Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
Teduglutide · 22 trials · 6 indications
An AE is defined as any untoward medical occurrence in a clinical investigation participant administered a drug; it does not necessarily have to 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.
An AE is defined as any untoward medical occurrence in a clinical investigation participant administered a drug; it does not necessarily have to have a causal relationship with this treatment. An SAE is defined as any untoward medical occurrence that at any dose: results in death, is life threatening, requires inpatient hospitalization or prolongation of existing hospitalization, results in persistent or significant disability/incapacity, leads to a congenital anomaly /birth defect, is the other important medical event.
An AE is defined as any untoward medical occurrence in a clinical investigation participant administered a drug; it does not necessarily have to have a causal relationship with this treatment. An AESI, whether serious or non-serious, is one of scientific and medical concern specific to the compound or program, for which ongoing monitoring and rapid communication by the investigator to the sponsor may be appropriate.
Vital signs include systolic and diastolic blood pressure, heart rate and body temperature.
A Z-score is 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. Z-score indicates the number of standard deviations away from the mean. A Z-score of 0 is equal to the mean of a reference population (i.e., healthy, age and sex-matched individuals). According to WHO Child Growth Standards used for assessment of this outcome measure, a weight for age Z-score below -2 indicates underweight.
A Z-score is 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. Z-score indicates the number of standard deviations away from the mean. A Z-score of 0 is equal to the mean of a reference population (i.e., healthy, age and sex-matched individuals). According to WHO Child Growth Standards used for assessment of this outcome measure, a height for age Z-score below -2 indicates stunted.
A Z-score is 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. Z-score indicates the number of standard deviations away from the mean. A Z-score of 0 is equal to the mean of a reference population (i.e., healthy, age and sex-matched individuals). According to the Food and Nutrition Technical Assistance (FANTA) Guide to Anthropometry used for assessment of this outcome measure, a head circumference for age Z-score below -2 indicates small head circumference.
A Z-score is 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. Z-score indicates the number of standard deviations away from the mean. A Z-score of 0 is equal to the mean of a reference population (i.e., healthy, age and sex-matched individuals). According to WHO Child Growth Standards used for assessment of this outcome measure, a weight for length Z-score below -2 indicates wasted (recent and severe weight loss).
Laboratory safety parameters included biochemistry, hematology, and urinalysis.
Urine and stool output was recorded and calculated in the output diary over a 48-hour period of parenteral support (PS) and enteral nutrition (EN) stability before every site visit and within 1 week of implementing a change in the PS prescription.
Urine and stool output was recorded and calculated in the output diary over a 48-hour period of PS and EN stability before every site visit and within 1 week of implementing a change in the PS prescription.
Number of participants who achieved at least 20% reduction from baseline in weight-normalized PS volume at EOT/ET (up to Week 24) were reported. EOT/ET was defined as the last available visit after the date of first dose (or randomization in standard of care treatment group) during the 24-week treatment period.
The weekly volume was calculated as (sum of daily volumes in the diary/number of days with values)\*7. Baseline data refer to the baseline of core studies (SHP633-306 \[NCT03663582\] or TED-C14-004 \[NCT02340819\]), however the time frame represents the duration for which participants were assessed in current study. The only data presented from the Core Studies in this report are the comparison of the Extension Study data with the Baseline data of the Core Study.
The weekly volume was calculated as (sum of daily volumes in the diary/number of days with values)\*7. Baseline data refer to the baseline of core studies (SHP633-306 \[NCT03663582\] or TED-C14-004 \[NCT02340819\]), however the time frame represents the duration for which participants were assessed in current study. The only data presented from the Core Studies in this report are the comparison of the Extension Study data with the Baseline data of the Core Study.
The weekly volume was calculated as (sum of daily volumes in the diary/number of days with values)\*7. Baseline data refer to the baseline of core studies (SHP633-306 \[NCT03663582\] or TED-C14-004 \[NCT02340819\]), however the time frame represents the duration for which participants were assessed in current study. The only data presented from the Core Studies in this report are the comparison of the Extension Study data with the Baseline data of the Core Study.
Baseline data refer to the baseline of core studies (SHP633-306 \[NCT03663582\] or TED-C14-004 \[NCT02340819\]), however the time frame represents the duration for which participants were assessed in current study. The only data presented from the Core Studies in this report are the comparison of the Extension Study data with the Baseline data of the Core Study.
Plasma citrulline was measured as an assessment of enterocyte mass. Plasma samples were analyzed using a validated high-performance liquid chromatography-tandem mass spectrometry (LC-MS/MS) method. Baseline data refer to the baseline of core studies (SHP633-306 \[NCT03663582\] or TED-C14-004 \[NCT02340819\]), however the time frame represents the duration for which participants were assessed in current study. The only data presented from the Core Studies in this report are the comparison of the Extension Study data with the Baseline data of the Core Study.
Baseline data refer to the baseline of core studies (SHP633-306 \[NCT03663582\] or TED-C14-004 \[NCT02340819\]), however the time frame represents the duration for which participants were assessed in current study. The only data presented from the Core Studies in this report are the comparison of the Extension Study data with the Baseline data of the Core Study.
Baseline data refer to the baseline of core studies (SHP633-306 \[NCT03663582\] or TED-C14-004 \[NCT02340819\]), however the time frame represents the duration for which participants were assessed in current study. The only data presented from the Core Studies in this report are the comparison of the Extension Study data with the Baseline data of the Core Study.
Baseline data refer to the baseline of core studies (SHP633-306 \[NCT03663582\] or TED-C14-004 \[NCT02340819\]), however the time frame represents the duration for which participants were assessed in current study. The only data presented from the Core Studies in this report are the comparison of the Extension Study data with the Baseline data of the Core Study.
Baseline data refer to the baseline of core studies (SHP633-306 \[NCT03663582\] or TED-C14-004 \[NCT02340819\]), however the time frame represents the duration for which participants were assessed in current study. The only data presented from the Core Studies in this report are the comparison of the Extension Study data with the Baseline data of the Core Study.
Baseline data refer to the baseline of core studies (SHP633-306 \[NCT03663582\] or TED-C14-004 \[NCT02340819\]), however the time frame represents the duration for which participants were assessed in current study. The only data presented from the Core Studies in this report are the comparison of the Extension Study data with the Baseline data of the Core Study.
Baseline data refer to the baseline of core studies (SHP633-306 \[NCT03663582\] or TED-C14-004 \[NCT02340819\]), however the time frame represents the duration for which participants were assessed in current study. The only data presented from the Core Studies in this report are the comparison of the Extension Study data with the Baseline data of the Core Study.
Change from baseline in weekly PS volume at EOT/ET was reported.
Percent change from baseline in weekly PS volume at EOT/ET was reported.
Percentage of participants who achieved at least 20% reduction from baseline in weekly PS volume at Week 20 was reported.
Percentage of participants who achieved at least 20% reduction from baseline in weekly PS volume at Week 24 was reported.
Percentage of participants who achieve at least 20% reduction from baseline in weekly PS at EOT/ET was reported.
Change from baseline in days per week of PS at EOT/ET was reported.
Plasma citrulline levels were measured as a biomarker of enterocyte mass. Change from baseline in plasma citrulline levels up to EOT/ET was reported.
Number of participants who were completely weaned off PS at Week 24/EOT was reported.
AUC0-t of teduglutide was reported.
Cmax of teduglutide was reported.
Tmax of teduglutide was reported.
T1/2 of teduglutide was reported.
CL/F of teduglutide was reported.
Vz/F of teduglutide was reported.
12-lead ECG was performed at the study center after the participant has been resting for at least 5 minutes. Number of participants with clinically significant abnormalities in 12-Lead ECG was reported.
Change from baseline in systolic and diastolic blood pressure at EOT/ET was reported.
Change from baseline in pulse rate at EOT/ET was reported.
Change from baseline in body temperature at EOT/ET was reported.
Change from baseline in hemoglobin at EOT/ET was reported.
Change from baseline in hematocrit at EOT/ET was reported.
Change from baseline in serum blood urea nitrogen at EOT/ET was reported.
Change from baseline in creatinine at EOT/ET was reported.
Change from baseline in urine sodium at EOT/ET was reported.
Number of participants who reported positive specific antibodies to teduglutide at EOT/ET was reported.
Change from baseline in 48-hour urine output at EOT/ET was reported.
Change from baseline in body weight at EOT/ET was reported.
Change from baseline in BMI at EOT/ET was reported.
GI specific tests included colonoscopy or sigmoidoscopy, abdominal ultrasound, upper GI series with small bowel follow-through (UGI/SBFT). Number of participants with abnormal clinically significant changes in gastrointestinal specific tests at Week 24/ET was reported.
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 \[NCT02980666\]).
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 \[NCT02980666\]).
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 \[NCT02980666\]).
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 \[NCT02980666\]).
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 \[NCT02980666\]).
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 \[NCT02980666\]).
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 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 \[NCT02980666\]).
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 \[NCT02980666\]).
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 \[NCT02980666\]).
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 \[NCT02980666\]).
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 \[NCT02980666\]).
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 \[NCT02980666\]).
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 \[NCT02980666\]).
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 \[NCT02980666\]).
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 \[NCT02980666\]).
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 \[NCT02980666\]).
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 \[NCT02980666\]).
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 \[NCT02980666\]).
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 \[NCT02980666\]).
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 \[NCT02980666\]).
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 \[NCT02980666\]).
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 \[NCT02980666\]).
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 \[NCT02980666\]).
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 \[NCT02980666\]).
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 \[NCT02980666\]).
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 \[NCT02980666\]).
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 \[NCT02980666\]).
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 \[NCT02980666\]).
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 \[NCT02980666\]).
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 \[NCT02980666\]).
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 \[NCT02980666\]).
Absolute change from baseline in PS volume at EOT (up to Week 24) based on dairy data was reported. EOT was defined as the last available measurement after the date of first dose during the 24-week treatment period. Here, milliliter per kilogram per day is abbreviated as mL/kg/day.
Percent change from baseline in PS volume at EOT (up to Week 24) based on dairy data was reported. EOT was defined as the last available measurement after the date of first dose during the 24-week treatment period.
Absolute change from baseline in PS caloric intake at EOT (up to Week 24) based on dairy data was reported. EOT was defined as the last available measurement after the date of first dose during the 24-week treatment period. Here, kilo-calories per kilogram per day was abbreviated as (kcal/kg/day).
Percent change from baseline in PS caloric intake at EOT (up to Week 24) based on dairy data was reported. EOT was defined as the last available measurement after the date of first dose during the 24-week treatment period.
Absolute change from baseline in plasma citrulline at EOT (up to Week 24) was reported. EOT was defined as the last available measurement after the date of first dose during the 24-week treatment period.
Percent change from baseline in plasma citrulline at EOT (up to Week 24) was reported. EOT was defined as the last available measurement after the date of first dose during the 24-week treatment period.
Absolute change from baseline in EN volume at EOT (up to Week 24) based on dairy data was reported. EOT was defined as the last available measurement after the date of first dose during the 24-week treatment period.
Percent change from baseline in EN volume at EOT (up to Week 24) based on dairy data was reported. EOT was defined as the last available measurement after the date of first dose during the 24-week treatment period.
Absolute change from baseline in EN caloric intake at EOT (up to Week 24) based on dairy data was reported. EOT was defined as the last available measurement after the date of first dose during the 24-week treatment period.
Percent change from baseline in EN caloric intake at EOT (up to Week 24) based on dairy data was reported. EOT was defined as the last available measurement after the date of first dose during the 24-week treatment period.
Number of participants who achieved at least 20% reduction in PS volume at Week 24 was reported.
Number of participants who achieved at least 20% reduction in PS volume at EOT (up to Week 24) was reported. EOT was defined as the last available measurement after the date of first dose during the 24-week treatment period.
Number of participants who achieved at least 100% reduction in complete weaning of PS volume at EOT (up to Week 24) was reported. EOT was defined as the last available measurement after the date of first dose during the 24-week treatment period.
Number of participants who achieved \>= 20% reduction in PS volume at Week 28 was reported.
Absolute change from EOT (up to Week 24) in PS volume at EOS (up to Week 28) based on dairy data was reported. EOT was defined as the last available measurement after the date of first dose during the 24-week treatment period.
Percent change from EOT (up to Week 24) in PS volume at EOS (up to Week 28) based on dairy data was reported. EOT was defined as the last available measurement after the date of first dose during the 24-week treatment period.
Absolute change from EOT (up to Week 24) in PS caloric intake at EOS (up to Week 28) based on dairy data was reported. EOT was defined as the last available measurement after the date of first dose during the 24-week treatment period.
Percent change from EOT (up to Week 24) in PS caloric intake at EOS (up to Week 28) based on dairy data was reported. EOT was defined as the last available measurement after the date of first dose during the 24-week treatment period.
Absolute change from EOT (up to Week 24) in plasma citrulline at EOS (up to Week 28) was reported. EOT was defined as the last available measurement after the date of first dose during the 24-week treatment period.
Percent change from EOT (up to Week 24) in plasma citrulline at EOS (up to Week 28) was reported. EOT was defined as the last available measurement after the date of first dose during the 24-week treatment period.
Absolute change from EOT (up to Week 24) in EN volume at EOS (up to Week 28) based on dairy data was reported. EOT was defined as the last available measurement after the date of first dose during the 24-week treatment period.
Percent change from EOT (up to Week 24) in EN volume at EOS (up to Week 28) based on dairy data was reported. EOT was defined as the last available measurement after the date of first dose during the 24-week treatment period.
Absolute change from EOT (up to Week 24) in EN caloric intake at EOS (up to Week 28) based on dairy data was reported. EOT was defined as the last available measurement after the date of first dose during the 24-week treatment period.
Percent change from EOT (up to Week 24) in EN caloric intake at EOS (up to Week 28) based on dairy data was reported. EOT was defined as the last available measurement after the date of first dose during the 24-week treatment period.
Absolute change from baseline in number of hours per day of PS Usage at EOT (up to Week 24) based on dairy data was reported. EOT was defined as the last available measurement after the date of first dose during the 24-week treatment period.
Absolute change from baseline in number of days per Week of PS usage at EOT (up to Week 24) based on dairy data was reported. EOT was defined as the last available measurement after the date of first dose during the 24-week treatment period.
Body weight was measured using age Z-score. A Z-score was defined as the deviation of the value for an individual from the mean value of the reference population divided by the standard deviation for the reference population. A negative Z-score indicates values lower than the mean while a positive Z-score indicates values higher than the mean. Change from baseline in body weight for age Z-score at Week 28 was reported.
Height was measured using age Z-score. A Z-score was defined as the deviation of the value for an individual from the mean value of the reference population divided by the standard deviation for the reference population. A negative Z-score indicates values lower than the mean while a positive Z-score indicates values higher than the mean. Change from baseline in height for age Z-score at Week 28 was reported.
Head circumference was measured using age Z-score. A Z-score was defined as the deviation of the value for an individual from the mean value of the reference population divided by the standard deviation for the reference population. A negative Z-score indicates values lower than the mean while a positive Z-score indicates values higher than the mean. Change from baseline in head circumference for age Z-score at Week 28 was reported.
Vital sign assessments included pulse rate, blood pressure, or body temperature. Number of participants with clinically significant changes in vital signs by the investigator were recorded as TEAEs.
12-lead ECG was performed. Any change in ECG assessments which were deemed to be clinically significant changes were recorded as TEAEs.
Clinical laboratory assessments included biochemistry, hematology, coagulation, urinalysis. The number of participants with clinically significant laboratory abnormalities were reported as TEAEs.
Average urine output was recorded in measured volume at Week 28 was recorded.
Change from baseline in the fecal output (Average number of stools per day) at Week 28 was recorded.
Number of participants with positive specific antibodies to teduglutide were used to summarize the presence of antibodies.
GI specific testing included colonoscopy or sigmoidoscopy, abdominal ultrasound, fecal occult blood testing, upper GI series with small bowel follow-through (UGI/SBFT). EOT was defined as the last available measurement after the date of first dose during the 24-week treatment period. Number of participants with clinically significant abnormal findings in gastrointestinal specific testing were reported.
Since only 2 sparse pharmacokinetics (PK) samples were collected during the study, PK parameters were not estimated and analyzed using this study samples. Therefore, no PK parameters were reported in this study.
Since only 2 sparse PK samples were collected during the study, PK parameters were not estimated and analyzed using this study samples. Therefore, no PK parameters were reported in this study.
Since only 2 sparse PK samples were collected during the study, PK parameters were not estimated and analyzed using this study samples. Therefore, no PK parameters were reported in this study.
Since only 2 sparse PK samples were collected during the study, PK parameters were not estimated and analyzed using this study samples. Therefore, no PK parameters were reported in this study.
Since only 2 sparse PK samples were collected during the study, PK parameters were not estimated and analyzed using this study samples. Therefore, no PK parameters were reported in this study.
Since only 2 sparse PK samples were collected during the study, PK parameters were not estimated and analyzed using this study samples. Therefore, no PK parameters were reported in this study.
An 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. A SAE was any untoward medical occurrence (whether considered to be related to investigational product or not) that at any dose: results in death, was life-threatening, requires inpatient hospitalization or prolongation of existing hospitalization, results in persistent or significant disability/incapacity, was a congenital abnormality/birth defect, was an important medical event. Number of participants with AEs, related AEs, serious adverse events (SAEs) and related SAEs of retrospective observation period were reported.
Height was measured using age Z-score. Z-score was calculated as (observed value - median value of the reference population) / standard deviation value of reference population. Centers for Disease Control and Prevention (age greater than or equal to \[\>=\] 2 years old) and World Health Organization (age less than \[\<\] 2 years old) Z-score calculation charts are 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 up to Week 168 of retrospective observation period was reported. Data for this outcome was not planned to be collected and analyzed in retrospective TED/TED group.
Body weight was measured using age Z-score. Z-score was calculated as (observed value - median value of the reference population) / standard deviation value of reference population. Centers for Disease Control and Prevention (age \>= 2 years old) and World Health Organization (age \< 2 years old) Z-score calculation charts are 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 up to Week 168 was reported. Data for this outcome was not planned to be collected and analyzed in retrospective TED/TED group.
BMI Z-score was calculated by using the retrospective height and weight data. Z-score was calculated as (observed value - median value of the reference population) / standard deviation value of reference population. Centers for Disease Control and Prevention (age \>= 2 years old) and World Health Organization (age \< 2 years old) Z-score calculation charts are 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 up to Week 168 of retrospective observation period was reported. Data for this outcome was not planned to be collected and analyzed in retrospective TED/TED group.
TEAEs are defined as AEs that started or worsened on or after the first dose of teduglutide treatment in the core study. A SAE was any untoward medical occurrence (whether considered to be related to investigational product or not) that at any dose: results in death, was life-threatening, required inpatient hospitalization or prolongation of existing hospitalization, results in persistent or significant disability/incapacity, was a congenital abnormality/birth defect, was an important medical event. AESI was an TEAE or TESAE of scientific and medical concern specific to the sponsor's product or program and for which ongoing monitoring and immediate notification by the investigator to the sponsor. Number of participants With TEAEs, treatment -emergent serious adverse events (TESAEs) and adverse events of special interest (AESI) of Prospective study period were reported.
Height was measured using age Z-score. Z-score was calculated as (observed value - median value of the reference population) / standard deviation value of reference population. Centers for Disease Control and Prevention (age \>= 2 years old) and World Health Organization (age \< 2 years old) Z-score calculation charts are 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 up to Cycle 6 Week 12 during the end of teduglutide treatment period in prospective study period was reported.
Body weight was measured using age Z-score. Z-score was calculated as (observed value - median value of the reference population) / standard deviation value of reference population. Centers for Disease Control and Prevention (age \>= 2 years old) and World Health Organization (age \< 2 years old) Z-score calculation charts are 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 up to Cycle 6 Week 24 during the end of teduglutide treatment period of prospective study period was reported.
BMI Z-score was calculated by using the height and weight data. Z-score was calculated as (observed value - median value of the reference population) / standard deviation value of reference population. Centers for Disease Control and Prevention (age \>= 2 years old) and World Health Organization (age \< 2 years old) Z-score calculation charts are 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 up to Cycle 6 Week 12 during the end of teduglutide treatment period of prospective study period was reported.
Average total urine output was calculated based on the daily data recorded in participants diaries over a 48-hour period of nutritional stability prior to each scheduled visit. Average total 48-Hour urine output up to Cycle 6 Week 12 during the end of teduglutide treatment period of prospective study period was reported. Here, mL/kg/day is abbreviated as milliliter per kilogram per day.
Average total urine output was calculated based on the daily data recorded in participants diaries over a 48-hour period of nutritional stability prior to each scheduled visit. Average total 48-Hour urine output up to Week 108 during the end of NTT period in prospective study period was reported.
Average number of stools per day was calculated based on the daily data recorded in participants diaries over a 48-hour period of nutritional stability prior to each scheduled visit. Average number of stools per day up to cycle 6 week 24 during the end of teduglutide treatment period in prospective study period was reported.
Average number of stools per day was calculated based on the daily data recorded in participants diaries over a 48-hour period of nutritional stability prior to each scheduled visit. Average number of stools per day up to Week 120 during the end of NTT period in prospective study period was reported.
Number of participants with positive specific antibodies to teduglutide were used to summarize the presence of antibodies.
Reduction in weight-normalized PN/IV volume was performed using both participant diary and investigator prescribed data. Number of participants who achieved at least a 20% reduction in weight-normalized PN/IV volume between the baseline and week 24/EOT visit were reported.
Absolute change from baseline in weekly PS volume at Week 24 was reported.
Percent change from baseline in weekly PS volume at Week 24 was reported.
Response was defined as the achievement of at least a 20% reduction from baseline (Visit 2) in weekly PS volume at Week 20 and again at Week 24.
Absolute change from baseline in number of days per Week of PS usage at Week 24 was reported.
Plasma citrulline was measured as an assessment of enterocyte mass. Absolute change from baseline in plasma citrulline levels at Week 24 was reported.
Plasma citrulline was measured as an assessment of enterocyte mass. Percent change from baseline in plasma citrulline at Week 24 was reported.
Enteral autonomy was defined as no prescribed PS and no use of PS recorded in the participant diary at the end of stage 2. Number of participants who achieved enteral autonomy at Week 24 was reported.
Absolute change from baseline in weekly PS volume at extension Month 24 was reported. Extension month 24 = 30 months of teduglutide treatment.
Percent change from baseline in weekly PS volume at extension Month 24 was reported. Extension month 24 = 30 months of teduglutide treatment.
Absolute change from baseline in number of days per Week of PS usage at extension Month 24 was reported. Extension month 24 = 30 months of teduglutide treatment.
Plasma citrulline was measured as an assessment of enterocyte mass. Absolute change from baseline in plasma citrulline levels at extension Month 24 was reported. Extension month 24 = 30 months of teduglutide treatment.
Plasma citrulline was measured as an assessment of enterocyte mass. Percent change from baseline in plasma citrulline at extension Month 24 was reported. Extension month 24 = 30 months of teduglutide treatment.
Absolute change from baseline in weekly PS volume at end of treatment was reported.
Percent change from baseline in weekly PS volume at end of treatment was reported.
Absolute change from baseline in number of days per week of PS usage at end of treatment was reported.
Plasma citrulline was measured as an assessment of enterocyte mass. Absolute change from baseline in plasma citrulline levels at end of treatment was reported.
Plasma citrulline was measured as an assessment of enterocyte mass. Percent change in plasma citrulline from baseline at end of treatment was reported.
Percent change in PN/IV from the Baseline Visit to Week 12 Visit.
Percent change in PN/IV from the Baseline Visit to End of Treatment Visit.
Percent change in PN/IV from the Baseline Visit to Week 16 Visit.
Absolute change in PN/IV from the Baseline Visit to Week 12 Visit.
Absolute change in PN/IV from the Baseline Visit to End of Treatment Visit.
Absolute change in PN/IV from the Baseline Visit to Week 16 Visit.
As the primary intent of this study was to collect additional safety data, this outcomes measure will provide a summary of the treatment emergent adverse events. Based on the start date of each subject in this study and the study end date, not all subjects reached 12 months.
The mean change from baseline in weekly PN.IV volume in percent change is shown by visit.
The mean change from baseline in weekly PN.IV volume in Liters is shown by visit.
Comparison of subjects treated with teduglutide to placebo who achieve a 20 to 100% reduction from baseline in weekly parenteral nutrition/intravenous fluid (PN/I.V.) volume at weeks 20 and 24.
For those subjects who received teduglutide (0.05 or 0.10 mg dose) in Study 004 and the same in Study 005, Parenteral Nutrition (PN) Use at Week 28 was compared to the Baseline Visit of Study 004 to calculate the 20% reduction in PN Use. For those subjects who received placebo in Study 004 and either teduglutide 0.05 or 0.10 mg dose in Study 005, PN Use at Week 28 was compared to the use of PN at Week 24 of Study 004 to calculate the 20% reduction in PN Use.
The intensity of the response relied on a reduction from Baseline in weekly parenteral nutrition (PN) volume (minimum reduction of 20% and a maximum of 100%). Duration of the response incorporated responses at Weeks(Wk) 16-20 and at Wk20-24. Zero (0 - lowest) assigned if \<20% reduction at Wk20-24 and reduction at Wk16-20 of \< 20%, 20-39%, or \>=40%. One (1) assigned if reduction of 20-39% at Wk20-24 but \< 20% at Wk16-20. Two(2) assigned if reductions of 40-99% at Wk20-24 AND \<20% at Wk16-20 OR 20-39% at Wk20-24 AND 20-39% at Wk16-20. Three (3) assigned if reductions of 100% at Wk20-24 AND \<20% at Wk16-20 OR 40-99% at Wk20-24 AND 20-39% at Wk16-20 OR 20-39% at Wk20-24 AND \>=40% at Wk16-20. Four (4) assigned if reductions of 100% at Wk20-24 AND 20-39% at Wk16-20 OR 40-99% at Wk20-24 AND \>=40% at Wk16-20.
Percent fistula volume will be reported from the participant
Cmax is the peak plasma concentration of a drug after administration, obtained directly from the plasma concentration-time curve.
The primary objective of this study is to assess the effects of 10 consecutive, daily, subcutaneous doses of 4 mg of teduglutide as compared with placebo on gastric emptying as assessed by acetaminophen absorption kinetics in healthy subjects.
AUC0-last of Teduglutide will be evaluated.
Cmax of Teduglutide will be evaluated.
t1/2 of Teduglutide will be evaluated.
| Arm | Type | Description |
|---|---|---|
| Teduglutide 0.05 milligram per kilogram (mg/kg) | EXPERIMENTAL | Participants will receive teduglutide 0.05 mg/kg subcutaneous (SC) injection, once daily into 1 of the 4 quadrants of the abdomen or either thigh or arm for 24 weeks. |
| Teduglutide | EXPERIMENTAL | Participants will receive 0.05 milligram per kilogram (mg/kg) subcutaneous (SC) injection of teduglutide into abdomen or into either the thigh or arm once daily (QD) in addition to standard medical therapy for 24 weeks. |
| Standard of Care (SOC) | OTHER | Participants will receive standard medical therapy for 24 weeks. |
| Teduglutide 0.05 mg | 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 until Teduglutide is commercially available, the participant's participation in this study is discontinued, or the study is discontinued. |
| Standard of Care (SOC) Treatment +/- Teduglutide | EXPERIMENTAL | TED 0.05mg/kg subcutaneous injections once daily as needed in addition to SOC treatment |
| 0.025 mg/kg/day Teduglutide | EXPERIMENTAL | 0.025 milligrams per kilogram per day (mg/kg/day) of teduglutide for 24 weeks. |
| 0.05 mg/kg/day Teduglutide | EXPERIMENTAL | 0.05 mg/kg/day of teduglutide for 24 weeks. |
| Standard of care | ACTIVE_COMPARATOR | Observational cohort for the 24-week treatment period and 4 week follow-up. The subjects in the standard of care group will follow the same visit schedule as the randomized subjects. |
| Arm 1 | EXPERIMENTAL | 0.05 mg/kg/day administered by subcutaneous injection over a 24-week period. |
| Placebo | PLACEBO_COMPARATOR | Matching subcutaneous dose of placebo to teduglutide |
| teduglutide 0.05 mg/kg/d | EXPERIMENTAL | 0.05 mg/kg/d teduglutide subcutaneous injection |
| teduglutide 0.10 mg/kg/d | EXPERIMENTAL | 0.10 mg/kg/d teduglutide subcutaneous injection |
| 2 | EXPERIMENTAL | teduglutide 0.05 mg/kg/d |
| 3 | EXPERIMENTAL | teduglutide 0.1 mg/kg/d |
| Standard of Care followed by Teduglutide | EXPERIMENTAL | Participants in this group will receive standard of care treatment for the first 8 weeks followed by daily Teduglutide for the next 8 weeks. Subjects with a favorable response will be offered an open label extension during which they will continue to receive teduglutide for up to another 12 months or permanent fistula closure. |
| Teduglutide followed by Standard of Care | EXPERIMENTAL | Participants in this group will receive daily Teduglutide treatment for the first 8 weeks followed by standard of care for the next 8 weeks. Subjects with a favorable response will be offered an open label extension during which they will continue to receive teduglutide for up to another 12 months or permanent fistula closure |
| 1 | EXPERIMENTAL | All subjects in the study dosed at 0.1 mg/kg teduglutide |
| Cohort 1 (Teduglutide 3 mg): Treatment A1, Then Treatment B1 (A1B1) | EXPERIMENTAL | Teduglutide, 3 mg, subcutaneous (SC) injection using a syringe, once on Day 1 of Treatment Period 1 (Treatment A1), followed by same dose SC pen injector, once on Day 1 of Treatment Period 2 (Treatment B1). A Washout Period of 7 days was maintained between Treatment Periods 1 and 2. Participants with \>=40.0 kg to \<=75.0 kg of weight were included in Cohort 1. |
| Cohort 1 (Teduglutide 3 mg): Treatment B1, Then Treatment A1 (B1A1) | EXPERIMENTAL | Teduglutide, 3 mg, SC pen injector, once on Day 1 of Treatment Period 1 (Treatment B1), followed by same dose SC injection using a syringe, once on Day 1 of Treatment Period 2 (Treatment A1). A Washout Period of 7 days was maintained between the Treatment Periods 1 and 2. Participants with \>=40.0 kg to \<=75.0 kg of weight were included in Cohort 1. |
| Cohort 2 (Teduglutide 4 mg): Treatment A2, Then Treatment B2 (A2B2) | EXPERIMENTAL | Teduglutide, 4 mg, SC injection using a syringe, once on Day 1 of Treatment Period 1 (Treatment A2), followed by same dose SC pen injector, once on Day 1 of Treatment Period 2 (Treatment B2). A Washout Period of 7 days was maintained between Treatment Periods 1 and 2. Participants \>75.0 kg to \<=120.0 kg of weight were included in Cohort 2. |
| Cohort 2 (Teduglutide 4 mg): Treatment B2, Then Treatment A2 (B2A2) | EXPERIMENTAL | Teduglutide, 4 mg, SC pen injector, once on Day 1 of Treatment Period 1 (Treatment B2), followed by same dose SC injection using a syringe, once on Day 1 of Treatment Period 2 (Treatment A2). A Washout Period of 7 days was maintained between Treatment Periods 1 and 2. Participants \>75.0 kg to \<=120.0 kg of weight were included in Cohort 2. |
| Participants with Moderate Hepatic Impairment | EXPERIMENTAL | Participants with moderate hepatic impairment (Child-Pugh score of 7-9) will receive 20 mg of teduglutide. |
| Healthy Volunteers | ACTIVE_COMPARATOR | Healthy volunteers with normal hepatic function matched to hepatic impaired participants by age, gender, BMI, and renal function as measured by creatinine will receive 20 mg of teduglutide. |
| cohort A | OTHER | 20 mg dose 20 mg/ML concentration and placebo |
| Cohort AR | OTHER | 20 mg in 50 mg/ml concentration and placebo |
| Cohort C | OTHER | 25 mg in 50mg/ml concentration and placebo |
| Cohort D | OTHER | 15 mg in 50mg/ml concentration and placebo |
| Cohort E | OTHER | 10 mg in 50mg/ml concentration and placebo |
| Cohort F | OTHER | 30mg in 50mg/ml concentration and placebo |
| Cohort H | OTHER | 50mg in 50mg/ml concentration and placebo |
| cohort I | OTHER | 80mg in 50mg/ml concentration and placebo |
| Name | Type | Description |
|---|---|---|
| Teduglutide | DRUG | Teduglutide 0.05 mg/kg SC injection. |
| Standard Medical Therapy | OTHER | Standard medical therapy will be administered for 24 weeks. |
| Syringe | DEVICE | Teduglutide will be administered using syringe (510k number: K980987). |
| Needle | DEVICE | Teduglutide will be administered using needle (510k number: K021475). |
| Vial Adapter for Device | DEVICE | Vial adapter for device is approved for use in Japan by PMDA. |
| SOC | OTHER | Standard safety assessments and adjustments in nutritional support will be provided for all subjects throughout the study, including any teduglutide treatment periods |
| Teduglutide 0.05mg/kg | DRUG | 0.05 mg/kg |
| Teduglutide 0.025 mg/kg | DRUG | 0.025 mg/kg |
| Standard of Care | OTHER | Observational cohort for the 24-week treatment period and 4 week follow-up. |
| placebo | DRUG | Matching daily subcutaneous dose of placebo to teduglutide for 24 weeks |
| teduglutide 0.05 mg/kg/d | DRUG | 0.05 mg/kg/d subcutaneous injection |
| teduglutide 0.10 mg/kg/d | DRUG | 0.10 mg/kg/d subcutaneous injection |
| Teduglutide 0.1 mg/kg/d | DRUG | Teduglutide 0.1 /g/kg/d daily injection subcutaneously into thigh or abdomen |
| Teduglutide (ALX-0600) | DRUG | 0.1 mg/kg injected subcutaneously daily |
| Syringe Injection | DEVICE | Teduglutide was administered using syringe. |
| Pen injector | DEVICE | Teduglutide was administered using pen injector. |
| tedguglutide | DRUG | lypholized powder reconstituted with sterile water and injection subcutaneously once a day in the abdomen. |
Inclusion Criteria: 1. Males or females 18 years of age or older at the time of signing the informed consent. 2. Intestinal failure due to SBS as a consequence of major intestinal resection (example, due to injury, volvulus, vascular disease, cancer, Crohn's disease). 3. Has undergone intestinal re...
| Company | Ticker | Trials | Lead Phase | Drugs |
|---|---|---|---|---|
| Takeda Pharmaceutical Co. Ltd. Sponsored ADR | TAK | 5 | PHASE3 | Teduglutide |
| Jaguar Health, Inc. | JAGX | 1 | PHASE2 | Crofelemer for |
| Smith & Nephew plc Sponsored ADR | SNN | 1 | - | Undisclosed |
Teduglutide is being studied for use in Crohn's Disease, Short Bowel Syndrome, Postoperative Fistula, and for pharmacokinetics and safety of elevated doses in Healthy Volunteers and Hepatic Impairment. It is a gastrointestinal therapeutic developed by Takeda Pharmaceutical Company Limited and is currently in Phase 2 clinical development.
Teduglutide targets the GLP2R receptor and acts as an agonist. This means it binds to and activates the glucagon-like peptide-2 receptor, which is involved in intestinal growth and function. The drug is being investigated for its potential effects in gastrointestinal conditions such as Short Bowel Syndrome and Crohn's Disease.
Teduglutide is developed by Takeda Pharmaceutical Company Limited, which trades under the ticker TAK. The company is conducting clinical trials for this investigational drug across multiple gastrointestinal indications, including Short Bowel Syndrome and Crohn's Disease.
Teduglutide is in Phase 2 clinical development. It is an investigational drug and has not been approved by the FDA. Across all studies, there have been 16 total trials, with 1 active and 15 completed, involving 541 participants total.
Teduglutide has been studied in several clinical trials, including NCT00819468, a Phase 1 pharmacokinetics study in hepatic impairment; NCT02682381, a Phase 3 study in children with Short Bowel Syndrome; NCT03596164, an extension study in Japanese adults; and NCT05027308, a study in Japanese children with Short Bowel Syndrome.
Teduglutide is a GLP2R agonist, meaning it activates the GLP2R receptor. It is not the same as the receptor itself; rather, it is a drug that targets and stimulates this receptor. This mechanism is being explored for treating gastrointestinal conditions like Short Bowel Syndrome.