Pediatric Teduglutide Registry (PTR)

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

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

Study Overview

• Status: Completed
• Conditions: Short Bowel Syndrome
• Intervention / Treatment: Drug: 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 Type: Observational
• Enrollment (Actual): 142

Contacts and Locations

• Study Contact: Name: Lissette Jimenez, MD; Phone Number: 6173554806; Email: lissette.jimenez@childrens.harvard.edu

Study Locations

• United States
  • Massachusetts
    • Boston, Massachusetts, United States, 02115
      • Boston Children's Hospital

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 1 year and older (Child, Adult, Older Adult)
• Accepts Healthy Volunteers: No

• Sampling Method: Non-Probability Sample

Study Population

Children with SBS on Teduglutide

Description

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.

Study Plan

How is the study designed?

Design Details

• Observational Models: Cohort
• Time Perspectives: Prospective

Number of groups / cohorts

2

Cohorts and Interventions

Group / Cohort	Intervention / Treatment
Cohort A; Participants with SBS who started Teduglutide after FDA approval (May 2019)	Drug: Teduglutide; Standard of care Teduglutide dosing; Other Names: Gattex
Cohort B; 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	Drug: Teduglutide; Standard of care Teduglutide dosing; Other Names: Gattex

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
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 Outcome Measures

Outcome Measure	Measure Description	Time Frame
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
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
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
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
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
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
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

Collaborators and Investigators

• Sponsor: Boston Children's Hospital
• Collaborators: Takeda
• Investigators: Principal Investigator: Lissette Jimenez, Boston Children's Hospital

Publications and helpful links

General Publications

• 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 24-Week, Phase III Study. JPEN J Parenter Enteral Nutr. 2020 May;44(4):621-631. doi: 10.1002/jpen.1690. Epub 2019 Sep 8.
• 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-Week, Open-Label, Multicenter Clinical Trial of Teduglutide in Pediatric Short Bowel Syndrome. J Pediatr. 2017 Feb;181:102-111.e5. doi: 10.1016/j.jpeds.2016.10.027. Epub 2016 Nov 15.

Study record dates

Study Major Dates

• Study Start (Actual): May 18, 2021
• Primary Completion (Actual): August 31, 2023
• Study Completion (Actual): August 31, 2023

Study Registration Dates

• First Submitted: March 29, 2021
• First Submitted That Met QC Criteria: April 1, 2021
• First Posted (Actual): April 5, 2021

Study Record Updates

• Last Update Posted (Estimated): December 11, 2023
• Last Update Submitted That Met QC Criteria: December 8, 2023
• Last Verified: December 1, 2023

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Digestive System Diseases; Pathologic Processes; Postoperative Complications; Gastrointestinal Diseases; Intestinal Diseases; Malabsorption Syndromes; Short Bowel Syndrome; Physiological Effects of Drugs; Gastrointestinal Agents; Protective Agents; Radiation-Protective Agents; Teduglutide
• Other Study ID Numbers: P00033923

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No
• product manufactured in and exported from the U.S.: No