Pediatric Teduglutide Registry (PTR)

Boston Children's Hospital

Status: Completed

Conditions: Short Bowel Syndrome

Treatments: Drug: Teduglutide

Study type: Observational

Funder types: Other; Industry

Identifiers

NCT04832087

P00033923

Details and patient eligibility

About

Pediatric specific post-marketing registry to evaluate the long-term safety and efficacy of teduglutide.

Full 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.

Enrollment: 142 patients

Sex: All

Ages: 1+ year old

Volunteers: No Healthy Volunteers

Inclusion and exclusion 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.

Exclusion Criteria:

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

Trial design

142 participants in 2 patient groups

Cohort A

Description:

Participants with SBS who started Teduglutide after FDA approval (May 2019)

Treatment:

Drug: Teduglutide

Cohort B

Description:

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

Treatment:

Drug: Teduglutide