PEDIATRIC TEDUGLUTIDE REGISTRY
This is a multicenter post-marketing registry for children with short bowel syndrome who started teduglutide as part of their standard of care. Teduglutide has been approved for use in the US for pediatric patients 1 year of age and older who are dependent on parenteral support. Published clinical trials in pediatrics thus far have been limited to 12 and 24 weeks in duration. This registry will provide additional data on the long-term efficacy and safety outcomes.
Industry Partner
Takeda Pharmaceutic International is our leading partner in this registry.
Principal Investigators
Dr. Lissette Jimenez
Dr. Christopher Duggan
Dr. Alexandra Carey
Statistical and Data Coordinating Center
Dr. Edie Weller
Dr. Suzanne Dahlberg
Clinical Sites and Partners
Boston Children’s Hospital is partnering with 15 hospitals and health centers to evaluate the safety and efficacy of teduglutide in children. This research is supported by Boston Children’s and a collaborative research funding grant from Takeda Pharmaceuticals U.S.A Inc. Central reporting will be through the Statistical and Data Coordinating Center at Boston Children's within the Institutional Centers for Clinical and Translational Research.
2021
Year Established
14
Hospitals and Health Centers
20+
Years of data management experience
160
Accrual goal
Participating Centers
Study Information
PURPOSE
The goal of this study is to evaluate the long-term efficacy and safety of teduglutide in children with short bowel syndrome. Published clinical trials in pediatrics thus far have been limited to 12 and 24 weeks in duration. A post-marketing registry is important in addressing long-term efficacy and safety outcomes in pediatric patients with short bowel syndrome who are undergoing intestinal adaptation, and who are at high risk for intestinal failure associated comorbidities.
DISEASE
Short bowel syndrome is a serious and chronic malabsorption disorder in which the body fails to absorb enough nutrients from food and drink to function normally. Short bowel syndrome is a congenital or acquired disease of the gastrointestinal tract. Consequently, patients with short bowel syndrome often require parenteral support to help support hydration and growth. However, parenteral support does not increase intestinal absorption and long-term use is associated with several complications.
TREATMENT
Teduglutide is a known GLP-2 receptor agonist that promotes mucosal surface growth. It is USFDA approved for use in children with short bowel syndrome for the indication of reducing parenteral nutrition support and increasing nutrient absorption.
Participants
Who can participate?
Patients who started therapy with teduglutide after FDA approval (May 17, 2019) or 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.
How long is the study?
Data will be collected up to 3 years from the start of teduglutide treatment or the last visit of the patient’s final teduglutide study visit, depending on which cohort the patient belongs to.
What does the study involve?
This is an observational study, so only clinical data/test results from the patient’s standard of care encounters are collected. Participation in this registry will not affect standard of care and patients will not be asked to attend additional clinical visits for the study.
What are the benefits to patients?
Research findings may help address the long-term efficacy and safety outcomes of teduglutide in children with short bowel syndrome.
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About Us
Principal Investigator
Co-Director Congenital Enteropathy Program; Attending Physician, Division of Gastroenterology, Hepatology, and Nutrition, Boston Children’s Hospital
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Co-investigator
Senior Physician, Division of Gastroenterology, Hepatology, and Nutrition; Director, Center for Nutrition; Medical Director, Center for Advanced Intestinal Rehabilitation, Boston Children’s Hospital.
Co-investigator
Director, Home Parenteral Nutrition Program; Attending Physician, Division of Gastroenterology, Hepatology, and Nutrition, Boston Children’s Hospital.
The Statistical and Data Coordinating Center
Statistical and Data Coordinating Center (SDCC) for this registry is located within the Institutional Centers for Clinical and Translational Research (ICCTR) at Boston Children’s Hospital. The ICCTR provides infrastructure support for the conduct of clinical research at the hospital and has provided support for many studies (>80 studies over 4 years, of which approximately 40% are multi-center). SDCC Members provide expertise in the following areas:
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Project Management
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Biostatistics
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Data Management
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Database Development and Programming
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Clinical Research Coordination
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Web design
SDCC Leadership Team
The team has more than 20 years of experience in the management of clinical trials, registry studies, and any corresponding correlative studies. The SDCC Leadership Team is responsible for administrative coordination, communication, study planning, data management, quality assurance and quality control procedures, statistical design, conduct, analysis, and reporting.
Edie Weller, PhD
Co-Director of the Statistical and Data Coordinating Center
Director of Biostatistics and Research Design Center,
Institutional Centers of Clinical and Translational Research
Boston Children’s Hospital
Suzanne Dahlberg, PhD
Co-Director of the Statistical and Data Coordinating Center
Biostatistics and Research Design Center, Institutional Centers of Clinical and Translational Research
Boston Children’s Hospital
Maggie Malsch, MSN, RN, CPHON
Co-Director of Study Operations
Clinical Research Operations Director, Institutional Centers of Clinical and Translational Research
Boston Children's Hospital
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Cindy Williams, DNP, RN, PNP, NE-BC
Co-Director of Study Operations
Nursing Director, ETU, Institutional Centers for Clinical and Translational Research
Daniel Elman, MS, MPH
Project Manager
Clinical Research Manager, Institutional Centers for Clinical and Translational Research