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

This study has been completed.
Sponsor:
Information provided by (Responsible Party):
Shire
ClinicalTrials.gov Identifier:
NCT01952080
First received: September 23, 2013
Last updated: November 10, 2015
Last verified: July 2015
Results First Received: August 12, 2015  
Study Type: Interventional
Study Design: Allocation: Non-Randomized;   Endpoint Classification: Safety Study;   Intervention Model: Single Group Assignment;   Masking: Open Label;   Primary Purpose: Treatment
Condition: Short Bowel Syndrome
Intervention: Drug: teduglutide

  Participant Flow
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Recruitment Details
Key information relevant to the recruitment process for the overall study, such as dates of the recruitment period and locations
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Pre-Assignment Details
Significant events and approaches for the overall study following participant enrollment, but prior to group assignment
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Reporting Groups
  Description
Standard of Care SOC- no teduglutide therapy
Teduglutide 0.0125 mg/kg/Day Cohort 1 - Teduglutide 0.0125 mg/kg/day
Teduglutide 0.025 mg/kg/Day Cohort 2 - Teduglutide 0.025 mg/kg/day
Teduglutide 0.05 mg/kg/Day Cohort 3 - Teduglutide 0.05 mg/kg/day

Participant Flow:   Overall Study
    Standard of Care     Teduglutide 0.0125 mg/kg/Day     Teduglutide 0.025 mg/kg/Day     Teduglutide 0.05 mg/kg/Day  
STARTED     5     8     14     15  
Treated     5     8     14     15  
COMPLETED     5     7     14     14  
NOT COMPLETED     0     1     0     1  
Withdrawal by Subject                 0                 0                 0                 1  
Protocol Non-compliance                 0                 1                 0                 0  



  Baseline Characteristics
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Population Description
Explanation of how the number of participants for analysis was determined. Includes whether analysis was per protocol, intention to treat, or another method. Also provides relevant details such as imputation technique, as appropriate.
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Reporting Groups
  Description
Standard of Care SOC- no teduglutide therapy
Teduglutide 0.0125 mg/kg/Day Cohort 1 - Teduglutide 0.0125 mg/kg/day
Teduglutide 0.025 mg/kg/Day Cohort 2 - Teduglutide 0.025 mg/kg/day
Teduglutide 0.05 mg/kg/Day Cohort 3 - Teduglutide 0.05 mg/kg/day
Total Total of all reporting groups

Baseline Measures
    Standard of Care     Teduglutide 0.0125 mg/kg/Day     Teduglutide 0.025 mg/kg/Day     Teduglutide 0.05 mg/kg/Day     Total  
Number of Participants  
[units: participants]
  5     8     14     15     42  
Age  
[units: years]
Mean (Standard Deviation)
  2.2  (0.45)     5.1  (4.55)     4.6  (3.43)     4.5  (3.16)     4.7  (3.5)  
Age, Customized  
[units: participants]
         
1 to 3 years     5     4     6     7     22  
4 to 12 years     0     3     7     7     17  
13 to 17 years     0     1     1     1     3  
Gender  
[units: participants]
         
Female     2     2     3     7     14  
Male     3     6     11     8     28  
Region of Enrollment  
[units: participants]
         
United States     5     8     14     12     39  
United Kingdom     0     0     0     3     3  



  Outcome Measures
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1.  Primary:   Percent Change in Parenteral Support [Parenteral Nutrition (PN)/Intravenous (IV)] Volume at Week 12   [ Time Frame: Baseline, Week 12 ]

2.  Primary:   Percent Change in Parenteral Support (PN/IV) Volume at End of Treatment   [ Time Frame: Baseline, End of Treatment ]

3.  Primary:   Percent Change in Parenteral Support (PN/IV) Volume at Week 16   [ Time Frame: Baseline, Week 16 ]

4.  Primary:   Absolute Change in Parenteral Support (PN/IV) Volume at Week 12   [ Time Frame: Baseline, Week 12 ]

5.  Primary:   Absolute Change in Parenteral Support (PN/IV) Volume at End of Treatment   [ Time Frame: Baseline, End of Treatment ]

6.  Primary:   Absolute Change in Parenteral Support (PN/IV) Volume at Week 16   [ Time Frame: Baseline, Week 16 ]

7.  Other Pre-specified:   Percent Change in Enteral Support (EN) Volume From Baseline at Week 12   [ Time Frame: Baseline, Week 12 ]

8.  Other Pre-specified:   Percent Change in Enteral Support (EN) Volume From Baseline at Week 16   [ Time Frame: Baseline, Week 16 ]

9.  Other Pre-specified:   Absolute Change in Enteral Support (EN) Volume From Baseline at Week 12   [ Time Frame: Baseline, Week 12 ]

10.  Other Pre-specified:   Absolute Change in Enteral Support (EN) Volume From Baseline at Week 16   [ Time Frame: Baseline, Week 16 ]


  Serious Adverse Events


  Other Adverse Events


  Limitations and Caveats
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Limitations of the study, such as early termination leading to small numbers of participants analyzed and technical problems with measurement leading to unreliable or uninterpretable data
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  More Information
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Certain Agreements:  
Principal Investigators are NOT employed by the organization sponsoring the study.
There is NOT an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.


Results Point of Contact:  
Name/Title: Global Clinical Development Operations
Organization: Shire
phone: 908-450-5300
e-mail: mgalen@shire.com



Responsible Party: Shire
ClinicalTrials.gov Identifier: NCT01952080     History of Changes
Other Study ID Numbers: TED-C13-003
Study First Received: September 23, 2013
Results First Received: August 12, 2015
Last Updated: November 10, 2015
Health Authority: United States: Food and Drug Administration