A Study of Teduglutide in Japanese Children With Short Bowel Syndrome Aged 4 Months or Older

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

Time frame · From first dose of study drug until follow-up visit (4 weeks after end of treatment [EOT]/end of termination [ET] {up to 47.3-51.3 weeks})

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.

Number of Participants With Serious Adverse Events (SAEs)

Time frame · From first dose of study drug until follow-up visit (4 weeks after EOT/ET [up to 47.3-51.3 weeks])

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.

Number of Participants With Adverse Events of Special Interest (AESIs)

Time frame · From first dose of study drug until follow-up visit (4 weeks after EOT/ET [up to 47.3-51.3 weeks])

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.

Number of Participants With Clinically Significant Abnormalities in Vital Signs Reported as an Adverse Event

Time frame · From first dose of study drug until follow-up visit (4 weeks after EOT/ET [up to 47.3-51.3 weeks])

Vital signs include systolic and diastolic blood pressure, heart rate and body temperature.

Change From Baseline in Body Weight Z-Score at EOT

Time frame · Baseline, EOT (up to 47.3-51.3 weeks)

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.

Change From Baseline in Height Z-Score at EOT

Time frame · Baseline, EOT (up to 47.3-51.3 weeks)

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.

Change From Baseline in Head Circumference Z-Score at EOT

Time frame · Baseline, EOT (up to 47.3-51.3 weeks)

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.

Change From Baseline in Weight-for-Length Z-Score at EOT

Time frame · Baseline, EOT (up to 47.3-51.3 weeks)

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).

Change From Baseline in PS Volume

Time frame · Baseline, Cycle 1 = Week 1, 2, 4, 8, 12, 16, 20, 24, and EOT, Cycle 2: Week 0, 1, 2, 4, 8, 12, 16, 20, 24, and EOT, Cycle 3 = Week 0, 1, 2, and EOT and overall EOT (for up to 47.3-51.3 weeks) [cycle length=28 weeks]

PS (parenteral nutrition or intravenous fluids) was considered for managing nutritional support in terms of volume and calories during the treatment period. An end of treatment (EOT) was defined as the last determination of endpoint of the last cycle.

Percent Change From Baseline in PS Volume

Time frame · Baseline, Cycle 1 = Week 1, 2, 4, 8, 12, 16, 20, 24, and EOT, Cycle 2 = Week 0, 1, 2, 4, 8, 12, 16, 20, 24, and EOT, Cycle 3 = Week 0, 1, 2, and EOT and overall EOT (for up to 47.3-51.3 weeks) [cycle length=28 weeks]

Percent change from baseline in PS volume was calculated as follows; (PS volume at each point \[Week 1, 2, 4, 8, 12, 16, 20, 24, and EOT\] - PS volume at baseline)/ PS volume at baseline \*100 (percent). PS (parenteral nutrition or intravenous fluids) was to be considered for managing nutritional support in terms of volume and calories during the treatment period. An EOT was defined as the last determination of endpoint of the last cycle.

Number of Participants Who Demonstrate at Least 20 Percent (%) Reduction From Baseline in PS Volume

Time frame · Baseline, Cycle 1 = Week 1, 2, 4, 8, 12, 16, 20, 24, and EOT, Cycle 2 = Week 0, 1, 2, 4, 8, 12, 16, 20, 24, and EOT, Cycle 3 = Week 0, 1, 2, and EOT and overall EOT (for up to 47.3-51.3 weeks) [cycle length=28 weeks]

PS (parenteral nutrition or intravenous fluids) was considered for managing nutritional support in terms of volume and calories during the treatment period. An EOT was defined as the last determination of endpoint of the last cycle.

Number of Participants Who Achieved Enteral Autonomy

Time frame · Cycle 1 = Week 1, 2, 4, 8, 12, 16, 20, 24, and EOT, Cycle 2 = Week 0, 1, 2, 4, 8, 12, 16, 20, 24, and EOT, Cycle 3 = Week 0, 1, 2, and EOT and overall EOT (for up to 47.3-51.3 weeks) [cycle length=28 weeks]

Achieving enteral autonomy is defined as complete weaning off PS. PS (parenteral nutrition or intravenous fluids) was to be considered for managing nutritional support in terms of volume and calories during the treatment period.

Change From Baseline in Number of Days Per Week of PS Usage at EOT

Time frame · Baseline, Cycle 1 = Week 1, 2, 4, 8, 12, 16, 20, 24, and EOT, Cycle 2 = Week 0, 1, 2, 4, 8, 12, 16, 20, 24, and EOT, Cycle 3 = Week 0, 1, 2, and EOT, and overall EOT (for 47.3-51.3 weeks) [cycle length=28 weeks]

PS (parenteral nutrition or intravenous fluids) was considered for managing nutritional support in terms of volume and calories during the treatment period.