Short Bowel Syndrome
Conditions
Brief summary
Pediatric specific post-marketing registry to evaluate the long-term safety and efficacy of teduglutide.
Detailed description
This will be a multi-center post-marketing pediatric registry study evaluating the efficacy and safety of teduglutide. This is an observational longitudinal registry of pediatric SBS patients who are using FDA approved teduglutide as per standard of care.
Interventions
DRUGTeduglutide
Standard of care Teduglutide dosing
Sponsors
Takeda
Boston Children's Hospital
Study design
Observational model
COHORT
Time perspective
PROSPECTIVE
Eligibility
Sex/Gender
ALL
Age
1 Years to No maximum
Healthy volunteers
No
Inclusion criteria
Cohort A: Inclusion Criteria: 1. Diagnosis of SBS defined as intestinal resection for congenital or acquired intestinal disease leading to dependence on PS for 90 days 2. Received teduglutide after FDA approval 3. Age ≥ 1 years and ≤ 18 years at start of teduglutide initiation 4. Weight ≥ 10 kg at start of teduglutide initiation 5. Dependent on PS at the time of teduglutide initiation 6. Subject data for primary outcome (PS energy intake in cal/kg/day) is available for medical chart abstraction at baseline (pretreatment) visit. 7. The subject or guardian signs and dates a written informed consent form and any required privacy authorization and minor provides any assent required prior to enrolment into the registry.
Exclusion criteria
1. Participation in the pediatric clinical trial studies of teduglutide (TED-C13-003, TED-C14-006 or SHP633-303, SHP633-304). 2. In the opinion of the investigator, the subject or guardian is at high risk for non-compliance. Cohort B: Inclusion Criteria: 1. Diagnosis of SBS defined as intestinal resection for congenital or acquired intestinal disease leading to dependence on PS for 90 days 2. Participated in clinical trial study (TED-C13-003, TED-C14-006) and/or extension study (SHP633-303, SHP633-304) 3. Currently receiving teduglutide 4. The subject or guardian signs and dates a written informed consent form and any required privacy authorization and minor provides any assent required prior to enrolment into the registry.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Percent change in calories from parenteral support (PS) (Kcal/kg/day) | Up to 3 years | Maximum percent change in calories (energy intake in kcal/kg/day) from parenteral support relative to pre- treatment (baseline) assessment up to 6 months, 1 year, 2 years and 3 years from start of therapy among Cohort A. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Percent change of PS infusions (number of days/week ) | Up to 3 years | Percent change of PS infusions (number of days/week relative to pre-treatment (baseline) assessment up to 6 months, 1 year, 2 years and 3 years from start of therapy among Cohort A |
| Proportion of subjects completely weaned from PS | Up to 3 years | Number (proportion) of children who wean completely from PS within 6 months, 1 year, 2 years and 3 years from start of therapy among Cohort A. |
| Adverse Events | Up to 3 years | Number (proportion) of subjects with adverse events among Cohort A. Number (proportion) of subjects with adverse events among Cohort B. |
| Percent change of (PS) volume (liters/week) | Up to 3 years | Percent change of PS volume (liters/week) relative to pre-treatment (baseline) assessment up to 6 months, 1 year, 2 years and 3 years from start of therapy among Cohort A |
| Height-for-age-Z-score (HAZ) | Up to 3 years | Mean change in Height-for-age-Z-score (HAZ), relative to pre-treatment (baseline) assessment up to 6 months, 1 year, 2 years and 3 years from start of therapy among Cohort A. |
| Body-Mass-Index Z-score (BMIZ) | Up to 3 years | Mean change in Body-Mass-Index Z-score (BMIZ), relative to pre-treatment (baseline) assessment up to 6 months, 1 year, 2 years and 3 years from start of therapy among Cohort A. |
| Weight-for-age Z-score (WAZ) | Up to 3 years | Mean change in Weight-for-age Z-score (WAZ), relative to pre-treatment (baseline) assessment up to 6 months, 1 year, 2 years and 3 years from start of therapy among Cohort A. |
Countries
United States
Outcome results
None listed