Skip to content

Trial · Interventional

CompletedPhase 3Results postedRegistry data

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

Overview

Status
Completed
Phase
Phase 3
Enrollment
59 participants (actual)
Sponsor
Shire · Industry
Design
Randomized · Triple masking
Countries
7 countries

United States, Belgium, Canada, Finland, Germany, Italy, United Kingdom

Start
23 Jun 2016
Primary completion
18 Aug 2017
Completion
18 Aug 2017
First posted
15 Feb 2016 (estimated)
Last update posted
9 Jun 2021
Conditions
Short Bowel Syndrome

Timeline

1 trial · 1 completed · 1 with posted results

  • Completed
  • Ongoing
  • Not yet recruiting
  • Stopped
  • Estimated dates (est.)
  • Results posted
201620172018
  1. Ph 3

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

    NCT02682381

    23 Jun 2016 to 18 Aug 2017

Registry summary

Text and fields below are the sponsor's entries at ClinicalTrials.gov, shown as posted.

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.

Interventions

  • Teduglutide 0.05mg/kgDrug
  • Teduglutide 0.025 mg/kgDrug
  • Standard of CareOther

Arms3

  • Experimental

    0.025 mg/kg/day Teduglutide

    0.025 milligrams per kilogram per day (mg/kg/day) of teduglutide for 24 weeks.

  • Experimental

    0.05 mg/kg/day Teduglutide

    0.05 mg/kg/day of teduglutide for 24 weeks.

  • Active comparator

    Standard of care

    Observational 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.

Primary outcomes1

Outcomes the sponsor listed as primary.

  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

    Time frame · Baseline through Week 24

    Registry description

    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.

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

    Time frame · From start of study treatment up to 28 weeks

    Registry description

    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.

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

    Time frame · Week 24

    Registry description

    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.

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

    Time frame · Baseline, Week 24

    Registry description

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

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

    Time frame · Baseline, Week 24

    Registry description

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

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

    Time frame · Baseline, Week 24

    Registry description

    Plasma citrulline level was reported.

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

    Time frame · Baseline, Week 24

    Registry description

    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.

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

    Time frame · Baseline, Week 24

    Registry description

    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.

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

    Time frame · Week 24, Week 28

    Registry description

    Change in PN/IV volume was reported.

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

    Time frame · Week 24, Week 28

    Registry description

    Change in PN/IV caloric intake was reported.

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

    Time frame · Week 24, Week 28

    Registry description

    Change in plasma citrulline level was reported.

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

    Time frame · Week 24, Week 28

    Registry description

    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.

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

    Time frame · Week 24, Week 28

    Registry description

    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.

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

    Time frame · Baseline, Week 28

    Registry description

    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.

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

    Time frame · Baseline, Week 28

    Registry description

    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.

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

    Time frame · Baseline, Week 28

    Registry description

    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.

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

    Time frame · Baseline, Week 28

    Registry description

    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.

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

    Time frame · Baseline, Week 28

    Registry description

    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.

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

    Time frame · Baseline, Week 24

    Registry description

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

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

    Time frame · Baseline, Week 24

    Registry description

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

Posted results

Values are shown as posted at ClinicalTrials.gov, with their units. PepBank has not converted, pooled or compared them.

Primary outcome results

Primary outcomeCount of participants· Baseline through Week 24

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

GroupValue (Participants)
0.025 mg/kg/Day Teduglutide13
0.05 mg/kg/Day Teduglutide18
Standard of Care1
Secondary and other outcome results(7)
Secondary outcomeCount of participants· From start of study treatment up to 28 weeks

Number of Participants With Treatment-emergent Adverse Events (TEAEs)

GroupValue (Participants)
0.025 mg/kg/Day Teduglutide24
0.05 mg/kg/Day Teduglutide25
Standard of Care9
Secondary outcomeCount of participants· Week 24

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

GroupValue (Participants)
0.025 mg/kg/Day Teduglutide2
0.05 mg/kg/Day Teduglutide3
Standard of Care0
Secondary outcomeMean· Baseline, Week 24

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

GroupValue (Milliliters per kilogram per day)Spread
0.025 mg/kg/Day Teduglutide-16.1610.52
0.05 mg/kg/Day Teduglutide-23.3017.50
Standard of Care-6.034.55
Secondary outcomeMean· Baseline, Week 24

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

GroupValue (Kilocalories per kilogram per day)Spread
0.025 mg/kg/Day Teduglutide-14.928.29
0.05 mg/kg/Day Teduglutide-18.9914.28
Standard of Care-0.464.95
Secondary outcomeMean· Baseline, Week 24

Change From Baseline in Plasma Citrulline Levels at Week 24

GroupValue (Micromoles per liter)Spread
0.025 mg/kg/Day Teduglutide7.78.50
0.05 mg/kg/Day Teduglutide12.012.00
Standard of Care0.17.79
Secondary outcomeMean· Baseline, Week 24

Change From Baseline in Enteral Nutrition Volume at Week 24

GroupValue (Milliliter per kilogram per day)Spread
0.025 mg/kg/Day Teduglutide7.6913.46
0.05 mg/kg/Day Teduglutide10.9616.59
Standard of Care0.745.91
Secondary outcomeMean· Baseline, Week 24

Change From Baseline in Enteral Nutrition Caloric Intake at Week 24

GroupValue (Kilocalorie per kilogram per day)Spread
0.025 mg/kg/Day Teduglutide8.4314.39
0.05 mg/kg/Day Teduglutide12.9818.93
Standard of Care4.2213.75

Adverse events, as posted

Adverse events as posted: participants affected out of participants at risk, by group
GroupDeathsSerious eventsOther events
0.025 mg/kg/Day TeduglutideParticipants received 0.025 mg/kg/day of teduglutide subcutaneously for 24 weeks along with standard medical therapy.0 / 2415 / 2424 / 24
0.05 mg/kg/Day TeduglutideParticipants received 0.05 mg/kg/day of teduglutide subcutaneously for 24 weeks along with standard medical therapy.0 / 2620 / 2623 / 26
Standard of CareParticipants received standard medical therapy.0 / 94 / 99 / 9

Each cell reads participants affected / participants at risk, as the registry posted them.

Registry data comes from ClinicalTrials.gov. Study results come from studies, not from the registry. How PepBank works

Studies1

Studies that cite this trial.

Studies with results

1 study on this page

Indexing hint: Clinical trial · 2023

3 results

Effects of Teduglutide on Diarrhea in Pediatric Patients with Short Bowel Syndrome-Associated Intestinal Failure.

Fifi et al. 2023Journal of pediatric gastroenterology and nutritioncited by 5

PMID 37889619PMC10583903DOI 10.1097/mpg.0000000000003922PUB-37889619

Free to read · publisher termsCC BY-NC-ND3 results

Eligibility

Who could join, as posted.

Sex
All
Age
from 0 Years up to 17 Years
Healthy volunteers
Not accepted
Inclusion and exclusion criteria, as posted
Inclusion Criteria: 1. Informed consent by a parent or guardian or emancipated minor prior to any study-related procedures 2. When applicable, an informed assent by the subject (as deemed appropriate by the Ethics Committee/Institutional Review Board) prior to any study-related procedures 3. Current history of SBS as a result of major intestinal resection, (eg, due to necrotizing enterocolitis, midgut volvulus, intestinal atresia, or gastroschisis) 4. Short bowel syndrome that requires PN/IV support that provides at least 30% of caloric and/or fluid/electrolyte needs prior to screening 5. 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. 6. 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 Exclusion Criteria: 1. Subjects who are not expected to be able to advance oral or tube feeding regimens 2. Serial transverse enteroplasty or any other bowel lengthening procedure performed within 3 months of screening 3. Known clinically significant untreated intestinal obstruction contributing to feeding intolerance and inability to reduce parenteral support 4. Unstable absorption due to cystic fibrosis or known DNA abnormalities 5. 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. 6. Evidence of clinically significant obstruction on upper GI series done within 6 months prior to screening. 7. 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). 8. 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. 9. 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. 10. Pregnant or lactating female subjects (in the teduglutide treatment arm only). 11. 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. 12. Previous use of teduglutide or native/synthetic glucagon-like peptide-2 (GLP-2) 13. Previous use of glucagon-like peptide-1 analog or human growth hormone within 3 months prior to screening 14. Previous use of octreotide, or dipeptidyl peptidase-4 (DPP-4) inhibitors within 3 months prior to screening 15. 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 16. 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 17. 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 18. 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 19. Body weight \< 10 kg at the screening and baseline visits 20. Signs of active severe or unstable, clinically significant hepatic impairment during the screening period, as indicated by any of the following laboratory test results : 1. Total bilirubin (TBL) ≥ 2 x upper limit of normal (ULN) 2. Aspartate aminotransferase (AST) ≥ 7x ULN 3. Alanine aminotransferase (ALT) ≥ 7x ULN For subjects with Gilbert's disease: 4. Indirect (unconjugated) bilirubin ≥ 2x ULN 21. 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. 22. 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 23. Unstable, clinically significant active, untreated pancreatic or biliary disease 24. 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.

PepBank is a research reference. Talk to a doctor before you use any peptide.