Clinical trial NCT01952080: A Pharmacokinetic, Safety, and Pharmacodynamic Study of Teduglutide in Pediatric Subjects With Short Bowel Syndrome
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
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE3
Conditions: Short Bowel Syndrome
Keywords: SBS
Pediatric SBS
Short Bowel Syndrome
Lead sponsor: Takeda
Sponsor class: INDUSTRY
First posted: 2013-09-27
Enrollment: 42
Sex: ALL
Minimum age (years): 1
Maximum age (years): 17
Standard ages: CHILD
Accepts healthy volunteers: no
Allocation: NON_RANDOMIZED
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

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.


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)

Primary outcomes:
measure: Percent Change in Parenteral Support [Parenteral Nutrition (PN)/Intravenous (IV)] Volume at Week 12
timeFrame: Baseline, Week 12
measure: Percent Change in Parenteral Support (PN/IV) Volume at End of Treatment
timeFrame: Baseline, End of Treatment
measure: Percent Change in Parenteral Support (PN/IV) Volume at Week 16
timeFrame: Baseline, Week 16
measure: Absolute Change in Parenteral Support (PN/IV) Volume at Week 12
timeFrame: Baseline, Week 12
measure: Absolute Change in Parenteral Support (PN/IV) Volume at End of Treatment
timeFrame: Baseline, End of Treatment
measure: Absolute Change in Parenteral Support (PN/IV) Volume at Week 16
timeFrame: Baseline, Week 16
Secondary outcomes:
Other outcomes:
measure: Percent Change in Enteral Support (EN) Volume From Baseline at Week 12
timeFrame: Baseline, Week 12
measure: Percent Change in Enteral Support (EN) Volume From Baseline at Week 16
timeFrame: Baseline, Week 16
measure: Absolute Change in Enteral Support (EN) Volume From Baseline at Week 12
timeFrame: Baseline, Week 12
measure: Absolute Change in Enteral Support (EN) Volume From Baseline at Week 16
timeFrame: Baseline, Week 16
Locations:
city: Birmingham
country: United States
facility: Children´s Hospital of Alabama
state: Alabama
city: Little Rock
country: United States
facility: Arkansas Children's Hospital
state: Arkansas
city: Los Angeles
country: United States
facility: Children's Hospital Los Angeles
state: California
city: Los Angeles
country: United States
facility: Mattel Children's Hospital UCLA, Department of Pediatric Gastroenterology
state: California
city: Orlando
country: United States
facility: Arnold Palmer Hospital for Children
state: Florida
city: Chicago
country: United States
facility: Ann & Robert H. Lurie Children's Hospital of Chicago
state: Illinois
city: Indianapolis
country: United States
facility: Riley Hospital for Children at IU Health
state: Indiana
city: Boston
country: United States
facility: Boston Children's Hospital
state: Massachusetts
city: Ann Arbor
country: United States
facility: Mott Children's Hospital, University of Michigan
state: Michigan
city: Kansas City
country: United States
facility: Childrens Mercy Hospitals & Clinics Pediatric Gastroenterology
state: Missouri
city: Omaha
country: United States
facility: University of Nebraska Medical Center
state: Nebraska
city: Bronx
country: United States
facility: Montefiore Medical Center
state: New York
city: Cincinnati
country: United States
facility: Cincinnati Children's Hospital Medical Center
state: Ohio
city: Memphis
country: United States
facility: University of Tennessee Health Science Center Le Bonheur Pediatric Specialists
state: Tennessee
city: Nashville
country: United States
facility: Monroe Carell, Jr. Children's Hospital at Vanderbilt
state: Tennessee
city: Houston
country: United States
facility: Texas Children's Hospital, Baylor College of Medicine
state: Texas
city: Seattle
country: United States
facility: Seattle Childrens Hospital Gastroenterology and Hepatology
state: Washington
city: Madison
country: United States
facility: University of Wisconsin School of Medicine and Public Health Surgery
state: Wisconsin
city: London
country: United Kingdom
facility: Great Ormond Street Hospital for Children
Study officials:
affiliation: Takeda
name: Study Director
role: STUDY_DIRECTOR
References:
citation: Carter BA, Cohran VC, Cole CR, Corkins MR, Dimmitt RA, Duggan C, Hill S, Horslen S, Lim JD, Mercer DF, Merritt RJ, Nichol PF, Sigurdsson L, Teitelbaum DH, Thompson J, Vanderpool C, Vaughan JF, Li B, Youssef NN, Venick RS, Kocoshis SA. Outcomes from a 12-Week, Open-Label, Multicenter Clinical Trial of Teduglutide in Pediatric Short Bowel Syndrome. J Pediatr. 2017 Feb;181:102-111.e5. doi: 10.1016/j.jpeds.2016.10.027. Epub 2016 Nov 15.