Phase 3
N=10
Study in Japanese Pediatric Subjects With Short Bowel Syndrome (SBS) Who Are Dependent on Parenteral Support
Short Bowel Syndrome
Bottom Line
View on ClinicalTrials.gov: NCT02980666 ↗Enrolled (actual)
10
Serious AEs
80.0%
Results posted
Feb 2021
Primary outcome: Primary: Absolute Change From Baseline in Parenteral Support (PS) Volume at End of Treatment (EOT) Based on Dairy Data — -11.8; -26.2 mL/kg/day
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 3
- Interventions
- Teduglutide (Drug)
- Age
- Pediatric · 0+ yrs
- Sex
- All
- Sponsor
- Shire
- Primary completion
- Jan 2020
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Absolute Change From Baseline in Parenteral Support (PS) Volume at End of Treatment (EOT) Based on Dairy Data |
-11.8; -26.2 | — |
| PRIMARY Percent Change From Baseline in Parenteral Support (PS) Volume at End of Treatment (EOT) Based on Dairy Data |
-27.7; -26.7 | — |
| PRIMARY Absolute Change From Baseline in Parenteral Support (PS) Caloric Intake at End of Treatment (EOT) Based on Dairy Data |
-7.2; -13.8 | — |
| PRIMARY Percent Change From Baseline in Parenteral Support (PS) Caloric Intake at End of Treatment (EOT) Based on Dairy Data |
-26.2; -25.7 | — |
| PRIMARY Absolute Change From Baseline in Plasma Citrulline at End of Treatment (EOT) |
3.2 | — |
| PRIMARY Percent Change From Baseline in Plasma Citrulline at End of Treatment (EOT) |
39.2 | — |
| PRIMARY Absolute Change From Baseline in Enteral Nutritional (EN) Volume at End of Treatment (EOT) Based on Dairy Data |
1.0; 0.6 | — |
| PRIMARY Percent Change From Baseline in Enteral Nutritional (EN) Volume at End of Treatment (EOT) Based on Dairy Data |
53.1; -23.2 | — |
| PRIMARY Absolute Change From Baseline in Enteral Nutritional (EN) Caloric Intake at End of Treatment (EOT) Based on Dairy Data |
0.8; 0.3 | — |
| PRIMARY Percent Change From Baseline in Enteral Nutritional (EN) Caloric Intake at End of Treatment (EOT) Based on Dairy Data |
56.8; -24.2 | — |
| PRIMARY Number of Participants Who Achieved At Least 20 Percent (%) Reduction in Parenteral Support (PS) Volume at Week 24 |
4; 1 | — |
| PRIMARY Number of Participants Who Achieved At Least 20 Percent (%) Reduction in Parenteral Support (PS) Volume at End of Treatment (EOT) |
4; 1 | — |
| PRIMARY Number of Participants Who Achieved 100 Percent (%) Reduction in Complete Weaning of Parenteral Support (PS) Volume at End of Treatment (EOT) |
1; 0 | — |
| PRIMARY Number of Participants Who Achieved Greater Than or Equal to (>=) 20 Percent (%) Reduction in Parenteral Support (PS) Volume at Week 28 |
3; 1 | — |
| PRIMARY Absolute Change From End of Treatment (EOT) in Parenteral Support (PS) Volume at End of Study (EOS) Based on Dairy Data |
3.9; -3.8 | — |
| PRIMARY Percent Change From End of Treatment (EOT) in Parenteral Support (PS) Volume at End of Study (EOS) Based on Dairy Data |
9.8; -5.5 | — |
| PRIMARY Absolute Change From End of Treatment (EOT) in Parenteral Support (PS) Caloric Intake at End of Study (EOS) Based on Dairy Data |
0.5; -4.0 | — |
| PRIMARY Percent Change From End of Treatment (EOT) in Parenteral Support (PS) Caloric Intake at End of Study (EOS) Based on Dairy Data |
3.5; -10.3 | — |
| PRIMARY Absolute Change From End of Treatment (EOT) in Plasma Citrulline at End of Study (EOS) |
-2.9 | — |
| PRIMARY Percent Change From End of Treatment (EOT) in Plasma Citrulline at End of Study (EOS) |
-20.0 | — |
| PRIMARY Absolute Change From End of Treatment (EOT) in Enteral Nutritional (EN) Volume at End of Study (EOS) Based on Dairy Data |
-1.5; -2.8 | — |
| PRIMARY Percent Change From End of Treatment (EOT) in Enteral Nutritional (EN) Volume at End of Study (EOS) Based on Dairy Data |
-22.1; -1.4 | — |
| PRIMARY Absolute Change From End of Treatment (EOT) in Enteral Nutritional (EN) Caloric Intake at End of Study (EOS) Based on Dairy Data |
-1.1; -1.4 | — |
| PRIMARY Percent Change From End of Treatment (EOT) in Enteral Nutritional (EN) Caloric Intake at End of Study (EOS) Based on Dairy Data |
-22.1; -3.0 | — |
| PRIMARY Absolute Change From Baseline in Number of Hours Per Day of Parenteral Support (PS) Usage at End of Treatment (EOT) Based on Dairy Data |
-2.1; 0.0 | — |
| PRIMARY Absolute Change From Baseline in Number of Days Per Week of Parenteral Support (PS) Usage at End of Treatment (EOT) Based on Dairy Data |
-0.9; 0.0 | — |
| PRIMARY Number of Participants With Treatment-emergent Adverse Events (TEAEs) |
8; 2 | — |
| PRIMARY Change From Baseline in Body Weight for Age Z-score at Week 28 |
0.177; 2.332 | — |
| PRIMARY Change From Baseline in Height for Age Z-score at Week 28 |
0.024; 1.056 | — |
| PRIMARY Change From Baseline in Head Circumference for Age Z-score at Week 28 |
1.559 | — |
| PRIMARY Number of Participants With Clinically Significant Changes in Vital Signs Reported as Treatment Emergent Adverse Events (TEAEs) |
0; 0 | — |
| PRIMARY Number of Participants With Clinically Significant Changes in Electrocardiogram (ECG) Reported as Treatment Emergent Adverse Events (TEAEs) |
0; 0 | — |
| PRIMARY Number of Participants With Clinically Significant Laboratory Abnormalities Reported as Treatment Emergent Adverse Events (TEAEs) |
0; 0 | — |
| PRIMARY Change From Baseline in the Average Urine Output at Week 28 |
-22.317; -48.879 | — |
| PRIMARY Change From Baseline in the Fecal Output at Week 28 |
-0.64; 2.50 | — |
| PRIMARY Number of Participants With Positive Specific Antibodies to Teduglutide |
1; 2 | — |
| PRIMARY Number of Participants With Clinically Significant Abnormal Findings in Gastrointestinal (GI) Specific Testing |
— | — |
| PRIMARY Area Under the Concentration-time Curve at Steady State (AUCtau,ss) of Teduglutide in Plasma |
— | — |
| PRIMARY Maximum Plasma Concentration at Steady-state (Cmax,ss) of Teduglutide in Plasma |
— | — |
| PRIMARY Minimum Plasma Concentration at Steady-state (Cmin.ss) of Teduglutide in Plasma |
— | — |
| PRIMARY Time to Reach Maximum Observed Drug Concentration (Tmax) of Teduglutide in Plasma |
— | — |
| PRIMARY Terminal-Phase Half-life (t1/2) of Teduglutide in Plasma |
— | — |
| PRIMARY Apparent Clearance (CL/F) of Teduglutide |
— | — |
| PRIMARY Apparent Volume of Distribution (V[Lambda z]/F) of Teduglutide |
— | — |
Summary
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).
Eligibility Criteria
Inclusion Criteria
- Informed consent by a parent or guardian prior to any study-related procedures
- When applicable, informed assent (as deemed appropriate by the Institutional Review Board) by the participant prior to any study-related procedures
- Male or female infant 4 to 1.5 not corrected with parenteral vitamin K
- Platelet count 4 milligram per deciliter (mg/dL) (>68 micromoles per liter [mcmol/L]) over a 2 week period
For children 1 to 15 years of age:
- Total bilirubin >= 2x upper limit of normal (ULN)
- Aspartate aminotransferase (AST) >= 7x ULN
- Alanine aminotransferase (ALT) >= 7x ULN
- Signs of known continuous active or unstable, clinically significant renal dysfunction shown by results of an estimated glomerular filtration rate below 50 milliliter per minute (mL/min)/1.73 meter (m)^2
- Parent(s)/guardian(s) and/or participants 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 participant at any undue risk, prevents completion of the study, or interferes with analysis of the study results.
Data sourced from ClinicalTrials.gov (NCT02980666). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.