Phase 3 N=3 Treatment

A Study of Teduglutide in Japanese Children With Short Bowel Syndrome Aged 4 Months or Older

Short Bowel Syndrome

Bottom Line

View on ClinicalTrials.gov: NCT05027308 ↗

Enrolled (actual)

Serious AEs

100.0%

Results posted

May 2024

Primary outcome: Primary: Number of Participants With Treatment-emergent Adverse Events (TEAEs) — 3 Participants

Study Design & Population

Study type: Interventional
Phase: Phase 3
Interventions: Teduglutide (Drug)
Age: Pediatric, Adult, Older Adult · 0+ yrs
Sex: All
Sponsor: Takeda
Primary completion: Sep 2023

Outcome Measures

Outcome	Result	p-value
PRIMARY Number of Participants With Treatment-emergent Adverse Events (TEAEs)	3	—
PRIMARY Number of Participants With Serious Adverse Events (SAEs)	3	—
PRIMARY Number of Participants With Adverse Events of Special Interest (AESIs)	—	—
PRIMARY Number of Participants With Clinically Significant Abnormalities in Vital Signs Reported as an Adverse Event	—	—
PRIMARY Change From Baseline in Body Weight Z-Score at EOT	1.637	—
PRIMARY Change From Baseline in Height Z-Score at EOT	0.590	—
PRIMARY Change From Baseline in Head Circumference Z-Score at EOT	1.130	—
PRIMARY Change From Baseline in Weight-for-Length Z-Score at EOT	4.240	—
PRIMARY Number of Participants With Any Laboratory Safety Finding Reported as an Adverse Event	1	—
PRIMARY Number of Participants With a Change in Urine Output Reported as an Adverse Event	1	—
PRIMARY Number of Participants With a Change in Stool Output Reported as an Adverse Event	—	—
SECONDARY Change From Baseline in PS Volume	3.13; 7.82; -1.31; 10.27; 5.83; 1.89	—
SECONDARY Percent Change From Baseline in PS Volume	4.82; 11.27; -1.51; 13.48; 6.97; 1.61	—
SECONDARY Number of Participants Who Demonstrate at Least 20 Percent (%) Reduction From Baseline in PS Volume	0; 0; 0; 0; 0; 1	—
SECONDARY Number of Participants Who Achieved Enteral Autonomy	0; 0; 0; 0; 0; 0	—
SECONDARY Change From Baseline in Number of Days Per Week of PS Usage at EOT	0.0; 0.0; 0.0; 0.0; 0.0; 0.0	—

Summary

The main aims of the study are to check for side effects from teduglutide. Participants will receive a daily injection of teduglutide just under the skin (subcutaneous) for 24 weeks. Then they are followed up for another 4 weeks. Participants may be able to repeat this treatment if they meet specific criteria. The study doctors will check for side effects from teduglutide until it becomes commercially available. The maximum duration of treatment is approximately 51.3 weeks.

Eligibility Criteria

Inclusion Criteria

Male or female pediatric patient of corrected gestational age 4 months or older.
Body weight at the time of screening and baseline visits of at least 5 kg and =50 mL/min/1.73 m^2), OR at least 10 kg and 1.5 not corrected with parenteral vitamin K
Platelet count 4 mg/dL (>68 mcr mol/L) over a 2-week period during screening
Total bilirubin >=2x upper limit of normal (ULN)
Aspartate aminotransferase (AST) >=3x ULN
Alanine aminotransferase (ALT) >=3x ULN

View full record on ClinicalTrials.gov →

Data sourced from ClinicalTrials.gov (NCT05027308). 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.