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Safety and Efficacy Study of Teduglutide in Subjects With Short Bowel Syndrome

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ClinicalTrials.gov Identifier: NCT00081458
Recruitment Status : Completed
First Posted : April 14, 2004
Results First Posted : July 15, 2013
Last Update Posted : December 8, 2015
Sponsor:
Information provided by (Responsible Party):
Shire

Study Type Interventional
Study Design Allocation: Randomized;   Intervention Model: Parallel Assignment;   Masking: Triple (Participant, Care Provider, Investigator);   Primary Purpose: Treatment
Condition Short Bowel Syndrome
Interventions Drug: Placebo
Drug: Teduglutide 0.05 mg/kg/d
Drug: Teduglutide 0.1 mg/kg/d
Enrollment 84
Recruitment Details First subject was screened 25May2004 and last subject evaluations were completed on 06Jul2007.
Pre-assignment Details Patients underwent stabilization period for parenteral nutrition prior to randomization
Arm/Group Title Placebo Teduglutide 0.05 mg/kg/d Teduglutide 0.1 mg/kg/d
Hide Arm/Group Description Placebo injected subcutaneously daily teduglutide 0.05 mg/kg/d injected subcutaneously teduglutide 0.1 mg/kg/d injected subcutaneously
Period Title: Overall Study
Started 16 35 33
Received Treatment 16 35 32 [1]
Completed 15 27 29
Not Completed 1 8 4
Reason Not Completed
Adverse Event             1             5             2
Withdrawal by Subject             0             3             1
Randomized but not dosed             0             0             1
[1]
1 subject was randomized but did not receive the study drug
Arm/Group Title Placebo Teduglutide 0.05 mg/kg/d Teduglutide 0.1 mg/kg/d Total
Hide Arm/Group Description Placebo injected subcutaneously daily teduglutide 0.05 mg/kg/d injected subcutaneously teduglutide 0.1 mg/kg/d injected subcutaneously Total of all reporting groups
Overall Number of Baseline Participants 16 35 32 83
Hide Baseline Analysis Population Description
84 subjects were initially randomized; however, one subject never received study therapy. As a result, the total participant who started flow period is 83.
Age, Continuous  
Mean (Standard Deviation)
Unit of measure:  Years
Number Analyzed 16 participants 35 participants 32 participants 83 participants
49.4  (15.1) 47.1  (14.2) 50.3  (14.0) 48.8  (14.2)
Age, Customized  
Measure Type: Number
Unit of measure:  Participants
Number Analyzed 16 participants 35 participants 32 participants 83 participants
< 35 years 3 7 5 15
35-44 years 2 5 4 11
45 - 54 years 3 12 10 25
> 55 years 8 11 13 32
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 16 participants 35 participants 32 participants 83 participants
Female
9
  56.3%
18
  51.4%
19
  59.4%
46
  55.4%
Male
7
  43.8%
17
  48.6%
13
  40.6%
37
  44.6%
Region of Enrollment  
Measure Type: Number
Unit of measure:  Participants
Number Analyzed 16 participants 35 participants 32 participants 83 participants
Belgium 1 1 0 2
Canada 1 2 5 8
Denmark 2 3 3 8
France 2 5 2 9
Germany 0 0 2 2
Netherlands 0 4 1 5
Poland 3 6 8 17
United Kingdom 0 3 1 4
United States 7 11 10 28
1.Primary Outcome
Title A Graded Response Score in Parenteral Nutrition (PN) Reduction
Hide Description

The intensity of the response relied on a reduction from Baseline in weekly parenteral nutrition (PN) volume (minimum reduction of 20% and a maximum of 100%). Duration of the response incorporated responses at Weeks(Wk) 16-20 and at Wk20-24.

Zero (0 - lowest) assigned if <20% reduction at Wk20-24 and reduction at Wk16-20 of < 20%, 20-39%, or >=40%.

One (1) assigned if reduction of 20-39% at Wk20-24 but < 20% at Wk16-20. Two(2) assigned if reductions of 40-99% at Wk20-24 AND <20% at Wk16-20 OR 20-39% at Wk20-24 AND 20-39% at Wk16-20.

Three (3) assigned if reductions of 100% at Wk20-24 AND <20% at Wk16-20 OR 40-99% at Wk20-24 AND 20-39% at Wk16-20 OR 20-39% at Wk20-24 AND >=40% at Wk16-20.

Four (4) assigned if reductions of 100% at Wk20-24 AND 20-39% at Wk16-20 OR 40-99% at Wk20-24 AND >=40% at Wk16-20.

Time Frame 6 months
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
Intent to Treat (ITT) analysis using a stepdown procedure that was stopped if the 0.10 mg/kg dose was not significantly better than placebo.
Arm/Group Title Placebo Teduglutide 0.05 mg/kg/d Teduglutide 0.1 mg/kg/d
Hide Arm/Group Description:
Placebo injectable subcutaneously daily into the thigh or abdomen
teduglutide 0.05 mg/kg/d injected subcutaneously
teduglutide 0.1 mg/kg/d injected subcutaneously
Overall Number of Participants Analyzed 16 35 32
Measure Type: Number
Unit of Measure: participants
0 (No Response) 15 19 24
1 0 6 2
2 1 6 4
4 0 2 2
5 (No longer need PN) 0 2 0
Show Statistical Analysis 1 Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Placebo, Teduglutide 0.05 mg/kg/d, Teduglutide 0.1 mg/kg/d
Comments [Not Specified]
Type of Statistical Test Superiority or Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.007
Comments [Not Specified]
Method ANCOVA
Comments [Not Specified]
2.Secondary Outcome
Title Number of Subjects Achieving Binary Response at Week 20, Maintained at Week 24
Hide Description An efficacy responder was defined as achieving at least a 20% reduction from Baseline to Week 20 and maintained at Week 24 in weekly actual PN infusion volume.
Time Frame 6 months of treatment
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Placebo Teduglutide 0.05 mg/kg/d Teduglutide 0.1 mg/kg/d
Hide Arm/Group Description:
Placebo injected subcutaneously daily into the thigh or abdomen
teduglutide 0.05 mg/kg/d injected subcutaneously
teduglutide 0.1 mg/kg/d injected subcutaneously
Overall Number of Participants Analyzed 16 35 32
Measure Type: Number
Unit of Measure: participants
Responder 1 16 8
Non-Responder 15 19 24
Show Statistical Analysis 1 Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Placebo, Teduglutide 0.05 mg/kg/d, Teduglutide 0.1 mg/kg/d
Comments An efficacy responder was defined as achieving at least a 20% reduction from Baseline to Week 20 and maintained at Week 24 in weekly actual PN infusion volume.
Type of Statistical Test Superiority or Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.005
Comments [Not Specified]
Method ANCOVA
Comments [Not Specified]
Time Frame 6 months
Adverse Event Reporting Description A total of 84 subjects were randomized in this study; however, one subject did not receive study drug. Thus, the safety population consists of 83 subjects.
 
Arm/Group Title Placebo Teduglutide 0.05 mg/kg/d Teduglutide 0.1 mg/kg/d
Hide Arm/Group Description Placebo injected subcutaneously daily teduglutide 0.05 mg/kg/d injected subcutaneously teduglutide 0.1 mg/kg/d injected subcutaneously
All-Cause Mortality
Placebo Teduglutide 0.05 mg/kg/d Teduglutide 0.1 mg/kg/d
Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%)
Total   --/--      --/--      --/--    
Show Serious Adverse Events Hide Serious Adverse Events
Placebo Teduglutide 0.05 mg/kg/d Teduglutide 0.1 mg/kg/d
Affected / at Risk (%) # Events Affected / at Risk (%) # Events Affected / at Risk (%) # Events
Total   5/16 (31.25%)      13/35 (37.14%)      11/32 (34.38%)    
Cardiac disorders       
Cardiac failure congestive   0/16 (0.00%)  0 1/35 (2.86%)  1 0/32 (0.00%)  0
Gastrointestinal disorders       
Abdominal pain   0/16 (0.00%)  0 1/35 (2.86%)  1 0/32 (0.00%)  0
Colonic stenosis   0/16 (0.00%)  0 1/35 (2.86%)  1 0/32 (0.00%)  0
Hemorrhoidal hemorrhage   0/16 (0.00%)  0 1/35 (2.86%)  1 0/32 (0.00%)  0
Intestinal obstruction   0/16 (0.00%)  0 0/35 (0.00%)  0 1/32 (3.13%)  1
Pancreatitis   0/16 (0.00%)  0 1/35 (2.86%)  1 0/32 (0.00%)  0
Short-bowel syndrome   0/16 (0.00%)  0 1/35 (2.86%)  1 0/32 (0.00%)  0
Small intestinal obstruction   0/16 (0.00%)  0 1/35 (2.86%)  1 1/32 (3.13%)  1
General disorders       
Catheter related complication   3/16 (18.75%)  3 0/35 (0.00%)  0 3/32 (9.38%)  3
Pyrexia   0/16 (0.00%)  0 2/35 (5.71%)  2 0/32 (0.00%)  0
Hepatobiliary disorders       
Cholecystitis   0/16 (0.00%)  0 1/35 (2.86%)  2 0/32 (0.00%)  0
Cholecystitis acute   0/16 (0.00%)  0 1/35 (2.86%)  1 0/32 (0.00%)  0
Gallbladder perforation   0/16 (0.00%)  0 1/35 (2.86%)  1 0/32 (0.00%)  0
Infections and infestations       
Bronchitis   0/16 (0.00%)  0 0/35 (0.00%)  0 1/32 (3.13%)  1
Catheter bacteraemia   1/16 (6.25%)  1 1/35 (2.86%)  2 0/32 (0.00%)  0
Catheter related infection   0/16 (0.00%)  0 0/35 (0.00%)  0 1/32 (3.13%)  1
Catheter sepsis   1/16 (6.25%)  3 3/35 (8.57%)  10 4/32 (12.50%)  5
Catheter site infection   0/16 (0.00%)  0 2/35 (5.71%)  2 0/32 (0.00%)  0
Fungemia   0/16 (0.00%)  0 1/35 (2.86%)  1 0/32 (0.00%)  0
Infectious mononucleosis   0/16 (0.00%)  0 1/35 (2.86%)  1 0/32 (0.00%)  0
Meningitis   0/16 (0.00%)  0 1/35 (2.86%)  1 0/32 (0.00%)  0
Pneumonia   0/16 (0.00%)  0 1/35 (2.86%)  1 0/32 (0.00%)  0
Sepsis   0/16 (0.00%)  0 1/35 (2.86%)  2 0/32 (0.00%)  0
Urinary tract infection   0/16 (0.00%)  0 1/35 (2.86%)  4 0/32 (0.00%)  0
Injury, poisoning and procedural complications       
Device failure   1/16 (6.25%)  1 0/35 (0.00%)  0 1/32 (3.13%)  1
Eye Injury   1/16 (6.25%)  1 0/35 (0.00%)  0 0/32 (0.00%)  0
Intestinal stoma complication   0/16 (0.00%)  0 0/35 (0.00%)  0 1/32 (3.13%)  1
Medical device complication   0/16 (0.00%)  0 1/35 (2.86%)  1 0/32 (0.00%)  0
Investigations       
Drug level increased   0/16 (0.00%)  0 1/35 (2.86%)  1 0/32 (0.00%)  0
Heart rate increased   0/16 (0.00%)  0 1/35 (2.86%)  1 0/32 (0.00%)  0
Musculoskeletal and connective tissue disorders       
Back pain   1/16 (6.25%)  1 0/35 (0.00%)  0 0/32 (0.00%)  0
Intervertebral discitis   1/16 (6.25%)  1 0/35 (0.00%)  0 0/32 (0.00%)  0
Pain in extremity   0/16 (0.00%)  0 0/35 (0.00%)  0 1/32 (3.13%)  1
Nervous system disorders       
Coma   0/16 (0.00%)  0 1/35 (2.86%)  1 0/32 (0.00%)  0
Dizziness   0/16 (0.00%)  0 1/35 (2.86%)  1 0/32 (0.00%)  0
Hypersomnia   0/16 (0.00%)  0 1/35 (2.86%)  1 0/32 (0.00%)  0
Respiratory, thoracic and mediastinal disorders       
Pneumothorax   1/16 (6.25%)  1 0/35 (0.00%)  0 0/32 (0.00%)  0
Surgical and medical procedures       
Catheterization venous   0/16 (0.00%)  0 0/35 (0.00%)  0 1/32 (3.13%)  1
Indicates events were collected by systematic assessment
Show Other (Not Including Serious) Adverse Events Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 5%
Placebo Teduglutide 0.05 mg/kg/d Teduglutide 0.1 mg/kg/d
Affected / at Risk (%) # Events Affected / at Risk (%) # Events Affected / at Risk (%) # Events
Total   15/16 (93.75%)      33/35 (94.29%)      31/32 (96.88%)    
Gastrointestinal disorders       
abdominal distension   0/16 (0.00%)  0 6/35 (17.14%)  7 3/32 (9.38%)  4
Abdominal pain   2/16 (12.50%)  2 7/35 (20.00%)  12 9/32 (28.13%)  11
diahrrea   0/16 (0.00%)  0 0/35 (0.00%)  0 3/32 (9.38%)  3
Dyspepsia   1/16 (6.25%)  1 0/35 (0.00%)  0 3/32 (9.38%)  4
Nausea   4/16 (25.00%)  4 5/35 (14.29%)  7 10/32 (31.25%)  13
Vomiting   2/16 (12.50%)  2 4/35 (11.43%)  8 6/32 (18.75%)  7
Abdominal Pain Upper   0/16 (0.00%)  0 4/35 (11.43%)  5 2/32 (6.25%)  4
General disorders       
Asthenia   0/16 (0.00%)  0 3/35 (8.57%)  3 2/32 (6.25%)  2
Catheter related complications   3/16 (18.75%)  3 1/35 (2.86%)  1 3/32 (9.38%)  4
Fatigue   2/16 (12.50%)  2 1/35 (2.86%)  1 5/32 (15.63%)  7
Injection site bruising   0/16 (0.00%)  0 1/35 (2.86%)  1 7/32 (21.88%)  8
Injection site erythema   0/16 (0.00%)  0 1/35 (2.86%)  1 6/32 (18.75%)  6
Injection site pain   0/16 (0.00%)  0 0/35 (0.00%)  0 4/32 (12.50%)  5
Pain   1/16 (6.25%)  1 0/35 (0.00%)  0 4/32 (12.50%)  4
Pyrexia   1/16 (6.25%)  1 3/35 (8.57%)  3 3/32 (9.38%)  6
Infections and infestations       
Catheter sepsis   2/16 (12.50%)  4 4/35 (11.43%)  12 4/32 (12.50%)  5
Catheter site infection   0/16 (0.00%)  0 3/35 (8.57%)  3 1/32 (3.13%)  1
Influenza   1/16 (6.25%)  1 3/35 (8.57%)  3 4/32 (12.50%)  4
Nasopharyngitis   2/16 (12.50%)  2 6/35 (17.14%)  9 5/32 (15.63%)  7
Urinary tract infection   3/16 (18.75%)  3 3/35 (8.57%)  7 5/32 (15.63%)  6
Injury, poisoning and procedural complications       
Intestinal stoma complications   0/16 (0.00%)  0 1/35 (2.86%)  1 5/32 (15.63%)  5
Musculoskeletal and connective tissue disorders       
Arthralgia   1/16 (6.25%)  1 2/35 (5.71%)  4 3/32 (9.38%)  5
Back pain   1/16 (6.25%)  2 0/35 (0.00%)  0 3/32 (9.38%)  3
Nervous system disorders       
Headache   1/16 (6.25%)  1 9/35 (25.71%)  29 7/32 (21.88%)  12
Respiratory, thoracic and mediastinal disorders       
Cough   0/16 (0.00%)  0 3/35 (8.57%)  3 1/32 (3.13%)  1
Indicates events were collected by systematic assessment
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.
Results Point of Contact
Name/Title: Head of Clinical Research
Organization: NPS Pharmaceuticals
Phone: 908-450-5300
EMail: info@npsp.com
Responsible Party: Shire
ClinicalTrials.gov Identifier: NCT00081458     History of Changes
Other Study ID Numbers: CL0600-004
First Submitted: April 13, 2004
First Posted: April 14, 2004
Results First Submitted: September 24, 2010
Results First Posted: July 15, 2013
Last Update Posted: December 8, 2015