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Completed

NCT Number: NCT03571516

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

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.

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Key information

Conditions

Short Bowel Syndrome Digestive System Diseases Gastrointestinal Diseases Intestinal Diseases Malabsorption Syndromes Pathologic Processes Pathological Conditions, Signs and Symptoms Postoperative Complications

Age range

4 month–12 month

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 3

Primary location

Helsingin yliopistollinen keskussairaala, Helsinki, Finland

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Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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:
  • International normalized ratio (INR) greater than (>) 1.5 not corrected with PN vitamin K
  • Platelet count <100×10^3/ microliter (mcL) due to portal hypertension
  • Presence of clinically significant gastric or esophageal varices
  • 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.

Treatment and study plan

Teduglutide

Drug

SC injection of 0.05 mg/kg teduglutide will be administered QD into abdomen or into either the thigh or arm for 24 weeks.

Standard Medical Therapy

Other

Standard medical therapy will be administered for 24 weeks.

Syringe

Device

Teduglutide will be administered using syringe (510k number: K980987).

needle

Device

Teduglutide will be administered using needle (510k number: K021475).

Primary outcomes

  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)

    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

  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

    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)

    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

    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

    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

    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

    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

    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

    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)

    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)

    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

    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)

    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)

    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)

    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)

    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)

    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)

    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)

    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)

    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)

    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.

  21. Number of Participants Who Achieved At Least 20 Percent (%) Increase 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)

    Number of participants who achieved at least 20% increase from baseline in weight-normalized EN volume at EOT/ET 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.

  22. Number of Participants Who Achieved At Least 20 Percent (%) Increase 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)

    Number of participants who achieved at least 20% increase from baseline in weight-normalized EN caloric intake at EOT/ET 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.

Sponsors and collaborators

Lead sponsor

Shire

Industry

Registry information

Official study title

A Randomized, Open-label, 24-Week Safety, Efficacy, and Pharmacokinetic Study of Teduglutide in Infants 4 to 12 Months of Age With Short Bowel Syndrome Who Are Dependent on Parenteral Support

Important dates

Study start
2018
Primary completion
2020
Study completion
2020
First posted
Jun 27, 2018
Registry last updated
May 11, 2021

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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