Pediatrics international : official journal of the Japan Pediatric Society · 2026
Teduglutide in pediatric patients under 10 kg with short bowel syndrome on parenteral support: An open-label study.
Клиническое исследование III фазыСтатьяМногоцентровое исследованиеНазвание и аннотация: Перевод в очереди
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Masumoto Kouji, Muto Mitsuru, Sasaki Takato, Shikamura Mitsuhiro, Tanaka Tomoko, Sakui Sho, Miyamoto Masakazu, Nakano Hiroya, Kondo Taisuke, Ieiri Satoshi
Аннотация
BACKGROUND: Patients with short bowel syndrome (SBS)-associated intestinal failure (SBS-IF) are dependent on parenteral support (PS). In Japan, teduglutide is the only GLP-2 analog medicine indicated for these patients. There are limited data for pediatric patients, especially those with a low body weight. This study evaluated the safety and efficacy of teduglutide in Japanese pediatric patients with SBS-IF (dependence on PS to provide ≥30% of fluid or caloric intake needs) who weighed less than 10 kg.
METHODS: This phase 3, open-label study enrolled Japanese pediatric patients with SBS-IF. Patients weighing less than 10 kg received teduglutide 0.05 mg/kg/day subcutaneously in 28-week treatment cycles (24 weeks of teduglutide treatment and 4 weeks of follow-up). Adverse events, changes in PS requirements, and growth parameters were assessed.
RESULTS: Three patients completed the study, with a mean teduglutide exposure duration of 48.9 weeks. All patients experienced treatment-emergent adverse events (TEAEs), but none were related to the study drug or led to death or treatment discontinuation. Clinically meaningful reductions in mean PS volume (13.1%) and mean PS caloric intake (46.6%) were observed at the end of treatment from baseline. One patient achieved a reduction in PS volume of ≥20% at the end of treatment from baseline. Growth parameters showed increases in weight and height/length-for-age z-scores at the end of treatment from baseline.
CONCLUSION: In the three patients with SBS-IF who weighed less than 10 kg, no new safety signals were observed following teduglutide treatment. Clinically meaningful reductions in PS were noted and there was no adverse impact on growth parameters.
TRIAL REGISTRATION: ClinicalTrials.gov registration number: NCT05027308.
METHODS: This phase 3, open-label study enrolled Japanese pediatric patients with SBS-IF. Patients weighing less than 10 kg received teduglutide 0.05 mg/kg/day subcutaneously in 28-week treatment cycles (24 weeks of teduglutide treatment and 4 weeks of follow-up). Adverse events, changes in PS requirements, and growth parameters were assessed.
RESULTS: Three patients completed the study, with a mean teduglutide exposure duration of 48.9 weeks. All patients experienced treatment-emergent adverse events (TEAEs), but none were related to the study drug or led to death or treatment discontinuation. Clinically meaningful reductions in mean PS volume (13.1%) and mean PS caloric intake (46.6%) were observed at the end of treatment from baseline. One patient achieved a reduction in PS volume of ≥20% at the end of treatment from baseline. Growth parameters showed increases in weight and height/length-for-age z-scores at the end of treatment from baseline.
CONCLUSION: In the three patients with SBS-IF who weighed less than 10 kg, no new safety signals were observed following teduglutide treatment. Clinically meaningful reductions in PS were noted and there was no adverse impact on growth parameters.
TRIAL REGISTRATION: ClinicalTrials.gov registration number: NCT05027308.