Back to resultsLong-term Study of Teduglutide in Pediatric Subjects With Short Bowel Syndrome Who Completed the TED-C13-003 Study
Primary Purpose
Short Bowel Syndrome
Status
Completed
Phase
Phase 3
Locations
International
Study Type
Interventional
Intervention
Teduglutide
SOC
Sponsored by
About this trial
This is an interventional treatment trial for Short Bowel Syndrome
Eligibility Criteria
Inclusion Criteria:
- Subject provides written informed consent (subject, parent or legal guardian and, as appropriate, subject informed assent) to participate in the study before completing any study-related procedures.
- Subject completed the TED-C13-003 study (including subjects in the standard of care treatment arm).
- Subject understands and is willing and able to fully adhere to study requirements as defined in this protocol.
Exclusion Criteria:
There are no exclusion criteria for this study.
Sites / Locations
- Childrens Hospital Los Angeles - RHU
- Mattel Children's Hospital UCLA
- Riley Hospital for Children
- Boston Children's Hospital
- Children's Mercy Hospitals and Clinics
- Children's Hospital at Montefiore
- Cincinnati Children's Hospital Medical Center
- Texas Children's Hospital
- Seattle Children's Hospital
- University of Wisconsin School of Medicine and Public Health
- Great Ormond Children's Hospital
Arms of the Study
Arm 1
Arm Type
Experimental
Arm Label
Standard of Care (SOC) Treatment +/- Teduglutide
Arm Description
TED 0.05mg/kg subcutaneous injections once daily as needed in addition to SOC treatment
Outcomes
Primary Outcome Measures
Number of Participants With Adverse Events (AEs) in Retrospective Observation Period
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.
Change From Baseline in Height for Age Z-score up to Week 168 of Retrospective Observation Period
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.
Change From Baseline in Body Weight for Age Z-score up to Week 168 of Retrospective Observation Period
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.
Change From Baseline in Body Mass Index (BMI) for Age Z-score up to Week 168 of Retrospective Observation Period
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.
Number of Participants With Treatment Emergent Adverse Events (TEAEs) of Prospective Study Period
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.
Change From Baseline in Height for Age Z-score up to Cycle 6 Week 12 During the End of Teduglutide Treatment Period of Prospective Study Period
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.
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
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.
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
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 48-Hour Urine Output up to Cycle 6 Week 12 During the End of Teduglutide Treatment Period of Prospective Study Period
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 48-Hour Urine Output up to Week 108 During the End of Non-Teduglutide Treatment (NTT) Period of Prospective Study Period
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 up to Cycle 6 Week 24 During the End of Teduglutide Treatment Period of Prospective Study Period
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 up to Week 120 During the End of NTT Period of Prospective Study Period
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 at EOS During the End of Teduglutide Treatment Period of Prospective Study Period
Number of participants with positive specific antibodies to teduglutide were used to summarize the presence of antibodies.
Secondary Outcome Measures
Number of Participants Achieved At Least 20 Percent (%) Reduction in Parenteral Support (PS) Volume at 12 Weeks Interval up to Week 156 of Retrospective Observation Period
Number of participants achieved at least 20% reduction in PS volume at 12 weeks interval up to Week 156 in retrospective observation period was reported.
Change From Baseline in PS Volume at 12 Weeks Interval up to Week 156 of Retrospective Observation Period
Change from baseline in PS volume at 12 weeks interval up to Week 156 in retrospective observation period was reported.
Percent Change From Baseline in PS Volume at 12 Weeks Interval up to Week 156 of Retrospective Observation Period
Percent change from baseline in PS volume at 12 weeks interval up to Week 156 of retrospective observation period was reported.
Change From Baseline in PS Caloric Intake at 12 Weeks Interval up to Week 156 of Retrospective Observation Period
Change from baseline in PS caloric intake at 12 weeks interval up to Week 156 of retrospective observation period was reported. Here, kilo-calories per kilogram per day was abbreviated as (kcal/kg/day).
Percent Change From Baseline in PS Caloric Intake at 12 Weeks Interval up to Week 156 of Retrospective Observation Period
Percent change from baseline in PS caloric intake at 12 weeks interval up to Week 156 of retrospective observation period was reported.
Change From Baseline in Number of Hours Per Day of PS Usage at 12 Weeks Interval up to Week 156 of Retrospective Observation Period
Data for this outcome measure was not analyzed and collected because changes from baseline for prescribed hours per day were not collected at baseline in the core study.
Percent Change From Baseline in Number of Hours Per Day of PS Usage at 12 Weeks Interval up to Week 156 of Retrospective Observation Period
Data for this outcome measure was not analyzed and collected because changes from baseline for prescribed hours per day were not collected at baseline in the core study. Hence, percent change was not assessed in this study.
Change From Baseline in Number of Days Per Week of PS Usage at 12 Weeks Interval up to Week 156 of Retrospective Observation Period
Change from baseline in number of days per Week of PS usage at 12 weeks interval up to Week 156 in retrospective observation period was reported.
Percent Change From Baseline in Number of Days Per Week of PS Usage at 12 Weeks Interval up to Week 156 of Retrospective Observation Period
Percent change from baseline in number of days per Week of PS usage at 12 weeks interval up to Week 156 in retrospective observation period was reported.
Number of Participants Who Achieved At Least 20, 50 and 75% Reduction in PS Volume at EOT of Each Cycle During the End of Teduglutide Treatment Period of Prospective Study Period
Number of participants who achieved at least 20, 50 and 75% reduction in PS volume at EOT of each cycle during the end of teduglutide treatment period in prospective study period was reported.
Change From Baseline in PS Volume at EOT of Each Cycle During the End of Teduglutide Treatment Period of Prospective Study Period
Change from baseline in PS volume at EOT of each cycle during the end of teduglutide treatment period in prospective study period was reported.
Percent Change From Baseline in PS Volume at EOT of Each Cycle During the End of Teduglutide Treatment Period of Prospective Study Period
Percent change from baseline in PS volume at EOT of each cycle during the end of teduglutide treatment period in prospective study period.
Change From Baseline in PS Caloric Intake at EOT of Each Cycle During the End of Teduglutide Treatment Period of Prospective Study Period
Change from baseline in PS caloric intake at EOT of each cycle during the end of teduglutide treatment period in prospective study period was reported.
Percent Change From Baseline in PS Caloric Intake at EOT of Each Cycle During the End of Teduglutide Treatment Period of Prospective Study Period
Percent change from baseline in PS caloric intake at EOT of each cycle during the end of teduglutide treatment period in prospective study period was reported.
Number of Participants Who Achieved 100% Reduction in Complete Weaning of PS Volume at EOT of Each Cycle During the End of Teduglutide Treatment Period of Prospective Study Period
Number of participants who achieved at least 100% reduction in complete weaning of PS volume at EOT of each cycle during the end of teduglutide treatment period in prospective study period was reported.
Change From Baseline in Number of Hours Per Day of PS Usage at EOT of Each Cycle During the End of Teduglutide Treatment Period of Prospective Study Period
Change from baseline in number of hours per day of PS usage at EOT of each cycle during the end of teduglutide treatment period in prospective study period was reported.
Percent Change From Baseline in Number of Hours Per Day of PS Usage at EOT of Each Cycle During the End of Teduglutide Treatment Period of Prospective Study Period
Percent change from baseline in number of hours per day of PS usage at EOT of each cycle during the end of teduglutide treatment period in prospective study period was reported.
Change From Baseline in Number of Days Per Week of PS Usage at EOT of Each Cycle During the End of Teduglutide Treatment Period of Prospective Study Period
Change from baseline in number of days per week of PS usage at EOT of each cycle during the end of teduglutide treatment period in prospective study period was reported.
Percent Change From Baseline in Number of Days Per Week of PS Usage at EOT of Each Cycle During the End of Teduglutide Treatment Period of Prospective Study Period
Percent change from baseline in number of days per week of PS usage at EOT of each cycle during the end of teduglutide treatment period in prospective study period was reported.
Full Information
1. Study Identification
Unique Protocol Identification Number
NCT02949362
Brief Title
Long-term Study of Teduglutide in Pediatric Subjects With Short Bowel Syndrome Who Completed the TED-C13-003 Study
Official Title
A Retrospective and Prospective, Open-label, Long-term Safety and Efficacy Study of Teduglutide in Pediatric Subjects With Short Bowel Syndrome Who Completed TED-C13-003
Study Type
Interventional
2. Study Status
Record Verification Date
May 2021
Overall Recruitment Status
Completed
Study Start Date
December 9, 2016 (Actual)
Primary Completion Date
July 14, 2020 (Actual)
Study Completion Date
July 14, 2020 (Actual)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Shire
4. Oversight
Data Monitoring Committee
Yes
5. Study Description
Brief Summary
The purpose of this study is to evaluate the safety and efficacy of teduglutide treatment of children with short bowel syndrome (SBS) who completed the TED-C13-003 study over a long-term period. It will evaluate how these children fared after the TED-C13-003 study ended. This study will also offer teduglutide treatment to eligible subjects, regardless of treatment received in TED-C13-003.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Short Bowel Syndrome
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 3
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
29 (Actual)
8. Arms, Groups, and Interventions
Arm Title
Standard of Care (SOC) Treatment +/- Teduglutide
Arm Type
Experimental
Arm Description
TED 0.05mg/kg subcutaneous injections once daily as needed in addition to SOC treatment
Intervention Type
Drug
Intervention Name(s)
Teduglutide
Intervention Description
0.05mg/kg
Intervention Type
Other
Intervention Name(s)
SOC
Intervention Description
Standard safety assessments and adjustments in nutritional support will be provided for all subjects throughout the study, including any teduglutide treatment periods
Primary Outcome Measure Information:
Title
Number of Participants With Adverse Events (AEs) in Retrospective Observation Period
Description
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.
Time Frame
From end of the core study (TED-C13-003 [NCT01952080]) up to the beginning of the prospective period (up to Week 168)
Title
Change From Baseline in Height for Age Z-score up to Week 168 of Retrospective Observation Period
Description
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.
Time Frame
Baseline (Baseline visit in the core study [TED-C13-003 (NCT01952080)]) up to the beginning of the prospective period (up to Week 168)
Title
Change From Baseline in Body Weight for Age Z-score up to Week 168 of Retrospective Observation Period
Description
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.
Time Frame
Baseline (Baseline visit in the core study [TED-C13-003 (NCT01952080)]) up to the beginning of the prospective period (up to Week 168)
Title
Change From Baseline in Body Mass Index (BMI) for Age Z-score up to Week 168 of Retrospective Observation Period
Description
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.
Time Frame
Baseline (Baseline visit in the core study [TED-C13-003 (NCT01952080)]) up to the beginning of the prospective period (up to Week 168)
Title
Number of Participants With Treatment Emergent Adverse Events (TEAEs) of Prospective Study Period
Description
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.
Time Frame
From the beginning of the prospective study period to End of Study (EOS) (up to Week 144)
Title
Change From Baseline in Height for Age Z-score up to Cycle 6 Week 12 During the End of Teduglutide Treatment Period of Prospective Study Period
Description
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.
Time Frame
Baseline (from the beginning of the prospective study period) up to Cycle 6 Week 12
Title
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
Description
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.
Time Frame
Baseline (from the beginning of the prospective study period) up to Cycle 6 Week 24
Title
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
Description
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.
Time Frame
Baseline (from the beginning of the prospective study period) up to Cycle 6 Week 12
Title
Average Total 48-Hour Urine Output up to Cycle 6 Week 12 During the End of Teduglutide Treatment Period of Prospective Study Period
Description
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.
Time Frame
Baseline (from the beginning of the prospective study period) up to Cycle 6 Week 12
Title
Average Total 48-Hour Urine Output up to Week 108 During the End of Non-Teduglutide Treatment (NTT) Period of Prospective Study Period
Description
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.
Time Frame
Baseline (from the beginning of the prospective study period) up to Week 108
Title
Average Number of Stools Per Day up to Cycle 6 Week 24 During the End of Teduglutide Treatment Period of Prospective Study Period
Description
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.
Time Frame
Baseline (from the beginning of the prospective study period) up to Cycle 6 Week 24
Title
Average Number of Stools Per Day up to Week 120 During the End of NTT Period of Prospective Study Period
Description
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.
Time Frame
Baseline (from the beginning of the prospective study period) up to Week 120
Title
Number of Participants With Positive Specific Antibodies at EOS During the End of Teduglutide Treatment Period of Prospective Study Period
Description
Number of participants with positive specific antibodies to teduglutide were used to summarize the presence of antibodies.
Time Frame
From the beginning of the prospective study period, EOS (up to Week 156)
Secondary Outcome Measure Information:
Title
Number of Participants Achieved At Least 20 Percent (%) Reduction in Parenteral Support (PS) Volume at 12 Weeks Interval up to Week 156 of Retrospective Observation Period
Description
Number of participants achieved at least 20% reduction in PS volume at 12 weeks interval up to Week 156 in retrospective observation period was reported.
Time Frame
At Weeks 12, 24, 36, 48, 60, 72, 84, 96, 108, 120, 132, 144, and 156
Title
Change From Baseline in PS Volume at 12 Weeks Interval up to Week 156 of Retrospective Observation Period
Description
Change from baseline in PS volume at 12 weeks interval up to Week 156 in retrospective observation period was reported.
Time Frame
Baseline (Baseline visit in the core study [TED-C13-003 (NCT01952080)]), Weeks 12, 24, 36, 48, 60, 72, 84, 96, 108, 120, 132, 144, and 156
Title
Percent Change From Baseline in PS Volume at 12 Weeks Interval up to Week 156 of Retrospective Observation Period
Description
Percent change from baseline in PS volume at 12 weeks interval up to Week 156 of retrospective observation period was reported.
Time Frame
Baseline (Baseline visit in the core study [TED-C13-003 (NCT01952080)]), Weeks 12, 24, 36, 48, 60, 72, 84, 96, 108, 120, 132, 144, and 156
Title
Change From Baseline in PS Caloric Intake at 12 Weeks Interval up to Week 156 of Retrospective Observation Period
Description
Change from baseline in PS caloric intake at 12 weeks interval up to Week 156 of retrospective observation period was reported. Here, kilo-calories per kilogram per day was abbreviated as (kcal/kg/day).
Time Frame
Baseline (Baseline visit in the core study [TED-C13-003 (NCT01952080)]), Weeks 12, 24, 36, 48, 60, 72, 84, 96, 108, 120, 132, 144, and 156
Title
Percent Change From Baseline in PS Caloric Intake at 12 Weeks Interval up to Week 156 of Retrospective Observation Period
Description
Percent change from baseline in PS caloric intake at 12 weeks interval up to Week 156 of retrospective observation period was reported.
Time Frame
Baseline (Baseline visit in the core study [TED-C13-003 (NCT01952080)]), Weeks 12, 24, 36, 48, 60, 72, 84, 96, 108, 120, 132, 144, and 156
Title
Change From Baseline in Number of Hours Per Day of PS Usage at 12 Weeks Interval up to Week 156 of Retrospective Observation Period
Description
Data for this outcome measure was not analyzed and collected because changes from baseline for prescribed hours per day were not collected at baseline in the core study.
Time Frame
Baseline (Baseline visit in the core study [TED-C13-003 (NCT01952080)]) up to Week 156
Title
Percent Change From Baseline in Number of Hours Per Day of PS Usage at 12 Weeks Interval up to Week 156 of Retrospective Observation Period
Description
Data for this outcome measure was not analyzed and collected because changes from baseline for prescribed hours per day were not collected at baseline in the core study. Hence, percent change was not assessed in this study.
Time Frame
Baseline (Baseline visit in the core study [TED-C13-003 (NCT01952080)]) up to Week 156
Title
Change From Baseline in Number of Days Per Week of PS Usage at 12 Weeks Interval up to Week 156 of Retrospective Observation Period
Description
Change from baseline in number of days per Week of PS usage at 12 weeks interval up to Week 156 in retrospective observation period was reported.
Time Frame
Baseline (Baseline visit in the core study [TED-C13-003 (NCT01952080)]), Weeks 12, 24, 36, 48, 60, 72, 84, 96, 108, 120, 132, 144, and 156
Title
Percent Change From Baseline in Number of Days Per Week of PS Usage at 12 Weeks Interval up to Week 156 of Retrospective Observation Period
Description
Percent change from baseline in number of days per Week of PS usage at 12 weeks interval up to Week 156 in retrospective observation period was reported.
Time Frame
Baseline (Baseline visit in the core study [TED-C13-003 (NCT01952080)]), Weeks 12, 24, 36, 48, 60, 72, 84, 96, 108, 120, 132, 144, and 156
Title
Number of Participants Who Achieved At Least 20, 50 and 75% Reduction in PS Volume at EOT of Each Cycle During the End of Teduglutide Treatment Period of Prospective Study Period
Description
Number of participants who achieved at least 20, 50 and 75% reduction in PS volume at EOT of each cycle during the end of teduglutide treatment period in prospective study period was reported.
Time Frame
At EOT of each Cycles 1 to 6 (up to Week 140) (length of each Cycle 1 to 5 = 24 weeks, length of Cycle 6 = 20 weeks)
Title
Change From Baseline in PS Volume at EOT of Each Cycle During the End of Teduglutide Treatment Period of Prospective Study Period
Description
Change from baseline in PS volume at EOT of each cycle during the end of teduglutide treatment period in prospective study period was reported.
Time Frame
Baseline (from the beginning of the prospective study period), EOT of each Cycles 1 to 6 (up to Week 136) (length of each Cycle 1 to 5 = 24 weeks, length of Cycle 6 = 16 weeks)
Title
Percent Change From Baseline in PS Volume at EOT of Each Cycle During the End of Teduglutide Treatment Period of Prospective Study Period
Description
Percent change from baseline in PS volume at EOT of each cycle during the end of teduglutide treatment period in prospective study period.
Time Frame
Baseline (from the beginning of the prospective study period), EOT of each Cycles 1 to 6 (up to Week 136) (length of each Cycle 1 to 5 = 24 weeks, length of Cycle 6 = 16 weeks)
Title
Change From Baseline in PS Caloric Intake at EOT of Each Cycle During the End of Teduglutide Treatment Period of Prospective Study Period
Description
Change from baseline in PS caloric intake at EOT of each cycle during the end of teduglutide treatment period in prospective study period was reported.
Time Frame
Baseline (from the beginning of the prospective study period), EOT of each Cycles 1 to 6 (up to Week 136) (length of each Cycle 1 to 5 = 24 weeks, length of Cycle 6 = 16 weeks)
Title
Percent Change From Baseline in PS Caloric Intake at EOT of Each Cycle During the End of Teduglutide Treatment Period of Prospective Study Period
Description
Percent change from baseline in PS caloric intake at EOT of each cycle during the end of teduglutide treatment period in prospective study period was reported.
Time Frame
Baseline (from the beginning of the prospective study period), EOT of each Cycles 1 to 6 (up to Week 136) (length of each Cycle 1 to 5 = 24 weeks, length of Cycle 6 = 16 weeks)
Title
Number of Participants Who Achieved 100% Reduction in Complete Weaning of PS Volume at EOT of Each Cycle During the End of Teduglutide Treatment Period of Prospective Study Period
Description
Number of participants who achieved at least 100% reduction in complete weaning of PS volume at EOT of each cycle during the end of teduglutide treatment period in prospective study period was reported.
Time Frame
At EOT of each Cycles 1 to 6 (up to Week 144) (length of each Cycle 1 to 6 = 24 weeks)
Title
Change From Baseline in Number of Hours Per Day of PS Usage at EOT of Each Cycle During the End of Teduglutide Treatment Period of Prospective Study Period
Description
Change from baseline in number of hours per day of PS usage at EOT of each cycle during the end of teduglutide treatment period in prospective study period was reported.
Time Frame
Baseline (from the beginning of the prospective study period), EOT of each Cycles 1 to 6 (up to Week 136) (length of each Cycle 1 to 5 = 24 weeks, length of Cycle 6 = 16 weeks)
Title
Percent Change From Baseline in Number of Hours Per Day of PS Usage at EOT of Each Cycle During the End of Teduglutide Treatment Period of Prospective Study Period
Description
Percent change from baseline in number of hours per day of PS usage at EOT of each cycle during the end of teduglutide treatment period in prospective study period was reported.
Time Frame
Baseline (from the beginning of the prospective study period), EOT of each Cycles 1 to 6 (up to Week 136) (length of each Cycle 1 to 5 = 24 weeks, length of Cycle 6 = 16 weeks)
Title
Change From Baseline in Number of Days Per Week of PS Usage at EOT of Each Cycle During the End of Teduglutide Treatment Period of Prospective Study Period
Description
Change from baseline in number of days per week of PS usage at EOT of each cycle during the end of teduglutide treatment period in prospective study period was reported.
Time Frame
Baseline (from the beginning of the prospective study period), EOT of each Cycles 1 to 6 (up to Week 136) (length of each Cycle 1 to 5 = 24 weeks, length of Cycle 6 = 16 weeks)
Title
Percent Change From Baseline in Number of Days Per Week of PS Usage at EOT of Each Cycle During the End of Teduglutide Treatment Period of Prospective Study Period
Description
Percent change from baseline in number of days per week of PS usage at EOT of each cycle during the end of teduglutide treatment period in prospective study period was reported.
Time Frame
Baseline (from the beginning of the prospective study period), EOT of each Cycles 1 to 6 (up to Week 136) (length of each Cycle 1 to 5 = 24 weeks, length of Cycle 6 = 16 weeks)
Other Pre-specified Outcome Measures:
Title
Change From Baseline In Pediatric Quality of Life Inventory (PedsQL) Generic Core Scale (GCS) Score at Week 12 and 24 of Each Treatment Cycle During the Teduglutide Treatment Periods of Prospective Study Period
Description
PedsQL GCS was used for quality of life assessment. It encompasses 4 dimensions of functioning (physical, emotional, social, school). Age groups are: Toddler (2-4 years), Young child (5-7 years), Child (8-12 years), and Teens (13-18 years). Depending on the participants age, the questionnaire may be completed by either the participant or the parent/caregiver as appropriate. For the Toddler group, the PedsQL GCS consisted of 21 items, using a 5-point Likert scale (0 to 4); for all other groups, the PedsQL GCS consisted of 23 items, with a 3-point Likert scale (0, 2, 4) for the young child, and a 5-point Likert scale for the child and teens groups. Scores are transformed on a scale from 0 to 100 where 0=100, 1=75, 2=50, 3=25, and 4=0. Higher scores indicate improved quality of life.
Time Frame
Baseline (from the beginning of the prospective study period), Week 12 and 24 of Cycles 1 to 6
Title
Change From Baseline In Pediatric Quality of Life Inventory (PedsQL) Generic Core Scale (GCS) Score at End of Last NTT Period During the NTT Periods in Prospective Study Period
Description
PedsQL GCS was used for quality of life assessment. It encompasses 4 dimensions of functioning (physical, emotional, social, school). Age groups are: Toddler (2-4 years), Young child (5-7 years), Child (8-12 years), and Teens (13-18 years). Depending on the participants age, the questionnaire may be completed by either the participant or the parent/caregiver as appropriate. For the Toddler group, the PedsQL GCS consisted of 21 items, using a 5-point Likert scale (0 to 4); for all other groups, the PedsQL GCS consisted of 23 items, with a 3-point Likert scale (0, 2, 4) for the young child, and a 5-point Likert scale for the child and teens groups. Scores are transformed on a scale from 0 to 100 where 0=100, 1=75, 2=50, 3=25, and 4=0. Higher scores indicate improved quality of life.
Time Frame
Baseline (from the beginning of the prospective study period), End of Last NTT period (up to Week 120)
Title
Change From Baseline In Pediatric Quality of Life Inventory (PedsQL) Family Impact Module Total Score at Week 12 and 24 of Each Treatment Cycle During the Teduglutide Treatment Periods of Prospective Study Period
Description
PedsQL Family Impact Module was composed of 36 items comprising Physical Functioning (6 items), Emotional Functioning (5 items), Social Functioning (4 items), Cognitive Functioning (5 items), Communication (3 items), Worry (5 items), Daily Activities (3 items) and Family Relationships (5 items). Total score was calculated as the sum of all the items over the number of items answered on all the scales. Scores are transformed on a scale from 0 to 100 were 0=100, 1=75, 2=50, 3=25, and 4=0. Higher scores indicate improved quality of life.
Time Frame
Baseline (from the beginning of the prospective study period), Week 12 and 24 of Cycles 1 to 6
Title
Change From Baseline In Pediatric Quality of Life Inventory (PedsQL) Family Impact Module Total Score at End of Last NTT Period During the NTT Periods in Prospective Study Period
Description
PedsQL Family Impact Module was composed of 36 items comprising Physical Functioning (6 items), Emotional Functioning (5 items), Social Functioning (4 items), Cognitive Functioning (5 items), Communication (3 items), Worry (5 items), Daily Activities (3 items) and Family Relationships (5 items). Total score was calculated as the sum of all the items over the number of items answered on all the scales. Scores are transformed on a scale from 0 to 100 were 0=100, 1=75, 2=50, 3=25, and 4=0. Higher scores indicate improved quality of life.
Time Frame
Baseline (from the beginning of to the end the prospective study period), End of Last NTT period (up to Week 120)
Title
Change From Baseline In Pediatric Quality of Life Inventory (PedsQL) Gastrointestinal Symptoms Module at Week 12 and 24 of Each Treatment Cycle During the Teduglutide Treatment Periods of Prospective Study Period
Description
PedsQL GI symptoms module was composed of 58 items, comprised of 10 different symptom scales that assess gastrointestinal symptom-related quality of life: food and drink limits, trouble swallowing, heartburn and reflux, nausea and vomiting, gas and bloating, constipation, blood in poop, and diarrhea. Only the scales of food and drink limits (6 items) and diarrhea (7 items) was used in this study. Subscale score was calculated as the sum of the items over the number of items answered in the scale. Scores are transformed on a scale from 0 to 100 were 0=100, 1=75, 2=50, 3=25, and 4=0. Higher scores indicate improved quality of life.
Time Frame
Baseline (from the beginning of the prospective study period), Week 12 and 24 of Cycles 1 to 6
Title
Change From Baseline In Pediatric Quality of Life Inventory (PedsQL) Gastrointestinal Symptoms Module at End of Last NTT Period During the NTT Periods in Prospective Study Period
Description
PedsQL GI symptoms module was composed of 58 items, comprised of 10 different symptom scales that assess gastrointestinal symptom-related quality of life: food and drink limits, trouble swallowing, heartburn and reflux, nausea and vomiting, gas and bloating, constipation, blood in poop, and diarrhea. Only the scales of food and drink limits (6 items) and diarrhea (7 items) was used in this study. Subscale score was calculated as the sum of the items over the number of items answered in the scale. Scores are transformed on a scale from 0 to 100 were 0=100, 1=75, 2=50, 3=25, and 4=0. Higher scores indicate improved quality of life.
Time Frame
Baseline (from the beginning of the prospective study period), End of Last NTT period (up to Week 120)
10. Eligibility
Sex
All
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria:
Subject provides written informed consent (subject, parent or legal guardian and, as appropriate, subject informed assent) to participate in the study before completing any study-related procedures.
Subject completed the TED-C13-003 study (including subjects in the standard of care treatment arm).
Subject understands and is willing and able to fully adhere to study requirements as defined in this protocol.
Exclusion Criteria:
There are no exclusion criteria for this study.
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Shire Study Physician
Organizational Affiliation
Shire
Official's Role
Principal Investigator
Facility Information:
Facility Name
Childrens Hospital Los Angeles - RHU
City
Los Angeles
State/Province
California
ZIP/Postal Code
90027
Country
United States
Facility Name
Mattel Children's Hospital UCLA
City
Los Angeles
State/Province
California
ZIP/Postal Code
90095
Country
United States
Facility Name
Riley Hospital for Children
City
Indianapolis
State/Province
Indiana
ZIP/Postal Code
46202
Country
United States
Facility Name
Boston Children's Hospital
City
Boston
State/Province
Massachusetts
ZIP/Postal Code
02115
Country
United States
Facility Name
Children's Mercy Hospitals and Clinics
City
Kansas City
State/Province
Missouri
ZIP/Postal Code
64108
Country
United States
Facility Name
Children's Hospital at Montefiore
City
Bronx
State/Province
New York
ZIP/Postal Code
10467
Country
United States
Facility Name
Cincinnati Children's Hospital Medical Center
City
Cincinnati
State/Province
Ohio
ZIP/Postal Code
45229
Country
United States
Facility Name
Texas Children's Hospital
City
Houston
State/Province
Texas
ZIP/Postal Code
77030
Country
United States
Facility Name
Seattle Children's Hospital
City
Seattle
State/Province
Washington
ZIP/Postal Code
98105
Country
United States
Facility Name
University of Wisconsin School of Medicine and Public Health
City
Madison
State/Province
Wisconsin
ZIP/Postal Code
53792
Country
United States
Facility Name
Great Ormond Children's Hospital
City
London
ZIP/Postal Code
WC1N 3JH
Country
United Kingdom
12. IPD Sharing Statement
Plan to Share IPD
Yes
IPD Sharing Plan Description
Takeda provides access to the de-identified individual participant data (IPD) for eligible studies to aid qualified researchers in addressing legitimate scientific objectives (Takeda's data sharing commitment is available on https://clinicaltrials.takeda.com/takedas-commitment?commitment=5). These IPDs will be provided in a secure research environment following approval of a data sharing request, and under the terms of a data sharing agreement.
IPD Sharing Access Criteria
IPD from eligible studies will be shared with qualified researchers according to the criteria and process described on https://vivli.org/ourmember/takeda/. For approved requests, the researchers will be provided access to anonymized data (to respect patient privacy in line with applicable laws and regulations) and with information necessary to address the research objectives under the terms of a data sharing agreement.
IPD Sharing URL
https://vivli.org/ourmember/takeda/
Links:
URL
https://clinicaltrials.takeda.com/study-detail/5f6b5fd94db2bf003ab470b2
Description
To obtain more information on the study, click here/on this link
Learn more about this trial
Long-term Study of Teduglutide in Pediatric Subjects With Short Bowel Syndrome Who Completed the TED-C13-003 Study
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