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Study of Teduglutide Effectiveness in Parenteral Nutrition (PN)-Dependent Short Bowel Syndrome (SBS) Subjects (STEPS)

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ClinicalTrials.gov Identifier: NCT00798967
Recruitment Status : Completed
First Posted : November 27, 2008
Results First Posted : February 1, 2012
Last Update Posted : December 8, 2015
Sponsor:
Collaborator:
Nycomed
Information provided by (Responsible Party):
Shire

Study Type Interventional
Study Design Allocation: Randomized;   Intervention Model: Parallel Assignment;   Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor);   Primary Purpose: Treatment
Condition Short Bowel Syndrome
Interventions Drug: teduglutide
Drug: placebo
Enrollment 86
Recruitment Details First Patient Screened - November 25, 2008; Last Patient Screened - July 13, 2010; First Patient Randomized - March 3, 2009; Last Patient Randomized - July 22, 2010; Locations - hospitals and transplant centers; Subjects must be on parenteral nutrition (PN) and/or intravenous (I.V.) fluids.
Pre-assignment Details Stage 1 was screening, optimization, and stabilization periods. At screening, if the PN/I.V. volume is not stable per protocol, s/he entered an optimization period (up to 8 weeks) to find the minimally tolerated stable volume of PN/I.V.. Prior to randomization, all entered 4-8 weeks of stabilization period on that volume of PN/I.V.
Arm/Group Title Teduglutide Placebo
Hide Arm/Group Description 0.05 mg/kg/day subcutaneous (sc) dose of teduglutide Matching sc dose of placebo to teduglutide
Period Title: Overall Study
Started 43 43
Discontinued During Dosing Period 4 4
Completed 39 39
Not Completed 4 4
Reason Not Completed
Adverse Event             2             3
Withdrawal by Subject             0             1
Physician Decision             1             0
Randomized in error by site             1             0
Arm/Group Title Teduglutide Placebo Total
Hide Arm/Group Description 0.05 mg/kg/day subcutaneous (sc) dose of teduglutide Matching sc dose of placebo to teduglutide Total of all reporting groups
Overall Number of Baseline Participants 43 43 86
Hide Baseline Analysis Population Description
[Not Specified]
Age, Continuous  
Mean (Standard Deviation)
Unit of measure:  Years
Number Analyzed 43 participants 43 participants 86 participants
50.9  (12.6) 49.7  (15.6) 50.3  (14.1)
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 43 participants 43 participants 86 participants
Female
22
  51.2%
24
  55.8%
46
  53.5%
Male
21
  48.8%
19
  44.2%
40
  46.5%
1.Primary Outcome
Title Responder
Hide Description Comparison of subjects treated with teduglutide to placebo who achieve a 20 to 100% reduction from baseline in weekly parenteral nutrition/intravenous fluid (PN/I.V.) volume at weeks 20 and 24.
Time Frame Weeks 20 and 24
Hide Outcome Measure Data
Hide Analysis Population Description
Percentages were based on the number of subjects in the Intent to Treat (ITT) population.
Arm/Group Title Teduglutide Placebo
Hide Arm/Group Description:
0.05 mg/kg/day subcutaneous (sc) dose of teduglutide
Matching sc dose of placebo to teduglutide
Overall Number of Participants Analyzed 43 43
Measure Type: Number
Unit of Measure: subjects
27 13
Show Statistical Analysis 1 Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Teduglutide, Placebo
Comments [Not Specified]
Type of Statistical Test Superiority or Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.002
Comments Cochran-Mantel-Haenszel (CMH) test adjusted for the randomization stratification variable (<= 6 or > 6 L/week of PN at baseline)
Method Cochran-Mantel-Haenszel
Comments [Not Specified]
2.Secondary Outcome
Title Absolute Change in PN/I.V. Volume From Baseline to Last Time Point
Hide Description Absolute change in the volume of PN/I.V. from baseline (Week 0) to the visit when the last data point was collected (week 4 through week 24, or earlier if the subject discontinued early).
Time Frame Week 0 to last visit when data was collected.
Hide Outcome Measure Data
Hide Analysis Population Description
Intent-to-Treat (ITT) was defined for efficacy analyses which included all randomized patients.
Arm/Group Title Teduglutide Placebo
Hide Arm/Group Description:
0.05 mg/kg/day sc dose of teduglutide
Matching sc dose of placebo to teduglutide
Overall Number of Participants Analyzed 40 43
Mean (Standard Deviation)
Unit of Measure: Liters/Week
-4.28  (3.81) -2.38  (2.79)
Show Statistical Analysis 1 Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Teduglutide, Placebo
Comments Last Dosing Visit
Type of Statistical Test Superiority or Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value < 0.001
Comments [Not Specified]
Method ANCOVA
Comments [Not Specified]
Time Frame Adverse event data were collected for each subject from the time informed consent was signed to end of study. For this reporting of adverse event, the most commonly reported treatment emergent adverse events (>=3 subjects at SOC level) are listed.
Adverse Event Reporting Description

All adverse experience reporting used the Safety population which consisted of 85 subjects who received at least one dose of study drug. There were 42 subjects treated with teduglutide, whereas 43 subjects received placebo.

Adverse experience monitoring was performed through investigator assessment and safety laboratory testing at every visit.

 
Arm/Group Title Teduglutide Placebo
Hide Arm/Group Description 0.05 mg/kg/day subcutaneous (sc) dose of teduglutide Matching sc dose of placebo to teduglutide
All-Cause Mortality
Teduglutide Placebo
Affected / at Risk (%) Affected / at Risk (%)
Total   --/--      --/--    
Show Serious Adverse Events Hide Serious Adverse Events
Teduglutide Placebo
Affected / at Risk (%) # Events Affected / at Risk (%) # Events
Total   15/42 (35.71%)      12/43 (27.91%)    
Gastrointestinal disorders     
Small intestinal stenosis  1  1/42 (2.38%)  1 0/43 (0.00%)  0
General disorders     
Catheter-related complication  1  1/42 (2.38%)  1 0/43 (0.00%)  0
Implant site extravasation  1  1/42 (2.38%)  1 1/43 (2.33%)  1
Hepatobiliary disorders     
Cholecystitis acute  1  1/42 (2.38%)  1 0/43 (0.00%)  0
Hepatitis cholestatic  1  0/42 (0.00%)  0 1/43 (2.33%)  1
Infections and infestations     
Adenovirus infection  1  1/42 (2.38%)  1 0/43 (0.00%)  0
Bacteremia  1  0/42 (0.00%)  0 3/43 (6.98%)  3
Catheter sepsis  1  1/42 (2.38%)  1 0/43 (0.00%)  0
Catheter-related infection  1  5/42 (11.90%)  5 1/43 (2.33%)  1
Central line infection  1  2/42 (4.76%)  2 3/43 (6.98%)  3
Infective thrombosis  1  1/42 (2.38%)  1 0/43 (0.00%)  0
Influenza  1  1/42 (2.38%)  1 0/43 (0.00%)  0
Pneumonia  1  1/42 (2.38%)  1 0/43 (0.00%)  0
Pneumonia influenzal  1  0/42 (0.00%)  0 1/43 (2.33%)  1
Rectal abscess  1  1/42 (2.38%)  1 0/43 (0.00%)  0
Soft tissue infection  1  0/42 (0.00%)  0 1/43 (2.33%)  1
Urinary tract infection  1  2/42 (4.76%)  2 1/43 (2.33%)  1
Viral infection  1  1/42 (2.38%)  1 0/43 (0.00%)  0
Injury, poisoning and procedural complications     
Device breakage  1  0/42 (0.00%)  0 1/43 (2.33%)  1
Device dislocation  1  0/42 (0.00%)  0 2/43 (4.65%)  2
Device failure  1  0/42 (0.00%)  0 1/43 (2.33%)  1
Device malfunction  1  0/42 (0.00%)  0 1/43 (2.33%)  1
Fall  1  0/42 (0.00%)  0 1/43 (2.33%)  1
Metabolism and nutrition disorders     
Dehydration  1  1/42 (2.38%)  1 0/43 (0.00%)  0
Psychiatric disorders     
Suicide attempt  1  0/42 (0.00%)  0 1/43 (2.33%)  1
Respiratory, thoracic and mediastinal disorders     
Pleural effusion  1  0/42 (0.00%)  0 1/43 (2.33%)  1
Vascular disorders     
Subclavian vein thrombosis  1  1/42 (2.38%)  1 0/43 (0.00%)  0
Indicates events were collected by systematic assessment
1
Term from vocabulary, MedDRA (12.0)
Show Other (Not Including Serious) Adverse Events Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 5%
Teduglutide Placebo
Affected / at Risk (%) # Events Affected / at Risk (%) # Events
Total   35/42 (83.33%)      34/43 (79.07%)    
Gastrointestinal disorders     
Abdominal distension  1  9/42 (21.43%)  14 1/43 (2.33%)  2
Abdominal pain  1  13/42 (30.95%)  14 10/43 (23.26%)  14
Diarrhea  1  3/42 (7.14%)  4 5/43 (11.63%)  7
Flatulence  1  5/42 (11.90%)  5 3/43 (6.98%)  3
Nausea  1  12/42 (28.57%)  19 8/43 (18.60%)  12
Vomiting  1  5/42 (11.90%)  5 4/43 (9.30%)  10
General disorders     
Edema peripheral  1  7/42 (16.67%)  8 2/43 (4.65%)  3
Fatigue  1  4/42 (9.52%)  5 3/43 (6.98%)  3
Pyrexia  1  4/42 (9.52%)  5 4/43 (9.30%)  5
Infections and infestations     
Central line systemic infections  1  7/42 (16.67%)  12 7/43 (16.28%)  8
Nasopharyngitis  1  3/42 (7.14%)  3 0/43 (0.00%)  0
Urinary tract infection  1  6/42 (14.29%)  6 4/43 (9.30%)  5
Injury, poisoning and procedural complications     
Gastrointestinal stoma complication  1  10/42 (23.81%)  11 3/43 (6.98%)  3
Investigations     
Weight increased  1  3/42 (7.14%)  3 3/43 (6.98%)  3
Respiratory, thoracic and mediastinal disorders     
Dyspnea  1  3/42 (7.14%)  3 0/43 (0.00%)  0
Indicates events were collected by systematic assessment
1
Term from vocabulary, MedDRA (12.0)
Certain Agreements
Principal Investigators are NOT employed by the organization sponsoring the study.
There IS 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.
The terms and conditions of NPS Pharmaceuticals agreements with its investigators may vary. However, NPS does not prohibit any investigator from publishing. Any publications from a single-site are postponed until the publication of the pooled data (i.e., data from all sites) in the clinical trial.
Results Point of Contact
Name/Title: Head, Clinical Development
Organization: NPS Pharmaceuticals
Phone: 908-450-5300
Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):
Responsible Party: Shire
ClinicalTrials.gov Identifier: NCT00798967     History of Changes
Other Study ID Numbers: CL0600-020
First Submitted: November 25, 2008
First Posted: November 27, 2008
Results First Submitted: December 23, 2011
Results First Posted: February 1, 2012
Last Update Posted: December 8, 2015