Pediatric Teduglutide Registry

Completed

• Conditions: Short Bowel Syndrome
• Interventions: Drug: Teduglutide

• Registration Number: NCT04832087
• Lead Sponsor: Boston Children's Hospital

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.

Study Timeline

• Study First Submitted: 2021-03-29
• Study First Submitted QC: 2021-04-01
• Study First Posted: 2021-04-05
• Study Start: 2021-05-18
• Primary Completion: 2023-08-31
• Study Completion: 2023-08-31
• Status Verified: 2023-12
• Last Update Submitted: 2023-12-08
• Last Update Posted: 2023-12-11

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 142

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.

Exclusion Criteria:

1. In the opinion of the investigator, the subject or guardian is at high risk for non-compliance.

Study & Design

• Study Type: OBSERVATIONAL
• Study Design: Not specified

Arm && Interventions

Group	Intervention	Description
Cohort A	Teduglutide	Participants with SBS who started Teduglutide after FDA approval (May 2019)
Cohort B	Teduglutide	Subjects who participated in any of the teduglutide pediatric clinical trial studies (TED-C13-003, TED-C14-006, SHP633-303, or SHP633-304) and subsequently started therapy with teduglutide

Primary Outcome Measures

Name	Time	Method
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 Outcome Measures

Name	Time	Method
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
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
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.
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.
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.

Trial Locations

Locations (1)

Boston Children's Hospital; 🇺🇸 Boston, Massachusetts, United States