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

NCT ID: NCT04394351

Last Updated: 2025-10-27

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: PHASE3
• Total Enrollment: 102 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2020-09-01
• Study Completion Date: 2024-05-14

Brief 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

Detailed Description

This is a 3-part study:

• Part A: Double-blind 16-week treatment period
• Part B: 36-week extended active treatment period
• Part C: Up to108 weeks open-label extension period

Conditions

Eosinophilic Esophagitis (EoE)

Study Design

• Allocation Method: RANDOMIZED
• Intervention Model: PARALLEL
• Primary Study Purpose: TREATMENT
• Blinding Strategy: QUADRUPLE

Study Groups

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

Group Type: EXPERIMENTAL

Dupilumab

Intervention Type: DRUG

Single-use, prefilled syringe

Matching Placebo

Intervention Type: DRUG

Matching formulation and regimen (depending on the weight tier) as dupilumab without the active substance

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

Group Type: EXPERIMENTAL

Dupilumab

Intervention Type: DRUG

Single-use, prefilled syringe

Matching Placebo

Intervention Type: DRUG

Matching formulation and regimen (depending on the weight tier) as dupilumab without the active substance

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

Group Type: EXPERIMENTAL

Dupilumab

Intervention Type: DRUG

Single-use, prefilled syringe

Matching Placebo

Intervention Type: DRUG

Matching formulation and regimen (depending on the weight tier) as dupilumab without the active substance

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

Group Type: EXPERIMENTAL

Dupilumab

Intervention Type: DRUG

Single-use, prefilled syringe

Matching Placebo

Intervention Type: DRUG

Matching formulation and regimen (depending on the weight tier) as dupilumab without the active substance

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.

Group Type: EXPERIMENTAL

Dupilumab

Intervention Type: DRUG

Single-use, prefilled syringe

Interventions

Dupilumab

Single-use, prefilled syringe

Intervention Type: DRUG

Matching Placebo

Matching formulation and regimen (depending on the weight tier) as dupilumab without the active substance

Intervention Type: DRUG

Other Intervention Names

•DUPIXENT; •REGN668; •SAR231893

Eligibility Criteria

Inclusion Criteria

1. A documented diagnosis of eosinophilic esophagitis (EoE)

2. Baseline endoscopic biopsies with a demonstration on central reading of intraepithelial eosinophilic infiltration

Exclusion Criteria

1. Body weight <5 kg or ≥60 kg at screening

2. Other causes of esophageal eosinophilia

3. Active Helicobacter pylori

4. History of Crohn's disease, ulcerative colitis, celiac disease, or prior esophageal surgery

5. Any esophageal stricture unable to be passed with a standard, diagnostic, upper endoscope or any critical esophageal stricture that requires dilation at screening

6. Treatment with swallowed topical corticosteroids within 8 weeks prior to baseline standard of care endoscopy

7. History of bleeding disorders or esophageal varices that, in the opinion of the investigator, would put the patient at undue risk for significant complications from an endoscopy procedure

8. Active parasitic infection or suspected parasitic infection

9. Known or suspected immunodeficiency disorder

Key Exclusion for Patients Re-Entering the Study (for Entry into Part C, as defined in protocol):

1. Patients who are ≥12 years old, weigh ≥40 kg (or minimum weight for which dupilumab is approved for EoE), and dupilumab is commercially available for the treatment of EoE in their country

2. Patients who, during their previous participation in this clinical trial, developed an SAE and/or AE deemed related to dupilumab, which in the opinion of the investigator or of the medical monitor could indicate that continued treatment with dupilumab may present an unreasonable risk for the patient

3. Patients who did not undergo endoscopy with biopsies at week 16 and/or week 52 or prior to receiving rescue treatment Note: If the endoscopy with biopsies could not occur due to COVID-19 restrictions and rescue treatment was needed to be initiated without delay, these patients will be eligible to participate in Part C

4. Patients who became pregnant during their previous participation in this dupilumab clinical trial

5. Patients who, during their previous participation in this trial, were prematurely withdrawn because of a protocol violation, poor compliance, or inability to complete required study assessments

• Minimum Eligible Age: 1 Year
• Maximum Eligible Age: 11 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Sanofi

INDUSTRY

Sponsor Role: collaborator

Regeneron Pharmaceuticals

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Clinical Trial Management

Role: STUDY_DIRECTOR

Regeneron Pharmaceuticals

Locations

Regeneron Study Site

Phoenix, Arizona, United States

Regeneron Study Site

Little Rock, Arkansas, United States

Regeneron Study Site

Los Angeles, California, United States

Regeneron Study Site

San Francisco, California, United States

Regeneron Study Site

Aurora, Colorado, United States

Regeneron Study Site

St. Petersburg, Florida, United States

Regeneron Study Site

Atlanta, Georgia, United States

Regeneron Study Site

Atlanta, Georgia, United States

Regeneron Study Site

Chicago, Illinois, United States

Regeneron Study Site

Indianapolis, Indiana, United States

Regeneron Study Site

Iowa City, Iowa, United States

Regeneron Study Site

Boston, Massachusetts, United States

Regeneron Study Site

Boston, Massachusetts, United States

Regeneron Study Site

Boston, Massachusetts, United States

Regeneron Study Site

Lincoln, Nebraska, United States

Regeneron Study Site

New York, New York, United States

Regeneron Study Site

New York, New York, United States

Regeneron Study Site

New York, New York, United States

Regeneron Study Site

Chapel Hill, North Carolina, United States

Regeneron Study Site

Cincinnati, Ohio, United States

Regeneron Study Site

Cleveland, Ohio, United States

Regeneron Study Site

Philadelphia, Pennsylvania, United States

Regeneron Study Site

Dallas, Texas, United States

Regeneron Study Site

Fort Worth, Texas, United States

Regeneron Study Site

Houston, Texas, United States

Regeneron Study Site

Milwaukee, Wisconsin, United States

Regeneron Study Site

London, Ontario, Canada

Countries

United States; Canada

References

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.

Reference Type: DERIVED

PMID: 38924731 (View on PubMed)

Provided Documents

Document Type: Study Protocol

View Document

Document Type: Statistical Analysis Plan

View Document

Other Identifiers

2019-003078-24

Identifier Type: EUDRACT_NUMBER

Identifier Source: secondary_id

R668-EE-1877

Identifier Type: -

Identifier Source: org_study_id