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.
Brief Title
A Pharmacokinetic, Safety, and Pharmacodynamic Study of Teduglutide in Pediatric Subjects With Short Bowel Syndrome
Categories
Completion Date
Completion Date Type
Actual
Conditions
Short Bowel Syndrome
Eligibility 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)
* 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)
Inclusion Criteria
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
* 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
Gender
All
Gender Based
false
Keywords
SBS
Pediatric SBS
Short Bowel Syndrome
Healthy Volunteers
No
Last Update Post Date
Last Update Post Date Type
Actual
Last Update Submit Date
Maximum Age
17 Years
Minimum Age
1 Year
NCT Id
NCT01952080
Org Class
Industry
Org Full Name
Takeda
Org Study Id
TED-C13-003
Overall Status
Completed
Phases
Phase 3
Primary Completion Date
Primary Completion Date Type
Actual
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
Primary Outcomes
Outcome Description
Percent change in PN/IV from the Baseline Visit to Week 12 Visit.
Outcome Measure
Percent Change in Parenteral Support [Parenteral Nutrition (PN)/Intravenous (IV)] Volume at Week 12
Outcome Time Frame
Baseline, Week 12
Outcome Description
Percent change in PN/IV from the Baseline Visit to End of Treatment Visit.
Outcome Measure
Percent Change in Parenteral Support (PN/IV) Volume at End of Treatment
Outcome Time Frame
Baseline, End of Treatment
Outcome Description
Percent change in PN/IV from the Baseline Visit to Week 16 Visit.
Outcome Measure
Percent Change in Parenteral Support (PN/IV) Volume at Week 16
Outcome Time Frame
Baseline, Week 16
Outcome Description
Absolute change in PN/IV from the Baseline Visit to Week 12 Visit.
Outcome Measure
Absolute Change in Parenteral Support (PN/IV) Volume at Week 12
Outcome Time Frame
Baseline, Week 12
Outcome Description
Absolute change in PN/IV from the Baseline Visit to End of Treatment Visit.
Outcome Measure
Absolute Change in Parenteral Support (PN/IV) Volume at End of Treatment
Outcome Time Frame
Baseline, End of Treatment
Outcome Description
Absolute change in PN/IV from the Baseline Visit to Week 16 Visit.
Outcome Measure
Absolute Change in Parenteral Support (PN/IV) Volume at Week 16
Outcome Time Frame
Baseline, Week 16
Start Date
Start Date Type
Actual
Status Verified Date
First Post Date
First Post Date Type
Estimated
First Submit Date
First Submit QC Date
Std Ages
Child
Maximum Age Number (converted to Years and rounded down)
17
Minimum Age Number (converted to Years and rounded down)
1
Investigators
Investigator Type
Principal Investigator
Investigator Name
John Thompson
Investigator Email
JOTHOMPS@montefiore.org
Investigator Phone
Categories Mesh Debug
Gastrointestinal (GI) Cancers --- INTESTINAL DISEASES
Digestive System --- INTESTINAL DISEASES
Gastrointestinal (GI) Cancers --- GASTROINTESTINAL DISEASES
Digestive System --- GASTROINTESTINAL DISEASES
Gastrointestinal (GI) Cancers --- DIGESTIVE SYSTEM DISEASES
Digestive System --- DIGESTIVE SYSTEM DISEASES
Liver --- DIGESTIVE SYSTEM DISEASES
MeSH Terms
SHORT BOWEL SYNDROME
MALABSORPTION SYNDROMES
INTESTINAL DISEASES
GASTROINTESTINAL DISEASES
DIGESTIVE SYSTEM DISEASES
POSTOPERATIVE COMPLICATIONS
PATHOLOGIC PROCESSES
PATHOLOGICAL CONDITIONS, SIGNS AND SYMPTOMS
TEDUGLUTIDE