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Outcomes from a 12-Week, Open-Label, Multicenter Clinical Trial of Teduglutide in Pediatric Short Bowel Syndrome
Beth A Carter1, Valeria C Cohran2, Conrad R Cole3
1Department of Pediatric Medicine, Gastroenterology, Hepatology and Nutrition, Texas Children's Hospital and Baylor College of Medicine, Houston, TX.
Insights
Teduglutide demonstrated good tolerability in children with short bowel syndrome-intestinal failure (SBS-IF). Higher doses showed promising reductions in parenteral nutrition and increased enteral feeding, with some patients achieving independence from PN.
Area of Science:
- Pediatric Gastroenterology
- Clinical Pharmacology
- Parenteral Nutrition
Background:
- Short bowel syndrome with intestinal failure (SBS-IF) significantly impacts pediatric patients' quality of life and nutritional status.
- Parenteral nutrition (PN) is a critical intervention but carries risks and burdens.
- Exploring novel therapeutic agents to improve outcomes in SBS-IF is essential.
Purpose of the Study:
- To evaluate the safety and efficacy of teduglutide in pediatric patients with SBS-IF.
- To assess the pharmacodynamics of teduglutide in this population.
- To determine optimal dosing for potential therapeutic benefits.
Main Methods:
- An open-label, 12-week study involving pediatric patients (1-17 years) with SBS-IF requiring PN.
- Sequential enrollment into three teduglutide dose cohorts (0.0125, 0.025, 0.05 mg/kg/d) or standard of care (SOC).
- Evaluation of treatment-emergent adverse events, PN volume, PN calories, and enteral nutrition (EN) volume.
Main Results:
- Teduglutide was generally well-tolerated, with most adverse events being mild to moderate.
- Significant reductions in PN volume (up to -45%) and calories (up to -52%) were observed with higher teduglutide doses (0.025 and 0.05 mg/kg/d).
- Median increases in EN volume ranged from 22% to 40% across teduglutide cohorts, compared to 11% with SOC. Four patients achieved PN independence.
Conclusions:
- Teduglutide is a well-tolerated treatment option for pediatric patients with SBS-IF.
- Doses of 0.025 or 0.05 mg/kg/d teduglutide showed encouraging trends in reducing PN dependency and enhancing EN intake.
- Further research with larger sample sizes and longer durations is warranted to confirm these findings.
Objective:
To determine safety and pharmacodynamics/efficacy of teduglutide in children with intestinal failure associated with short bowel syndrome (SBS-IF).
Study Design:
This 12-week, open-label study enrolled patients aged 1-17 years with SBS-IF who required parenteral nutrition (PN) and showed minimal or no advance in enteral nutrition (EN) feeds. Patients enrolled sequentially into 3 teduglutide cohorts (0.0125 mg/kg/d [n = 8], 0.025 mg/kg/d [n = 14], 0.05 mg/kg/d [n = 15]) or received standard of care (SOC, n = 5). Descriptive summary statistics were used.
Results:
All patients experienced ≥1 treatment-emergent adverse event; most were mild or moderate. No serious teduglutide-related treatment-emergent adverse events occurred. Between baseline and week 12, prescribed PN volume and calories (kcal/kg/d) changed by a median of -41% and -45%, respectively, with 0.025 mg/kg/d teduglutide and by -25% and -52% with 0.05 mg/kg/d teduglutide. In contrast, PN volume and calories changed by 0% and -6%, respectively, with 0.0125 mg/kg/d teduglutide and by 0% and -1% with SOC. Per patient diary data, EN volume increased by a median of 22%, 32%, and 40% in the 0.0125, 0.025, and 0.05 mg/kg/d cohorts, respectively, and by 11% with SOC. Four patients achieved independence from PN, 3 in the 0.05 mg/kg/d cohort and 1 in the 0.025 mg/kg/d cohort. Study limitations included its short-term, open-label design, and small sample size.
Conclusions:
Teduglutide was well tolerated in pediatric patients with SBS-IF. Teduglutide 0.025 or 0.05 mg/kg/d was associated with trends toward reductions in PN requirements and advancements in EN feeding in children with SBS-IF.
Trial Registration:
ClinicalTrials.gov:NCT01952080; EudraCT: 2013-004588-30.
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