A Study in Japanese Children With Short Bowel Syndrome Who Completed SHP633-302

NCTNCT03268811Other idSHP633-305Other id2021-005404-36

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Overview

Status: Completed
Phase: Phase 3
Enrollment: 9 participants (actual)
Sponsor: Shire · Industry
Design: Allocation not applicable · Open label (no masking)
Countries: 1 country
Japan
Start: 23 Aug 2017
Primary completion: 2 Nov 2021
Completion: 2 Nov 2021
First posted: 31 Aug 2017
Last update posted: 24 May 2022
Conditions: Short Bowel Syndrome

Timeline

1 trial · 1 completed · 1 with posted results

Completed
Ongoing
Not yet recruiting
Stopped
Estimated dates (est.)
Results posted

201720182019202020212022

Ph 3
A Study in Japanese Children With Short Bowel Syndrome Who Completed SHP633-302
NCT03268811
23 Aug 2017 to 2 Nov 2021

Registry summary

Text and fields below are the sponsor's entries at ClinicalTrials.gov, shown as posted.

The purpose of this clinical study is to evaluate the long-term safety and tolerability of teduglutide treatment in Japanese pediatric participants with short bowel syndrome (SBS) who completed Study SHP633-302 (NCT02980666).

Interventions

TeduglutideDrug

Arms1

Experimental
Teduglutide
Participants will receive teduglutide 0.05 milligram per kilogram (mg/kg) subcutaneous (SC) injection once daily into 1 of the 4 quadrants of the abdomen or either thigh or arm for 24-week intervals. If a participant deteriorates during a follow-up period, the participant may be evaluated immediately for additional teduglutide treatment (24-week interval) until teduglutide is commercially available for each participant, the participant's participation in this study is discontinued, or the study is discontinued.

Primary outcomes33

Outcomes the sponsor listed as primary.

Number of Participants With Treatment-emergent Adverse Events (TEAEs)
Time frame · From Baseline up to follow-up (up to 50 months)
Registry description
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 (IP) in the core study (SHP633-302 \[NCT02980666\]) or this extension study. Number of participants with TEAEs were reported. Baseline refers to the baseline value of the core study (SHP633-302 \[NCT02980666\]).
Number of Participants With Clinically Significant Abnormalities in Vital Signs
Time frame · From Baseline up to follow-up (up to 50 months)
Registry description
Vital sign assessments included pulse rate, blood pressure (systolic and diastolic blood pressure) and body temperature. Number of participants with clinically significant abnormalities in vital signs which were deemed clinically significant by the investigator were reported. Baseline refers to the baseline value of the core study (SHP633-302 \[NCT02980666\]).
Number of Participants With Clinically Significant Abnormalities in Laboratory Parameters
Time frame · From Baseline up to follow-up (up to 50 months)
Registry description
Clinical laboratory parameters included biochemistry, hematology and urinalysis. Number of participants with clinically significant abnormalities in laboratory parameters which were deemed clinically significant by the investigator were reported. Baseline refers to the baseline value of the core study (SHP633-302 \[NCT02980666\]).
Percent Change From Baseline in Average Total Urine Output at End of Treatment (EOT) of Last Cycle During Teduglutide Treatment
Time frame · Baseline, EOT of last cycle (up to Month 45) (Each Cycle 1 to 8 = 24 weeks, and Cycle 9 = 4 weeks)
Registry description
Average total urine output was recorded over a 48-hour period of parental support (PS) stability before every scheduled site visit and within 1 week of implementing any PS adjustment. The Average daily urine output milliliter per kilogram per day (mL/kg/day) was calculated as: (Total urine output over 48 hours / 2) / body weight (kilogram \[kg\]) where total urine output was calculated as the sum of the urine output in milliliter (mL) and the urine-only diaper weights in gram (g) (1g = 1mL) for the participant collected on the output diary form of electronic case report from (eCRF). Percent change from baseline in average total urine output at EOT of last cycle during teduglutide treatment was reported. Baseline refers to the baseline value of the core study (SHP633-302 \[NCT02980666\]).
Percent Change From Baseline in Average Number of Stools Per Day at EOT of Last Cycle During Teduglutide Treatment
Time frame · Baseline, EOT of last cycle (up to Month 45) (Each Cycle 1 to 8 = 24 weeks, and Cycle 9 = 4 weeks)
Registry description
Fecal output was recorded over a 48-hour period of PS stability before every scheduled site visit and within 1 week of implementing any PS adjustment. The average daily fecal output was summarized by the average number of stools per day. The average number of stools per day was calculated as (sum of the daily data in a 48-hour period/2). Percent change from baseline in average number of stools per day at EOT of last cycle during teduglutide treatment was reported. Baseline refers to the baseline value of the core study (SHP633-302 \[NCT02980666\]).
Percent Change From Baseline in Average Stool/Mixed Stool Diaper Weight at EOT of Last Cycle During Teduglutide Treatment
Time frame · Baseline, EOT of last cycle (up to Month 45) (Each Cycle 1 to 8 = 24 weeks, and Cycle 9 = 4 weeks)
Registry description
Fecal output was recorded over a 48-hour period of PS stability before every scheduled site visit and within 1 week of implementing any PS adjustment. The average daily fecal output was summarized by the average stool/mixed stool diaper weight (gram per kilogram per day \[g/kg/day\]). The body weight was used to calculate the daily stool/mixed stool diaper weight (g/kg/day). Percent change from baseline in average stool/mixed stool diaper weight at EOT of last cycle during teduglutide treatment was reported. Baseline refers to the baseline value of the core study (SHP633-302 \[NCT02980666\]).
Percent Change From Baseline in Average Total Ostomy Output at EOT of Last Cycle During Teduglutide Treatment
Time frame · Baseline, EOT of last cycle (up to Month 45) (Each Cycle 1 to 8 = 24 weeks, and Cycle 9 = 4 weeks)
Registry description
Fecal output was recorded over a 48-hour period of PS stability before every scheduled site visit and within 1 week of implementing any PS adjustment. The body weight was used to calculate the average total ostomy output per day (mL/kg/day) using a formula analogous to that used to calculate the average daily urine output. Percent change from baseline in average total ostomy output at EOT of last cycle during teduglutide treatment was reported. Baseline refers to the baseline value of the core study (SHP633-302 \[NCT02980666\]).
Percent Change From Baseline in Average Bristol Stool Form Score at EOT of Last Cycle During Teduglutide Treatment
Time frame · Baseline, EOT of last cycle (up to Month 45) (Each Cycle 1 to 8 = 24 weeks, and Cycle 9 = 4 weeks)
Registry description
Fecal output was recorded over a 48-hour period of PS stability before every scheduled site visit and within 1 week of implementing any PS adjustment. The average daily fecal output was summarized the average typical stool form score using Bristol Stool Form Scale. The average typical stool form score was calculated as (sum of the daily data in a 48-hour period / 2). Typical Stool Form based on Bristol Stool Form Scale: 1 - Separate hard lumps, hard to pass, 2 - Sausage-shaped, but lumpy, 3 - Like a sausage but with cracks on the surface, 4- Like a sausage or snake, smooth and soft, 5- Soft blobs with clear-cut edges, 6- Fluffy pieces with ragged edges, a mushy stool, 7- Watery, no solid pieces. Entirely liquid. Baseline refers to the baseline value of the core study (SHP633-302 \[NCT02980666\]). A negative change from baseline indicates improvement.
Number of Participants With Anti-drug Antibodies (ADAs) (Including Neutralizing Antibodies) at EOT of Last Cycle During Teduglutide Treatment
Time frame · Baseline, EOT of last cycle (up to Month 45) (Each Cycle 1 to 8 = 24 weeks, and Cycle 9 = 4 weeks)
Registry description
Number of participants with ADAs (including NAbs) to teduglutide were used to summarize the presence of antibodies. The participants who tested positive only for ADAs (including NAbs) were reported. Baseline refers to the baseline value of the core study (SHP633-302 \[NCT02980666\]).
Number of Participants With Clinically Significant Changes in Gastrointestinal (GI) Specific Testing
Time frame · Baseline, EOT of last cycle (up to Month 45) (Each Cycle 1 to 8 = 24 weeks, and Cycle 9 = 4 weeks)
Registry description
GI specific testing included colonoscopy or sigmoidoscopy, abdominal ultrasound, fecal occult blood testing, upper GI series with small bowel follow-through (UGI/SBFT). Number of participants with clinically significant changes findings in gastrointestinal specific testing were reported. Baseline refers to the baseline value of the core study (SHP633-302 \[NCT02980666\]).
Change From Baseline in Body Weight For Age Z-Score at EOT of Last Cycle During Teduglutide Treatment
Time frame · Baseline, EOT of last cycle (up to Month 45) (Each Cycle 1 to 8 = 24 weeks, and Cycle 9 = 4 weeks)
Registry description
Body weight was measured using Age Z-score. A Z-score was 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. Centers for Disease Control and Prevention (age \>= 2 years old) and World Health Organization (age \< 2 years old) Z-score calculation charts were used for calculation. 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 EOT of last cycle during teduglutide treatment was reported. Baseline refers to the baseline value of the core study (SHP633-302 \[NCT02980666\]).
Change From Baseline in Height For Age Z-Score at EOT of Cycle 1 During Teduglutide Treatment
Time frame · Baseline, EOT of Cycle 1 (up to 24 Weeks)
Registry description
Height was measured using Age Z-Score. A Z-score was 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. Centers for Disease Control and Prevention (age \>= 2 years old) and World Health Organization (age \< 2 years old) Z-score calculation charts were used for calculation. 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 EOT of cycle 1 during teduglutide treatment was reported. Baseline refers to the baseline value of the core study (SHP633-302 \[NCT02980666\]).
Change From Baseline in Height For Age Z-Score at EOT of Cycle 2 During Teduglutide Treatment
Time frame · Baseline, EOT of Cycle 2 (up to 48 weeks)
Registry description
Height was measured using Age Z-Score. A Z-score was 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. Centers for Disease Control and Prevention (age \>= 2 years old) and World Health Organization (age \< 2 years old) Z-score calculation charts were used for calculation. 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 EOT of Cycle 2 during teduglutide treatment was reported. Baseline refers to the baseline value of the core study (SHP633-302 \[NCT02980666\]).
Change From Baseline in Height For Age Z-Score at EOT of Cycle 3 During Teduglutide Treatment
Time frame · Baseline, EOT of Cycle 3 (up to 72 weeks)
Registry description
Height was measured using Age Z-Score. A Z-score was 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. Centers for Disease Control and Prevention (age \>= 2 years old) and World Health Organization (age \< 2 years old) Z-score calculation charts were used for calculation. 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 EOT of Cycle 3 during teduglutide treatment was reported. Baseline refers to the baseline value of the core study (SHP633-302 \[NCT02980666\]).
Change From Baseline in Height For Age Z-Score at EOT of Cycle 4 During Teduglutide Treatment
Time frame · Baseline, EOT of Cycle 4 (up to 96 weeks)
Registry description
Height was measured using Age Z-Score. A Z-score was 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. Centers for Disease Control and Prevention (age \>= 2 years old) and World Health Organization (age \< 2 years old) Z-score calculation charts were used for calculation. 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 EOT of Cycle 4 during teduglutide treatment was reported. Baseline refers to the baseline value of the core study (SHP633-302 \[NCT02980666\]).
Change From Baseline in Height For Age Z-Score at EOT of Cycle 5 During Teduglutide Treatment
Time frame · Baseline, EOT of Cycle 5 (up to 120 weeks)
Registry description
Height was measured using Age Z-Score. A Z-score was 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. Centers for Disease Control and Prevention (age \>= 2 years old) and World Health Organization (age \< 2 years old) Z-score calculation charts were used for calculation. 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 EOT of Cycle 5 during teduglutide treatment was reported. Baseline refers to the baseline value of the core study (SHP633-302 \[NCT02980666\]).
Change From Baseline in Height For Age Z-Score at EOT of Cycle 6 During Teduglutide Treatment
Time frame · Baseline, EOT of Cycle 6 (up to 144 weeks)
Registry description
Height was measured using Age Z-Score. A Z-score was 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. Centers for Disease Control and Prevention (age \>= 2 years old) and World Health Organization (age \< 2 years old) Z-score calculation charts were used for calculation. 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 EOT of Cycle 6 during teduglutide treatment was reported. Baseline refers to the baseline value of the core study (SHP633-302 \[NCT02980666\]).
Change From Baseline in Height For Age Z-Score at EOT of Cycle 7 During Teduglutide Treatment
Time frame · Baseline, EOT of Cycle 7 (up to 168 weeks)
Registry description
Height was measured using Age Z-Score. A Z-score was 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. Centers for Disease Control and Prevention (age \>= 2 years old) and World Health Organization (age \< 2 years old) Z-score calculation charts were used for calculation. 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 EOT of Cycle 7 during teduglutide treatment was reported. Baseline refers to the baseline value of the core study (SHP633-302 \[NCT02980666\]).
Change From Baseline in Height For Age Z-Score at EOT of Cycle 8 During Teduglutide Treatment
Time frame · Baseline, EOT of Cycle 8 (up to 192 weeks)
Registry description
Height was measured using Age Z-Score. A Z-score was 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. Centers for Disease Control and Prevention (age \>= 2 years old) and World Health Organization (age \< 2 years old) Z-score calculation charts were used for calculation. 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 EOT of Cycle 8 during teduglutide treatment was reported. Baseline refers to the baseline value of the core study (SHP633-302 \[NCT02980666\]).
Change From Baseline in Height For Age Z-Score at EOT of Cycle 9 During Teduglutide Treatment
Time frame · Baseline, EOT of Cycle 9 (up to 196 weeks)
Registry description
Height was measured using Age Z-Score. A Z-score was 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. Centers for Disease Control and Prevention (age \>= 2 years old) and World Health Organization (age \< 2 years old) Z-score calculation charts were used for calculation. 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 EOT of Cycle 9 during teduglutide treatment was reported. Baseline refers to the baseline value of the core study (SHP633-302 \[NCT02980666\]).
Change From Baseline in Head Circumference for Age Z-score at EOT of Cycle 1 During Teduglutide Treatment
Time frame · Baseline, EOT of Cycle 1 (up to 24 weeks)
Registry description
Head circumference was measured using Age Z-score. A Z-score was 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. Centers for Disease Control and Prevention (age \>= 2 years old) and World Health Organization (age \< 2 years old) Z-score calculation charts were used for calculation. 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 EOT of Cycle 1 during teduglutide treatment was reported. Baseline refers to the baseline value of the core study (SHP633-302 \[NCT02980666\]).
Change From Baseline in Head Circumference for Age Z-score at EOT of Cycle 2 During Teduglutide Treatment
Time frame · Baseline, EOT of Cycle 2 (up to 48 weeks)
Registry description
Head circumference was measured using Age Z-score. A Z-score was 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. Centers for Disease Control and Prevention (age \>= 2 years old) and World Health Organization (age \< 2 years old) Z-score calculation charts were used for calculation. 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 EOT of Cycle 2 during teduglutide treatment was reported. Baseline refers to the baseline value of the core study (SHP633-302 \[NCT02980666\]).
Change From Baseline in Head Circumference for Age Z-score at EOT of Cycle 3 During Teduglutide Treatment
Time frame · Baseline, EOT of Cycle 3 (up to 72 weeks)
Registry description
Head circumference was measured using Age Z-score. A Z-score was 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. Centers for Disease Control and Prevention (age \>= 2 years old) and World Health Organization (age \< 2 years old) Z-score calculation charts were used for calculation. 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 EOT of Cycle 3 during teduglutide treatment was reported. Baseline refers to the baseline value of the core study (SHP633-302 \[NCT02980666\]).
Change From Baseline in Head Circumference for Age Z-score at EOT of Cycle 4 During Teduglutide Treatment
Time frame · Baseline, EOT of Cycle 4 (up to 76 weeks)
Registry description
Head circumference was measured using Age Z-score. A Z-score was 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. Centers for Disease Control and Prevention (age \>= 2 years old) and World Health Organization (age \< 2 years old) Z-score calculation charts were used for calculation. 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 EOT of Cycle 4 during teduglutide treatment was reported. Baseline refers to the baseline value of the core study (SHP633-302 \[NCT02980666\]).
Change From Baseline in Body Mass Index (BMI) for Age Z-score at EOT of Cycle 1 During Teduglutide Treatment
Time frame · Baseline, EOT of Cycle 1 (up to 24 weeks)
Registry description
BMI Z-score was calculated by using the height and weight Age Z-score. A Z-score was 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. Centers for Disease Control and Prevention (age \>= 2 years old) and World Health Organization (age \< 2 years old) Z-score calculation charts were used for calculation. 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 BMI for Age Z-score at EOT of Cycle 1 during teduglutide treatment was reported. Baseline refers to the baseline value of the core study (SHP633-302 \[NCT02980666\]).
Change From Baseline in BMI for Age Z-score at EOT of Cycle 2 During Teduglutide Treatment
Time frame · Baseline, EOT of Cycle 2 (up to 48 weeks)
Registry description
BMI Z-score was calculated by using the height and weight Age Z-score. A Z-score was 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. Centers for Disease Control and Prevention (age \>= 2 years old) and World Health Organization (age \< 2 years old) Z-score calculation charts were used for calculation. 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 BMI for Age Z-score at EOT of Cycle 2 during teduglutide treatment was reported. Baseline refers to the baseline value of the core study (SHP633-302 \[NCT02980666\]).
Change From Baseline in BMI for Age Z-score at EOT of Cycle 3 During Teduglutide Treatment
Time frame · Baseline, EOT of Cycle 3 (up to 72 weeks)
Registry description
BMI Z-score was calculated by using the height and weight Age Z-score. A Z-score was 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. Centers for Disease Control and Prevention (age \>= 2 years old) and World Health Organization (age \< 2 years old) Z-score calculation charts were used for calculation. 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 BMI for Age Z-score at EOT of Cycle 3 during teduglutide treatment was reported. Baseline refers to the baseline value of the core study (SHP633-302 \[NCT02980666\]).
Change From Baseline in BMI for Age Z-score at EOT of Cycle 4 During Teduglutide Treatment
Time frame · Baseline, EOT of Cycle 4 (up to 96 weeks)
Registry description
BMI Z-score was calculated by using the height and weight Age Z-score. A Z-score was 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. Centers for Disease Control and Prevention (age \>= 2 years old) and World Health Organization (age \< 2 years old) Z-score calculation charts were used for calculation. 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 BMI for Age Z-score at EOT of Cycle 4 during teduglutide treatment was reported. Baseline refers to the baseline value of the core study (SHP633-302 \[NCT02980666\]).
Change From Baseline in BMI for Age Z-score at EOT of Cycle 5 During Teduglutide Treatment
Time frame · Baseline, EOT of Cycle 5 (up to 120 weeks)
Registry description
BMI Z-score was calculated by using the height and weight Age Z-score. A Z-score was 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. Centers for Disease Control and Prevention (age \>= 2 years old) and World Health Organization (age \< 2 years old) Z-score calculation charts were used for calculation. 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 BMI for Age Z-score at EOT of Cycle 5 during teduglutide treatment was reported. Baseline refers to the baseline value of the core study (SHP633-302 \[NCT02980666\]).
Change From Baseline in BMI for Age Z-score at EOT of Cycle 6 During Teduglutide Treatment
Time frame · Baseline, EOT of Cycle 6 (up to 144 weeks)
Registry description
BMI Z-score was calculated by using the height and weight Age Z-score. A Z-score was 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. Centers for Disease Control and Prevention (age \>= 2 years old) and World Health Organization (age \< 2 years old) Z-score calculation charts were used for calculation. 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 BMI for Age Z-score at EOT of Cycle 6 during teduglutide treatment was reported. Baseline refers to the baseline value of the core study (SHP633-302 \[NCT02980666\]).
Change From Baseline in BMI for Age Z-score at EOT of Cycle 7 During Teduglutide Treatment
Time frame · Baseline, EOT of Cycle 7 (up to 168 weeks)
Registry description
BMI Z-score was calculated by using the height and weight Age Z-score. A Z-score was 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. Centers for Disease Control and Prevention (age \>= 2 years old) and World Health Organization (age \< 2 years old) Z-score calculation charts were used for calculation. 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 BMI for Age Z-score at EOT of Cycle 7 during teduglutide treatment was reported. Baseline refers to the baseline value of the core study (SHP633-302 \[NCT02980666\]).
Change From Baseline in BMI for Age Z-score at EOT of Cycle 8 During Teduglutide Treatment
Time frame · Baseline, EOT of Cycle 8 (up to 192 weeks)
Registry description
BMI Z-score was calculated by using the height and weight Age Z-score. A Z-score was 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. Centers for Disease Control and Prevention (age \>= 2 years old) and World Health Organization (age \< 2 years old) Z-score calculation charts were used for calculation. 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 BMI for Age Z-score at EOT of Cycle 8 during teduglutide treatment was reported. Baseline refers to the baseline value of the core study (SHP633-302 \[NCT02980666\]).
Change From Baseline in BMI for Age Z-score at EOT of Cycle 9 During Teduglutide Treatment
Time frame · Baseline, EOT of Cycle 9 (up to 196 weeks)
Registry description
BMI Z-score was calculated by using the height and weight Age Z-score. A Z-score was 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. Centers for Disease Control and Prevention (age \>= 2 years old) and World Health Organization (age \< 2 years old) Z-score calculation charts were used for calculation. 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 BMI for Age Z-score at EOT of Cycle 9 during teduglutide treatment was reported. Baseline refers to the baseline value of the core study (SHP633-302 \[NCT02980666\]).

Secondary outcomes(9)

Number of Participants Who Achieved At Least 20 Percent (%) Reduction From Baseline in Participants Diary Parenteral Support (PS) Volume at EOT of Each Cycle During Teduglutide Treatment
Time frame · Baseline, EOT of each cycle 1, 2, 3, 4, 5, 6, 7, 8 and 9 (Each Cycle 1 to 8 = 24 weeks, and Cycle 9 = 4 weeks)
Registry description
PS (parenteral nutrition or intravenous fluids) was considered for managing nutritional support in terms of volume during the treatment period. Percent reduction in weight-normalized diary PS values from baseline was calculated using the formula: % reduction in PS value at the visit = (\[average daily value at the scheduled visit - average daily value at baseline of the core study (SHP633-302 \[NCT02980666\]) / average daily value at baseline of the core study (SHP633-302 \[NCT02980666\])) \*100. Number of participants who achieved at least 20% reduction from baseline in participants diary PS volume at EOT of each cycle during teduglutide treatment were reported. Baseline refers to the baseline value of the core study (SHP633-302 \[NCT02980666\]).
Number of Participants Who Achieved At Least 20 Percent (%) Reduction From Baseline in Investigator Prescribed PS Volume at EOT of Each Cycle During Teduglutide Treatment
Time frame · Baseline, EOT of each cycle 1, 2, 3, 4, 5, 6, 7, 8 and 9 (Each Cycle 1 to 8 = 24 weeks, and Cycle 9 = 4 weeks)
Registry description
PS (parenteral nutrition or intravenous fluids) was considered for managing nutritional support in terms of volume during the treatment period. Percent reduction in weight-normalized prescribed PS values from baseline was calculated using the formula: % reduction in PS value at the visit =(\[average daily value at the scheduled visit - average daily value at baseline of the core study (SHP633-302 \[NCT02980666\]) / average daily value at baseline of the core study (SHP633-302 \[NCT02980666\])) \*100. Number of participants who achieved at least 20% reduction from baseline in Investigator prescribed PS volume at EOT of each cycle during teduglutide treatment were reported. Baseline refers to the baseline value of the core study (SHP633-302 \[NCT02980666\]).
Change From Baseline in Participant Diary PS Volume at EOT of Each Cycle During Teduglutide Treatment
Time frame · Baseline, EOT of each cycle 1, 2, 3, 4, 5, 6, 7, 8 and 9 (Each Cycle 1 to 8 = 24 weeks, and Cycle 9 = 4 weeks)
Registry description
PS (parenteral nutrition or intravenous fluids) was considered for managing nutritional support in terms of volume during the treatment period. Change from baseline in participants diary PS volume at EOT of each cycle during teduglutide treatment was reported. Baseline refers to the baseline value of the core study (SHP633-302 \[NCT02980666\]).
Percent Change From Baseline in Participant Diary PS Volume at EOT of Each Cycle During Teduglutide Treatment
Time frame · Baseline, EOT of each cycle 1, 2, 3, 4, 5, 6, 7, 8 and 9 (Each Cycle 1 to 8 = 24 weeks, and Cycle 9 = 4 weeks)
Registry description
PS (parenteral nutrition or intravenous fluids) was considered for managing nutritional support in terms of volume during the treatment period. Percent change from baseline in participants diary PS volume at EOT of each cycle during teduglutide treatment was reported. Baseline refers to the baseline value of the core study (SHP633-302 \[NCT02980666\]).
Change From Baseline in Investigator Prescribed PS Volume at EOT of Each Cycle During Teduglutide Treatment
Time frame · Baseline, EOT of each cycle 1, 2, 3, 4, 5, 6, 7, 8 and 9 (Each Cycle 1 to 8 = 24 weeks, and Cycle 9 = 4 weeks)
Registry description
PS (parenteral nutrition or intravenous fluids) was considered for managing nutritional support in terms of volume during the treatment period. Change from baseline in investigator prescribed PS volume at EOT of each cycle during teduglutide treatment was reported. Baseline refers to the baseline value of the core study (SHP633-302 \[NCT02980666\]).
Percent Change From Baseline in Investigator Prescribed PS Volume at EOT of Each Cycle During Teduglutide Treatment
Time frame · Baseline, EOT of each cycle 1, 2, 3, 4, 5, 6, 7, 8 and 9 (Each Cycle 1 to 8 = 24 weeks, and Cycle 9 = 4 weeks)
Registry description
PS (parenteral nutrition or intravenous fluids) was considered for managing nutritional support in terms of volume during the treatment period. Percent change from baseline in investigator prescribed PS volume at EOT of each cycle during teduglutide treatment was reported. Baseline refers to the baseline value of the core study (SHP633-302 \[NCT02980666\]).
Number of Participants Who Achieved Enteral Autonomy PS Volume at EOT of Each Cycle During Teduglutide Treatment
Time frame · Baseline, EOT of each cycle 1, 2, 3, 4, 5, 6, 7, 8 and 9 (Each Cycle 1 to 8 = 24 weeks, and Cycle 9 = 4 weeks)
Registry description
Enteral autonomy (completely weaned off PS) was defined as the first visit where there is no use of PS for the 7 days prior to the visit and there is no prescribed PS at that visit, and the participants remains off PS for the remainder of the treatment period of that cycle. Number of participants who achieved enteral autonomy off PS volume at EOT of each cycle during teduglutide treatment were reported. Baseline refers to the baseline value of the core study (SHP633-302 \[NCT02980666\]).
Change From Baseline in Days Per Week of Diary PS Usage at EOT of Each Cycle During Teduglutide Treatment
Time frame · Baseline, EOT of each cycle 1, 2, 3, 4, 5, 6, 7, 8 and 9 (Each Cycle 1 to 8 = 24 weeks, and Cycle 9 = 4 weeks)
Registry description
Days per week of diary PS was calculated as: Days per week of actual PS = (number of days with non-zero values for PS volume within the 7 days prior to the visit / number of days for which any PS intake data was recorded within the 7 days prior to the visit) \* 7. Change from baseline in days per week of diary PS usage at EOT of each cycle during teduglutide treatment was reported. Baseline refers to the baseline value of the core study (SHP633-302 \[NCT02980666\]).
Change From Baseline in Days Per Week of Prescribed PS Usage at EOT of Each Cycle During Teduglutide Treatment
Time frame · Baseline, EOT of each cycle 1, 2, 3, 4, 5, 6, 7, 8 and 9 (Each Cycle 1 to 8 = 24 weeks, and Cycle 9 = 4 weeks)
Registry description
Change from baseline in days per week of prescribed PS usage at EOT of each cycle during teduglutide treatment was reported. Days per week of diary PS was calculated as: Days per week of actual PS = (number of days with non-zero values for PS volume within the 7 days prior to the visit / number of days for which any PS intake data was recorded within the 7 days prior to the visit) \* 7. Change from baseline in days Per week of prescribed PS usage at EOT of each cycle during teduglutide treatment was reported. Baseline refers to the baseline value of the core study (SHP633-302 \[NCT02980666\]).

Posted results

Values are shown as posted at ClinicalTrials.gov, with their units. PepBank has not converted, pooled or compared them.

Primary outcome results

Primary outcomeCount of participants· From Baseline up to follow-up (up to 50 months)

Number of Participants With Treatment-emergent Adverse Events (TEAEs)

Group	Value (Participants)
Total Children (Aged: 1 to 15 Years)	7
Infants (Corrected Gestational Age: 4 to < 12 Months)	2

Primary outcomeCount of participants· From Baseline up to follow-up (up to 50 months)

Number of Participants With Clinically Significant Abnormalities in Vital Signs

Group	Value (Participants)
Total Children (Aged: 1 to 15 Years)	0
Infants (Corrected Gestational Age: 4 to < 12 Months)	0

Primary outcomeCount of participants· From Baseline up to follow-up (up to 50 months)