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

NCT01952080 | Completed Phase 3 Results DMC

• 1 other identifier: TED-C13-003
• Study Type: interventional
• Target: 42
• Locations: 2 countries
• Sites: 19

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.

Conditions

Short Bowel Syndrome

Keywords

SBS; Pediatric SBS; Short Bowel Syndrome

Outcome Measures

Primary Outcomes (6)

• 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

• 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

• 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

• 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

• 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

• 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

Other Outcomes (4)

• Percent Change in Enteral Support (EN) Volume From Baseline at Week 12

  Baseline, Week 12

• Percent Change in Enteral Support (EN) Volume From Baseline at Week 16

  Baseline, Week 16

• Absolute Change in Enteral Support (EN) Volume From Baseline at Week 12

  Baseline, Week 12

Study Arms (2)

teduglutide

EXPERIMENTAL

Open label teduglutide, subcutaneously injected.

Drug: teduglutide

Standard of Care

NO INTERVENTION

Interventions

teduglutide DRUG

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

teduglutide

Eligibility Criteria

• Age: 1 Year - 17 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• 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

You may not qualify if:

• 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)

Sponsors & Collaborators

• Shire: lead

Study Sites (19)

Children´s Hospital of Alabama

Birmingham, Alabama, 35233, United States

Location

Arkansas Children's Hospital

Little Rock, Arkansas, 72202, United States

Location

Children's Hospital Los Angeles

Los Angeles, California, 90024, United States

Location

Mattel Children's Hospital UCLA, Department of Pediatric Gastroenterology

Los Angeles, California, 90095, United States

Location

Arnold Palmer Hospital for Children

Orlando, Florida, 32806, United States

Location

Ann & Robert H. Lurie Children's Hospital of Chicago

Chicago, Illinois, 60611, United States

Location

Riley Hospital for Children at IU Health

Indianapolis, Indiana, 46202, United States

Location

Boston Children's Hospital

Boston, Massachusetts, 02115, United States

Location

Mott Children's Hospital, University of Michigan

Ann Arbor, Michigan, 48109, United States

Location

Childrens Mercy Hospitals & Clinics Pediatric Gastroenterology

Kansas City, Missouri, 64108, United States

Location

University of Nebraska Medical Center

Omaha, Nebraska, 68198, United States

Location

Montefiore Medical Center

The Bronx, New York, 10467, United States

Location

Cincinnati Children's Hospital Medical Center

Cincinnati, Ohio, 45229, United States

Location

University of Tennessee Health Science Center Le Bonheur Pediatric Specialists

Memphis, Tennessee, 38103, United States

Location

Monroe Carell, Jr. Children's Hospital at Vanderbilt

Nashville, Tennessee, 37232, United States

Location

Texas Children's Hospital, Baylor College of Medicine

Houston, Texas, 77030, United States

Location

Seattle Childrens Hospital Gastroenterology and Hepatology

Seattle, Washington, 98105, United States

Location

University of Wisconsin School of Medicine and Public Health Surgery

Madison, Wisconsin, 53792, United States

Location

Great Ormond Street Hospital for Children

London, United Kingdom

Location

Related Publications (2)

• Fifi A, Raphael BP, Terreri B, Uddin S, Kaufman SS. Effects of Teduglutide on Diarrhea in Pediatric Patients with Short Bowel Syndrome-Associated Intestinal Failure. J Pediatr Gastroenterol Nutr. 2023 Nov 1;77(5):666-671. doi: 10.1097/MPG.0000000000003922. Epub 2023 Aug 22.

  PMID: 37889619 DERIVED

• 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.

  PMID: 27855998 DERIVED

MeSH Terms

Conditions

Short Bowel Syndrome

Interventions

teduglutide

Condition Hierarchy (Ancestors)

Malabsorption Syndromes; Intestinal Diseases; Gastrointestinal Diseases; Digestive System Diseases; Postoperative Complications; Pathologic Processes; Pathological Conditions, Signs and Symptoms

Results Point of Contact

• Title: Study Director
• Organization: Shire

ClinicalTransparency@shire.com

+1 866 842 5335

Study Officials

• Study Director

  Takeda

  STUDY DIRECTOR

Publication Agreements

• PI is Sponsor Employee: No
• Restrictive Agreement: No

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: September 23, 2013
• First Posted: September 27, 2013
• Study Start: November 14, 2013
• Primary Completion: January 9, 2015
• Study Completion: January 9, 2015
• Last Updated: June 9, 2021
• Results First Posted: September 14, 2015

Record last verified: 2021-05

Locations

GB (1); US (18)