Trial Outcomes & Findings for Safety, Efficacy and Pharmacokinetic Study of Teduglutide in Infants 4 to 12 Months of Age With Short Bowel Syndrome (NCT NCT03571516)

Last Updated: 2021-05-11

Results Overview

Number of participants who achieved at least 20% reduction from baseline in weight-normalized PS volume at EOT/ET (up to Week 24) were reported. EOT/ET was defined as the last available visit after the date of first dose (or randomization in standard of care treatment group) during the 24-week treatment period.

Recruitment status

COMPLETED

Study phase

PHASE3

Target enrollment

10 participants

Primary outcome timeframe

Baseline, EOT/ET (up to Week 24)

Results posted on

2021-05-11

Participant Flow

The study was conducted at Finland and United Kingdom between 31 August 2018 (first participant first visit) and 24 September 2020 (last participant last visit).

A total of 10 participants were enrolled into the study, of which 8 participants completed the study.

Participant milestones

Participant milestones
Measure	Teduglutide (TED) Participants (infants) 4 to 12 months of corrected gestational aged received 0.05 milligram per kilogram (mg/kg) of teduglutide subcutaneous (SC) injection QD in addition to standard medical therapy for 24 weeks.	Standard of Care (SOC) Participants (infants) 4 to 12 months of corrected gestational aged received standard medical therapy for 24 weeks.
Overall Study STARTED	5	5
Overall Study COMPLETED	4	4
Overall Study NOT COMPLETED	1	1

Reasons for withdrawal

Reasons for withdrawal
Measure	Teduglutide (TED) Participants (infants) 4 to 12 months of corrected gestational aged received 0.05 milligram per kilogram (mg/kg) of teduglutide subcutaneous (SC) injection QD in addition to standard medical therapy for 24 weeks.	Standard of Care (SOC) Participants (infants) 4 to 12 months of corrected gestational aged received standard medical therapy for 24 weeks.
Overall Study Severe diarrhea	1	0
Overall Study Withdrawal by Subject	0	1

Baseline Characteristics

Safety, Efficacy and Pharmacokinetic Study of Teduglutide in Infants 4 to 12 Months of Age With Short Bowel Syndrome

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Teduglutide (TED) n=5 Participants Participants (infants) 4 to 12 months of corrected gestational aged received 0.05 milligram per kilogram (mg/kg) of teduglutide subcutaneous (SC) injection QD in addition to standard medical therapy for 24 weeks.	Standard of Care (SOC) n=5 Participants Participants (infants) 4 to 12 months of corrected gestational aged received standard medical therapy for 24 weeks.	Total n=10 Participants Total of all reporting groups
Race (NIH/OMB) More than one race	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Race (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Age, Continuous	8.5 Months STANDARD_DEVIATION 3.09 • n=5 Participants	8.3 Months STANDARD_DEVIATION 2.65 • n=7 Participants	8.4 Months STANDARD_DEVIATION 2.71 • n=5 Participants
Sex: Female, Male Female	1 Participants n=5 Participants	2 Participants n=7 Participants	3 Participants n=5 Participants
Sex: Female, Male Male	4 Participants n=5 Participants	3 Participants n=7 Participants	7 Participants n=5 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	5 Participants n=5 Participants	5 Participants n=7 Participants	10 Participants n=5 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Race (NIH/OMB) Asian	1 Participants n=5 Participants	1 Participants n=7 Participants	2 Participants n=5 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Race (NIH/OMB) Black or African American	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Race (NIH/OMB) White	4 Participants n=5 Participants	4 Participants n=7 Participants	8 Participants n=5 Participants

PRIMARY outcome

Timeframe: Baseline, EOT/ET (up to Week 24)

Population: Intent-to-Treat (ITT) set consisted of all participants randomized in the study.

Outcome measures

Outcome measures
Measure	Teduglutide (TED) n=5 Participants Participants (infants) 4 to 12 months of corrected gestational aged received 0.05 milligram per kilogram (mg/kg) of teduglutide subcutaneous (SC) injection QD in addition to standard medical therapy for 24 weeks.	Standard of Care (SOC) n=5 Participants Participants (infants) 4 to 12 months of corrected gestational aged received standard medical therapy for 24 weeks.
Number of Participants Who Achieved At Least 20 Percent (%) Reduction From Baseline in Weight-normalized Parenteral Support (PS) Volume at End of Treatment/Early Termination (EOT/ET) Diary Data: EOT/ET	3 Participants	1 Participants
Number of Participants Who Achieved At Least 20 Percent (%) Reduction From Baseline in Weight-normalized Parenteral Support (PS) Volume at End of Treatment/Early Termination (EOT/ET) Prescribed Data: EOT/ET	3 Participants	3 Participants

SECONDARY outcome

Timeframe: Baseline: Pre-dose,1, 4 hours post-dose, and 2 hours post-dose at Week 7

Population: Pharmacokinetic (PK) set consisted of all participants who received at least 1 dose of teduglutide and had at least 1 evaluable and interpretable post-dose PK concentration value. Here, the number of participants analyzed signifies participants who were evaluable for this outcome measure and number analyzed signifies participants who were evaluable at specific time point. Data was not planned to be collected and analyzed for SOC arm.

Mean plasma concentration of teduglutide was reported.

Outcome measures

Outcome measures
Measure	Teduglutide (TED) n=4 Participants Participants (infants) 4 to 12 months of corrected gestational aged received 0.05 milligram per kilogram (mg/kg) of teduglutide subcutaneous (SC) injection QD in addition to standard medical therapy for 24 weeks.	Standard of Care (SOC) Participants (infants) 4 to 12 months of corrected gestational aged received standard medical therapy for 24 weeks.
Plasma Concentration of Teduglutide at Nominal Time Points (Baseline at Pre-dose, and 1 Hour and 4 Hours Post-dose; 2 Hours Post-dose at Week 7) Baseline: At Pre-dose	0.00 Nanogram per milliliter (ng/mL) Interval 0.0 to 0.0	—
Plasma Concentration of Teduglutide at Nominal Time Points (Baseline at Pre-dose, and 1 Hour and 4 Hours Post-dose; 2 Hours Post-dose at Week 7) Baseline: At 1 hour	16.300 Nanogram per milliliter (ng/mL) Interval 7.25 to 25.7	—
Plasma Concentration of Teduglutide at Nominal Time Points (Baseline at Pre-dose, and 1 Hour and 4 Hours Post-dose; 2 Hours Post-dose at Week 7) Baseline: At 4 hours	8.385 Nanogram per milliliter (ng/mL) Interval 3.86 to 22.5	—
Plasma Concentration of Teduglutide at Nominal Time Points (Baseline at Pre-dose, and 1 Hour and 4 Hours Post-dose; 2 Hours Post-dose at Week 7) Week 7: At 2 hours	16.950 Nanogram per milliliter (ng/mL) Interval 14.8 to 19.1	—

SECONDARY outcome

Timeframe: From start of study treatment up to end of study (EOS) (up to Week 28)

Population: Safety set consisted of all participants who met the following criteria: Teduglutide treatment arm: participants who received at least 1 dose of teduglutide and had undergone at least 1 post baseline safety assessment; Standard of care treatment arm: participants who had undergone at least 1 post baseline safety assessment.

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 are defined as AEs that start or deteriorate on or after the date of the first dose of investigational product.

Outcome measures

Outcome measures
Measure	Teduglutide (TED) n=5 Participants Participants (infants) 4 to 12 months of corrected gestational aged received 0.05 milligram per kilogram (mg/kg) of teduglutide subcutaneous (SC) injection QD in addition to standard medical therapy for 24 weeks.	Standard of Care (SOC) n=5 Participants Participants (infants) 4 to 12 months of corrected gestational aged received standard medical therapy for 24 weeks.
Number of Participants With Treatment-emergent Adverse Events (TEAEs)	5 Participants	5 Participants

SECONDARY outcome

Timeframe: Baseline, Week 24

Body weight was measured using Z-score. Z-score was calculated as (observed value - median value of the reference population)/standard deviation value of 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 Z-score at Week 24 was reported.

Outcome measures

Outcome measures
Measure	Teduglutide (TED) n=4 Participants Participants (infants) 4 to 12 months of corrected gestational aged received 0.05 milligram per kilogram (mg/kg) of teduglutide subcutaneous (SC) injection QD in addition to standard medical therapy for 24 weeks.	Standard of Care (SOC) n=3 Participants Participants (infants) 4 to 12 months of corrected gestational aged received standard medical therapy for 24 weeks.
Change From Baseline in Body Weight Z-score at Week 24	-0.408 Z-score Standard Deviation 0.377	-0.289 Z-score Standard Deviation 0.278

SECONDARY outcome

Timeframe: Baseline, Week 24

Length was measured using Z-score. Z-score was calculated as (observed value - median value of the reference population)/standard deviation value of 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 length Z-score at Week 24 was reported.

Outcome measures

Outcome measures
Measure	Teduglutide (TED) n=4 Participants Participants (infants) 4 to 12 months of corrected gestational aged received 0.05 milligram per kilogram (mg/kg) of teduglutide subcutaneous (SC) injection QD in addition to standard medical therapy for 24 weeks.	Standard of Care (SOC) n=2 Participants Participants (infants) 4 to 12 months of corrected gestational aged received standard medical therapy for 24 weeks.
Change From Baseline in Length Z-Score at Week 24	-0.274 Z-Score Standard Deviation 1.258	-0.422 Z-Score Standard Deviation 0.384

SECONDARY outcome

Timeframe: Baseline, Week 24

Head circumference was measured using Z-score. Z-score was calculated as (observed value - median value of the reference population)/standard deviation value of 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 Z-score at Week 24 was reported.

Outcome measures

Outcome measures
Measure	Teduglutide (TED) n=4 Participants Participants (infants) 4 to 12 months of corrected gestational aged received 0.05 milligram per kilogram (mg/kg) of teduglutide subcutaneous (SC) injection QD in addition to standard medical therapy for 24 weeks.	Standard of Care (SOC) n=2 Participants Participants (infants) 4 to 12 months of corrected gestational aged received standard medical therapy for 24 weeks.
Change From Baseline in Head Circumference Z-Score at Week 24	-0.544 Z-Score Standard Deviation 0.446	-0.167 Z-Score Standard Deviation 0.690

SECONDARY outcome

Timeframe: Baseline, Week 24

Weight-for-length was measured using Z-score. Z-score was calculated as (observed value - median value of the reference population)/standard deviation value of 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 weight-for-length Z-score at Week 24 was reported.

Outcome measures

Outcome measures
Measure	Teduglutide (TED) n=4 Participants Participants (infants) 4 to 12 months of corrected gestational aged received 0.05 milligram per kilogram (mg/kg) of teduglutide subcutaneous (SC) injection QD in addition to standard medical therapy for 24 weeks.	Standard of Care (SOC) n=3 Participants Participants (infants) 4 to 12 months of corrected gestational aged received standard medical therapy for 24 weeks.
Change From Baseline in Weight-for-Length Z-Score at Week 24	-0.447 Z-Score Standard Deviation 1.042	-0.058 Z-Score Standard Deviation 0.992

SECONDARY outcome

Timeframe: Baseline, Week 24

Average total urine output was recorded over a 48-hour period of nutritional stability at Week 24 was reported. Here, milliliter per kilogram per day is abbreviated as mL/kg/day.

Outcome measures

Outcome measures
Measure	Teduglutide (TED) n=3 Participants Participants (infants) 4 to 12 months of corrected gestational aged received 0.05 milligram per kilogram (mg/kg) of teduglutide subcutaneous (SC) injection QD in addition to standard medical therapy for 24 weeks.	Standard of Care (SOC) n=3 Participants Participants (infants) 4 to 12 months of corrected gestational aged received standard medical therapy for 24 weeks.
Change From Baseline in Average Total Urine Output at Week 24	-0.61 mL/kg/day Standard Deviation 14.548	10.25 mL/kg/day Standard Deviation 17.383

SECONDARY outcome

Timeframe: Baseline, Week 24

Change from baseline in the fecal output (average number of stools per day) at Week 24 was reported.

Outcome measures

Outcome measures
Measure	Teduglutide (TED) n=3 Participants Participants (infants) 4 to 12 months of corrected gestational aged received 0.05 milligram per kilogram (mg/kg) of teduglutide subcutaneous (SC) injection QD in addition to standard medical therapy for 24 weeks.	Standard of Care (SOC) n=3 Participants Participants (infants) 4 to 12 months of corrected gestational aged received standard medical therapy for 24 weeks.
Change From Baseline in Fecal Output at Week 24	-3.33 Average number of stools per day Standard Deviation 3.547	1.67 Average number of stools per day Standard Deviation 1.756

SECONDARY outcome

Timeframe: Baseline, EOS (up to week 28)

Population: Safety set: all participants who met the following criteria: Teduglutide treatment arm: participants who received at least 1 dose of teduglutide and undergone at least 1 post baseline safety assessment; Standard of care treatment arm: participants who had undergone at least 1 post baseline safety assessment. Here, number of participants analyzed signifies participants who were evaluable for this outcome measure. Data for this outcome was not planned to be collected and analyzed for SOC group.

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

Outcome measures

Outcome measures
Measure	Teduglutide (TED) n=3 Participants Participants (infants) 4 to 12 months of corrected gestational aged received 0.05 milligram per kilogram (mg/kg) of teduglutide subcutaneous (SC) injection QD in addition to standard medical therapy for 24 weeks.	Standard of Care (SOC) Participants (infants) 4 to 12 months of corrected gestational aged received standard medical therapy for 24 weeks.
Number of Participants With Positive Specific Antibodies to Teduglutide	0 Participants	—

SECONDARY outcome

Timeframe: Baseline, EOT/ET (up to Week 24)

Population: ITT set consisted of all participants randomized in the study.

Number of participants who achieved at least 20% reduction from baseline in weight-normalized PS caloric intake at EOT/ET (up to Week 24) were reported. EOT/ET was defined as the last available visit after the date of first dose (or randomization in standard of care treatment group) during the 24-week treatment period.

Outcome measures

Outcome measures
Measure	Teduglutide (TED) n=5 Participants Participants (infants) 4 to 12 months of corrected gestational aged received 0.05 milligram per kilogram (mg/kg) of teduglutide subcutaneous (SC) injection QD in addition to standard medical therapy for 24 weeks.	Standard of Care (SOC) n=5 Participants Participants (infants) 4 to 12 months of corrected gestational aged received standard medical therapy for 24 weeks.
Number of Participants Who Achieved At Least 20 Percent (%) Reduction From Baseline in Weight-normalized Parenteral Support (PS) Caloric Intake at End of Treatment/Early Termination (EOT/ET) Diary Data: EOT/ET	3 Participants	1 Participants
Number of Participants Who Achieved At Least 20 Percent (%) Reduction From Baseline in Weight-normalized Parenteral Support (PS) Caloric Intake at End of Treatment/Early Termination (EOT/ET) Prescribed Data: EOT/ET	3 Participants	3 Participants

SECONDARY outcome

Timeframe: Week 24

Population: ITT set consisted of all participants randomized in the study. Here, the number of participants analyzed signifies participants who were evaluable for this outcome measure.

Number of participants who achieved 100% reduction in complete weaning off (enteral autonomy) PS volume at Week 24 were reported.

Outcome measures

Outcome measures
Measure	Teduglutide (TED) n=4 Participants Participants (infants) 4 to 12 months of corrected gestational aged received 0.05 milligram per kilogram (mg/kg) of teduglutide subcutaneous (SC) injection QD in addition to standard medical therapy for 24 weeks.	Standard of Care (SOC) n=4 Participants Participants (infants) 4 to 12 months of corrected gestational aged received standard medical therapy for 24 weeks.
Number of Participants Who Achieved 100 Percent (%) Reduction in Complete Weaning Off (Enteral Autonomy) Parenteral Support (PS) Volume at Week 24	0 Participants	0 Participants

SECONDARY outcome

Timeframe: EOS (up to Week 28)

Population: ITT set consisted of all participants randomized in the study. Here, the number of participants analyzed signifies participants who were evaluable for this outcome measure.

Number of participants who achieved 100% reduction in complete weaning off (enteral autonomy) PS volume at EOS (up to Week 28) were reported.

Outcome measures

Outcome measures
Measure	Teduglutide (TED) n=4 Participants Participants (infants) 4 to 12 months of corrected gestational aged received 0.05 milligram per kilogram (mg/kg) of teduglutide subcutaneous (SC) injection QD in addition to standard medical therapy for 24 weeks.	Standard of Care (SOC) n=5 Participants Participants (infants) 4 to 12 months of corrected gestational aged received standard medical therapy for 24 weeks.
Number of Participants Who Achieved 100 Percent (%) Reduction in Complete Weaning Off (Enteral Autonomy) Parenteral Support (PS) Volume at End of Study (EOS)	0 Participants	0 Participants

SECONDARY outcome

Timeframe: Baseline, EOT/ET (up to Week 24)

Population: ITT set consisted of all participants randomized in the study. Here, the number analyzed signifies participants who were evaluable for this outcome at specific time points.

Change from baseline in weight-normalized PS volume at EOT/ET (up to Week 24) was reported. EOT/ET was defined as the last available visit after the date of first dose (or randomization in standard of care treatment group) during the 24-week treatment period.

Outcome measures

Outcome measures
Measure	Teduglutide (TED) n=5 Participants Participants (infants) 4 to 12 months of corrected gestational aged received 0.05 milligram per kilogram (mg/kg) of teduglutide subcutaneous (SC) injection QD in addition to standard medical therapy for 24 weeks.	Standard of Care (SOC) n=5 Participants Participants (infants) 4 to 12 months of corrected gestational aged received standard medical therapy for 24 weeks.
Change From Baseline in Weight-normalized Parenteral Support (PS) Volume at End of Treatment/Early Termination (EOT/ET) Diary Data: EOT/ET	-21.54 mL/kg/day Standard Deviation 28.909	-9.51 mL/kg/day Standard Deviation 7.497
Change From Baseline in Weight-normalized Parenteral Support (PS) Volume at End of Treatment/Early Termination (EOT/ET) Prescribed Data: EOT/ET	-22.90 mL/kg/day Standard Deviation 26.940	-14.90 mL/kg/day Standard Deviation 12.323

SECONDARY outcome

Timeframe: Baseline, EOT/ET (up to Week 24)

Population: ITT set consisted of all participants randomized in the study. Here, the number analyzed signifies participants who were evaluable for this outcome at specific time points.

Percent change from baseline in weight-normalized PS volume at EOT/ET (up to Week 24) was reported. EOT/ET was defined as the last available visit after the date of first dose (or randomization in standard of care treatment group) during the 24-week treatment period.

Outcome measures

Outcome measures
Measure	Teduglutide (TED) n=5 Participants Participants (infants) 4 to 12 months of corrected gestational aged received 0.05 milligram per kilogram (mg/kg) of teduglutide subcutaneous (SC) injection QD in addition to standard medical therapy for 24 weeks.	Standard of Care (SOC) n=5 Participants Participants (infants) 4 to 12 months of corrected gestational aged received standard medical therapy for 24 weeks.
Percent Change From Baseline in Weight-normalized Parenteral Support (PS) Volume at End of Treatment/Early Termination (EOT/ET) Diary Data: EOT/ET	-24.77 Percent change Standard Deviation 34.723	-16.75 Percent change Standard Deviation 16.392
Percent Change From Baseline in Weight-normalized Parenteral Support (PS) Volume at End of Treatment/Early Termination (EOT/ET) Prescribed Data: EOT/ET	-27.28 Percent change Standard Deviation 33.518	-22.39 Percent change Standard Deviation 17.198

SECONDARY outcome

Timeframe: Baseline, EOT/ET (up to Week 24)

Population: ITT set consisted of all participants randomized in the study. Here, the number analyzed signifies participants who were evaluable for this outcome at specific time points.

Change from baseline in weight-normalized PS caloric intake at EOT/ET (up to Week 24) were reported. EOT/ET was defined as the last available visit after the date of first dose (or randomization in standard of care treatment group) during the 24-week treatment period. Here, kilo-calories per kilogram per day was abbreviated as (kcal/kg/day).

Outcome measures

Outcome measures
Measure	Teduglutide (TED) n=5 Participants Participants (infants) 4 to 12 months of corrected gestational aged received 0.05 milligram per kilogram (mg/kg) of teduglutide subcutaneous (SC) injection QD in addition to standard medical therapy for 24 weeks.	Standard of Care (SOC) n=5 Participants Participants (infants) 4 to 12 months of corrected gestational aged received standard medical therapy for 24 weeks.
Change From Baseline in Weight-normalized Parenteral Support (PS) Caloric Intake at End of Treatment/Early Termination (EOT/ET) Diary Data: EOT/ET	-16.14 kcal/kg/day Standard Deviation 17.547	-6.10 kcal/kg/day Standard Deviation 10.386
Change From Baseline in Weight-normalized Parenteral Support (PS) Caloric Intake at End of Treatment/Early Termination (EOT/ET) Prescribed Data: EOT/ET	-15.31 kcal/kg/day Standard Deviation 17.839	-20.40 kcal/kg/day Standard Deviation 21.024

SECONDARY outcome

Timeframe: Baseline, EOT/ET (up to Week 24)

Population: ITT set consisted of all participants randomized in the study. Here, the number analyzed signifies participants who were evaluable for this outcome at specific time points.

Percent change from baseline in weight-normalized PS caloric intake at EOT/ET (up to Week 24) were reported. EOT/ET was defined as the last available visit after the date of first dose (or randomization in standard of care treatment group) during the 24-week treatment period.

Outcome measures

Outcome measures
Measure	Teduglutide (TED) n=5 Participants Participants (infants) 4 to 12 months of corrected gestational aged received 0.05 milligram per kilogram (mg/kg) of teduglutide subcutaneous (SC) injection QD in addition to standard medical therapy for 24 weeks.	Standard of Care (SOC) n=5 Participants Participants (infants) 4 to 12 months of corrected gestational aged received standard medical therapy for 24 weeks.
Percent Change From Baseline in Weight-normalized Parenteral Support (PS) Caloric Intake at End of Treatment/Early Termination (EOT/ET) Diary Data: EOT/ET	-27.00 Percent change Standard Deviation 29.473	-13.68 Percent change Standard Deviation 21.873
Percent Change From Baseline in Weight-normalized Parenteral Support (PS) Caloric Intake at End of Treatment/Early Termination (EOT/ET) Prescribed Data: EOT/ET	-27.81 Percent change Standard Deviation 30.777	-38.86 Percent change Standard Deviation 39.893

SECONDARY outcome

Timeframe: Baseline, EOT/ET (up to Week 24)

Population: ITT set consisted of all participants randomized in the study. Here, the number of participants analyzed signifies participants who were evaluable for this outcome measure and number analyzed signifies participants who were evaluable at specific time point.

Change from baseline in weight-normalized EN volume at EOT/ET (up to Week 24) was reported. EOT/ET was defined as the last available visit after the date of first dose (or randomization in standard of care treatment group) during the 24-week treatment period.

Outcome measures

Outcome measures
Measure	Teduglutide (TED) n=4 Participants Participants (infants) 4 to 12 months of corrected gestational aged received 0.05 milligram per kilogram (mg/kg) of teduglutide subcutaneous (SC) injection QD in addition to standard medical therapy for 24 weeks.	Standard of Care (SOC) n=5 Participants Participants (infants) 4 to 12 months of corrected gestational aged received standard medical therapy for 24 weeks.
Change From Baseline in Weight-normalized Enteral Nutrition (EN) Volume at End of Treatment/Early Termination (EOT/ET) Prescribed Data: EOT/ET	-1.28 mL/kg/day Standard Deviation 2.563	2.27 mL/kg/day Standard Deviation 22.232
Change From Baseline in Weight-normalized Enteral Nutrition (EN) Volume at End of Treatment/Early Termination (EOT/ET) Diary Data: EOT/ET	16.14 mL/kg/day Standard Deviation 18.683	-15.25 mL/kg/day Standard Deviation 31.496

SECONDARY outcome

Timeframe: Baseline, EOT/ET (up to Week 24)

Percent change from baseline in weight-normalized EN volume at EOT/ET (up to Week 24) was reported. EOT/ET was defined as the last available visit after the date of first dose (or randomization in standard of care treatment group) during the 24-week treatment period.

Outcome measures

Outcome measures
Measure	Teduglutide (TED) n=2 Participants Participants (infants) 4 to 12 months of corrected gestational aged received 0.05 milligram per kilogram (mg/kg) of teduglutide subcutaneous (SC) injection QD in addition to standard medical therapy for 24 weeks.	Standard of Care (SOC) n=4 Participants Participants (infants) 4 to 12 months of corrected gestational aged received standard medical therapy for 24 weeks.
Percent Change From Baseline in Weight-normalized Enteral Nutrition (EN) Volume at End of Treatment/Early Termination (EOT/ET) Diary Data: EOT/ET	273.20 Percent change Standard Deviation 246.784	-44.25 Percent change Standard Deviation 78.847
Percent Change From Baseline in Weight-normalized Enteral Nutrition (EN) Volume at End of Treatment/Early Termination (EOT/ET) Prescribed Data: EOT/ET	-16.40 Percent change Standard Deviation NA Here, standard deviation was not analyzed due to single participant.	14.80 Percent change Standard Deviation 69.834

SECONDARY outcome

Timeframe: Baseline, EOT/ET (up to Week 24)

Change from baseline in weight-normalized EN caloric intake at EOT/ET (up to Week 24) were reported. EOT/ET was defined as the last available visit after the date of first dose (or randomization in standard of care treatment group) during the 24-week treatment period.

Outcome measures

Outcome measures
Measure	Teduglutide (TED) n=4 Participants Participants (infants) 4 to 12 months of corrected gestational aged received 0.05 milligram per kilogram (mg/kg) of teduglutide subcutaneous (SC) injection QD in addition to standard medical therapy for 24 weeks.	Standard of Care (SOC) n=5 Participants Participants (infants) 4 to 12 months of corrected gestational aged received standard medical therapy for 24 weeks.
Change From Baseline in Weight-normalized Enteral Nutrition (EN) Caloric Intake at End of Treatment/Early Termination (EOT/ET) Diary Data: EOT/ET	9.08 kcal/kg/day Standard Deviation 10.662	-9.38 kcal/kg/day Standard Deviation 21.402
Change From Baseline in Weight-normalized Enteral Nutrition (EN) Caloric Intake at End of Treatment/Early Termination (EOT/ET) Prescribed Data: EOT/ET	-1.15 kcal/kg/day Standard Deviation 2.306	3.11 kcal/kg/day Standard Deviation 16.285

SECONDARY outcome

Timeframe: Baseline, EOT/ET (up to Week 24)

Percent change from baseline in weight-normalized EN caloric intake at EOT/ET (up to Week 24) were reported. EOT/ET was defined as the last available visit after the date of first dose (or randomization in standard of care treatment group) during the 24-week treatment period.

Outcome measures

Outcome measures
Measure	Teduglutide (TED) n=2 Participants Participants (infants) 4 to 12 months of corrected gestational aged received 0.05 milligram per kilogram (mg/kg) of teduglutide subcutaneous (SC) injection QD in addition to standard medical therapy for 24 weeks.	Standard of Care (SOC) n=4 Participants Participants (infants) 4 to 12 months of corrected gestational aged received standard medical therapy for 24 weeks.
Percent Change From Baseline in Weight-normalized Enteral Nutrition (EN) Caloric Intake at End of Treatment/Early Termination (EOT/ET) Diary Data: EOT/ET	207.06 Percent change Standard Deviation 153.159	-44.25 Percent change Standard Deviation 78.847
Percent Change From Baseline in Weight-normalized Enteral Nutrition (EN) Caloric Intake at End of Treatment/Early Termination (EOT/ET) Prescribed Data: EOT/ET	-16.40 Percent change Standard Deviation NA Here, standard deviation was not analyzed due to single participant.	24.18 Percent change Standard Deviation 78.115

SECONDARY outcome

Timeframe: Baseline, EOT/ET (up to Week 24)

Population: ITT set consisted of all participants randomized in the study.

Number of participants who achieved at least 20% increase from baseline in weight-normalized EN volume at EOT/ET was reported. EOT/ET was defined as the last available visit after the date of first dose (or randomization in standard of care treatment group) during the 24-week treatment period.

Outcome measures

Outcome measures
Measure	Teduglutide (TED) n=5 Participants Participants (infants) 4 to 12 months of corrected gestational aged received 0.05 milligram per kilogram (mg/kg) of teduglutide subcutaneous (SC) injection QD in addition to standard medical therapy for 24 weeks.	Standard of Care (SOC) n=5 Participants Participants (infants) 4 to 12 months of corrected gestational aged received standard medical therapy for 24 weeks.
Number of Participants Who Achieved At Least 20 Percent (%) Increase From Baseline in Weight-normalized Enteral Nutrition (EN) Volume at End of Treatment/Early Termination (EOT/ET) Diary Data: EOT/ET	2 Participants	0 Participants
Number of Participants Who Achieved At Least 20 Percent (%) Increase From Baseline in Weight-normalized Enteral Nutrition (EN) Volume at End of Treatment/Early Termination (EOT/ET) Prescribed Data: EOT/ET	0 Participants	2 Participants

SECONDARY outcome

Timeframe: Baseline, EOT/ET (up to Week 24)

Population: ITT set consisted of all participants randomized in the study.

Number of participants who achieved at least 20% increase from baseline in weight-normalized EN caloric intake at EOT/ET was reported. EOT/ET was defined as the last available visit after the date of first dose (or randomization in standard of care treatment group) during the 24-week treatment period.

Outcome measures

Outcome measures
Measure	Teduglutide (TED) n=5 Participants Participants (infants) 4 to 12 months of corrected gestational aged received 0.05 milligram per kilogram (mg/kg) of teduglutide subcutaneous (SC) injection QD in addition to standard medical therapy for 24 weeks.	Standard of Care (SOC) n=5 Participants Participants (infants) 4 to 12 months of corrected gestational aged received standard medical therapy for 24 weeks.
Number of Participants Who Achieved At Least 20 Percent (%) Increase From Baseline in Weight-normalized Enteral Nutrition (EN) Caloric Intake at End of Treatment/Early Termination (EOT/ET) Prescribed Data: EOT/ET	0 Participants	2 Participants
Number of Participants Who Achieved At Least 20 Percent (%) Increase From Baseline in Weight-normalized Enteral Nutrition (EN) Caloric Intake at End of Treatment/Early Termination (EOT/ET) Diary Data: EOT/ET	2 Participants	0 Participants

Adverse Events

Teduglutide (TED)

Serious events: 4 serious events

Other events: 5 other events

Deaths: 0 deaths

Standard of Care (SOC)

Serious events: 3 serious events

Other events: 5 other events

Deaths: 0 deaths

Serious adverse events

Serious adverse events
Measure	Teduglutide (TED) n=5 participants at risk Participants (infants) 4 to 12 months of corrected gestational aged received 0.05 milligram per kilogram (mg/kg) of teduglutide subcutaneous (SC) injection QD in addition to standard medical therapy for 24 weeks.	Standard of Care (SOC) n=5 participants at risk Participants (infants) 4 to 12 months of corrected gestational aged received standard medical therapy for 24 weeks.
General disorders Pyrexia	20.0% 1/5 • Number of events 2 • From start of study treatment up to end of study (EOS) (up to Week 28)	0.00% 0/5 • From start of study treatment up to end of study (EOS) (up to Week 28)
Immune system disorders Immunisation reaction	20.0% 1/5 • Number of events 1 • From start of study treatment up to end of study (EOS) (up to Week 28)	0.00% 0/5 • From start of study treatment up to end of study (EOS) (up to Week 28)
Infections and infestations Device related infection	0.00% 0/5 • From start of study treatment up to end of study (EOS) (up to Week 28)	40.0% 2/5 • Number of events 2 • From start of study treatment up to end of study (EOS) (up to Week 28)
Investigations Transaminases increased	0.00% 0/5 • From start of study treatment up to end of study (EOS) (up to Week 28)	20.0% 1/5 • Number of events 1 • From start of study treatment up to end of study (EOS) (up to Week 28)
Metabolism and nutrition disorders Metabolic acidosis	0.00% 0/5 • From start of study treatment up to end of study (EOS) (up to Week 28)	20.0% 1/5 • Number of events 1 • From start of study treatment up to end of study (EOS) (up to Week 28)
Nervous system disorders Ataxia	0.00% 0/5 • From start of study treatment up to end of study (EOS) (up to Week 28)	20.0% 1/5 • Number of events 1 • From start of study treatment up to end of study (EOS) (up to Week 28)
Product Issues Device breakage	20.0% 1/5 • Number of events 1 • From start of study treatment up to end of study (EOS) (up to Week 28)	0.00% 0/5 • From start of study treatment up to end of study (EOS) (up to Week 28)
Product Issues Device leakage	20.0% 1/5 • Number of events 1 • From start of study treatment up to end of study (EOS) (up to Week 28)	0.00% 0/5 • From start of study treatment up to end of study (EOS) (up to Week 28)
Product Issues Device occlusion	0.00% 0/5 • From start of study treatment up to end of study (EOS) (up to Week 28)	20.0% 1/5 • Number of events 1 • From start of study treatment up to end of study (EOS) (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	Teduglutide (TED) n=5 participants at risk Participants (infants) 4 to 12 months of corrected gestational aged received 0.05 milligram per kilogram (mg/kg) of teduglutide subcutaneous (SC) injection QD in addition to standard medical therapy for 24 weeks.	Standard of Care (SOC) n=5 participants at risk Participants (infants) 4 to 12 months of corrected gestational aged received standard medical therapy for 24 weeks.
Blood and lymphatic system disorders Anaemia	20.0% 1/5 • Number of events 1 • From start of study treatment up to end of study (EOS) (up to Week 28)	0.00% 0/5 • From start of study treatment up to end of study (EOS) (up to Week 28)
Blood and lymphatic system disorders Iron deficiency anaemia	20.0% 1/5 • Number of events 1 • From start of study treatment up to end of study (EOS) (up to Week 28)	0.00% 0/5 • From start of study treatment up to end of study (EOS) (up to Week 28)
Gastrointestinal disorders Abdominal discomfort	20.0% 1/5 • Number of events 2 • From start of study treatment up to end of study (EOS) (up to Week 28)	0.00% 0/5 • From start of study treatment up to end of study (EOS) (up to Week 28)
Gastrointestinal disorders Abdominal distension	20.0% 1/5 • Number of events 3 • From start of study treatment up to end of study (EOS) (up to Week 28)	0.00% 0/5 • From start of study treatment up to end of study (EOS) (up to Week 28)
Gastrointestinal disorders Abnormal faeces	20.0% 1/5 • Number of events 1 • From start of study treatment up to end of study (EOS) (up to Week 28)	0.00% 0/5 • From start of study treatment up to end of study (EOS) (up to Week 28)
Gastrointestinal disorders Constipation	0.00% 0/5 • From start of study treatment up to end of study (EOS) (up to Week 28)	20.0% 1/5 • Number of events 1 • From start of study treatment up to end of study (EOS) (up to Week 28)
Gastrointestinal disorders Diarrhoea	40.0% 2/5 • Number of events 3 • From start of study treatment up to end of study (EOS) (up to Week 28)	0.00% 0/5 • From start of study treatment up to end of study (EOS) (up to Week 28)
Gastrointestinal disorders Faeces discoloured	20.0% 1/5 • Number of events 1 • From start of study treatment up to end of study (EOS) (up to Week 28)	0.00% 0/5 • From start of study treatment up to end of study (EOS) (up to Week 28)
Gastrointestinal disorders Flatulence	20.0% 1/5 • Number of events 1 • From start of study treatment up to end of study (EOS) (up to Week 28)	0.00% 0/5 • From start of study treatment up to end of study (EOS) (up to Week 28)
Gastrointestinal disorders Frequent bowel movements	40.0% 2/5 • Number of events 2 • From start of study treatment up to end of study (EOS) (up to Week 28)	0.00% 0/5 • From start of study treatment up to end of study (EOS) (up to Week 28)
Gastrointestinal disorders Gastrointestinal sounds abnormal	20.0% 1/5 • Number of events 1 • From start of study treatment up to end of study (EOS) (up to Week 28)	0.00% 0/5 • From start of study treatment up to end of study (EOS) (up to Week 28)
Gastrointestinal disorders Mucous stools	20.0% 1/5 • Number of events 1 • From start of study treatment up to end of study (EOS) (up to Week 28)	0.00% 0/5 • From start of study treatment up to end of study (EOS) (up to Week 28)
Gastrointestinal disorders Retching	20.0% 1/5 • Number of events 1 • From start of study treatment up to end of study (EOS) (up to Week 28)	0.00% 0/5 • From start of study treatment up to end of study (EOS) (up to Week 28)
Gastrointestinal disorders Teething	0.00% 0/5 • From start of study treatment up to end of study (EOS) (up to Week 28)	40.0% 2/5 • Number of events 3 • From start of study treatment up to end of study (EOS) (up to Week 28)
Gastrointestinal disorders Vomiting	60.0% 3/5 • Number of events 8 • From start of study treatment up to end of study (EOS) (up to Week 28)	20.0% 1/5 • Number of events 2 • From start of study treatment up to end of study (EOS) (up to Week 28)
General disorders Pyrexia	20.0% 1/5 • Number of events 1 • From start of study treatment up to end of study (EOS) (up to Week 28)	20.0% 1/5 • Number of events 1 • From start of study treatment up to end of study (EOS) (up to Week 28)
General disorders Secretion discharge	20.0% 1/5 • Number of events 1 • From start of study treatment up to end of study (EOS) (up to Week 28)	0.00% 0/5 • From start of study treatment up to end of study (EOS) (up to Week 28)
Infections and infestations Gastroenteritis norovirus	20.0% 1/5 • Number of events 1 • From start of study treatment up to end of study (EOS) (up to Week 28)	0.00% 0/5 • From start of study treatment up to end of study (EOS) (up to Week 28)
Infections and infestations Hand-foot-and-mouth disease	0.00% 0/5 • From start of study treatment up to end of study (EOS) (up to Week 28)	20.0% 1/5 • Number of events 1 • From start of study treatment up to end of study (EOS) (up to Week 28)
Infections and infestations Medical device site infection	20.0% 1/5 • Number of events 1 • From start of study treatment up to end of study (EOS) (up to Week 28)	0.00% 0/5 • From start of study treatment up to end of study (EOS) (up to Week 28)
Infections and infestations Nasopharyngitis	40.0% 2/5 • Number of events 2 • From start of study treatment up to end of study (EOS) (up to Week 28)	0.00% 0/5 • From start of study treatment up to end of study (EOS) (up to Week 28)
Infections and infestations Respiratory tract infection viral	20.0% 1/5 • Number of events 2 • From start of study treatment up to end of study (EOS) (up to Week 28)	0.00% 0/5 • From start of study treatment up to end of study (EOS) (up to Week 28)
Infections and infestations Upper respiratory tract infection	20.0% 1/5 • Number of events 1 • From start of study treatment up to end of study (EOS) (up to Week 28)	40.0% 2/5 • Number of events 2 • From start of study treatment up to end of study (EOS) (up to Week 28)
Infections and infestations Viral infection	20.0% 1/5 • Number of events 1 • From start of study treatment up to end of study (EOS) (up to Week 28)	20.0% 1/5 • Number of events 1 • From start of study treatment up to end of study (EOS) (up to Week 28)
Injury, poisoning and procedural complications Contusion	0.00% 0/5 • From start of study treatment up to end of study (EOS) (up to Week 28)	20.0% 1/5 • Number of events 1 • From start of study treatment up to end of study (EOS) (up to Week 28)
Injury, poisoning and procedural complications Lip injury	20.0% 1/5 • Number of events 1 • From start of study treatment up to end of study (EOS) (up to Week 28)	0.00% 0/5 • From start of study treatment up to end of study (EOS) (up to Week 28)
Injury, poisoning and procedural complications Skin abrasion	0.00% 0/5 • From start of study treatment up to end of study (EOS) (up to Week 28)	20.0% 1/5 • Number of events 1 • From start of study treatment up to end of study (EOS) (up to Week 28)
Investigations Alanine aminotransferase increased	20.0% 1/5 • Number of events 2 • From start of study treatment up to end of study (EOS) (up to Week 28)	0.00% 0/5 • From start of study treatment up to end of study (EOS) (up to Week 28)
Investigations Blood iron decreased	0.00% 0/5 • From start of study treatment up to end of study (EOS) (up to Week 28)	20.0% 1/5 • Number of events 1 • From start of study treatment up to end of study (EOS) (up to Week 28)
Investigations Faecal volume increased	20.0% 1/5 • Number of events 1 • From start of study treatment up to end of study (EOS) (up to Week 28)	0.00% 0/5 • From start of study treatment up to end of study (EOS) (up to Week 28)
Investigations Respiratory rate increased	0.00% 0/5 • From start of study treatment up to end of study (EOS) (up to Week 28)	20.0% 1/5 • Number of events 1 • From start of study treatment up to end of study (EOS) (up to Week 28)
Investigations Serum ferritin decreased	20.0% 1/5 • Number of events 1 • From start of study treatment up to end of study (EOS) (up to Week 28)	0.00% 0/5 • From start of study treatment up to end of study (EOS) (up to Week 28)
Investigations Transaminases increased	0.00% 0/5 • From start of study treatment up to end of study (EOS) (up to Week 28)	20.0% 1/5 • Number of events 2 • From start of study treatment up to end of study (EOS) (up to Week 28)
Metabolism and nutrition disorders Decreased appetite	20.0% 1/5 • Number of events 2 • From start of study treatment up to end of study (EOS) (up to Week 28)	0.00% 0/5 • From start of study treatment up to end of study (EOS) (up to Week 28)
Metabolism and nutrition disorders Hypoglycaemia	0.00% 0/5 • From start of study treatment up to end of study (EOS) (up to Week 28)	20.0% 1/5 • Number of events 1 • From start of study treatment up to end of study (EOS) (up to Week 28)
Metabolism and nutrition disorders Hypophagia	20.0% 1/5 • Number of events 1 • From start of study treatment up to end of study (EOS) (up to Week 28)	0.00% 0/5 • From start of study treatment up to end of study (EOS) (up to Week 28)
Product Issues Device breakage	20.0% 1/5 • Number of events 1 • From start of study treatment up to end of study (EOS) (up to Week 28)	20.0% 1/5 • Number of events 1 • From start of study treatment up to end of study (EOS) (up to Week 28)
Product Issues Device occlusion	20.0% 1/5 • Number of events 1 • From start of study treatment up to end of study (EOS) (up to Week 28)	0.00% 0/5 • From start of study treatment up to end of study (EOS) (up to Week 28)
Psychiatric disorders Irritability	20.0% 1/5 • Number of events 1 • From start of study treatment up to end of study (EOS) (up to Week 28)	0.00% 0/5 • From start of study treatment up to end of study (EOS) (up to Week 28)
Psychiatric disorders Sleep disorder	20.0% 1/5 • Number of events 1 • From start of study treatment up to end of study (EOS) (up to Week 28)	0.00% 0/5 • From start of study treatment up to end of study (EOS) (up to Week 28)
Respiratory, thoracic and mediastinal disorders Cough	20.0% 1/5 • Number of events 1 • From start of study treatment up to end of study (EOS) (up to Week 28)	20.0% 1/5 • Number of events 1 • From start of study treatment up to end of study (EOS) (up to Week 28)
Respiratory, thoracic and mediastinal disorders Rhinorrhoea	20.0% 1/5 • Number of events 1 • From start of study treatment up to end of study (EOS) (up to Week 28)	20.0% 1/5 • Number of events 1 • From start of study treatment up to end of study (EOS) (up to Week 28)
Skin and subcutaneous tissue disorders Dermatitis diaper	20.0% 1/5 • Number of events 2 • From start of study treatment up to end of study (EOS) (up to Week 28)	0.00% 0/5 • From start of study treatment up to end of study (EOS) (up to Week 28)
Skin and subcutaneous tissue disorders Eczema	20.0% 1/5 • Number of events 1 • From start of study treatment up to end of study (EOS) (up to Week 28)	0.00% 0/5 • From start of study treatment up to end of study (EOS) (up to Week 28)
Skin and subcutaneous tissue disorders Rash papular	0.00% 0/5 • From start of study treatment up to end of study (EOS) (up to Week 28)	20.0% 1/5 • Number of events 1 • From start of study treatment up to end of study (EOS) (up to Week 28)
Vascular disorders Hypertension	0.00% 0/5 • From start of study treatment up to end of study (EOS) (up to Week 28)	20.0% 1/5 • Number of events 1 • From start of study treatment up to end of study (EOS) (up to Week 28)