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

Conditions

• Eosinophilic Esophagitis (EoE)

Eligibility Criteria

Sex

ALL

Age

1 Year - 11 Years

Healthy Volunteers

Not accepted

Interventions

• Dupilumab — DRUG
  Single-use, prefilled syringe
• Matching Placebo — DRUG
  Matching formulation and regimen (depending on the weight tier) as dupilumab without the active substance

Study Details

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

Key Dates

Start date

Sep 1, 2020

Status verified

Oct 2025

Primary completion

Jun 2, 2022

Completion

May 14, 2024

Study Design

Enrollment

102 participants (actual)

Allocation

RANDOMIZED

Intervention model

PARALLEL

Primary purpose

TREATMENT

Arms

• Experimental: Part A - High Dose
  Part A consists of a 16-week double-blind treatment period. Patients will be randomized to receive dupilumab or placebo subcutaneous (SC) administration at tiered dosing regimens based on body weight
• Experimental: Part A - Low Dose
  Part A consists of a 16-week double-blind treatment period. Patients will be randomized to receive dupilumab or placebo subcutaneous (SC) administration at tiered dosing regimens based on body weight
• Experimental: Part B - High Dose
  Part B consists of a 36-week extended active treatment period. All patients to receive subcutaneous (SC) administration at tiered dosing regimens based on body weight
• Experimental: Part B - Low Dose
  Part B consists of a 36-week extended active treatment period. All patients to receive subcutaneous (SC) administration at tiered dosing regimens based on body weight
• Experimental: Part C - High Dose
  Part C consists of up to 108-week open-label extension period. All patients will receive higher exposure dupilumab subcutaneous (SC) administration at tiered dosing regimens based on body weight. No matching placebo administered in Part C.

Primary Outcome Measure

Part A: Percentage of Participants Achieving Peak Esophageal Intraepithelial Eosinophil Count of Less Than or Equal to (≤) 6 Eosinophils/High Power Field (Eos/Hpf) at Week 16 [ Time Frame: At Week 16 ]

Locations (26)

Find similar trials in Phoenix, AZ

Related Studies

• Esophageal String Test Monitoring to Monitor Eosinophilic Esophagitis During Oral Immunotherapy
  Recruiting · Children's Hospital of Philadelphia · Philadelphia, Pennsylvania
• Effect of Dupilumab on the Muscle Function of the Esophagus (food Pipe) in Participants with Eosinophilic Esophagitis (EoE)
  PHASE4 · Recruiting · University of California, San Diego · La Jolla, California
• A U.S. Registry of Eosinophilic Esophagitis Pediatric, Adolescent and Adult Patients Treated With DUPIXENT® As Standard of Care
  Recruiting · Regeneron Pharmaceuticals · Phoenix, Arizona
• A Study of Dupilumab in Small Children With an Allergic Condition of the Esophagus (Food Pipe): Eosinophilic Esophagitis
  PHASE3 · Recruiting · Regeneron Pharmaceuticals · Phoenix, Arizona