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

This study has been completed.
Sponsor:
Information provided by (Responsible Party):
Shire
ClinicalTrials.gov Identifier:
NCT01952080
First received: September 23, 2013
Last updated: November 10, 2015
Last verified: July 2015
  Purpose
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.

Condition Intervention Phase
Short Bowel Syndrome
Drug: teduglutide
Phase 3

Study Type: Interventional
Study Design: Allocation: Non-Randomized
Endpoint Classification: Safety Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
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

Resource links provided by NLM:


Further study details as provided by Shire:

Primary Outcome Measures:
  • Percent Change in Parenteral Support [Parenteral Nutrition (PN)/Intravenous (IV)] Volume at Week 12 [ Time Frame: Baseline, Week 12 ] [ Designated as safety issue: No ]
    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 [ Time Frame: Baseline, End of Treatment ] [ Designated as safety issue: No ]
    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 [ Time Frame: Baseline, Week 16 ] [ Designated as safety issue: No ]
    Percent change in PN/IV from the Baseline Visit to Week 16 Visit.

  • Absolute Change in Parenteral Support (PN/IV) Volume at Week 12 [ Time Frame: Baseline, Week 12 ] [ Designated as safety issue: No ]
    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 [ Time Frame: Baseline, End of Treatment ] [ Designated as safety issue: No ]
    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 [ Time Frame: Baseline, Week 16 ] [ Designated as safety issue: No ]
    Absolute change in PN/IV from the Baseline Visit to Week 16 Visit.


Other Outcome Measures:
  • Percent Change in Enteral Support (EN) Volume From Baseline at Week 12 [ Time Frame: Baseline, Week 12 ] [ Designated as safety issue: No ]
    Percent change in enteral support requirements at Week 12 (liters/week)

  • Percent Change in Enteral Support (EN) Volume From Baseline at Week 16 [ Time Frame: Baseline, Week 16 ] [ Designated as safety issue: No ]
    Percent change in enteral support requirements at Week 16 (liters/week)

  • Absolute Change in Enteral Support (EN) Volume From Baseline at Week 12 [ Time Frame: Baseline, Week 12 ] [ Designated as safety issue: No ]
    Absolute change in enteral support requirements at Week 12 (liters/week)

  • Absolute Change in Enteral Support (EN) Volume From Baseline at Week 16 [ Time Frame: Baseline, Week 16 ] [ Designated as safety issue: No ]
    Absolute change in enteral support requirements at Week 16 (liters/week)


Enrollment: 42
Study Start Date: November 2013
Study Completion Date: January 2015
Primary Completion Date: January 2015 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
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
No Intervention: Standard of Care

  Eligibility

Ages Eligible for Study:   1 Year to 17 Years   (Child)
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
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)
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT01952080

  Show 19 Study Locations
Sponsors and Collaborators
Shire
Investigators
Study Director: Executive Director Clinical Development, GI NPS Pharma
  More Information

Responsible Party: Shire
ClinicalTrials.gov Identifier: NCT01952080     History of Changes
Other Study ID Numbers: TED-C13-003 
Study First Received: September 23, 2013
Results First Received: August 12, 2015
Last Updated: November 10, 2015
Health Authority: United States: Food and Drug Administration

Keywords provided by Shire:
Short Bowel Syndrome
SBS
Pediatric SBS

Additional relevant MeSH terms:
Syndrome
Short Bowel Syndrome
Disease
Pathologic Processes
Malabsorption Syndromes
Intestinal Diseases
Gastrointestinal Diseases
Digestive System Diseases
Postoperative Complications

ClinicalTrials.gov processed this record on September 30, 2016