Trial Outcomes & Findings for Study in Japanese Pediatric Subjects With Short Bowel Syndrome (SBS) Who Are Dependent on Parenteral Support (NCT NCT02980666)

Results Overview

GI specific testing included colonoscopy or sigmoidoscopy, abdominal ultrasound, fecal occult blood testing, upper GI series with small bowel follow-through (UGI/SBFT). EOT was defined as the last available measurement after the date of first dose during the 24-week treatment period. Number of participants with clinically significant abnormal findings in gastrointestinal specific testing were reported.

Recruitment status

COMPLETED

Study phase

PHASE3

Target enrollment

10 participants

Primary outcome timeframe

Baseline, EOT (up to Week 24)

Results posted on

2022-02-02

Participant Flow

The study was conducted at 6 centers in Japan between 13 January 2017 (first participant first visit) and 21 January 2020 (last participant last visit).

A total of 10 Japanese study participants were enrolled into the study, including 8 children ages 1 through 15 years of age and 2 infants aged 4 months through \< 12 months of corrected gestational age. All the participants received treatment and completed the study.

Participant milestones

Participant milestones
Measure	Total Children (Aged: 1 to 15 Years) Participants aged from 1 through 15 years received teduglutide 0.05 milligram per kilogram per day (mg/kg/day) subcutaneous (SC) injection once daily for 24 weeks and completed the study at Week 28.	Infants (Corrected Gestational Age: 4 to < 12 Months) Participants (Infants) from 4 through \< 12 months of corrected gestational age received teduglutide 0.05 mg/kg/day SC injection once daily for 24 weeks and completed the study at Week 28.
Overall Study STARTED	8	2
Overall Study COMPLETED	8	2
Overall Study NOT COMPLETED	0	0

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

Study in Japanese Pediatric Subjects With Short Bowel Syndrome (SBS) Who Are Dependent on Parenteral Support

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Total Children (Aged: 1 to 15 Years) n=8 Participants Participants aged from 1 through 15 years received teduglutide 0.05 milligram per kilogram per day (mg/kg/day) subcutaneous (SC) injection once daily for 24 weeks and completed the study at Week 28.	Infants (Corrected Gestational Age: 4 to < 12 Months) n=2 Participants Participants (Infants) from 4 through \< 12 months of corrected gestational age received teduglutide 0.05 mg/kg/day SC injection once daily for 24 weeks and completed the study at Week 28.	Total n=10 Participants Total of all reporting groups
Age, Customized	8 Participants n=39 Participants	2 Participants n=41 Participants	10 Participants n=35 Participants
Sex: Female, Male Female	1 Participants n=39 Participants	1 Participants n=41 Participants	2 Participants n=35 Participants
Sex: Female, Male Male	7 Participants n=39 Participants	1 Participants n=41 Participants	8 Participants n=35 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	0 Participants n=39 Participants	0 Participants n=41 Participants	0 Participants n=35 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	8 Participants n=39 Participants	2 Participants n=41 Participants	10 Participants n=35 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	0 Participants n=39 Participants	0 Participants n=41 Participants	0 Participants n=35 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=39 Participants	0 Participants n=41 Participants	0 Participants n=35 Participants
Race (NIH/OMB) Asian	8 Participants n=39 Participants	2 Participants n=41 Participants	10 Participants n=35 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=39 Participants	0 Participants n=41 Participants	0 Participants n=35 Participants
Race (NIH/OMB) Black or African American	0 Participants n=39 Participants	0 Participants n=41 Participants	0 Participants n=35 Participants
Race (NIH/OMB) White	0 Participants n=39 Participants	0 Participants n=41 Participants	0 Participants n=35 Participants
Race (NIH/OMB) More than one race	0 Participants n=39 Participants	0 Participants n=41 Participants	0 Participants n=35 Participants
Race (NIH/OMB) Unknown or Not Reported	0 Participants n=39 Participants	0 Participants n=41 Participants	0 Participants n=35 Participants

PRIMARY outcome

Timeframe: Baseline, EOT (up to Week 24)

Population: ITT population consisted of all participants who were enrolled into the study and met all eligible criteria for the study.

Absolute change from baseline in PS volume at EOT (up to Week 24) based on dairy data was reported. EOT was defined as the last available measurement after the date of first dose during the 24-week treatment period. Here, milliliter per kilogram per day is abbreviated as mL/kg/day.

Outcome measures

Outcome measures
Measure	Total Children (Aged: 1 to 15 Years) n=8 Participants Participants aged from 1 through 15 years received teduglutide 0.05 milligram per kilogram per day (mg/kg/day) subcutaneous (SC) injection once daily for 24 weeks and completed the study at Week 28.	Infants (Corrected Gestational Age: 4 to < 12 Months) n=2 Participants Participants (Infants) from 4 through \< 12 months of corrected gestational age received teduglutide 0.05 mg/kg/day SC injection once daily for 24 weeks and completed the study at Week 28.
Absolute Change From Baseline in Parenteral Support (PS) Volume at End of Treatment (EOT) Based on Dairy Data	-11.8 mL/kg/day Standard Deviation 8.47	-26.2 mL/kg/day Standard Deviation 13.61

PRIMARY outcome

Timeframe: Baseline, EOT (up to Week 24)

Population: ITT population consisted of all participants who were enrolled into the study and met all eligible criteria for the study.

Percent change from baseline in PS volume at EOT (up to Week 24) based on dairy data was reported. EOT was defined as the last available measurement after the date of first dose during the 24-week treatment period.

Outcome measures

Outcome measures
Measure	Total Children (Aged: 1 to 15 Years) n=8 Participants Participants aged from 1 through 15 years received teduglutide 0.05 milligram per kilogram per day (mg/kg/day) subcutaneous (SC) injection once daily for 24 weeks and completed the study at Week 28.	Infants (Corrected Gestational Age: 4 to < 12 Months) n=2 Participants Participants (Infants) from 4 through \< 12 months of corrected gestational age received teduglutide 0.05 mg/kg/day SC injection once daily for 24 weeks and completed the study at Week 28.
Percent Change From Baseline in Parenteral Support (PS) Volume at End of Treatment (EOT) Based on Dairy Data	-27.7 Percent Change Standard Deviation 31.79	-26.7 Percent Change Standard Deviation 15.14

PRIMARY outcome

Timeframe: Baseline, EOT (up to Week 24)

Population: ITT population consisted of all participants who were enrolled into the study and met all eligible criteria for the study.

Absolute change from baseline in PS caloric intake at EOT (up to Week 24) based on dairy data was reported. EOT was defined as the last available measurement after the date of first dose during the 24-week treatment period. Here, kilo-calories per kilogram per day was abbreviated as (kcal/kg/day).

Outcome measures

Outcome measures
Measure	Total Children (Aged: 1 to 15 Years) n=8 Participants Participants aged from 1 through 15 years received teduglutide 0.05 milligram per kilogram per day (mg/kg/day) subcutaneous (SC) injection once daily for 24 weeks and completed the study at Week 28.	Infants (Corrected Gestational Age: 4 to < 12 Months) n=2 Participants Participants (Infants) from 4 through \< 12 months of corrected gestational age received teduglutide 0.05 mg/kg/day SC injection once daily for 24 weeks and completed the study at Week 28.
Absolute Change From Baseline in Parenteral Support (PS) Caloric Intake at End of Treatment (EOT) Based on Dairy Data	-7.2 kcal/kg/day Standard Deviation 8.70	-13.8 kcal/kg/day Standard Deviation 3.17

PRIMARY outcome

Timeframe: Baseline, EOT (up to Week 24)

Population: ITT population consisted of all participants who were enrolled into the study and met all eligible criteria for this study.

Percent change from baseline in PS caloric intake at EOT (up to Week 24) based on dairy data was reported. EOT was defined as the last available measurement after the date of first dose during the 24-week treatment period.

Outcome measures

Outcome measures
Measure	Total Children (Aged: 1 to 15 Years) n=8 Participants Participants aged from 1 through 15 years received teduglutide 0.05 milligram per kilogram per day (mg/kg/day) subcutaneous (SC) injection once daily for 24 weeks and completed the study at Week 28.	Infants (Corrected Gestational Age: 4 to < 12 Months) n=2 Participants Participants (Infants) from 4 through \< 12 months of corrected gestational age received teduglutide 0.05 mg/kg/day SC injection once daily for 24 weeks and completed the study at Week 28.
Percent Change From Baseline in Parenteral Support (PS) Caloric Intake at End of Treatment (EOT) Based on Dairy Data	-26.2 Percent change Standard Deviation 33.00	-25.7 Percent change Standard Deviation 2.73

PRIMARY outcome

Timeframe: Baseline, EOT (up to Week 24)

Population: ITT population consisted of all participants who were enrolled into the study and met all eligible criteria for this study. Data for this outcome measure was not planned to be collected and analyzed for infants.

Absolute change from baseline in plasma citrulline at EOT (up to Week 24) was reported. EOT was defined as the last available measurement after the date of first dose during the 24-week treatment period.

Outcome measures

Outcome measures
Measure	Total Children (Aged: 1 to 15 Years) n=8 Participants Participants aged from 1 through 15 years received teduglutide 0.05 milligram per kilogram per day (mg/kg/day) subcutaneous (SC) injection once daily for 24 weeks and completed the study at Week 28.	Infants (Corrected Gestational Age: 4 to < 12 Months) Participants (Infants) from 4 through \< 12 months of corrected gestational age received teduglutide 0.05 mg/kg/day SC injection once daily for 24 weeks and completed the study at Week 28.
Absolute Change From Baseline in Plasma Citrulline at End of Treatment (EOT)	3.2 Micromoles per liter (mcmol/L) Standard Deviation 4.28	—

PRIMARY outcome

Timeframe: Baseline, EOT (up to Week 24)

Population: ITT population consisted of all participants who were enrolled into the study and met all eligible criteria for this study. Data for this outcome measure was not planned to be collected and analyzed for infants.

Percent change from baseline in plasma citrulline at EOT (up to Week 24) was reported. EOT was defined as the last available measurement after the date of first dose during the 24-week treatment period.

Outcome measures

Outcome measures
Measure	Total Children (Aged: 1 to 15 Years) n=8 Participants Participants aged from 1 through 15 years received teduglutide 0.05 milligram per kilogram per day (mg/kg/day) subcutaneous (SC) injection once daily for 24 weeks and completed the study at Week 28.	Infants (Corrected Gestational Age: 4 to < 12 Months) Participants (Infants) from 4 through \< 12 months of corrected gestational age received teduglutide 0.05 mg/kg/day SC injection once daily for 24 weeks and completed the study at Week 28.
Percent Change From Baseline in Plasma Citrulline at End of Treatment (EOT)	39.2 Percent change Standard Deviation 39.46	—

PRIMARY outcome

Timeframe: Baseline, EOT (up to Week 24)

Population: ITT population consisted of all participants who were enrolled into the study and met all eligible criteria for this study.

Absolute change from baseline in EN volume at EOT (up to Week 24) based on dairy data was reported. EOT was defined as the last available measurement after the date of first dose during the 24-week treatment period.

Outcome measures

Outcome measures
Measure	Total Children (Aged: 1 to 15 Years) n=8 Participants Participants aged from 1 through 15 years received teduglutide 0.05 milligram per kilogram per day (mg/kg/day) subcutaneous (SC) injection once daily for 24 weeks and completed the study at Week 28.	Infants (Corrected Gestational Age: 4 to < 12 Months) n=2 Participants Participants (Infants) from 4 through \< 12 months of corrected gestational age received teduglutide 0.05 mg/kg/day SC injection once daily for 24 weeks and completed the study at Week 28.
Absolute Change From Baseline in Enteral Nutritional (EN) Volume at End of Treatment (EOT) Based on Dairy Data	1.0 mL/kg/day Standard Deviation 7.14	0.6 mL/kg/day Standard Deviation 2.15

PRIMARY outcome

Timeframe: Baseline, EOT (up to Week 24)

Population: ITT population consisted of all participants who were enrolled into the study and met all eligible criteria for this study. Here, the number of participants analyzed refer to the participants evaluable for this outcome measure.

Percent change from baseline in EN volume at EOT (up to Week 24) based on dairy data was reported. EOT was defined as the last available measurement after the date of first dose during the 24-week treatment period.

Outcome measures

Outcome measures
Measure	Total Children (Aged: 1 to 15 Years) n=4 Participants Participants aged from 1 through 15 years received teduglutide 0.05 milligram per kilogram per day (mg/kg/day) subcutaneous (SC) injection once daily for 24 weeks and completed the study at Week 28.	Infants (Corrected Gestational Age: 4 to < 12 Months) n=2 Participants Participants (Infants) from 4 through \< 12 months of corrected gestational age received teduglutide 0.05 mg/kg/day SC injection once daily for 24 weeks and completed the study at Week 28.
Percent Change From Baseline in Enteral Nutritional (EN) Volume at End of Treatment (EOT) Based on Dairy Data	53.1 Percent change Standard Deviation 111.01	-23.2 Percent change Standard Deviation 36.89

PRIMARY outcome

Timeframe: Baseline, EOT (up to Week 24)

Population: ITT population consisted of all participants who were enrolled into the study and met all eligible criteria for this study.

Absolute change from baseline in EN caloric intake at EOT (up to Week 24) based on dairy data was reported. EOT was defined as the last available measurement after the date of first dose during the 24-week treatment period.

Outcome measures

Outcome measures
Measure	Total Children (Aged: 1 to 15 Years) n=8 Participants Participants aged from 1 through 15 years received teduglutide 0.05 milligram per kilogram per day (mg/kg/day) subcutaneous (SC) injection once daily for 24 weeks and completed the study at Week 28.	Infants (Corrected Gestational Age: 4 to < 12 Months) n=2 Participants Participants (Infants) from 4 through \< 12 months of corrected gestational age received teduglutide 0.05 mg/kg/day SC injection once daily for 24 weeks and completed the study at Week 28.
Absolute Change From Baseline in Enteral Nutritional (EN) Caloric Intake at End of Treatment (EOT) Based on Dairy Data	0.8 kcal/kg/day Standard Deviation 5.60	0.3 kcal/kg/day Standard Deviation 1.03

PRIMARY outcome

Timeframe: Baseline, EOT (up to Week 24)

Population: ITT population consisted of all participants who were enrolled into the study and met all eligible criteria for this study. Here, the number of participants analyzed refer to the participants evaluable for this outcome measure.

Percent change from baseline in EN caloric intake at EOT (up to Week 24) based on dairy data was reported. EOT was defined as the last available measurement after the date of first dose during the 24-week treatment period.

Outcome measures

Outcome measures
Measure	Total Children (Aged: 1 to 15 Years) n=4 Participants Participants aged from 1 through 15 years received teduglutide 0.05 milligram per kilogram per day (mg/kg/day) subcutaneous (SC) injection once daily for 24 weeks and completed the study at Week 28.	Infants (Corrected Gestational Age: 4 to < 12 Months) n=2 Participants Participants (Infants) from 4 through \< 12 months of corrected gestational age received teduglutide 0.05 mg/kg/day SC injection once daily for 24 weeks and completed the study at Week 28.
Percent Change From Baseline in Enteral Nutritional (EN) Caloric Intake at End of Treatment (EOT) Based on Dairy Data	56.8 Percent change Standard Deviation 117.88	-24.2 Percent change Standard Deviation 38.06

PRIMARY outcome

Timeframe: Week 24

Population: ITT population consisted of all participants who were enrolled into the study and met all eligible criteria for this study. Here, the number of participants analyzed refer to the participants evaluable for this outcome measure.

Number of participants who achieved at least 20% reduction in PS volume at Week 24 was reported.

Outcome measures

Outcome measures
Measure	Total Children (Aged: 1 to 15 Years) n=7 Participants Participants aged from 1 through 15 years received teduglutide 0.05 milligram per kilogram per day (mg/kg/day) subcutaneous (SC) injection once daily for 24 weeks and completed the study at Week 28.	Infants (Corrected Gestational Age: 4 to < 12 Months) n=2 Participants Participants (Infants) from 4 through \< 12 months of corrected gestational age received teduglutide 0.05 mg/kg/day SC injection once daily for 24 weeks and completed the study at Week 28.
Number of Participants Who Achieved At Least 20 Percent (%) Reduction in Parenteral Support (PS) Volume at Week 24	4 Participants	1 Participants

PRIMARY outcome

Timeframe: EOT (up to Week 24)

Population: ITT population consisted of all participants who were enrolled into the study and met all eligible criteria for this study.

Number of participants who achieved at least 20% reduction in PS volume at EOT (up to Week 24) was reported. EOT was defined as the last available measurement after the date of first dose during the 24-week treatment period.

Outcome measures

Outcome measures
Measure	Total Children (Aged: 1 to 15 Years) n=8 Participants Participants aged from 1 through 15 years received teduglutide 0.05 milligram per kilogram per day (mg/kg/day) subcutaneous (SC) injection once daily for 24 weeks and completed the study at Week 28.	Infants (Corrected Gestational Age: 4 to < 12 Months) n=2 Participants Participants (Infants) from 4 through \< 12 months of corrected gestational age received teduglutide 0.05 mg/kg/day SC injection once daily for 24 weeks and completed the study at Week 28.
Number of Participants Who Achieved At Least 20 Percent (%) Reduction in Parenteral Support (PS) Volume at End of Treatment (EOT)	4 Participants	1 Participants

PRIMARY outcome

Timeframe: EOT (up to Week 24)

Population: ITT population consisted of all participants who were enrolled into the study and met all eligible criteria for this study.

Number of participants who achieved at least 100% reduction in complete weaning of PS volume at EOT (up to Week 24) was reported. EOT was defined as the last available measurement after the date of first dose during the 24-week treatment period.

Outcome measures

Outcome measures
Measure	Total Children (Aged: 1 to 15 Years) n=8 Participants Participants aged from 1 through 15 years received teduglutide 0.05 milligram per kilogram per day (mg/kg/day) subcutaneous (SC) injection once daily for 24 weeks and completed the study at Week 28.	Infants (Corrected Gestational Age: 4 to < 12 Months) n=2 Participants Participants (Infants) from 4 through \< 12 months of corrected gestational age received teduglutide 0.05 mg/kg/day SC injection once daily for 24 weeks and completed the study at Week 28.
Number of Participants Who Achieved 100 Percent (%) Reduction in Complete Weaning of Parenteral Support (PS) Volume at End of Treatment (EOT)	1 Participants	0 Participants

PRIMARY outcome

Timeframe: Week 28

Population: ITT population consisted of all participants who were enrolled into the study and met all eligible criteria for this study.

Number of participants who achieved \>= 20% reduction in PS volume at Week 28 was reported.

Outcome measures

Outcome measures
Measure	Total Children (Aged: 1 to 15 Years) n=8 Participants Participants aged from 1 through 15 years received teduglutide 0.05 milligram per kilogram per day (mg/kg/day) subcutaneous (SC) injection once daily for 24 weeks and completed the study at Week 28.	Infants (Corrected Gestational Age: 4 to < 12 Months) n=2 Participants Participants (Infants) from 4 through \< 12 months of corrected gestational age received teduglutide 0.05 mg/kg/day SC injection once daily for 24 weeks and completed the study at Week 28.
Number of Participants Who Achieved Greater Than or Equal to (>=) 20 Percent (%) Reduction in Parenteral Support (PS) Volume at Week 28	3 Participants	1 Participants

PRIMARY outcome

Timeframe: EOT (up to Week 24), EOS (up to Week 28)

Population: ITT population consisted of all participants who were enrolled into the study and met all eligible criteria for this study.

Absolute change from EOT (up to Week 24) in PS volume at EOS (up to Week 28) based on dairy data was reported. EOT was defined as the last available measurement after the date of first dose during the 24-week treatment period.

Outcome measures

Outcome measures
Measure	Total Children (Aged: 1 to 15 Years) n=8 Participants Participants aged from 1 through 15 years received teduglutide 0.05 milligram per kilogram per day (mg/kg/day) subcutaneous (SC) injection once daily for 24 weeks and completed the study at Week 28.	Infants (Corrected Gestational Age: 4 to < 12 Months) n=2 Participants Participants (Infants) from 4 through \< 12 months of corrected gestational age received teduglutide 0.05 mg/kg/day SC injection once daily for 24 weeks and completed the study at Week 28.
Absolute Change From End of Treatment (EOT) in Parenteral Support (PS) Volume at End of Study (EOS) Based on Dairy Data	3.9 mL/kg/day Standard Deviation 7.06	-3.8 mL/kg/day Standard Deviation 0.67

PRIMARY outcome

Timeframe: EOT (up to Week 24), EOS (up to Week 28)

Population: ITT population consisted of all participants who were enrolled into the study and met all eligible criteria for this study. Here, the number of participants analyzed refer to the participants evaluable for this outcome measure.

Percent change from EOT (up to Week 24) in PS volume at EOS (up to Week 28) based on dairy data was reported. EOT was defined as the last available measurement after the date of first dose during the 24-week treatment period.

Outcome measures

Outcome measures
Measure	Total Children (Aged: 1 to 15 Years) n=7 Participants Participants aged from 1 through 15 years received teduglutide 0.05 milligram per kilogram per day (mg/kg/day) subcutaneous (SC) injection once daily for 24 weeks and completed the study at Week 28.	Infants (Corrected Gestational Age: 4 to < 12 Months) n=2 Participants Participants (Infants) from 4 through \< 12 months of corrected gestational age received teduglutide 0.05 mg/kg/day SC injection once daily for 24 weeks and completed the study at Week 28.
Percent Change From End of Treatment (EOT) in Parenteral Support (PS) Volume at End of Study (EOS) Based on Dairy Data	9.8 Percent change Standard Deviation 19.59	-5.5 Percent change Standard Deviation 2.35

PRIMARY outcome

Timeframe: EOT (up to Week 24), EOS (up to Week 28)

Population: ITT population consisted of all participants who were enrolled into the study and met all eligible criteria for this study.

Absolute change from EOT (up to Week 24) in PS caloric intake at EOS (up to Week 28) based on dairy data was reported. EOT was defined as the last available measurement after the date of first dose during the 24-week treatment period.

Outcome measures

Outcome measures
Measure	Total Children (Aged: 1 to 15 Years) n=8 Participants Participants aged from 1 through 15 years received teduglutide 0.05 milligram per kilogram per day (mg/kg/day) subcutaneous (SC) injection once daily for 24 weeks and completed the study at Week 28.	Infants (Corrected Gestational Age: 4 to < 12 Months) n=2 Participants Participants (Infants) from 4 through \< 12 months of corrected gestational age received teduglutide 0.05 mg/kg/day SC injection once daily for 24 weeks and completed the study at Week 28.
Absolute Change From End of Treatment (EOT) in Parenteral Support (PS) Caloric Intake at End of Study (EOS) Based on Dairy Data	0.5 kcal/kg/day Standard Deviation 6.16	-4.0 kcal/kg/day Standard Deviation 1.38

PRIMARY outcome

Timeframe: EOT (up to Week 24), EOS (up to Week 28)

Population: ITT population consisted of all participants who were enrolled into the study and met all eligible criteria for this study. Here, the number of participants analyzed refer to the participants evaluable for this outcome measure.

Percent change from EOT (up to Week 24) in PS caloric intake at EOS (up to Week 28) based on dairy data was reported. EOT was defined as the last available measurement after the date of first dose during the 24-week treatment period.

Outcome measures

Outcome measures
Measure	Total Children (Aged: 1 to 15 Years) n=7 Participants Participants aged from 1 through 15 years received teduglutide 0.05 milligram per kilogram per day (mg/kg/day) subcutaneous (SC) injection once daily for 24 weeks and completed the study at Week 28.	Infants (Corrected Gestational Age: 4 to < 12 Months) n=2 Participants Participants (Infants) from 4 through \< 12 months of corrected gestational age received teduglutide 0.05 mg/kg/day SC injection once daily for 24 weeks and completed the study at Week 28.
Percent Change From End of Treatment (EOT) in Parenteral Support (PS) Caloric Intake at End of Study (EOS) Based on Dairy Data	3.5 Percent change Standard Deviation 19.17	-10.3 Percent change Standard Deviation 4.40

PRIMARY outcome

Timeframe: EOT (up to Week 24), EOS (up to Week 28)

Population: ITT population consisted of all participants who were enrolled into the study and met all eligible criteria for this study. Data for this outcome measure was not planned to be collected and analyzed for infants.

Absolute change from EOT (up to Week 24) in plasma citrulline at EOS (up to Week 28) was reported. EOT was defined as the last available measurement after the date of first dose during the 24-week treatment period.

Outcome measures

Outcome measures
Measure	Total Children (Aged: 1 to 15 Years) n=8 Participants Participants aged from 1 through 15 years received teduglutide 0.05 milligram per kilogram per day (mg/kg/day) subcutaneous (SC) injection once daily for 24 weeks and completed the study at Week 28.	Infants (Corrected Gestational Age: 4 to < 12 Months) Participants (Infants) from 4 through \< 12 months of corrected gestational age received teduglutide 0.05 mg/kg/day SC injection once daily for 24 weeks and completed the study at Week 28.
Absolute Change From End of Treatment (EOT) in Plasma Citrulline at End of Study (EOS)	-2.9 Micromoles (mcM) Standard Deviation 2.87	—

PRIMARY outcome

Timeframe: EOT (up to Week 24), EOS (up to Week 28)

Population: ITT population consisted of all participants who were enrolled into the study and met all eligible criteria for this study. Data for this outcome measure was not planned to be collected and analyzed for infants.

Percent change from EOT (up to Week 24) in plasma citrulline at EOS (up to Week 28) was reported. EOT was defined as the last available measurement after the date of first dose during the 24-week treatment period.

Outcome measures

Outcome measures
Measure	Total Children (Aged: 1 to 15 Years) n=8 Participants Participants aged from 1 through 15 years received teduglutide 0.05 milligram per kilogram per day (mg/kg/day) subcutaneous (SC) injection once daily for 24 weeks and completed the study at Week 28.	Infants (Corrected Gestational Age: 4 to < 12 Months) Participants (Infants) from 4 through \< 12 months of corrected gestational age received teduglutide 0.05 mg/kg/day SC injection once daily for 24 weeks and completed the study at Week 28.
Percent Change From End of Treatment (EOT) in Plasma Citrulline at End of Study (EOS)	-20.0 Percent change Standard Deviation 24.16	—

PRIMARY outcome

Timeframe: EOT (up to Week 24), EOS (up to Week 28)

Population: ITT population consisted of all participants who were enrolled into the study and met all eligible criteria for this study.

Absolute change from EOT (up to Week 24) in EN volume at EOS (up to Week 28) based on dairy data was reported. EOT was defined as the last available measurement after the date of first dose during the 24-week treatment period.

Outcome measures

Outcome measures
Measure	Total Children (Aged: 1 to 15 Years) n=8 Participants Participants aged from 1 through 15 years received teduglutide 0.05 milligram per kilogram per day (mg/kg/day) subcutaneous (SC) injection once daily for 24 weeks and completed the study at Week 28.	Infants (Corrected Gestational Age: 4 to < 12 Months) n=2 Participants Participants (Infants) from 4 through \< 12 months of corrected gestational age received teduglutide 0.05 mg/kg/day SC injection once daily for 24 weeks and completed the study at Week 28.
Absolute Change From End of Treatment (EOT) in Enteral Nutritional (EN) Volume at End of Study (EOS) Based on Dairy Data	-1.5 mL/kg/day Standard Deviation 3.24	-2.8 mL/kg/day Standard Deviation 3.98

PRIMARY outcome

Timeframe: EOT (up to Week 24), EOS (up to Week 28)

Population: ITT population consisted of all participants who were enrolled into the study and met all eligible criteria for this study. Here, the number of participants analyzed refer to the participants evaluable for this outcome measure.

Percent change from EOT (up to Week 24) in EN volume at EOS (up to Week 28) based on dairy data was reported. EOT was defined as the last available measurement after the date of first dose during the 24-week treatment period.

Outcome measures

Outcome measures
Measure	Total Children (Aged: 1 to 15 Years) n=4 Participants Participants aged from 1 through 15 years received teduglutide 0.05 milligram per kilogram per day (mg/kg/day) subcutaneous (SC) injection once daily for 24 weeks and completed the study at Week 28.	Infants (Corrected Gestational Age: 4 to < 12 Months) n=2 Participants Participants (Infants) from 4 through \< 12 months of corrected gestational age received teduglutide 0.05 mg/kg/day SC injection once daily for 24 weeks and completed the study at Week 28.
Percent Change From End of Treatment (EOT) in Enteral Nutritional (EN) Volume at End of Study (EOS) Based on Dairy Data	-22.1 Percent change Standard Deviation 28.06	-1.4 Percent change Standard Deviation 8.25

PRIMARY outcome

Timeframe: EOT (up to Week 24), EOS (up to Week 28)

Population: ITT population consisted of all participants who were enrolled into the study and met all eligible criteria for this study.

Absolute change from EOT (up to Week 24) in EN caloric intake at EOS (up to Week 28) based on dairy data was reported. EOT was defined as the last available measurement after the date of first dose during the 24-week treatment period.

Outcome measures

Outcome measures
Measure	Total Children (Aged: 1 to 15 Years) n=8 Participants Participants aged from 1 through 15 years received teduglutide 0.05 milligram per kilogram per day (mg/kg/day) subcutaneous (SC) injection once daily for 24 weeks and completed the study at Week 28.	Infants (Corrected Gestational Age: 4 to < 12 Months) n=2 Participants Participants (Infants) from 4 through \< 12 months of corrected gestational age received teduglutide 0.05 mg/kg/day SC injection once daily for 24 weeks and completed the study at Week 28.
Absolute Change From End of Treatment (EOT) in Enteral Nutritional (EN) Caloric Intake at End of Study (EOS) Based on Dairy Data	-1.1 kcal/kg/day Standard Deviation 2.64	-1.4 kcal/kg/day Standard Deviation 1.97

PRIMARY outcome

Timeframe: EOT (up to Week 24), EOS (up to Week 28)

Population: ITT population consisted of all participants who were enrolled into the study and met all eligible criteria for this study. Here, the number of participants analyzed refer to the participants evaluable for this outcome measure.

Percent change from EOT (up to Week 24) in EN caloric intake at EOS (up to Week 28) based on dairy data was reported. EOT was defined as the last available measurement after the date of first dose during the 24-week treatment period.

Outcome measures

Outcome measures
Measure	Total Children (Aged: 1 to 15 Years) n=4 Participants Participants aged from 1 through 15 years received teduglutide 0.05 milligram per kilogram per day (mg/kg/day) subcutaneous (SC) injection once daily for 24 weeks and completed the study at Week 28.	Infants (Corrected Gestational Age: 4 to < 12 Months) n=2 Participants Participants (Infants) from 4 through \< 12 months of corrected gestational age received teduglutide 0.05 mg/kg/day SC injection once daily for 24 weeks and completed the study at Week 28.
Percent Change From End of Treatment (EOT) in Enteral Nutritional (EN) Caloric Intake at End of Study (EOS) Based on Dairy Data	-22.1 Percent change Standard Deviation 28.16	-3.0 Percent change Standard Deviation 6.03

PRIMARY outcome

Timeframe: Baseline, EOT (up to Week 24)

Population: ITT population consisted of all participants who were enrolled into the study and met all eligible criteria for this study.

Absolute change from baseline in number of hours per day of PS Usage at EOT (up to Week 24) based on dairy data was reported. EOT was defined as the last available measurement after the date of first dose during the 24-week treatment period.

Outcome measures

Outcome measures
Measure	Total Children (Aged: 1 to 15 Years) n=8 Participants Participants aged from 1 through 15 years received teduglutide 0.05 milligram per kilogram per day (mg/kg/day) subcutaneous (SC) injection once daily for 24 weeks and completed the study at Week 28.	Infants (Corrected Gestational Age: 4 to < 12 Months) n=2 Participants Participants (Infants) from 4 through \< 12 months of corrected gestational age received teduglutide 0.05 mg/kg/day SC injection once daily for 24 weeks and completed the study at Week 28.
Absolute Change From Baseline in Number of Hours Per Day of Parenteral Support (PS) Usage at End of Treatment (EOT) Based on Dairy Data	-2.1 Hours per day (hours/day) Standard Deviation 4.16	0.0 Hours per day (hours/day) Standard Deviation 0.00

PRIMARY outcome

Timeframe: Baseline, EOT (up to Week 24)

Population: ITT population consisted of all participants who were enrolled into the study and met all eligible criteria for this study.

Absolute change from baseline in number of days per Week of PS usage at EOT (up to Week 24) based on dairy data was reported. EOT was defined as the last available measurement after the date of first dose during the 24-week treatment period.

Outcome measures

Outcome measures
Measure	Total Children (Aged: 1 to 15 Years) n=8 Participants Participants aged from 1 through 15 years received teduglutide 0.05 milligram per kilogram per day (mg/kg/day) subcutaneous (SC) injection once daily for 24 weeks and completed the study at Week 28.	Infants (Corrected Gestational Age: 4 to < 12 Months) n=2 Participants Participants (Infants) from 4 through \< 12 months of corrected gestational age received teduglutide 0.05 mg/kg/day SC injection once daily for 24 weeks and completed the study at Week 28.
Absolute Change From Baseline in Number of Days Per Week of Parenteral Support (PS) Usage at End of Treatment (EOT) Based on Dairy Data	-0.9 Days per week Standard Deviation 2.47	0.0 Days per week Standard Deviation 0.00

PRIMARY outcome

Timeframe: From start of study drug administration up to EOS (up to Week 28)

Population: Safety population consisted of all participants in the ITT population who received at least 1 administration of investigational product with any safety follow-up.

An Adverse Event (AE) was any untoward medical occurrence in a clinical investigation participant administered a pharmaceutical product and that does not necessarily have a causal relationship with this treatment. TEAEs were defined as any AEs whose onset occurred, severity worsened, or intensity increased after receiving the investigational product.

Outcome measures

Outcome measures
Measure	Total Children (Aged: 1 to 15 Years) n=8 Participants Participants aged from 1 through 15 years received teduglutide 0.05 milligram per kilogram per day (mg/kg/day) subcutaneous (SC) injection once daily for 24 weeks and completed the study at Week 28.	Infants (Corrected Gestational Age: 4 to < 12 Months) n=2 Participants Participants (Infants) from 4 through \< 12 months of corrected gestational age received teduglutide 0.05 mg/kg/day SC injection once daily for 24 weeks and completed the study at Week 28.
Number of Participants With Treatment-emergent Adverse Events (TEAEs)	8 Participants	2 Participants

PRIMARY outcome

Timeframe: Baseline, Week 28

Population: Safety population consisted of all participants in the ITT population who received at least 1 administration of investigational product with any safety follow-up.

Body weight was measured using age Z-score. A Z-score was defined as the deviation of the value for an individual from the mean value of the reference population divided by the standard deviation for the reference population. A negative Z-score indicates values lower than the mean while a positive Z-score indicates values higher than the mean. Change from baseline in body weight for age Z-score at Week 28 was reported.

Outcome measures

Outcome measures
Measure	Total Children (Aged: 1 to 15 Years) n=8 Participants Participants aged from 1 through 15 years received teduglutide 0.05 milligram per kilogram per day (mg/kg/day) subcutaneous (SC) injection once daily for 24 weeks and completed the study at Week 28.	Infants (Corrected Gestational Age: 4 to < 12 Months) n=2 Participants Participants (Infants) from 4 through \< 12 months of corrected gestational age received teduglutide 0.05 mg/kg/day SC injection once daily for 24 weeks and completed the study at Week 28.
Change From Baseline in Body Weight for Age Z-score at Week 28	0.177 Z-score Standard Deviation 0.3625	2.332 Z-score Standard Deviation 1.1503

PRIMARY outcome

Timeframe: Baseline, Week 28

Population: Safety population consisted of all participants in the ITT population who received at least 1 administration of investigational product with any safety follow-up.

Height was measured using age Z-score. A Z-score was defined as the deviation of the value for an individual from the mean value of the reference population divided by the standard deviation for the reference population. A negative Z-score indicates values lower than the mean while a positive Z-score indicates values higher than the mean. Change from baseline in height for age Z-score at Week 28 was reported.

Outcome measures

Outcome measures
Measure	Total Children (Aged: 1 to 15 Years) n=8 Participants Participants aged from 1 through 15 years received teduglutide 0.05 milligram per kilogram per day (mg/kg/day) subcutaneous (SC) injection once daily for 24 weeks and completed the study at Week 28.	Infants (Corrected Gestational Age: 4 to < 12 Months) n=2 Participants Participants (Infants) from 4 through \< 12 months of corrected gestational age received teduglutide 0.05 mg/kg/day SC injection once daily for 24 weeks and completed the study at Week 28.
Change From Baseline in Height for Age Z-score at Week 28	0.024 Z-score Standard Deviation 0.4841	1.056 Z-score Standard Deviation 0.6968

PRIMARY outcome

Timeframe: Baseline, Week 28

Population: Safety population consisted of all participants in the ITT population who received at least 1 administration of investigational product with any safety follow-up. Data for this outcome measure was not planned to be collected and analyzed for Total Children (Aged: 1 to 15 Years).

Head circumference was measured using age Z-score. A Z-score was defined as the deviation of the value for an individual from the mean value of the reference population divided by the standard deviation for the reference population. A negative Z-score indicates values lower than the mean while a positive Z-score indicates values higher than the mean. Change from baseline in head circumference for age Z-score at Week 28 was reported.

Outcome measures

Outcome measures
Measure	Total Children (Aged: 1 to 15 Years) Participants aged from 1 through 15 years received teduglutide 0.05 milligram per kilogram per day (mg/kg/day) subcutaneous (SC) injection once daily for 24 weeks and completed the study at Week 28.	Infants (Corrected Gestational Age: 4 to < 12 Months) n=2 Participants Participants (Infants) from 4 through \< 12 months of corrected gestational age received teduglutide 0.05 mg/kg/day SC injection once daily for 24 weeks and completed the study at Week 28.
Change From Baseline in Head Circumference for Age Z-score at Week 28	—	1.559 Z-score Standard Deviation 0.6116

PRIMARY outcome

Timeframe: From start of study drug administration up to EOS (up to Week 28)

Population: Safety population consisted of all participants in the ITT population who received at least 1 administration of investigational product with any safety follow-up.

Vital sign assessments included pulse rate, blood pressure, or body temperature. Number of participants with clinically significant changes in vital signs by the investigator were recorded as TEAEs.

Outcome measures

Outcome measures
Measure	Total Children (Aged: 1 to 15 Years) n=8 Participants Participants aged from 1 through 15 years received teduglutide 0.05 milligram per kilogram per day (mg/kg/day) subcutaneous (SC) injection once daily for 24 weeks and completed the study at Week 28.	Infants (Corrected Gestational Age: 4 to < 12 Months) n=2 Participants Participants (Infants) from 4 through \< 12 months of corrected gestational age received teduglutide 0.05 mg/kg/day SC injection once daily for 24 weeks and completed the study at Week 28.
Number of Participants With Clinically Significant Changes in Vital Signs Reported as Treatment Emergent Adverse Events (TEAEs)	0 Participants	0 Participants

PRIMARY outcome

Timeframe: From start of study drug administration up to EOS (up to Week 28)

Population: Safety population consisted of all participants in the ITT population who received at least 1 administration of investigational product with any safety follow-up.

12-lead ECG was performed. Any change in ECG assessments which were deemed to be clinically significant changes were recorded as TEAEs.

Outcome measures

Outcome measures
Measure	Total Children (Aged: 1 to 15 Years) n=8 Participants Participants aged from 1 through 15 years received teduglutide 0.05 milligram per kilogram per day (mg/kg/day) subcutaneous (SC) injection once daily for 24 weeks and completed the study at Week 28.	Infants (Corrected Gestational Age: 4 to < 12 Months) n=2 Participants Participants (Infants) from 4 through \< 12 months of corrected gestational age received teduglutide 0.05 mg/kg/day SC injection once daily for 24 weeks and completed the study at Week 28.
Number of Participants With Clinically Significant Changes in Electrocardiogram (ECG) Reported as Treatment Emergent Adverse Events (TEAEs)	0 Participants	0 Participants

PRIMARY outcome

Timeframe: From start of study drug administration up to EOS (up to Week 28)

Population: Safety population consisted of all participants in the ITT population who received at least 1 administration of investigational product with any safety follow-up.

Clinical laboratory assessments included biochemistry, hematology, coagulation, urinalysis. The number of participants with clinically significant laboratory abnormalities were reported as TEAEs.

Outcome measures

Outcome measures
Measure	Total Children (Aged: 1 to 15 Years) n=8 Participants Participants aged from 1 through 15 years received teduglutide 0.05 milligram per kilogram per day (mg/kg/day) subcutaneous (SC) injection once daily for 24 weeks and completed the study at Week 28.	Infants (Corrected Gestational Age: 4 to < 12 Months) n=2 Participants Participants (Infants) from 4 through \< 12 months of corrected gestational age received teduglutide 0.05 mg/kg/day SC injection once daily for 24 weeks and completed the study at Week 28.
Number of Participants With Clinically Significant Laboratory Abnormalities Reported as Treatment Emergent Adverse Events (TEAEs)	0 Participants	0 Participants

PRIMARY outcome

Timeframe: Baseline, Week 28

Population: Safety population consisted of all participants in the ITT population who received at least 1 administration of investigational product with any safety follow-up. Here, the number of participants analyzed refer to the participants evaluable for this outcome measure.

Average urine output was recorded in measured volume at Week 28 was recorded.

Outcome measures

Outcome measures
Measure	Total Children (Aged: 1 to 15 Years) n=7 Participants Participants aged from 1 through 15 years received teduglutide 0.05 milligram per kilogram per day (mg/kg/day) subcutaneous (SC) injection once daily for 24 weeks and completed the study at Week 28.	Infants (Corrected Gestational Age: 4 to < 12 Months) n=1 Participants Participants (Infants) from 4 through \< 12 months of corrected gestational age received teduglutide 0.05 mg/kg/day SC injection once daily for 24 weeks and completed the study at Week 28.
Change From Baseline in the Average Urine Output at Week 28	-22.317 mL/kg/day Standard Deviation 34.8252	-48.879 mL/kg/day Standard Deviation NA Standard deviation was not estimated as only single participant was analyzed.

PRIMARY outcome

Timeframe: Baseline, Week 28

Population: Safety population consisted of all participants in the ITT population who received at least 1 administration of investigational product with any safety follow-up. Here, the number of participants analyzed refer to the participants evaluable for this outcome measure.

Change from baseline in the fecal output (Average number of stools per day) at Week 28 was recorded.

Outcome measures

Outcome measures
Measure	Total Children (Aged: 1 to 15 Years) n=7 Participants Participants aged from 1 through 15 years received teduglutide 0.05 milligram per kilogram per day (mg/kg/day) subcutaneous (SC) injection once daily for 24 weeks and completed the study at Week 28.	Infants (Corrected Gestational Age: 4 to < 12 Months) n=1 Participants Participants (Infants) from 4 through \< 12 months of corrected gestational age received teduglutide 0.05 mg/kg/day SC injection once daily for 24 weeks and completed the study at Week 28.
Change From Baseline in the Fecal Output at Week 28	-0.64 Average number of stools per day Standard Deviation 1.547	2.50 Average number of stools per day Standard Deviation NA Standard deviation was not estimated as only single participant was analyzed.

PRIMARY outcome

Timeframe: From start of study drug administration up to EOS (up to Week 28)

Population: Safety population consisted of all participants in the ITT population who received at least 1 administration of investigational product with any safety follow-up.

Number of participants with positive specific antibodies to teduglutide were used to summarize the presence of antibodies.

Outcome measures

Outcome measures
Measure	Total Children (Aged: 1 to 15 Years) n=8 Participants Participants aged from 1 through 15 years received teduglutide 0.05 milligram per kilogram per day (mg/kg/day) subcutaneous (SC) injection once daily for 24 weeks and completed the study at Week 28.	Infants (Corrected Gestational Age: 4 to < 12 Months) n=2 Participants Participants (Infants) from 4 through \< 12 months of corrected gestational age received teduglutide 0.05 mg/kg/day SC injection once daily for 24 weeks and completed the study at Week 28.
Number of Participants With Positive Specific Antibodies to Teduglutide	1 Participants	2 Participants

PRIMARY outcome

Timeframe: Baseline, EOT (up to Week 24)

Population: Safety population consisted of all participants in the ITT population who received at least 1 administration of investigational product with any safety follow-up. Data for this outcome was not planned to be collected and analyzed for infants.

GI specific testing included colonoscopy or sigmoidoscopy, abdominal ultrasound, fecal occult blood testing, upper GI series with small bowel follow-through (UGI/SBFT). EOT was defined as the last available measurement after the date of first dose during the 24-week treatment period. Number of participants with clinically significant abnormal findings in gastrointestinal specific testing were reported.

Outcome measures

Outcome measures
Measure	Total Children (Aged: 1 to 15 Years) n=8 Participants Participants aged from 1 through 15 years received teduglutide 0.05 milligram per kilogram per day (mg/kg/day) subcutaneous (SC) injection once daily for 24 weeks and completed the study at Week 28.	Infants (Corrected Gestational Age: 4 to < 12 Months) Participants (Infants) from 4 through \< 12 months of corrected gestational age received teduglutide 0.05 mg/kg/day SC injection once daily for 24 weeks and completed the study at Week 28.
Number of Participants With Clinically Significant Abnormal Findings in Gastrointestinal (GI) Specific Testing	0 Participants	—

Adverse Events

Total Children (Aged: 1 - 15 Years)

Serious events: 6 serious events

Other events: 8 other events

Deaths: 0 deaths

Infants (Corrected Gestational Age: 4 to < 12 Months)

Serious events: 2 serious events

Other events: 1 other events

Deaths: 0 deaths

Serious adverse events

Serious adverse events
Measure	Total Children (Aged: 1 - 15 Years) n=8 participants at risk Participants aged from 1 through 15 years received teduglutide 0.05 mg/kg/day SC injection once daily for 24 weeks and completed the study at Week 28.	Infants (Corrected Gestational Age: 4 to < 12 Months) n=2 participants at risk Participants (Infants) from 4 through \< 12 months of corrected gestational age received teduglutide 0.05 mg/kg/day SC injection once daily for 24 weeks and completed the study at Week 28.
Gastrointestinal disorders Abdominal pain	12.5% 1/8 • Number of events 1 • From start of study drug administration up to end of the study (up to Week 28)	0.00% 0/2 • From start of study drug administration up to end of the study (up to Week 28)
Gastrointestinal disorders Enterocolitis	25.0% 2/8 • Number of events 2 • From start of study drug administration up to end of the study (up to Week 28)	0.00% 0/2 • From start of study drug administration up to end of the study (up to Week 28)
General disorders Pyrexia	0.00% 0/8 • From start of study drug administration up to end of the study (up to Week 28)	100.0% 2/2 • Number of events 4 • From start of study drug administration up to end of the study (up to Week 28)
Immune system disorders Anaphylactic reaction	12.5% 1/8 • Number of events 1 • From start of study drug administration up to end of the study (up to Week 28)	0.00% 0/2 • From start of study drug administration up to end of the study (up to Week 28)
Infections and infestations Adenovirus infection	12.5% 1/8 • Number of events 1 • From start of study drug administration up to end of the study (up to Week 28)	0.00% 0/2 • From start of study drug administration up to end of the study (up to Week 28)
Infections and infestations Device related infection	37.5% 3/8 • Number of events 4 • From start of study drug administration up to end of the study (up to Week 28)	50.0% 1/2 • Number of events 2 • From start of study drug administration up to end of the study (up to Week 28)
Infections and infestations Gastroenteritis	12.5% 1/8 • Number of events 1 • From start of study drug administration up to end of the study (up to Week 28)	0.00% 0/2 • From start of study drug administration up to end of the study (up to Week 28)
Infections and infestations Hand-foot-and-mouth disease	12.5% 1/8 • Number of events 1 • From start of study drug administration up to end of the study (up to Week 28)	0.00% 0/2 • From start of study drug administration up to end of the study (up to Week 28)
Infections and infestations Medical device site infection	25.0% 2/8 • Number of events 3 • From start of study drug administration up to end of the study (up to Week 28)	0.00% 0/2 • From start of study drug administration up to end of the study (up to Week 28)
Infections and infestations Upper respiratory tract infection	0.00% 0/8 • From start of study drug administration up to end of the study (up to Week 28)	50.0% 1/2 • Number of events 2 • From start of study drug administration up to end of the study (up to Week 28)
Product Issues Device damage	12.5% 1/8 • Number of events 1 • From start of study drug administration up to end of the study (up to Week 28)	0.00% 0/2 • From start of study drug administration up to end of the study (up to Week 28)
Respiratory, thoracic and mediastinal disorders Upper respiratory tract inflammation	0.00% 0/8 • From start of study drug administration up to end of the study (up to Week 28)	50.0% 1/2 • Number of events 1 • From start of study drug administration up to end of the study (up to Week 28)

Other adverse events

Additional Information

Study Director

Shire

Phone: +1 866 842 5335

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee If a multicenter publication is not submitted within twelve (12) months after conclusion, abandonment or 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.
Publication restrictions are in place

Restriction type: OTHER

Measure	Total Children (Aged: 1 - 15 Years) n=8 participants at risk Participants aged from 1 through 15 years received teduglutide 0.05 mg/kg/day SC injection once daily for 24 weeks and completed the study at Week 28.	Infants (Corrected Gestational Age: 4 to < 12 Months) n=2 participants at risk Participants (Infants) from 4 through \< 12 months of corrected gestational age received teduglutide 0.05 mg/kg/day SC injection once daily for 24 weeks and completed the study at Week 28.
Gastrointestinal disorders Dental caries	12.5% 1/8 • Number of events 1 • From start of study drug administration up to end of the study (up to Week 28)	0.00% 0/2 • From start of study drug administration up to end of the study (up to Week 28)
Gastrointestinal disorders Enteritis	12.5% 1/8 • Number of events 1 • From start of study drug administration up to end of the study (up to Week 28)	0.00% 0/2 • From start of study drug administration up to end of the study (up to Week 28)
Gastrointestinal disorders Enterocolitis	0.00% 0/8 • From start of study drug administration up to end of the study (up to Week 28)	50.0% 1/2 • Number of events 1 • From start of study drug administration up to end of the study (up to Week 28)
Gastrointestinal disorders Gastritis	12.5% 1/8 • Number of events 1 • From start of study drug administration up to end of the study (up to Week 28)	0.00% 0/2 • From start of study drug administration up to end of the study (up to Week 28)
Gastrointestinal disorders Abdominal distension	12.5% 1/8 • Number of events 1 • From start of study drug administration up to end of the study (up to Week 28)	0.00% 0/2 • From start of study drug administration up to end of the study (up to Week 28)
Gastrointestinal disorders Abdominal pain	12.5% 1/8 • Number of events 1 • From start of study drug administration up to end of the study (up to Week 28)	0.00% 0/2 • From start of study drug administration up to end of the study (up to Week 28)
Gastrointestinal disorders Constipation	25.0% 2/8 • Number of events 2 • From start of study drug administration up to end of the study (up to Week 28)	0.00% 0/2 • From start of study drug administration up to end of the study (up to Week 28)
Gastrointestinal disorders Proctalgia	12.5% 1/8 • Number of events 1 • From start of study drug administration up to end of the study (up to Week 28)	0.00% 0/2 • From start of study drug administration up to end of the study (up to Week 28)
General disorders Catheter site granuloma	12.5% 1/8 • Number of events 1 • From start of study drug administration up to end of the study (up to Week 28)	0.00% 0/2 • From start of study drug administration up to end of the study (up to Week 28)
General disorders Injection site bruising	25.0% 2/8 • Number of events 9 • From start of study drug administration up to end of the study (up to Week 28)	0.00% 0/2 • From start of study drug administration up to end of the study (up to Week 28)
General disorders Injection site erythema	37.5% 3/8 • Number of events 12 • From start of study drug administration up to end of the study (up to Week 28)	0.00% 0/2 • From start of study drug administration up to end of the study (up to Week 28)
General disorders Injection site haematoma	12.5% 1/8 • Number of events 1 • From start of study drug administration up to end of the study (up to Week 28)	0.00% 0/2 • From start of study drug administration up to end of the study (up to Week 28)
General disorders Injection site induration	12.5% 1/8 • Number of events 1 • From start of study drug administration up to end of the study (up to Week 28)	0.00% 0/2 • From start of study drug administration up to end of the study (up to Week 28)
General disorders Injection site pain	12.5% 1/8 • Number of events 1 • From start of study drug administration up to end of the study (up to Week 28)	0.00% 0/2 • From start of study drug administration up to end of the study (up to Week 28)
General disorders Injection site rash	12.5% 1/8 • Number of events 4 • From start of study drug administration up to end of the study (up to Week 28)	0.00% 0/2 • From start of study drug administration up to end of the study (up to Week 28)
General disorders Injection site reaction	12.5% 1/8 • Number of events 1 • From start of study drug administration up to end of the study (up to Week 28)	0.00% 0/2 • From start of study drug administration up to end of the study (up to Week 28)
General disorders Pyrexia	25.0% 2/8 • Number of events 3 • From start of study drug administration up to end of the study (up to Week 28)	0.00% 0/2 • From start of study drug administration up to end of the study (up to Week 28)
Immune system disorders Food allergy	12.5% 1/8 • Number of events 1 • From start of study drug administration up to end of the study (up to Week 28)	0.00% 0/2 • From start of study drug administration up to end of the study (up to Week 28)
Infections and infestations Device related infection	12.5% 1/8 • Number of events 1 • From start of study drug administration up to end of the study (up to Week 28)	0.00% 0/2 • From start of study drug administration up to end of the study (up to Week 28)
Infections and infestations Gingivitis	12.5% 1/8 • Number of events 1 • From start of study drug administration up to end of the study (up to Week 28)	0.00% 0/2 • From start of study drug administration up to end of the study (up to Week 28)
Infections and infestations Hand-foot-and-mouth disease	12.5% 1/8 • Number of events 1 • From start of study drug administration up to end of the study (up to Week 28)	0.00% 0/2 • From start of study drug administration up to end of the study (up to Week 28)
Infections and infestations Influenza	12.5% 1/8 • Number of events 1 • From start of study drug administration up to end of the study (up to Week 28)	0.00% 0/2 • From start of study drug administration up to end of the study (up to Week 28)
Infections and infestations Medical device site infection	25.0% 2/8 • Number of events 2 • From start of study drug administration up to end of the study (up to Week 28)	0.00% 0/2 • From start of study drug administration up to end of the study (up to Week 28)
Infections and infestations Otitis media	0.00% 0/8 • From start of study drug administration up to end of the study (up to Week 28)	50.0% 1/2 • Number of events 1 • From start of study drug administration up to end of the study (up to Week 28)
Infections and infestations Pharyngitis streptococcal	12.5% 1/8 • Number of events 1 • From start of study drug administration up to end of the study (up to Week 28)	0.00% 0/2 • From start of study drug administration up to end of the study (up to Week 28)
Infections and infestations Rhinitis	12.5% 1/8 • Number of events 1 • From start of study drug administration up to end of the study (up to Week 28)	0.00% 0/2 • From start of study drug administration up to end of the study (up to Week 28)
Infections and infestations Viral upper respiratory tract infection	75.0% 6/8 • Number of events 6 • From start of study drug administration up to end of the study (up to Week 28)	50.0% 1/2 • Number of events 1 • From start of study drug administration up to end of the study (up to Week 28)
Injury, poisoning and procedural complications Frostbite	12.5% 1/8 • Number of events 1 • From start of study drug administration up to end of the study (up to Week 28)	0.00% 0/2 • From start of study drug administration up to end of the study (up to Week 28)
Injury, poisoning and procedural complications Gastrostomy tube site complication	12.5% 1/8 • Number of events 2 • From start of study drug administration up to end of the study (up to Week 28)	0.00% 0/2 • From start of study drug administration up to end of the study (up to Week 28)
Injury, poisoning and procedural complications Vaccination complication	0.00% 0/8 • From start of study drug administration up to end of the study (up to Week 28)	50.0% 1/2 • Number of events 1 • From start of study drug administration up to end of the study (up to Week 28)
Investigations Amylase increased	12.5% 1/8 • Number of events 1 • From start of study drug administration up to end of the study (up to Week 28)	0.00% 0/2 • From start of study drug administration up to end of the study (up to Week 28)
Metabolism and nutrition disorders Dehydration	12.5% 1/8 • Number of events 1 • From start of study drug administration up to end of the study (up to Week 28)	0.00% 0/2 • From start of study drug administration up to end of the study (up to Week 28)
Metabolism and nutrition disorders Hyperlipasaemia	12.5% 1/8 • Number of events 1 • From start of study drug administration up to end of the study (up to Week 28)	0.00% 0/2 • From start of study drug administration up to end of the study (up to Week 28)
Metabolism and nutrition disorders Hypertriglyceridaemia	12.5% 1/8 • Number of events 1 • From start of study drug administration up to end of the study (up to Week 28)	0.00% 0/2 • From start of study drug administration up to end of the study (up to Week 28)
Metabolism and nutrition disorders Hyperzincaemia	12.5% 1/8 • Number of events 1 • From start of study drug administration up to end of the study (up to Week 28)	0.00% 0/2 • From start of study drug administration up to end of the study (up to Week 28)
Nervous system disorders Headache	12.5% 1/8 • Number of events 1 • From start of study drug administration up to end of the study (up to Week 28)	0.00% 0/2 • From start of study drug administration up to end of the study (up to Week 28)
Product Issues Device damage	25.0% 2/8 • Number of events 2 • From start of study drug administration up to end of the study (up to Week 28)	0.00% 0/2 • From start of study drug administration up to end of the study (up to Week 28)
Reproductive system and breast disorders Balanoposthitis	12.5% 1/8 • Number of events 1 • From start of study drug administration up to end of the study (up to Week 28)	0.00% 0/2 • From start of study drug administration up to end of the study (up to Week 28)
Respiratory, thoracic and mediastinal disorders Upper respiratory tract inflammation	0.00% 0/8 • From start of study drug administration up to end of the study (up to Week 28)	50.0% 1/2 • Number of events 2 • From start of study drug administration up to end of the study (up to Week 28)
Skin and subcutaneous tissue disorders Dermatitis contact	0.00% 0/8 • From start of study drug administration up to end of the study (up to Week 28)	50.0% 1/2 • Number of events 1 • From start of study drug administration up to end of the study (up to Week 28)
Skin and subcutaneous tissue disorders Dermatitis diaper	12.5% 1/8 • Number of events 1 • From start of study drug administration up to end of the study (up to Week 28)	50.0% 1/2 • Number of events 2 • From start of study drug administration up to end of the study (up to Week 28)
Skin and subcutaneous tissue disorders Dry skin	25.0% 2/8 • Number of events 2 • From start of study drug administration up to end of the study (up to Week 28)	50.0% 1/2 • Number of events 1 • From start of study drug administration up to end of the study (up to Week 28)
Skin and subcutaneous tissue disorders Eczema	12.5% 1/8 • Number of events 1 • From start of study drug administration up to end of the study (up to Week 28)	0.00% 0/2 • From start of study drug administration up to end of the study (up to Week 28)
Skin and subcutaneous tissue disorders Rash	50.0% 4/8 • Number of events 5 • From start of study drug administration up to end of the study (up to Week 28)	0.00% 0/2 • From start of study drug administration up to end of the study (up to Week 28)