Back to resultsA Pharmacokinetic, Safety, and Pharmacodynamic Study of Teduglutide in Pediatric Subjects With Short Bowel Syndrome
Primary Purpose
Short Bowel Syndrome
Status
Completed
Phase
Phase 3
Locations
International
Study Type
Interventional
Intervention
teduglutide
Sponsored by
About this trial
This is an interventional treatment trial for Short Bowel Syndrome focused on measuring SBS, Pediatric SBS, Short Bowel Syndrome
Eligibility Criteria
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)
Sites / Locations
- Children´s Hospital of Alabama
- Arkansas Children's Hospital
- Children's Hospital Los Angeles
- Mattel Children's Hospital UCLA, Department of Pediatric Gastroenterology
- Arnold Palmer Hospital for Children
- Ann & Robert H. Lurie Children's Hospital of Chicago
- Riley Hospital for Children at IU Health
- Boston Children's Hospital
- Mott Children's Hospital, University of Michigan
- Childrens Mercy Hospitals & Clinics Pediatric Gastroenterology
- University of Nebraska Medical Center
- Montefiore Medical Center
- Cincinnati Children's Hospital Medical Center
- University of Tennessee Health Science Center Le Bonheur Pediatric Specialists
- Monroe Carell, Jr. Children's Hospital at Vanderbilt
- Texas Children's Hospital, Baylor College of Medicine
- Seattle Childrens Hospital Gastroenterology and Hepatology
- University of Wisconsin School of Medicine and Public Health Surgery
- Great Ormond Street Hospital for Children
Arms of the Study
Arm 1
Arm 2
Arm Type
Experimental
No Intervention
Arm Label
teduglutide
Standard of Care
Arm Description
Open label teduglutide, subcutaneously injected.
Outcomes
Primary Outcome Measures
Percent Change in Parenteral Support [Parenteral Nutrition (PN)/Intravenous (IV)] Volume at Week 12
Percent change in PN/IV from the Baseline Visit to Week 12 Visit.
Percent Change in Parenteral Support (PN/IV) Volume at End of Treatment
Percent change in PN/IV from the Baseline Visit to End of Treatment Visit.
Percent Change in Parenteral Support (PN/IV) Volume at Week 16
Percent change in PN/IV from the Baseline Visit to Week 16 Visit.
Absolute Change in Parenteral Support (PN/IV) Volume at Week 12
Absolute change in PN/IV from the Baseline Visit to Week 12 Visit.
Absolute Change in Parenteral Support (PN/IV) Volume at End of Treatment
Absolute change in PN/IV from the Baseline Visit to End of Treatment Visit.
Absolute Change in Parenteral Support (PN/IV) Volume at Week 16
Absolute change in PN/IV from the Baseline Visit to Week 16 Visit.
Secondary Outcome Measures
Full Information
1. Study Identification
Unique Protocol Identification Number
NCT01952080
Brief Title
A Pharmacokinetic, Safety, and Pharmacodynamic Study of Teduglutide in Pediatric Subjects With Short Bowel Syndrome
Official Title
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
Study Type
Interventional
2. Study Status
Record Verification Date
May 2021
Overall Recruitment Status
Completed
Study Start Date
November 14, 2013 (Actual)
Primary Completion Date
January 9, 2015 (Actual)
Study Completion Date
January 9, 2015 (Actual)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Shire
4. Oversight
Data Monitoring Committee
Yes
5. Study Description
Brief Summary
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.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Short Bowel Syndrome
Keywords
SBS, Pediatric SBS, Short Bowel Syndrome
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 3
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
Non-Randomized
Enrollment
42 (Actual)
8. Arms, Groups, and Interventions
Arm Title
teduglutide
Arm Type
Experimental
Arm Description
Open label teduglutide, subcutaneously injected.
Arm Title
Standard of Care
Arm Type
No Intervention
Intervention Type
Drug
Intervention Name(s)
teduglutide
Intervention Description
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
Primary Outcome Measure Information:
Title
Percent Change in Parenteral Support [Parenteral Nutrition (PN)/Intravenous (IV)] Volume at Week 12
Description
Percent change in PN/IV from the Baseline Visit to Week 12 Visit.
Time Frame
Baseline, Week 12
Title
Percent Change in Parenteral Support (PN/IV) Volume at End of Treatment
Description
Percent change in PN/IV from the Baseline Visit to End of Treatment Visit.
Time Frame
Baseline, End of Treatment
Title
Percent Change in Parenteral Support (PN/IV) Volume at Week 16
Description
Percent change in PN/IV from the Baseline Visit to Week 16 Visit.
Time Frame
Baseline, Week 16
Title
Absolute Change in Parenteral Support (PN/IV) Volume at Week 12
Description
Absolute change in PN/IV from the Baseline Visit to Week 12 Visit.
Time Frame
Baseline, Week 12
Title
Absolute Change in Parenteral Support (PN/IV) Volume at End of Treatment
Description
Absolute change in PN/IV from the Baseline Visit to End of Treatment Visit.
Time Frame
Baseline, End of Treatment
Title
Absolute Change in Parenteral Support (PN/IV) Volume at Week 16
Description
Absolute change in PN/IV from the Baseline Visit to Week 16 Visit.
Time Frame
Baseline, Week 16
Other Pre-specified Outcome Measures:
Title
Percent Change in Enteral Support (EN) Volume From Baseline at Week 12
Description
Percent change in enteral support requirements at Week 12 (liters/week)
Time Frame
Baseline, Week 12
Title
Percent Change in Enteral Support (EN) Volume From Baseline at Week 16
Description
Percent change in enteral support requirements at Week 16 (liters/week)
Time Frame
Baseline, Week 16
Title
Absolute Change in Enteral Support (EN) Volume From Baseline at Week 12
Description
Absolute change in enteral support requirements at Week 12 (liters/week)
Time Frame
Baseline, Week 12
Title
Absolute Change in Enteral Support (EN) Volume From Baseline at Week 16
Description
Absolute change in enteral support requirements at Week 16 (liters/week)
Time Frame
Baseline, Week 16
10. Eligibility
Sex
All
Minimum Age & Unit of Time
1 Year
Maximum Age & Unit of Time
17 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
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)
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Study Director
Organizational Affiliation
Takeda
Official's Role
Study Director
Facility Information:
Facility Name
Children´s Hospital of Alabama
City
Birmingham
State/Province
Alabama
ZIP/Postal Code
35233
Country
United States
Facility Name
Arkansas Children's Hospital
City
Little Rock
State/Province
Arkansas
ZIP/Postal Code
72202
Country
United States
Facility Name
Children's Hospital Los Angeles
City
Los Angeles
State/Province
California
ZIP/Postal Code
90024
Country
United States
Facility Name
Mattel Children's Hospital UCLA, Department of Pediatric Gastroenterology
City
Los Angeles
State/Province
California
ZIP/Postal Code
90095
Country
United States
Facility Name
Arnold Palmer Hospital for Children
City
Orlando
State/Province
Florida
ZIP/Postal Code
32806
Country
United States
Facility Name
Ann & Robert H. Lurie Children's Hospital of Chicago
City
Chicago
State/Province
Illinois
ZIP/Postal Code
60611
Country
United States
Facility Name
Riley Hospital for Children at IU Health
City
Indianapolis
State/Province
Indiana
ZIP/Postal Code
46202
Country
United States
Facility Name
Boston Children's Hospital
City
Boston
State/Province
Massachusetts
ZIP/Postal Code
02115
Country
United States
Facility Name
Mott Children's Hospital, University of Michigan
City
Ann Arbor
State/Province
Michigan
ZIP/Postal Code
48109
Country
United States
Facility Name
Childrens Mercy Hospitals & Clinics Pediatric Gastroenterology
City
Kansas City
State/Province
Missouri
ZIP/Postal Code
64108
Country
United States
Facility Name
University of Nebraska Medical Center
City
Omaha
State/Province
Nebraska
ZIP/Postal Code
68198
Country
United States
Facility Name
Montefiore Medical Center
City
Bronx
State/Province
New York
ZIP/Postal Code
10467
Country
United States
Facility Name
Cincinnati Children's Hospital Medical Center
City
Cincinnati
State/Province
Ohio
ZIP/Postal Code
45229
Country
United States
Facility Name
University of Tennessee Health Science Center Le Bonheur Pediatric Specialists
City
Memphis
State/Province
Tennessee
ZIP/Postal Code
38103
Country
United States
Facility Name
Monroe Carell, Jr. Children's Hospital at Vanderbilt
City
Nashville
State/Province
Tennessee
ZIP/Postal Code
37232
Country
United States
Facility Name
Texas Children's Hospital, Baylor College of Medicine
City
Houston
State/Province
Texas
ZIP/Postal Code
77030
Country
United States
Facility Name
Seattle Childrens Hospital Gastroenterology and Hepatology
City
Seattle
State/Province
Washington
ZIP/Postal Code
98105
Country
United States
Facility Name
University of Wisconsin School of Medicine and Public Health Surgery
City
Madison
State/Province
Wisconsin
ZIP/Postal Code
53792
Country
United States
Facility Name
Great Ormond Street Hospital for Children
City
London
Country
United Kingdom
12. IPD Sharing Statement
Citations:
PubMed Identifier
27855998
Citation
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.
Results Reference
derived
Learn more about this trial
A Pharmacokinetic, Safety, and Pharmacodynamic Study of Teduglutide in Pediatric Subjects With Short Bowel Syndrome
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