Trial Outcomes & Findings for Dupilumab and Milk OIT for the Treatment of Cow's Milk Allergy (NCT NCT04148352)
NCT ID: NCT04148352
Last Updated: 2026-01-07
Results Overview
DBPCFC double-blind, placebo-controlled food challenge; OIT oral immunotherapy; withdrawals are imputed as failures
Recruitment status
TERMINATED
Study phase
PHASE2
Target enrollment
33 participants
Primary outcome timeframe
Week 18
Results posted on
2026-01-07
Participant Flow
Of 33 enrolled, 8 participants failed at screening and 2 withdrew from the study prior to randomization, for a total of 10 participants consented but not randomized
Participant milestones
| Measure |
Active Dupilumab and Milk Oral Immunotherapy (OIT)
Dupilumab every two to four weeks for 4 weeks, followed by dupilumab every two to four weeks plus milk OIT for 12 weeks, then milk OIT alone for 8 weeks
|
Placebo for Dupilumab and Milk OIT
Placebo for dupilumab every two to four weeks for 4 weeks, followed by placebo for dupilumab every two to four weeks plus milk OIT for 12 weeks, then milk OIT alone for 8 weeks
|
|---|---|---|
|
Overall Study
STARTED
|
11
|
12
|
|
Overall Study
Week 18 Double-Blind, Placebo-Controlled Food Challenge (DBPCFC)
|
11
|
8
|
|
Overall Study
Week 24 DBPCFC
|
11
|
6
|
|
Overall Study
COMPLETED
|
11
|
6
|
|
Overall Study
NOT COMPLETED
|
0
|
6
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
Dupilumab and Milk OIT for the Treatment of Cow's Milk Allergy
Baseline characteristics by cohort
| Measure |
Active Dupilumab and Milk OIT
n=11 Participants
Dupilumab every two to four weeks for 4 weeks, followed by dupilumab every two to four weeks plus milk OIT for 12 weeks, then milk OIT alone for 8 weeks
|
Placebo for Dupilumab and Milk OIT
n=12 Participants
Placebo for dupilumab every two to four weeks for 4 weeks, followed by placebo for dupilumab every two to four weeks plus milk OIT for 12 weeks, then milk OIT alone for 8 weeks
|
Total
n=23 Participants
Total of all reporting groups
|
|---|---|---|---|
|
Age, Customized
4 - 10 years
|
4 Participants
n=37 Participants
|
6 Participants
n=56 Participants
|
10 Participants
n=95 Participants
|
|
Age, Customized
11 - 17 years
|
4 Participants
n=37 Participants
|
3 Participants
n=56 Participants
|
7 Participants
n=95 Participants
|
|
Age, Customized
18+ years
|
3 Participants
n=37 Participants
|
3 Participants
n=56 Participants
|
6 Participants
n=95 Participants
|
|
Sex: Female, Male
Female
|
3 Participants
n=37 Participants
|
2 Participants
n=56 Participants
|
5 Participants
n=95 Participants
|
|
Sex: Female, Male
Male
|
8 Participants
n=37 Participants
|
10 Participants
n=56 Participants
|
18 Participants
n=95 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
0 Participants
n=37 Participants
|
3 Participants
n=56 Participants
|
3 Participants
n=95 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
11 Participants
n=37 Participants
|
9 Participants
n=56 Participants
|
20 Participants
n=95 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=37 Participants
|
0 Participants
n=56 Participants
|
0 Participants
n=95 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=37 Participants
|
0 Participants
n=56 Participants
|
0 Participants
n=95 Participants
|
|
Race (NIH/OMB)
Asian
|
1 Participants
n=37 Participants
|
3 Participants
n=56 Participants
|
4 Participants
n=95 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=37 Participants
|
0 Participants
n=56 Participants
|
0 Participants
n=95 Participants
|
|
Race (NIH/OMB)
Black or African American
|
1 Participants
n=37 Participants
|
0 Participants
n=56 Participants
|
1 Participants
n=95 Participants
|
|
Race (NIH/OMB)
White
|
7 Participants
n=37 Participants
|
5 Participants
n=56 Participants
|
12 Participants
n=95 Participants
|
|
Race (NIH/OMB)
More than one race
|
2 Participants
n=37 Participants
|
4 Participants
n=56 Participants
|
6 Participants
n=95 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=37 Participants
|
0 Participants
n=56 Participants
|
0 Participants
n=95 Participants
|
|
History of Atopy
Asthma
|
7 Participants
n=37 Participants
|
8 Participants
n=56 Participants
|
15 Participants
n=95 Participants
|
|
History of Atopy
Allergic Rhinitis
|
6 Participants
n=37 Participants
|
6 Participants
n=56 Participants
|
12 Participants
n=95 Participants
|
|
History of Atopy
Atopic Disease
|
9 Participants
n=37 Participants
|
7 Participants
n=56 Participants
|
16 Participants
n=95 Participants
|
PRIMARY outcome
Timeframe: Week 18Population: Intention to treat population: all randomized patients, analyzed in the treatment group they were assigned to, regardless of whether they actually received the treatment, dropped out, or deviated from the protocol.
DBPCFC double-blind, placebo-controlled food challenge; OIT oral immunotherapy; withdrawals are imputed as failures
Outcome measures
| Measure |
Active Dupilumab and Milk OIT
n=11 Participants
Dupilumab every two to four weeks for 4 weeks, followed by dupilumab every two to four weeks plus milk OIT for 12 weeks, then milk OIT alone for 8 weeks
|
Placebo for Dupilumab and Milk OIT
n=12 Participants
Placebo for dupilumab every two to four weeks for 4 weeks, followed by placebo for dupilumab every two to four weeks plus milk OIT for 12 weeks, then milk OIT alone for 8 weeks
|
|---|---|---|
|
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
|
8 Participants
|
5 Participants
|
SECONDARY outcome
Timeframe: Week 18Population: Intention to treat population
DBPCFC double-blind, placebo-controlled food challenge; withdrawals are imputed as failures
Outcome measures
| Measure |
Active Dupilumab and Milk OIT
n=11 Participants
Dupilumab every two to four weeks for 4 weeks, followed by dupilumab every two to four weeks plus milk OIT for 12 weeks, then milk OIT alone for 8 weeks
|
Placebo for Dupilumab and Milk OIT
n=12 Participants
Placebo for dupilumab every two to four weeks for 4 weeks, followed by placebo for dupilumab every two to four weeks plus milk OIT for 12 weeks, then milk OIT alone for 8 weeks
|
|---|---|---|
|
Proportion of Participants Who Tolerate at Least 1040 mg Cumulative Milk Protein at Week 18 DBPCFC
|
9 Participants
|
6 Participants
|
SECONDARY outcome
Timeframe: Week 18Population: Intention to treat population
DBPCFC double-blind, placebo-controlled food challenge; withdrawals are imputed as failures
Outcome measures
| Measure |
Active Dupilumab and Milk OIT
n=11 Participants
Dupilumab every two to four weeks for 4 weeks, followed by dupilumab every two to four weeks plus milk OIT for 12 weeks, then milk OIT alone for 8 weeks
|
Placebo for Dupilumab and Milk OIT
n=12 Participants
Placebo for dupilumab every two to four weeks for 4 weeks, followed by placebo for dupilumab every two to four weeks plus milk OIT for 12 weeks, then milk OIT alone for 8 weeks
|
|---|---|---|
|
Proportion of Participants Who Tolerate 4040 mg Cumulative Milk Protein at Week 18 DBPCFC
|
5 Participants
|
3 Participants
|
SECONDARY outcome
Timeframe: week 24Population: Intention to treat population
DBPCFC double-blind, placebo-controlled food challenge; withdrawals are imputed as failures
Outcome measures
| Measure |
Active Dupilumab and Milk OIT
n=11 Participants
Dupilumab every two to four weeks for 4 weeks, followed by dupilumab every two to four weeks plus milk OIT for 12 weeks, then milk OIT alone for 8 weeks
|
Placebo for Dupilumab and Milk OIT
n=12 Participants
Placebo for dupilumab every two to four weeks for 4 weeks, followed by placebo for dupilumab every two to four weeks plus milk OIT for 12 weeks, then milk OIT alone for 8 weeks
|
|---|---|---|
|
Proportion of Participants Who Tolerate at Least 1040 mg Cumulative Milk Protein at Week 24 DBPCFC
|
8 Participants
|
5 Participants
|
SECONDARY outcome
Timeframe: Week 24Population: Intention to treat population
DBPCFC double-blind, placebo-controlled food challenge; withdrawals are imputed as failures
Outcome measures
| Measure |
Active Dupilumab and Milk OIT
n=11 Participants
Dupilumab every two to four weeks for 4 weeks, followed by dupilumab every two to four weeks plus milk OIT for 12 weeks, then milk OIT alone for 8 weeks
|
Placebo for Dupilumab and Milk OIT
n=12 Participants
Placebo for dupilumab every two to four weeks for 4 weeks, followed by placebo for dupilumab every two to four weeks plus milk OIT for 12 weeks, then milk OIT alone for 8 weeks
|
|---|---|---|
|
Proportion of Participants Who Tolerate at Least 2040 mg Cumulative Milk Protein at Week 24 DBPCFC
|
7 Participants
|
5 Participants
|
SECONDARY outcome
Timeframe: Week 24Population: Intention to treat population
DBPCFC double-blind, placebo-controlled food challenge; withdrawals are imputed as failures
Outcome measures
| Measure |
Active Dupilumab and Milk OIT
n=11 Participants
Dupilumab every two to four weeks for 4 weeks, followed by dupilumab every two to four weeks plus milk OIT for 12 weeks, then milk OIT alone for 8 weeks
|
Placebo for Dupilumab and Milk OIT
n=12 Participants
Placebo for dupilumab every two to four weeks for 4 weeks, followed by placebo for dupilumab every two to four weeks plus milk OIT for 12 weeks, then milk OIT alone for 8 weeks
|
|---|---|---|
|
Proportion of Participants Who Tolerate 4040 mg Cumulative Milk Protein at Week 24 DBPCFC
|
5 Participants
|
3 Participants
|
SECONDARY outcome
Timeframe: Baseline and week 18Population: Participants with week 18 data
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.
Outcome measures
| Measure |
Active Dupilumab and Milk OIT
n=11 Participants
Dupilumab every two to four weeks for 4 weeks, followed by dupilumab every two to four weeks plus milk OIT for 12 weeks, then milk OIT alone for 8 weeks
|
Placebo for Dupilumab and Milk OIT
n=8 Participants
Placebo for dupilumab every two to four weeks for 4 weeks, followed by placebo for dupilumab every two to four weeks plus milk OIT for 12 weeks, then milk OIT alone for 8 weeks
|
|---|---|---|
|
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
|
1.632 log10 mg cumulative tolerated protein
Standard Deviation 0.888
|
1.899 log10 mg cumulative tolerated protein
Standard Deviation 0.600
|
|
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
Change at week 18
|
1.574 log10 mg cumulative tolerated protein
Standard Deviation 0.880
|
1.127 log10 mg cumulative tolerated protein
Standard Deviation 0.618
|
SECONDARY outcome
Timeframe: Baseline and week 24Population: Participants with week 24 data
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.
Outcome measures
| Measure |
Active Dupilumab and Milk OIT
n=11 Participants
Dupilumab every two to four weeks for 4 weeks, followed by dupilumab every two to four weeks plus milk OIT for 12 weeks, then milk OIT alone for 8 weeks
|
Placebo for Dupilumab and Milk OIT
n=6 Participants
Placebo for dupilumab every two to four weeks for 4 weeks, followed by placebo for dupilumab every two to four weeks plus milk OIT for 12 weeks, then milk OIT alone for 8 weeks
|
|---|---|---|
|
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
|
1.632 log10 mg cumulative tolerated protein
Standard Deviation 0.888
|
1.898 log10 mg cumulative tolerated protein
Standard Deviation 0.691
|
|
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
Change at week 24
|
1.559 log10 mg cumulative tolerated protein
Standard Deviation 1.153
|
1.366 log10 mg cumulative tolerated protein
Standard Deviation 0.490
|
Adverse Events
Active Dupilumab and Milk OIT
Serious events: 0 serious events
Other events: 10 other events
Deaths: 0 deaths
Placebo for Dupilumab and Milk OIT
Serious events: 0 serious events
Other events: 11 other events
Deaths: 0 deaths
Serious adverse events
Adverse event data not reported
Other adverse events
| Measure |
Active Dupilumab and Milk OIT
n=11 participants at risk
Dupilumab every two to four weeks for 4 weeks, followed by dupilumab every two to four weeks plus milk OIT for 12 weeks, then milk OIT alone for 8 weeks
|
Placebo for Dupilumab and Milk OIT
n=12 participants at risk
Dupilumab every two to four weeks for 4 weeks, followed by dupilumab every two to four weeks plus milk OIT for 12 weeks, then milk OIT alone for 8 weeks
|
|---|---|---|
|
Gastrointestinal disorders
Gastrointestinal Food Challenge Reactions Allergic not related to OIT
|
63.6%
7/11 • From enrollment until end of follow-up, up to 24 weeks
|
58.3%
7/12 • From enrollment until end of follow-up, up to 24 weeks
|
|
Skin and subcutaneous tissue disorders
Skin Food Challenge Reactions Allergic not related to OIT
|
54.5%
6/11 • From enrollment until end of follow-up, up to 24 weeks
|
75.0%
9/12 • From enrollment until end of follow-up, up to 24 weeks
|
|
Respiratory, thoracic and mediastinal disorders
Respiratory Food Challenge Reactions Allergic not related to OIT
|
63.6%
7/11 • From enrollment until end of follow-up, up to 24 weeks
|
58.3%
7/12 • From enrollment until end of follow-up, up to 24 weeks
|
|
Vascular disorders
Vascular Allergic AEs related to OIT
|
9.1%
1/11 • From enrollment until end of follow-up, up to 24 weeks
|
8.3%
1/12 • From enrollment until end of follow-up, up to 24 weeks
|
|
Gastrointestinal disorders
Gastrointestinal Allergic AEs related to OIT
|
90.9%
10/11 • From enrollment until end of follow-up, up to 24 weeks
|
91.7%
11/12 • From enrollment until end of follow-up, up to 24 weeks
|
|
Skin and subcutaneous tissue disorders
Skin Allergic AEs related to OIT
|
72.7%
8/11 • From enrollment until end of follow-up, up to 24 weeks
|
75.0%
9/12 • From enrollment until end of follow-up, up to 24 weeks
|
|
Respiratory, thoracic and mediastinal disorders
Respiratory Allergic AEs related to OIT
|
54.5%
6/11 • From enrollment until end of follow-up, up to 24 weeks
|
83.3%
10/12 • From enrollment until end of follow-up, up to 24 weeks
|
|
General disorders
General Allergic AEs related to OIT
|
36.4%
4/11 • From enrollment until end of follow-up, up to 24 weeks
|
25.0%
3/12 • From enrollment until end of follow-up, up to 24 weeks
|
|
Nervous system disorders
Nervous system Allergic AEs related to OIT
|
9.1%
1/11 • From enrollment until end of follow-up, up to 24 weeks
|
8.3%
1/12 • From enrollment until end of follow-up, up to 24 weeks
|
|
Eye disorders
Eye Allergic AEs related to OIT
|
18.2%
2/11 • From enrollment until end of follow-up, up to 24 weeks
|
8.3%
1/12 • From enrollment until end of follow-up, up to 24 weeks
|
|
Immune system disorders
Immune system Allergic AEs related to OIT
|
9.1%
1/11 • From enrollment until end of follow-up, up to 24 weeks
|
16.7%
2/12 • From enrollment until end of follow-up, up to 24 weeks
|
|
Nervous system disorders
Nervous System Food Challenge Reactions Allergic not related to OIT
|
9.1%
1/11 • From enrollment until end of follow-up, up to 24 weeks
|
25.0%
3/12 • From enrollment until end of follow-up, up to 24 weeks
|
|
Psychiatric disorders
Psychiatric Food Challenge Reactions Allergic not related to OIT
|
18.2%
2/11 • From enrollment until end of follow-up, up to 24 weeks
|
0.00%
0/12 • From enrollment until end of follow-up, up to 24 weeks
|
|
Eye disorders
Eye Food Challenge Reactions Allergic not related to OIT
|
9.1%
1/11 • From enrollment until end of follow-up, up to 24 weeks
|
0.00%
0/12 • From enrollment until end of follow-up, up to 24 weeks
|
|
Immune system disorders
Immune System Food Challenge Reactions Allergic not related to OIT
|
9.1%
1/11 • From enrollment until end of follow-up, up to 24 weeks
|
16.7%
2/12 • From enrollment until end of follow-up, up to 24 weeks
|
|
General disorders
General/administration site Non-Allergic not related to OIT
|
36.4%
4/11 • From enrollment until end of follow-up, up to 24 weeks
|
25.0%
3/12 • From enrollment until end of follow-up, up to 24 weeks
|
|
Eye disorders
Eye Non-Allergic not related to OIT
|
9.1%
1/11 • From enrollment until end of follow-up, up to 24 weeks
|
0.00%
0/12 • From enrollment until end of follow-up, up to 24 weeks
|
|
Gastrointestinal disorders
Gastrointestinal Non-Allergic not related to OIT
|
18.2%
2/11 • From enrollment until end of follow-up, up to 24 weeks
|
8.3%
1/12 • From enrollment until end of follow-up, up to 24 weeks
|
|
Infections and infestations
Infection Non-Allergic not related to OIT
|
27.3%
3/11 • From enrollment until end of follow-up, up to 24 weeks
|
33.3%
4/12 • From enrollment until end of follow-up, up to 24 weeks
|
|
Nervous system disorders
Nervous Non-Allergic not related to OIT
|
27.3%
3/11 • From enrollment until end of follow-up, up to 24 weeks
|
8.3%
1/12 • From enrollment until end of follow-up, up to 24 weeks
|
|
Respiratory, thoracic and mediastinal disorders
Respiratory Non-Allergic not related to OIT
|
36.4%
4/11 • From enrollment until end of follow-up, up to 24 weeks
|
33.3%
4/12 • From enrollment until end of follow-up, up to 24 weeks
|
|
Skin and subcutaneous tissue disorders
Skin Non-Allergic not related to OIT
|
0.00%
0/11 • From enrollment until end of follow-up, up to 24 weeks
|
16.7%
2/12 • From enrollment until end of follow-up, up to 24 weeks
|
|
Renal and urinary disorders
Renal/Urinary Non-Allergic not related to OIT
|
9.1%
1/11 • From enrollment until end of follow-up, up to 24 weeks
|
0.00%
0/12 • From enrollment until end of follow-up, up to 24 weeks
|
|
Injury, poisoning and procedural complications
Injury Non-Allergic not related to OIT
|
0.00%
0/11 • From enrollment until end of follow-up, up to 24 weeks
|
8.3%
1/12 • From enrollment until end of follow-up, up to 24 weeks
|
|
Ear and labyrinth disorders
Ear Non-Allergic not related to OIT
|
9.1%
1/11 • From enrollment until end of follow-up, up to 24 weeks
|
0.00%
0/12 • From enrollment until end of follow-up, up to 24 weeks
|
Additional Information
Sayantani B. Sindher, MD
Sean N Parker Center for Allergy & Asthma Research at Stanford University
Phone: 650-521-7237
Email: [email protected]
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place