Trial Outcomes & Findings for Baked Egg or Egg Oral Immunotherapy for Children With Egg Allergy (NCT NCT01846208)

Last Updated: 2019-07-26

Results Overview

Sustained unresponsiveness - able to successfully consume 7444 mg egg white protein in a desensitization OFC and, after an 8-10 week egg-free interval, were also able to successfully consume 7444 mg egg white protein in an OFC after up to 2 years of therapy.

Recruitment status

COMPLETED

Study phase

PHASE2

Target enrollment

92 participants

Primary outcome timeframe

2 Years

Results posted on

2019-07-26

Participant Flow

Study recruitment began in July 2013 and accrual closed in August 2015.

Participant milestones

Participant milestones
Measure	Baked Egg Randomized Subjects who passed a baked egg oral food challenge (OFC) at baseline were randomized to receive baked egg in the form of home-baked goods and "safe" commercial products with up to four oral food challenges as directed by the protocol. Baked Egg: Predetermined food substances with known amounts of Baked Egg (egg protein) with standardized dosing/consumption instructions.	Egg OIT Randomized Subjects who passed a baked egg oral food challenge (OFC) at baseline were randomized to receive egg oral immunotherapy (OIT) in the form of egg white solid with up to four oral food challenges as directed by protocol. Egg Oral Immunotherapy: Commercially available egg white solid dispensed by the central manufacturer. Study product will be dispensed in vials for low doses, capsules for mid-range doses, and bulk powder with dosing scoops for the higher doses.	Egg OIT Assigned Subjects who failed a baked egg oral food challenge (OFC) at baseline were assigned to receive egg oral immunotherapy (OIT) in the form of egg white solid with up to four oral food challenges as directed by protocol. Egg Oral Immunotherapy: Commercially available egg white solid dispensed by the central manufacturer. Study product will be dispensed in vials for low doses, capsules for mid-range doses, and bulk powder with dosing scoops for the higher doses.
Overall Study STARTED	28	24	40
Overall Study Initiatiated Treatment	27	23	39
Overall Study Year 1 Desensitization OFC	23	20	34
Overall Study Year 2 Desensitization OFC	19	20	33
Overall Study Year 2 Sustained Unresponsiveness OFC	4	18	21
Overall Study COMPLETED	19	20	32
Overall Study NOT COMPLETED	9	4	8

Reasons for withdrawal

Reasons for withdrawal
Measure	Baked Egg Randomized Subjects who passed a baked egg oral food challenge (OFC) at baseline were randomized to receive baked egg in the form of home-baked goods and "safe" commercial products with up to four oral food challenges as directed by the protocol. Baked Egg: Predetermined food substances with known amounts of Baked Egg (egg protein) with standardized dosing/consumption instructions.	Egg OIT Randomized Subjects who passed a baked egg oral food challenge (OFC) at baseline were randomized to receive egg oral immunotherapy (OIT) in the form of egg white solid with up to four oral food challenges as directed by protocol. Egg Oral Immunotherapy: Commercially available egg white solid dispensed by the central manufacturer. Study product will be dispensed in vials for low doses, capsules for mid-range doses, and bulk powder with dosing scoops for the higher doses.	Egg OIT Assigned Subjects who failed a baked egg oral food challenge (OFC) at baseline were assigned to receive egg oral immunotherapy (OIT) in the form of egg white solid with up to four oral food challenges as directed by protocol. Egg Oral Immunotherapy: Commercially available egg white solid dispensed by the central manufacturer. Study product will be dispensed in vials for low doses, capsules for mid-range doses, and bulk powder with dosing scoops for the higher doses.
Overall Study Withdrew prior to treatment	1	1	1
Overall Study Dosing Symptoms	1	2	6
Overall Study Withdrawal by Subject	5	0	0
Overall Study Non-compliance	1	1	0
Overall Study Developed Eosinophilic Esophagitis (EoE)	1	0	0
Overall Study Refused SU OFC	0	0	1

Baseline Characteristics

One participant in Egg OIT Assigned arm had no plasma

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Baked Egg Randomized n=27 Participants Subjects who passed a baked egg oral food challenge (OFC) at baseline were randomized to receive baked egg in the form of home-baked goods and "safe" commercial products with up to four oral food challenges as directed by the protocol. Baked Egg: Predetermined food substances with known amounts of Baked Egg (egg protein) with standardized dosing/consumption instructions.	Egg OIT Randomized n=23 Participants Subjects who passed a baked egg oral food challenge (OFC) at baseline were randomized to receive egg oral immunotherapy (OIT) in the form of egg white solid with up to four oral food challenges as directed by protocol. Egg Oral Immunotherapy: Commercially available egg white solid dispensed by the central manufacturer. Study product will be dispensed in vials for low doses, capsules for mid-range doses, and bulk powder with dosing scoops for the higher doses.	Egg OIT Assigned n=39 Participants Subjects who failed a baked egg oral food challenge (OFC) at baseline were assigned to receive egg oral immunotherapy (OIT) in the form of egg white solid with up to four oral food challenges as directed by protocol. Egg Oral Immunotherapy: Commercially available egg white solid dispensed by the central manufacturer. Study product will be dispensed in vials for low doses, capsules for mid-range doses, and bulk powder with dosing scoops for the higher doses.	Total n=89 Participants Total of all reporting groups
Age, Continuous	7.2 years STANDARD_DEVIATION 3.0 • n=27 Participants	9.1 years STANDARD_DEVIATION 3.1 • n=23 Participants	8.8 years STANDARD_DEVIATION 2.4 • n=39 Participants	8.4 years STANDARD_DEVIATION 2.8 • n=89 Participants
Sex: Female, Male Female	12 Participants n=27 Participants	6 Participants n=23 Participants	14 Participants n=39 Participants	32 Participants n=89 Participants
Sex: Female, Male Male	15 Participants n=27 Participants	17 Participants n=23 Participants	25 Participants n=39 Participants	57 Participants n=89 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=27 Participants	0 Participants n=23 Participants	0 Participants n=39 Participants	0 Participants n=89 Participants
Race (NIH/OMB) Asian	2 Participants n=27 Participants	2 Participants n=23 Participants	4 Participants n=39 Participants	8 Participants n=89 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=27 Participants	0 Participants n=23 Participants	0 Participants n=39 Participants	0 Participants n=89 Participants
Race (NIH/OMB) Black or African American	1 Participants n=27 Participants	2 Participants n=23 Participants	0 Participants n=39 Participants	3 Participants n=89 Participants
Race (NIH/OMB) White	23 Participants n=27 Participants	18 Participants n=23 Participants	31 Participants n=39 Participants	72 Participants n=89 Participants
Race (NIH/OMB) More than one race	1 Participants n=27 Participants	1 Participants n=23 Participants	4 Participants n=39 Participants	6 Participants n=89 Participants
Race (NIH/OMB) Unknown or Not Reported	0 Participants n=27 Participants	0 Participants n=23 Participants	0 Participants n=39 Participants	0 Participants n=89 Participants
Race/Ethnicity, Customized Non-Hispanic or Non-Latino origin	25 Participants n=27 Participants	23 Participants n=23 Participants	39 Participants n=39 Participants	87 Participants n=89 Participants
Race/Ethnicity, Customized Hispanic or Latino origin	2 Participants n=27 Participants	0 Participants n=23 Participants	0 Participants n=39 Participants	2 Participants n=89 Participants
Other Food Allergy History No/Unknown	1 Participants n=27 Participants	3 Participants n=23 Participants	8 Participants n=39 Participants	12 Participants n=89 Participants
Other Food Allergy History Yes	26 Participants n=27 Participants	20 Participants n=23 Participants	31 Participants n=39 Participants	77 Participants n=89 Participants
Asthma Severity History No History	9 Participants n=27 Participants	10 Participants n=23 Participants	13 Participants n=39 Participants	32 Participants n=89 Participants
Asthma Severity History Mild intermittent	16 Participants n=27 Participants	10 Participants n=23 Participants	24 Participants n=39 Participants	50 Participants n=89 Participants
Asthma Severity History Mild persistent	1 Participants n=27 Participants	2 Participants n=23 Participants	2 Participants n=39 Participants	5 Participants n=89 Participants
Asthma Severity History Moderate persistent	1 Participants n=27 Participants	1 Participants n=23 Participants	0 Participants n=39 Participants	2 Participants n=89 Participants
Allergic GI Disease	0 Participants n=27 Participants	0 Participants n=23 Participants	0 Participants n=39 Participants	0 Participants n=89 Participants
Allergic Rhinitis	24 Participants n=27 Participants	18 Participants n=23 Participants	31 Participants n=39 Participants	73 Participants n=89 Participants
Atopic Dermatitis History	18 Participants n=27 Participants	13 Participants n=23 Participants	24 Participants n=39 Participants	55 Participants n=89 Participants
Atopic Dermatitis Score	0.0 units on a scale n=27 Participants	0.0 units on a scale n=23 Participants	0.0 units on a scale n=39 Participants	0.0 units on a scale n=89 Participants
Baseline Egg IgE	9.77 kUA/L n=27 Participants • One participant in Egg OIT Assigned arm had no plasma	12.30 kUA/L n=23 Participants • One participant in Egg OIT Assigned arm had no plasma	26.25 kUA/L n=38 Participants • One participant in Egg OIT Assigned arm had no plasma	15.60 kUA/L n=88 Participants • One participant in Egg OIT Assigned arm had no plasma
Baseline Total IgE	809.00 kUA/L n=27 Participants • One participant in Egg OIT Assigned arm had no plasma	873.00 kUA/L n=23 Participants • One participant in Egg OIT Assigned arm had no plasma	960.00 kUA/L n=38 Participants • One participant in Egg OIT Assigned arm had no plasma	885.50 kUA/L n=88 Participants • One participant in Egg OIT Assigned arm had no plasma
Baseline Egg IgG4	0.84 mgA/L n=27 Participants • One participant in Egg OIT Assigned arm had no plasma	0.81 mgA/L n=23 Participants • One participant in Egg OIT Assigned arm had no plasma	0.28 mgA/L n=38 Participants • One participant in Egg OIT Assigned arm had no plasma	0.64 mgA/L n=88 Participants • One participant in Egg OIT Assigned arm had no plasma
Baseline Egg %IgE	1.86 % IgE n=27 Participants • One participant in Egg OIT Assigned arm had no plasma	1.80 % IgE n=23 Participants • One participant in Egg OIT Assigned arm had no plasma	3.06 % IgE n=38 Participants • One participant in Egg OIT Assigned arm had no plasma	2.13 % IgE n=88 Participants • One participant in Egg OIT Assigned arm had no plasma
Baseline Ratio IgG4/IgE	24.94 ratio n=27 Participants • One participant in Egg OIT Assigned arm had no plasma	25.75 ratio n=23 Participants • One participant in Egg OIT Assigned arm had no plasma	5.01 ratio n=38 Participants • One participant in Egg OIT Assigned arm had no plasma	11.18 ratio n=88 Participants • One participant in Egg OIT Assigned arm had no plasma
Baseline Ovalbumin IgE	5.53 kUA/L n=27 Participants • One participant in Egg OIT Assigned arm had no plasma	5.85 kUA/L n=23 Participants • One participant in Egg OIT Assigned arm had no plasma	16.10 kUA/L n=38 Participants • One participant in Egg OIT Assigned arm had no plasma	8.88 kUA/L n=88 Participants • One participant in Egg OIT Assigned arm had no plasma
Baseline Ovomucoid IgE	6.94 kUA/L n=27 Participants • One participant in Egg OIT Assigned arm had no plasma	6.93 kUA/L n=23 Participants • One participant in Egg OIT Assigned arm had no plasma	16.40 kUA/L n=38 Participants • One participant in Egg OIT Assigned arm had no plasma	9.76 kUA/L n=88 Participants • One participant in Egg OIT Assigned arm had no plasma
Baseline Ovalbumin IgG4	0.64 mgA/L n=27 Participants • One participant in Egg OIT Assigned arm had no plasma	0.53 mgA/L n=23 Participants • One participant in Egg OIT Assigned arm had no plasma	0.33 mgA/L n=38 Participants • One participant in Egg OIT Assigned arm had no plasma	0.49 mgA/L n=88 Participants • One participant in Egg OIT Assigned arm had no plasma
Baseline Ovomucoid IgG4	0.25 mgA/L n=27 Participants • One participant in Egg OIT Assigned arm had no plasma	0.15 mgA/L n=23 Participants • One participant in Egg OIT Assigned arm had no plasma	0.09 mgA/L n=38 Participants • One participant in Egg OIT Assigned arm had no plasma	0.11 mgA/L n=88 Participants • One participant in Egg OIT Assigned arm had no plasma
Baseline Egg Skin Prick Test (SPT)	7.50 mms n=27 Participants	11.00 mms n=23 Participants	12.00 mms n=39 Participants	11.00 mms n=89 Participants
Baseline Milk SPT	1.00 mms n=27 Participants	3.00 mms n=23 Participants	3.50 mms n=39 Participants	2.50 mms n=89 Participants
Baseline Peanut SPT	10.50 mms n=27 Participants • One Egg OIT Assigned subject refused to complete the peanut SPT at baseline	11.00 mms n=23 Participants • One Egg OIT Assigned subject refused to complete the peanut SPT at baseline	10.50 mms n=38 Participants • One Egg OIT Assigned subject refused to complete the peanut SPT at baseline	11.00 mms n=88 Participants • One Egg OIT Assigned subject refused to complete the peanut SPT at baseline
Baseline Tree SPT	2.50 mms n=27 Participants	4.50 mms n=23 Participants	3.00 mms n=39 Participants	3.50 mms n=89 Participants
Baseline Grass SPT	4.50 mms n=27 Participants	0.00 mms n=23 Participants	2.50 mms n=39 Participants	2.50 mms n=89 Participants
Baseline Weed SPT	1.50 mms n=27 Participants	0.00 mms n=23 Participants	0.00 mms n=39 Participants	0.00 mms n=89 Participants
Baseline Mold SPT	0.50 mms n=27 Participants	1.00 mms n=23 Participants	0.00 mms n=39 Participants	0.00 mms n=89 Participants
Baseline Cockroach SPT	0.00 mms n=27 Participants	0.00 mms n=23 Participants	0.00 mms n=39 Participants	0.00 mms n=89 Participants

PRIMARY outcome

Timeframe: 2 Years

Population: All treated participants were included in the analysis.

Outcome measures

Outcome measures
Measure	Baked Egg Randomized n=27 Participants Subjects who passed a baked egg oral food challenge (OFC) at baseline were randomized to receive baked egg in the form of home-baked goods and "safe" commercial products with up to four oral food challenges as directed by the protocol. Baked Egg: Predetermined food substances with known amounts of Baked Egg (egg protein) with standardized dosing/consumption instructions.	Egg OIT Randomized n=23 Participants Subjects who passed a baked egg oral food challenge (OFC) at baseline were randomized to receive egg oral immunotherapy (OIT) in the form of egg white solid with up to four oral food challenges as directed by protocol. Egg Oral Immunotherapy: Commercially available egg white solid dispensed by the central manufacturer. Study product will be dispensed in vials for low doses, capsules for mid-range doses, and bulk powder with dosing scoops for the higher doses.	Egg OIT Assigned n=39 Participants Subjects who failed a baked egg oral food challenge (OFC) at baseline were assigned to receive egg oral immunotherapy (OIT) in the form of egg white solid with up to four oral food challenges as directed by protocol. Egg Oral Immunotherapy: Commercially available egg white solid dispensed by the central manufacturer. Study product will be dispensed in vials for low doses, capsules for mid-range doses, and bulk powder with dosing scoops for the higher doses.
Sustained Unresponsiveness to Egg Consumption at 2 Years.	3 Participants	10 Participants	7 Participants

SECONDARY outcome

Timeframe: 1 Year and 2 Years

Development of desensitization to able to successfully consume \>=4444 mg egg white protein during a desensitization OFC on therapy at 1 year and 2 years.

Outcome measures

Outcome measures
Measure	Baked Egg Randomized n=27 Participants Subjects who passed a baked egg oral food challenge (OFC) at baseline were randomized to receive baked egg in the form of home-baked goods and "safe" commercial products with up to four oral food challenges as directed by the protocol. Baked Egg: Predetermined food substances with known amounts of Baked Egg (egg protein) with standardized dosing/consumption instructions.	Egg OIT Randomized n=23 Participants Subjects who passed a baked egg oral food challenge (OFC) at baseline were randomized to receive egg oral immunotherapy (OIT) in the form of egg white solid with up to four oral food challenges as directed by protocol. Egg Oral Immunotherapy: Commercially available egg white solid dispensed by the central manufacturer. Study product will be dispensed in vials for low doses, capsules for mid-range doses, and bulk powder with dosing scoops for the higher doses.	Egg OIT Assigned n=39 Participants Subjects who failed a baked egg oral food challenge (OFC) at baseline were assigned to receive egg oral immunotherapy (OIT) in the form of egg white solid with up to four oral food challenges as directed by protocol. Egg Oral Immunotherapy: Commercially available egg white solid dispensed by the central manufacturer. Study product will be dispensed in vials for low doses, capsules for mid-range doses, and bulk powder with dosing scoops for the higher doses.
Desensitization to >= 4.444 Grams Egg White Solid. Desensitized Success at Year 1	8 Participants	17 Participants	22 Participants
Desensitization to >= 4.444 Grams Egg White Solid. Desensitized Success at Year 2	6 Participants	20 Participants	27 Participants

SECONDARY outcome

Timeframe: up to 3 years

Population: All treated participants

Incidence of all serious adverse events during the study. No statistical analyses were performed since there were no events in 2 of the 3 treatment groups and only 1 event in the third so it would not be meaningful.

Outcome measures

Outcome measures
Measure	Baked Egg Randomized n=27 Participants Subjects who passed a baked egg oral food challenge (OFC) at baseline were randomized to receive baked egg in the form of home-baked goods and "safe" commercial products with up to four oral food challenges as directed by the protocol. Baked Egg: Predetermined food substances with known amounts of Baked Egg (egg protein) with standardized dosing/consumption instructions.	Egg OIT Randomized n=23 Participants Subjects who passed a baked egg oral food challenge (OFC) at baseline were randomized to receive egg oral immunotherapy (OIT) in the form of egg white solid with up to four oral food challenges as directed by protocol. Egg Oral Immunotherapy: Commercially available egg white solid dispensed by the central manufacturer. Study product will be dispensed in vials for low doses, capsules for mid-range doses, and bulk powder with dosing scoops for the higher doses.	Egg OIT Assigned n=39 Participants Subjects who failed a baked egg oral food challenge (OFC) at baseline were assigned to receive egg oral immunotherapy (OIT) in the form of egg white solid with up to four oral food challenges as directed by protocol. Egg Oral Immunotherapy: Commercially available egg white solid dispensed by the central manufacturer. Study product will be dispensed in vials for low doses, capsules for mid-range doses, and bulk powder with dosing scoops for the higher doses.
Incidence of All Serious Adverse Events	0 Participants	0 Participants	1 Participants

SECONDARY outcome

Timeframe: 3 years after randomization

Population: Participants who completed the long term follow-up questionnaire 3 years after randomization are included.

Number of participants who reported consumption of concentrated (unbaked) egg in their diet on the long-term follow-up questionnaire 3 years after randomization, indicating unrestricted consumption of unbaked egg. This is a qualitative questionnaire asking participants about egg in their diet, symptoms, and what treatment they received for their allergic reactions.

Outcome measures

Outcome measures
Measure	Baked Egg Randomized n=17 Participants Subjects who passed a baked egg oral food challenge (OFC) at baseline were randomized to receive baked egg in the form of home-baked goods and "safe" commercial products with up to four oral food challenges as directed by the protocol. Baked Egg: Predetermined food substances with known amounts of Baked Egg (egg protein) with standardized dosing/consumption instructions.	Egg OIT Randomized n=19 Participants Subjects who passed a baked egg oral food challenge (OFC) at baseline were randomized to receive egg oral immunotherapy (OIT) in the form of egg white solid with up to four oral food challenges as directed by protocol. Egg Oral Immunotherapy: Commercially available egg white solid dispensed by the central manufacturer. Study product will be dispensed in vials for low doses, capsules for mid-range doses, and bulk powder with dosing scoops for the higher doses.	Egg OIT Assigned n=30 Participants Subjects who failed a baked egg oral food challenge (OFC) at baseline were assigned to receive egg oral immunotherapy (OIT) in the form of egg white solid with up to four oral food challenges as directed by protocol. Egg Oral Immunotherapy: Commercially available egg white solid dispensed by the central manufacturer. Study product will be dispensed in vials for low doses, capsules for mid-range doses, and bulk powder with dosing scoops for the higher doses.
Number of Participants With Unrestricted Consumption of Unbaked Egg	4 Participants	14 Participants	12 Participants

OTHER_PRE_SPECIFIED outcome

Timeframe: 2 Years

Population: Data not reported because these are for tertiary objectives that are exploratory.

Changes in egg-specific IgE and IgG4, changes in SPT mean wheal diameters, basophil reactivity, Th2 and Treg values.

Outcome measures

Outcome data not reported

Adverse Events

Baked Egg Randomized

Serious events: 0 serious events

Other events: 27 other events

Deaths: 0 deaths

Egg OIT Randomized

Serious events: 0 serious events

Other events: 23 other events

Deaths: 0 deaths

Egg OIT Assigned

Serious events: 1 serious events

Other events: 39 other events

Deaths: 0 deaths

Serious adverse events

Serious adverse events
Measure	Baked Egg Randomized n=27 participants at risk Subjects who passed a baked egg oral food challenge (OFC) at baseline were randomized to receive baked egg in the form of home-baked goods and "safe" commercial products with up to four oral food challenges as directed by the protocol. Baked Egg: Predetermined food substances with known amounts of Baked Egg (egg protein) with standardized dosing/consumption instructions.	Egg OIT Randomized n=23 participants at risk Subjects who passed a baked egg oral food challenge (OFC) at baseline were randomized to receive egg oral immunotherapy (OIT) in the form of egg white solid with up to four oral food challenges as directed by protocol. Egg Oral Immunotherapy: Commercially available egg white solid dispensed by the central manufacturer. Study product will be dispensed in vials for low doses, capsules for mid-range doses, and bulk powder with dosing scoops for the higher doses.	Egg OIT Assigned n=39 participants at risk Subjects who failed a baked egg oral food challenge (OFC) at baseline were assigned to receive egg oral immunotherapy (OIT) in the form of egg white solid with up to four oral food challenges as directed by protocol. Egg Oral Immunotherapy: Commercially available egg white solid dispensed by the central manufacturer. Study product will be dispensed in vials for low doses, capsules for mid-range doses, and bulk powder with dosing scoops for the higher doses.
Infections and infestations Pneumonia	0.00% 0/27 • up to 3 years, through last clinic visit. \* Unsolicited - collected by non-systematic assessment \+ Solicited (dosing symptoms from daily logs) - collected by systematic assessment	0.00% 0/23 • up to 3 years, through last clinic visit. \* Unsolicited - collected by non-systematic assessment \+ Solicited (dosing symptoms from daily logs) - collected by systematic assessment	2.6% 1/39 • up to 3 years, through last clinic visit. \* Unsolicited - collected by non-systematic assessment \+ Solicited (dosing symptoms from daily logs) - collected by systematic assessment

Other adverse events

Additional Information

Dr. Hugh A. Sampson

Icahn School of Medicine at Mount Sinai

Phone: 212-659-9426

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place