Short Bowel Syndrome
Conditions
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.
Interventions
DRUGTeduglutide
0.05mg/kg
OTHERSOC
Standard safety assessments and adjustments in nutritional support will be provided for all subjects throughout the study, including any teduglutide treatment periods
Sponsors
Shire
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Healthy volunteers
No
Inclusion criteria
1. 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. 2. Subject completed the TED-C13-003 study (including subjects in the standard of care treatment arm). 3. Subject understands and is willing and able to fully adhere to study requirements as defined in this protocol.
Exclusion criteria
There are no
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Average Number of Stools Per Day up to Week 120 During the End of NTT Period of Prospective Study Period | Baseline (from the beginning of the prospective study period) up to Week 120 | 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. |
| 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 | Baseline (from the beginning of the prospective study period) up to Cycle 6 Week 12 | 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. |
| 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 | Baseline (from the beginning of the prospective study period) up to Cycle 6 Week 24 | 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. |
| Average Number of Stools Per Day up to Cycle 6 Week 24 During the End of Teduglutide Treatment Period of Prospective Study Period | Baseline (from the beginning of the prospective study period) up to Cycle 6 Week 24 | 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 Total 48-Hour Urine Output up to Week 108 During the End of Non-Teduglutide Treatment (NTT) Period of Prospective Study Period | Baseline (from the beginning of the prospective study period) up to Week 108 | 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 Total 48-Hour Urine Output up to Cycle 6 Week 12 During the End of Teduglutide Treatment Period of Prospective Study Period | Baseline (from the beginning of the prospective study period) up to Cycle 6 Week 12 | 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. |
| Number of Participants With Positive Specific Antibodies at EOS During the End of Teduglutide Treatment Period of Prospective Study Period | From the beginning of the prospective study period, EOS (up to Week 156) | Number of participants with positive specific antibodies to teduglutide were used to summarize the presence of antibodies. |
| Number of Participants With Adverse Events (AEs) in Retrospective Observation Period | From end of the core study (TED-C13-003 [NCT01952080]) up to the beginning of the prospective period (up to Week 168) | 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 | Baseline (Baseline visit in the core study [TED-C13-003 (NCT01952080)]) up to the beginning of the prospective period (up to Week 168) | 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 | Baseline (Baseline visit in the core study [TED-C13-003 (NCT01952080)]) up to the beginning of the prospective period (up to Week 168) | 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 | Baseline (Baseline visit in the core study [TED-C13-003 (NCT01952080)]) up to the beginning of the prospective period (up to Week 168) | 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 | From the beginning of the prospective study period to End of Study (EOS) (up to Week 144) | 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 | Baseline (from the beginning of the prospective study period) up to Cycle 6 Week 12 | 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. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Change From Baseline in PS Caloric Intake at EOT of Each Cycle During the End of Teduglutide Treatment Period of Prospective Study Period | 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) | 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 | At EOT of each Cycles 1 to 6 (up to Week 144) (length of each Cycle 1 to 6 = 24 weeks) | 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 | 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) | 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 | 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) | 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 | 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) | 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 | 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) | 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. |
| Percent Change From Baseline in PS Caloric Intake at EOT of Each Cycle During the End of Teduglutide Treatment Period of Prospective Study Period | 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) | 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 Achieved At Least 20 Percent (%) Reduction in Parenteral Support (PS) Volume at 12 Weeks Interval up to Week 156 of Retrospective Observation Period | At Weeks 12, 24, 36, 48, 60, 72, 84, 96, 108, 120, 132, 144, and 156 | 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 | 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 | 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 | 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 | 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 | 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 | 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 | 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 | 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 | Baseline (Baseline visit in the core study [TED-C13-003 (NCT01952080)]) up to Week 156 | 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 | Baseline (Baseline visit in the core study [TED-C13-003 (NCT01952080)]) up to Week 156 | 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 | 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 | 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 | 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 | 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 | 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) | 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 | 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) | 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 | 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) | Percent change from baseline in PS volume at EOT of each cycle during the end of teduglutide treatment period in prospective study period. |
Other
| Measure | Time frame | Description |
|---|---|---|
| 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 | Baseline (from the beginning of the prospective study period), Week 12 and 24 of Cycles 1 to 6 | 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. |
| 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 | Baseline (from the beginning of the prospective study period), End of Last NTT period (up to Week 120) | 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. |
| 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 | Baseline (from the beginning of the prospective study period), Week 12 and 24 of Cycles 1 to 6 | 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. |
| 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 | Baseline (from the beginning of to the end the prospective study period), End of Last NTT period (up to Week 120) | 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. |
| 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 | Baseline (from the beginning of the prospective study period), Week 12 and 24 of Cycles 1 to 6 | 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. |
| 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 | Baseline (from the beginning of the prospective study period), End of Last NTT period (up to Week 120) | 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. |
Countries
United Kingdom, United States
Participant flow
Recruitment details
Participants who completed TED-C13-003 (NCT01952080) were enrolled in this study which was conducted at 11 sites in the United States and United Kingdom between 09 December 2016 (first participant first visit) and 14 July 2020 (last participant last visit).
Pre-assignment details
A total of 29 participants were enrolled in this study, 29 participants consented to retrospective period. Of these, 24 enrolled into Retro TED/NTT (did not receive teduglutide) and 5 into Retro TED/TED (received teduglutide). Out of 29 participants of retrospective period, 24 enrolled into prospective period (ANY TED group), of these, 19 in TED/TED and 5 in TED/NTT.
Participants by arm
| Arm | Count |
|---|---|
| Retrospective TED/NTT Group Participants received teduglutide 0.05 milligrams per kilogram (mg/kg) subcutaneous injection once daily in TED-C13-003 (NCT01952080) and did not receive teduglutide in retrospective period in the current study SHP633-303. | 24 |
| Retrospective TED/TED Group Participants who received teduglutide 0.05 mg/kg subcutaneous injection once daily in TED-C13-003 (NCT01952080) and also received teduglutide in retrospective period in the current study SHP633-303. | 5 |
| Total | 29 |
Withdrawals & dropouts
| Period | Reason | FG000 | FG001 | FG002 | FG003 |
|---|---|---|---|---|---|
| Prospective Period | Discontinued the study early but didn't complete an ET Visit. | 0 | 0 | 1 | 0 |
| Prospective Period | Other | 0 | 0 | 0 | 4 |
| Retrospective Period | Inform consent not signed by participant | 4 | 1 | 0 | 0 |
Baseline characteristics
| Characteristic | Retrospective TED/TED Group | Total | Retrospective TED/NTT Group |
|---|---|---|---|
| Age, Continuous | 8.2 Years STANDARD_DEVIATION 5.5 | 4.7 Years STANDARD_DEVIATION 3.71 | 4.0 Years STANDARD_DEVIATION 2.88 |
| Race/Ethnicity, Customized Ethnicity Hispanic or Latino | 1 Participants | 10 Participants | 9 Participants |
| Race/Ethnicity, Customized Ethnicity Not allowed based on local regulations | 1 Participants | 1 Participants | 0 Participants |
| Race/Ethnicity, Customized Ethnicity Not Hispanic or Latino | 3 Participants | 18 Participants | 15 Participants |
| Race/Ethnicity, Customized Race Asian | 1 Participants | 1 Participants | 0 Participants |
| Race/Ethnicity, Customized Race Black or African American | 1 Participants | 3 Participants | 2 Participants |
| Race/Ethnicity, Customized Race Other | 0 Participants | 2 Participants | 2 Participants |
| Race/Ethnicity, Customized Race White | 3 Participants | 23 Participants | 20 Participants |
| Sex: Female, Male Female | 0 Participants | 9 Participants | 9 Participants |
| Sex: Female, Male Male | 5 Participants | 20 Participants | 15 Participants |
Adverse events
| Event type | EG000 affected / at risk | EG001 affected / at risk | EG002 affected / at risk | EG003 affected / at risk |
|---|---|---|---|---|
| deaths Total, all-cause mortality | 0 / 24 | 0 / 5 | 0 / 5 | 0 / 19 |
| other Total, other adverse events | 1 / 24 | 1 / 5 | 4 / 5 | 18 / 19 |
| serious Total, serious adverse events | 23 / 24 | 4 / 5 | 3 / 5 | 17 / 19 |
Outcome results
Primary
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.
Time frame: Baseline (from the beginning of the prospective study period) up to Cycle 6 Week 24
Population: Safety population included all enrolled participants who provided informed consent for the prospective portion and met all the inclusion criteria. Here, overall number of participants analyzed signifies participants who were evaluable for this outcome measure. Data for this outcome was not planned to be collected and analyzed in prospective TED/NTT group.
| Arm | Measure | Value (MEAN) |
|---|---|---|
| Retrospective TED/NTT Group | Average Number of Stools Per Day up to Cycle 6 Week 24 During the End of Teduglutide Treatment Period of Prospective Study Period | 5.50 Stools per day |
Primary
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.
Time frame: Baseline (from the beginning of the prospective study period) up to Week 120
Population: Safety population included all enrolled participants who provided informed consent for the prospective portion and met all the inclusion criteria. Here, overall number of participants analyzed signifies participants who were evaluable for this outcome measure. Data were not planned to be collected and analyzed for the prospective TED/TED group.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Retrospective TED/NTT Group | Average Number of Stools Per Day up to Week 120 During the End of NTT Period of Prospective Study Period | 3.75 Stools per day | Standard Deviation 3.182 |
Primary
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.
Time frame: Baseline (from the beginning of the prospective study period) up to Cycle 6 Week 12
Population: Safety population included all enrolled participants who provided informed consent for the prospective portion and met all the inclusion criteria. Here, overall number of participants analyzed signifies participants who were evaluable for this outcome measure. Data were not planned to be collected and analyzed for the prospective TED/NTT group.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Retrospective TED/NTT Group | Average Total 48-Hour Urine Output up to Cycle 6 Week 12 During the End of Teduglutide Treatment Period of Prospective Study Period | 14.38 mL/kg/day | Standard Deviation 12.712 |
Primary
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.
Time frame: Baseline (from the beginning of the prospective study period) up to Week 108
Population: Safety population included all enrolled participants who provided informed consent for the prospective portion and met all the inclusion criteria. Here, overall number of participants analyzed signifies participants who were evaluable for this outcome measure. Data were not planned to be collected and analyzed for the prospective TED/TED group.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Retrospective TED/NTT Group | Average Total 48-Hour Urine Output up to Week 108 During the End of Non-Teduglutide Treatment (NTT) Period of Prospective Study Period | 22.86 mL/kg/day | Standard Deviation 9.956 |
Primary
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.
Time frame: Baseline (from the beginning of the prospective study period) up to Cycle 6 Week 12
Population: Safety population included all enrolled participants who provided informed consent for the prospective portion and met all the inclusion criteria. Here, overall number of participants analyzed signifies participants who were evaluable for this outcome measure. Data were not planned to be collected and analyzed for the prospective TED/NTT group.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Retrospective TED/NTT Group | 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 | -0.68 Z-score | Standard Deviation 0.349 |
Primary
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.
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)
Population: RETRO participants: all participants who consented to participation in this extension study and provided data for the retrospective period of the protocol. Here, overall number of participants analyzed signifies participants who were evaluable for this outcome measure. Data were not planned to be collected and analyzed for the retrospective TED/TED group.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Retrospective TED/NTT Group | Change From Baseline in Body Mass Index (BMI) for Age Z-score up to Week 168 of Retrospective Observation Period | -0.283 Z-score | Standard Deviation 0.0646 |
Primary
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.
Time frame: Baseline (from the beginning of the prospective study period) up to Cycle 6 Week 24
Population: Safety population included all enrolled participants who provided informed consent for the prospective portion and met all the inclusion criteria. Here, overall number of participants analyzed signifies participants who were evaluable for this outcome measure. Data were not planned to be collected and analyzed for the prospective TED/NTT group.
| Arm | Measure | Value (MEAN) |
|---|---|---|
| Retrospective TED/NTT Group | 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 | -0.66 Z-score |
Primary
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.
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)
Population: RETRO participants: all participants who consented to participation in this extension study and provided data for the retrospective period of the protocol. Here, overall number of participants analyzed signifies participants who were evaluable for this outcome measure. Data were not planned to be collected and analyzed for the retrospective TED/TED group.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Retrospective TED/NTT Group | Change From Baseline in Body Weight for Age Z-score up to Week 168 of Retrospective Observation Period | -0.181 Z-score | Standard Deviation 0.0601 |
Primary
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.
Time frame: Baseline (from the beginning of the prospective study period) up to Cycle 6 Week 12
Population: Safety population included all enrolled participants who provided informed consent for the prospective portion and met all the inclusion criteria. Here, overall number of participants analyzed signifies participants who were evaluable for this outcome measure. Data were not planned to be collected and analyzed for the prospective TED/NTT group.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Retrospective TED/NTT Group | 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 | 0.05 Z-score | Standard Deviation 1.068 |
Primary
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.
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)
Population: RETRO participants: all participants who consented to participation in this extension study and provided data for the retrospective period of the protocol. Here, overall number of participants analyzed signifies participants who were evaluable for this outcome measure. Data were not planned to be collected and analyzed for the retrospective TED/TED group.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Retrospective TED/NTT Group | Change From Baseline in Height for Age Z-score up to Week 168 of Retrospective Observation Period | 0.128 Z-score | Standard Deviation 0.0505 |
Primary
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.
Time frame: From end of the core study (TED-C13-003 [NCT01952080]) up to the beginning of the prospective period (up to Week 168)
Population: Retrospective participants (RETRO): all participants who consented to participation in this extension study and provided data for the retrospective period of the protocol.
| Arm | Measure | Group | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|---|
| Retrospective TED/NTT Group | Number of Participants With Adverse Events (AEs) in Retrospective Observation Period | Participants with AEs | 23 Participants |
| Retrospective TED/NTT Group | Number of Participants With Adverse Events (AEs) in Retrospective Observation Period | Participants with Related AEs | 23 Participants |
| Retrospective TED/NTT Group | Number of Participants With Adverse Events (AEs) in Retrospective Observation Period | Participants with SAEs | 23 Participants |
| Retrospective TED/NTT Group | Number of Participants With Adverse Events (AEs) in Retrospective Observation Period | Participants with Related SAE | 0 Participants |
| Retrospective TED/TED Group | Number of Participants With Adverse Events (AEs) in Retrospective Observation Period | Participants with Related SAE | 0 Participants |
| Retrospective TED/TED Group | Number of Participants With Adverse Events (AEs) in Retrospective Observation Period | Participants with AEs | 4 Participants |
| Retrospective TED/TED Group | Number of Participants With Adverse Events (AEs) in Retrospective Observation Period | Participants with SAEs | 4 Participants |
| Retrospective TED/TED Group | Number of Participants With Adverse Events (AEs) in Retrospective Observation Period | Participants with Related AEs | 4 Participants |
Primary
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.
Time frame: From the beginning of the prospective study period, EOS (up to Week 156)
Population: Safety population included all enrolled participants who provided informed consent for the prospective portion and met all the inclusion criteria. Here, overall number of participants analyzed signifies participants who were evaluable for this outcome measure. Data for this outcome was not planned to be collected and analyzed in prospective TED/NTT group.
| Arm | Measure | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|
| Retrospective TED/NTT Group | Number of Participants With Positive Specific Antibodies at EOS During the End of Teduglutide Treatment Period of Prospective Study Period | 1 Participants |
Primary
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.
Time frame: From the beginning of the prospective study period to End of Study (EOS) (up to Week 144)
Population: Safety population included all enrolled participants who provided informed consent for the prospective portion and met all the inclusion criteria.
| Arm | Measure | Group | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|---|
| Retrospective TED/NTT Group | Number of Participants With Treatment Emergent Adverse Events (TEAEs) of Prospective Study Period | Participants with TEAEs | 4 Participants |
| Retrospective TED/NTT Group | Number of Participants With Treatment Emergent Adverse Events (TEAEs) of Prospective Study Period | Participants with TESAEs | 3 Participants |
| Retrospective TED/NTT Group | Number of Participants With Treatment Emergent Adverse Events (TEAEs) of Prospective Study Period | Participants with AESI | 0 Participants |
| Retrospective TED/TED Group | Number of Participants With Treatment Emergent Adverse Events (TEAEs) of Prospective Study Period | Participants with TEAEs | 19 Participants |
| Retrospective TED/TED Group | Number of Participants With Treatment Emergent Adverse Events (TEAEs) of Prospective Study Period | Participants with TESAEs | 17 Participants |
| Retrospective TED/TED Group | Number of Participants With Treatment Emergent Adverse Events (TEAEs) of Prospective Study Period | Participants with AESI | 0 Participants |
Secondary
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.
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
Population: RETRO participants: all participants who consented to participation in this extension study and provided data for the retrospective period of the protocol. Here, number analyzed signifies participants who were evaluable for this outcome at specific time point.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Retrospective TED/NTT Group | 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 at Week 120 | -0.95 Days per week | Standard Deviation 2.459 |
| Retrospective TED/NTT Group | 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 at Week 12 | -0.38 Days per week | Standard Deviation 1.469 |
| Retrospective TED/NTT Group | 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 at Week 24 | -0.29 Days per week | Standard Deviation 1.429 |
| Retrospective TED/NTT Group | 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 at Week 36 | -0.43 Days per week | Standard Deviation 1.562 |
| Retrospective TED/NTT Group | 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 at Week 48 | -0.61 Days per week | Standard Deviation 2.017 |
| Retrospective TED/NTT Group | 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 at Week 60 | -0.61 Days per week | Standard Deviation 2.017 |
| Retrospective TED/NTT Group | 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 at Week 72 | -0.64 Days per week | Standard Deviation 2.06 |
| Retrospective TED/NTT Group | 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 at Week 84 | -0.64 Days per week | Standard Deviation 2.06 |
| Retrospective TED/NTT Group | 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 at Week 96 | -1.17 Days per week | Standard Deviation 2.665 |
| Retrospective TED/NTT Group | 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 at Week 108 | -0.88 Days per week | Standard Deviation 2.365 |
| Retrospective TED/NTT Group | 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 at Week 132 | -1.00 Days per week | Standard Deviation 2.542 |
| Retrospective TED/NTT Group | 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 at Week 144 | 0.00 Days per week | Standard Deviation 0 |
| Retrospective TED/NTT Group | 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 at Week 156 | 0.00 Days per week | Standard Deviation 0 |
| Retrospective TED/TED Group | 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 at Week 144 | -3.00 Days per week | — |
| Retrospective TED/TED Group | 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 at Week 84 | -1.40 Days per week | Standard Deviation 2.966 |
| Retrospective TED/TED Group | 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 at Week 12 | -1.20 Days per week | Standard Deviation 2.683 |
| Retrospective TED/TED Group | 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 at Week 132 | -5.25 Days per week | Standard Deviation 2.062 |
| Retrospective TED/TED Group | 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 at Week 24 | -1.00 Days per week | Standard Deviation 2 |
| Retrospective TED/TED Group | 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 at Week 96 | -2.00 Days per week | Standard Deviation 3.536 |
| Retrospective TED/TED Group | 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 at Week 36 | -2.60 Days per week | Standard Deviation 2.793 |
| Retrospective TED/TED Group | 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 at Week 156 | -3.00 Days per week | — |
| Retrospective TED/TED Group | 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 at Week 48 | -4.50 Days per week | Standard Deviation 2.082 |
| Retrospective TED/TED Group | 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 at Week 108 | -3.80 Days per week | Standard Deviation 3.701 |
| Retrospective TED/TED Group | 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 at Week 60 | -3.00 Days per week | Standard Deviation 3.536 |
| Retrospective TED/TED Group | 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 at Week 120 | -3.80 Days per week | Standard Deviation 3.701 |
| Retrospective TED/TED Group | 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 at Week 72 | -1.40 Days per week | Standard Deviation 2.966 |
Secondary
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.
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)
Population: Safety population included all enrolled participants who provided informed consent for the prospective portion and met all the inclusion criteria. Here, overall number of participants analyzed signifies participants who were evaluable for this outcome measure and number analyzed signifies participants who were evaluable at specific time point. Data were not planned to be collected and analyzed for the prospective TED/NTT group.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Retrospective TED/NTT Group | 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 at Cycle 1: EOT | -0.60 Days per week | Standard Deviation 1.892 |
| Retrospective TED/NTT Group | 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 at Cycle 2: EOT | -1.93 Days per week | Standard Deviation 2.84 |
| Retrospective TED/NTT Group | 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 at Cycle 3: EOT | -3.03 Days per week | Standard Deviation 3.257 |
| Retrospective TED/NTT Group | 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 at Cycle 4: EOT | -2.99 Days per week | Standard Deviation 3.61 |
| Retrospective TED/NTT Group | 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 at Cycle 5: EOT | -3.83 Days per week | Standard Deviation 3.969 |
| Retrospective TED/NTT Group | 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 at Cycle 6: EOT | -4.37 Days per week | Standard Deviation 3.618 |
Secondary
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.
Time frame: Baseline (Baseline visit in the core study [TED-C13-003 (NCT01952080)]) up to Week 156
Population: 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.
Secondary
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.
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)
Population: Safety population included all enrolled participants who provided informed consent for the prospective portion and met all the inclusion criteria. Here, overall number of participants analyzed signifies participants who were evaluable for this outcome measure and number analyzed signifies participants who were evaluable at specific time point. Data were not planned to be collected and analyzed for the prospective TED/NTT group.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Retrospective TED/NTT Group | 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 at Cycle 1: EOT | -3.40 Hours per day | Standard Deviation 3.752 |
| Retrospective TED/NTT Group | 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 at Cycle 2: EOT | -4.80 Hours per day | Standard Deviation 5.36 |
| Retrospective TED/NTT Group | 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 at Cycle 3: EOT | -6.69 Hours per day | Standard Deviation 4.247 |
| Retrospective TED/NTT Group | 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 at Cycle 4: EOT | -6.48 Hours per day | Standard Deviation 4.881 |
| Retrospective TED/NTT Group | 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 at Cycle 5: EOT | -6.71 Hours per day | Standard Deviation 6.175 |
| Retrospective TED/NTT Group | 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 at Cycle 6: EOT | -8.08 Hours per day | Standard Deviation 6.075 |
Secondary
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).
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
Population: RETRO participants: all participants who consented to participation in this extension study and provided data for the retrospective period of the protocol. Here, overall number of participants analyzed signifies participants who were evaluable for this outcome and number analyzed signifies participants who were evaluable at specific time point.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Retrospective TED/NTT Group | Change From Baseline in PS Caloric Intake at 12 Weeks Interval up to Week 156 of Retrospective Observation Period | Change at Week 36 | -8.38 kcal/kg/day | Standard Deviation 17.148 |
| Retrospective TED/NTT Group | Change From Baseline in PS Caloric Intake at 12 Weeks Interval up to Week 156 of Retrospective Observation Period | Change at Week 96 | -10.08 kcal/kg/day | Standard Deviation 23.998 |
| Retrospective TED/NTT Group | Change From Baseline in PS Caloric Intake at 12 Weeks Interval up to Week 156 of Retrospective Observation Period | Change at Week 60 | -10.55 kcal/kg/day | Standard Deviation 23.136 |
| Retrospective TED/NTT Group | Change From Baseline in PS Caloric Intake at 12 Weeks Interval up to Week 156 of Retrospective Observation Period | Change at Week 108 | -7.20 kcal/kg/day | Standard Deviation 22.875 |
| Retrospective TED/NTT Group | Change From Baseline in PS Caloric Intake at 12 Weeks Interval up to Week 156 of Retrospective Observation Period | Change at Week 24 | -3.96 kcal/kg/day | Standard Deviation 16.97 |
| Retrospective TED/NTT Group | Change From Baseline in PS Caloric Intake at 12 Weeks Interval up to Week 156 of Retrospective Observation Period | Change at Week 120 | -7.98 kcal/kg/day | Standard Deviation 21.425 |
| Retrospective TED/NTT Group | Change From Baseline in PS Caloric Intake at 12 Weeks Interval up to Week 156 of Retrospective Observation Period | Change at Week 72 | -12.64 kcal/kg/day | Standard Deviation 23.642 |
| Retrospective TED/NTT Group | Change From Baseline in PS Caloric Intake at 12 Weeks Interval up to Week 156 of Retrospective Observation Period | Change at Week 132 | -12.93 kcal/kg/day | Standard Deviation 18.744 |
| Retrospective TED/NTT Group | Change From Baseline in PS Caloric Intake at 12 Weeks Interval up to Week 156 of Retrospective Observation Period | Change at Week 48 | -9.84 kcal/kg/day | Standard Deviation 22.144 |
| Retrospective TED/NTT Group | Change From Baseline in PS Caloric Intake at 12 Weeks Interval up to Week 156 of Retrospective Observation Period | Change at Week 144 | -11.97 kcal/kg/day | Standard Deviation 17.977 |
| Retrospective TED/NTT Group | Change From Baseline in PS Caloric Intake at 12 Weeks Interval up to Week 156 of Retrospective Observation Period | Change at Week 84 | -9.70 kcal/kg/day | Standard Deviation 24.86 |
| Retrospective TED/NTT Group | Change From Baseline in PS Caloric Intake at 12 Weeks Interval up to Week 156 of Retrospective Observation Period | Change at Week 156 | -6.55 kcal/kg/day | Standard Deviation 4.708 |
| Retrospective TED/NTT Group | Change From Baseline in PS Caloric Intake at 12 Weeks Interval up to Week 156 of Retrospective Observation Period | Change at Week 12 | -10.15 kcal/kg/day | Standard Deviation 13.026 |
| Retrospective TED/TED Group | Change From Baseline in PS Caloric Intake at 12 Weeks Interval up to Week 156 of Retrospective Observation Period | Change at Week 156 | -25.67 kcal/kg/day | — |
| Retrospective TED/TED Group | Change From Baseline in PS Caloric Intake at 12 Weeks Interval up to Week 156 of Retrospective Observation Period | Change at Week 12 | -2.64 kcal/kg/day | Standard Deviation 18.739 |
| Retrospective TED/TED Group | Change From Baseline in PS Caloric Intake at 12 Weeks Interval up to Week 156 of Retrospective Observation Period | Change at Week 24 | -7.25 kcal/kg/day | Standard Deviation 11.916 |
| Retrospective TED/TED Group | Change From Baseline in PS Caloric Intake at 12 Weeks Interval up to Week 156 of Retrospective Observation Period | Change at Week 36 | -6.42 kcal/kg/day | Standard Deviation 24.547 |
| Retrospective TED/TED Group | Change From Baseline in PS Caloric Intake at 12 Weeks Interval up to Week 156 of Retrospective Observation Period | Change at Week 48 | -18.11 kcal/kg/day | Standard Deviation 7.896 |
| Retrospective TED/TED Group | Change From Baseline in PS Caloric Intake at 12 Weeks Interval up to Week 156 of Retrospective Observation Period | Change at Week 60 | -10.53 kcal/kg/day | Standard Deviation 18.538 |
| Retrospective TED/TED Group | Change From Baseline in PS Caloric Intake at 12 Weeks Interval up to Week 156 of Retrospective Observation Period | Change at Week 72 | -0.05 kcal/kg/day | Standard Deviation 23.552 |
| Retrospective TED/TED Group | Change From Baseline in PS Caloric Intake at 12 Weeks Interval up to Week 156 of Retrospective Observation Period | Change at Week 84 | -5.24 kcal/kg/day | Standard Deviation 23.064 |
| Retrospective TED/TED Group | Change From Baseline in PS Caloric Intake at 12 Weeks Interval up to Week 156 of Retrospective Observation Period | Change at Week 96 | -15.09 kcal/kg/day | Standard Deviation 18.604 |
| Retrospective TED/TED Group | Change From Baseline in PS Caloric Intake at 12 Weeks Interval up to Week 156 of Retrospective Observation Period | Change at Week 108 | -19.05 kcal/kg/day | Standard Deviation 18.229 |
| Retrospective TED/TED Group | Change From Baseline in PS Caloric Intake at 12 Weeks Interval up to Week 156 of Retrospective Observation Period | Change at Week 120 | -17.18 kcal/kg/day | Standard Deviation 23.169 |
| Retrospective TED/TED Group | Change From Baseline in PS Caloric Intake at 12 Weeks Interval up to Week 156 of Retrospective Observation Period | Change at Week 132 | -27.59 kcal/kg/day | Standard Deviation 5.114 |
| Retrospective TED/TED Group | Change From Baseline in PS Caloric Intake at 12 Weeks Interval up to Week 156 of Retrospective Observation Period | Change at Week 144 | -25.53 kcal/kg/day | — |
Secondary
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.
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)
Population: Safety population included all enrolled participants who provided informed consent for the prospective portion and met all the inclusion criteria. Here, number analyzed signifies participants who were evaluable for this outcome at specific time point. Data were not planned to be collected and analyzed for the prospective TED/NTT group.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Retrospective TED/NTT Group | Change From Baseline in PS Caloric Intake at EOT of Each Cycle During the End of Teduglutide Treatment Period of Prospective Study Period | Change at Cycle 1: EOT | -17.66 kcal/kg/day | Standard Deviation 17.649 |
| Retrospective TED/NTT Group | Change From Baseline in PS Caloric Intake at EOT of Each Cycle During the End of Teduglutide Treatment Period of Prospective Study Period | Change at Cycle 2: EOT | -19.95 kcal/kg/day | Standard Deviation 23.402 |
| Retrospective TED/NTT Group | Change From Baseline in PS Caloric Intake at EOT of Each Cycle During the End of Teduglutide Treatment Period of Prospective Study Period | Change at Cycle 3: EOT | -27.66 kcal/kg/day | Standard Deviation 19.668 |
| Retrospective TED/NTT Group | Change From Baseline in PS Caloric Intake at EOT of Each Cycle During the End of Teduglutide Treatment Period of Prospective Study Period | Change at Cycle 4: EOT | -24.05 kcal/kg/day | Standard Deviation 22.746 |
| Retrospective TED/NTT Group | Change From Baseline in PS Caloric Intake at EOT of Each Cycle During the End of Teduglutide Treatment Period of Prospective Study Period | Change at Cycle 5: EOT | -26.98 kcal/kg/day | Standard Deviation 27.465 |
| Retrospective TED/NTT Group | Change From Baseline in PS Caloric Intake at EOT of Each Cycle During the End of Teduglutide Treatment Period of Prospective Study Period | Change at Cycle 6: EOT | -27.53 kcal/kg/day | Standard Deviation 32.486 |
Secondary
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.
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
Population: RETRO participants: all participants who consented to participation in this extension study and provided data for the retrospective period of the protocol. Here, number analyzed signifies participants who were evaluable for this outcome at specific time point.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Retrospective TED/NTT Group | Change From Baseline in PS Volume at 12 Weeks Interval up to Week 156 of Retrospective Observation Period | Change at Week 36 | -10.65 mL/kg/day | Standard Deviation 20.877 |
| Retrospective TED/NTT Group | Change From Baseline in PS Volume at 12 Weeks Interval up to Week 156 of Retrospective Observation Period | Change at Week 96 | -12.87 mL/kg/day | Standard Deviation 37.996 |
| Retrospective TED/NTT Group | Change From Baseline in PS Volume at 12 Weeks Interval up to Week 156 of Retrospective Observation Period | Change at Week 60 | -13.85 mL/kg/day | Standard Deviation 30.219 |
| Retrospective TED/NTT Group | Change From Baseline in PS Volume at 12 Weeks Interval up to Week 156 of Retrospective Observation Period | Change at Week 108 | -10.35 mL/kg/day | Standard Deviation 29.37 |
| Retrospective TED/NTT Group | Change From Baseline in PS Volume at 12 Weeks Interval up to Week 156 of Retrospective Observation Period | Change at Week 24 | -9.27 mL/kg/day | Standard Deviation 21.642 |
| Retrospective TED/NTT Group | Change From Baseline in PS Volume at 12 Weeks Interval up to Week 156 of Retrospective Observation Period | Change at Week 120 | -12.21 mL/kg/day | Standard Deviation 27.48 |
| Retrospective TED/NTT Group | Change From Baseline in PS Volume at 12 Weeks Interval up to Week 156 of Retrospective Observation Period | Change at Week 72 | -13.65 mL/kg/day | Standard Deviation 30.577 |
| Retrospective TED/NTT Group | Change From Baseline in PS Volume at 12 Weeks Interval up to Week 156 of Retrospective Observation Period | Change at Week 132 | -19.20 mL/kg/day | Standard Deviation 26.171 |
| Retrospective TED/NTT Group | Change From Baseline in PS Volume at 12 Weeks Interval up to Week 156 of Retrospective Observation Period | Change at Week 48 | -12.24 mL/kg/day | Standard Deviation 29.646 |
| Retrospective TED/NTT Group | Change From Baseline in PS Volume at 12 Weeks Interval up to Week 156 of Retrospective Observation Period | Change at Week 144 | -16.79 mL/kg/day | Standard Deviation 29.62 |
| Retrospective TED/NTT Group | Change From Baseline in PS Volume at 12 Weeks Interval up to Week 156 of Retrospective Observation Period | Change at Week 84 | -8.74 mL/kg/day | Standard Deviation 36.078 |
| Retrospective TED/NTT Group | Change From Baseline in PS Volume at 12 Weeks Interval up to Week 156 of Retrospective Observation Period | Change at Week 156 | -7.46 mL/kg/day | Standard Deviation 1.931 |
| Retrospective TED/NTT Group | Change From Baseline in PS Volume at 12 Weeks Interval up to Week 156 of Retrospective Observation Period | Change at Week 12 | -14.45 mL/kg/day | Standard Deviation 17.77 |
| Retrospective TED/TED Group | Change From Baseline in PS Volume at 12 Weeks Interval up to Week 156 of Retrospective Observation Period | Change at Week 156 | -34.43 mL/kg/day | — |
| Retrospective TED/TED Group | Change From Baseline in PS Volume at 12 Weeks Interval up to Week 156 of Retrospective Observation Period | Change at Week 12 | -12.84 mL/kg/day | Standard Deviation 17.694 |
| Retrospective TED/TED Group | Change From Baseline in PS Volume at 12 Weeks Interval up to Week 156 of Retrospective Observation Period | Change at Week 24 | -13.49 mL/kg/day | Standard Deviation 14.53 |
| Retrospective TED/TED Group | Change From Baseline in PS Volume at 12 Weeks Interval up to Week 156 of Retrospective Observation Period | Change at Week 36 | -17.91 mL/kg/day | Standard Deviation 17.069 |
| Retrospective TED/TED Group | Change From Baseline in PS Volume at 12 Weeks Interval up to Week 156 of Retrospective Observation Period | Change at Week 48 | -26.12 mL/kg/day | Standard Deviation 8.044 |
| Retrospective TED/TED Group | Change From Baseline in PS Volume at 12 Weeks Interval up to Week 156 of Retrospective Observation Period | Change at Week 60 | -21.35 mL/kg/day | Standard Deviation 12.285 |
| Retrospective TED/TED Group | Change From Baseline in PS Volume at 12 Weeks Interval up to Week 156 of Retrospective Observation Period | Change at Week 72 | -11.41 mL/kg/day | Standard Deviation 22.03 |
| Retrospective TED/TED Group | Change From Baseline in PS Volume at 12 Weeks Interval up to Week 156 of Retrospective Observation Period | Change at Week 84 | -15.65 mL/kg/day | Standard Deviation 19.603 |
| Retrospective TED/TED Group | Change From Baseline in PS Volume at 12 Weeks Interval up to Week 156 of Retrospective Observation Period | Change at Week 96 | -27.34 mL/kg/day | Standard Deviation 16.531 |
| Retrospective TED/TED Group | Change From Baseline in PS Volume at 12 Weeks Interval up to Week 156 of Retrospective Observation Period | Change at Week 108 | -31.83 mL/kg/day | Standard Deviation 14.872 |
| Retrospective TED/TED Group | Change From Baseline in PS Volume at 12 Weeks Interval up to Week 156 of Retrospective Observation Period | Change at Week 120 | -30.48 mL/kg/day | Standard Deviation 19.043 |
| Retrospective TED/TED Group | Change From Baseline in PS Volume at 12 Weeks Interval up to Week 156 of Retrospective Observation Period | Change at Week 132 | -37.70 mL/kg/day | Standard Deviation 12.296 |
| Retrospective TED/TED Group | Change From Baseline in PS Volume at 12 Weeks Interval up to Week 156 of Retrospective Observation Period | Change at Week 144 | -34.32 mL/kg/day | — |
Secondary
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.
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)
Population: Safety population included all enrolled participants who provided informed consent for the prospective portion and met all the inclusion criteria. Here, overall number of participants analyzed signifies participants who were evaluable for this outcome measure and number analyzed signifies participants who were evaluable at specific time point. Data were not planned to be collected and analyzed for the prospective TED/NTT group.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Retrospective TED/NTT Group | Change From Baseline in PS Volume at EOT of Each Cycle During the End of Teduglutide Treatment Period of Prospective Study Period | Change at Cycle 1: EOT | -25.18 mL/kg/day | Standard Deviation 19.915 |
| Retrospective TED/NTT Group | Change From Baseline in PS Volume at EOT of Each Cycle During the End of Teduglutide Treatment Period of Prospective Study Period | Change at Cycle 2: EOT | -28.59 mL/kg/day | Standard Deviation 29.856 |
| Retrospective TED/NTT Group | Change From Baseline in PS Volume at EOT of Each Cycle During the End of Teduglutide Treatment Period of Prospective Study Period | Change at Cycle 3: EOT | -40.05 mL/kg/day | Standard Deviation 29.711 |
| Retrospective TED/NTT Group | Change From Baseline in PS Volume at EOT of Each Cycle During the End of Teduglutide Treatment Period of Prospective Study Period | Change at Cycle 4: EOT | -40.10 mL/kg/day | Standard Deviation 30.359 |
| Retrospective TED/NTT Group | Change From Baseline in PS Volume at EOT of Each Cycle During the End of Teduglutide Treatment Period of Prospective Study Period | Change at Cycle 5: EOT | -40.49 mL/kg/day | Standard Deviation 24.259 |
| Retrospective TED/NTT Group | Change From Baseline in PS Volume at EOT of Each Cycle During the End of Teduglutide Treatment Period of Prospective Study Period | Change at Cycle 6: EOT | -47.57 mL/kg/day | Standard Deviation 26.243 |
Secondary
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.
Time frame: At Weeks 12, 24, 36, 48, 60, 72, 84, 96, 108, 120, 132, 144, and 156
Population: RETRO participants: all participants who consented to participation in this extension study and provided data for the retrospective period of the protocol. Here, number analyzed signifies participants who were evaluable for this outcome at specific time point.
| Arm | Measure | Group | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|---|
| Retrospective TED/NTT Group | 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 | At Week 36 | 9 Participants |
| Retrospective TED/NTT Group | 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 | At Week 96 | 10 Participants |
| Retrospective TED/NTT Group | 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 | At Week 60 | 9 Participants |
| Retrospective TED/NTT Group | 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 | At Week 108 | 9 Participants |
| Retrospective TED/NTT Group | 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 | At Week 24 | 9 Participants |
| Retrospective TED/NTT Group | 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 | At Week 120 | 10 Participants |
| Retrospective TED/NTT Group | 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 | At Week 72 | 6 Participants |
| Retrospective TED/NTT Group | 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 | At Week 132 | 7 Participants |
| Retrospective TED/NTT Group | 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 | At Week 48 | 9 Participants |
| Retrospective TED/NTT Group | 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 | At Week 144 | 3 Participants |
| Retrospective TED/NTT Group | 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 | At Week 84 | 7 Participants |
| Retrospective TED/NTT Group | 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 | At Week 156 | 0 Participants |
| Retrospective TED/NTT Group | 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 | At Week 12 | 12 Participants |
| Retrospective TED/TED Group | 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 | At Week 156 | 1 Participants |
| Retrospective TED/TED Group | 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 | At Week 12 | 2 Participants |
| Retrospective TED/TED Group | 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 | At Week 24 | 2 Participants |
| Retrospective TED/TED Group | 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 | At Week 36 | 4 Participants |
| Retrospective TED/TED Group | 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 | At Week 48 | 4 Participants |
| Retrospective TED/TED Group | 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 | At Week 60 | 4 Participants |
| Retrospective TED/TED Group | 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 | At Week 72 | 2 Participants |
| Retrospective TED/TED Group | 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 | At Week 84 | 2 Participants |
| Retrospective TED/TED Group | 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 | At Week 96 | 5 Participants |
| Retrospective TED/TED Group | 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 | At Week 108 | 5 Participants |
| Retrospective TED/TED Group | 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 | At Week 120 | 4 Participants |
| Retrospective TED/TED Group | 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 | At Week 132 | 4 Participants |
| Retrospective TED/TED Group | 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 | At Week 144 | 1 Participants |
Secondary
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.
Time frame: At EOT of each Cycles 1 to 6 (up to Week 144) (length of each Cycle 1 to 6 = 24 weeks)
Population: Safety population included all enrolled participants who provided informed consent for the prospective portion and met all the inclusion criteria. Here, number analyzed signifies participants who were evaluable for this outcome at specific time point. Data were not planned to be collected and analyzed for the prospective TED/NTT group.
| Arm | Measure | Group | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|---|
| Retrospective TED/NTT Group | 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 | Cycle 1: EOT | 1 Participants |
| Retrospective TED/NTT Group | 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 | Cycle 2: EOT | 3 Participants |
| Retrospective TED/NTT Group | 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 | Cycle 3: EOT | 6 Participants |
| Retrospective TED/NTT Group | 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 | Cycle 4: EOT | 5 Participants |
| Retrospective TED/NTT Group | 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 | Cycle 5: EOT | 4 Participants |
| Retrospective TED/NTT Group | 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 | Cycle 6: EOT | 3 Participants |
Secondary
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.
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)
Population: Safety population included all enrolled participants who provided informed consent for the prospective portion and met all the inclusion criteria. Here, number analyzed signifies participants who were evaluable for this outcome at specific time point. Data were not planned to be collected and analyzed for the prospective TED/NTT group.
| Arm | Measure | Group | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|---|
| Retrospective TED/NTT Group | 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 | Cycle 1: EOT at >= 20% | 13 Participants |
| Retrospective TED/NTT Group | 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 | Cycle 2: EOT at >= 20% | 12 Participants |
| Retrospective TED/NTT Group | 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 | Cycle 3: EOT at >= 20% | 10 Participants |
| Retrospective TED/NTT Group | 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 | Cycle 4: EOT at >= 20% | 9 Participants |
| Retrospective TED/NTT Group | 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 | Cycle 5: EOT at >= 20% | 7 Participants |
| Retrospective TED/NTT Group | 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 | Cycle 6: EOT at >= 20% | 5 Participants |
| Retrospective TED/NTT Group | 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 | Cycle 1: EOT at >= 50% | 5 Participants |
| Retrospective TED/NTT Group | 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 | Cycle 2: EOT at >= 50% | 10 Participants |
| Retrospective TED/NTT Group | 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 | Cycle 3: EOT at >= 50% | 9 Participants |
| Retrospective TED/NTT Group | 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 | Cycle 4: EOT at >= 50% | 9 Participants |
| Retrospective TED/NTT Group | 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 | Cycle 5: EOT at >= 50% | 5 Participants |
| Retrospective TED/NTT Group | 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 | Cycle 6: EOT at >= 50% | 4 Participants |
| Retrospective TED/NTT Group | 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 | Cycle 1: EOT at >= 75% | 2 Participants |
| Retrospective TED/NTT Group | 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 | Cycle 2: EOT at >= 75% | 4 Participants |
| Retrospective TED/NTT Group | 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 | Cycle 3: EOT at >= 75% | 6 Participants |
| Retrospective TED/NTT Group | 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 | Cycle 4: EOT at >= 75% | 5 Participants |
| Retrospective TED/NTT Group | 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 | Cycle 5: EOT at >= 75% | 4 Participants |
| Retrospective TED/NTT Group | 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 | Cycle 6: EOT at >= 75% | 3 Participants |
Secondary
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.
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
Population: RETRO participants: all participants who consented to participation in this extension study and provided data for the retrospective period of the protocol. Here, number analyzed signifies participants who were evaluable for this outcome at specific time point.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Retrospective TED/NTT Group | 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 at Week 156 | 0.00 Percent change | Standard Deviation 0 |
| Retrospective TED/NTT Group | 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 at Week 12 | -6.25 Percent change | Standard Deviation 22.421 |
| Retrospective TED/NTT Group | 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 at Week 24 | -4.17 Percent change | Standard Deviation 20.412 |
| Retrospective TED/NTT Group | 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 at Week 36 | -6.21 Percent change | Standard Deviation 22.309 |
| Retrospective TED/NTT Group | 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 at Week 48 | -8.70 Percent change | Standard Deviation 28.81 |
| Retrospective TED/NTT Group | 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 at Week 60 | -8.70 Percent change | Standard Deviation 28.81 |
| Retrospective TED/NTT Group | 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 at Week 72 | -9.09 Percent change | Standard Deviation 29.424 |
| Retrospective TED/NTT Group | 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 at Week 84 | -9.09 Percent change | Standard Deviation 29.424 |
| Retrospective TED/NTT Group | 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 at Week 96 | -16.67 Percent change | Standard Deviation 38.069 |
| Retrospective TED/NTT Group | 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 at Week 108 | -12.50 Percent change | Standard Deviation 33.783 |
| Retrospective TED/NTT Group | 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 at Week 120 | -13.64 Percent change | Standard Deviation 35.125 |
| Retrospective TED/NTT Group | 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 at Week 132 | -14.29 Percent change | Standard Deviation 36.314 |
| Retrospective TED/NTT Group | 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 at Week 144 | 0.00 Percent change | Standard Deviation 0 |
| Retrospective TED/TED Group | 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 at Week 120 | -52.00 Percent change | Standard Deviation 57.407 |
| Retrospective TED/TED Group | 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 at Week 84 | -17.71 Percent change | Standard Deviation 45.821 |
| Retrospective TED/TED Group | 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 at Week 12 | -14.86 Percent change | Standard Deviation 41.913 |
| Retrospective TED/TED Group | 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 at Week 144 | -42.86 Percent change | — |
| Retrospective TED/TED Group | 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 at Week 24 | -14.29 Percent change | Standard Deviation 28.571 |
| Retrospective TED/TED Group | 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 at Week 96 | -26.29 Percent change | Standard Deviation 53.886 |
| Retrospective TED/TED Group | 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 at Week 36 | -34.86 Percent change | Standard Deviation 44.648 |
| Retrospective TED/TED Group | 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 at Week 132 | -75.00 Percent change | Standard Deviation 29.451 |
| Retrospective TED/TED Group | 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 at Week 48 | -64.29 Percent change | Standard Deviation 29.738 |
| Retrospective TED/TED Group | 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 at Week 108 | -52.00 Percent change | Standard Deviation 57.407 |
| Retrospective TED/TED Group | 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 at Week 60 | -40.57 Percent change | Standard Deviation 54.638 |
| Retrospective TED/TED Group | 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 at Week 156 | -42.86 Percent change | — |
| Retrospective TED/TED Group | 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 at Week 72 | -17.71 Percent change | Standard Deviation 45.821 |
Secondary
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.
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)
Population: Safety population included all enrolled participants who provided informed consent for the prospective portion and met all the inclusion criteria. Here, overall number of participants analyzed signifies participants who were evaluable for this outcome measure and number analyzed signifies participants who were evaluable at specific time point. Data were not planned to be collected and analyzed for the prospective TED/NTT group.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Retrospective TED/NTT Group | 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 at Cycle 3: EOT | -44.51 Percent change | Standard Deviation 47.929 |
| Retrospective TED/NTT Group | 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 at Cycle 1: EOT | -9.01 Percent change | Standard Deviation 27.472 |
| Retrospective TED/NTT Group | 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 at Cycle 2: EOT | -29.24 Percent change | Standard Deviation 40.525 |
| Retrospective TED/NTT Group | 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 at Cycle 4: EOT | -42.38 Percent change | Standard Deviation 51.929 |
| Retrospective TED/NTT Group | 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 at Cycle 5: EOT | -54.29 Percent change | Standard Deviation 57.404 |
| Retrospective TED/NTT Group | 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 at Cycle 6: EOT | -62.86 Percent change | Standard Deviation 51.11 |
Secondary
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.
Time frame: Baseline (Baseline visit in the core study [TED-C13-003 (NCT01952080)]) up to Week 156
Population: 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.
Secondary
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.
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)
Population: Safety population included all enrolled participants who provided informed consent for the prospective portion and met all the inclusion criteria. Here, overall number of participants analyzed signifies participants who were evaluable for this outcome measure and number analyzed signifies participants who were evaluable at specific time point. Data were not planned to be collected and analyzed for the prospective TED/NTT group.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Retrospective TED/NTT Group | 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 at Cycle 1: EOT | -26.32 Percent change | Standard Deviation 29.732 |
| Retrospective TED/NTT Group | 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 at Cycle 2: EOT | -40.47 Percent change | Standard Deviation 40.476 |
| Retrospective TED/NTT Group | 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 at Cycle 3: EOT | -57.61 Percent change | Standard Deviation 36.973 |
| Retrospective TED/NTT Group | 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 at Cycle 4: EOT | -55.87 Percent change | Standard Deviation 42.693 |
| Retrospective TED/NTT Group | 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 at Cycle 5: EOT | -58.85 Percent change | Standard Deviation 52.717 |
| Retrospective TED/NTT Group | 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 at Cycle 6: EOT | -66.39 Percent change | Standard Deviation 46.042 |
Secondary
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.
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
Population: RETRO participants: all participants who consented to participation in this extension study and provided data for the retrospective period of the protocol. Here, overall number of participants analyzed signifies participants who were evaluable for this outcome and number analyzed signifies participants who were evaluable at specific time point.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Retrospective TED/NTT Group | Percent Change From Baseline in PS Caloric Intake at 12 Weeks Interval up to Week 156 of Retrospective Observation Period | Percent change at Week 36 | -18.29 Percent change | Standard Deviation 41.07 |
| Retrospective TED/NTT Group | Percent Change From Baseline in PS Caloric Intake at 12 Weeks Interval up to Week 156 of Retrospective Observation Period | Percent change at Week 96 | -21.12 Percent change | Standard Deviation 46.005 |
| Retrospective TED/NTT Group | Percent Change From Baseline in PS Caloric Intake at 12 Weeks Interval up to Week 156 of Retrospective Observation Period | Percent change at Week 60 | -20.63 Percent change | Standard Deviation 44.903 |
| Retrospective TED/NTT Group | Percent Change From Baseline in PS Caloric Intake at 12 Weeks Interval up to Week 156 of Retrospective Observation Period | Percent change at Week 108 | -16.41 Percent change | Standard Deviation 46.034 |
| Retrospective TED/NTT Group | Percent Change From Baseline in PS Caloric Intake at 12 Weeks Interval up to Week 156 of Retrospective Observation Period | Percent change at Week 24 | -10.12 Percent change | Standard Deviation 42.431 |
| Retrospective TED/NTT Group | Percent Change From Baseline in PS Caloric Intake at 12 Weeks Interval up to Week 156 of Retrospective Observation Period | Percent change at Week 120 | -18.79 Percent change | Standard Deviation 45.774 |
| Retrospective TED/NTT Group | Percent Change From Baseline in PS Caloric Intake at 12 Weeks Interval up to Week 156 of Retrospective Observation Period | Percent change at Week 72 | -25.48 Percent change | Standard Deviation 45.035 |
| Retrospective TED/NTT Group | Percent Change From Baseline in PS Caloric Intake at 12 Weeks Interval up to Week 156 of Retrospective Observation Period | Percent change at Week 132 | -29.37 Percent change | Standard Deviation 40.17 |
| Retrospective TED/NTT Group | Percent Change From Baseline in PS Caloric Intake at 12 Weeks Interval up to Week 156 of Retrospective Observation Period | Percent change at Week 48 | -21.03 Percent change | Standard Deviation 43.924 |
| Retrospective TED/NTT Group | Percent Change From Baseline in PS Caloric Intake at 12 Weeks Interval up to Week 156 of Retrospective Observation Period | Percent change at Week 144 | -25.25 Percent change | Standard Deviation 33.618 |
| Retrospective TED/NTT Group | Percent Change From Baseline in PS Caloric Intake at 12 Weeks Interval up to Week 156 of Retrospective Observation Period | Percent change at Week 84 | -20.42 Percent change | Standard Deviation 45.287 |
| Retrospective TED/NTT Group | Percent Change From Baseline in PS Caloric Intake at 12 Weeks Interval up to Week 156 of Retrospective Observation Period | Percent change at Week 156 | -15.66 Percent change | Standard Deviation 7.7 |
| Retrospective TED/NTT Group | Percent Change From Baseline in PS Caloric Intake at 12 Weeks Interval up to Week 156 of Retrospective Observation Period | Percent change at Week 12 | -24.91 Percent change | Standard Deviation 29.001 |
| Retrospective TED/TED Group | Percent Change From Baseline in PS Caloric Intake at 12 Weeks Interval up to Week 156 of Retrospective Observation Period | Percent change at Week 156 | -60.69 Percent change | — |
| Retrospective TED/TED Group | Percent Change From Baseline in PS Caloric Intake at 12 Weeks Interval up to Week 156 of Retrospective Observation Period | Percent change at Week 12 | 7.64 Percent change | Standard Deviation 82.061 |
| Retrospective TED/TED Group | Percent Change From Baseline in PS Caloric Intake at 12 Weeks Interval up to Week 156 of Retrospective Observation Period | Percent change at Week 24 | -21.29 Percent change | Standard Deviation 37.759 |
| Retrospective TED/TED Group | Percent Change From Baseline in PS Caloric Intake at 12 Weeks Interval up to Week 156 of Retrospective Observation Period | Percent change at Week 36 | -3.38 Percent change | Standard Deviation 118.167 |
| Retrospective TED/TED Group | Percent Change From Baseline in PS Caloric Intake at 12 Weeks Interval up to Week 156 of Retrospective Observation Period | Percent change at Week 48 | -59.31 Percent change | Standard Deviation 38.042 |
| Retrospective TED/TED Group | Percent Change From Baseline in PS Caloric Intake at 12 Weeks Interval up to Week 156 of Retrospective Observation Period | Percent change at Week 60 | -25.65 Percent change | Standard Deviation 83.47 |
| Retrospective TED/TED Group | Percent Change From Baseline in PS Caloric Intake at 12 Weeks Interval up to Week 156 of Retrospective Observation Period | Percent change at Week 72 | 23.26 Percent change | Standard Deviation 99.38 |
| Retrospective TED/TED Group | Percent Change From Baseline in PS Caloric Intake at 12 Weeks Interval up to Week 156 of Retrospective Observation Period | Percent change at Week 84 | 4.41 Percent change | Standard Deviation 102.162 |
| Retrospective TED/TED Group | Percent Change From Baseline in PS Caloric Intake at 12 Weeks Interval up to Week 156 of Retrospective Observation Period | Percent change at Week 96 | -36.49 Percent change | Standard Deviation 72.357 |
| Retrospective TED/TED Group | Percent Change From Baseline in PS Caloric Intake at 12 Weeks Interval up to Week 156 of Retrospective Observation Period | Percent change at Week 108 | -50.24 Percent change | Standard Deviation 70.513 |
| Retrospective TED/TED Group | Percent Change From Baseline in PS Caloric Intake at 12 Weeks Interval up to Week 156 of Retrospective Observation Period | Percent change at Week 120 | -38.58 Percent change | Standard Deviation 97.75 |
| Retrospective TED/TED Group | Percent Change From Baseline in PS Caloric Intake at 12 Weeks Interval up to Week 156 of Retrospective Observation Period | Percent change at Week 132 | -82.01 Percent change | Standard Deviation 21.165 |
| Retrospective TED/TED Group | Percent Change From Baseline in PS Caloric Intake at 12 Weeks Interval up to Week 156 of Retrospective Observation Period | Percent change at Week 144 | -60.36 Percent change | — |
Secondary
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.
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)
Population: Safety population included all enrolled participants who provided informed consent for the prospective portion and met all the inclusion criteria. Here, number analyzed signifies participants who were evaluable for this outcome at specific time point. Data were not planned to be collected and analyzed for the prospective TED/NTT group.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Retrospective TED/NTT Group | 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 at Cycle 1: EOT | -33.50 Percent change | Standard Deviation 40.439 |
| Retrospective TED/NTT Group | 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 at Cycle 2: EOT | -42.06 Percent change | Standard Deviation 44.077 |
| Retrospective TED/NTT Group | 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 at Cycle 3: EOT | -62.47 Percent change | Standard Deviation 37.677 |
| Retrospective TED/NTT Group | 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 at Cycle 4: EOT | -49.51 Percent change | Standard Deviation 54.041 |
| Retrospective TED/NTT Group | 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 at Cycle 5: EOT | -54.08 Percent change | Standard Deviation 64.002 |
| Retrospective TED/NTT Group | 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 at Cycle 6: EOT | -59.00 Percent change | Standard Deviation 57.267 |
Secondary
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.
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
Population: RETRO participants: all participants who consented to participation in this extension study and provided data for the retrospective period of the protocol. Here, number analyzed signifies participants who were evaluable for this outcome at specific time point.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Retrospective TED/NTT Group | Percent Change From Baseline in PS Volume at 12 Weeks Interval up to Week 156 of Retrospective Observation Period | Percent change at Week 36 | -13.21 Percent change | Standard Deviation 34.172 |
| Retrospective TED/NTT Group | Percent Change From Baseline in PS Volume at 12 Weeks Interval up to Week 156 of Retrospective Observation Period | Percent change at Week 96 | -12.04 Percent change | Standard Deviation 51.631 |
| Retrospective TED/NTT Group | Percent Change From Baseline in PS Volume at 12 Weeks Interval up to Week 156 of Retrospective Observation Period | Percent change at Week 60 | -16.15 Percent change | Standard Deviation 39.491 |
| Retrospective TED/NTT Group | Percent Change From Baseline in PS Volume at 12 Weeks Interval up to Week 156 of Retrospective Observation Period | Percent change at Week 108 | -10.62 Percent change | Standard Deviation 48.82 |
| Retrospective TED/NTT Group | Percent Change From Baseline in PS Volume at 12 Weeks Interval up to Week 156 of Retrospective Observation Period | Percent change at Week 24 | -10.72 Percent change | Standard Deviation 33.081 |
| Retrospective TED/NTT Group | Percent Change From Baseline in PS Volume at 12 Weeks Interval up to Week 156 of Retrospective Observation Period | Percent change at Week 120 | -12.89 Percent change | Standard Deviation 49.366 |
| Retrospective TED/NTT Group | Percent Change From Baseline in PS Volume at 12 Weeks Interval up to Week 156 of Retrospective Observation Period | Percent change at Week 72 | -14.19 Percent change | Standard Deviation 40.558 |
| Retrospective TED/NTT Group | Percent Change From Baseline in PS Volume at 12 Weeks Interval up to Week 156 of Retrospective Observation Period | Percent change at Week 132 | -20.24 Percent change | Standard Deviation 47.431 |
| Retrospective TED/NTT Group | Percent Change From Baseline in PS Volume at 12 Weeks Interval up to Week 156 of Retrospective Observation Period | Percent change at Week 48 | -14.04 Percent change | Standard Deviation 39.39 |
| Retrospective TED/NTT Group | Percent Change From Baseline in PS Volume at 12 Weeks Interval up to Week 156 of Retrospective Observation Period | Percent change at Week 144 | -7.70 Percent change | Standard Deviation 44.17 |
| Retrospective TED/NTT Group | Percent Change From Baseline in PS Volume at 12 Weeks Interval up to Week 156 of Retrospective Observation Period | Percent change at Week 84 | -8.67 Percent change | Standard Deviation 47.108 |
| Retrospective TED/NTT Group | Percent Change From Baseline in PS Volume at 12 Weeks Interval up to Week 156 of Retrospective Observation Period | Percent change at Week 156 | -17.21 Percent change | Standard Deviation 3.164 |
| Retrospective TED/NTT Group | Percent Change From Baseline in PS Volume at 12 Weeks Interval up to Week 156 of Retrospective Observation Period | Percent change at Week 12 | -24.50 Percent change | Standard Deviation 27.853 |
| Retrospective TED/TED Group | Percent Change From Baseline in PS Volume at 12 Weeks Interval up to Week 156 of Retrospective Observation Period | Percent change at Week 156 | -65.42 Percent change | — |
| Retrospective TED/TED Group | Percent Change From Baseline in PS Volume at 12 Weeks Interval up to Week 156 of Retrospective Observation Period | Percent change at Week 12 | -24.88 Percent change | Standard Deviation 35.649 |
| Retrospective TED/TED Group | Percent Change From Baseline in PS Volume at 12 Weeks Interval up to Week 156 of Retrospective Observation Period | Percent change at Week 24 | -32.10 Percent change | Standard Deviation 31.645 |
| Retrospective TED/TED Group | Percent Change From Baseline in PS Volume at 12 Weeks Interval up to Week 156 of Retrospective Observation Period | Percent change at Week 36 | -43.09 Percent change | Standard Deviation 42.753 |
| Retrospective TED/TED Group | Percent Change From Baseline in PS Volume at 12 Weeks Interval up to Week 156 of Retrospective Observation Period | Percent change at Week 48 | -65.18 Percent change | Standard Deviation 31.65 |
| Retrospective TED/TED Group | Percent Change From Baseline in PS Volume at 12 Weeks Interval up to Week 156 of Retrospective Observation Period | Percent change at Week 60 | -53.61 Percent change | Standard Deviation 37.141 |
| Retrospective TED/TED Group | Percent Change From Baseline in PS Volume at 12 Weeks Interval up to Week 156 of Retrospective Observation Period | Percent change at Week 72 | -14.11 Percent change | Standard Deviation 58.452 |
| Retrospective TED/TED Group | Percent Change From Baseline in PS Volume at 12 Weeks Interval up to Week 156 of Retrospective Observation Period | Percent change at Week 84 | -30.25 Percent change | Standard Deviation 39.598 |
| Retrospective TED/TED Group | Percent Change From Baseline in PS Volume at 12 Weeks Interval up to Week 156 of Retrospective Observation Period | Percent change at Week 96 | -61.19 Percent change | Standard Deviation 29.335 |
| Retrospective TED/TED Group | Percent Change From Baseline in PS Volume at 12 Weeks Interval up to Week 156 of Retrospective Observation Period | Percent change at Week 108 | -73.43 Percent change | Standard Deviation 29.298 |
| Retrospective TED/TED Group | Percent Change From Baseline in PS Volume at 12 Weeks Interval up to Week 156 of Retrospective Observation Period | Percent change at Week 120 | -69.61 Percent change | Standard Deviation 39.194 |
| Retrospective TED/TED Group | Percent Change From Baseline in PS Volume at 12 Weeks Interval up to Week 156 of Retrospective Observation Period | Percent change at Week 132 | -85.92 Percent change | Standard Deviation 17.457 |
| Retrospective TED/TED Group | Percent Change From Baseline in PS Volume at 12 Weeks Interval up to Week 156 of Retrospective Observation Period | Percent change at Week 144 | -65.20 Percent change | — |
Secondary
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.
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)
Population: Safety population included all enrolled participants who provided informed consent for the prospective portion and met all the inclusion criteria. Here, overall number of participants analyzed signifies participants who were evaluable for this outcome measure and number analyzed signifies participants who were evaluable at specific time point. Data were not planned to be collected and analyzed for the prospective TED/NTT group.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Retrospective TED/NTT Group | 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: Cycle 1: EOT | -38.02 Percent change | Standard Deviation 28.611 |
| Retrospective TED/NTT Group | 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: Cycle 2: EOT | -45.58 Percent change | Standard Deviation 48.15 |
| Retrospective TED/NTT Group | 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: Cycle 3: EOT | -62.16 Percent change | Standard Deviation 41.805 |
| Retrospective TED/NTT Group | 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: Cycle 4: EOT | -62.92 Percent change | Standard Deviation 38.466 |
| Retrospective TED/NTT Group | 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: Cycle 5: EOT | -73.49 Percent change | Standard Deviation 34.593 |
| Retrospective TED/NTT Group | 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: Cycle 6: EOT | -78.75 Percent change | Standard Deviation 33.949 |
Other Pre-specified
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
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)
Population: Safety population included all enrolled participants who provided informed consent for the prospective portion and met all the inclusion criteria. Data was not collected and analyzed at this time point, for this respective arms.
Other Pre-specified
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
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
Population: Safety population included all enrolled participants who provided informed consent for the prospective portion and met all the inclusion criteria. Here, number analyzed signifies participants who were evaluable for this outcome at specific time point. Data were not planned to be collected and analyzed for the prospective TED/NTT group.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Retrospective TED/NTT Group | 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 | Change at Cycle 1 Week 12 | 1.39 Score on a scale | Standard Deviation 15.742 |
| Retrospective TED/NTT Group | 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 | Change at Cycle 1 Week 24 | 1.56 Score on a scale | Standard Deviation 13.372 |
| Retrospective TED/NTT Group | 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 | Change at Cycle 2 Week 12 | 0.10 Score on a scale | Standard Deviation 13.622 |
| Retrospective TED/NTT Group | 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 | Change at Cycle 2 Week 24 | 1.90 Score on a scale | Standard Deviation 11.664 |
| Retrospective TED/NTT Group | 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 | Change at Cycle 3 Week 12 | 2.72 Score on a scale | Standard Deviation 15.051 |
| Retrospective TED/NTT Group | 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 | Change at Cycle 3 Week 24 | 3.90 Score on a scale | Standard Deviation 16.018 |
| Retrospective TED/NTT Group | 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 | Change at Cycle 4 Week 12 | 2.14 Score on a scale | Standard Deviation 20.762 |
| Retrospective TED/NTT Group | 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 | Change at Cycle 4 Week 24 | 4.66 Score on a scale | Standard Deviation 23.546 |
| Retrospective TED/NTT Group | 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 | Change at Cycle 5 Week 12 | 0.11 Score on a scale | Standard Deviation 14.105 |
| Retrospective TED/NTT Group | 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 | Change at Cycle 5 Week 24 | 2.23 Score on a scale | Standard Deviation 16.099 |
| Retrospective TED/NTT Group | 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 | Change at Cycle 6 Week 12 | 5.13 Score on a scale | Standard Deviation 18.084 |
| Retrospective TED/NTT Group | 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 | Change at Cycle 6 Week 24 | -17.86 Score on a scale | — |
Other Pre-specified
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
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)
Population: Safety population included all enrolled participants who provided informed consent for the prospective portion and met all the inclusion criteria. Data was not collected and analyzed at this time point, for this respective arms.
| Arm | Measure | Group | Value |
|---|---|---|---|
| Unknown | 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 | Food and Drink Limits: End of last NTT Period | — |
| Unknown | 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 | Diarrhea: End of last NTT Period | — |
Other Pre-specified
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
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
Population: Safety population included all enrolled participants who provided informed consent for the prospective portion and met all the inclusion criteria. Here, overall number of participants analyzed refer to the participants evaluable for this outcome measure and number analyzed refer to participants evaluable for this outcome at at specific time point at specific time point. Data for this outcome was not planned to be collected and analyzed in prospective TED/NTT group.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Retrospective TED/NTT Group | 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 | Diarrhea: Change at Cycle 4 Week 12 | 9.13 Score on a scale | Standard Deviation 18.653 |
| Retrospective TED/NTT Group | 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 | Food and Drink Limits: Change at Cycle 1 Week 12 | -2.08 Score on a scale | Standard Deviation 30.46 |
| Retrospective TED/NTT Group | 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 | Food and Drink Limits: Change at Cycle 1 Week 24 | -7.06 Score on a scale | Standard Deviation 27.524 |
| Retrospective TED/NTT Group | 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 | Food and Drink Limits: Change at Cycle 2 Week 12 | -0.83 Score on a scale | Standard Deviation 29.723 |
| Retrospective TED/NTT Group | 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 | Food and Drink Limits: Change at Cycle 2 Week 24 | 9.31 Score on a scale | Standard Deviation 26.292 |
| Retrospective TED/NTT Group | 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 | Food and Drink Limits: Change at Cycle 3 Week 12 | 1.19 Score on a scale | Standard Deviation 35.784 |
| Retrospective TED/NTT Group | 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 | Food and Drink Limits: Change at Cycle 3 Week 24 | -0.08 Score on a scale | Standard Deviation 29.511 |
| Retrospective TED/NTT Group | 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 | Food and Drink Limits: Change at Cycle 4 Week 12 | -4.63 Score on a scale | Standard Deviation 33.75 |
| Retrospective TED/NTT Group | 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 | Food and Drink Limits: Change at Cycle 4 Week 24 | 1.04 Score on a scale | Standard Deviation 35.895 |
| Retrospective TED/NTT Group | 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 | Food and Drink Limits: Change at Cycle 5 Week 12 | 1.19 Score on a scale | Standard Deviation 21.343 |
| Retrospective TED/NTT Group | 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 | Food and Drink Limits: Change at Cycle 5 Week 24 | -17.36 Score on a scale | Standard Deviation 44.986 |
| Retrospective TED/NTT Group | 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 | Food and Drink Limits: Change at Cycle 6 Week 12 | -1.39 Score on a scale | Standard Deviation 30.142 |
| Retrospective TED/NTT Group | 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 | Food and Drink Limits: Change at Cycle 6 Week 24 | -45.83 Score on a scale | — |
| Retrospective TED/NTT Group | 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 | Diarrhea: Change at Cycle 1 Week 12 | 7.79 Score on a scale | Standard Deviation 23.137 |
| Retrospective TED/NTT Group | 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 | Diarrhea: Change at Cycle 1 Week 24 | 9.33 Score on a scale | Standard Deviation 23.279 |
| Retrospective TED/NTT Group | 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 | Diarrhea: Change at Cycle 2 Week 12 | 5.48 Score on a scale | Standard Deviation 15.144 |
| Retrospective TED/NTT Group | 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 | Diarrhea: Change at Cycle 2 Week 24 | 6.72 Score on a scale | Standard Deviation 19.88 |
| Retrospective TED/NTT Group | 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 | Diarrhea: Change at Cycle 3 Week 12 | 13.27 Score on a scale | Standard Deviation 22.124 |
| Retrospective TED/NTT Group | 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 | Diarrhea: Change at Cycle 3 Week 24 | 5.19 Score on a scale | Standard Deviation 21.569 |
| Retrospective TED/NTT Group | 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 | Diarrhea: Change at Cycle 4 Week 24 | 12.95 Score on a scale | Standard Deviation 26.448 |
| Retrospective TED/NTT Group | 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 | Diarrhea: Change at Cycle 5 Week 12 | 10.71 Score on a scale | Standard Deviation 23.237 |
| Retrospective TED/NTT Group | 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 | Diarrhea: Change at Cycle 5 Week 24 | 8.33 Score on a scale | Standard Deviation 30.612 |
| Retrospective TED/NTT Group | 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 | Diarrhea: Change at Cycle 6 Week 12 | 5.95 Score on a scale | Standard Deviation 22.961 |
| Retrospective TED/NTT Group | 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 | Diarrhea: Change at Cycle 6 Week 24 | -7.14 Score on a scale | — |
Other Pre-specified
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
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)
Population: Safety population included all enrolled participants who provided informed consent for the prospective portion and met all the inclusion criteria. Data was not collected and analyzed at this time point, for this respective arms.
| Arm | Measure | Group | Value |
|---|---|---|---|
| Unknown | 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 | Physical Functioning: End of last NTT period | — |
| Unknown | 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 | Emotional Functioning: End of last NTT period | — |
| Unknown | 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 | Social Functioning: End of last NTT period | — |
| Unknown | 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 | School Functioning: End of last NTT period | — |
Other Pre-specified
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
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
Population: Safety population included all enrolled participants who provided informed consent for the prospective portion and met all the inclusion criteria. Here, overall number of participants analyzed signifies participants who were evaluable for this outcome measure and number analyzed signifies participants who were evaluable at specific time point. Data were not planned to be collected and analyzed for the prospective TED/NTT group.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Retrospective TED/NTT Group | 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 | Physical Functioning: Change at Cycle 1 Week 12 | -0.39 Score on a scale | Standard Deviation 24.326 |
| Retrospective TED/NTT Group | 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 | Physical Functioning: Change at Cycle 1 Week 24 | -0.83 Score on a scale | Standard Deviation 10.726 |
| Retrospective TED/NTT Group | 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 | Physical Functioning: Change at Cycle 2 Week 12 | 2.34 Score on a scale | Standard Deviation 15.717 |
| Retrospective TED/NTT Group | 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 | Physical Functioning: Change at Cycle 2 Week 24 | 2.40 Score on a scale | Standard Deviation 16.296 |
| Retrospective TED/NTT Group | 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 | Physical Functioning: Change at Cycle 3 Week 12 | -0.57 Score on a scale | Standard Deviation 16.167 |
| Retrospective TED/NTT Group | 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 | Physical Functioning: Change at Cycle 3 Week 24 | -1.74 Score on a scale | Standard Deviation 25.963 |
| Retrospective TED/NTT Group | 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 | Physical Functioning: Change at Cycle 4 Week 12 | -1.74 Score on a scale | Standard Deviation 22.378 |
| Retrospective TED/NTT Group | 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 | Physical Functioning: Change at Cycle 4 Week 24 | 1.79 Score on a scale | Standard Deviation 19.998 |
| Retrospective TED/NTT Group | 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 | Physical Functioning: Change at Cycle 5 Week 12 | -7.50 Score on a scale | Standard Deviation 19.961 |
| Retrospective TED/NTT Group | 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 | Physical Functioning: Change at Cycle 5 Week 24 | -2.34 Score on a scale | Standard Deviation 14.292 |
| Retrospective TED/NTT Group | 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 | Physical Functioning: Change at Cycle 6 Week 12 | -6.25 Score on a scale | Standard Deviation 22.244 |
| Retrospective TED/NTT Group | 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 | Physical Functioning: Change at Cycle 6 Week 24 | 3.13 Score on a scale | — |
| Retrospective TED/NTT Group | 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 | Emotional Functioning: Change at Cycle 1 Week 12 | 4.17 Score on a scale | Standard Deviation 18.843 |
| Retrospective TED/NTT Group | 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 | Emotional Functioning: Change at Cycle 1 Week 24 | -4.67 Score on a scale | Standard Deviation 24.511 |
| Retrospective TED/NTT Group | 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 | Emotional Functioning: Change at Cycle 2 Week 12 | -3.33 Score on a scale | Standard Deviation 28.591 |
| Retrospective TED/NTT Group | 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 | Emotional Functioning: Change at Cycle 2 Week 24 | -0.38 Score on a scale | Standard Deviation 21.454 |
| Retrospective TED/NTT Group | 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 | Emotional Functioning: Change at Cycle 3 Week 12 | 7.05 Score on a scale | Standard Deviation 29.661 |
| Retrospective TED/NTT Group | 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 | Emotional Functioning: Change at Cycle 3 Week 24 | -8.33 Score on a scale | Standard Deviation 36.12 |
| Retrospective TED/NTT Group | 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 | Emotional Functioning: Change at Cycle 4 Week 12 | 2.78 Score on a scale | Standard Deviation 33.481 |
| Retrospective TED/NTT Group | 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 | Emotional Functioning: Change at Cycle 4 Week 24 | -0.71 Score on a scale | Standard Deviation 35.786 |
| Retrospective TED/NTT Group | 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 | Emotional Functioning: Change at Cycle 5 Week 12 | -11.00 Score on a scale | Standard Deviation 40.566 |
| Retrospective TED/NTT Group | 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 | Emotional Functioning: Cycle 5 Week 24 | -17.50 Score on a scale | Standard Deviation 37.804 |
| Retrospective TED/NTT Group | 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 | Emotional Functioning: Change at Cycle 6 Week 12 | -5.63 Score on a scale | Standard Deviation 56.065 |
| Retrospective TED/NTT Group | 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 | Emotional Functioning: Change at Cycle 6 Week 24 | 17.50 Score on a scale | — |
| Retrospective TED/NTT Group | 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 | Social Functioning: Change at Cycle 1 Week 12 | -15.00 Score on a scale | — |
| Retrospective TED/NTT Group | 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 | Social Functioning: Change at Cycle 1 Week 24 | -10.00 Score on a scale | — |
| Retrospective TED/NTT Group | 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 | Social Functioning: Change at Cycle 2 Week 12 | -5.00 Score on a scale | — |
| Retrospective TED/NTT Group | 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 | Social Functioning: Change at Cycle 2 Week 24 | 0.00 Score on a scale | — |
| Retrospective TED/NTT Group | 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 | Social Functioning: Change at Cycle 3 Week 12 | 10.00 Score on a scale | — |
| Retrospective TED/NTT Group | 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 | Social Functioning: Change at Cycle 3 Week 24 | 0.00 Score on a scale | — |
| Retrospective TED/NTT Group | 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 | Social Functioning: Change at Cycle 4 Week 12 | 10.00 Score on a scale | — |
| Retrospective TED/NTT Group | 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 | Social Functioning: Change at Cycle 4 Week 24 | 10.00 Score on a scale | — |
| Retrospective TED/NTT Group | 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 | Social Functioning: Change at Cycle 5 Week 12 | 10.00 Score on a scale | — |
| Retrospective TED/NTT Group | 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 | Social Functioning: Change at Cycle 5 Week 24 | 10.00 Score on a scale | — |
| Retrospective TED/NTT Group | 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 | Social Functioning: Change at Cycle 6 Week 12 | 10.00 Score on a scale | — |
| Retrospective TED/NTT Group | 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 | School Functioning: Change at Cycle 1 Week 12 | 5.67 Score on a scale | Standard Deviation 19.445 |
| Retrospective TED/NTT Group | 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 | School Functioning: Change at Cycle 1 Week 24 | 2.00 Score on a scale | Standard Deviation 20.16 |
| Retrospective TED/NTT Group | 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 | School Functioning: Change at Cycle 2 Week 12 | -5.00 Score on a scale | Standard Deviation 24.027 |
| Retrospective TED/NTT Group | 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 | School Functioning: Change at Cycle 2 Week 24 | 8.75 Score on a scale | Standard Deviation 16.394 |
| Retrospective TED/NTT Group | 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 | School Functioning: Change at Cycle 3 Week 12 | 3.64 Score on a scale | Standard Deviation 23.355 |
| Retrospective TED/NTT Group | 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 | School Functioning: Change at Cycle 3 Week 24 | -3.33 Score on a scale | Standard Deviation 22.5 |
| Retrospective TED/NTT Group | 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 | School Functioning: Change at Cycle 4 Week 12 | 0.00 Score on a scale | Standard Deviation 15 |
| Retrospective TED/NTT Group | 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 | School Functioning: Change at Cycle 4 Week 24 | 13.57 Score on a scale | Standard Deviation 8.018 |
| Retrospective TED/NTT Group | 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 | School Functioning: Change at Cycle 5 Week 12 | 15.00 Score on a scale | Standard Deviation 11.18 |
| Retrospective TED/NTT Group | 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 | School Functioning: Change at Cycle 5 Week 24 | 18.75 Score on a scale | Standard Deviation 8.539 |
| Retrospective TED/NTT Group | 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 | School Functioning: Change at Cycle 6 Week 12 | 13.75 Score on a scale | Standard Deviation 4.787 |
| Retrospective TED/NTT Group | 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 | School Functioning: Change at Cycle 6 Week 24 | 20.00 Score on a scale | — |