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

Phase 3CompletedNCT01952080

Shire42 enrolled

Overview

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.

Study Type

INTERVENTIONAL

Allocation

NON_RANDOMIZED

Purpose

TREATMENT

Masking

NONE

Enrollment

Conditions

Short Bowel Syndrome

Interventions

teduglutideDRUG

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

Eligibility

Sex: ALLMin age: 1 YearMax age: 17 Years

Medical Language ↔ Plain English

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)

Locations (19)

Children´s Hospital of Alabama

Birmingham, Alabama, United States

Outcomes

Primary Outcomes

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.

Time frame: 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.

Time frame: 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.

Time frame: 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.

Time frame: 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.

Time frame: 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.

Time frame: Baseline, Week 16

Data from ClinicalTrials.gov

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