Hen Egg Allergy
Conditions
Keywords
CODIT™ (characterized oral desensitization immunotherapy™), Hen Egg Allergic Children, Desensitization, AR201, Allergy, Hen Egg Allergy, Characterized Hen Egg Allergen, Oral Immunotherapy, CHEA (Characterized Hen Egg Allergen), Food Allergy
Brief summary
To determine the efficacy and safety of AR201 in a characterized oral desensitization immunotherapy (CODIT™) regimen in hen egg-allergic subjects aged 4 to 26 years, inclusive.
Detailed description
This is a phase 2, randomized, double-blind, placebo-controlled study of the efficacy and safety of AR201 in a CODIT regimen in hen egg-allergic subjects aged 4 to 26 years, inclusive. The study was conducted at 8 sites in the United States.
Interventions
BIOLOGICALAR201 powder
AR201 powder (Hen Egg allergen formulation) provided in capsules
BIOLOGICALPlacebo powder
Placebo powder provided in capsules
Sponsors
Aimmune Therapeutics, Inc.
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
TRIPLE (Subject, Caregiver, Investigator)
Masking description
Double-Blinded
Intervention model description
2:1 randomization
Eligibility
Sex/Gender
ALL
Age
4 Years to 26 Years
Healthy volunteers
No
Inclusion criteria
Key Inclusion Criteria: * Age 4 to 26 years, inclusive * Written informed consent from subjects, as appropriate per local requirements, and parent/guardian * Written assent from subjects who are minors, as appropriate per local requirements * History of physician-diagnosed hen egg allergy * Elevated egg specific IgE and/or mean wheal diameter on SPT to egg * Experience dose-limiting symptoms at or before the 300 mg challenge dose of dried egg white protein on Screening DBPCFC * Use of effective birth control by sexually active female subjects of childbearing potential Key
Exclusion criteria
* History of severe or life-threatening anaphylaxis within 60 days before screening * History of non-IgE mediated hen egg allergy such as food protein-induced enterocolitis syndrome (FPIES) * History of eosinophilic esophagitis (EoE), other eosinophilic gastrointestinal disease, chronic, recurrent, or severe gastroesophageal reflux disease (GERD), symptoms of dysphagia or recurrent gastrointestinal symptoms of unknown etiology * History of a mast cell disorder including systemic mastocytosis, urticaria pigmentosa, chronic idiopathic or chronic physical urticaria beyond simple dermatographism (e.g., cold urticaria, cholinergic urticaria), and hereditary or idiopathic angioedema * History of chronic disease (except asthma, atopic dermatitis, or allergic rhinitis) which may require a change in immunosuppressive medication regimen or chronic corticosteroid use * History of cardiovascular disease, including uncontrolled or inadequately controlled hypertension * Developing dose-limiting symptoms to placebo challenge during the Screening DBPCFC * Having the same place of residence as another subject in the study * Any other condition that, in the opinion of the Investigator, precludes participation for reasons of safety
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Proportion of Subjects Tolerating a Single Highest Dose of at Least 1000 mg Dried Egg White Protein With no More Than Mild Symptoms at the Exit Double-blind Placebo Controlled Food Challenge (DBPCFC) | 9-12 months |
Secondary
| Measure | Time frame |
|---|---|
| Number of Participants by Maximum Dose Achieved With no or Mild Symptoms at Exit DBPCFC | 9-12 months |
Countries
United States
Participant flow
Participants by arm
| Arm | Count |
|---|---|
| AR201 Powder Subjects were randomized to active arm of AIME01 and administered AR201 in escalating doses for approximately 6 months, followed by a maintenance dose for approximately 12 weeks
AR201 powder: AR201 powder (Hen Egg allergen formulation) provided in capsules | 9 |
| Placebo Powder Subjects were randomized to placebo arm of AIME01 and administered placebo for approximately 6 months, followed by maintenance placebo for approximately 12 weeks.
Placebo powder: Placebo powder provided in capsules | 6 |
| Total | 15 |
Withdrawals & dropouts
| Period | Reason | FG000 | FG001 |
|---|---|---|---|
| Overall Study | Sponsor discontinued study | 3 | 3 |
Baseline characteristics
| Characteristic | AR201 Powder | Total | Placebo Powder |
|---|---|---|---|
| Age, Continuous | 13.0 years | 7.0 years | 5.5 years |
| Ethnicity (NIH/OMB) Hispanic or Latino | 1 Participants | 1 Participants | 0 Participants |
| Ethnicity (NIH/OMB) Not Hispanic or Latino | 8 Participants | 14 Participants | 6 Participants |
| Ethnicity (NIH/OMB) Unknown or Not Reported | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) American Indian or Alaska Native | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) Asian | 2 Participants | 2 Participants | 0 Participants |
| Race (NIH/OMB) Black or African American | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) More than one race | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) Native Hawaiian or Other Pacific Islander | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) Unknown or Not Reported | 0 Participants | 2 Participants | 2 Participants |
| Race (NIH/OMB) White | 7 Participants | 11 Participants | 4 Participants |
| Region of Enrollment United States | 9 participants | 15 participants | 6 participants |
| Sex: Female, Male Female | 5 Participants | 8 Participants | 3 Participants |
| Sex: Female, Male Male | 4 Participants | 7 Participants | 3 Participants |
Adverse events
| Event type | EG000 affected / at risk | EG001 affected / at risk |
|---|---|---|
| deaths Total, all-cause mortality | 0 / 9 | 0 / 6 |
| other Total, other adverse events | 9 / 9 | 5 / 6 |
| serious Total, serious adverse events | 1 / 9 | 0 / 6 |
Outcome results
Primary
Proportion of Subjects Tolerating a Single Highest Dose of at Least 1000 mg Dried Egg White Protein With no More Than Mild Symptoms at the Exit Double-blind Placebo Controlled Food Challenge (DBPCFC)
Time frame: 9-12 months
Population: The study was prematurely terminated due to difficulties in meeting enrollment goals due to the Coronavirus Disease 2019 (COVID-19) pandemic. As a consequence, all prespecified efficacy endpoint analysis were not performed, and the exit DBPCFC was optional and limited to subjects who completed at least 9 months of study treatment including at least 4 weeks at 300 mg/day, and tolerated the 300 mg/day dose for approximately 2 consecutive weeks before the exit DBPCFC.
| Arm | Measure | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|
| AR201 Powder | Proportion of Subjects Tolerating a Single Highest Dose of at Least 1000 mg Dried Egg White Protein With no More Than Mild Symptoms at the Exit Double-blind Placebo Controlled Food Challenge (DBPCFC) | 1 Participants |
| Placebo Powder | Proportion of Subjects Tolerating a Single Highest Dose of at Least 1000 mg Dried Egg White Protein With no More Than Mild Symptoms at the Exit Double-blind Placebo Controlled Food Challenge (DBPCFC) | 0 Participants |
Secondary
Number of Participants by Maximum Dose Achieved With no or Mild Symptoms at Exit DBPCFC
Time frame: 9-12 months
Population: The study was prematurely terminated due to difficulties in meeting enrollment goals due to the COVID-19 pandemic. As a consequence, all prespecified efficacy endpoint analysis were not performed, and the exit DBPCFC was optional and limited to subjects who completed at least 9 months of study treatment including at least 4 weeks at 300 mg/day, and tolerated the 300 mg/day dose for approximately 2 consecutive weeks before the exit DBPCFC.
| Arm | Measure | Category | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|---|
| AR201 Powder | Number of Participants by Maximum Dose Achieved With no or Mild Symptoms at Exit DBPCFC | 10 mg | 0 Participants |
| AR201 Powder | Number of Participants by Maximum Dose Achieved With no or Mild Symptoms at Exit DBPCFC | 300 mg | 2 Participants |
| AR201 Powder | Number of Participants by Maximum Dose Achieved With no or Mild Symptoms at Exit DBPCFC | 3 mg | 0 Participants |
| AR201 Powder | Number of Participants by Maximum Dose Achieved With no or Mild Symptoms at Exit DBPCFC | 600 mg | 0 Participants |
| AR201 Powder | Number of Participants by Maximum Dose Achieved With no or Mild Symptoms at Exit DBPCFC | 30 mg | 0 Participants |
| AR201 Powder | Number of Participants by Maximum Dose Achieved With no or Mild Symptoms at Exit DBPCFC | 1000 mg | 0 Participants |
| AR201 Powder | Number of Participants by Maximum Dose Achieved With no or Mild Symptoms at Exit DBPCFC | 1 mg | 0 Participants |
| AR201 Powder | Number of Participants by Maximum Dose Achieved With no or Mild Symptoms at Exit DBPCFC | 2000 mg | 1 Participants |
| AR201 Powder | Number of Participants by Maximum Dose Achieved With no or Mild Symptoms at Exit DBPCFC | 100 mg | 0 Participants |
| AR201 Powder | Number of Participants by Maximum Dose Achieved With no or Mild Symptoms at Exit DBPCFC | Did not have an exit DBPCFC | 6 Participants |
| AR201 Powder | Number of Participants by Maximum Dose Achieved With no or Mild Symptoms at Exit DBPCFC | None | 0 Participants |
| Placebo Powder | Number of Participants by Maximum Dose Achieved With no or Mild Symptoms at Exit DBPCFC | Did not have an exit DBPCFC | 6 Participants |
| Placebo Powder | Number of Participants by Maximum Dose Achieved With no or Mild Symptoms at Exit DBPCFC | None | 0 Participants |
| Placebo Powder | Number of Participants by Maximum Dose Achieved With no or Mild Symptoms at Exit DBPCFC | 1 mg | 0 Participants |
| Placebo Powder | Number of Participants by Maximum Dose Achieved With no or Mild Symptoms at Exit DBPCFC | 3 mg | 0 Participants |
| Placebo Powder | Number of Participants by Maximum Dose Achieved With no or Mild Symptoms at Exit DBPCFC | 10 mg | 0 Participants |
| Placebo Powder | Number of Participants by Maximum Dose Achieved With no or Mild Symptoms at Exit DBPCFC | 30 mg | 0 Participants |
| Placebo Powder | Number of Participants by Maximum Dose Achieved With no or Mild Symptoms at Exit DBPCFC | 100 mg | 0 Participants |
| Placebo Powder | Number of Participants by Maximum Dose Achieved With no or Mild Symptoms at Exit DBPCFC | 300 mg | 0 Participants |
| Placebo Powder | Number of Participants by Maximum Dose Achieved With no or Mild Symptoms at Exit DBPCFC | 600 mg | 0 Participants |
| Placebo Powder | Number of Participants by Maximum Dose Achieved With no or Mild Symptoms at Exit DBPCFC | 1000 mg | 0 Participants |
| Placebo Powder | Number of Participants by Maximum Dose Achieved With no or Mild Symptoms at Exit DBPCFC | 2000 mg | 0 Participants |