Short Bowel SyndromePhase 3STEPSNCT00798967
Study of Teduglutide Effectiveness in Parenteral Nutrition (PN)-Dependent Short Bowel Syndrome (SBS) Subjects
A Phase 3 study testing teduglutide in people with Short Bowel Syndrome, run by Shire at 35 sites in 10 countries.
Trial 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. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Short Bowel Syndrome
- What is being tested?
- teduglutide
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 86 participants
- Where
- 35 sitesin 10 countries
- Study dates
- 25 Nov 2008to 25 Jan 2011
- Phase
- Phase 3
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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
Who cannot join
- 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
Show all 17Show fewer
- 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
Trial locations and contacts
35 sites in 10 countries.
35 sites in 10 countries
United States11 sites
Emory University School of Medicine
Atlanta, Georgia, 30322
Cleveland Clinic Foundation
Cleveland, Ohio, 44195
Mayo Clinic Jacksonville
Jacksonville, Florida, 32224
Kansas University Medical Center
Kansas City, Kansas, 66160
Scripps Clinic & Research Foundation
La Jolla, California, 92037
Vanderbilt University Medical Center
Nashville, Tennessee, 37232
Mount Sinai Medical Center
New York, New York, 10029
University of Pennslyvania
Philadelphia, Pennsylvania, 19104
University Of Pittsburgh Medical Center
Pittsburgh, Pennsylvania, 15213
Rhode Island Hospital
Providence, Rhode Island, 02903
Washington Hospital Center
Washington D.C., District of Columbia, 20010
Canada4 sites
Critical Care Research, Royal Alexandra Hospital
Edmonton, Alberta, T5H 3V9
Hamilton Health Sciences Centre
Hamilton, Ontario, L8N 3Z5
Polyclinic Family and Specialty Medicine Facility
North York, Ontario, M3H 5S4
University Health Network - Toronto General Hospital
Toronto, Ontario, M5G 2N2
Poland4 sites
Wojewodzki Szpital Specjalistyczny im. M. Pirogowa, Pracownia Leczenia Zywieniowego
Lodz, 90-531
Samodzielny Publiczny Szpital Kliniczny nr 4, Klinika Chirurgii Ogolnej i Transplantacyjnej
Lublin, 20-954
Wojewodzki Szpital Specjalistyczny,
Olsztyn, 10-561
Samodzielny Publiczny Szpital Kliniczny im. prof W. Orlowskiego CMKP, Oddzial Kliniczny Zywienia i Chirurgii
Warsaw, 00-416
France3 sites
Hôpital Beaujon
Clichy, 92110
Hôpital Croix Rousse Unité de Nutrition Clinique Intensive
Lyon
Hôpital de l'Archet Pôle Digestif
Nice, 06100
Germany3 sites
Med. Klinik m.S. Hepatologie und Gastroenterologie
Berlin, State of Berlin, 13353
Israelitisches Krankenhaus
Hamburg, Free and Hanseatic City of Hamburg, 22297
Universitaetsklinikum Tuebingen
Tübingen, Baden-Wurttemberg, 72076
Italy3 sites
Policlinico Sant'Orsola - Malpighi centro insufficienza intestinale
Bologna, 40138
Azienda Universitaria Policlinico Federico II
Naples, 80131
Azienda Ospedaliera Universitaria S. Giovanni Battista - Le Molinette
Torino, TO, 10126
United Kingdom3 sites
St. Mark's Hospital Northwick Park
Harrow, GT LON, HA1 3UJ
University College Hospital
London, GT LON, NW1 2BU
Green Area, Main Hospital Salford Royal Hospitals NHS Foundation Trust
Manchester, GT MAN, M6 8HD
Spain2 sites
Hospital Universitario de Bellvitge
Barcelona, 08907
Hospital Universitario 12 de Octubre
Madrid, 28041
Denmark1 site
Rigshospitalet - Abdominalcentret, Hepatologisk Kilinik A
Copenhagen, 2100
Netherlands1 site
Universitair Medisch Centrum St. Radboud
Nijmegen, 6525 GA
About this study
Official title: A 24-Week Study of the Efficacy and Safety of Teduglutide in Subjects With Parenteral Nutrition-Dependent Short Bowel Syndrome
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.
- Conditions
- Short Bowel Syndrome
- Being tested
- teduglutide, placebo
- Keywords
- SBS, short bowel syndrome, TPN, parenteral nutrition, PN
- Medical subject headings
- Short Bowel Syndrome, Hyperphagia
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
teduglutideDrug
Also called GATTEX, ALX-0600
0.05 mg/kg/day sc injection for 24 weeks
placeboDrug
Matching daily subcutaneous dose of placebo to teduglutide for 24 weeks
| Study group | Role | What they receive |
|---|---|---|
| Teduglutide | Experimental | 0.05 mg/kg/day sc dose of teduglutide Receives: Drug: teduglutide |
| Placebo | Placebo comparator | Matching subcutaneous dose of placebo to teduglutide Receives: Drug: placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: Weeks 20 and 24
Secondary outcomes (1)
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).
Measured: Week 0 to last visit when data was collected.
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted27 Nov 2008 (estimated)
- Study start25 Nov 2008
- Primary completion25 Jan 2011
- Study completion25 Jan 2011
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Publications (8)Papers the sponsor has linked to this study.
- Other trials for Short Bowel SyndromeStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 3
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 86 participants (actual)
Sponsor and investigators
- Lead sponsor
- Shire
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- Nycomed
- Responsible party
- Sponsor
- Investigators
- Study Director, Study director, Takeda
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00798967
- Sponsor's protocol ID
- CL0600-020
- Other IDs
- 2008-006193-15, 2008
- Acronym
- STEPS
- Status last verified
- May 2021
- First submitted
- 25 Nov 2008
- Last updated
- 3 Jun 2021
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
Publications
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.
PubMed 16099790Jeppesen 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.
PubMed 31784300Jeppesen 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.
PubMed 29174926Fujioka 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.
PubMed 27507402Vipperla 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.
PubMed 24134154Jeppesen 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.
PubMed 23587733Berghofer 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.
PubMed 23274148Jeppesen 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.
PubMed 22982184
Study at a glance
From the registry record, last updated by the sponsor on 3 Jun 2021. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A 24-Week Study of the Efficacy and Safety of Teduglutide in Subjects With Parenteral Nutrition-Dependent Short Bowel Syndrome
- ClinicalTrials.gov ID
- NCT00798967
- Acronym / sponsor ID
- STEPS / CL0600-020
- Conditions
- Short Bowel Syndrome
- Keywords
- SBS, short bowel syndrome, TPN, parenteral nutrition, PN
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 86 participants (actual)
- Lead sponsor
- ShireIndustry (pharma, biotech, devices)
- Collaborators
- Nycomed
- Study start
- 25 Nov 2008 (actual)
- Primary completion
- 25 Jan 2011 (actual)
- Study completion
- 25 Jan 2011 (actual)
- First posted
- 27 Nov 2008 (estimated)
- Results
- Posted 1 Feb 2012
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Teduglutide | Experimental | Drug: teduglutide0.05 mg/kg/day sc dose of teduglutide |
| Placebo | Placebo comparator | Drug: placeboMatching subcutaneous dose of placebo to teduglutide |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (7 listed)
- 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
Key exclusion criteria (17 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| teduglutideAlso: GATTEX, ALX-06000.05 mg/kg/day sc injection for 24 weeks | Drug | Teduglutide |
| placeboMatching daily subcutaneous dose of placebo to teduglutide for 24 weeks | Drug | Placebo |
Primary outcome
Responder
Time frame: Weeks 20 and 24
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.
1 secondary outcome
Absolute Change in PN/I.V. Volume From Baseline to Last Time Point
Time frame: Week 0 to last visit when data was collected.
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 3 Jun 2021.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Results publication: PubMed 16099790Jeppesen 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.
- Derived from this study: PubMed 31784300Jeppesen 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.
- Derived from this study: PubMed 29174926Jeppesen 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.
- Derived from this study: PubMed 27507402Fujioka 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.
- Derived from this study: PubMed 24134154Vipperla 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.
- Derived from this study: PubMed 23587733Jeppesen 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.
- Derived from this study: PubMed 23274148Berghofer 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.
- Derived from this study: PubMed 22982184Jeppesen 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.
- HPN supportLink provided by the sponsor.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Study Director study director
Takeda
35 sites in 10 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing teduglutide in people with Short Bowel Syndrome, run by Shire at 35 sites in 10 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 7 inclusion and 17 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 3 Jun 2021. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 35 sites in United States, Canada, Poland, France, Germany and 5 more countries. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 1 Feb 2012). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Responder (time frame: Weeks 20 and 24). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT00798967, which also lists 8 linked publications.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 3 Jun 2021.