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

Study Status: Completed
Recruit Status: Completed
Study Type: Interventional

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

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.

Detailed Summary:
Sponsor: Shire

Current Primary Outcome:

• 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.
• 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.
• 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.
• 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.
• 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.
• 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.

Original Primary Outcome:

• Safety [ Time Frame: 12 weeks ]
  Number of reported adverse events after 12 weeks of teduglutide
• Pharmacodynamics [ Time Frame: 12 weeks ]
  Change in parenteral support requirements after 12 weeks of teduglutide (liters/week)
• Pharmacodynamics [ Time Frame: 12 weeks ]
  Change in enteral support requirements after 12 weeks of teduglutide (liters/week)

Current Secondary Outcome:

Original Secondary Outcome:

Information By: Shire

Dates:
Date Received: September 23, 2013
Date Started: November 2013
Date Completion:
Last Updated: November 10, 2015
Last Verified: July 2015