Real-World AR101 Market-Supporting Experience Study in Peanut-Allergic Children (RAMSES)

Study Overview
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Brief Summary
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This is a multicenter, randomized, double-blind, placebo-controlled safety study of AR101 using the characterized oral desensitization immunotherapy (CODIT™) regimen in peanut-allergic children.

Detailed Description
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The primary objective is to assess the safety and tolerability of AR101 when used in a CODIT™ regimen for approximately 6 months in peanut-allergic children children aged 4 to 17 years, inclusive.

Official Title
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Real-World AR101 Market-Supporting Experience Study in Peanut-Allergic Children Ages 4 to 17 Years (RAMSES)

Conditions
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Peanut Allergy

Intervention / Treatment
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* Biological: AR101
* Biological: Placebo


Participation Criteria
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Eligibility Criteria
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Key Inclusion Criteria: Ages 4 to 17 years, inclusive History of physician-diagnosed peanut allergy that includes allergic signs and symptoms within two hours of known oral exposure to peanut Mean peanut wheal diameter on SPT of at least 8mm and elevated psIgE of at least 14 kUA/L at screening Written informed consent from the subject's parent/guardian Written assent from the subject as appropriate (per local regulatory requirements) Use of effective birth control by sexually active female subjects of childbearing potential Key Exclusion Criteria: Subjects in whom the clinical diagnosis of peanut allergy is uncertain Severe or uncontrolled asthma History of cardiovascular disease, including uncontrolled or inadequately controlled hypertension History of severe or life-threatening episode of anaphylaxis or anaphylactic shock within 60 days of screening 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 undiagnosed etiology History of a mast cell disorder, including mastocytosis, urticaria pigmentosa, chronic idiopathic or chronic physical urticaria beyond simple dermatographism (e.g., cold urticaria, cholinergic urticaria), and hereditary or idiopathic angioedema Any other condition that, in the opinion of the Investigator, precludes participation for reasons of safety

Ages Eligible for Study
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Minimum Age: 4 Years
Maximum Age: 17 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Allocation: Randomized
Intervention Model: Parallel Assignment
Interventional Model Description: 2:1 randomization to AR101 or placebo
Masking: Triple


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Active Comparator: AR101 Powder Provided in Capsules<br>Study product formulated to contain peanut protein at different dosage strengths for use as defined in the protocol | Biological: AR101<br>* AR101 powder provided in capsules<br>|
| Placebo Comparator: Placebo powder<br>Placebo formulation in pull-apart capsules containing only excipients color-matched to AR101 study product. | Biological: Placebo<br>* Placebo powder provided in capsules<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Frequency of Treatment Emergent Adverse Events, Including Serious Adverse Events | Frequency of Treatment Emergent Adverse Events, including Serious Adverse Events, during the overall study period. Treatment-emergent adverse events were defined as adverse events with onset after the first dose of study product. | Approximately 6 months | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Frequency of Premature Discontinuation of Dosing Due to Adverse Events |  | Approximately 6 months | 
| Frequency of Premature Discontinuation of Dosing Due to Chronic/Recurrent Gastrointestinal Adverse Events |  | Approximately 6 months | 
| Proportion of Chronic/Recurrent Gastrointestinal Adverse Events Resolving <2, Between 2-4, Between 4-12, and ≥ 12 Weeks Following Discontinuation of Dosing |  | Approximately 6 months | 
| Frequency of Allergic Hypersensitivity Adverse Events Normalized for Duration of Treatment |  | Approximately 6 months | 
| Frequency of Anaphylaxis as Defined in the Protocol | Anaphylaxis is likely when any 1 of the 3 following sets of criteria is fulfilled: Acute onset of an illness (minutes to hours) with involvement of: (a) Skin/mucosal tissue (eg, generalized hives, itch/flush, swollen lips/tongue/uvula); AND (b) Airway compromise (eg, dyspnea, stridor, wheeze/bronchospasm, hypoxia, reduced PEFR); AND/OR (c) Reduced BP or associated symptoms (eg, hypotonia, syncope, incontinence). Two or more of the following that occur rapidly after exposure to the allergen (minutes to hours): (a) Skin/mucosal tissue; (b) Airway compromise; (c) Reduced BP or associated symptoms; (d) Persistent GI symptoms (eg, nausea, vomiting, crampy abdominal pain). Reduced BP after exposure to the allergen (minutes to hours). Infants and children: low systolic BP (age-specific) or > 30% drop in systolic BP; Adults: systolic BP < 90 mm Hg or > 30% drop from their baseline. | Approximately 6 months | 
| Frequency of Epinephrine Use as Rescue Medication |  | Approximately 6 months | 
| Frequency of Accidental Ingestion of Peanut and Other Allergenic Foods |  | Approximately 6 months | 
| Change of Asthma Control Using the Asthma Control Test (ACT) Questionnaire Total Score From Baseline to Interim (80 mg), End of Up-Dosing (300 mg) and Study Exit, Ages 4-11 | The Asthma Control Test (ACT) Questionnaire ACT for subjects aged 4-11 years included 4 questions for the subject and 3 questions for the parent; the total score (sum of 7 questions) ranged from 0 (worst control) to 27 (total control) | Baseline, Interim dose (approx. 10 weeks), End of Up-Dosing (20 to 40 weeks), Study Exit (14 days after End of Up-Dosing, usually approx. 6 months) | 
| Change of Asthma Control Using the Asthma Control Test (ACT) Questionnaire Total Score From Baseline to Interim (80 mg), End of Up-Dosing (300 mg) and Study Exit, Ages 12-17 | The Asthma Control Test (ACT) Questionnaire ACT for subjects aged 12-17 years consisted of 5 questions, and the total score (sum of 5 questions) ranged from 5 (worst control) to 25 (total control). | Baseline, Interim dose (approx. 10 weeks), End of Up-Dosing (20 to 40 weeks), Study Exit (14 days after End of Up-Dosing, usually approx. 6 months) | 
| Frequency of Adverse Events That Led to Early Withdrawal |  | Approximately 6 months | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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Characterized Oral Desensitization Immunotherapy (CODIT™), AR101, Characterized Peanut Allergen, CPNA (Characterized Peanut Allergen), OIT (oral immunotherapy), Peanut Allergy, Peanut-Allergic Children, Desensitization, ARC007