Study to Investigate the Efficacy and Safety of Dupilumab in Pediatric Patients With Active Eosinophilic Esophagitis (EoE)

Summary

The Primary objective is to demonstrate the efficacy of dupilumab treatment compared with placebo in pediatric patients with active eosinophilic esophagitis (EoE) based on histologic improvement meeting validated histologic criteria. The Secondary objectives are: * To demonstrate the efficacy of dupilumab compared to placebo in pediatric patients with active EoE after 16 weeks of treatment as assessed by endoscopic visual measurements of disease activity using the Eosinophilic Esophagitis-Endoscopic Reference Score (EoE-EREFS) and histologic abnormalities as measured by the EoE Histology Scoring System (EoE-HSS) * To evaluate the safety, tolerability, and immunogenicity of dupilumab treatment for up to 16 weeks in pediatric patients with active EoE * To evaluate the effects of dupilumab on transcriptomic signatures associated with EoE and type 2 inflammation * To study the effects of dupilumab on the type 2 inflammation gene expression signature * To evaluate the concentration-time profile of functional dupilumab in serum in this population * To assess efficacy of long-term (up to 160 weeks) dupilumab treatment * To assess the impact of dupilumab treatment on changes in weight and growth during the extended active period and open-label extension period of the study * To assess safety, tolerability, and immunogenicity of long-term (up to 160 weeks) dupilumab treatment * To evaluate the impact of dupilumab treatment on EoE signs and symptoms

Timeline

Start

2020-09-01

Primary completion

2022-06-02

Completion

2024-05-14

Publications

• Lim WK, Wipperman MF, Rothenberg ME, Collins MH, Hamon SC, Chehade M, Spergel JM, Gayvert KM, Ehmann PJ, Horowitz JE, Farrell AB, Dellon ES, Maloney J, Radin A, Louisias M, Wolfe K, Akinlade B, Ajithdoss DK, Herman G, Yancopoulos GD, Hamilton JD. Dupilumab normalizes the eosinophilic esophagitis disease transcriptome. J Allergy Clin Immunol. 2026 Apr 22:S0091-6749(26)00263-0. doi: 10.1016/j.jaci.2026.01.033. Online ahead of print.
• Chehade M, McCann E, Spergel J, Yaworsky A, Lamoureux RE, Litcher-Kelly L, Burbridge C, Sutter C, Newton L, Liu R, Tilton ST, Kamat S, Dellon ES. Validation of the Novel Pediatric Eosinophilic Esophagitis Sign/Symptom Questionnaire-Caregiver Version in EoE KIDS. J Allergy Clin Immunol Pract. 2026 Apr;14(4):899-906. doi: 10.1016/j.jaip.2026.01.001. Epub 2026 Jan 12.
• Chehade M, Dellon ES, Spergel JM, Collins MH, Rothenberg ME, Pesek RD, Hirano I, Liu R, Laws E, Mortensen E, Martincova R, Shabbir A, McCann E, Kamal MA, Kosloski MP, Hamilton JD, Samuely C, Lim WK, Wipperman MF, Farrell A, Patel N, Yancopoulos GD, Glotfelty L, Maloney J. Dupilumab for Eosinophilic Esophagitis in Patients 1 to 11 Years of Age. N Engl J Med. 2024 Jun 27;390(24):2239-2251. doi: 10.1056/NEJMoa2312282.

Drugs

Indications