Short Bowel SyndromePhase 3NCT03268811

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

A Phase 3 study testing Teduglutide in people with Short Bowel Syndrome, run by Shire at 6 sites in Japan.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Trial summary

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). Read more about this study

Who is it for?: Any age
Conditions the study is for: Short Bowel Syndrome
What is being tested?: Teduglutide
Type of treatment: Injection or infusion
Could I receive a placebo?: No, everyone receives an active treatment
How many people: 9 participants
Where: 6 sitesJapan
Study dates: 23 Aug 2017to 2 Nov 2021
Phase: Phase 3
Are there results?: Yes, posted in the registrySummary in the doctors' view

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

Ages Any age
All sexes
No healthy volunteers
Child, Adult, Older adult

Who may be able to join

Informed consent by a parent or guardian prior to any study-related procedures.
When applicable, informed assent (as deemed appropriate by the Institutional Review Board [IRB]) by the participant prior to any study-related procedures.
Participant completed Study SHP633-302 (NCT02980666).
Participant (and/or parent/guardian) understands and is willing and able to fully adhere to study requirements as defined in this protocol.

Who cannot join

There are no exclusion criteria for this study.

Trial locations and contacts

6 sites in 1 country.

City, postcode or hospital

6 sites in 1 country

Japan6 sites

Akita University Hospital
Akita, Akita, 010-8543
Kyushu University Hospital
Fukuoka, Fukuoka, 812-8582
Kagoshima University Hospital
Kagoshima, Kagoshima-ken, 890-8520
Tohoku University Hospital
Sendai, Miyagi, 980-8574
Showa University Hospital
Shinagawa-ku, Tokyo-To, 142-8666
Tsukuba University Hospital
Tsukuba, Ibaraki, 305-8576

Other trials in Japan

About this study

Official title: A Prospective, Open-label, Long-term Safety and Efficacy Study of Teduglutide in Japanese Pediatric Subjects With Short Bowel Syndrome Who Completed SHP633-302

Conditions: Short Bowel Syndrome
Being tested: Teduglutide
Medical subject headings: Short Bowel Syndrome

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

TeduglutideDrug
Teduglutide 0.05 mg/kg SC injection will be administered once daily into 1 of the 4 quadrants of the abdomen or either thigh or arm.

Study groups in NCT03268811
Study group	Role	What they receive
Teduglutide	Experimental	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. Receives: Drug: Teduglutide

What is being measured

The primary outcome is the main question the study is designed to answer.

Number of Participants With Treatment-emergent Adverse Events (TEAEs)
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]).
Measured: From Baseline up to follow-up (up to 50 months)
Number of Participants With Clinically Significant Abnormalities in Vital Signs
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]).
Measured: From Baseline up to follow-up (up to 50 months)
Number of Participants With Clinically Significant Abnormalities in Laboratory Parameters
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]).
Measured: From Baseline up to follow-up (up to 50 months)
Percent Change From Baseline in Average Total Urine Output at End of Treatment (EOT) of Last Cycle During Teduglutide Treatment
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]).
Measured: Baseline, EOT of last cycle (up to Month 45) (Each Cycle 1 to 8 = 24 weeks, and Cycle 9 = 4 weeks)
Percent Change From Baseline in Average Number of Stools Per Day at EOT of Last Cycle During Teduglutide Treatment
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]).
Measured: Baseline, EOT of last cycle (up to Month 45) (Each Cycle 1 to 8 = 24 weeks, and Cycle 9 = 4 weeks)
Percent Change From Baseline in Average Stool/Mixed Stool Diaper Weight at EOT of Last Cycle During Teduglutide Treatment
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]).
Measured: Baseline, EOT of last cycle (up to Month 45) (Each Cycle 1 to 8 = 24 weeks, and Cycle 9 = 4 weeks)
Percent Change From Baseline in Average Total Ostomy Output at EOT of Last Cycle During Teduglutide Treatment
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]).
Measured: Baseline, EOT of last cycle (up to Month 45) (Each Cycle 1 to 8 = 24 weeks, and Cycle 9 = 4 weeks)
Percent Change From Baseline in Average Bristol Stool Form Score at EOT of Last Cycle During Teduglutide Treatment
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.
Measured: Baseline, EOT of last cycle (up to Month 45) (Each Cycle 1 to 8 = 24 weeks, and Cycle 9 = 4 weeks)
Number of Participants With Anti-drug Antibodies (ADAs) (Including Neutralizing Antibodies) at EOT of Last Cycle During Teduglutide Treatment
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]).
Measured: Baseline, EOT of last cycle (up to Month 45) (Each Cycle 1 to 8 = 24 weeks, and Cycle 9 = 4 weeks)
Number of Participants With Clinically Significant Changes in Gastrointestinal (GI) Specific Testing
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]).
Measured: Baseline, EOT of last cycle (up to Month 45) (Each Cycle 1 to 8 = 24 weeks, and Cycle 9 = 4 weeks)
Change From Baseline in Body Weight For Age Z-Score at EOT of Last Cycle During Teduglutide Treatment
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]).
Measured: Baseline, EOT of last cycle (up to Month 45) (Each Cycle 1 to 8 = 24 weeks, and Cycle 9 = 4 weeks)
Change From Baseline in Height For Age Z-Score at EOT of Cycle 1 During Teduglutide Treatment
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]).
Measured: Baseline, EOT of Cycle 1 (up to 24 Weeks)
Change From Baseline in Height For Age Z-Score at EOT of Cycle 2 During Teduglutide Treatment
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]).
Measured: Baseline, EOT of Cycle 2 (up to 48 weeks)
Change From Baseline in Height For Age Z-Score at EOT of Cycle 3 During Teduglutide Treatment
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]).
Measured: Baseline, EOT of Cycle 3 (up to 72 weeks)
Change From Baseline in Height For Age Z-Score at EOT of Cycle 4 During Teduglutide Treatment
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]).
Measured: Baseline, EOT of Cycle 4 (up to 96 weeks)
Change From Baseline in Height For Age Z-Score at EOT of Cycle 5 During Teduglutide Treatment
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]).
Measured: Baseline, EOT of Cycle 5 (up to 120 weeks)
Change From Baseline in Height For Age Z-Score at EOT of Cycle 6 During Teduglutide Treatment
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]).
Measured: Baseline, EOT of Cycle 6 (up to 144 weeks)
Change From Baseline in Height For Age Z-Score at EOT of Cycle 7 During Teduglutide Treatment
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]).
Measured: Baseline, EOT of Cycle 7 (up to 168 weeks)
Change From Baseline in Height For Age Z-Score at EOT of Cycle 8 During Teduglutide Treatment
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]).
Measured: Baseline, EOT of Cycle 8 (up to 192 weeks)
Change From Baseline in Height For Age Z-Score at EOT of Cycle 9 During Teduglutide Treatment
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]).
Measured: Baseline, EOT of Cycle 9 (up to 196 weeks)
Change From Baseline in Head Circumference for Age Z-score at EOT of Cycle 1 During Teduglutide Treatment
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]).
Measured: Baseline, EOT of Cycle 1 (up to 24 weeks)
Change From Baseline in Head Circumference for Age Z-score at EOT of Cycle 2 During Teduglutide Treatment
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]).
Measured: Baseline, EOT of Cycle 2 (up to 48 weeks)
Change From Baseline in Head Circumference for Age Z-score at EOT of Cycle 3 During Teduglutide Treatment
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]).
Measured: Baseline, EOT of Cycle 3 (up to 72 weeks)
Change From Baseline in Head Circumference for Age Z-score at EOT of Cycle 4 During Teduglutide Treatment
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]).
Measured: Baseline, EOT of Cycle 4 (up to 76 weeks)
Change From Baseline in Body Mass Index (BMI) for Age Z-score at EOT of Cycle 1 During Teduglutide Treatment
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]).
Measured: Baseline, EOT of Cycle 1 (up to 24 weeks)
Change From Baseline in BMI for Age Z-score at EOT of Cycle 2 During Teduglutide Treatment
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]).
Measured: Baseline, EOT of Cycle 2 (up to 48 weeks)
Change From Baseline in BMI for Age Z-score at EOT of Cycle 3 During Teduglutide Treatment
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]).
Measured: Baseline, EOT of Cycle 3 (up to 72 weeks)
Change From Baseline in BMI for Age Z-score at EOT of Cycle 4 During Teduglutide Treatment
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]).
Measured: Baseline, EOT of Cycle 4 (up to 96 weeks)
Change From Baseline in BMI for Age Z-score at EOT of Cycle 5 During Teduglutide Treatment
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]).
Measured: Baseline, EOT of Cycle 5 (up to 120 weeks)
Change From Baseline in BMI for Age Z-score at EOT of Cycle 6 During Teduglutide Treatment
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]).
Measured: Baseline, EOT of Cycle 6 (up to 144 weeks)
Change From Baseline in BMI for Age Z-score at EOT of Cycle 7 During Teduglutide Treatment
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]).
Measured: Baseline, EOT of Cycle 7 (up to 168 weeks)
Change From Baseline in BMI for Age Z-score at EOT of Cycle 8 During Teduglutide Treatment
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]).
Measured: Baseline, EOT of Cycle 8 (up to 192 weeks)
Change From Baseline in BMI for Age Z-score at EOT of Cycle 9 During Teduglutide Treatment
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]).
Measured: Baseline, EOT of Cycle 9 (up to 196 weeks)

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
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]).
Measured: 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)
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
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]).
Measured: 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)
Change From Baseline in Participant Diary PS Volume at EOT of Each Cycle During Teduglutide Treatment
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]).
Measured: 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)
Percent Change From Baseline in Participant Diary PS Volume at EOT of Each Cycle During Teduglutide Treatment
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]).
Measured: 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)
Change From Baseline in Investigator Prescribed PS Volume at EOT of Each Cycle During Teduglutide Treatment
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]).
Measured: 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)
Percent Change From Baseline in Investigator Prescribed PS Volume at EOT of Each Cycle During Teduglutide Treatment
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]).
Measured: 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)
Number of Participants Who Achieved Enteral Autonomy PS Volume at EOT of Each Cycle During Teduglutide Treatment
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]).
Measured: 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)
Change From Baseline in Days Per Week of Diary PS Usage at EOT of Each Cycle During Teduglutide Treatment
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]).
Measured: 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)
Change From Baseline in Days Per Week of Prescribed PS Usage at EOT of Each Cycle During Teduglutide Treatment
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]).
Measured: 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)

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

23 Aug 20172 Nov 2021

First posted31 Aug 2017
Study start23 Aug 2017
Primary completion2 Nov 2021
Study completion2 Nov 2021

Resources

Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
Publications (1)Papers the sponsor has linked to this study.
Other trials for Short Bowel SyndromeStudies of the same condition, open and under way.
What the trial phases meanHow a treatment moves from first tests in people to approval.

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design

Study type: Interventional
Phase: Phase 3
Primary purpose: Treatment
Allocation: Not applicable
Intervention model: Single group
Masking: None (open label)
Enrolment: 9 participants (actual)

IDs, dates and publications

ClinicalTrials.gov ID: NCT03268811
Sponsor's protocol ID: SHP633-305
Other IDs: 2021-005404-36
Status last verified: Apr 2022
First submitted: 10 Aug 2017
Last updated: 24 May 2022
Results posted: Yes, on ClinicalTrials.gov
Data monitoring committee: Yes
FDA-regulated drug: Yes
FDA-regulated device: No
Individual data sharing: No: De-identified individual participant data from this particular study will not be shared as there is a reasonable likelihood that individual patients could be re-identified (due to the limited number of study participants/study sites, …).

Publications

Chiba M, Masumoto K, Kaji T, Matsuura T, Morii M, Fagbemi A, Hill S, Pakarinen MP, Protheroe S, Urs A, Chen ST, Sakui S, Udagawa E, Wada M. Efficacy and Safety of Teduglutide in Infants and Children With Short Bowel Syndrome Dependent on Parenteral Support. J Pediatr Gastroenterol Nutr. 2023 Sep 1;77(3):339-346. doi: 10.1097/MPG.0000000000003867. Epub 2023 Jun 26.
PubMed 37364133

Want to know more?

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Talk to your doctor first and take this page with you. Only the study team can confirm whether you can join.

Questions to ask the study team

What is the study trying to find out?
What would I need to do, and how often would I visit?
Could I receive a placebo instead of the treatment?
What are the possible risks and benefits for me?
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Can I keep seeing my own doctor during the study?
What happens when the study ends?

See locations

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Efficacy and Safety of PJ009 in Patients With Short Bowel Syndrome Requiring Parenteral Nutrition
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Efficacy and Safety of TRA16 Administered Orally in Patients With Enteric Hyperoxaluria (EH).
Enteric Hyperoxaluria
RecruitingPhase 3
Efficacy And Safety Evaluation of Glepaglutide in Treatment of SBS
Short Bowel Syndrome
Active, not recruitingObservational study
Registry for Participants With Short Bowel Syndrome
Short Bowel Syndrome

All Short Bowel Syndrome trials

Study at a glance

From the registry record, last updated by the sponsor on 24 May 2022. Recruitment status is as the sponsor reported it: confirm with the study team.

Official title: A Prospective, Open-label, Long-term Safety and Efficacy Study of Teduglutide in Japanese Pediatric Subjects With Short Bowel Syndrome Who Completed SHP633-302
ClinicalTrials.gov ID: NCT03268811
Acronym / sponsor ID: SHP633-305
Conditions: Short Bowel Syndrome
Phase: Phase 3
Study type: Interventional
Recruitment status: Completed
Enrolment: 9 participants (actual)
Lead sponsor: ShireIndustry (pharma, biotech, devices)
Collaborators: Takeda
Study start: 23 Aug 2017 (actual)
Primary completion: 2 Nov 2021 (actual)
Study completion: 2 Nov 2021 (actual)
First posted: 31 Aug 2017 (actual)
Results: Posted 24 May 2022

Clinical summary

The research question as the sponsor registered it. This describes what the study plans to test, not what it found.

Study design

Primary purpose: Treatment
Allocation: Not applicable
Intervention model: Single group
Masking: None (open label)
Number of arms: 1

Arm	Type	Receives
Teduglutide	Experimental	Drug: TeduglutideParticipants 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.

Who may qualify

Sex: All
Healthy volunteers: Not accepted
Age groups: Child, Adult, Older adult

Key inclusion criteria (4 listed)

Informed consent by a parent or guardian prior to any study-related procedures.
When applicable, informed assent (as deemed appropriate by the Institutional Review Board [IRB]) by the participant prior to any study-related procedures.
Participant completed Study SHP633-302 (NCT02980666).
Participant (and/or parent/guardian) understands and is willing and able to fully adhere to study requirements as defined in this protocol.

Key exclusion criteria (1 listed)

There are no exclusion criteria for this study.

Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.

Interventions

Intervention	Type	Arms
TeduglutideTeduglutide 0.05 mg/kg SC injection will be administered once daily into 1 of the 4 quadrants of the abdomen or either thigh or arm.	Drug	Teduglutide

Primary outcome

Number of Participants With Treatment-emergent Adverse Events (TEAEs)
Time frame: From Baseline up to follow-up (up to 50 months)
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)
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)
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)
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)
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)
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)
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)
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)
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)
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)
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)
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)
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)
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)
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)
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)
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)
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)
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)
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)
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)
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)
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)
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)
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)
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)
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)
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)
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)
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)
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)
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)
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]).

9 secondary outcomes

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)
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)
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)
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)
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)
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)
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)
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)
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)

Results and safety

Results posted to the registry on 24 May 2022, as reported by the sponsor. Figures are copied from the record, not re-analysed. Full results on ClinicalTrials.gov.

Participant flow

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

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

Time frame: From Baseline up to follow-up (up to 50 months). Count of participants. Unit: Participants

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

Primary outcome: Number of Participants With Clinically Significant Abnormalities in Vital Signs

Time frame: From Baseline up to follow-up (up to 50 months). Count of participants. Unit: Participants

Measure	Total Children (Aged: 1 to 15 Years) n = 7	Infants (Corrected Gestational Age: 4 to < 12 Months) n = 2
Value	0	0

Primary outcome: Number of Participants With Clinically Significant Abnormalities in Laboratory Parameters

Time frame: From Baseline up to follow-up (up to 50 months). Count of participants. Unit: Participants

Measure	Total Children (Aged: 1 to 15 Years) n = 7	Infants (Corrected Gestational Age: 4 to < 12 Months) n = 2
Biochemistry	5	1
Hematology	1	0
Urinalysis.	0	0

Primary outcome: 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). Mean (Standard Deviation). Unit: Percent change

Measure	Total Children (Aged: 1 to 15 Years) n = 6	Infants (Corrected Gestational Age: 4 to < 12 Months) n = 2
Value	-37.228 (42.9396)	146.881 (295.8061)

Population analysed: The safety population included all enrolled participants in the study and who received at least one dose of teduglutide (in study SHP633-302 \[NCT02980666\] or SHP633-305 \[NCT03268811\]). Here, "Overall number of participants analyzed" signifies those participants who were evaluable for this outcome measure.

9 secondary outcomes are reported in the full results.

Adverse events

Time frame: From Baseline up to follow-up (up to 50 months). Other adverse events reported at 5% frequency or more

Group	Serious adverse events	Other adverse events	Deaths
Total Children (Aged: 1 to 15 Years)	7 of 7 (100%)	7 of 7 (100%)	0 of 7
Infants (Corrected Gestational Age: 4 to < 12 Months)	2 of 2 (100%)	2 of 2 (100%)	0 of 2

Loading the posted results from ClinicalTrials.gov…

Research links

Official record on ClinicalTrials.govLast updated by the sponsor 24 May 2022.
Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
Derived from this study: PubMed 37364133Chiba M, Masumoto K, Kaji T, Matsuura T, Morii M, Fagbemi A, Hill S, Pakarinen MP, Protheroe S, Urs A, Chen ST, Sakui S, Udagawa E, Wada M. Efficacy and Safety of Teduglutide in Infants and Children With Short Bowel Syndrome Dependent on Parenteral Support. J Pediatr Gastroenterol Nutr. 2023 Sep 1;77(3):339-346. doi: 10.1097/MPG.0000000000003867. Epub 2023 Jun 26.
To obtain more information on the study, click here/on this linkLink provided by the sponsor.
Individual participant dataSharing planned: No. De-identified individual participant data from this particular study will not be shared as there is a reasonable likelihood that individual patients could be re-identified (due to the limited number of study participants/study sites, …).

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Referral and contact

AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.

Study Director study director

Shire

6 sites in 1 country: see every site and its status.

Questions about this trial

What is this clinical trial studying?

A Phase 3 study testing Teduglutide in people with Short Bowel Syndrome, run by Shire at 6 sites in Japan.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 4 inclusion and 1 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: finished, as of the sponsor's last update on 24 May 2022. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 6 sites in Japan. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

Yes. Results are posted in the registry (first posted 24 May 2022). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Number of Participants With Treatment-emergent Adverse Events (TEAEs) (time frame: From Baseline up to follow-up (up to 50 months)). The study lists 33 primary outcomes in all. It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT03268811, which also lists 1 linked publication.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 24 May 2022.