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

Study Overview
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Brief Summary
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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.

Official Title
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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

Conditions
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Short Bowel Syndrome

Intervention / Treatment
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* Drug: teduglutide


Participation Criteria
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Eligibility Criteria
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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)

Ages Eligible for Study
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Minimum Age: 1 Year
Maximum Age: 17 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Allocation: Non-Randomized
Intervention Model: Single Group Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: teduglutide<br>Open label teduglutide, subcutaneously injected. | Drug: teduglutide<br>* 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<br>|
| No Intervention: Standard of Care<br> | |

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| 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 | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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SBS, Pediatric SBS, Short Bowel Syndrome