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

Primary Purpose

Short Bowel Syndrome

Status
Completed
Phase
Phase 3
Locations
International
Study Type
Interventional
Intervention
teduglutide
placebo
Sponsored by
Shire
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Short Bowel Syndrome focused on measuring SBS, short bowel syndrome, TPN, parenteral nutrition, PN

Eligibility Criteria

18 Years - undefined (Adult, Older Adult)All SexesDoes not accept healthy volunteers

Inclusion Criteria:

  • Signed and dated informed consent prior to any study-related procedures are performed
  • Men and women 18 years of age or older at the time of informed consent signing
  • Intestinal failure resulting in Short Bowel Syndrome
  • At least 12 months of continuous PN dependency
  • 12 weeks of clinical remission of Crohn's disease (CD) prior to dosing
  • PN required at least 3 times weekly
  • A stable PN volume for four weeks prior to dosing

Exclusion Criteria:

  • History of cancer or clinically significant lymphoproliferative disease with fewer than 5 years documented disease-free state
  • Participation in clinical study within 30 days for drug or 90 days for antibody
  • Use of native GLP-2 or human growth hormone (HGH) within 6 months of screening
  • Use of iv glutamine within 30 days prior to screening
  • Use of teduglutide
  • CD patients who have been treated with biological therapy within 6 months of screening
  • IBD patients who require chronic systemic immunosuppressant therapy
  • More than 4 SBS- or PN-related hospitalizations within 12 months of screening
  • Unplanned hospitalization within one month of screening
  • Pregnant or lactating women
  • Body weight > 88kg
  • Body mass index (BMI) < 15 kg/m2
  • Severe hepatic impairment or disturbed renal function
  • Female subjects who are not surgically sterile or postmenopausal or who are not using medically acceptable methods of birth control during and for 30 days after the treatment period
  • Not capable of understanding or not willing to adhere to the study visit schedules and other protocol requirements
  • Any condition or circumstance that is the investigator's opinion would put the subject at any undue risk, prevent completion of the study, or interfere with the analysis of the study results
  • Significant active, uncontrolled, untreated systemic diseases

Sites / Locations

  • Scripps Clinic & Research Foundation
  • Washington Hospital Center
  • Mayo Clinic Jacksonville
  • Emory University School of Medicine
  • Kansas University Medical Center
  • Mount Sinai Medical Center
  • Cleveland Clinic Foundation
  • University of Pennslyvania
  • University Of Pittsburgh Medical Center
  • Rhode Island Hospital
  • Vanderbilt University Medical Center
  • Critical Care Research, Royal Alexandra Hospital
  • Hamilton Health Sciences Centre
  • Polyclinic Family and Specialty Medicine Facility
  • University Health Network - Toronto General Hospital
  • Rigshospitalet - Abdominalcentret, Hepatologisk Kilinik A
  • Hôpital Beaujon
  • Hôpital Croix Rousse Unité de Nutrition Clinique Intensive
  • Hôpital de l'Archet Pôle Digestif
  • Med. Klinik m.S. Hepatologie und Gastroenterologie
  • Universitaetsklinikum Tuebingen
  • Israelitisches Krankenhaus
  • Azienda Ospedaliera Universitaria S. Giovanni Battista - Le Molinette
  • Policlinico Sant'Orsola - Malpighi centro insufficienza intestinale
  • Azienda Universitaria Policlinico Federico II
  • Universitair Medisch Centrum St. Radboud
  • Wojewodzki Szpital Specjalistyczny im. M. Pirogowa, Pracownia Leczenia Zywieniowego
  • Samodzielny Publiczny Szpital Kliniczny nr 4, Klinika Chirurgii Ogolnej i Transplantacyjnej
  • Wojewodzki Szpital Specjalistyczny,
  • Samodzielny Publiczny Szpital Kliniczny im. prof W. Orlowskiego CMKP, Oddzial Kliniczny Zywienia i Chirurgii
  • Hospital Universitario de Bellvitge
  • Hospital Universitario 12 de Octubre
  • St. Mark's Hospital Northwick Park
  • University College Hospital
  • Green Area, Main Hospital Salford Royal Hospitals NHS Foundation Trust

Arms of the Study

Arm 1

Arm 2

Arm Type

Experimental

Placebo Comparator

Arm Label

Teduglutide

Placebo

Arm Description

0.05 mg/kg/day sc dose of teduglutide

Matching subcutaneous dose of placebo to teduglutide

Outcomes

Primary Outcome Measures

Responder
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.

Secondary Outcome Measures

Absolute Change in PN/I.V. Volume From Baseline to Last Time Point
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).

Full Information

First Posted
November 25, 2008
Last Updated
May 13, 2021
Sponsor
Shire
Collaborators
Nycomed
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1. Study Identification

Unique Protocol Identification Number
NCT00798967
Brief Title
Study of Teduglutide Effectiveness in Parenteral Nutrition (PN)-Dependent Short Bowel Syndrome (SBS) Subjects
Acronym
STEPS
Official Title
A 24-Week Study of the Efficacy and Safety of Teduglutide in Subjects With Parenteral Nutrition-Dependent Short Bowel Syndrome
Study Type
Interventional

2. Study Status

Record Verification Date
May 2021
Overall Recruitment Status
Completed
Study Start Date
November 25, 2008 (Actual)
Primary Completion Date
January 25, 2011 (Actual)
Study Completion Date
January 25, 2011 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Shire
Collaborators
Nycomed

4. Oversight

Data Monitoring Committee
Yes

5. Study Description

Brief Summary
Teduglutide is an investigative medicine being evaluated as a possible treatment for people with parenteral nutrition (PN) dependent Short Bowel Syndrome (SBS). Teduglutide is similar to a protein the body makes. When people have SBS, their bodies do not make enough of the protein and they have trouble getting nutrients and fluids from the food they eat and drink. This study was designed to provide evidence of efficacy, safety, and tolerability of teduglutide 0.05 mg/kg daily in SBS subjects.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Short Bowel Syndrome
Keywords
SBS, short bowel syndrome, TPN, parenteral nutrition, PN

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 3
Interventional Study Model
Parallel Assignment
Masking
ParticipantCare ProviderInvestigatorOutcomes Assessor
Allocation
Randomized
Enrollment
86 (Actual)

8. Arms, Groups, and Interventions

Arm Title
Teduglutide
Arm Type
Experimental
Arm Description
0.05 mg/kg/day sc dose of teduglutide
Arm Title
Placebo
Arm Type
Placebo Comparator
Arm Description
Matching subcutaneous dose of placebo to teduglutide
Intervention Type
Drug
Intervention Name(s)
teduglutide
Other Intervention Name(s)
GATTEX, ALX-0600
Intervention Description
0.05 mg/kg/day sc injection for 24 weeks
Intervention Type
Drug
Intervention Name(s)
placebo
Intervention Description
Matching daily subcutaneous dose of placebo to teduglutide for 24 weeks
Primary Outcome Measure Information:
Title
Responder
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
Secondary Outcome Measure Information:
Title
Absolute Change in PN/I.V. Volume From Baseline to Last Time Point
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.

10. Eligibility

Sex
All
Minimum Age & Unit of Time
18 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria: Signed and dated informed consent prior to any study-related procedures are performed Men and women 18 years of age or older at the time of informed consent signing Intestinal failure resulting in Short Bowel Syndrome At least 12 months of continuous PN dependency 12 weeks of clinical remission of Crohn's disease (CD) prior to dosing PN required at least 3 times weekly A stable PN volume for four weeks prior to dosing Exclusion Criteria: History of cancer or clinically significant lymphoproliferative disease with fewer than 5 years documented disease-free state Participation in clinical study within 30 days for drug or 90 days for antibody Use of native GLP-2 or human growth hormone (HGH) within 6 months of screening Use of iv glutamine within 30 days prior to screening Use of teduglutide CD patients who have been treated with biological therapy within 6 months of screening IBD patients who require chronic systemic immunosuppressant therapy More than 4 SBS- or PN-related hospitalizations within 12 months of screening Unplanned hospitalization within one month of screening Pregnant or lactating women Body weight > 88kg Body mass index (BMI) < 15 kg/m2 Severe hepatic impairment or disturbed renal function Female subjects who are not surgically sterile or postmenopausal or who are not using medically acceptable methods of birth control during and for 30 days after the treatment period Not capable of understanding or not willing to adhere to the study visit schedules and other protocol requirements Any condition or circumstance that is the investigator's opinion would put the subject at any undue risk, prevent completion of the study, or interfere with the analysis of the study results Significant active, uncontrolled, untreated systemic diseases
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Study Director
Organizational Affiliation
Takeda
Official's Role
Study Director
Facility Information:
Facility Name
Scripps Clinic & Research Foundation
City
La Jolla
State/Province
California
ZIP/Postal Code
92037
Country
United States
Facility Name
Washington Hospital Center
City
Washington
State/Province
District of Columbia
ZIP/Postal Code
20010
Country
United States
Facility Name
Mayo Clinic Jacksonville
City
Jacksonville
State/Province
Florida
ZIP/Postal Code
32224
Country
United States
Facility Name
Emory University School of Medicine
City
Atlanta
State/Province
Georgia
ZIP/Postal Code
30322
Country
United States
Facility Name
Kansas University Medical Center
City
Kansas City
State/Province
Kansas
ZIP/Postal Code
66160
Country
United States
Facility Name
Mount Sinai Medical Center
City
New York
State/Province
New York
ZIP/Postal Code
10029
Country
United States
Facility Name
Cleveland Clinic Foundation
City
Cleveland
State/Province
Ohio
ZIP/Postal Code
44195
Country
United States
Facility Name
University of Pennslyvania
City
Philadelphia
State/Province
Pennsylvania
ZIP/Postal Code
19104
Country
United States
Facility Name
University Of Pittsburgh Medical Center
City
Pittsburgh
State/Province
Pennsylvania
ZIP/Postal Code
15213
Country
United States
Facility Name
Rhode Island Hospital
City
Providence
State/Province
Rhode Island
ZIP/Postal Code
02903
Country
United States
Facility Name
Vanderbilt University Medical Center
City
Nashville
State/Province
Tennessee
ZIP/Postal Code
37232
Country
United States
Facility Name
Critical Care Research, Royal Alexandra Hospital
City
Edmonton
State/Province
Alberta
ZIP/Postal Code
T5H 3V9
Country
Canada
Facility Name
Hamilton Health Sciences Centre
City
Hamilton
State/Province
Ontario
ZIP/Postal Code
L8N 3Z5
Country
Canada
Facility Name
Polyclinic Family and Specialty Medicine Facility
City
North York
State/Province
Ontario
ZIP/Postal Code
M3H 5S4
Country
Canada
Facility Name
University Health Network - Toronto General Hospital
City
Toronto
State/Province
Ontario
ZIP/Postal Code
M5G 2N2
Country
Canada
Facility Name
Rigshospitalet - Abdominalcentret, Hepatologisk Kilinik A
City
København
ZIP/Postal Code
2100
Country
Denmark
Facility Name
Hôpital Beaujon
City
Clichy
ZIP/Postal Code
92110
Country
France
Facility Name
Hôpital Croix Rousse Unité de Nutrition Clinique Intensive
City
Lyon
Country
France
Facility Name
Hôpital de l'Archet Pôle Digestif
City
Nice Cedex 3
ZIP/Postal Code
06100
Country
France
Facility Name
Med. Klinik m.S. Hepatologie und Gastroenterologie
City
Berlin
State/Province
BE
ZIP/Postal Code
13353
Country
Germany
Facility Name
Universitaetsklinikum Tuebingen
City
Tuebingen
State/Province
BW
ZIP/Postal Code
72076
Country
Germany
Facility Name
Israelitisches Krankenhaus
City
Hamburg
State/Province
HH
ZIP/Postal Code
22297
Country
Germany
Facility Name
Azienda Ospedaliera Universitaria S. Giovanni Battista - Le Molinette
City
Torino
State/Province
TO
ZIP/Postal Code
10126
Country
Italy
Facility Name
Policlinico Sant'Orsola - Malpighi centro insufficienza intestinale
City
Bologna
ZIP/Postal Code
40138
Country
Italy
Facility Name
Azienda Universitaria Policlinico Federico II
City
Napoli
ZIP/Postal Code
80131
Country
Italy
Facility Name
Universitair Medisch Centrum St. Radboud
City
Nijmegen
ZIP/Postal Code
6525 GA
Country
Netherlands
Facility Name
Wojewodzki Szpital Specjalistyczny im. M. Pirogowa, Pracownia Leczenia Zywieniowego
City
Lodz
ZIP/Postal Code
90-531
Country
Poland
Facility Name
Samodzielny Publiczny Szpital Kliniczny nr 4, Klinika Chirurgii Ogolnej i Transplantacyjnej
City
Lublin
ZIP/Postal Code
20-954
Country
Poland
Facility Name
Wojewodzki Szpital Specjalistyczny,
City
Olsztyn
ZIP/Postal Code
10-561
Country
Poland
Facility Name
Samodzielny Publiczny Szpital Kliniczny im. prof W. Orlowskiego CMKP, Oddzial Kliniczny Zywienia i Chirurgii
City
Warsaw
ZIP/Postal Code
00-416
Country
Poland
Facility Name
Hospital Universitario de Bellvitge
City
Barcelona
ZIP/Postal Code
08907
Country
Spain
Facility Name
Hospital Universitario 12 de Octubre
City
Madrid
ZIP/Postal Code
28041
Country
Spain
Facility Name
St. Mark's Hospital Northwick Park
City
Harrow
State/Province
Gt Lon
ZIP/Postal Code
HA1 3UJ
Country
United Kingdom
Facility Name
University College Hospital
City
London
State/Province
Gt Lon
ZIP/Postal Code
NW1 2BU
Country
United Kingdom
Facility Name
Green Area, Main Hospital Salford Royal Hospitals NHS Foundation Trust
City
Manchester
State/Province
Gt Man
ZIP/Postal Code
M6 8HD
Country
United Kingdom

12. IPD Sharing Statement

Citations:
PubMed Identifier
16099790
Citation
Jeppesen PB, Sanguinetti EL, Buchman A, Howard L, Scolapio JS, Ziegler TR, Gregory J, Tappenden KA, Holst J, Mortensen PB. Teduglutide (ALX-0600), a dipeptidyl peptidase IV resistant glucagon-like peptide 2 analogue, improves intestinal function in short bowel syndrome patients. Gut. 2005 Sep;54(9):1224-31. doi: 10.1136/gut.2004.061440.
Results Reference
result
PubMed Identifier
31784300
Citation
Jeppesen PB, Gabe SM, Seidner DL, Lee HM, Olivier C. Citrulline correlations in short bowel syndrome-intestinal failure by patient stratification: Analysis of 24 weeks of teduglutide treatment from a randomized controlled study. Clin Nutr. 2020 Aug;39(8):2479-2486. doi: 10.1016/j.clnu.2019.11.001. Epub 2019 Nov 9.
Results Reference
derived
PubMed Identifier
29174926
Citation
Jeppesen PB, Gabe SM, Seidner DL, Lee HM, Olivier C. Factors Associated With Response to Teduglutide in Patients With Short-Bowel Syndrome and Intestinal Failure. Gastroenterology. 2018 Mar;154(4):874-885. doi: 10.1053/j.gastro.2017.11.023. Epub 2017 Nov 22.
Results Reference
derived
PubMed Identifier
27507402
Citation
Fujioka K, Jeejeebhoy K, Pape UF, Li B, Youssef NN, Schneider SM. Patients With Short Bowel on Narcotics During 2 Randomized Trials Have Abdominal Complaints Independent of Teduglutide. JPEN J Parenter Enteral Nutr. 2017 Nov;41(8):1419-1422. doi: 10.1177/0148607116663481. Epub 2016 Aug 9.
Results Reference
derived
PubMed Identifier
24134154
Citation
Vipperla K, O'Keefe SJ. Study of teduglutide effectiveness in parenteral nutrition-dependent short-bowel syndrome subjects. Expert Rev Gastroenterol Hepatol. 2013 Nov;7(8):683-7. doi: 10.1586/17474124.2013.842894. Epub 2013 Oct 17.
Results Reference
derived
PubMed Identifier
23587733
Citation
Jeppesen PB, Pertkiewicz M, Forbes A, Pironi L, Gabe SM, Joly F, Messing B, Loth S, Youssef NN, Heinze H, Berghofer P. Quality of life in patients with short bowel syndrome treated with the new glucagon-like peptide-2 analogue teduglutide--analyses from a randomised, placebo-controlled study. Clin Nutr. 2013 Oct;32(5):713-21. doi: 10.1016/j.clnu.2013.03.016. Epub 2013 Mar 28.
Results Reference
derived
PubMed Identifier
23274148
Citation
Berghofer P, Fragkos KC, Baxter JP, Forbes A, Joly F, Heinze H, Loth S, Pertkiewicz M, Messing B, Jeppesen PB. Development and validation of the disease-specific Short Bowel Syndrome-Quality of Life (SBS-QoL) scale. Clin Nutr. 2013 Oct;32(5):789-96. doi: 10.1016/j.clnu.2012.12.001. Epub 2012 Dec 12.
Results Reference
derived
PubMed Identifier
22982184
Citation
Jeppesen PB, Pertkiewicz M, Messing B, Iyer K, Seidner DL, O'keefe SJ, Forbes A, Heinze H, Joelsson B. Teduglutide reduces need for parenteral support among patients with short bowel syndrome with intestinal failure. Gastroenterology. 2012 Dec;143(6):1473-1481.e3. doi: 10.1053/j.gastro.2012.09.007. Epub 2012 Sep 11.
Results Reference
derived

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

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