome
Title Change From Baseline in Plasma Citrulline Levels at Week 24
Hide Description Plasma citrulline level was reported.
Time Frame Baseline, Week 24
Hide Outcome Measure Data
Hide Analysis Population Description
ITT population with number of participants evaluable for this outcome measure.
Arm/Group Title 0.025 mg/kg/Day Teduglutide 0.05 mg/kg/Day Teduglutide Standard of Care
Hide Arm/Group Description:
Participants received 0.025 mg/kg/day of teduglutide subcutaneously for 24 weeks along with standard medical therapy.
Participants received 0.05 mg/kg/day of teduglutide subcutaneously for 24 weeks along with standard medical therapy.
Participants received standard medical therapy.
Overall Number of Participants Analyzed 21 24 8
Mean (Standard Deviation)
Unit of Measure: Micromoles per liter
7.7  (8.50) 12.0  (12.00) 0.1  (7.79)
7.Secondary Outcome
Title Change From Baseline in Enteral Nutrition Volume at Week 24
Hide Description Enteral nutrition was defined as specialized formula taken orally or by tube feeding, and excluded table foods and other fluids. Change in enteral nutrition volume was reported.
Time Frame Baseline, Week 24
Hide Outcome Measure Data
Hide Analysis Population Description
ITT population included all enrolled participants.
Arm/Group Title 0.025 mg/kg/Day Teduglutide 0.05 mg/kg/Day Teduglutide Standard of Care
Hide Arm/Group Description:
Participants received 0.025 mg/kg/day of teduglutide subcutaneously for 24 weeks along with standard medical therapy.
Participants received 0.05 mg/kg/day of teduglutide subcutaneously for 24 weeks along with standard medical therapy.
Participants received standard medical therapy.
Overall Number of Participants Analyzed 24 26 9
Mean (Standard Deviation)
Unit of Measure: Milliliter per kilogram per day
Participant Diary Number Analyzed 18 participants 25 participants 9 participants
7.69  (13.46) 10.96  (16.59) 0.74  (5.91)
Investigator Prescribed Number Analyzed 18 participants 21 participants 5 participants
7.67  (17.77) 8.17  (17.87) 0.33  (0.90)
8.Secondary Outcome
Title Change From Baseline in Enteral Nutrition Caloric Intake at Week 24
Hide Description Enteral nutrition was defined as specialized formula taken orally or by tube feeding, and excluded table foods and other fluids. Change in enteral nutrition caloric intake was reported.
Time Frame Baseline, Week 24
Hide Outcome Measure Data
Hide Analysis Population Description
ITT population included all enrolled participants.
Arm/Group Title 0.025 mg/kg/Day Teduglutide 0.05 mg/kg/Day Teduglutide Standard of Care
Hide Arm/Group Description:
Participants received 0.025 mg/kg/day of teduglutide subcutaneously for 24 weeks along with standard medical therapy.
Participants received 0.05 mg/kg/day of teduglutide subcutaneously for 24 weeks along with standard medical therapy.
Participants received standard medical therapy.
Overall Number of Participants Analyzed 24 26 9
Mean (Standard Deviation)
Unit of Measure: Kilocalorie per kilogram per day
Participant Diary Number Analyzed 18 participants 25 participants 9 participants
8.43  (14.39) 12.98  (18.93) 4.22  (13.75)
Investigator Prescribed Number Analyzed 18 participants 21 participants 5 participants
7.29  (15.88) 11.47  (21.13) 6.66  (14.77)
9.Secondary Outcome
Title Change From Week 24 in Parenteral Nutrition Intravenous (PN/IV) Volume at Week 28
Hide Description Change in PN/IV volume was reported.
Time Frame Week 24, Week 28
Hide Outcome Measure Data
Hide Analysis Population Description
ITT population included all enrolled participants.
Arm/Group Title 0.025 mg/kg/Day Teduglutide 0.05 mg/kg/Day Teduglutide Standard of Care
Hide Arm/Group Description:
Participants received 0.025 mg/kg/day of teduglutide subcutaneously for 24 weeks along with standard medical therapy.
Participants received 0.05 mg/kg/day of teduglutide subcutaneously for 24 weeks along with standard medical therapy.
Participants received standard medical therapy.
Overall Number of Participants Analyzed 24 26 9
Mean (Standard Deviation)
Unit of Measure: Milliliter per kilogram per day
Participant Diary Number Analyzed 24 participants 26 participants 8 participants
2.63  (7.80) 1.52  (12.68) -2.99  (4.94)
Investigator Prescribed Number Analyzed 24 participants 26 participants 9 participants
2.13  (6.95) -0.35  (5.96) -0.91  (2.18)
10.Secondary Outcome
Title Change From Week 24 in Parenteral Nutrition Intravenous (PN/IV) Caloric Intake at Week 28
Hide Description Change in PN/IV caloric intake was reported.
Time Frame Week 24, Week 28
Hide Outcome Measure Data
Hide Analysis Population Description
ITT population included all enrolled participants.
Arm/Group Title 0.025 mg/kg/Day Teduglutide 0.05 mg/kg/Day Teduglutide Standard of Care
Hide Arm/Group Description:
Participants received 0.025 mg/kg/day of teduglutide subcutaneously for 24 weeks along with standard medical therapy.
Participants received 0.05 mg/kg/day of teduglutide subcutaneously for 24 weeks along with standard medical therapy.
Participants received standard medical therapy.
Overall Number of Participants Analyzed 24 26 9
Mean (Standard Deviation)
Unit of Measure: Kilocalorie per kilogram per day
Participant Diary Number Analyzed 24 participants 26 participants 8 participants
0.88  (2.94) -0.91  (7.14) -4.21  (10.30)
Investigator Prescribed Number Analyzed 24 participants 26 participants 9 participants
0.55  (3.42) 0.08  (6.13) -4.34  (11.22)
11.Secondary Outcome
Title Change From Week 24 in Plasma Citrulline Levels at Week 28
Hide Description Change in plasma citrulline level was reported.
Time Frame Week 24, Week 28
Hide Outcome Measure Data
Hide Analysis Population Description
ITT population with number of participants evaluable for this outcome measure.
Arm/Group Title 0.025 mg/kg/Day Teduglutide 0.05 mg/kg/Day Teduglutide Standard of Care
Hide Arm/Group Description:
Participants received 0.025 mg/kg/day of teduglutide subcutaneously for 24 weeks along with standard medical therapy.
Participants received 0.05 mg/kg/day of teduglutide subcutaneously for 24 weeks along with standard medical therapy.
Participants received standard medical therapy.
Overall Number of Participants Analyzed 24 25 9
Mean (Standard Deviation)
Unit of Measure: Micromoles per liter
-6.0  (9.26) -9.5  (10.33) 1.8  (6.48)
12.Secondary Outcome
Title Change From Week 24 in Enteral Nutrition Volume at Week 28
Hide Description Enteral nutrition was defined as specialized formula taken orally or by tube feeding, and excluded table foods and other fluids. Change in enteral nutrition volume was reported.
Time Frame Week 24, Week 28
Hide Outcome Measure Data
Hide Analysis Population Description
ITT population included all enrolled participants.
Arm/Group Title 0.025 mg/kg/Day Teduglutide 0.05 mg/kg/Day Teduglutide Standard of Care
Hide Arm/Group Description:
Participants received 0.025 mg/kg/day of teduglutide subcutaneously for 24 weeks along with standard medical therapy.
Participants received 0.05 mg/kg/day of teduglutide subcutaneously for 24 weeks along with standard medical therapy.
Participants received standard medical therapy.
Overall Number of Participants Analyzed 24 26 9
Mean (Standard Deviation)
Unit of Measure: Milliliter per kilogram per day
Participant Diary Number Analyzed 23 participants 26 participants 8 participants
-0.68  (3.86) 1.38  (8.41) -0.75  (6.78)
Investigator Prescribed Number Analyzed 18 participants 22 participants 5 participants
1.67  (5.73) 0.68  (6.65) 0.39  (0.57)
13.Secondary Outcome
Title Change From Week 24 in Enteral Nutrition Caloric Intake at Week 28
Hide Description Enteral nutrition was defined as specialized formula taken orally or by tube feeding, and excluded table foods and other fluids. Change in enteral nutrition caloric intake was reported.
Time Frame Week 24, Week 28
Hide Outcome Measure Data
Hide Analysis Population Description
ITT population included all enrolled participants.
Arm/Group Title 0.025 mg/kg/Day Teduglutide 0.05 mg/kg/Day Teduglutide Standard of Care
Hide Arm/Group Description:
Participants received 0.025 mg/kg/day of teduglutide subcutaneously for 24 weeks along with standard medical therapy.
Participants received 0.05 mg/kg/day of teduglutide subcutaneously for 24 weeks along with standard medical therapy.
Participants received standard medical therapy.
Overall Number of Participants Analyzed 24 26 9
Mean (Standard Deviation)
Unit of Measure: Kilocalorie per kilogram per day
Participant Diary Number Analyzed 23 participants 26 participants 8 participants
-1.07  (4.36) -0.14  (7.73) -0.42  (6.13)
Investigator Prescribed Number Analyzed 18 participants 22 participants 5 participants
1.28  (4.30) -0.11  (3.77) 0.45  (0.64)
14.Secondary Outcome
Title Change From Baseline in Body Weight Z-score at Week 28
Hide Description Body weight z-score is a measure of relative weight adjusted for child age and sex. The Z-score indicates the number of standard deviations away from a reference population in the same age range and with the same sex. A Z-score of 0 is equal to the mean. Negative numbers indicate values lower than the mean and positive numbers indicate values higher than the mean.
Time Frame Baseline, Week 28
Hide Outcome Measure Data
Hide Analysis Population Description
Safety analysis population included all participants who received at least 1 dose of teduglutide and had undergone at least 1 post-baseline safety assessment in teduglutide treatment group or participants who had undergone at least 1 post-baseline safety assessment in standard of care treatment group.
Arm/Group Title 0.025 mg/kg/Day Teduglutide 0.05 mg/kg/Day Teduglutide Standard of Care
Hide Arm/Group Description:
Participants received 0.025 mg/kg/day of teduglutide subcutaneously for 24 weeks along with standard medical therapy.
Participants received 0.05 mg/kg/day of teduglutide subcutaneously for 24 weeks along with standard medical therapy.
Participants received standard medical therapy.
Overall Number of Participants Analyzed 24 26 9
Mean (Standard Deviation)
Unit of Measure: Z-score
-0.12  (0.41) -0.18  (0.59) -0.05  (0.37)
15.Secondary Outcome
Title Change From Baseline in Body Height Z-score at Week 28
Hide Description Body height z-score is a measure of relative height adjusted for child age and sex. The Z-score indicates the number of standard deviations away from a reference population in the same age range and with the same sex. A Z-score of 0 is equal to the mean. Negative numbers indicate values lower than the mean and positive numbers indicate values higher than the mean.
Time Frame Baseline, Week 28
Hide Outcome Measure Data
Hide Analysis Population Description
Safety analysis population with number of participants evaluable for this outcome measure.
Arm/Group Title 0.025 mg/kg/Day Teduglutide 0.05 mg/kg/Day Teduglutide Standard of Care
Hide Arm/Group Description:
Participants received 0.025 mg/kg/day of teduglutide subcutaneously for 24 weeks along with standard medical therapy.
Participants received 0.05 mg/kg/day of teduglutide subcutaneously for 24 weeks along with standard medical therapy.
Participants received standard medical therapy.
Overall Number of Participants Analyzed 24 25 9
Mean (Standard Deviation)
Unit of Measure: Z-score
0  (0.29) 0.05  (0.45) 0.16  (0.66)
16.Secondary Outcome
Title Change From Baseline in Head Circumference Z-score at Week 28
Hide Description Head circumference z-score is a measure of relative head circumference adjusted for child age and sex. The Z-score indicates the number of standard deviations away from a reference population in the same age range and with the same sex. A Z-score of 0 is equal to the mean. Negative numbers indicate values lower than the mean and positive numbers indicate values higher than the mean. Head circumference was collected only for participants of less than or equal to (<=) 36 months of age at the time of measurement.
Time Frame Baseline, Week 28
Hide Outcome Measure Data
Hide Analysis Population Description
Safety analysis population with number of participants evaluable for this outcome measure.
Arm/Group Title 0.025 mg/kg/Day Teduglutide 0.05 mg/kg/Day Teduglutide Standard of Care
Hide Arm/Group Description:
Participants received 0.025 mg/kg/day of teduglutide subcutaneously for 24 weeks along with standard medical therapy.
Participants received 0.05 mg/kg/day of teduglutide subcutaneously for 24 weeks along with standard medical therapy.
Participants received standard medical therapy.
Overall Number of Participants Analyzed 0 0 1
Mean (Standard Deviation)
Unit of Measure: Z-score
-0.014 [1]   (NA)
[1]
"NA" signifies that the standard deviation was not calculated due to less number of participants.
17.Secondary Outcome
Title Change From Baseline in Body Mass Index (BMI) Z-score at Week 28
Hide Description BMI z-score is a measure of relative BMI adjusted for child age and sex. The Z-score indicates the number of standard deviations away from a reference population in the same age range and with the same sex. A Z-score of 0 is equal to the mean. Negative numbers indicate values lower than the mean and positive numbers indicate values higher than the mean.
Time Frame Baseline, Week 28
Hide Outcome Measure Data
Hide Analysis Population Description
Safety analysis population with number of participants evaluable for this outcome measure.
Arm/Group Title 0.025 mg/kg/Day Teduglutide 0.05 mg/kg/Day Teduglutide Standard of Care
Hide Arm/Group Description:
Participants received 0.025 mg/kg/day of teduglutide subcutaneously for 24 weeks along with standard medical therapy.
Participants received 0.05 mg/kg/day of teduglutide subcutaneously for 24 weeks along with standard medical therapy.
Participants received standard medical therapy.
Overall Number of Participants Analyzed 24 25 9
Mean (Standard Deviation)
Unit of Measure: Z-score
-0.13  (0.57) -0.22  (0.70) -0.25  (1.42)
18.Secondary Outcome
Title Change From Baseline in Participants' Stool Consistency at Week 28
Hide Description Stool consistency was assessed by typical stool form based on Bristol Stool Form Scale: 1 - Separate hard lumps, hard to pass, 2 - Sausage-shaped, but lumpy, 3 - Like a sausage but with cracks on the surface, 4 - Like a sausage or snake, smooth and soft, 5 - Soft blobs with clear-cut edges, 6 - Fluffy pieces with ragged edges, a mushy stool, 7 - Watery, no solid pieces, entirely liquid.
Time Frame Baseline, Week 28
Hide Outcome Measure Data
Hide Analysis Population Description
Safety analysis population with number of participants evaluable for this outcome measure.
Arm/Group Title 0.025 mg/kg/Day Teduglutide 0.05 mg/kg/Day Teduglutide Standard of Care
Hide Arm/Group Description:
Participants received 0.025 mg/kg/day of teduglutide subcutaneously for 24 weeks along with standard medical therapy.
Participants received 0.05 mg/kg/day of teduglutide subcutaneously for 24 weeks along with standard medical therapy.
Participants received standard medical therapy.
Overall Number of Participants Analyzed 8 16 2
Mean (Standard Deviation)
Unit of Measure: Score on a scale
-1.3  (1.77) -1.4  (1.38) -3.3  (0.35)
19.Secondary Outcome
Title Change From Baseline in Hours Per Day of Parenteral Nutrition Intravenous (PN/IV) Support at Week 24
Hide Description The mean duration of the PN/IV infusions in hours, on the days when PN/IV was administered was reported.
Time Frame Baseline, Week 24
Hide Outcome Measure Data
Hide Analysis Population Description
ITT population included all enrolled participants.
Arm/Group Title 0.025 mg/kg/Day Teduglutide 0.05 mg/kg/Day Teduglutide Standard of Care
Hide Arm/Group Description:
Participants received 0.025 mg/kg/day of teduglutide subcutaneously for 24 weeks along with standard medical therapy.
Participants received 0.05 mg/kg/day of teduglutide subcutaneously for 24 weeks along with standard medical therapy.
Participants received standard medical therapy.
Overall Number of Participants Analyzed 24 26 9
Mean (Standard Deviation)
Unit of Measure: Hours per day (hour/day)
Participant Diary Number Analyzed 22 participants 26 participants 9 participants
-2.47  (2.73) -3.03  (3.84) -0.21  (0.69)
Investigator Prescribed Number Analyzed 24 participants 26 participants 9 participants
-1.48  (3.59) -1.79  (3.52) 0.11  (0.33)
20.Secondary Outcome
Title Change From Baseline in Days Per Week of Parenteral Nutrition Intravenous (PN/IV) Support at Week 24
Hide Description The number of days per week of PN/IV infusions were reported.
Time Frame Baseline, Week 24
Hide Outcome Measure Data
Hide Analysis Population Description
ITT population included all enrolled participants.
Arm/Group Title 0.025 mg/kg/Day Teduglutide 0.05 mg/kg/Day Teduglutide Standard of Care
Hide Arm/Group Description:
Participants received 0.025 mg/kg/day of teduglutide subcutaneously for 24 weeks along with standard medical therapy.
Participants received 0.05 mg/kg/day of teduglutide subcutaneously for 24 weeks along with standard medical therapy.
Participants received standard medical therapy.
Overall Number of Participants Analyzed 24 26 9
Mean (Standard Deviation)
Unit of Measure: Days per week (Days/week)
Participant Diary Number Analyzed 22 participants 26 participants 9 participants
-0.88  (1.78) -1.34  (2.24) 0  (0)
Investigator Prescribed Number Analyzed 24 participants 26 participants 9 participants
-0.79  (1.62) -1.42  (2.32) 0  (0)
Time Frame From start of study treatment up to 28 weeks
Adverse Event Reporting Description [Not Specified]
 
Arm/Group Title 0.025 mg/kg/Day Teduglutide 0.05 mg/kg/Day Teduglutide Standard of Care
Hide Arm/Group Description Participants received 0.025 mg/kg/day of teduglutide subcutaneously for 24 weeks along with standard medical therapy. Participants received 0.05 mg/kg/day of teduglutide subcutaneously for 24 weeks along with standard medical therapy. Participants received standard medical therapy.
All-Cause Mortality
0.025 mg/kg/Day Teduglutide 0.05 mg/kg/Day Teduglutide Standard of Care
Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%)
Total   0/24 (0.00%)      0/26 (0.00%)      0/9 (0.00%)    
Show Serious Adverse Events Hide Serious Adverse Events
0.025 mg/kg/Day Teduglutide 0.05 mg/kg/Day Teduglutide Standard of Care
Affected / at Risk (%) # Events Affected / at Risk (%) # Events Affected / at Risk (%) # Events
Total   15/24 (62.50%)      20/26 (76.92%)      4/9 (44.44%)    
Gastrointestinal disorders       
Diarrhoea * 1  0/24 (0.00%)  0 1/26 (3.85%)  1 0/9 (0.00%)  0
Faecaloma * 1  1/24 (4.17%)  1 0/26 (0.00%)  0 0/9 (0.00%)  0
Haematemesis * 1  0/24 (0.00%)  0 1/26 (3.85%)  1 0/9 (0.00%)  0
Ileus * 1  1/24 (4.17%)  1 0/26 (0.00%)  0 0/9 (0.00%)  0
Vomiting * 1  1/24 (4.17%)  1 0/26 (0.00%)  0 0/9 (0.00%)  0
General disorders       
Pyrexia * 1  4/24 (16.67%)  6 7/26 (26.92%)  8 1/9 (11.11%)  3
Hepatobiliary disorders       
Cholestasis * 1  1/24 (4.17%)  1 0/26 (0.00%)  0 0/9 (0.00%)  0
Infections and infestations       
Bacteraemia * 1  0/24 (0.00%)  0 1/26 (3.85%)  1 0/9 (0.00%)  0
Catheter site infection * 1  0/24 (0.00%)  0 3/26 (11.54%)  3 0/9 (0.00%)  0
Corona virus infection * 1  0/24 (0.00%)  0 1/26 (3.85%)  1 1/9 (11.11%)  1
Device related infection * 1  1/24 (4.17%)  2 3/26 (11.54%)  3 0/9 (0.00%)  0
Device related sepsis * 1  1/24 (4.17%)  1 0/26 (0.00%)  0 0/9 (0.00%)  0
Fungaemia * 1  0/24 (0.00%)  0 0/26 (0.00%)  0 1/9 (11.11%)  1
Gastritis viral * 1  0/24 (0.00%)  0 1/26 (3.85%)  1 0/9 (0.00%)  0
Gastroenteritis * 1  0/24 (0.00%)  0 1/26 (3.85%)  1 0/9 (0.00%)  0
Influenza * 1  2/24 (8.33%)  2 2/26 (7.69%)  2 0/9 (0.00%)  0
Metapneumovirus infection * 1  0/24 (0.00%)  0 1/26 (3.85%)  1 0/9 (0.00%)  0
Orchitis * 1  1/24 (4.17%)  1 0/26 (0.00%)  0 0/9 (0.00%)  0
Otitis media * 1  0/24 (0.00%)  0 1/26 (3.85%)  1 0/9 (0.00%)  0
Otitis media acute * 1  0/24 (0.00%)  0 0/26 (0.00%)  0 1/9 (11.11%)  1
Parainfluenzae virus infection * 1  1/24 (4.17%)  1 0/26 (0.00%)  0 0/9 (0.00%)  0
Pneumonia * 1  0/24 (0.00%)  0 1/26 (3.85%)  1 0/9 (0.00%)  0
Respiratory syncytial virus infection * 1  0/24 (0.00%)  0 1/26 (3.85%)  1 0/9 (0.00%)  0
Roseola * 1  0/24 (0.00%)  0 0/26 (0.00%)  0 1/9 (11.11%)  1
Rotavirus infection * 1  0/24 (0.00%)  0 1/26 (3.85%)  1 0/9 (0.00%)  0
Subcutaneous abscess * 1  0/24 (0.00%)  0 1/26 (3.85%)  1 0/9 (0.00%)  0
Upper respiratory tract infection * 1  2/24 (8.33%)  2 2/26 (7.69%)  2 0/9 (0.00%)  0
Urinary tract infection * 1  1/24 (4.17%)  2 0/26 (0.00%)  0 1/9 (11.11%)  1
Viral infection * 1  0/24 (0.00%)  0 2/26 (7.69%)  2 1/9 (11.11%)  1
Viral upper respiratory tract infection * 1  0/24 (0.00%)  0 0/26 (0.00%)  0 1/9 (11.11%)  1
Investigations       
Blood urea increased * 1  1/24 (4.17%)  1 0/26 (0.00%)  0 0/9 (0.00%)  0
Transaminases increased * 1  0/24 (0.00%)  0 1/26 (3.85%)  1 0/9 (0.00%)  0
Metabolism and nutrition disorders       
Acidosis * 1  0/24 (0.00%)  0 1/26 (3.85%)  1 0/9 (0.00%)  0
Dehydration * 1  4/24 (16.67%)  4 0/26 (0.00%)  0 0/9 (0.00%)  0
Hypokalaemia * 1  1/24 (4.17%)  1 1/26 (3.85%)  1 0/9 (0.00%)  0
Metabolic acidosis * 1  1/24 (4.17%)  1 1/26 (3.85%)  1 0/9 (0.00%)  0
Product Issues       
Device breakage * 1 [1]  2/24 (8.33%)  3 2/26 (7.69%)  2 0/9 (0.00%)  0
Device dislocation * 1 [1]  1/24 (4.17%)  1 0/26 (0.00%)  0 0/9 (0.00%)  0
Device issue * 1 [1]  0/24 (0.00%)  0 1/26 (3.85%)  1 0/9 (0.00%)  0
Device occlusion * 1 [1]  0/24 (0.00%)  0 1/26 (3.85%)  1 0/9 (0.00%)  0
Respiratory, thoracic and mediastinal disorders       
Asthma * 1  0/24 (0.00%)  0 1/26 (3.85%)  1 0/9 (0.00%)  0
Hypocapnia * 1  0/24 (0.00%)  0 1/26 (3.85%)  1 0/9 (0.00%)  0
Vascular disorders       
Superior vena cava syndrome * 1  0/24 (0.00%)  0 1/26 (3.85%)  1 0/9 (0.00%)  0
1
Term from vocabulary, MedDRA 19.1
*
Indicates events were collected by non-systematic assessment
[1]
TEAEs coded to Product issues were related to central line complications, and not due to complications of the investigational product and ancillary supplies.
Show Other (Not Including Serious) Adverse Events Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 5%
0.025 mg/kg/Day Teduglutide 0.05 mg/kg/Day Teduglutide Standard of Care
Affected / at Risk (%) # Events Affected / at Risk (%) # Events Affected / at Risk (%) # Events
Total   24/24 (100.00%)      23/26 (88.46%)      9/9 (100.00%)    
Blood and lymphatic system disorders       
Lymphadenopathy * 1  0/24 (0.00%)  0 1/26 (3.85%)  1 1/9 (11.11%)  1
Ear and labyrinth disorders       
Ear pain * 1  1/24 (4.17%)  1 1/26 (3.85%)  1 1/9 (11.11%)  1
Gastrointestinal disorders       
Abdominal distension * 1  0/24 (0.00%)  0 2/26 (7.69%)  2 0/9 (0.00%)  0
Abdominal pain * 1  4/24 (16.67%)  5 6/26 (23.08%)  7 0/9 (0.00%)  0
Abdominal pain lower * 1  2/24 (8.33%)  2 0/26 (0.00%)  0 0/9 (0.00%)  0
Abdominal pain upper * 1  3/24 (12.50%)  8 3/26 (11.54%)  3 1/9 (11.11%)  1
Diarrhoea * 1  8/24 (33.33%)  9 2/26 (7.69%)  3 1/9 (11.11%)  1
Nausea * 1  3/24 (12.50%)  3 3/26 (11.54%)  3 1/9 (11.11%)  1
Perianal erythema * 1  0/24 (0.00%)  0 1/26 (3.85%)  1 1/9 (11.11%)  2
Proctalgia * 1  0/24 (0.00%)  0 1/26 (3.85%)  1 1/9 (11.11%)  2
Vomiting * 1  10/24 (41.67%)  23 8/26 (30.77%)  17 5/9 (55.56%)  7
General disorders       
Catheter site erythema * 1  0/24 (0.00%)  0 1/26 (3.85%)  1 2/9 (22.22%)  2
Catheter site related reaction * 1  0/24 (0.00%)  0 0/26 (0.00%)  0 1/9 (11.11%)  1
Injection site bruising * 1  3/24 (12.50%)  4 1/26 (3.85%)  1 0/9 (0.00%)  0
Medical device site pain * 1  0/24 (0.00%)  0 2/26 (7.69%)  2 1/9 (11.11%)  1
Pain * 1  2/24 (8.33%)  2 1/26 (3.85%)  1 0/9 (0.00%)  0
Pyrexia * 1  5/24 (20.83%)  6 6/26 (23.08%)  9 3/9 (33.33%)  4
Hepatobiliary disorders       
Drug-Induced liver injury * 1  0/24 (0.00%)  0 0/26 (0.00%)  0 1/9 (11.11%)  1
Immune system disorders       
Seasonal allergy * 1  1/24 (4.17%)  1 2/26 (7.69%)  2 0/9 (0.00%)  0
Infections and infestations       
Cellulitis * 1  2/24 (8.33%)  3 0/26 (0.00%)  0 0/9 (0.00%)  0
Conjunctivitis * 1  3/24 (12.50%)  3 1/26 (3.85%)  1 0/9 (0.00%)  0
Device related infection * 1  0/24 (0.00%)  0 2/26 (7.69%)  2 0/9 (0.00%)  0
Ear infection * 1  1/24 (4.17%)  1 3/26 (11.54%)  3 1/9 (11.11%)  1
Gastroenteritis viral * 1  3/24 (12.50%)  3 0/26 (0.00%)  0 0/9 (0.00%)  0
Gastrointestinal bacterial overgrowth * 1  2/24 (8.33%)  6 0/26 (0.00%)  0 0/9 (0.00%)  0
Nasopharyngitis * 1  4/24 (16.67%)  4 6/26 (23.08%)  9 2/9 (22.22%)  2
Otitis media * 1  0/24 (0.00%)  0 0/26 (0.00%)  0 1/9 (11.11%)  1
Pharyngitis * 1  0/24 (0.00%)  0 2/26 (7.69%)  2 0/9 (0.00%)  0
Respiratory tract infection * 1  0/24 (0.00%)  0 2/26 (7.69%)  3 0/9 (0.00%)  0
Respiratory tract infection viral * 1  0/24 (0.00%)  0 0/26 (0.00%)  0 1/9 (11.11%)  2
Rhinitis * 1  1/24 (4.17%)  1 5/26 (19.23%)  6 0/9 (0.00%)  0
Upper respiratory tract infection * 1  5/24 (20.83%)  7 6/26 (23.08%)  8 4/9 (44.44%)  5
Urinary tract infection * 1  2/24 (8.33%)  4 1/26 (3.85%)  1 0/9 (0.00%)  0
Urinary tract infection bacterial * 1  0/24 (0.00%)  0 0/26 (0.00%)  0 1/9 (11.11%)  1
Viral infection * 1  3/24 (12.50%)  4 1/26 (3.85%)  2 1/9 (11.11%)  1
Viral upper respiratory tract infection * 1  0/24 (0.00%)  0 0/26 (0.00%)  0 1/9 (11.11%)  1
Injury, poisoning and procedural complications       
Anaesthetic complication neurological * 1  0/24 (0.00%)  0 0/26 (0.00%)  0 1/9 (11.11%)  1
Stoma site erythema * 1  0/24 (0.00%)  0 2/26 (7.69%)  3 0/9 (0.00%)  0
Investigations       
Alanine aminotransferase increased * 1  7/24 (29.17%)  7 2/26 (7.69%)  2 0/9 (0.00%)  0
Aspartate aminotransferase increased * 1  5/24 (20.83%)  5 0/26 (0.00%)  0 0/9 (0.00%)  0
Blood bicarbonate decreased * 1  4/24 (16.67%)  5 0/26 (0.00%)  0 0/9 (0.00%)  0
Blood triglycerides increased * 1  2/24 (8.33%)  2 1/26 (3.85%)  1 0/9 (0.00%)  0
Gamma-Glutamyltransferase increased * 1  2/24 (8.33%)  2 0/26 (0.00%)  0 0/9 (0.00%)  0
Lymph node palpable * 1  2/24 (8.33%)  2 0/26 (0.00%)  0 1/9 (11.11%)  1
Transaminases increased * 1  0/24 (0.00%)  0 0/26 (0.00%)  0 1/9 (11.11%)  1
Metabolism and nutrition disorders       
Acidosis * 1  2/24 (8.33%)  2 0/26 (0.00%)  0 0/9 (0.00%)  0
Dehydration * 1  5/24 (20.83%)  9 1/26 (3.85%)  1 0/9 (0.00%)  0
Metabolic acidosis * 1  2/24 (8.33%)  4 0/26 (0.00%)  0 0/9 (0.00%)  0
Musculoskeletal and connective tissue disorders       
Muscle spasms * 1  1/24 (4.17%)  1 0/26 (0.00%)  0 1/9 (11.11%)  1
Pain in extremity * 1  2/24 (8.33%)  2 0/26 (0.00%)  0 0/9 (0.00%)  0
Nervous system disorders       
Dizziness * 1  0/24 (0.00%)  0 0/26 (0.00%)  0 1/9 (11.11%)  2
Headache * 1  3/24 (12.50%)  4 5/26 (19.23%)  7 1/9 (11.11%)  3
Product Issues       
Device breakage * 1  2/24 (8.33%)  2 2/26 (7.69%)  2 0/9 (0.00%)  0
Device occlusion * 1  3/24 (12.50%)  3 0/26 (0.00%)  0 0/9 (0.00%)  0
Psychiatric disorders       
Anxiety * 1  0/24 (0.00%)  0 0/26 (0.00%)  0 1/9 (11.11%)  1
Renal and urinary disorders       
Nephrolithiasis * 1  0/24 (0.00%)  0 1/26 (3.85%)  1 1/9 (11.11%)  1
Respiratory, thoracic and mediastinal disorders       
Cough * 1  2/24 (8.33%)  2 10/26 (38.46%)  11 3/9 (33.33%)  4
Hyperventilation * 1  0/24 (0.00%)  0 0/26 (0.00%)  0 1/9 (11.11%)  1
Nasal congestion * 1  1/24 (4.17%)  1 1/26 (3.85%)  1 1/9 (11.11%)  2
Oropharyngeal pain * 1  0/24 (0.00%)  0 1/26 (3.85%)  1 1/9 (11.11%)  1
Rhinorrhoea * 1  3/24 (12.50%)  3 0/26 (0.00%)  0 1/9 (11.11%)  1
Skin and subcutaneous tissue disorders       
Dermatitis diaper * 1  2/24 (8.33%)  20 0/26 (0.00%)  0 0/9 (0.00%)  0
Eczema * 1  1/24 (4.17%)  1 1/26 (3.85%)  1 1/9 (11.11%)  1
Excessive granulation tissue * 1  0/24 (0.00%)  0 1/26 (3.85%)  1 1/9 (11.11%)  1
Rash * 1  1/24 (4.17%)  1 1/26 (3.85%)  4 1/9 (11.11%)  1
Red man syndrome * 1  0/24 (0.00%)  0 0/26 (0.00%)  0 1/9 (11.11%)  1
1
Term from vocabulary, MedDRA 19.1
*
Indicates events were collected by non-systematic assessment
Certain Agreements
Principal Investigators are NOT employed by the organization sponsoring the study.
There IS an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.
If a multicenter publication is not submitted within twelve (12) months after conclusion, abandonmentor termination of the Study at all sites, or after Sponsor confirms there shall be no multicenter Study publication, the Institution and/or such Principal Investigator may publish the results from the Institution site individually.
Results Point of Contact
Name/Title: Study Physician
Organization: Shire
Phone: 1 866-842-5335
Responsible Party: Shire
ClinicalTrials.gov Identifier: NCT02682381     History of Changes
Other Study ID Numbers: TED-C14-006
First Submitted: January 27, 2016
First Posted: February 15, 2016
Results First Submitted: August 3, 2018
Results First Posted: October 16, 2018
Last Update Posted: May 16, 2019