The main aims of the study are to check for side effects from teduglutide. Participants will receive a daily injection of teduglutide just under the skin (subcutaneous) for 24 weeks. Then they are followed up for another 4 weeks. Participants may be able to repeat this treatment if they meet specific criteria. The study doctors will check for side effects from teduglutide until it becomes commercially available. The maximum duration of treatment is approximately 51.3 weeks.
A study of teduglutide in Japanese children with short bowel syndrome aged 4 months or older.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
3
Teduglutide 0.05 mg/kg SC injection
University of Tsukuba Hospital
Tsukuba, Ibaraki, Japan
Tohoku University Hospital
Sendai, Miyagi, Japan
Showa University Hospital
Shinagawa-ku, Tokyo, Japan
Akita University Hospital
Akita, Japan
Number of Participants With Treatment-emergent Adverse Events (TEAEs)
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.
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})
Number of Participants With Serious Adverse Events (SAEs)
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.
Time frame: From first dose of study drug until follow-up visit (4 weeks after EOT/ET [up to 47.3-51.3 weeks])
Number of Participants With Adverse Events of Special Interest (AESIs)
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.
Time frame: From first dose of study drug until follow-up visit (4 weeks after EOT/ET [up to 47.3-51.3 weeks])
Number of Participants With Clinically Significant Abnormalities in Vital Signs Reported as an Adverse Event
Vital signs include systolic and diastolic blood pressure, heart rate and body temperature.
Time frame: From first dose of study drug until follow-up visit (4 weeks after EOT/ET [up to 47.3-51.3 weeks])
This platform is for informational purposes only and does not constitute medical advice. Always consult a qualified healthcare professional.
Kyushu University Hospital
Fukuoka, Japan
Kagoshiha University Hospital
Kagoshima, Japan
Change From Baseline in Body Weight Z-Score at EOT
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.
Time frame: Baseline, EOT (up to 47.3-51.3 weeks)
Change From Baseline in Height Z-Score at EOT
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.
Time frame: Baseline, EOT (up to 47.3-51.3 weeks)
Change From Baseline in Head Circumference Z-Score at EOT
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.
Time frame: Baseline, EOT (up to 47.3-51.3 weeks)
Change From Baseline in Weight-for-Length Z-Score at EOT
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).
Time frame: Baseline, EOT (up to 47.3-51.3 weeks)
Number of Participants With Any Laboratory Safety Finding Reported as an Adverse Event
Laboratory safety parameters included biochemistry, hematology, and urinalysis.
Time frame: From first dose of study drug until follow-up visit (4 weeks after EOT/ET [up to 47.3-51.3 weeks])
Number of Participants With a Change in Urine Output Reported as an Adverse Event
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.
Time frame: From first dose of study drug until follow-up visit (4 weeks after EOT/ET [up to 47.3-51.3 weeks])
Number of Participants With a Change in Stool Output Reported as an Adverse Event
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.
Time frame: From first dose of study drug until follow-up visit (4 weeks after EOT/ET [up to 47.3-51.3 weeks])
Change From Baseline in PS Volume
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.
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
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.
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]
Number of Participants Who Demonstrate at Least 20 Percent (%) Reduction From Baseline in PS Volume
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.
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]
Number of Participants Who Achieved Enteral Autonomy
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.
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]
Change From Baseline in Number of Days Per Week of PS Usage at EOT
PS (parenteral nutrition or intravenous fluids) was considered for managing nutritional support in terms of volume and calories during the treatment period.
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]