Efficacy of Dupilumab on Facilitated Food Introduction in Eosinophilic Esophagitis

NCT05247866 | Completed Phase 4 Results FDA Drug

• 1 other identifier: 21-019416
• Study Type: interventional
• Target: 21
• Locations: 1 country
• Sites: 1

Brief Summary

Eosinophilic Esophagitis (EoE) is a food driven non-immunoglobulin E (IgE) mediated disease involving eosinophils and type 2 inflammation. Current therapies include diet and the off label use of medications including proton pump inhibitors, topical steroids or biologics. Food elimination creates a decrease quality of life in many children. The goal of the study is to examine a T2 inhibitor (dupilumab) can allow successful reintroduction of allergic EoE foods into the diet. This is a single site study, enrolling subjects 6 to 25 years of age.

Conditions

Eosinophilic Esophagitis

Outcome Measures

Primary Outcomes (3)

• Esophageal Eosinophilia (Number of Eosinophils in the Esophagus)

  Eosinophils per high power field on esophageal biopsy

  week 24

• Esophageal Eosinophilia (Number of Eosinophils in the Esophagus)

  Eosinophils per high power field on esophageal biopsy

  week 48

• Esophageal Eosinophilia (Number of Eosinophils in the Esophagus)

  Eosinophils per high power field on esophageal biopsy

  week 36

Secondary Outcomes (3)

• Pediatric Eosinophilic Esophagitis Symptom Score (Total Score Based on Pediatric Symptom Score)

  week 24

• Pediatric Eosinophilic Esophagitis Symptom Score (Total Score Based on Pediatric Symptom Score)

  week 36

• Pediatric Eosinophilic Esophagitis Symptom Score (Total Score Based on Pediatric Symptom Score)

  week 48

Other Outcomes (12)

• Rate of Maintenance of Remission (Less Than <6 Eos/Hpf) in Esophageal Biopsy

  week 24

• Rate of Maintenance of Remission (Less Than <6 Eos/Hpf) in Esophageal Biopsy

  week 36

• Rate of Maintenance of Remission (Less Than <6 Eos/Hpf) in Esophageal Biopsy

  week 48

Study Arms (1)

Study Group

EXPERIMENTAL

30 patients will be given dupilumab (dose based on current approved doses or used in current EoE clinical trial, q weekly dosing) and monitored for clinical response after 12 weeks of therapy) Dosing: \>12 years of age \> 40 kg 300 SQ weekly 30- 40 kg 300 mg SQ Q2 weeks 15-29.9 kg 200 mg SQ Q2 weeks 6-11 years of age 5-15 kg 100 mg SQ Q2W 15-30 kg 200 mg SQ Q2W \>30-60 kg 300 mg SQ Q2W

Dietary Supplement: Eosinophilic Esophagitis (EoE) food introduction-1st; Dietary Supplement: Eosinophilic Esophagitis (E0E) food introduction-2nd; Dietary Supplement: Eosinophilic Esophagitis (EoE) Food introduction-3rd dose; Drug: Dupilumab

Interventions

Eosinophilic Esophagitis (EoE) food introduction-1st DIETARY_SUPPLEMENT

1 serving size of food proven to induce histologic and clinical symptoms in EoE

Study Group

Eosinophilic Esophagitis (E0E) food introduction-2nd DIETARY_SUPPLEMENT

Either 1 serving size of different food (not food in arm 1) to induce histologic and clinical symptoms in EoE or Increase the food in arm 1 to 2 serving size a day

Study Group

Eosinophilic Esophagitis (EoE) Food introduction-3rd dose DIETARY_SUPPLEMENT

Either 1 serving size of different food (not food in arm 1 or 2) to induce histologic and clinical symptoms in EoE or Increase the food in arm 2 to 2 serving size a day or unlimited amounts of food arm 1

Study Group

Dupilumab DRUG

All patients will receive open label dupilumab at the following doses \>12 years of age \> 40 kg 300 subcutaneous (SQ) weekly 6-11 years of age 5-15 kg 100 mg SQ every 2 weeks (Q2W) 15-30 kg 200 mg SQ Q2W \>30-60 kg 300 mg SQ Q2W

Study Group

Eligibility Criteria

• Age: 6 Years - 25 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

• Males or females age 6 to 25 years
• Diagnosis of Eosinophilic Esophagitis based on the most recent international consensus definition (Dellon et al, Gastroenterology 2019)
• a) History of endoscopy with a peak count of \>15 eosinophils per high powered field meeting consensus criteria for Eosinophilic Esophagitis1
• History of either milk, egg, soy or wheat induced EoE based on the following criteria in the last two years
• Addition of a single food lead to exacerbation of esophageal eosinophilia (increase of greater than 15 eos/hpf) or
• Removal of a single food lead to normalization of biopsy (esophageal eosinophilia showed less than 6 eos/hpf) AND
• History of either milk, egg, soy or wheat induced EoE based on introduction of the food and symptoms in the last 12 months
• Weight \> 10 kg
• Ability to remain on stable dose of Proton Pump Inhibitor (PPI) therapy throughout the study
• Girls \> 11 years of age must have a negative urine/serum pregnancy test.
• Parental/guardian permission (informed consent) and if appropriate, child assent.

You may not qualify if:

• Tracheo-esophageal fistulas, inflammatory bowel disease, Barrett's disease, or other significant inflammatory disease of the gastrointestinal tract
• Biopsy evidence of eosinophilic infiltration in any other organ system
• History of significant esophageal procedures e.g. sclerotherapy or esophagectomy
• Systemic immunosuppressant usage in prior 3 months
• Narrow caliber esophagus defined as the inability to pass a 9.5 mm endoscopy into the esophagus
• IgE mediated reaction to food (milk, egg, soy or wheat) being introduced in the last 12 months
• Therapy with biologic molecule (e.g. omalizumab, infliximab) in prior 12 months
• Any factors that may pose a significant risk for undergoing anesthesia/sedation
• Subjects undergoing any type of immunotherapy to any food (oral immunotherapy, sublingual immunotherapy, specific oral tolerance induction) within 3 months prior to Visit 1.
• Active IgE- mediated milk, egg, wheat or soy allergy based on skin test or history (if those foods are being introduced back into the diet).
• Allergy or known hypersensitivity to the dupilumab.
• Subjects (or parents of subjects) with obvious excessive anxiety and unlikely to cope with the conditions of an upper Endoscopy and biopsy.
• Past or current disease(s), which in the opinion of the Investigator or the Sponsor, may affect the subject's participation in this study, including but not limited to active autoimmune disorders, immunodeficiency, malignancy, uncontrolled diseases (hypertension, psychiatric (especially anxiety), cardiac), or other disorders (e.g., liver, gastrointestinal, kidney, cardiovascular, pulmonary disease, or blood disorders).
• Participation in another clinical intervention study in the three months prior to Visit 1.
• Subjects unable to follow the protocol and the protocol requirements.

Sponsors & Collaborators

• Children's Hospital of Philadelphia: lead
• Regeneron Pharmaceuticals: collaborator
• Genzyme, a Sanofi Company: collaborator

Study Sites (1)

Children's Hospital of Philadelphia

Philadelphia, Pennsylvania, 19104, United States

Location

Related Publications (6)

• Rothenberg ME, Dellon ES, Collins MH, Hirano I, Chehade M, Bredenoord AJ, Lucendo AJ, Spergel JM, Sun X, Hamilton JD, Mortensen E, Laws E, Maloney J, Mannent LP, McCann E, Liu X, Glotfelty L, Shabbir A. Efficacy and safety of dupilumab up to 52 weeks in adults and adolescents with eosinophilic oesophagitis (LIBERTY EoE TREET study): a multicentre, double-blind, randomised, placebo-controlled, phase 3 trial. Lancet Gastroenterol Hepatol. 2023 Nov;8(11):990-1004. doi: 10.1016/S2468-1253(23)00204-2. Epub 2023 Aug 31.

  PMID: 37660704 BACKGROUND

• McCann E, Chehade M, Spergel JM, Yaworsky A, Symonds T, Stokes J, Tilton ST, Sun X, Kamat S. Validation of the novel Eosinophilic Esophagitis Impact Questionnaire. J Patient Rep Outcomes. 2023 Nov 27;7(1):120. doi: 10.1186/s41687-023-00654-z.

  PMID: 38010430 BACKGROUND

• Dellon ES, Rothenberg ME, Collins MH, Hirano I, Chehade M, Bredenoord AJ, Lucendo AJ, Spergel JM, Aceves S, Sun X, Kosloski MP, Kamal MA, Hamilton JD, Beazley B, McCann E, Patel K, Mannent LP, Laws E, Akinlade B, Amin N, Lim WK, Wipperman MF, Ruddy M, Patel N, Weinreich DR, Yancopoulos GD, Shumel B, Maloney J, Giannelou A, Shabbir A. Dupilumab in Adults and Adolescents with Eosinophilic Esophagitis. N Engl J Med. 2022 Dec 22;387(25):2317-2330. doi: 10.1056/NEJMoa2205982.

  PMID: 36546624 BACKGROUND

• Spergel BL, Ruffner MA, Godwin BC, Liacouras CA, Cianferoni A, Gober L, Hill DA, Brown-Whitehorn TF, Chaiboonma K, Aceves SA, Muir AM, Spergel JM. Improvement in eosinophilic esophagitis when using dupilumab for other indications or compassionate use. Ann Allergy Asthma Immunol. 2022 May;128(5):589-593. doi: 10.1016/j.anai.2022.01.019. Epub 2022 Jan 25.

  PMID: 35085819 BACKGROUND

• Dellon ES, Liacouras CA, Molina-Infante J, Furuta GT, Spergel JM, Zevit N, Spechler SJ, Attwood SE, Straumann A, Aceves SS, Alexander JA, Atkins D, Arva NC, Blanchard C, Bonis PA, Book WM, Capocelli KE, Chehade M, Cheng E, Collins MH, Davis CM, Dias JA, Di Lorenzo C, Dohil R, Dupont C, Falk GW, Ferreira CT, Fox A, Gonsalves NP, Gupta SK, Katzka DA, Kinoshita Y, Menard-Katcher C, Kodroff E, Metz DC, Miehlke S, Muir AB, Mukkada VA, Murch S, Nurko S, Ohtsuka Y, Orel R, Papadopoulou A, Peterson KA, Philpott H, Putnam PE, Richter JE, Rosen R, Rothenberg ME, Schoepfer A, Scott MM, Shah N, Sheikh J, Souza RF, Strobel MJ, Talley NJ, Vaezi MF, Vandenplas Y, Vieira MC, Walker MM, Wechsler JB, Wershil BK, Wen T, Yang GY, Hirano I, Bredenoord AJ. Updated International Consensus Diagnostic Criteria for Eosinophilic Esophagitis: Proceedings of the AGREE Conference. Gastroenterology. 2018 Oct;155(4):1022-1033.e10. doi: 10.1053/j.gastro.2018.07.009. Epub 2018 Sep 6.

  PMID: 30009819 BACKGROUND

• Wolfset N, Muir AB, Benitez AJ, Williams D, De La Torre I, Ruffner MA, Hill DA, Cazeau C, Angello JT, Radwan A, Durrani S, deMarchi S, Spergel JM. Efficacy of Dupilumab on Facilitated Food Reintroduction in Eosinophilic Esophagitis. Clin Gastroenterol Hepatol. 2025 Sep 30:S1542-3565(25)00745-1. doi: 10.1016/j.cgh.2025.08.025. Online ahead of print.

  PMID: 41038415 DERIVED

MeSH Terms

Conditions

Eosinophilic Esophagitis

Interventions

dupilumab

Condition Hierarchy (Ancestors)

Esophagitis; Esophageal Diseases; Gastrointestinal Diseases; Digestive System Diseases; Gastroenteritis; Eosinophilia; Leukocyte Disorders; Hematologic Diseases; Hemic and Lymphatic Diseases; Hypersensitivity, Immediate; Hypersensitivity; Immune System Diseases

Limitations and Caveats

The study was only completed in pediatrics. If is unclear if the results will extend to adults.

Results Point of Contact

• Title: Jonathan Spergel
• Organization: Children's Hospital of Philadelphia

spergel@chop.edu

2155902549

Study Officials

• Jonathan Spergel, MD, PhD

  Children's Hospital of Philadelphia

  PRINCIPAL INVESTIGATOR

Publication Agreements

• PI is Sponsor Employee: No
• Restriction Type: LTE60
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 4
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Model Details: All patients receive active therapy

Study Record Dates

• First Submitted: December 21, 2021
• First Posted: February 21, 2022
• Study Start: June 16, 2022
• Primary Completion: January 21, 2025
• Study Completion: January 21, 2025
• Last Updated: November 10, 2025
• Results First Posted: November 10, 2025

Record last verified: 2025-10

Data Sharing

• IPD Sharing: Will share
• Shared Documents: STUDY PROTOCOL
• Time Frame: The data will be released after the data is published.

Locations

US (1)