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

Short Bowel Syndrome Clinical Trial

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

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT01952080
• Other study ID #: TED-C13-003
• Status: Completed
• Phase: Phase 3
• Start date: November 14, 2013
• Est. completion date: January 9, 2015

Study information

• Verified date: May 2021
• Source: Takeda
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 42
• Est. completion date: January 9, 2015
• Est. primary completion date: January 9, 2015
• Accepts healthy volunteers: No
• Gender: All
• Age group: 1 Year to 17 Years

Eligibility

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)

Related Conditions & MeSH terms

• Short Bowel Syndrome
• Syndrome

Intervention

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

Locations

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

Sponsors (1)

Lead Sponsor	Collaborator
Shire

Countries where clinical trial is conducted

United States,  United Kingdom, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Other	Percent Change in Enteral Support (EN) Volume From Baseline at Week 12	Percent change in enteral support requirements at Week 12 (liters/week)	Baseline, Week 12
Other	Percent Change in Enteral Support (EN) Volume From Baseline at Week 16	Percent change in enteral support requirements at Week 16 (liters/week)	Baseline, Week 16
Other	Absolute Change in Enteral Support (EN) Volume From Baseline at Week 12	Absolute change in enteral support requirements at Week 12 (liters/week)	Baseline, Week 12
Other	Absolute Change in Enteral Support (EN) Volume From Baseline at Week 16	Absolute change in enteral support requirements at Week 16 (liters/week)	Baseline, Week 16
Primary	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.	Baseline, Week 12
Primary	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.	Baseline, End of Treatment
Primary	Percent Change in Parenteral Support (PN/IV) Volume at Week 16	Percent change in PN/IV from the Baseline Visit to Week 16 Visit.	Baseline, Week 16
Primary	Absolute Change in Parenteral Support (PN/IV) Volume at Week 12	Absolute change in PN/IV from the Baseline Visit to Week 12 Visit.	Baseline, Week 12
Primary	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.	Baseline, End of Treatment
Primary	Absolute Change in Parenteral Support (PN/IV) Volume at Week 16	Absolute change in PN/IV from the Baseline Visit to Week 16 Visit.	Baseline, Week 16