Pediatric Teduglutide Registry

Short Bowel Syndrome Clinical Trial

— PTR  

Official title:

Pediatric Teduglutide Registry: Clinical Use of Teduglutide in Children With Intestinal Failure, a Multicenter Post-marketing Evaluation

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT04832087
• Other study ID #: P00033923
• Status: Completed
• Start date: May 18, 2021
• Est. completion date: August 31, 2023

Study information

• Verified date: December 2023
• Source: Boston Children's Hospital
• Contact: n/a
• Is FDA regulated: No
• Study type: Observational [Patient Registry]

Clinical Trial Summary

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

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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 142
• Est. completion date: August 31, 2023
• Est. primary completion date: August 31, 2023
• Accepts healthy volunteers: No
• Gender: All
• Age group: 1 Year and older

Eligibility

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.

Related Conditions & MeSH terms

• Short Bowel Syndrome

Intervention

Drug:
• Teduglutide
Standard of care Teduglutide dosing

Locations

Country	Name	City	State
United States	Boston Children's Hospital	Boston	Massachusetts

Sponsors (2)

Lead Sponsor	Collaborator
Boston Children's Hospital	Takeda

Country where clinical trial is conducted

United States, 

References & Publications (2)

Carter BA, Cohran VC, Cole CR, Corkins MR, Dimmitt RA, Duggan C, Hill S, Horslen S, Lim JD, Mercer DF, Merritt RJ, Nichol PF, Sigurdsson L, Teitelbaum DH, Thompson J, Vanderpool C, Vaughan JF, Li B, Youssef NN, Venick RS, Kocoshis SA. Outcomes from a 12-W — View Citation

Kocoshis SA, Merritt RJ, Hill S, Protheroe S, Carter BA, Horslen S, Hu S, Kaufman SS, Mercer DF, Pakarinen MP, Venick RS, Wales PW, Grimm AA. Safety and Efficacy of Teduglutide in Pediatric Patients With Intestinal Failure due to Short Bowel Syndrome: A 2 — View Citation

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Percent change in calories from parenteral support (PS) (Kcal/kg/day)	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.	Up to 3 years
Secondary	Percent change of (PS) volume (liters/week)	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	Up to 3 years
Secondary	Percent change of PS infusions (number of days/week )	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	Up to 3 years
Secondary	Proportion of subjects completely weaned from PS	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.	Up to 3 years
Secondary	Adverse Events	Number (proportion) of subjects with adverse events among Cohort A. Number (proportion) of subjects with adverse events among Cohort B.	Up to 3 years
Secondary	Weight-for-age Z-score (WAZ)	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.	Up to 3 years
Secondary	Height-for-age-Z-score (HAZ)	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.	Up to 3 years
Secondary	Body-Mass-Index Z-score (BMIZ)	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.	Up to 3 years