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

This study is currently recruiting participants. (see Contacts and Locations)
Verified May 2014 by NPS Pharmaceuticals
Sponsor:
Information provided by (Responsible Party):
NPS Pharmaceuticals
ClinicalTrials.gov Identifier:
NCT01952080
First received: September 23, 2013
Last updated: May 16, 2014
Last verified: May 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 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

Contacts
Contact: Clinical Operations 908-450-5300 info@npsp.com

Locations
United States, Alabama
Children´s of Alabama Recruiting
Brimingham, Alabama, United States, 35233
Contact: Donna Bartle    205-638-2925      
Principal Investigator: Reed Dimmitt, MD         
United States, Arkansas
Arkansas Children's Hospital Recruiting
Little Rock, Arkansas, United States, 72202
Contact: Michelle Hart    501-364-4490      
Principal Investigator: Juliana Vaughan, MD         
United States, California
Mattel Children's Hospital UCLA, Department of Pediatric Gastroenterology Recruiting
Los Angeles, California, United States, 90095
Contact: Susan Feist    310-825-4349      
Principal Investigator: Robert Venick, MD         
Rady Children's Hospital- San Diego Not yet recruiting
San Diego, California, United States, 92123
Contact: Katheryn Smith    858-966-8152      
Principal Investigator: Rebecca Cherry, MD         
United States, Illinois
Ann & Robert H. Lurie Children's Hospital of Chicago Recruiting
Chicago, Illinois, United States, 60611
Contact: Kristen Johnson    312-227-4200      
Principal Investigator: Valeria Cohran, MD         
United States, Indiana
Riley Hospital for Children at IU Health Recruiting
Indianapolis, Indiana, United States, 46202
Contact: Ann Klipsch    317-944-9605      
Principal Investigator: Charles P Vanderpool, MD         
United States, Massachusetts
Boston Children's Hospital Recruiting
Boston, Massachusetts, United States, 02115
Contact: Sivan Kassiff    617-730-0494      
Principal Investigator: Christopher Duggan, MD         
United States, Michigan
Mott Children's Hospital, University of Michigan Recruiting
Ann Arbor, Michigan, United States, 48109
Contact: Ann M Mehringer    734-936-8464      
Principal Investigator: Daniel H Teitelbaum, MD         
United States, Missouri
Childrens Mercy Hospitals & Clinics Pediatric Gastroenterology Recruiting
Kansas City, Missouri, United States, 64108
Contact: Corey Schurman    816-802-1167      
Principal Investigator: Joel Lim, MD         
United States, Nebraska
University of Nebraska Medical Center Recruiting
Omaha, Nebraska, United States, 68198
Contact: Bridget Bauman    402-559-6955      
Principal Investigator: David Mercer, MD         
United States, New York
Montefiore Medical Center Recruiting
Bronx, New York, United States, 10467
Contact: Gaelle Erisnor    718-741-2557      
Principal Investigator: John F Thompson, MD         
United States, Ohio
Cincinnati Children's Hospital Medical Center Recruiting
Cincinnati, Ohio, United States, 45229
Contact: Crystal Slaughter    513-636-0137      
Principal Investigator: Samuel Kokoshis, MD         
Sub-Investigator: Conrad Cole, MD         
United States, Tennessee
University of Tennessee Health Science Center Le Bonheur Pediatric Specialists Recruiting
Memphis, Tennessee, United States, 38103
Contact: Ila MacDonald    901-287-5366      
Principal Investigator: Mark Corkin, MD         
Monroe Carell, Jr. Children's Hospital at Vanderbilt Not yet recruiting
Nashville, Tennessee, United States, 37232
Contact: Cynthia Womack-Ramirez    615-322-4137      
Principal Investigator: Kelly Thomsen, MD         
United States, Texas
Texas Children's Hospital, Baylor College of Medicine Recruiting
Houston, Texas, United States, 77030
Contact: Brooke Bessard    832-824-4267      
Principal Investigator: Beth Carter, MD         
United States, Washington
Seattle Childrens Hospital Gastroenterology and Hepatology Recruiting
Seattle, Washington, United States, 98105
Contact: Heather Vendettuoli    206-987-0055      
Principal Investigator: Simon Horslen, MD         
United States, Wisconsin
University of Wisconsin School of Medicine and Public Health Surgery Recruiting
Madison, Wisconsin, United States, 53792
Contact: Lauren Howard    608-265-6648      
Principal Investigator: Peter F Nichol, MD         
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: May 16, 2014
Health Authority: United States: Food and Drug Administration

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

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

ClinicalTrials.gov processed this record on July 28, 2014