Trial Outcomes & Findings for Immune Basis and Clinical Implications of Threshold-based Phenotypes of Peanut Allergy (NCT NCT03907397)
NCT ID: NCT03907397
Last Updated: 2025-02-07
Results Overview
The difference between the treatment and comparison (avoidance) groups in the percentage of children who by the endpoint double-blind, placebo-controlled oral food challenge (DBPCFC) tolerate a dose at least 2 steps higher than their baseline DBPCFC or 9043 mg of peanut protein. Due to missing data in the primary endpoint, the protocol \& SAP specified a priori that missing data would be imputed using multiple imputation and results based on 30 completed-imputed data sets would be combined using Rubin's rule. In the Peanut Protein group, the observed data was so strong that participants with missing data were also imputed as success across all imputations (hence, 100% success rate). In the Peanut Avoidance group, there was more variability in the proportion of successes across imputations and these varying proportions were averaged. Missing data were imputed and results were pooled.
COMPLETED
PHASE2
73 participants
72 weeks
2025-02-07
Participant Flow
From August 2019 to April 2022 at one clinical site in the US. A total of 129 participants were assessed for eligibility and 73 fulfilled criteria for randomization.
Participant milestones
| Measure |
Peanut Protein
Ingests peanut - . Depending upon reaction threshold, participants may begin with different starting amounts of store bought peanut butter measured with study-supplied kitchen measuring spoons.
Peanut Protein: up to 3 level teaspoons peanut butter or equivalent (approximately 3400 mg)
|
Avoidance
Avoids peanut, standard care
Continued peanut avoidance: Standard of care avoidance of peanut
|
|---|---|---|
|
Overall Study
STARTED
|
38
|
35
|
|
Overall Study
COMPLETED
|
30
|
29
|
|
Overall Study
NOT COMPLETED
|
8
|
6
|
Reasons for withdrawal
| Measure |
Peanut Protein
Ingests peanut - . Depending upon reaction threshold, participants may begin with different starting amounts of store bought peanut butter measured with study-supplied kitchen measuring spoons.
Peanut Protein: up to 3 level teaspoons peanut butter or equivalent (approximately 3400 mg)
|
Avoidance
Avoids peanut, standard care
Continued peanut avoidance: Standard of care avoidance of peanut
|
|---|---|---|
|
Overall Study
Lost to Follow-up
|
0
|
1
|
|
Overall Study
Physician Decision
|
2
|
1
|
|
Overall Study
Withdrawal by Subject
|
5
|
4
|
|
Overall Study
withdrew after Day 1 of the primary endpoint desensitization visit.
|
1
|
0
|
Baseline Characteristics
one sample did not have enough plasma for the IgG run.
Baseline characteristics by cohort
| Measure |
Peanut Protein
n=38 Participants
Ingests peanut - . Depending upon reaction threshold, participants may begin with different starting amounts of store bought peanut butter measured with study-supplied kitchen measuring spoons.
Peanut Protein: up to 3 level teaspoons peanut butter or equivalent (approximately 3400 mg)
|
Avoidance
n=35 Participants
Avoids peanut, standard care
Continued peanut avoidance: Standard of care avoidance of peanut
|
Total
n=73 Participants
Total of all reporting groups
|
|---|---|---|---|
|
Age, Continuous
|
6.0 years
n=38 Participants
|
7.0 years
n=35 Participants
|
7.0 years
n=73 Participants
|
|
Sex: Female, Male
Female
|
19 Participants
n=38 Participants
|
9 Participants
n=35 Participants
|
28 Participants
n=73 Participants
|
|
Sex: Female, Male
Male
|
19 Participants
n=38 Participants
|
26 Participants
n=35 Participants
|
45 Participants
n=73 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
5 Participants
n=38 Participants
|
2 Participants
n=35 Participants
|
7 Participants
n=73 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
33 Participants
n=38 Participants
|
33 Participants
n=35 Participants
|
66 Participants
n=73 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=38 Participants
|
0 Participants
n=35 Participants
|
0 Participants
n=73 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=38 Participants
|
0 Participants
n=35 Participants
|
0 Participants
n=73 Participants
|
|
Race (NIH/OMB)
Asian
|
10 Participants
n=38 Participants
|
4 Participants
n=35 Participants
|
14 Participants
n=73 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=38 Participants
|
0 Participants
n=35 Participants
|
0 Participants
n=73 Participants
|
|
Race (NIH/OMB)
Black or African American
|
1 Participants
n=38 Participants
|
0 Participants
n=35 Participants
|
1 Participants
n=73 Participants
|
|
Race (NIH/OMB)
White
|
18 Participants
n=38 Participants
|
24 Participants
n=35 Participants
|
42 Participants
n=73 Participants
|
|
Race (NIH/OMB)
More than one race
|
9 Participants
n=38 Participants
|
7 Participants
n=35 Participants
|
16 Participants
n=73 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=38 Participants
|
0 Participants
n=35 Participants
|
0 Participants
n=73 Participants
|
|
Skin prick test
|
8.0 mm
n=38 Participants
|
9.0 mm
n=35 Participants
|
9.0 mm
n=73 Participants
|
|
Peanut IgE level
|
5.5 kUA/L
n=38 Participants
|
4.6 kUA/L
n=35 Participants
|
5.1 kUA/L
n=73 Participants
|
|
Peanut IgG4 level
|
0.4 mgA/L
n=37 Participants • one sample did not have enough plasma for the IgG run.
|
0.4 mgA/L
n=35 Participants • one sample did not have enough plasma for the IgG run.
|
0.4 mgA/L
n=72 Participants • one sample did not have enough plasma for the IgG run.
|
|
Ara h2 IgE level
|
3.8 kUA/L
n=38 Participants
|
2.6 kUA/L
n=35 Participants
|
2.9 kUA/L
n=73 Participants
|
|
Ara h2 IgG4 level
|
0.020 mgA/L
n=37 Participants • one sample did not have enough plasma for the IgG run.
|
0.050 mgA/L
n=35 Participants • one sample did not have enough plasma for the IgG run.
|
0.030 mgA/L
n=72 Participants • one sample did not have enough plasma for the IgG run.
|
PRIMARY outcome
Timeframe: 72 weeksThe difference between the treatment and comparison (avoidance) groups in the percentage of children who by the endpoint double-blind, placebo-controlled oral food challenge (DBPCFC) tolerate a dose at least 2 steps higher than their baseline DBPCFC or 9043 mg of peanut protein. Due to missing data in the primary endpoint, the protocol \& SAP specified a priori that missing data would be imputed using multiple imputation and results based on 30 completed-imputed data sets would be combined using Rubin's rule. In the Peanut Protein group, the observed data was so strong that participants with missing data were also imputed as success across all imputations (hence, 100% success rate). In the Peanut Avoidance group, there was more variability in the proportion of successes across imputations and these varying proportions were averaged. Missing data were imputed and results were pooled.
Outcome measures
| Measure |
Peanut Protein
n=38 Participants
Ingests peanut - . Depending upon reaction threshold, participants may begin with different starting amounts of store bought peanut butter measured with study-supplied kitchen measuring spoons.
Peanut Protein: up to 3 level teaspoons peanut butter or equivalent (approximately 3400 mg)
|
Avoidance
n=35 Participants
Avoids peanut, standard care
Continued peanut avoidance: Standard of care avoidance of peanut
|
|---|---|---|
|
Percentage of Children That Tolerate the Full Challenge
|
100 percentage of participants
|
21 percentage of participants
|
SECONDARY outcome
Timeframe: 96 weeksThe percentage of children who achieve sustained unresponsiveness or natural tolerance during the study.
Outcome measures
| Measure |
Peanut Protein
n=38 Participants
Ingests peanut - . Depending upon reaction threshold, participants may begin with different starting amounts of store bought peanut butter measured with study-supplied kitchen measuring spoons.
Peanut Protein: up to 3 level teaspoons peanut butter or equivalent (approximately 3400 mg)
|
Avoidance
n=35 Participants
Avoids peanut, standard care
Continued peanut avoidance: Standard of care avoidance of peanut
|
|---|---|---|
|
The Percentage of Children That Achieve Sustained Unresponsiveness
|
68.4 percentage of participants
Interval 53.6 to 83.2
|
8.6 percentage of participants
Interval 0.0 to 17.9
|
SECONDARY outcome
Timeframe: 96 weeksSafety parameter assessed by number of participants with acute allergic reactions which includes anaphylaxis or gastrointestinal side effects.
Outcome measures
| Measure |
Peanut Protein
n=38 Participants
Ingests peanut - . Depending upon reaction threshold, participants may begin with different starting amounts of store bought peanut butter measured with study-supplied kitchen measuring spoons.
Peanut Protein: up to 3 level teaspoons peanut butter or equivalent (approximately 3400 mg)
|
Avoidance
n=35 Participants
Avoids peanut, standard care
Continued peanut avoidance: Standard of care avoidance of peanut
|
|---|---|---|
|
Number of Children With Acute Allergic Reactions
|
25 Participants
|
11 Participants
|
SECONDARY outcome
Timeframe: baseline and at 72 weeksThe Food Allergy Quality of Life-Parental Burden (FAQL-PB) Scale is a 17-item instrument. It utilizes a 7-point Likert scale ranging from 0 (not troubled) to 6 (extremely troubled). The number circled for each question is summed to provide a total continuous score range of 0 to 102 with a higher score indicating greater burden on the family.
Outcome measures
| Measure |
Peanut Protein
n=38 Participants
Ingests peanut - . Depending upon reaction threshold, participants may begin with different starting amounts of store bought peanut butter measured with study-supplied kitchen measuring spoons.
Peanut Protein: up to 3 level teaspoons peanut butter or equivalent (approximately 3400 mg)
|
Avoidance
n=35 Participants
Avoids peanut, standard care
Continued peanut avoidance: Standard of care avoidance of peanut
|
|---|---|---|
|
Mean Change in Food Allergy Quality of Life Parental Burden Instrument
|
-7.78 score on a scale
Interval -13.1 to -2.47
|
-9.23 score on a scale
Interval -14.71 to -3.74
|
SECONDARY outcome
Timeframe: Baseline and 72 weeksChange in Skin Prick Test (SPT) mean wheal size at 72 weeks as compared to baseline. A skin test reaction is considered positive if the wheal size for the antigen is at least 3 mm larger than the wheal size of the negative control (saline).
Outcome measures
| Measure |
Peanut Protein
n=38 Participants
Ingests peanut - . Depending upon reaction threshold, participants may begin with different starting amounts of store bought peanut butter measured with study-supplied kitchen measuring spoons.
Peanut Protein: up to 3 level teaspoons peanut butter or equivalent (approximately 3400 mg)
|
Avoidance
n=35 Participants
Avoids peanut, standard care
Continued peanut avoidance: Standard of care avoidance of peanut
|
|---|---|---|
|
Mean Change in SPT Wheal Size
|
-3.05 mm
Interval -4.27 to -1.84
|
-0.48 mm
Interval -1.74 to 0.78
|
SECONDARY outcome
Timeframe: Baseline and 72 weeksMean Change in Peanut-specific IgE level at week 72 as compared to baseline.
Outcome measures
| Measure |
Peanut Protein
n=38 Participants
Ingests peanut - . Depending upon reaction threshold, participants may begin with different starting amounts of store bought peanut butter measured with study-supplied kitchen measuring spoons.
Peanut Protein: up to 3 level teaspoons peanut butter or equivalent (approximately 3400 mg)
|
Avoidance
n=35 Participants
Avoids peanut, standard care
Continued peanut avoidance: Standard of care avoidance of peanut
|
|---|---|---|
|
Mean Change in Peanut-specific IgE
|
-0.22 kUA/L
Interval -0.45 to 0.02
|
0.40 kUA/L
Interval 0.15 to 0.64
|
SECONDARY outcome
Timeframe: Baseline and 72 weeksMean Change in Peanut-specific IgG4 level at week 72 as compared to baseline.
Outcome measures
| Measure |
Peanut Protein
n=38 Participants
Ingests peanut - . Depending upon reaction threshold, participants may begin with different starting amounts of store bought peanut butter measured with study-supplied kitchen measuring spoons.
Peanut Protein: up to 3 level teaspoons peanut butter or equivalent (approximately 3400 mg)
|
Avoidance
n=35 Participants
Avoids peanut, standard care
Continued peanut avoidance: Standard of care avoidance of peanut
|
|---|---|---|
|
Mean Change in Peanut-specific IgG4
|
3.11 mgA/L
Interval 2.41 to 3.81
|
0.52 mgA/L
Interval -0.2 to 1.24
|
Adverse Events
Peanut Protein
Avoidance
Serious adverse events
Adverse event data not reported
Other adverse events
| Measure |
Peanut Protein
n=38 participants at risk
Ingests peanut - . Depending upon reaction threshold, participants may begin with different starting amounts of store bought peanut butter measured with study-supplied kitchen measuring spoons.
Peanut Protein: up to 3 level teaspoons peanut butter or equivalent (approximately 3400 mg)
|
Avoidance
n=35 participants at risk
Avoids peanut, standard care
Continued peanut avoidance: Standard of care avoidance of peanut
|
|---|---|---|
|
Gastrointestinal disorders
Abdominal Discomfort
|
23.7%
9/38 • 96 weeks
|
2.9%
1/35 • 96 weeks
|
|
Gastrointestinal disorders
Abdominal Pain
|
10.5%
4/38 • 96 weeks
|
2.9%
1/35 • 96 weeks
|
|
Gastrointestinal disorders
Nausea
|
5.3%
2/38 • 96 weeks
|
0.00%
0/35 • 96 weeks
|
|
Gastrointestinal disorders
Oral Pruritus
|
44.7%
17/38 • 96 weeks
|
22.9%
8/35 • 96 weeks
|
|
Gastrointestinal disorders
Vomiting
|
21.1%
8/38 • 96 weeks
|
5.7%
2/35 • 96 weeks
|
|
Gastrointestinal disorders
Vomiting Projectile
|
2.6%
1/38 • 96 weeks
|
0.00%
0/35 • 96 weeks
|
|
Immune system disorders
Anaphylactic Reaction
|
0.00%
0/38 • 96 weeks
|
5.7%
2/35 • 96 weeks
|
|
Immune system disorders
Lip Edema
|
2.6%
1/38 • 96 weeks
|
2.9%
1/35 • 96 weeks
|
|
Immune system disorders
Pruritus Allergic
|
2.6%
1/38 • 96 weeks
|
11.4%
4/35 • 96 weeks
|
|
Immune system disorders
Urticaria
|
26.3%
10/38 • 96 weeks
|
17.1%
6/35 • 96 weeks
|
|
Respiratory, thoracic and mediastinal disorders
Cough
|
5.3%
2/38 • 96 weeks
|
2.9%
1/35 • 96 weeks
|
|
Respiratory, thoracic and mediastinal disorders
Rhinorrhea
|
10.5%
4/38 • 96 weeks
|
5.7%
2/35 • 96 weeks
|
|
Respiratory, thoracic and mediastinal disorders
Throat Tightness
|
7.9%
3/38 • 96 weeks
|
5.7%
2/35 • 96 weeks
|
|
Respiratory, thoracic and mediastinal disorders
Wheezing
|
0.00%
0/38 • 96 weeks
|
2.9%
1/35 • 96 weeks
|
|
Skin and subcutaneous tissue disorders
Dermatitis Atopic
|
5.3%
2/38 • 96 weeks
|
2.9%
1/35 • 96 weeks
|
|
Skin and subcutaneous tissue disorders
Erythema
|
5.3%
2/38 • 96 weeks
|
2.9%
1/35 • 96 weeks
|
Additional Information
Dr. Scott Sicherer
Icahn School of Medicine at Mount Sinai
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place