Short Bowel SyndromePhase 3NCT01952080

A Pharmacokinetic, Safety, and Pharmacodynamic Study of Teduglutide in Pediatric Subjects With Short Bowel Syndrome

A Phase 3 study testing teduglutide in people with Short Bowel Syndrome, run by Shire at 19 sites in 2 countries.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record

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Trial summary

Who is it for?: Ages 1 year to 17 years
Conditions the study is for: Short Bowel Syndrome
What is being tested?: teduglutide
Type of treatment: Injection or infusion
Could I receive a placebo?: No, everyone receives an active treatment
How many people: 42 participants
Where: 19 sitesin 2 countries
Study dates: 14 Nov 2013to 9 Jan 2015
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 1 year to 17 years
All sexes
No healthy volunteers
Child

Who may be able to join

Current history of SBS as a result of major intestinal resection, (eg, due to necrotizing enterocolitis, midgut volvulus, intestinal atresia, or gastroschisis) for at least 12 months prior to screening
Short bowel syndrome that requires parenteral nutrition/intravenous (PN/IV) support
Stable PN/IV support for at least 3 months prior to enrollment based upon the opinion of the investigator

Who cannot join

Serial transverse enteroplasty or any other bowel lengthening procedure performed within the past 3 months
Unstable absorption due to cystic fibrosis, untreated Hirschsprung's disease or known DNA abnormalities (ie, Familial Adenomatous Polyposis, Fanconi syndrome)
Evidence of obstruction on upper gastrointestinal (GI) series done within 6 months prior to screening
Major gastrointestinal surgical intervention within 3 months prior to screening (insertion of feeding tube or endoscopic procedure is allowed)

Trial locations and contacts

19 sites in 2 countries.

Country

City, postcode or hospital

19 sites in 2 countries

United States18 sites

Mott Children's Hospital, University of Michigan
Ann Arbor, Michigan, 48109
Children´s Hospital of Alabama
Birmingham, Alabama, 35233
Boston Children's Hospital
Boston, Massachusetts, 02115
Ann & Robert H. Lurie Children's Hospital of Chicago
Chicago, Illinois, 60611
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, 45229
Texas Children's Hospital, Baylor College of Medicine
Houston, Texas, 77030
Riley Hospital for Children at IU Health
Indianapolis, Indiana, 46202
Childrens Mercy Hospitals & Clinics Pediatric Gastroenterology
Kansas City, Missouri, 64108
Arkansas Children's Hospital
Little Rock, Arkansas, 72202
Children's Hospital Los Angeles
Los Angeles, California, 90024
Mattel Children's Hospital UCLA, Department of Pediatric Gastroenterology
Los Angeles, California, 90095
University of Wisconsin School of Medicine and Public Health Surgery
Madison, Wisconsin, 53792
University of Tennessee Health Science Center Le Bonheur Pediatric Specialists
Memphis, Tennessee, 38103
Monroe Carell, Jr. Children's Hospital at Vanderbilt
Nashville, Tennessee, 37232
University of Nebraska Medical Center
Omaha, Nebraska, 68198
Arnold Palmer Hospital for Children
Orlando, Florida, 32806
Seattle Childrens Hospital Gastroenterology and Hepatology
Seattle, Washington, 98105
Montefiore Medical Center
The Bronx, New York, 10467

Other trials in United States

United Kingdom1 site

Great Ormond Street Hospital for Children
London

Other trials in United Kingdom

About this study

Official title: A 12-Week Pharmacokinetic, Safety, and Pharmacodynamic Study of Teduglutide in Pediatric Subjects Aged 1 Year Through 17 Years, With Short Bowel Syndrome Who Are Dependent on Parenteral Support

This will be an open label, 4-cohort study. Non-randomized subjects will receive teduglutide in each of the 3 active cohorts. An attempt will be made to enroll additional subjects into an observational cohort who will receive standard of care. Three doses of teduglutide are to be investigated for 12 weeks. All subjects will be screened prior to the start of treatment (SOT) to establish baseline characteristics including safety, eligibility and nutritional support parameters.

Conditions: Short Bowel Syndrome
Being tested: teduglutide
Keywords: SBS, Pediatric SBS, Short Bowel Syndrome
Medical subject headings: Short Bowel Syndrome

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

teduglutideDrug
Open label intervention. Each subject will be assigned to receive 0.0125, 0.025, or 0.05 mg/kg/day for the duration of the study

Study groups in NCT01952080
Study group	Role	What they receive
teduglutide	Experimental	Open label teduglutide, subcutaneously injected. Receives: Drug: teduglutide
Standard of Care	No intervention	Not described

What is being measured

The primary outcome is the main question the study is designed to answer.

Percent Change in Parenteral Support [Parenteral Nutrition (PN)/Intravenous (IV)] Volume at Week 12
Percent change in PN/IV from the Baseline Visit to Week 12 Visit.
Measured: Baseline, Week 12
Percent Change in Parenteral Support (PN/IV) Volume at End of Treatment
Percent change in PN/IV from the Baseline Visit to End of Treatment Visit.
Measured: Baseline, End of Treatment
Percent Change in Parenteral Support (PN/IV) Volume at Week 16
Percent change in PN/IV from the Baseline Visit to Week 16 Visit.
Measured: Baseline, Week 16
Absolute Change in Parenteral Support (PN/IV) Volume at Week 12
Absolute change in PN/IV from the Baseline Visit to Week 12 Visit.
Measured: Baseline, Week 12
Absolute Change in Parenteral Support (PN/IV) Volume at End of Treatment
Absolute change in PN/IV from the Baseline Visit to End of Treatment Visit.
Measured: Baseline, End of Treatment
Absolute Change in Parenteral Support (PN/IV) Volume at Week 16
Absolute change in PN/IV from the Baseline Visit to Week 16 Visit.
Measured: Baseline, Week 16

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

14 Nov 20139 Jan 2015

First posted27 Sep 2013 (estimated)
Study start14 Nov 2013
Primary completion9 Jan 2015
Study completion9 Jan 2015

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 (2)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: Non-randomised
Intervention model: Single group
Masking: None (open label)
Enrolment: 42 participants (actual)

IDs, dates and publications

ClinicalTrials.gov ID: NCT01952080
Sponsor's protocol ID: TED-C13-003
Status last verified: May 2021
First submitted: 23 Sep 2013
Last updated: 9 Jun 2021
Results posted: Yes, on ClinicalTrials.gov
Data monitoring committee: Yes

Publications

Fifi A, Raphael BP, Terreri B, Uddin S, Kaufman SS. Effects of Teduglutide on Diarrhea in Pediatric Patients with Short Bowel Syndrome-Associated Intestinal Failure. J Pediatr Gastroenterol Nutr. 2023 Nov 1;77(5):666-671. doi: 10.1097/MPG.0000000000003922. Epub 2023 Aug 22.
PubMed 37889619
Carter BA, Cohran VC, Cole CR, Corkins MR, Dimmitt RA, Duggan C, Hill S, Horslen S, Lim JD, Mercer DF, Merritt RJ, Nichol PF, Sigurdsson L, Teitelbaum DH, Thompson J, Vanderpool C, Vaughan JF, Li B, Youssef NN, Venick RS, Kocoshis SA. Outcomes from a 12-Week, Open-Label, Multicenter Clinical Trial of Teduglutide in Pediatric Short Bowel Syndrome. J Pediatr. 2017 Feb;181:102-111.e5. doi: 10.1016/j.jpeds.2016.10.027. Epub 2016 Nov 15.
PubMed 27855998

Want to know more?

The sponsor has not listed a contact. The official record may have more details.

Talk to your doctor first and take this page with you. Only the study team can confirm whether you can join.

Questions to ask the study team

What is the study trying to find out?
What would I need to do, and how often would I visit?
Could I receive a placebo instead of the treatment?
What are the possible risks and benefits for me?
Will it cost me anything? Are travel or time paid for?
Can I keep seeing my own doctor during the study?
What happens when the study ends?

See locations

RecruitingObservational study
Rare Disease Patient Registry & Natural History Study - Coordination of Rare Diseases at Sanford
Rare Disorders, Undiagnosed Disorders
Active, not recruitingPhase 3
Efficacy and Safety of PJ009 in Patients With Short Bowel Syndrome Requiring Parenteral Nutrition
Short Bowel Syndrome
Not yet recruitingPhase 2
Efficacy and Safety of TRA16 Administered Orally in Patients With Enteric Hyperoxaluria (EH).
Enteric Hyperoxaluria
RecruitingPhase 3
Efficacy And Safety Evaluation of Glepaglutide in Treatment of SBS
Short Bowel Syndrome
Active, not recruitingObservational study
Registry for Participants With Short Bowel Syndrome
Short Bowel Syndrome

All Short Bowel Syndrome trials

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 12-Week Pharmacokinetic, Safety, and Pharmacodynamic Study of Teduglutide in Pediatric Subjects Aged 1 Year Through 17 Years, With Short Bowel Syndrome Who Are Dependent on Parenteral Support
ClinicalTrials.gov ID: NCT01952080
Acronym / sponsor ID: TED-C13-003
Conditions: Short Bowel Syndrome
Keywords: SBS, Pediatric SBS, Short Bowel Syndrome
Phase: Phase 3
Study type: Interventional
Recruitment status: Completed
Enrolment: 42 participants (actual)
Lead sponsor: ShireIndustry (pharma, biotech, devices)
Study start: 14 Nov 2013 (actual)
Primary completion: 9 Jan 2015 (actual)
Study completion: 9 Jan 2015 (actual)
First posted: 27 Sep 2013 (estimated)
Results: Posted 14 Sep 2015

Clinical summary

The research question as the sponsor registered it. This describes what the study plans to test, not what it found.

Study design

Primary purpose: Treatment
Allocation: Non-randomised
Intervention model: Single group
Masking: None (open label)
Number of arms: 2

Arm	Type	Receives
teduglutide	Experimental	Drug: teduglutideOpen label teduglutide, subcutaneously injected.
Standard of Care	No intervention	–

Who may qualify

Ages: 1 year to 17 years
Sex: All
Healthy volunteers: Not accepted
Age groups: Child

Key inclusion criteria (3 listed)

Current history of SBS as a result of major intestinal resection, (eg, due to necrotizing enterocolitis, midgut volvulus, intestinal atresia, or gastroschisis) for at least 12 months prior to screening
Short bowel syndrome that requires parenteral nutrition/intravenous (PN/IV) support
Stable PN/IV support for at least 3 months prior to enrollment based upon the opinion of the investigator

Key exclusion criteria (4 listed)

Serial transverse enteroplasty or any other bowel lengthening procedure performed within the past 3 months
Unstable absorption due to cystic fibrosis, untreated Hirschsprung's disease or known DNA abnormalities (ie, Familial Adenomatous Polyposis, Fanconi syndrome)
Evidence of obstruction on upper gastrointestinal (GI) series done within 6 months prior to screening
Major gastrointestinal surgical intervention within 3 months prior to screening (insertion of feeding tube or endoscopic procedure is allowed)

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
teduglutideOpen label intervention. Each subject will be assigned to receive 0.0125, 0.025, or 0.05 mg/kg/day for the duration of the study	Drug	teduglutide

Primary outcome

Percent Change in Parenteral Support [Parenteral Nutrition (PN)/Intravenous (IV)] Volume at Week 12
Time frame: Baseline, Week 12
Percent change in PN/IV from the Baseline Visit to Week 12 Visit.
Percent Change in Parenteral Support (PN/IV) Volume at End of Treatment
Time frame: Baseline, End of Treatment
Percent change in PN/IV from the Baseline Visit to End of Treatment Visit.
Percent Change in Parenteral Support (PN/IV) Volume at Week 16
Time frame: Baseline, Week 16
Percent change in PN/IV from the Baseline Visit to Week 16 Visit.
Absolute Change in Parenteral Support (PN/IV) Volume at Week 12
Time frame: Baseline, Week 12
Absolute change in PN/IV from the Baseline Visit to Week 12 Visit.
Absolute Change in Parenteral Support (PN/IV) Volume at End of Treatment
Time frame: Baseline, End of Treatment
Absolute change in PN/IV from the Baseline Visit to End of Treatment Visit.
Absolute Change in Parenteral Support (PN/IV) Volume at Week 16
Time frame: Baseline, Week 16
Absolute change in PN/IV from the Baseline Visit to Week 16 Visit.

Results and safety

Results posted to the registry on 14 Sep 2015, as reported by the sponsor. Figures are copied from the record, not re-analysed. Full results on ClinicalTrials.gov.

Participant flow

Group	Started	Completed
Standard of Care	5	5
Teduglutide 0.0125 mg/kg/Day	8	7
Teduglutide 0.025 mg/kg/Day	14	14
Teduglutide 0.05 mg/kg/Day	15	14

Primary outcome: Percent Change in Parenteral Support [Parenteral Nutrition (PN)/Intravenous (IV)] Volume at Week 12

Time frame: Baseline, Week 12. Mean (Standard Deviation). Unit: percent change

Measure	Standard of Care n = 5	Teduglutide 0.0125 mg/kg/Day n = 7	Teduglutide 0.025 mg/kg/Day n = 13	Teduglutide 0.05 mg/kg/Day n = 14
Value	7.38 (12.756)	-9.95 (21.625)	-37.34 (26.422)	-39.11 (40.792)

Population analysed: Percent change in PN/IV volume from baseline to Week 12 based on prescribed data - Intent-to-Treat Population (ITT), defined as all participants who were enrolled in the study.

Primary outcome: Percent Change in Parenteral Support (PN/IV) Volume at End of Treatment

Time frame: Baseline, End of Treatment. Mean (Standard Deviation). Unit: percent change

Measure	Standard of Care n = 5	Teduglutide 0.0125 mg/kg/Day n = 8	Teduglutide 0.025 mg/kg/Day n = 14	Teduglutide 0.05 mg/kg/Day n = 15
Value	7.38 (12.756)	-8.60 (20.380)	-35.61 (26.198)	-36.50 (40.585)

Population analysed: Percent change in PN/IV volume from baseline to End of Treatment based on prescribed data - Intent-to-Treat Population

Primary outcome: Percent Change in Parenteral Support (PN/IV) Volume at Week 16

Time frame: Baseline, Week 16. Mean (Standard Deviation). Unit: percent change

Measure	Standard of Care n = 5	Teduglutide 0.0125 mg/kg/Day n = 7	Teduglutide 0.025 mg/kg/Day n = 14	Teduglutide 0.05 mg/kg/Day n = 14
Value	3.92 (16.616)	-11.25 (21.196)	-33.85 (27.017)	-31.80 (39.264)

Population analysed: Percent change in PN/IV volume from baseline to Week 16 based on prescribed data - Intent-to-Treat Population

Primary outcome: Absolute Change in Parenteral Support (PN/IV) Volume at Week 12

Time frame: Baseline, Week 12. Mean (Standard Deviation). Unit: Liters/week

Measure	Standard of Care n = 5	Teduglutide 0.0125 mg/kg/Day n = 7	Teduglutide 0.025 mg/kg/Day n = 13	Teduglutide 0.05 mg/kg/Day n = 14
Value	0.43 (0.746)	-0.50 (0.910)	-2.78 (1.985)	-2.57 (3.564)

Population analysed: Absolute change in PN/IV volume from baseline to Week 12 based on prescribed data - Intent-to-Treat Population

Adverse events

Time frame: Adverse event data were collected from the first patient consent signed through last subject last visit for a duration of 421 days.. Other adverse events reported at 5% frequency or more

Group	Serious adverse events	Other adverse events	Deaths
Standard of Care	3 of 5 (60%)	5 of 5 (100%)	Not reported
Teduglutide 0.0125 mg/kg/Day	3 of 8 (37.5%)	8 of 8 (100%)	Not reported
Teduglutide 0.025 mg/kg/Day	6 of 14 (42.9%)	14 of 14 (100%)	Not reported
Teduglutide 0.05 mg/kg/Day	8 of 15 (53.3%)	15 of 15 (100%)	Not reported

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.
Derived from this study: PubMed 37889619Fifi A, Raphael BP, Terreri B, Uddin S, Kaufman SS. Effects of Teduglutide on Diarrhea in Pediatric Patients with Short Bowel Syndrome-Associated Intestinal Failure. J Pediatr Gastroenterol Nutr. 2023 Nov 1;77(5):666-671. doi: 10.1097/MPG.0000000000003922. Epub 2023 Aug 22.
Derived from this study: PubMed 27855998Carter BA, Cohran VC, Cole CR, Corkins MR, Dimmitt RA, Duggan C, Hill S, Horslen S, Lim JD, Mercer DF, Merritt RJ, Nichol PF, Sigurdsson L, Teitelbaum DH, Thompson J, Vanderpool C, Vaughan JF, Li B, Youssef NN, Venick RS, Kocoshis SA. Outcomes from a 12-Week, Open-Label, Multicenter Clinical Trial of Teduglutide in Pediatric Short Bowel Syndrome. J Pediatr. 2017 Feb;181:102-111.e5. doi: 10.1016/j.jpeds.2016.10.027. Epub 2016 Nov 15.

Related trials for Short Bowel Syndrome

RecruitingObservational study
Rare Disease Patient Registry & Natural History Study - Coordination of Rare Diseases at Sanford
Rare Disorders, Undiagnosed Disorders
Active, not recruitingPhase 3
Efficacy and Safety of PJ009 in Patients With Short Bowel Syndrome Requiring Parenteral Nutrition
Short Bowel Syndrome
Not yet recruitingPhase 2
Efficacy and Safety of TRA16 Administered Orally in Patients With Enteric Hyperoxaluria (EH).
Enteric Hyperoxaluria
RecruitingPhase 3
Efficacy And Safety Evaluation of Glepaglutide in Treatment of SBS
Short Bowel Syndrome
Active, not recruitingObservational study
Registry for Participants With Short Bowel Syndrome
Short Bowel Syndrome

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

19 sites in 2 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 19 sites in 2 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 3 inclusion and 4 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 19 sites in United States, United Kingdom. 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. 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 14 Sep 2015). 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?

Percent Change in Parenteral Support [Parenteral Nutrition (PN)/Intravenous (IV)] Volume at Week 12 (time frame: Baseline, Week 12). The study lists 6 primary outcomes in all. 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 NCT01952080, which also lists 2 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; AzTrials last refreshed its copy on 8 Nov 2026.