Phase 3
N=29
Long-term Study of Teduglutide in Pediatric Subjects With Short Bowel Syndrome Who Completed the TED-C13-003 Study
Short Bowel Syndrome
Bottom Line
View on ClinicalTrials.gov: NCT02949362 ↗Enrolled (actual)
29
Serious AEs
88.7%
Results posted
Jun 2021
Primary outcome: Primary: Number of Participants With Adverse Events (AEs) in Retrospective Observation Period — 23; 4; 23; 4 Participants
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 3
- Interventions
- Teduglutide (Drug); SOC (Other)
- Age
- Pediatric, Adult, Older Adult
- Sex
- All
- Sponsor
- Shire
- Primary completion
- Jul 2020
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Number of Participants With Adverse Events (AEs) in Retrospective Observation Period |
23; 4; 23; 4; 23; 4 | — |
| PRIMARY Change From Baseline in Height for Age Z-score up to Week 168 of Retrospective Observation Period |
0.128 | — |
| PRIMARY Change From Baseline in Body Weight for Age Z-score up to Week 168 of Retrospective Observation Period |
-0.181 | — |
| PRIMARY Change From Baseline in Body Mass Index (BMI) for Age Z-score up to Week 168 of Retrospective Observation Period |
-0.283 | — |
| PRIMARY Number of Participants With Treatment Emergent Adverse Events (TEAEs) of Prospective Study Period |
4; 19; 3; 17; 0; 0 | — |
| 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 |
0.05 | — |
| 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 |
-0.66 | — |
| 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 |
-0.68 | — |
| PRIMARY 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 | — |
| PRIMARY 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 | — |
| PRIMARY 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 | — |
| PRIMARY Average Number of Stools Per Day up to Week 120 During the End of NTT Period of Prospective Study Period |
3.75 | — |
| PRIMARY Number of Participants With Positive Specific Antibodies at EOS During the End of Teduglutide Treatment Period of Prospective Study Period |
1 | — |
| 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 |
12; 2; 9; 2; 9; 4 | — |
| SECONDARY Change From Baseline in PS Volume at 12 Weeks Interval up to Week 156 of Retrospective Observation Period |
-14.45; -12.84; -9.27; -13.49; -10.65; -17.91 | — |
| SECONDARY Percent Change From Baseline in PS Volume at 12 Weeks Interval up to Week 156 of Retrospective Observation Period |
-24.50; -24.88; -10.72; -32.10; -13.21; -43.09 | — |
| SECONDARY Change From Baseline in PS Caloric Intake at 12 Weeks Interval up to Week 156 of Retrospective Observation Period |
-10.15; -2.64; -3.96; -7.25; -8.38; -6.42 | — |
| SECONDARY Percent Change From Baseline in PS Caloric Intake at 12 Weeks Interval up to Week 156 of Retrospective Observation Period |
-24.91; 7.64; -10.12; -21.29; -18.29; -3.38 | — |
| 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 |
— | — |
| 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 |
— | — |
| 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 |
-0.38; -1.20; -0.29; -1.00; -0.43; -2.60 | — |
| 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 |
-6.25; -14.86; -4.17; -14.29; -6.21; -34.86 | — |
| 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 |
13; 12; 10; 9; 7; 5 | — |
| SECONDARY Change From Baseline in PS Volume at EOT of Each Cycle During the End of Teduglutide Treatment Period of Prospective Study Period |
-25.18; -28.59; -40.05; -40.10; -40.49; -47.57 | — |
| SECONDARY Percent Change From Baseline in PS Volume at EOT of Each Cycle During the End of Teduglutide Treatment Period of Prospective Study Period |
-38.02; -45.58; -62.16; -62.92; -73.49; -78.75 | — |
| SECONDARY Change From Baseline in PS Caloric Intake at EOT of Each Cycle During the End of Teduglutide Treatment Period of Prospective Study Period |
-17.66; -19.95; -27.66; -24.05; -26.98; -27.53 | — |
| 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 |
-33.50; -42.06; -62.47; -49.51; -54.08; -59.00 | — |
| 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 |
1; 3; 6; 5; 4; 3 | — |
| 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 |
-3.40; -4.80; -6.69; -6.48; -6.71; -8.08 | — |
| 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 |
-26.32; -40.47; -57.61; -55.87; -58.85; -66.39 | — |
| 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 |
-0.60; -1.93; -3.03; -2.99; -3.83; -4.37 | — |
| 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 |
-9.01; -29.24; -44.51; -42.38; -54.29; -62.86 | — |
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.
Eligibility Criteria
Inclusion Criteria
- Subject provides written informed consent (subject, parent or legal guardian and, as appropriate, subject informed assent) to participate in the study before completing any study-related procedures.
- Subject completed the TED-C13-003 study (including subjects in the standard of care treatment arm).
- Subject understands and is willing and able to fully adhere to study requirements as defined in this protocol.
Exclusion Criteria
There are no exclusion criteria for this study.
Data sourced from ClinicalTrials.gov (NCT02949362). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.