Short Bowel SyndromePhase 3NCT00081458
Safety and Efficacy Study of Teduglutide in Subjects With Short Bowel Syndrome
A Phase 3 study testing Teduglutide 0.05 mg/kg/d and Teduglutide 0.1 mg/kg/d in people with Short Bowel Syndrome, run by Shire at 32 sites in 9 countries.
Trial summary
The purpose of this study is to evaluate the efficacy, safety, tolerability, and pharmacokinetics (PK) of teduglutide compared with placebo in subjects with parenteral nutrition (PN)-dependent short bowel syndrome (SBS). 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 0.05 mg/kg/d and Teduglutide 0.1 mg/kg/d
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 84 participants
- Where
- 32 sitesin 9 countries
- Study dates
- 25 May 2004to 6 Jul 2007
- 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
- Men and women, aged 18 years of age or older at the time of signing the informed consent form (ICF)
- SBS as a result of major intestinal resection resulting in at least 12 months intravenous feeding
- Body weight must be less than 90 kg
- At baseline, subjects must require PN treatment to meet their caloric or electrolyte needs due to ongoing malabsorption at least 3 times weekly and to be on a stable PN regimen for 4 weeks before dosing
- Body mass index (BMI) 18 to 27 kg/m2
- Adequate hepatic and renal function
Who cannot join
- History of cancer or clinically significant lymphoproliferative disease with fewer than 5 years documented disease-free state
- History of alcohol or drug abuse (within previous year)
- Participation in a clinical study within 30 days prior to signing the ICF, or concurrent participation in any clinical study
- Clinically significant laboratory abnormalities at the time of randomization
- Previous use of teduglutide (ALX-0600)
- Prior use of native GLP-2 within 3 months of screening visit
Show all 10Show fewer
- Hospital admission within 1 month prior to screening visit
- Pregnant or lactating women
- Any condition or circumstance, which in the investigator's opinion would put the subject at any undue risk, prevent completion of the study, or interfere with analysis of the study results.
- Presence of excluded disease: Radiation enteritis, Scleroderma, Celiac disease, Refractory/Tropical sprue, Pseudo-obstruction, Active inflammatory bowel disease (IBD), Pre-malignant/malignant change in colonoscopy biopsy or polypectomy, Surgery scheduled within the time frame of the study, Human immunodeficiency virus (HIV) positive test, Immunological disorders, Possible allergies to teduglutide or its constituents, Significant, active, uncontrolled, untreated systemic diseases
Trial locations and contacts
32 sites in 9 countries.
32 sites in 9 countries
United States14 sites
Albany Medical Center
Albany, New York, 12208
Emory University Hospital
Atlanta, Georgia, 30322
Medical University of South Carolina
Charleston, South Carolina, 29425
Northwestern Center for Clinical Research
Chicago, Illinois, 60611
The Cleveland Clinic Foundation
Cleveland, Ohio, 44195
University of Kansas Medical Center
Kansas City, Kansas, 66160
Mount Sinai School of Medicine
New York, New York, 10029
University of Nebraska Medical Center
Omaha, Nebraska, 68198
University of Pennsylvania - Penn Nursing
Philadelphia, Pennsylvania, 19104
University of Pittsburgh Medical Center
Pittsburgh, Pennsylvania, 15213
Rhode Island Hospital
Providence, Rhode Island, 02903
University of Rochester Medical Center
Rochester, New York, 14642
Mayo Clinic Scottsdale
Scottsdale, Arizona, 85259
Georgetown University
Washington D.C., District of Columbia, 20007
Canada4 sites
Royal Alexandra Hospital
Edmonton, Alberta, T5H4B9
St. Michael's Hospital
Toronto, Ontario, M5B1W8
Toronto General Hospital
Toronto, Ontario, M5G2N2
St. Paul's Hospital
Vancouver, British Columbia, V6Z1Y6
France4 sites
Hôpital Claude-Huriez
Lille, 59037
Hôpital de la Croix-Rousse
Lyon, 69317
Hôpital Edouard Herriot
Lyon, 69437
Hôpital Beaujon
Paris, 92110
Germany3 sites
Charité University Hospital
Berlin, 10117
Charité-Universitätsmedizin Berlin
Berlin, 13353
Universitätsklinikum Frankfurt
Frankfurt, 60590
Poland2 sites
Pracownia Żywienia Klinicznego
Olsztyn, 10-651
Samodzielny Publiczny Szpital Kliniczny-Im.prof W.Orłowskiego CMKP
Warsaw, 00-416
United Kingdom2 sites
St. Mark's Hospital
Harrow, HA13UJ
Hope Hospital
Salford, Greater Manchester, M68HD
About this study
Official title: A Study of the Efficacy and Safety of Teduglutide in Subjects With Parenteral Nutrition-Dependent Short Bowel Syndrome
The purpose of this study is to evaluate the efficacy, safety, tolerability, and pharmacokinetics (PK) of teduglutide compared with placebo in subjects with parenteral nutrition (PN)-dependent short bowel syndrome (SBS).
Read the detailed description
Teduglutide is an analog of glucagon-like peptide 2 (GLP-2), a naturally occurring hormone that regulates the growth, proliferation, and maintenance of cells lining the gastrointestinal tract. Teduglutide has been shown in animal studies and previous human clinical trials to increase the size and number of these cells, thereby increasing the absorptive surface area of the intestines.
The multicenter, double-blind, international Phase III trial will randomly assign approximately 80 patients to receive daily subcutaneous injections of 0.05 milligrams or 0.10 milligrams of teduglutide per kilogram of body weight, or a placebo. Dosing will continue for a period of six months. The primary endpoint in the study is a reduction in the use of intravenous feeding, which is often required to sustain life in patients with SBS.
- Conditions
- Short Bowel Syndrome
- Being tested
- Placebo, Teduglutide 0.05 mg/kg/d, Teduglutide 0.1 mg/kg/d
- Keywords
- Short Bowel Syndrome, Parenteral Nutrition, SBS
- 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.
PlaceboDrug
placebo injectable subcutaneously daily into thigh or abdomen
Teduglutide 0.05 mg/kg/dDrug
Also called GATTEX
Teduglutide 0.05 mg/kg/d daily injectable subcutaneously into the thigh or abdomen
Teduglutide 0.1 mg/kg/dDrug
Also called GATTEX
Teduglutide 0.1 /g/kg/d daily injection subcutaneously into thigh or abdomen
| Study group | Role | What they receive |
|---|---|---|
| placebo | Placebo comparator | Placebo injectable subcutaneously daily into the thigh or abdomen Receives: Drug: Placebo |
| 2 | Experimental | teduglutide 0.05 mg/kg/d Receives: Drug: Teduglutide 0.05 mg/kg/d |
| 3 | Experimental | teduglutide 0.1 mg/kg/d Receives: Drug: Teduglutide 0.1 mg/kg/d |
What is being measured
The primary outcome is the main question the study is designed to answer.
A Graded Response Score in Parenteral Nutrition (PN) Reduction
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.
Measured: 6 months
Secondary outcomes (1)
Number of Subjects Achieving Binary Response at Week 20, Maintained at Week 24
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.
Measured: 6 months of treatment
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted14 Apr 2004 (estimated)
- Study start25 May 2004
- Primary completion6 Jul 2007
- Study completion6 Jul 2007
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 (6)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
- Triple blind
- Who is masked
- Participant, Care provider, Investigator
- Enrolment
- 84 participants (actual)
Sponsor and investigators
- Lead sponsor
- Shire
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Study Director, Study director, Takeda
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00081458
- Sponsor's protocol ID
- CL0600-004
- Other IDs
- 2004-000438-35, 2004
- Status last verified
- May 2021
- First submitted
- 13 Apr 2004
- Last updated
- 9 Jun 2021
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
Publications
Dudrick SJ, Latifi R, Fosnocht DE. Management of the short-bowel syndrome. Surg Clin North Am. 1991 Jun;71(3):625-43. doi: 10.1016/s0039-6109(16)45438-1.
PubMed 1904648Rombeau JL, Rolandelli RH. Enteral and parenteral nutrition in patients with enteric fistulas and short bowel syndrome. Surg Clin North Am. 1987 Jun;67(3):551-71. doi: 10.1016/s0039-6109(16)44232-5.
PubMed 3109044Shanbhogue LK, Molenaar JC. Short bowel syndrome: metabolic and surgical management. Br J Surg. 1994 Apr;81(4):486-99. doi: 10.1002/bjs.1800810404.
PubMed 8205419Vanderhoof JA, Langnas AN. Short-bowel syndrome in children and adults. Gastroenterology. 1997 Nov;113(5):1767-78. doi: 10.1053/gast.1997.v113.pm9352883.
PubMed 9352883Jeppesen PB, Gilroy R, Pertkiewicz M, Allard JP, Messing B, O'Keefe SJ. Randomised placebo-controlled trial of teduglutide in reducing parenteral nutrition and/or intravenous fluid requirements in patients with short bowel syndrome. Gut. 2011 Jul;60(7):902-14. doi: 10.1136/gut.2010.218271. Epub 2011 Feb 11.
PubMed 21317170Fujioka 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 27507402
Study at a glance
From the registry record, last updated by the sponsor on 9 Jun 2021. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Study of the Efficacy and Safety of Teduglutide in Subjects With Parenteral Nutrition-Dependent Short Bowel Syndrome
- ClinicalTrials.gov ID
- NCT00081458
- Acronym / sponsor ID
- CL0600-004
- Conditions
- Short Bowel Syndrome
- Keywords
- Short Bowel Syndrome, Parenteral Nutrition, SBS
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 84 participants (actual)
- Lead sponsor
- ShireIndustry (pharma, biotech, devices)
- Study start
- 25 May 2004 (actual)
- Primary completion
- 6 Jul 2007 (actual)
- Study completion
- 6 Jul 2007 (actual)
- First posted
- 14 Apr 2004 (estimated)
- Results
- Posted 15 Jul 2013
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The purpose of this study is to evaluate the efficacy, safety, tolerability, and pharmacokinetics (PK) of teduglutide compared with placebo in subjects with parenteral nutrition (PN)-dependent short bowel syndrome (SBS).
Detailed description
Teduglutide is an analog of glucagon-like peptide 2 (GLP-2), a naturally occurring hormone that regulates the growth, proliferation, and maintenance of cells lining the gastrointestinal tract. Teduglutide has been shown in animal studies and previous human clinical trials to increase the size and number of these cells, thereby increasing the absorptive surface area of the intestines.
The multicenter, double-blind, international Phase III trial will randomly assign approximately 80 patients to receive daily subcutaneous injections of 0.05 milligrams or 0.10 milligrams of teduglutide per kilogram of body weight, or a placebo. Dosing will continue for a period of six months. The primary endpoint in the study is a reduction in the use of intravenous feeding, which is often required to sustain life in patients with SBS.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Triple blind (participant, care provider, investigator)
- Number of arms
- 3
| Arm | Type | Receives |
|---|---|---|
| placebo | Placebo comparator | Drug: PlaceboPlacebo injectable subcutaneously daily into the thigh or abdomen |
| 2 | Experimental | Drug: Teduglutide 0.05 mg/kg/dteduglutide 0.05 mg/kg/d |
| 3 | Experimental | Drug: Teduglutide 0.1 mg/kg/dteduglutide 0.1 mg/kg/d |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (6 listed)
- Men and women, aged 18 years of age or older at the time of signing the informed consent form (ICF)
- SBS as a result of major intestinal resection resulting in at least 12 months intravenous feeding
- Body weight must be less than 90 kg
- At baseline, subjects must require PN treatment to meet their caloric or electrolyte needs due to ongoing malabsorption at least 3 times weekly and to be on a stable PN regimen for 4 weeks before dosing
- Body mass index (BMI) 18 to 27 kg/m2
Key exclusion criteria (10 listed)
- History of cancer or clinically significant lymphoproliferative disease with fewer than 5 years documented disease-free state
- History of alcohol or drug abuse (within previous year)
- Participation in a clinical study within 30 days prior to signing the ICF, or concurrent participation in any clinical study
- Clinically significant laboratory abnormalities at the time of randomization
- Previous use of teduglutide (ALX-0600)
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 |
|---|---|---|
| Placeboplacebo injectable subcutaneously daily into thigh or abdomen | Drug | placebo |
| Teduglutide 0.05 mg/kg/dAlso: GATTEXTeduglutide 0.05 mg/kg/d daily injectable subcutaneously into the thigh or abdomen | Drug | 2 |
| Teduglutide 0.1 mg/kg/dAlso: GATTEXTeduglutide 0.1 /g/kg/d daily injection subcutaneously into thigh or abdomen | Drug | 3 |
Primary outcome
A Graded Response Score in Parenteral Nutrition (PN) Reduction
Time frame: 6 months
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.
1 secondary outcome
Number of Subjects Achieving Binary Response at Week 20, Maintained at Week 24
Time frame: 6 months of treatment
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 9 Jun 2021.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Background: PubMed 1904648Dudrick SJ, Latifi R, Fosnocht DE. Management of the short-bowel syndrome. Surg Clin North Am. 1991 Jun;71(3):625-43. doi: 10.1016/s0039-6109(16)45438-1.
- Background: PubMed 3109044Rombeau JL, Rolandelli RH. Enteral and parenteral nutrition in patients with enteric fistulas and short bowel syndrome. Surg Clin North Am. 1987 Jun;67(3):551-71. doi: 10.1016/s0039-6109(16)44232-5.
- Background: PubMed 8205419Shanbhogue LK, Molenaar JC. Short bowel syndrome: metabolic and surgical management. Br J Surg. 1994 Apr;81(4):486-99. doi: 10.1002/bjs.1800810404.
- Background: PubMed 9352883Vanderhoof JA, Langnas AN. Short-bowel syndrome in children and adults. Gastroenterology. 1997 Nov;113(5):1767-78. doi: 10.1053/gast.1997.v113.pm9352883.
- Results publication: PubMed 21317170Jeppesen PB, Gilroy R, Pertkiewicz M, Allard JP, Messing B, O'Keefe SJ. Randomised placebo-controlled trial of teduglutide in reducing parenteral nutrition and/or intravenous fluid requirements in patients with short bowel syndrome. Gut. 2011 Jul;60(7):902-14. doi: 10.1136/gut.2010.218271. Epub 2011 Feb 11.
- 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.
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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
32 sites in 9 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing Teduglutide 0.05 mg/kg/d and Teduglutide 0.1 mg/kg/d in people with Short Bowel Syndrome, run by Shire at 32 sites in 9 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 6 inclusion and 10 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 9 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 32 sites in United States, Canada, France, Germany, Poland and 4 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 15 Jul 2013). 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?
A Graded Response Score in Parenteral Nutrition (PN) Reduction (time frame: 6 months). 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 NCT00081458, which also lists 6 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 9 Jun 2021.