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NCT02980666

Study in Japanese Pediatric Subjects With Short Bowel Syndrome (SBS) Who Are Dependent on Parenteral Support

Completed Phase 3 Results posted Last updated 2 February 2022
What this trial tests

Phase 3 trial testing Teduglutide in Short Bowel Syndrome in 10 participants. Completed in 21 January 2020.

Timeline
13 January 2017
Primary endpoint
21 January 2020
21 January 2020

Quick facts

Lead sponsorShire
PhasePhase 3
StatusCompleted
Study typeINTERVENTIONAL
Allocationna
Designsingle group
Maskingnone
Primary purposetreatment
Enrollment10
Start date13 January 2017
Primary completion21 January 2020
Estimated completion21 January 2020
Sites5 locations across Japan

Drugs / interventions tested

Conditions studied

Sponsor

Shire — full company profile →

Who can join

Adults 4 Months to 15, any sex, with Short Bowel Syndrome. Patients with the condition only — healthy volunteers not accepted.

Results — posted to ClinicalTrials.gov

Per-arm endpoint measurements with 95% confidence intervals where reported. Source: trial results section.

Absolute Change From Baseline in Parenteral Support (PS) Volume at End of Treatment (EOT) Based on Dairy Data Primary · Baseline, EOT (up to Week 24)

Absolute change from baseline in PS volume at EOT (up to Week 24) based on dairy data was reported. EOT was defined as the last available measurement after the date of first dose during the 24-week treatment period. Here, milliliter per kilogram per day is abbreviated as mL/kg/day.

GroupValue95% CI
Total Children (Aged: 1 to 15 Years)-11.8± 8.47
Infants (Corrected Gestational Age: 4 to < 12 Months)-26.2± 13.61
Percent Change From Baseline in Parenteral Support (PS) Volume at End of Treatment (EOT) Based on Dairy Data Primary · Baseline, EOT (up to Week 24)

Percent change from baseline in PS volume at EOT (up to Week 24) based on dairy data was reported. EOT was defined as the last available measurement after the date of first dose during the 24-week treatment period.

GroupValue95% CI
Total Children (Aged: 1 to 15 Years)-27.7± 31.79
Infants (Corrected Gestational Age: 4 to < 12 Months)-26.7± 15.14
Absolute Change From Baseline in Parenteral Support (PS) Caloric Intake at End of Treatment (EOT) Based on Dairy Data Primary · Baseline, EOT (up to Week 24)

Absolute change from baseline in PS caloric intake at EOT (up to Week 24) based on dairy data was reported. EOT was defined as the last available measurement after the date of first dose during the 24-week treatment period. Here, kilo-calories per kilogram per day was abbreviated as (kcal/kg/day).

GroupValue95% CI
Total Children (Aged: 1 to 15 Years)-7.2± 8.70
Infants (Corrected Gestational Age: 4 to < 12 Months)-13.8± 3.17
Percent Change From Baseline in Parenteral Support (PS) Caloric Intake at End of Treatment (EOT) Based on Dairy Data Primary · Baseline, EOT (up to Week 24)

Percent change from baseline in PS caloric intake at EOT (up to Week 24) based on dairy data was reported. EOT was defined as the last available measurement after the date of first dose during the 24-week treatment period.

GroupValue95% CI
Total Children (Aged: 1 to 15 Years)-26.2± 33.00
Infants (Corrected Gestational Age: 4 to < 12 Months)-25.7± 2.73
Absolute Change From Baseline in Plasma Citrulline at End of Treatment (EOT) Primary · Baseline, EOT (up to Week 24)

Absolute change from baseline in plasma citrulline at EOT (up to Week 24) was reported. EOT was defined as the last available measurement after the date of first dose during the 24-week treatment period.

GroupValue95% CI
Total Children (Aged: 1 to 15 Years)3.2± 4.28
Percent Change From Baseline in Plasma Citrulline at End of Treatment (EOT) Primary · Baseline, EOT (up to Week 24)

Percent change from baseline in plasma citrulline at EOT (up to Week 24) was reported. EOT was defined as the last available measurement after the date of first dose during the 24-week treatment period.

GroupValue95% CI
Total Children (Aged: 1 to 15 Years)39.2± 39.46
Absolute Change From Baseline in Enteral Nutritional (EN) Volume at End of Treatment (EOT) Based on Dairy Data Primary · Baseline, EOT (up to Week 24)

Absolute change from baseline in EN volume at EOT (up to Week 24) based on dairy data was reported. EOT was defined as the last available measurement after the date of first dose during the 24-week treatment period.

GroupValue95% CI
Total Children (Aged: 1 to 15 Years)1.0± 7.14
Infants (Corrected Gestational Age: 4 to < 12 Months)0.6± 2.15
Percent Change From Baseline in Enteral Nutritional (EN) Volume at End of Treatment (EOT) Based on Dairy Data Primary · Baseline, EOT (up to Week 24)

Percent change from baseline in EN volume at EOT (up to Week 24) based on dairy data was reported. EOT was defined as the last available measurement after the date of first dose during the 24-week treatment period.

GroupValue95% CI
Total Children (Aged: 1 to 15 Years)53.1± 111.01
Infants (Corrected Gestational Age: 4 to < 12 Months)-23.2± 36.89
Absolute Change From Baseline in Enteral Nutritional (EN) Caloric Intake at End of Treatment (EOT) Based on Dairy Data Primary · Baseline, EOT (up to Week 24)

Absolute change from baseline in EN caloric intake at EOT (up to Week 24) based on dairy data was reported. EOT was defined as the last available measurement after the date of first dose during the 24-week treatment period.

GroupValue95% CI
Total Children (Aged: 1 to 15 Years)0.8± 5.60
Infants (Corrected Gestational Age: 4 to < 12 Months)0.3± 1.03
Percent Change From Baseline in Enteral Nutritional (EN) Caloric Intake at End of Treatment (EOT) Based on Dairy Data Primary · Baseline, EOT (up to Week 24)

Percent change from baseline in EN caloric intake at EOT (up to Week 24) based on dairy data was reported. EOT was defined as the last available measurement after the date of first dose during the 24-week treatment period.

GroupValue95% CI
Total Children (Aged: 1 to 15 Years)56.8± 117.88
Infants (Corrected Gestational Age: 4 to < 12 Months)-24.2± 38.06
Number of Participants Who Achieved At Least 20 Percent (%) Reduction in Parenteral Support (PS) Volume at Week 24 Primary · Week 24

Number of participants who achieved at least 20% reduction in PS volume at Week 24 was reported.

GroupValue95% CI
Total Children (Aged: 1 to 15 Years)4
Infants (Corrected Gestational Age: 4 to < 12 Months)1
Number of Participants Who Achieved At Least 20 Percent (%) Reduction in Parenteral Support (PS) Volume at End of Treatment (EOT) Primary · EOT (up to Week 24)

Number of participants who achieved at least 20% reduction in PS volume at EOT (up to Week 24) was reported. EOT was defined as the last available measurement after the date of first dose during the 24-week treatment period.

GroupValue95% CI
Total Children (Aged: 1 to 15 Years)4
Infants (Corrected Gestational Age: 4 to < 12 Months)1

Adverse events — posted to ClinicalTrials.gov

Time frame: From start of study drug administration up to end of the study (up to Week 28). Reporting threshold: 5%. Adverse-event reports describe events observed during the trial — not all are caused by the drug.

Total Children (Aged: 1 - 15 Years)
Serious: 6/8 (75%)
Deaths: 0/8
Infants (Corrected Gestational Age: 4 to < 12 Months)
Serious: 2/2 (100%)
Deaths: 0/2

Serious adverse events (12 terms)

ReactionSystemTotal Children (Aged: 1 - …Infants (Corrected Gestati…
Device related infectionInfections and infestations
EnterocolitisGastrointestinal disorders
PyrexiaGeneral disorders
Medical device site infectionInfections and infestations
Abdominal painGastrointestinal disorders
Anaphylactic reactionImmune system disorders
Adenovirus infectionInfections and infestations
GastroenteritisInfections and infestations
Hand-foot-and-mouth diseaseInfections and infestations
Upper respiratory tract infectionInfections and infestations
Device damageProduct Issues
Upper respiratory tract inflammationRespiratory, thoracic and mediastinal disorders
Other adverse events (44 terms — click to expand)

ReactionSystemTotal Children (Aged: 1 - …Infants (Corrected Gestati…
Viral upper respiratory tract infectionInfections and infestations
RashSkin and subcutaneous tissue disorders
Injection site erythemaGeneral disorders
ConstipationGastrointestinal disorders
Injection site bruisingGeneral disorders
PyrexiaGeneral disorders
Medical device site infectionInfections and infestations
Device damageProduct Issues
Dry skinSkin and subcutaneous tissue disorders
Abdominal distensionGastrointestinal disorders
Abdominal painGastrointestinal disorders
Dental cariesGastrointestinal disorders
EnteritisGastrointestinal disorders
EnterocolitisGastrointestinal disorders
GastritisGastrointestinal disorders
ProctalgiaGastrointestinal disorders
Catheter site granulomaGeneral disorders
Injection site haematomaGeneral disorders
Injection site indurationGeneral disorders
Injection site painGeneral disorders
Injection site rashGeneral disorders
Injection site reactionGeneral disorders
Food allergyImmune system disorders
Device related infectionInfections and infestations
GingivitisInfections and infestations
Hand-foot-and-mouth diseaseInfections and infestations
InfluenzaInfections and infestations
Otitis mediaInfections and infestations
Pharyngitis streptococcalInfections and infestations
RhinitisInfections and infestations
FrostbiteInjury, poisoning and procedural complications
Gastrostomy tube site complicationInjury, poisoning and procedural complications
Vaccination complicationInjury, poisoning and procedural complications
Amylase increasedInvestigations
DehydrationMetabolism and nutrition disorders
HyperlipasaemiaMetabolism and nutrition disorders
HypertriglyceridaemiaMetabolism and nutrition disorders
HyperzincaemiaMetabolism and nutrition disorders
HeadacheNervous system disorders
BalanoposthitisReproductive system and breast disorders

Most-reported serious reactions: Device related infection, Enterocolitis, Pyrexia, Medical device site infection, Abdominal pain, Anaphylactic reaction, Adenovirus infection, Gastroenteritis.

Data from ClinicalTrials.gov NCT02980666 adverse events section.

Sponsor's own description

The purpose of this study is to determine if an investigational treatment (teduglutide) is safe and effective in Japanese children (age 4 months through 15 years of age) with SBS who are dependent on parenteral support. This study will also evaluate how teduglutide moves through the body (pharmacokinetics) and how it affects the body (pharmacodynamics).

Publications & conference data

2 peer-reviewed publications reference this trial (live from Europe PMC):

  1. Efficacy and Safety of Teduglutide in Infants and Children With Short Bowel Syndrome Dependent on Parenteral Support.
    Chiba M, Masumoto K, Kaji T, Matsuura T, et al · · 2023 · cited 15× · PMID 37364133 · DOI 10.1097/mpg.0000000000003867
  2. [Use of teduglutide in short bowel syndrome in infants, children and adolescents: Position paper of the working group "Chronic intestinal failure" of the Society for Paediatric Gastroenterology and Nutrition (GPGE)].
    Posovszky C, de Laffolie J, Henning S, Ballauf A, et al · · 2026 · PMID 41672430 · DOI 10.1055/a-2757-3525

Verify or expand the search:

Other trials of Teduglutide

Trials testing the same drug.

Other recruiting trials for Short Bowel Syndrome

Currently open trials in the same condition.

Other Shire trials

Trials by the same sponsor.

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Data sources for this page

Drug Landscape aggregates and links these public records for informational use only. Always verify against the primary source before clinical or regulatory decisions. Canonical URL: https://druglandscape.com/trial/NCT02980666.

Primary sources · FDA · ClinicalTrials.gov · EMA · SEC EDGAR · ChEMBL · Wikidata · full sourcing