Dupilumab and Milk OIT for the Treatment of Cow's Milk Allergy

NCT04148352 | Terminated Phase 2 Results DMC FDA Drug

• 1 other identifier: IRB-52976
• Study Type: interventional
• Target: 33
• Locations: 1 country
• Sites: 3

Brief Summary

This is a phase 2, multicenter, randomized, double-blind, parallel group, 2 arm study in approximately 40 subjects aged 4 to 50 years, inclusive, who are allergic to cow's milk. The primary objective is to assess whether dupilumab as an adjunct to milk oral immunotherapy (OIT) compared to placebo improves the safety of milk OIT and rates of desensitization, defined as an increase in the proportion of subjects who pass a double-blind placebo-controlled food challenge (DBPCFC) to at least 2040 mg cumulative milk protein at week 18.

Conditions

Allergies Food Milk

Keywords

Food allergy; Milk allergy; Oral Immunotherapy

Outcome Measures

Primary Outcomes (1)

• Proportion of Participants Treated With Dupilumab Plus Milk Protein OIT vs Placebo Plus Milk Protein OIT Who Tolerate at Least 2040 mg (Cumulative) Cow's Milk Protein During DBPCFC to Milk at Week 18

  DBPCFC double-blind, placebo-controlled food challenge; OIT oral immunotherapy; withdrawals are imputed as failures

  Week 18

Secondary Outcomes (7)

• Proportion of Participants Who Tolerate at Least 1040 mg Cumulative Milk Protein at Week 18 DBPCFC

  Week 18

• Proportion of Participants Who Tolerate 4040 mg Cumulative Milk Protein at Week 18 DBPCFC

  Week 18

• Proportion of Participants Who Tolerate at Least 1040 mg Cumulative Milk Protein at Week 24 DBPCFC

  week 24

• Proportion of Participants Who Tolerate at Least 2040 mg Cumulative Milk Protein at Week 24 DBPCFC

  Week 24

• Proportion of Participants Who Tolerate 4040 mg Cumulative Milk Protein at Week 24 DBPCFC

  Week 24

Study Arms (2)

Dupilumab

EXPERIMENTAL

24-week treatment period, which includes a 4-week run-in period with dupilumab followed by 12 weeks of treatment with dupilumab in combination with a gradual up-dosing of milk protein OIT, then followed by 8 weeks of milk OIT dosing with no dupilumab

Drug: Dupilumab

Placebo

PLACEBO COMPARATOR

24-week treatment period, which includes a 4-week run-in period with placebo for dupilumab followed by 12 weeks of treatment with placebo for dupilumab in combination with a gradual up-dosing of milk protein OIT, then followed by 8 weeks of milk OIT dosing with no placebo

Other: Placebo

Interventions

Dupilumab DRUG

Dupilumab injected every 2 weeks for 18 weeks

Dupilumab

Placebo OTHER

Placebo injected every 2 weeks for 18 weeks

Placebo

Eligibility Criteria

• Age: 4 Years - 50 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

• Age 4 to 50 years (inclusive)
• Clinical history of allergy to cow's milk or milk-containing foods
• Serum IgE to milk of \>4 kUA/L within the last 12 months and/or a SPT to milk ≥6 mm compared to a negative control
• Experience clinical reaction at or before 444 mg cumulative protein dose of cow's milk protein on Screening DBPCFC
• No clinical reaction observed during the placebo (oat) Screening DBPCFC
• Subjects with other known food allergies must agree to eliminate these other food items from their diet so as not to confound the safety and efficacy data from the study
• Use of effective birth control by female participants of childbearing potential

You may not qualify if:

• Any previous exposure to dupilumab
• Known hypersensitivity to dupilumab or any of its excipients
• Known hypersensitivity to epinephrine or any of its excipients
• Allergy to oat (placebo in DBPCFC)
• History of severe anaphylaxis to cow's milk, defined as neurological compromise or requiring intubation
• Recent history of frequent severe, life-threatening episodes of anaphylaxis or anaphylactic shock as defined as 3 or more episodes of anaphylaxis within the past year
• Inability to tolerate biological (antibody) therapies
• Body weight \<5 kg at the time of screening
• History of eosinophilic esophagitis (EoE), other eosinophilic gastrointestinal disease, chronic, recurrent, or severe gastroesophageal reflux disease (GERD), symptoms of dysphagia (e.g., difficulty swallowing, food "getting stuck"), or recurrent gastrointestinal symptoms of undiagnosed etiology
• History of cardiovascular disease, including uncontrolled or inadequately controlled hypertension
• History of a mast cell disorder
• Established diagnosis of a primary immunodeficiency disorder
• Severe asthma or mild or moderate asthma if uncontrolled or difficult to control
• Current participation or within the last 4 months in any other interventional study
• Use of medication such as beta-blockers (oral), angiotensin-converting enzyme (ACE) inhibitors, angiotensin-receptor blockers (ARB) or calcium channel blockers

Sponsors & Collaborators

• Andrew J Long, PharmD: lead
• Robert Levin Charitable Fund: collaborator
• Regeneron Pharmaceuticals: collaborator

Study Sites (3)

Phoenix Children's Hospital

Phoenix, Arizona, 85016, United States

Location

Mayo Clinic

Scottsdale, Arizona, 85259, United States

Location

Sean N. Parker Center for Allergy & Asthma Research at Stanford University

Palo Alto, California, 94304, United States

Location

Related Publications (1)

• Ghelli C, Costanzo G, Canonica GW, Heffler E, Paoletti G. New evidence in food allergies treatment. Curr Opin Allergy Clin Immunol. 2024 Aug 1;24(4):251-256. doi: 10.1097/ACI.0000000000000999. Epub 2024 May 30.

  PMID: 38814736 DERIVED

MeSH Terms

Conditions

Milk Hypersensitivity; Food Hypersensitivity

Interventions

dupilumab

Condition Hierarchy (Ancestors)

Hypersensitivity, Immediate; Hypersensitivity; Immune System Diseases

Limitations and Caveats

Study findings are limited by the small sample size and high attrition. Screen failures and early loss to follow-up reduced power and may limit the ability to draw definitive conclusions about the intervention's effect.

Results Point of Contact

• Title: Sayantani B. Sindher, MD
• Organization: Sean N Parker Center for Allergy & Asthma Research at Stanford University

snpcenterallergy_inquiry@stanford.edu

650-521-7237

Study Officials

• Andrew Long, PharmD

  Stanford, Sean N. Parker Center for Allergy & Asthma Research

  STUDY DIRECTOR

Publication Agreements

• PI is Sponsor Employee: No
• Restrictive Agreement: No

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: TRIPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: SPONSOR INVESTIGATOR
• PI Title: Pharmacist

Model Details: dupilumab or placebo arm

Study Record Dates

• First Submitted: October 30, 2019
• First Posted: November 1, 2019
• Study Start: October 18, 2021
• Primary Completion: December 17, 2024
• Study Completion: February 25, 2025
• Last Updated: January 7, 2026
• Results First Posted: January 7, 2026

Record last verified: 2025-12

Data Sharing

• IPD Sharing: Will not share

Locations

US (3)