A Pharmacokinetic, Safety, and Pharmacodynamic Study of Teduglutide in Pediatric Subjects With Short Bowel Syndrome

May 14, 2021 updated by: Shire

A 12-Week Pharmacokinetic, Safety, and Pharmacodynamic Study of Teduglutide in Pediatric Subjects Aged 1 Year Through 17 Years, With Short Bowel Syndrome Who Are Dependent on Parenteral Support

This will be an open label, 4-cohort study. Non-randomized subjects will receive teduglutide in each of the 3 active cohorts. An attempt will be made to enroll additional subjects into an observational cohort who will receive standard of care. Three doses of teduglutide are to be investigated for 12 weeks. All subjects will be screened prior to the start of treatment (SOT) to establish baseline characteristics including safety, eligibility and nutritional support parameters.

Study Overview

Status

Completed

Conditions

Intervention / Treatment

Study Type

Interventional

Enrollment (Actual)

42

Phase

  • Phase 3

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • United Kingdom
      • London, United Kingdom
        • Great Ormond Street Hospital for Children
  • United States
    • Alabama
      • Birmingham, Alabama, United States, 35233
        • Children´s Hospital of Alabama
    • Arkansas
      • Little Rock, Arkansas, United States, 72202
        • Arkansas Children's Hospital
    • California
      • Los Angeles, California, United States, 90024
        • Children's Hospital Los Angeles
      • Los Angeles, California, United States, 90095
        • Mattel Children's Hospital UCLA, Department of Pediatric Gastroenterology
    • Florida
      • Orlando, Florida, United States, 32806
        • Arnold Palmer Hospital for Children
    • Illinois
      • Chicago, Illinois, United States, 60611
        • Ann & Robert H. Lurie Children's Hospital of Chicago
    • Indiana
      • Indianapolis, Indiana, United States, 46202
        • Riley Hospital for Children at IU Health
    • Massachusetts
      • Boston, Massachusetts, United States, 02115
        • Boston Children's Hospital
    • Michigan
      • Ann Arbor, Michigan, United States, 48109
        • Mott Children's Hospital, University of Michigan
    • Missouri
      • Kansas City, Missouri, United States, 64108
        • Childrens Mercy Hospitals & Clinics Pediatric Gastroenterology
    • Nebraska
      • Omaha, Nebraska, United States, 68198
        • University of Nebraska Medical Center
    • New York
      • Bronx, New York, United States, 10467
        • Montefiore Medical Center
    • Ohio
      • Cincinnati, Ohio, United States, 45229
        • Cincinnati Children's Hospital Medical Center
    • Tennessee
      • Memphis, Tennessee, United States, 38103
        • University of Tennessee Health Science Center Le Bonheur Pediatric Specialists
      • Nashville, Tennessee, United States, 37232
        • Monroe Carell, Jr. Children's Hospital at Vanderbilt
    • Texas
      • Houston, Texas, United States, 77030
        • Texas Children's Hospital, Baylor College of Medicine
    • Washington
      • Seattle, Washington, United States, 98105
        • Seattle Childrens Hospital Gastroenterology and Hepatology
    • Wisconsin
      • Madison, Wisconsin, United States, 53792
        • University of Wisconsin School of Medicine and Public Health Surgery

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

1 year to 17 years (CHILD)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Description

Key Inclusion Criteria:

  • Current history of SBS as a result of major intestinal resection, (eg, due to necrotizing enterocolitis, midgut volvulus, intestinal atresia, or gastroschisis) for at least 12 months prior to screening
  • Short bowel syndrome that requires parenteral nutrition/intravenous (PN/IV) support
  • Stable PN/IV support for at least 3 months prior to enrollment based upon the opinion of the investigator

Key Exclusion Criteria:

  • Serial transverse enteroplasty or any other bowel lengthening procedure performed within the past 3 months
  • Unstable absorption due to cystic fibrosis, untreated Hirschsprung's disease or known DNA abnormalities (ie, Familial Adenomatous Polyposis, Fanconi syndrome)
  • Evidence of obstruction on upper gastrointestinal (GI) series done within 6 months prior to screening
  • Major gastrointestinal surgical intervention within 3 months prior to screening (insertion of feeding tube or endoscopic procedure is allowed)

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: TREATMENT
  • Allocation: NON_RANDOMIZED
  • Interventional Model: SINGLE_GROUP
  • Masking: NONE

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
NO_INTERVENTION: Standard of Care
EXPERIMENTAL: teduglutide
Open label teduglutide, subcutaneously injected.
Drug: teduglutide
Open label intervention. Each subject will be assigned to receive 0.0125, 0.025, or 0.05 mg/kg/day for the duration of the study

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Percent Change in Parenteral Support [Parenteral Nutrition (PN)/Intravenous (IV)] Volume at Week 12
Time Frame: Baseline, Week 12
Percent change in PN/IV from the Baseline Visit to Week 12 Visit.
Baseline, Week 12
Percent Change in Parenteral Support (PN/IV) Volume at End of Treatment
Time Frame: Baseline, End of Treatment
Percent change in PN/IV from the Baseline Visit to End of Treatment Visit.
Baseline, End of Treatment
Percent Change in Parenteral Support (PN/IV) Volume at Week 16
Time Frame: Baseline, Week 16
Percent change in PN/IV from the Baseline Visit to Week 16 Visit.
Baseline, Week 16
Absolute Change in Parenteral Support (PN/IV) Volume at Week 12
Time Frame: Baseline, Week 12
Absolute change in PN/IV from the Baseline Visit to Week 12 Visit.
Baseline, Week 12
Absolute Change in Parenteral Support (PN/IV) Volume at End of Treatment
Time Frame: Baseline, End of Treatment
Absolute change in PN/IV from the Baseline Visit to End of Treatment Visit.
Baseline, End of Treatment
Absolute Change in Parenteral Support (PN/IV) Volume at Week 16
Time Frame: Baseline, Week 16
Absolute change in PN/IV from the Baseline Visit to Week 16 Visit.
Baseline, Week 16

Other Outcome Measures

Outcome Measure
Measure Description
Time Frame
Percent Change in Enteral Support (EN) Volume From Baseline at Week 12
Time Frame: Baseline, Week 12
Percent change in enteral support requirements at Week 12 (liters/week)
Baseline, Week 12
Percent Change in Enteral Support (EN) Volume From Baseline at Week 16
Time Frame: Baseline, Week 16
Percent change in enteral support requirements at Week 16 (liters/week)
Baseline, Week 16
Absolute Change in Enteral Support (EN) Volume From Baseline at Week 12
Time Frame: Baseline, Week 12
Absolute change in enteral support requirements at Week 12 (liters/week)
Baseline, Week 12
Absolute Change in Enteral Support (EN) Volume From Baseline at Week 16
Time Frame: Baseline, Week 16
Absolute change in enteral support requirements at Week 16 (liters/week)
Baseline, Week 16

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Shire

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (ACTUAL)

November 14, 2013

Primary Completion (ACTUAL)

January 9, 2015

Study Completion (ACTUAL)

January 9, 2015

Study Registration Dates

First Submitted

September 23, 2013

First Submitted That Met QC Criteria

September 24, 2013

First Posted (ESTIMATE)

September 27, 2013

Study Record Updates

Last Update Posted (ACTUAL)

June 9, 2021

Last Update Submitted That Met QC Criteria

May 14, 2021

Last Verified

May 1, 2021

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • TED-C13-003

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

Clinical Trials on Short Bowel Syndrome

Clinical Trials on teduglutide

Search Similar Trials

Sponsors and Collaborators

Medical Conditions

Drug Interventions

CROs by country

CROs in South Africa

Conditions

Rare Diseases

Drug Interventions

Dietary Supplements

Sponsor/Collaborators

Locations

3
Subscribe