Trial Outcomes & Findings for Sublingual Immunotherapy for Peanut Allergy and Induction of Tolerance (NCT NCT01373242)

NCT ID: NCT01373242

Last Updated: 2019-06-12

Results Overview

An estimate of the dose as assessed by the 48th month DBPCFC reported in mg peanut protein, also called population sensitization threshold, predicted to provoke reactions in 5% of the peanut-allergic population. This will also give population level no observed adverse event level (NOAEL) and lowest observed adverse event level (LOAEL) that would define interval of consecutive administered dose levels within which the population sensitization threshold lies. NOAEL is the highest dose observed not to produce any adverse effect and LOAEL is the lowest dose that is observed to produce an adverse effect.

Recruitment status

COMPLETED

Study phase

PHASE1/PHASE2

Target enrollment

54 participants

Primary outcome timeframe

48 - 52 months

Results posted on

2019-06-12

Participant Flow

Peanut allergic children ages 1-11 years

Participant milestones

Participant milestones
Measure
Peanut (Liquid Peanut Extract) SLIT
All subjects will receive peanut SLIT upon enrollment for at least 48 months and then undergo a desensitization DBPCFC. Subjects will then be randomized to be off treatment for a period between 1 to 17 weeks. Subjects will then undergo a final DBPCFC. Liquid peanut extract (Peanut SLIT): Liquid peanut extract will be administered under the tongue
Overall Study
STARTED
54
Overall Study
COMPLETED
38
Overall Study
NOT COMPLETED
16

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

Sublingual Immunotherapy for Peanut Allergy and Induction of Tolerance

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure
Peanut ( Liquid Peanut Extract) SLIT
n=54 Participants
All subjects will receive peanut SLIT upon enrollment for at least the first 48 months. After the desensitization DBPCFC after at least 48 months of treatment, subjects will be randomized off treatment from 1 to 17 weeks. Subjects will then undergo another DBPCFC. Liquid peanut extract (Peanut SLIT): Liquid peanut extract will be administered under the tongue
Age, Categorical
<=18 years
54 Participants
n=5 Participants
Age, Categorical
Between 18 and 65 years
0 Participants
n=5 Participants
Age, Categorical
>=65 years
0 Participants
n=5 Participants
Age, Continuous
7.05 years
n=5 Participants
Sex: Female, Male
Female
20 Participants
n=5 Participants
Sex: Female, Male
Male
34 Participants
n=5 Participants
Race (NIH/OMB)
American Indian or Alaska Native
0 Participants
n=5 Participants
Race (NIH/OMB)
Asian
4 Participants
n=5 Participants
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
0 Participants
n=5 Participants
Race (NIH/OMB)
Black or African American
0 Participants
n=5 Participants
Race (NIH/OMB)
White
49 Participants
n=5 Participants
Race (NIH/OMB)
More than one race
1 Participants
n=5 Participants
Race (NIH/OMB)
Unknown or Not Reported
0 Participants
n=5 Participants
Region of Enrollment
United States
54 Participants
n=5 Participants
Peanut-specific IgE (kU/L)
85.6 kU/L
n=5 Participants
Peanut skin prick test wheal size (mm)
16 mm
n=5 Participants

PRIMARY outcome

Timeframe: 48 - 52 months

An estimate of the dose as assessed by the 48th month DBPCFC reported in mg peanut protein, also called population sensitization threshold, predicted to provoke reactions in 5% of the peanut-allergic population. This will also give population level no observed adverse event level (NOAEL) and lowest observed adverse event level (LOAEL) that would define interval of consecutive administered dose levels within which the population sensitization threshold lies. NOAEL is the highest dose observed not to produce any adverse effect and LOAEL is the lowest dose that is observed to produce an adverse effect.

Outcome measures

Outcome measures
Measure
Peanut (Liquid Peanut Extract) SLIT
n=54 Participants
All subjects will receive peanut SLIT upon enrollment for at least 48 months and then undergo a desensitization DBPCFC. Subjects will then be randomized to be off treatment for a period between 1 to 17 weeks. Subjects will then undergo a final DBPCFC. Liquid peanut extract (Peanut SLIT): Liquid peanut extract will be administered under the tongue
Non-desensitized After Peanut SLIT
All subjects will receive peanut SLIT upon enrollment for at least 48 months and then undergo a desensitization DBPCFC. 'Non-desensitized' defined as experiencing dose limiting symptoms during the 5000mg desensitization DBPCFC.
Population Sensitization Threshold in mg Peanut Protein Predicted to Provoke Reactions in 5% of the Peanut-allergic Population
NOAEL threshold
160 mg
Population Sensitization Threshold in mg Peanut Protein Predicted to Provoke Reactions in 5% of the Peanut-allergic Population
LOAEL threshold
300 mg

PRIMARY outcome

Timeframe: 48-52 months

An estimate of the dose as assessed by the 48th month DBPCFC reported in mg peanut protein, also called population sensitization threshold, predicted to provoke reactions in 10% of the peanut-allergic population. This will also give population level no observed adverse event level (NOAEL) and lowest observed adverse event level (LOAEL) that would define interval of consecutive administered dose levels within which the population sensitization threshold lies. NOAEL is the highest dose observed not to produce any adverse effect and LOAEL is the lowest dose that is observed to produce an adverse effect.

Outcome measures

Outcome measures
Measure
Peanut (Liquid Peanut Extract) SLIT
n=54 Participants
All subjects will receive peanut SLIT upon enrollment for at least 48 months and then undergo a desensitization DBPCFC. Subjects will then be randomized to be off treatment for a period between 1 to 17 weeks. Subjects will then undergo a final DBPCFC. Liquid peanut extract (Peanut SLIT): Liquid peanut extract will be administered under the tongue
Non-desensitized After Peanut SLIT
All subjects will receive peanut SLIT upon enrollment for at least 48 months and then undergo a desensitization DBPCFC. 'Non-desensitized' defined as experiencing dose limiting symptoms during the 5000mg desensitization DBPCFC.
Population Sensitization Threshold in mg Peanut Protein Predicted to Provoke Reactions in 10% of the Peanut-allergic Population
NOAEL threshold
800 mg
Population Sensitization Threshold in mg Peanut Protein Predicted to Provoke Reactions in 10% of the Peanut-allergic Population
LOAEL threshold
750 mg

PRIMARY outcome

Timeframe: 52 months

A population estimate of time to loss of desensitization will be calculated for a subject's true sensitivity threshold (LOAEL) to reduce by half, also called half-life of sensitivity threshold. This is calculated based on the Kaplan-Meier estimator of the survival function using study participant data.

Outcome measures

Outcome measures
Measure
Peanut (Liquid Peanut Extract) SLIT
n=54 Participants
All subjects will receive peanut SLIT upon enrollment for at least 48 months and then undergo a desensitization DBPCFC. Subjects will then be randomized to be off treatment for a period between 1 to 17 weeks. Subjects will then undergo a final DBPCFC. Liquid peanut extract (Peanut SLIT): Liquid peanut extract will be administered under the tongue
Non-desensitized After Peanut SLIT
All subjects will receive peanut SLIT upon enrollment for at least 48 months and then undergo a desensitization DBPCFC. 'Non-desensitized' defined as experiencing dose limiting symptoms during the 5000mg desensitization DBPCFC.
Population Estimate of Time for a Subject's True Sensitivity Threshold to Reduce by Half.
17 weeks

PRIMARY outcome

Timeframe: 52 months

A population estimate of time to loss of desensitization will be calculated for a subject's true sensitivity threshold (LOAEL) to maintain at the same dose level during the double-blind, placebo-controlled food challenge. Food challenge dose levels are as follows: 100 mg, 200 mg, 500 mg, 800 mg, 1300 mg, 2100 mg. This is calculated based on the Kaplan-Meier estimator of the survival function using study participant data.

Outcome measures

Outcome measures
Measure
Peanut (Liquid Peanut Extract) SLIT
n=54 Participants
All subjects will receive peanut SLIT upon enrollment for at least 48 months and then undergo a desensitization DBPCFC. Subjects will then be randomized to be off treatment for a period between 1 to 17 weeks. Subjects will then undergo a final DBPCFC. Liquid peanut extract (Peanut SLIT): Liquid peanut extract will be administered under the tongue
Non-desensitized After Peanut SLIT
All subjects will receive peanut SLIT upon enrollment for at least 48 months and then undergo a desensitization DBPCFC. 'Non-desensitized' defined as experiencing dose limiting symptoms during the 5000mg desensitization DBPCFC.
Population Estimate of a Subject's True Sensitivity Threshold to Maintain at the Same Dose Level During DBPCFC
13 weeks

PRIMARY outcome

Timeframe: 52 months

A population estimate of time to loss of desensitization will be calculated for a subject's true sensitivity threshold (LOAEL) to decrease by one dose level during the double-blind, placebo-controlled food challenge. Food challenge dose levels are as follows: 100 mg, 200 mg, 500 mg, 800 mg, 1300 mg, 2100 mg. This is calculated based on the Kaplan-Meier estimator of the survival function using study participant data.

Outcome measures

Outcome measures
Measure
Peanut (Liquid Peanut Extract) SLIT
n=54 Participants
All subjects will receive peanut SLIT upon enrollment for at least 48 months and then undergo a desensitization DBPCFC. Subjects will then be randomized to be off treatment for a period between 1 to 17 weeks. Subjects will then undergo a final DBPCFC. Liquid peanut extract (Peanut SLIT): Liquid peanut extract will be administered under the tongue
Non-desensitized After Peanut SLIT
All subjects will receive peanut SLIT upon enrollment for at least 48 months and then undergo a desensitization DBPCFC. 'Non-desensitized' defined as experiencing dose limiting symptoms during the 5000mg desensitization DBPCFC.
Population Estimate of a Subject's True Sensitivity Threshold to Decrease by One Dose Level of During the DBPCFC
14 weeks

SECONDARY outcome

Timeframe: 48 months

Number of participants with adverse events (AEs) and serious adverse events (SAEs) during the SLIT treatment portion of the study.

Outcome measures

Outcome measures
Measure
Peanut (Liquid Peanut Extract) SLIT
n=54 Participants
All subjects will receive peanut SLIT upon enrollment for at least 48 months and then undergo a desensitization DBPCFC. Subjects will then be randomized to be off treatment for a period between 1 to 17 weeks. Subjects will then undergo a final DBPCFC. Liquid peanut extract (Peanut SLIT): Liquid peanut extract will be administered under the tongue
Non-desensitized After Peanut SLIT
All subjects will receive peanut SLIT upon enrollment for at least 48 months and then undergo a desensitization DBPCFC. 'Non-desensitized' defined as experiencing dose limiting symptoms during the 5000mg desensitization DBPCFC.
Number of Participants With Adverse Events and Serious Adverse Events During the Study.
Adverse events
53 Participants
Number of Participants With Adverse Events and Serious Adverse Events During the Study.
Serious adverse events
0 Participants

SECONDARY outcome

Timeframe: 48 months

With published concerns for the development of eosinophilic gastrointestinal disease after food immunotherapy, will report the number of participants with gastrointestinal and possible gastrointestinal eosinophilic adverse events during SLIT therapy

Outcome measures

Outcome measures
Measure
Peanut (Liquid Peanut Extract) SLIT
n=54 Participants
All subjects will receive peanut SLIT upon enrollment for at least 48 months and then undergo a desensitization DBPCFC. Subjects will then be randomized to be off treatment for a period between 1 to 17 weeks. Subjects will then undergo a final DBPCFC. Liquid peanut extract (Peanut SLIT): Liquid peanut extract will be administered under the tongue
Non-desensitized After Peanut SLIT
All subjects will receive peanut SLIT upon enrollment for at least 48 months and then undergo a desensitization DBPCFC. 'Non-desensitized' defined as experiencing dose limiting symptoms during the 5000mg desensitization DBPCFC.
Number of Participants With Gastrointestinal and Possible Gastrointestinal Eosinophilic Adverse Events.
Gastrointestinal adverse events
16 Participants
Number of Participants With Gastrointestinal and Possible Gastrointestinal Eosinophilic Adverse Events.
Possible eosinophilic gastrointestinal AEs
2 Participants

SECONDARY outcome

Timeframe: 48 months

Comparative estimates of changes in immune parameters (wheal size in mm during peanut skin prick test) from baseline through end of treatment among subjects who were induced with clinical desensitization (ingesting 5000mg on DBPCFC without symptoms) versus those who failed (developing clinical symptoms after ingesting 5000mg on DBPCFC).

Outcome measures

Outcome measures
Measure
Peanut (Liquid Peanut Extract) SLIT
n=17 Participants
All subjects will receive peanut SLIT upon enrollment for at least 48 months and then undergo a desensitization DBPCFC. Subjects will then be randomized to be off treatment for a period between 1 to 17 weeks. Subjects will then undergo a final DBPCFC. Liquid peanut extract (Peanut SLIT): Liquid peanut extract will be administered under the tongue
Non-desensitized After Peanut SLIT
n=37 Participants
All subjects will receive peanut SLIT upon enrollment for at least 48 months and then undergo a desensitization DBPCFC. 'Non-desensitized' defined as experiencing dose limiting symptoms during the 5000mg desensitization DBPCFC.
Change in Peanut Skin Test Wheal Over Time Among Subjects Who Were Induced With Clinical Desensitization Versus Those Who Failed
Baseline wheal size
14.5 mm
Interval 2.0 to 27.0
16.25 mm
Interval 5.5 to 36.5
Change in Peanut Skin Test Wheal Over Time Among Subjects Who Were Induced With Clinical Desensitization Versus Those Who Failed
48 month wheal size
7.25 mm
Interval 1.0 to 14.0
9.25 mm
Interval 2.0 to 25.5

SECONDARY outcome

Timeframe: 48 months

Comparative estimates of changes in immune parameters (serum levels of peanut specific IgE in kU/L) from baseline through end of treatment among subjects who were induced with clinical desensitization (ingesting 5000mg on DBPCFC without symptoms) versus those who failed (developing clinical symptoms after ingesting 5000mg on DBPCFC).

Outcome measures

Outcome measures
Measure
Peanut (Liquid Peanut Extract) SLIT
n=17 Participants
All subjects will receive peanut SLIT upon enrollment for at least 48 months and then undergo a desensitization DBPCFC. Subjects will then be randomized to be off treatment for a period between 1 to 17 weeks. Subjects will then undergo a final DBPCFC. Liquid peanut extract (Peanut SLIT): Liquid peanut extract will be administered under the tongue
Non-desensitized After Peanut SLIT
n=37 Participants
All subjects will receive peanut SLIT upon enrollment for at least 48 months and then undergo a desensitization DBPCFC. 'Non-desensitized' defined as experiencing dose limiting symptoms during the 5000mg desensitization DBPCFC.
Change in Peanut IgE Over Time Among Subjects Who Were Induced With Clinical Desensitization Versus Those Who Failed
Baseline peanut IgE
107 kU/L
Interval 0.3 to 1139.4
84.7 kU/L
Interval 10.9 to 1804.0
Change in Peanut IgE Over Time Among Subjects Who Were Induced With Clinical Desensitization Versus Those Who Failed
48 month peanut IgE
12.1 kU/L
Interval 0.05 to 484.0
11.2 kU/L
Interval 1.4 to 970.0

SECONDARY outcome

Timeframe: 48 months

Comparative estimates of changes in immune parameters (serum levels of peanut-specific IgG4 in mg/L) from baseline through end of treatment among subjects who were induced with clinical desensitization (ingesting 5000mg on DBPCFC without symptoms) versus those who failed (developing clinical symptoms after ingesting 5000mg on DBPCFC).

Outcome measures

Outcome measures
Measure
Peanut (Liquid Peanut Extract) SLIT
n=17 Participants
All subjects will receive peanut SLIT upon enrollment for at least 48 months and then undergo a desensitization DBPCFC. Subjects will then be randomized to be off treatment for a period between 1 to 17 weeks. Subjects will then undergo a final DBPCFC. Liquid peanut extract (Peanut SLIT): Liquid peanut extract will be administered under the tongue
Non-desensitized After Peanut SLIT
n=37 Participants
All subjects will receive peanut SLIT upon enrollment for at least 48 months and then undergo a desensitization DBPCFC. 'Non-desensitized' defined as experiencing dose limiting symptoms during the 5000mg desensitization DBPCFC.
Change in Peanut IgG4 Over Time Among Subjects Who Were Induced With Clinical Desensitization Versus Those Who Failed
Baseline peanut IgG4
0.47 mg/L
Interval 0.02 to 6.38
0.37 mg/L
Interval 0.02 to 19.65
Change in Peanut IgG4 Over Time Among Subjects Who Were Induced With Clinical Desensitization Versus Those Who Failed
48 month peanut IgG4
9.69 mg/L
Interval 0.04 to 80.0
4.39 mg/L
Interval 0.05 to 57.4

Adverse Events

Peanut (Liquid Peanut Extract) SLIT

Serious events: 0 serious events
Other events: 53 other events
Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Other adverse events
Measure
Peanut (Liquid Peanut Extract) SLIT
n=54 participants at risk
All subjects will receive peanut SLIT upon enrollment for at least 48 months and then undergo a desensitization DBPCFC. Subjects will then be randomized to be off treatment for a period between 1 to 17 weeks. Subjects will then undergo a final DBPCFC. Liquid peanut extract (Peanut SLIT): Liquid peanut extract will be administered under the tongue
Gastrointestinal disorders
Vomiting
14.8%
8/54 • Adverse event data collected over 48 month peanut SLIT treatment period.
Gastrointestinal disorders
Diarrhea
5.6%
3/54 • Adverse event data collected over 48 month peanut SLIT treatment period.
Skin and subcutaneous tissue disorders
Rash
7.4%
4/54 • Adverse event data collected over 48 month peanut SLIT treatment period.
Respiratory, thoracic and mediastinal disorders
Runny nose
1.9%
1/54 • Adverse event data collected over 48 month peanut SLIT treatment period.
Respiratory, thoracic and mediastinal disorders
Wheezing
3.7%
2/54 • Adverse event data collected over 48 month peanut SLIT treatment period.
Skin and subcutaneous tissue disorders
Oropharyngeal itch
66.7%
36/54 • Adverse event data collected over 48 month peanut SLIT treatment period.
Respiratory, thoracic and mediastinal disorders
Sneezing
5.6%
3/54 • Adverse event data collected over 48 month peanut SLIT treatment period.
Respiratory, thoracic and mediastinal disorders
Coughing
5.6%
3/54 • Adverse event data collected over 48 month peanut SLIT treatment period.
Skin and subcutaneous tissue disorders
Itchy nose
1.9%
1/54 • Adverse event data collected over 48 month peanut SLIT treatment period.
Skin and subcutaneous tissue disorders
Skin itch
25.9%
14/54 • Adverse event data collected over 48 month peanut SLIT treatment period.
Skin and subcutaneous tissue disorders
Lip swelling
27.8%
15/54 • Adverse event data collected over 48 month peanut SLIT treatment period.
Skin and subcutaneous tissue disorders
Eye swelling
3.7%
2/54 • Adverse event data collected over 48 month peanut SLIT treatment period.
Skin and subcutaneous tissue disorders
Eye itch
3.7%
2/54 • Adverse event data collected over 48 month peanut SLIT treatment period.
Skin and subcutaneous tissue disorders
Eye tearing
3.7%
2/54 • Adverse event data collected over 48 month peanut SLIT treatment period.
Skin and subcutaneous tissue disorders
Hives
18.5%
10/54 • Adverse event data collected over 48 month peanut SLIT treatment period.
Gastrointestinal disorders
Belly pain
29.6%
16/54 • Adverse event data collected over 48 month peanut SLIT treatment period.
Skin and subcutaneous tissue disorders
Nasal congestion
1.9%
1/54 • Adverse event data collected over 48 month peanut SLIT treatment period.

Additional Information

Edwin Kim, MD; Director UNC Food Allergy Initiative

University of North Carolina at Chapel Hill

Phone: 919-537-3193

Results disclosure agreements

  • Principal investigator is a sponsor employee
  • Publication restrictions are in place