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TerminatedNCT04148352Updated Jan 7, 2026Results posted

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

A Phase 2 interventional study of Dupilumab and Placebo in Allergies Food Milk, sponsored by Andrew J Long, PharmD. Terminated at 3 sites in United States. Open to participants aged 4 Years to 50 Years. Per ClinicalTrials.gov, last updated 2026-01-07.

Sponsored by Andrew J Long, PharmD · Phase 2, Interventional, and Treatment

Why this study was terminated
Resources not available to continue study
Phase
Phase 2
Study type
Interventional
Enrollment
33
Allocation
Randomized
Ages
4 Years to 50 Years
Sex
All
01

Study 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.

02

Conditions studied

  • Allergies Food Milk

Keywords

  • Food allergy
  • Milk allergy
  • Oral Immunotherapy
03

Who can participate

Ages eligible
4 Years to 50 Years
Sexes eligible
All
Accepts healthy volunteers
No

Inclusion criteria

  • 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

Exclusion criteria

Exclusion Criteria:

  • 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
  • Pregnant or breastfeeding women
04

Study design

Phase
Phase 2
Primary purpose
Treatment
Allocation
Randomized
Intervention model
Parallel assignment
Masking
Triple (Participant, Care provider, Investigator)
Enrollment
33 participants (actual)

Study arms

  • Experimental
    Dupilumab

    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 comparator
    Placebo

    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

  • DrugDupilumab

    Dupilumab injected every 2 weeks for 18 weeks

  • OtherPlacebo

    Placebo injected every 2 weeks for 18 weeks

05

What researchers measure

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

    Time frame: Week 18

Secondary outcomes

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

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

    Time frame: Week 18

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

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

    Time frame: Week 18

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

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

    Time frame: week 24

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

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

    Time frame: Week 24

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

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

    Time frame: Week 24

  6. Change in the Cumulative Tolerated Dose (CTD) of Milk Protein During DBPCFC From Baseline to Week 18 Across Cohorts as Pairwise Comparisons of All Treatment Groups

    Baseline values reflect the log10 of cumulative tolerated dose (CTD) of milk protein. The change metric reflects the order of magnitude of paired differences in CTD between baseline and week 18.

    Time frame: Baseline and week 18

  7. Change in the Cumulative Tolerated Dose (CTD) of Milk Protein During DBPCFC From Baseline to Week 24 Across Cohorts as Pairwise Comparisons of All Treatment Groups

    Baseline values reflect the log10 of cumulative tolerated dose (CTD) of milk protein. The change metric reflects the order of magnitude of paired differences in CTD between baseline and week 24.

    Time frame: Baseline and week 24

06

Results

Posted Jan 7, 2026
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.

Participant flow

Participant flow — Overall Study
MilestoneActive Dupilumab and Milk Oral Immunotherapy (OIT)Placebo for Dupilumab and Milk OIT
Started1112
Week 18 double-blind, placebo-controlled food challenge (dbpcfc)118
Week 24 dbpcfc116
Completed116
Not completed06

Outcome measures

PrimaryProportion 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

Time frame:
Week 18
Reported as:
Count of participants · Participants
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
ParticipantsActive Dupilumab and Milk OITPlacebo for Dupilumab and Milk OIT
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 1885
SecondaryProportion of Participants Who Tolerate at Least 1040 mg Cumulative Milk Protein at Week 18 DBPCFC

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

Time frame:
Week 18
Reported as:
Count of participants · Participants
Proportion of Participants Who Tolerate at Least 1040 mg Cumulative Milk Protein at Week 18 DBPCFC
ParticipantsActive Dupilumab and Milk OITPlacebo for Dupilumab and Milk OIT
Proportion of Participants Who Tolerate at Least 1040 mg Cumulative Milk Protein at Week 18 DBPCFC96
SecondaryProportion of Participants Who Tolerate 4040 mg Cumulative Milk Protein at Week 18 DBPCFC

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

Time frame:
Week 18
Reported as:
Count of participants · Participants
Proportion of Participants Who Tolerate 4040 mg Cumulative Milk Protein at Week 18 DBPCFC
ParticipantsActive Dupilumab and Milk OITPlacebo for Dupilumab and Milk OIT
Proportion of Participants Who Tolerate 4040 mg Cumulative Milk Protein at Week 18 DBPCFC53
SecondaryProportion of Participants Who Tolerate at Least 1040 mg Cumulative Milk Protein at Week 24 DBPCFC

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

Time frame:
week 24
Reported as:
Count of participants · Participants
Proportion of Participants Who Tolerate at Least 1040 mg Cumulative Milk Protein at Week 24 DBPCFC
ParticipantsActive Dupilumab and Milk OITPlacebo for Dupilumab and Milk OIT
Proportion of Participants Who Tolerate at Least 1040 mg Cumulative Milk Protein at Week 24 DBPCFC85
SecondaryProportion of Participants Who Tolerate at Least 2040 mg Cumulative Milk Protein at Week 24 DBPCFC

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

Time frame:
Week 24
Reported as:
Count of participants · Participants
Proportion of Participants Who Tolerate at Least 2040 mg Cumulative Milk Protein at Week 24 DBPCFC
ParticipantsActive Dupilumab and Milk OITPlacebo for Dupilumab and Milk OIT
Proportion of Participants Who Tolerate at Least 2040 mg Cumulative Milk Protein at Week 24 DBPCFC75
SecondaryProportion of Participants Who Tolerate 4040 mg Cumulative Milk Protein at Week 24 DBPCFC

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

Time frame:
Week 24
Reported as:
Count of participants · Participants
Proportion of Participants Who Tolerate 4040 mg Cumulative Milk Protein at Week 24 DBPCFC
ParticipantsActive Dupilumab and Milk OITPlacebo for Dupilumab and Milk OIT
Proportion of Participants Who Tolerate 4040 mg Cumulative Milk Protein at Week 24 DBPCFC53
SecondaryChange in the Cumulative Tolerated Dose (CTD) of Milk Protein During DBPCFC From Baseline to Week 18 Across Cohorts as Pairwise Comparisons of All Treatment Groups

Baseline values reflect the log10 of cumulative tolerated dose (CTD) of milk protein. The change metric reflects the order of magnitude of paired differences in CTD between baseline and week 18.

Time frame:
Baseline and week 18
Reported as:
Mean · log10 mg cumulative tolerated protein
Change in the Cumulative Tolerated Dose (CTD) of Milk Protein During DBPCFC From Baseline to Week 18 Across Cohorts as Pairwise Comparisons of All Treatment Groups
log10 mg cumulative tolerated proteinActive Dupilumab and Milk OITPlacebo for Dupilumab and Milk OIT
Baseline1.632 ± 0.8881.899 ± 0.600
Change at week 181.574 ± 0.8801.127 ± 0.618
SecondaryChange in the Cumulative Tolerated Dose (CTD) of Milk Protein During DBPCFC From Baseline to Week 24 Across Cohorts as Pairwise Comparisons of All Treatment Groups

Baseline values reflect the log10 of cumulative tolerated dose (CTD) of milk protein. The change metric reflects the order of magnitude of paired differences in CTD between baseline and week 24.

Time frame:
Baseline and week 24
Reported as:
Mean · log10 mg cumulative tolerated protein
Change in the Cumulative Tolerated Dose (CTD) of Milk Protein During DBPCFC From Baseline to Week 24 Across Cohorts as Pairwise Comparisons of All Treatment Groups
log10 mg cumulative tolerated proteinActive Dupilumab and Milk OITPlacebo for Dupilumab and Milk OIT
Baseline1.632 ± 0.8881.898 ± 0.691
Change at week 241.559 ± 1.1531.366 ± 0.490

Adverse events

Collected over From enrollment until end of follow-up, up to 24 weeks. Non-serious events are listed at a 0% frequency threshold.

Adverse event summary by group
GroupDeathsSeriousOther
Active Dupilumab and Milk OIT0/11 (0%)0/11 (0%)10/11 (90.9%)
Placebo for Dupilumab and Milk OIT0/12 (0%)0/12 (0%)11/12 (91.7%)
Most frequent other events
Showing 10 of 25
Most frequent other events
EventActive Dupilumab and Milk OITPlacebo for Dupilumab and Milk OIT
Gastrointestinal Allergic AEs related to OITGastrointestinal disorders10/1111/12
Respiratory Allergic AEs related to OITRespiratory, thoracic and mediastinal disorders6/1110/12
Skin Allergic AEs related to OITSkin and subcutaneous tissue disorders8/119/12
Skin Food Challenge Reactions Allergic not related to OITSkin and subcutaneous tissue disorders6/119/12
Gastrointestinal Food Challenge Reactions Allergic not related to OITGastrointestinal disorders7/117/12
Respiratory Food Challenge Reactions Allergic not related to OITRespiratory, thoracic and mediastinal disorders7/117/12
General Allergic AEs related to OITGeneral disorders4/113/12
General/administration site Non-Allergic not related to OITGeneral disorders4/113/12
Respiratory Non-Allergic not related to OITRespiratory, thoracic and mediastinal disorders4/114/12
Infection Non-Allergic not related to OITInfections and infestations3/114/12

Baseline characteristics

Age, Customized
Age, Customized(Participants)Active Dupilumab and Milk OITPlacebo for Dupilumab and Milk OITTotal
4 - 10 years4610
11 - 17 years437
18+ years336
Sex: Female, Male
Sex: Female, Male(Participants)Active Dupilumab and Milk OITPlacebo for Dupilumab and Milk OITTotal
Female325
Male81018
Ethnicity (NIH/OMB)
Ethnicity (NIH/OMB)(Participants)Active Dupilumab and Milk OITPlacebo for Dupilumab and Milk OITTotal
Hispanic or Latino033
Not Hispanic or Latino11920
Unknown or Not Reported000
Race (NIH/OMB)
Race (NIH/OMB)(Participants)Active Dupilumab and Milk OITPlacebo for Dupilumab and Milk OITTotal
American Indian or Alaska Native000
Asian134
Native Hawaiian or Other Pacific Islander000
Black or African American101
White7512
More than one race246
Unknown or Not Reported000
History of Atopy
History of Atopy(Participants)Active Dupilumab and Milk OITPlacebo for Dupilumab and Milk OITTotal
Asthma7815
Allergic Rhinitis6612
Atopic Disease9716
07

Study locations

3 sites
  • Phoenix Children's Hospital
    Phoenix, Arizona 85016, United States
  • Mayo Clinic
    Scottsdale, Arizona 85259, United States
  • Sean N. Parker Center for Allergy & Asthma Research at Stanford University
    Palo Alto, California 94304, United States
08

References and documents

Publications

  • 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. PubMed 38814736 ↗

Study documents

  • Protocol and statistical analysis plan · Oct 22, 2024

Documents are hosted by the registry — open the source record to download them.

Individual participant data

Plan to share: No

09

Registry details

Key details

Study ID
NCT04148352
Lead sponsor
Andrew J Long, PharmD
Collaborators
Robert Levin Charitable Fund, Regeneron Pharmaceuticals
Responsible party
Andrew J Long, PharmD (Pharmacist, Stanford University) — Sponsor-investigator
First posted
Nov 1, 2019
Start date
Oct 18, 2021
Primary completion
Dec 17, 2024
Completion
Feb 25, 2025
Results posted
Jan 7, 2026
Last update
Jan 7, 2026

Study contacts

Andrew Long, PharmD
study director · Stanford, Sean N. Parker Center for Allergy & Asthma Research

Oversight

Data monitoring committee
Yes
FDA-regulated drug
Yes
FDA-regulated device
No
View the source record on ClinicalTrials.gov ↗

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