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

This study is ongoing, but not recruiting participants.
Sponsor:
Information provided by (Responsible Party):
NPS Pharmaceuticals
ClinicalTrials.gov Identifier:
NCT01952080
First received: September 23, 2013
Last updated: September 12, 2014
Last verified: September 2014
  Purpose

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.


Condition Intervention Phase
Short Bowel Syndrome
Drug: teduglutide
Phase 3

Study Type: Interventional
Study Design: Endpoint Classification: Safety Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
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

Resource links provided by NLM:


Further study details as provided by NPS Pharmaceuticals:

Primary Outcome Measures:
  • Safety [ Time Frame: 12 weeks ] [ Designated as safety issue: No ]
    Number of reported adverse events after 12 weeks of teduglutide

  • Pharmacodynamics [ Time Frame: 12 weeks ] [ Designated as safety issue: No ]
    Change in parenteral support requirements after 12 weeks of teduglutide (liters/week)

  • Pharmacodynamics [ Time Frame: 12 weeks ] [ Designated as safety issue: No ]
    Change in enteral support requirements after 12 weeks of teduglutide (liters/week)


Estimated Enrollment: 36
Study Start Date: November 2013
Estimated Study Completion Date: March 2015
Estimated Primary Completion Date: December 2014 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: teduglutide
Open label teduglutide, subcutaneously injected.
Drug: teduglutide

  Eligibility

Ages Eligible for Study:   1 Year to 17 Years
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
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)
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT01952080

Locations
United States, Alabama
Children´s Hospital of Alabama
Birmingham, Alabama, United States, 35233
United States, Arkansas
Arkansas Children's Hospital
Little Rock, Arkansas, United States, 72202
United States, California
Mattel Children's Hospital UCLA, Department of Pediatric Gastroenterology
Los Angeles, California, United States, 90095
Rady Children's Hospital- San Diego
San Diego, California, United States, 92123
United States, Illinois
Ann & Robert H. Lurie Children's Hospital of Chicago
Chicago, Illinois, United States, 60611
United States, Indiana
Riley Hospital for Children at IU Health
Indianapolis, Indiana, United States, 46202
United States, Massachusetts
Boston Children's Hospital
Boston, Massachusetts, United States, 02115
United States, Michigan
Mott Children's Hospital, University of Michigan
Ann Arbor, Michigan, United States, 48109
United States, Missouri
Childrens Mercy Hospitals & Clinics Pediatric Gastroenterology
Kansas City, Missouri, United States, 64108
United States, Nebraska
University of Nebraska Medical Center
Omaha, Nebraska, United States, 68198
United States, New York
Montefiore Medical Center
Bronx, New York, United States, 10467
United States, Ohio
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, United States, 45229
United States, Tennessee
University of Tennessee Health Science Center Le Bonheur Pediatric Specialists
Memphis, Tennessee, United States, 38103
Monroe Carell, Jr. Children's Hospital at Vanderbilt
Nashville, Tennessee, United States, 37232
United States, Texas
Texas Children's Hospital, Baylor College of Medicine
Houston, Texas, United States, 77030
United States, Washington
Seattle Childrens Hospital Gastroenterology and Hepatology
Seattle, Washington, United States, 98105
United States, Wisconsin
University of Wisconsin School of Medicine and Public Health Surgery
Madison, Wisconsin, United States, 53792
Sponsors and Collaborators
NPS Pharmaceuticals
Investigators
Study Director: Executive Director Clinical Development, GI NPS Pharmaceuticals
  More Information

No publications provided

Responsible Party: NPS Pharmaceuticals
ClinicalTrials.gov Identifier: NCT01952080     History of Changes
Other Study ID Numbers: TED-C13-003
Study First Received: September 23, 2013
Last Updated: September 12, 2014
Health Authority: United States: Food and Drug Administration

Keywords provided by NPS Pharmaceuticals:
Short Bowel Syndrome
SBS
Pediatric SBS

Additional relevant MeSH terms:
Syndrome
Short Bowel Syndrome
Disease
Pathologic Processes
Malabsorption Syndromes
Intestinal Diseases
Gastrointestinal Diseases
Digestive System Diseases
Postoperative Complications

ClinicalTrials.gov processed this record on September 18, 2014