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Short Bowel SyndromePhase 3NCT02682381

Short Bowel Syndrome Research Study for Children Up To 17 Years of Age on Parenteral Nutrition

A Phase 3 study testing Teduglutide 0.05mg/kg and Teduglutide 0.025 mg/kg in people with Short Bowel Syndrome, run by Shire at 27 sites in 7 countries.

Completed

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

Official record
Search all trials

Trial summary

Teduglutide is approved for treatment of adults with short bowel syndrome (SBS). The purpose of this study is to evaluate the safety and efficacy of teduglutide in children up to the age of 17 with SBS who are dependent on parenteral support. Read more about this study

Who is it for?
Ages 0 to 17 years
Conditions the study is for
Short Bowel Syndrome
What is being tested?
Teduglutide 0.05mg/kg and Teduglutide 0.025 mg/kg
Could I receive a placebo?
No, everyone receives an active treatment
How many people
59 participants
Where
27 sitesin 7 countries
Study dates
23 Jun 2016to 18 Aug 2017
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 0 to 17 years
  • All sexes
  • No healthy volunteers
  • Child

Who may be able to join

  • Informed consent by a parent or guardian or emancipated minor prior to any study-related procedures
  • When applicable, an informed assent by the subject (as deemed appropriate by the Ethics Committee/Institutional Review Board) prior to any study-related procedures
  • Current history of SBS as a result of major intestinal resection, (eg, due to necrotizing enterocolitis, midgut volvulus, intestinal atresia, or gastroschisis)
  • Short bowel syndrome that requires PN/IV support that provides at least 30% of caloric and/or fluid/electrolyte needs prior to screening
  • Stable PN/IV support, defined as inability to significantly reduce PN/IV support, usually associated with minimal or no advance in enteral feeds (ie, 10% or less change in PN or advance in feeds) for at least 3 months prior to and during screening, as assessed by the investigator.
  • Sexually active female subjects of child-bearing potential (in the teduglutide treatment arm only) must use medically acceptable methods of birth control during and 4 weeks after the treatment period

Who cannot join

  • Subjects who are not expected to be able to advance oral or tube feeding regimens
  • Serial transverse enteroplasty or any other bowel lengthening procedure performed within 3 months of screening
  • Known clinically significant untreated intestinal obstruction contributing to feeding intolerance and inability to reduce parenteral support
  • Unstable absorption due to cystic fibrosis or known DNA abnormalities
  • Severe, known dysmotility syndrome, such as pseudo-obstruction or persistent, severe, active gastroschisis-related dysmotility, that is the primary contributing factor to feeding intolerance and inability to reduce parenteral support, prior to screening. Dysmotility is defined as severe if it is expected to limit the advancement of enteral feeding.
  • Evidence of clinically significant obstruction on upper GI series done within 6 months prior to screening.
Show all 29Show fewer
  • Major GI surgical intervention including significant intestinal resection within 3 months prior to the screening visit (insertion of feeding tube, anastomotic ulcer repair, minor intestinal resections ≤ 10 cm, or endoscopic procedure is allowed).
  • Unstable cardiac disease, congenital heart disease or cyanotic disease, with the exception of subjects who had undergone ventricular or atrial septal defect repair, and patent ductus arteriosus (PDA) ligation.
  • History of cancer or clinically significant lymphoproliferative disease, not including resected cutaneous basal or squamous cell carcinoma, or in situ non aggressive and surgically resected cancer.
  • Pregnant or lactating female subjects (in the teduglutide treatment arm only).
  • Participation in a clinical study using an experimental drug (other than glutamine or Omegaven) within 3 months or 5.5 half-lives of the experimental drug, whichever is longer, prior to screening, and for the duration of the study.
  • Previous use of teduglutide or native/synthetic glucagon-like peptide-2 (GLP-2)
  • Previous use of glucagon-like peptide-1 analog or human growth hormone within 3 months prior to screening
  • Previous use of octreotide, or dipeptidyl peptidase-4 (DPP-4) inhibitors within 3 months prior to screening
  • Subjects with active Crohn's disease who had been treated with biological therapy (eg, antitumor necrosis factor [anti-TNF]) within the 6 months prior to the screening visit
  • Subjects with inflammatory bowel disease (IBD) who require chronic systemic immunosuppressant therapy that had been introduced or changed during the 3 months prior to screening
  • More than 3 SBS-related or PN-related hospital admissions (eg, documented infection-related catheter sepsis, clots, bowel obstruction, severe water-electrolyte disturbances) within 3 months prior to the screening visit
  • Any major unscheduled hospital admission which affects parenteral support requirements within 1 month prior to or during screening, excluding uncomplicated treatment of bacteremia, central line replacement/repair, or issues of similar magnitude in an otherwise stable subject
  • Body weight < 10 kg at the screening and baseline visits
  • Signs of active severe or unstable, clinically significant hepatic impairment during the screening period, as indicated by any of the following laboratory test results :
  • Total bilirubin (TBL) ≥ 2 x upper limit of normal (ULN)
  • Aspartate aminotransferase (AST) ≥ 7x ULN
  • Alanine aminotransferase (ALT) ≥ 7x ULN
  • For subjects with Gilbert's disease:
  • Indirect (unconjugated) bilirubin ≥ 2x ULN
  • Signs of known continuous active or unstable, clinically significant renal dysfunction shown by results of an estimated glomerular filtration rate (eGFR) below 50 mL/min/1.73 m2.
  • Parent(s) and/or subjects who are not capable of understanding or not willing to adhere to the study visit schedule and other protocol requirements
  • Unstable, clinically significant active, untreated pancreatic or biliary disease
  • Any condition, disease, illness, or circumstance that in the investigator's opinion puts the subject at any undue risk, prevents completion of the study, or interferes with analysis of the study results.

Trial locations and contacts

27 sites in 7 countries.

27 sites in 7 countries

United States18 sites
  • 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

  • Cleveland Clinic Pediatric Specialists

    Cleveland, Ohio, 44195

  • Children's Medical Center Dallas

    Dallas, Texas, 75235

  • Duke Medical Center

    Durham, North Carolina, 27710

  • Texas Children's Hospital

    Houston, Texas, 77030

  • Riley Hospital for Children

    Indianapolis, Indiana, 46202

  • Children's Hospital Los Angeles - RHU

    Los Angeles, California, 90027

  • UCLA Dept. of Medicine

    Los Angeles, California, 90095

  • University of Wisconsin School of Medicine and Public Health

    Madison, Wisconsin, 53792

  • Children's Hospital GI Nutrition

    New York, New York, 10032

  • The Nebraska Medical Center

    Omaha, Nebraska, 68198

  • University of Pennsylvania Medical Center

    Philadelphia, Pennsylvania, 19104

  • UCSF Benioff Children's Hospital

    San Francisco, California, 94158

  • Seattle Children's Hospital

    Seattle, Washington, 98105

  • Montefiore Medical Center Child Spc

    The Bronx, New York, 10467

  • Georgetown Children's Research Network

    Washington D.C., District of Columbia, 20007

Other trials in United States

Canada3 sites
  • Walter C. Mackenzie Health Science Center

    Edmonton, Alberta, T6G 1C9

  • The Hospital for Sick Children

    Toronto, Ontario, M5G 1X8

  • British Columbia Children's & Women's Hospital Center

    Vancouver, British Columbia, V6H 3V4

Other trials in Canada

United Kingdom2 sites
  • Birmingham Children's Hospital

    Birmingham, B4 6NH

  • Great Ormond Street Hospital for Children

    London, Greater London, WC1N 3JH

Other trials in United Kingdom

Belgium1 site
  • Cliniques Universitaires Saint-Luc

    Brussels, 1200

Other trials in Belgium

Finland1 site
  • Helsingin yliopistollinen keskussairaala

    Helsinki, 00290

Other trials in Finland

Germany1 site
  • Universitaetsklinikum Tuebingen

    Tübingen, Baden Wuertternberg, 72076

Other trials in Germany

Italy1 site
  • Ospedale Pediatrico Bambino Gesu

    Roma, 00165

Other trials in Italy

About this study

Official title: A 24-Week Double-blind, Safety, Efficacy, and Pharmacodynamic Study Investigating Two Doses of Teduglutide in Pediatric Subjects Through 17 Years of Age With Short Bowel Syndrome Who Are Dependent on Parenteral Support

Teduglutide is approved for treatment of adults with short bowel syndrome (SBS). The purpose of this study is to evaluate the safety and efficacy of teduglutide in children up to the age of 17 with SBS who are dependent on parenteral support. Subjects may choose whether to receive the study drug or to participate in a standard-of-care arm. All participants who complete the study may be eligible to receive the study drug in a long-term extension study.

Being tested
Teduglutide 0.05mg/kg, Teduglutide 0.025 mg/kg, Standard of Care
Keywords
Teduglutide, pediatric, GLP-2, parenteral support, Short Bowel Syndrome, short gut syndrome, short gut, SBS, parenteral nutrition
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.

  • Teduglutide 0.05mg/kgDrug

    0.05 mg/kg

  • Teduglutide 0.025 mg/kgDrug

    0.025 mg/kg

  • Standard of CareOther

    Observational cohort for the 24-week treatment period and 4 week follow-up.

Study groups in NCT02682381
Study groupRoleWhat they receive
0.025 mg/kg/day TeduglutideExperimental0.025 milligrams per kilogram per day (mg/kg/day) of teduglutide for 24 weeks.
Receives: Drug: Teduglutide 0.025 mg/kg
0.05 mg/kg/day TeduglutideExperimental0.05 mg/kg/day of teduglutide for 24 weeks.
Receives: Drug: Teduglutide 0.05mg/kg
Standard of careActive comparatorObservational cohort for the 24-week treatment period and 4 week follow-up. The subjects in the standard of care group will follow the same visit schedule as the randomized subjects.
Receives: Other: Standard of Care

What is being measured

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

  1. Number of Participants Who Achieved at Least a 20 Percent (%) Reduction in Weight-Normalized Average Daily Parenteral Nutrition Intravenous (PN/IV) Volume at Week 24

    Reduction in weight-normalized PN/IV volume was performed using both participant diary and investigator prescribed data. Number of participants who achieved at least a 20% reduction in weight-normalized PN/IV volume between the baseline and week 24/EOT visit were reported.

    Measured: Baseline through Week 24

Secondary outcomes (19)
  1. Number of Participants With Treatment-emergent Adverse Events (TEAEs)

    An adverse event (AE) was defined as any untoward medical occurrence in a clinical investigation participant administered a pharmaceutical product and that did not necessarily have a causal relationship with this treatment. TEAEs were defined as AEs that started or worsened on or after the date of first dose for treatment groups and those that started or worsened on or after the baseline visit for standard of care group.

    Measured: From start of study treatment up to 28 weeks

  2. Number of Participants Who Were Completely Weaned Off Parenteral Nutrition Intravenous (PN/IV) Support at Week 24

    A participant was considered to have achieved independence from PN/IV support (completely weaned off PN/IV) if the investigator prescribed no PN/IV at EOT and there was no use of PN/IV recorded in the participant diary during the week prior to EOT.

    Measured: Week 24

  3. Change From Baseline in Parenteral Nutrition Intravenous (PN/IV) Volume at Week 24

    Change in PN/IV volume was reported based on the participant diary and the investigator prescribed data.

    Measured: Baseline, Week 24

  4. Change From Baseline in Parenteral Nutrition Intravenous (PN/IV) Caloric Intake at Week 24

    Change in PN/IV caloric intake was reported based on the participant diary and the investigator prescribed data.

    Measured: Baseline, Week 24

  5. Change From Baseline in Plasma Citrulline Levels at Week 24

    Plasma citrulline level was reported.

    Measured: Baseline, Week 24

  6. Change From Baseline in Enteral Nutrition Volume at Week 24

    Enteral nutrition was defined as specialized formula taken orally or by tube feeding, and excluded table foods and other fluids. Change in enteral nutrition volume was reported.

    Measured: Baseline, Week 24

  7. Change From Baseline in Enteral Nutrition Caloric Intake at Week 24

    Enteral nutrition was defined as specialized formula taken orally or by tube feeding, and excluded table foods and other fluids. Change in enteral nutrition caloric intake was reported.

    Measured: Baseline, Week 24

  8. Change From Week 24 in Parenteral Nutrition Intravenous (PN/IV) Volume at Week 28

    Change in PN/IV volume was reported.

    Measured: Week 24, Week 28

  9. Change From Week 24 in Parenteral Nutrition Intravenous (PN/IV) Caloric Intake at Week 28

    Change in PN/IV caloric intake was reported.

    Measured: Week 24, Week 28

  10. Change From Week 24 in Plasma Citrulline Levels at Week 28

    Change in plasma citrulline level was reported.

    Measured: Week 24, Week 28

  11. Change From Week 24 in Enteral Nutrition Volume at Week 28

    Enteral nutrition was defined as specialized formula taken orally or by tube feeding, and excluded table foods and other fluids. Change in enteral nutrition volume was reported.

    Measured: Week 24, Week 28

  12. Change From Week 24 in Enteral Nutrition Caloric Intake at Week 28

    Enteral nutrition was defined as specialized formula taken orally or by tube feeding, and excluded table foods and other fluids. Change in enteral nutrition caloric intake was reported.

    Measured: Week 24, Week 28

  13. Change From Baseline in Body Weight Z-score at Week 28

    Body weight z-score is a measure of relative weight adjusted for child age and sex. The Z-score indicates the number of standard deviations away from a reference population in the same age range and with the same sex. A Z-score of 0 is equal to the mean. Negative numbers indicate values lower than the mean and positive numbers indicate values higher than the mean.

    Measured: Baseline, Week 28

  14. Change From Baseline in Body Height Z-score at Week 28

    Body height z-score is a measure of relative height adjusted for child age and sex. The Z-score indicates the number of standard deviations away from a reference population in the same age range and with the same sex. A Z-score of 0 is equal to the mean. Negative numbers indicate values lower than the mean and positive numbers indicate values higher than the mean.

    Measured: Baseline, Week 28

  15. Change From Baseline in Head Circumference Z-score at Week 28

    Head circumference z-score is a measure of relative head circumference adjusted for child age and sex. The Z-score indicates the number of standard deviations away from a reference population in the same age range and with the same sex. A Z-score of 0 is equal to the mean. Negative numbers indicate values lower than the mean and positive numbers indicate values higher than the mean. Head circumference was collected only for participants of less than or equal to (<=) 36 months of age at the time of measurement.

    Measured: Baseline, Week 28

  16. Change From Baseline in Body Mass Index (BMI) Z-score at Week 28

    BMI z-score is a measure of relative BMI adjusted for child age and sex. The Z-score indicates the number of standard deviations away from a reference population in the same age range and with the same sex. A Z-score of 0 is equal to the mean. Negative numbers indicate values lower than the mean and positive numbers indicate values higher than the mean.

    Measured: Baseline, Week 28

  17. Change From Baseline in Participants' Stool Consistency at Week 28

    Stool consistency was assessed by typical stool form based on Bristol Stool Form Scale: 1 - Separate hard lumps, hard to pass, 2 - Sausage-shaped, but lumpy, 3 - Like a sausage but with cracks on the surface, 4 - Like a sausage or snake, smooth and soft, 5 - Soft blobs with clear-cut edges, 6 - Fluffy pieces with ragged edges, a mushy stool, 7 - Watery, no solid pieces, entirely liquid.

    Measured: Baseline, Week 28

  18. Change From Baseline in Hours Per Day of Parenteral Nutrition Intravenous (PN/IV) Support at Week 24

    The mean duration of the PN/IV infusions in hours, on the days when PN/IV was administered was reported.

    Measured: Baseline, Week 24

  19. Change From Baseline in Days Per Week of Parenteral Nutrition Intravenous (PN/IV) Support at Week 24

    The number of days per week of PN/IV infusions were reported.

    Measured: Baseline, Week 24

Timeline

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

  1. First posted15 Feb 2016 (estimated)
  2. Study start23 Jun 2016
  3. Primary completion18 Aug 2017
  4. Study completion18 Aug 2017

Resources

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
Single group
Masking
Triple blind
Who is masked
Participant, Care provider, Investigator
Enrolment
59 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT02682381
Sponsor's protocol ID
TED-C14-006
Status last verified
May 2021
First submitted
27 Jan 2016
Last updated
9 Jun 2021
Data monitoring committee
Yes
Individual data sharing
Yes: Takeda provides access to the de-identified individual participant data (IPD) for eligible studies to aid qualified researchers in addressing legitimate scientific objectives (Takeda's data sharing commitment is available on https://clinicaltrials.takeda.com/takedas-commitment?commitment=5). These IPDs will be provided in a secure research environment following approval of a data sharing request, and under the terms of a data sharing agreement.

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

Questions about this trial

What is this clinical trial studying?

A Phase 3 study testing Teduglutide 0.05mg/kg and Teduglutide 0.025 mg/kg in people with Short Bowel Syndrome, run by Shire at 27 sites in 7 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 29 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 27 sites in United States, Canada, United Kingdom, Belgium, Finland and 2 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. 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 16 Oct 2018). 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?

Number of Participants Who Achieved at Least a 20 Percent (%) Reduction in Weight-Normalized Average Daily Parenteral Nutrition Intravenous (PN/IV) Volume at Week 24 (time frame: Baseline through Week 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 NCT02682381, which also lists 1 linked publication.

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.

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