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Trial · Interventional

CompletedPhase 3Results postedRegistry data

Safety, Efficacy and Pharmacokinetic Study of Teduglutide in Infants 4 to 12 Months of Age With Short Bowel Syndrome

NCTNCT03571516 (opens ClinicalTrials.gov in a new tab)Other idSHP633-301Other id2017-003606-40Other id2017
Open at ClinicalTrials.govAsk about this trial

Overview

Status
Completed
Phase
Phase 3
Enrollment
10 participants (actual)
Sponsor
Shire · Industry
Design
Randomized · Open label (no masking)
Countries
4 countries

Finland, France, Italy, United Kingdom

Start
31 Aug 2018
Primary completion
24 Sept 2020
Completion
24 Sept 2020
First posted
27 Jun 2018
Last update posted
11 May 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
2018201920202021
  1. Ph 3

    Safety, Efficacy and Pharmacokinetic Study of Teduglutide in Infants 4 to 12 Months of Age With Short Bowel Syndrome

    NCT03571516

    31 Aug 2018 to 24 Sept 2020

Registry summary

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

The purpose of the study is to evaluate the safety, efficacy/pharmacodynamics (PD) and pharmacokinetics (PK) of teduglutide treatment in infants with short bowel syndrome (SBS) dependent on parenteral (PN) support.

Interventions

  • TeduglutideDrug
  • Standard Medical TherapyOther
  • SyringeDevice
  • NeedleDevice

Arms2

  • Experimental

    Teduglutide

    Participants will receive 0.05 milligram per kilogram (mg/kg) subcutaneous (SC) injection of teduglutide into abdomen or into either the thigh or arm once daily (QD) in addition to standard medical therapy for 24 weeks.

  • Other

    Standard of Care (SOC)

    Participants will receive standard medical therapy for 24 weeks.

Primary outcomes1

Outcomes the sponsor listed as primary.

  1. Number of Participants Who Achieved At Least 20 Percent (%) Reduction From Baseline in Weight-normalized Parenteral Support (PS) Volume at End of Treatment/Early Termination (EOT/ET)

    Time frame · Baseline, EOT/ET (up to Week 24)

    Registry description

    Number of participants who achieved at least 20% reduction from baseline in weight-normalized PS volume at EOT/ET (up to Week 24) were reported. EOT/ET was defined as the last available visit after the date of first dose (or randomization in standard of care treatment group) during the 24-week treatment period.

Secondary outcomes(20)

  1. Plasma Concentration of Teduglutide at Nominal Time Points (Baseline at Pre-dose, and 1 Hour and 4 Hours Post-dose; 2 Hours Post-dose at Week 7)

    Time frame · Baseline: Pre-dose,1, 4 hours post-dose, and 2 hours post-dose at Week 7

    Registry description

    Mean plasma concentration of teduglutide was reported.

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

    Time frame · From start of study treatment up to end of study (EOS) (up to Week 28)

    Registry description

    An adverse event (AE) was any untoward medical occurrence in a clinical investigation participant administered a pharmaceutical product and that does not necessarily have a causal relationship with this treatment. TEAEs are defined as AEs that start or deteriorate on or after the date of the first dose of investigational product.

  3. Change From Baseline in Body Weight Z-score at Week 24

    Time frame · Baseline, Week 24

    Registry description

    Body weight was measured using Z-score. Z-score was calculated as (observed value - median value of the reference population)/standard deviation value of reference population. A negative Z-score indicates values lower than the mean while a positive Z-score indicates values higher than the mean. Change from baseline in body weight Z-score at Week 24 was reported.

  4. Change From Baseline in Length Z-Score at Week 24

    Time frame · Baseline, Week 24

    Registry description

    Length was measured using Z-score. Z-score was calculated as (observed value - median value of the reference population)/standard deviation value of reference population. A negative Z-score indicates values lower than the mean while a positive Z-score indicates values higher than the mean. Change from baseline in length Z-score at Week 24 was reported.

  5. Change From Baseline in Head Circumference Z-Score at Week 24

    Time frame · Baseline, Week 24

    Registry description

    Head circumference was measured using Z-score. Z-score was calculated as (observed value - median value of the reference population)/standard deviation value of reference population. A negative Z-score indicates values lower than the mean while a positive Z-score indicates values higher than the mean. Change from baseline in head circumference Z-score at Week 24 was reported.

  6. Change From Baseline in Weight-for-Length Z-Score at Week 24

    Time frame · Baseline, Week 24

    Registry description

    Weight-for-length was measured using Z-score. Z-score was calculated as (observed value - median value of the reference population)/standard deviation value of reference population. A negative Z-score indicates values lower than the mean while a positive Z-score indicates values higher than the mean. Change from baseline in weight-for-length Z-score at Week 24 was reported.

  7. Change From Baseline in Average Total Urine Output at Week 24

    Time frame · Baseline, Week 24

    Registry description

    Average total urine output was recorded over a 48-hour period of nutritional stability at Week 24 was reported. Here, milliliter per kilogram per day is abbreviated as mL/kg/day.

  8. Change From Baseline in Fecal Output at Week 24

    Time frame · Baseline, Week 24

    Registry description

    Change from baseline in the fecal output (average number of stools per day) at Week 24 was reported.

  9. Number of Participants With Positive Specific Antibodies to Teduglutide

    Time frame · Baseline, EOS (up to week 28)

    Registry description

    Number of participants with positive specific antibodies to teduglutide were used to summarize the presence of antibodies.

  10. Number of Participants Who Achieved At Least 20 Percent (%) Reduction From Baseline in Weight-normalized Parenteral Support (PS) Caloric Intake at End of Treatment/Early Termination (EOT/ET)

    Time frame · Baseline, EOT/ET (up to Week 24)

    Registry description

    Number of participants who achieved at least 20% reduction from baseline in weight-normalized PS caloric intake at EOT/ET (up to Week 24) were reported. EOT/ET was defined as the last available visit after the date of first dose (or randomization in standard of care treatment group) during the 24-week treatment period.

  11. Number of Participants Who Achieved 100 Percent (%) Reduction in Complete Weaning Off (Enteral Autonomy) Parenteral Support (PS) Volume at Week 24

    Time frame · Week 24

    Registry description

    Number of participants who achieved 100% reduction in complete weaning off (enteral autonomy) PS volume at Week 24 were reported.

  12. Number of Participants Who Achieved 100 Percent (%) Reduction in Complete Weaning Off (Enteral Autonomy) Parenteral Support (PS) Volume at End of Study (EOS)

    Time frame · EOS (up to Week 28)

    Registry description

    Number of participants who achieved 100% reduction in complete weaning off (enteral autonomy) PS volume at EOS (up to Week 28) were reported.

  13. Change From Baseline in Weight-normalized Parenteral Support (PS) Volume at End of Treatment/Early Termination (EOT/ET)

    Time frame · Baseline, EOT/ET (up to Week 24)

    Registry description

    Change from baseline in weight-normalized PS volume at EOT/ET (up to Week 24) was reported. EOT/ET was defined as the last available visit after the date of first dose (or randomization in standard of care treatment group) during the 24-week treatment period.

  14. Percent Change From Baseline in Weight-normalized Parenteral Support (PS) Volume at End of Treatment/Early Termination (EOT/ET)

    Time frame · Baseline, EOT/ET (up to Week 24)

    Registry description

    Percent change from baseline in weight-normalized PS volume at EOT/ET (up to Week 24) was reported. EOT/ET was defined as the last available visit after the date of first dose (or randomization in standard of care treatment group) during the 24-week treatment period.

  15. Change From Baseline in Weight-normalized Parenteral Support (PS) Caloric Intake at End of Treatment/Early Termination (EOT/ET)

    Time frame · Baseline, EOT/ET (up to Week 24)

    Registry description

    Change from baseline in weight-normalized PS caloric intake at EOT/ET (up to Week 24) were reported. EOT/ET was defined as the last available visit after the date of first dose (or randomization in standard of care treatment group) during the 24-week treatment period. Here, kilo-calories per kilogram per day was abbreviated as (kcal/kg/day).

  16. Percent Change From Baseline in Weight-normalized Parenteral Support (PS) Caloric Intake at End of Treatment/Early Termination (EOT/ET)

    Time frame · Baseline, EOT/ET (up to Week 24)

    Registry description

    Percent change from baseline in weight-normalized PS caloric intake at EOT/ET (up to Week 24) were reported. EOT/ET was defined as the last available visit after the date of first dose (or randomization in standard of care treatment group) during the 24-week treatment period.

  17. Change From Baseline in Weight-normalized Enteral Nutrition (EN) Volume at End of Treatment/Early Termination (EOT/ET)

    Time frame · Baseline, EOT/ET (up to Week 24)

    Registry description

    Change from baseline in weight-normalized EN volume at EOT/ET (up to Week 24) was reported. EOT/ET was defined as the last available visit after the date of first dose (or randomization in standard of care treatment group) during the 24-week treatment period.

  18. Percent Change From Baseline in Weight-normalized Enteral Nutrition (EN) Volume at End of Treatment/Early Termination (EOT/ET)

    Time frame · Baseline, EOT/ET (up to Week 24)

    Registry description

    Percent change from baseline in weight-normalized EN volume at EOT/ET (up to Week 24) was reported. EOT/ET was defined as the last available visit after the date of first dose (or randomization in standard of care treatment group) during the 24-week treatment period.

  19. Change From Baseline in Weight-normalized Enteral Nutrition (EN) Caloric Intake at End of Treatment/Early Termination (EOT/ET)

    Time frame · Baseline, EOT/ET (up to Week 24)

    Registry description

    Change from baseline in weight-normalized EN caloric intake at EOT/ET (up to Week 24) were reported. EOT/ET was defined as the last available visit after the date of first dose (or randomization in standard of care treatment group) during the 24-week treatment period.

  20. Percent Change From Baseline in Weight-normalized Enteral Nutrition (EN) Caloric Intake at End of Treatment/Early Termination (EOT/ET)

    Time frame · Baseline, EOT/ET (up to Week 24)

    Registry description

    Percent change from baseline in weight-normalized EN caloric intake at EOT/ET (up to Week 24) were reported. EOT/ET was defined as the last available visit after the date of first dose (or randomization in standard of care treatment group) during the 24-week treatment period.

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, EOT/ET (up to Week 24)

Number of Participants Who Achieved At Least 20 Percent (%) Reduction From Baseline in Weight-normalized Parenteral Support (PS) Volume at End of Treatment/Early Termination (EOT/ET)

GroupValue (Participants)
Teduglutide (TED)3
Standard of Care (SOC)1
Secondary and other outcome results(7)
Secondary outcomeMedian· Baseline: Pre-dose,1, 4 hours post-dose, and 2 hours post-dose at Week 7

Plasma Concentration of Teduglutide at Nominal Time Points (Baseline at Pre-dose, and 1 Hour and 4 Hours Post-dose; 2 Hours Post-dose at Week 7)

GroupValue (Nanogram per milliliter (ng/mL))
Teduglutide (TED)0.00
Secondary outcomeCount of participants· From start of study treatment up to end of study (EOS) (up to Week 28)

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

GroupValue (Participants)
Teduglutide (TED)5
Standard of Care (SOC)5
Secondary outcomeMean· Baseline, Week 24

Change From Baseline in Body Weight Z-score at Week 24

GroupValue (Z-score)Spread
Teduglutide (TED)-0.4080.377
Standard of Care (SOC)-0.2890.278
Secondary outcomeMean· Baseline, Week 24

Change From Baseline in Length Z-Score at Week 24

GroupValue (Z-Score)Spread
Teduglutide (TED)-0.2741.258
Standard of Care (SOC)-0.4220.384
Secondary outcomeMean· Baseline, Week 24

Change From Baseline in Head Circumference Z-Score at Week 24

GroupValue (Z-Score)Spread
Teduglutide (TED)-0.5440.446
Standard of Care (SOC)-0.1670.690
Secondary outcomeMean· Baseline, Week 24

Change From Baseline in Weight-for-Length Z-Score at Week 24

GroupValue (Z-Score)Spread
Teduglutide (TED)-0.4471.042
Standard of Care (SOC)-0.0580.992
Secondary outcomeMean· Baseline, Week 24

Change From Baseline in Average Total Urine Output at Week 24

GroupValue (mL/kg/day)Spread
Teduglutide (TED)-0.6114.548
Standard of Care (SOC)10.2517.383

Adverse events, as posted

Adverse events as posted: participants affected out of participants at risk, by group
GroupDeathsSerious eventsOther events
Teduglutide (TED)Participants (infants) 4 to 12 months of corrected gestational aged received 0.05 milligram per kilogram (mg/kg) of teduglutide subcutaneous (SC) injection QD in addition to standard medical therapy for 24 weeks.0 / 54 / 55 / 5
Standard of Care (SOC)Participants (infants) 4 to 12 months of corrected gestational aged received standard medical therapy for 24 weeks.0 / 53 / 55 / 5

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.

More studies

1 study on this page

Indexing hint: Randomized controlled trial · 2023

Corrected

Efficacy and Safety of Teduglutide in Infants and Children With Short Bowel Syndrome Dependent on Parenteral Support.

Chiba et al. 2023Journal of pediatric gastroenterology and nutritioncited by 21

PMID 37364133PMC10412081DOI 10.1097/mpg.0000000000003867PUB-37364133

Free to read · publisher termsCC BY-NC-ND
Read at sourceRead the notice

Eligibility

Who could join, as posted.

Sex
All
Age
from 4 Months up to 12 Months
Healthy volunteers
Not accepted
Inclusion and exclusion criteria, as posted
Inclusion Criteria: * Informed consent by the parent or legal guardian. * Male or female infant 4 to 12 months corrected gestational age at screening. * Weight at least 5 kilogram (kg) and weight-for-length Z-score greater than -2 at screening and baseline. * Short bowel syndrome with dependence on parenteral support to provide at least 50% of fluid or caloric needs. * Stable PN requirements for at least 1 month prior to screening, defined as a less than or equal to (\<=) 10% change in the weight-normalized PN total fluid and caloric intake, despite attempts to wean PN, not withstanding transient instability for events such as sepsis or interruption of central venous access. * Parent or legal guardian understands and is willing and able to fully adhere to study requirements as defined in this protocol. Exclusion Criteria: * Previous treatment with teduglutide. * Intestinal malabsorption due to a genetic condition, such as cystic fibrosis, microvillus inclusion disease, etc. * 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 PN support, prior to screening. Dysmotility is defined as severe if it is expected to limit the advancement of enteral feeding. * Inability to advance oral or enteral feeding due to lack of access to the gut, such as oral aversion in the absence of a feeding tube. * Intestinal obstruction or clinically significant intestinal stenosis. * Major gastrointestinal surgical intervention, such as serial transverse enteroplasty or major intestinal resection or anastomosis, within 3 months prior to screening or planned during the study period. * Unstable cardiac disease. * Renal dysfunction, defined as estimated glomerular filtration rate less than (\<) 50 milliliter per minute (mL/min) per 1.73 square meter (m\^2). * Biliary obstruction, stenosis, or malformation. * Clinically significant pancreatic disease. * Severe hepatic dysfunction or portal hypertension, defined by at least 2 of the following parameters: 1. International normalized ratio (INR) greater than (\>) 1.5 not corrected with PN vitamin K 2. Platelet count \<100×10\^3/ microliter (mcL) due to portal hypertension 3. Presence of clinically significant gastric or esophageal varices 4. Documented cirrhosis * Persistent cholestasis defined as conjugated bilirubin \>4 milligram per deciliter (mg/dL) (\>68 micromoles per liter \[mcmol/L\]) over a 2 week period. * More than 3 serious complications of intestinal failure (example \[e.g.\], catheter-associated bloodstream infections, interruption of nutrition due to feeding intolerance, catheter-associated thrombosis, severe fluid or electrolyte disturbances) within 1 month prior to or during screening. * A history of cancer or a known cancer predisposition syndrome, such as juvenile polyposis or Beckwith-Wiedemann syndrome, or first degree relative with early onset of gastrointestinal cancer (including hepatobiliary and pancreatic cancers). * Concurrent treatment with glucagon-like peptide-1 (GLP-1); glucagon-like peptide-2 (GLP-2); insulin-like growth factor-1 (IGF-1); growth hormone, somatostatin, or analogs of these hormones; or glutamine. * Participation in a clinical study using an experimental drug within 3 months or 5.5 half-lives of the experimental drug, whichever is longer. * Known or suspected intolerance or hypersensitivity to the investigational product, closely-related compounds, or any of the stated ingredients. * Any condition, disease, illness, or circumstance that, in the investigator's opinion, puts the participant at any undue risk, prevents completion of the study, or interferes with analysis of the study results.

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