| E.1 Medical condition or disease under investigation |
| E.1.1 | Medical condition(s) being investigated |
| Peanut Allergy |
| Alergia al cacahuete |
|
| E.1.1.1 | Medical condition in easily understood language |
| Allergy to peanuts or peanut-containing foods |
| Alergia al cacahuete o comida que contenga cacahuete |
|
| E.1.1.2 | Therapeutic area | Diseases [C] - Immune System Diseases [C20] |
| MedDRA Classification |
| E.1.2 Medical condition or disease under investigation |
| E.1.2 | Version | 20.0 |
| E.1.2 | Level | PT |
| E.1.2 | Classification code | 10016946 |
| E.1.2 | Term | Food allergy |
| E.1.2 | System Organ Class | 10021428 - Immune system disorders |
|
| E.1.3 | Condition being studied is a rare disease | No |
| E.2 Objective of the trial |
| E.2.1 | Main objective of the trial |
| To assess proxy- and self- reported disease-specific HRQOL of peanut-allergic subjects aged 4 to 17 years, inclusive, receiving AR101 in combination with standard of care versus standard of care alone for approximately 18 months. |
| Evaluar la CVRS específica de la enfermedad evaluada por el propio sujeto o por un allegado en sujetos alérgicos al cacahuete de 4 a 17 años de edad, ambos inclusive, que reciban AR101 en combinación con el tratamiento habitual o solo el tratamiento habitual durante aproximadamente 18 meses. |
|
| E.2.2 | Secondary objectives of the trial |
- Safety and tolerability of AR101
To characterize changes over the course of the study in the following:
- Disease-specific HRQOL of parents/caregivers
- Relationship between clinical efficacy of AR101 (change in level of sensitization to peanut allergen) and HRQOL of subjects and parents/caregivers |
- Seguridad y tolerabilidad del AR101
Caracterizar los cambios observados durante el estudio en los siguientes aspectos:
- CVRS de progenitores/cuidadores específica de la enfermedad.
- Relación entre la eficacia clínica de AR101 (variación del grado de sensibilización a los alérgenos del cacahuete) y la CVRS de los sujetos y de sus progenitores/cuidadores. |
|
| E.2.3 | Trial contains a sub-study | No |
| E.3 | Principal inclusion criteria |
1. Aged 4 to 17 years, inclusive, at screening
2. Written informed consent from subjects, as appropriate per local requirements, and legal
guardian/parent (or both parents where required by local authorities) of subjects who are
minors.
3. Written assent from subjects who are minors, as appropriate per local requirements
4. Written informed consent from the parent/caregiver who will complete relevant questionnaires during the study
5. History of physician-diagnosed immunoglobulin (Ig) E-mediated peanut allergy that includes the onset of characteristic signs and symptoms of allergy within 2 hours of known oral exposure to peanut or peanut-containing food. In general, characteristic signs and symptoms of IgE-mediated allergic reactions are objective and affect the target organs of skin, gastrointestinal (GI) tract, upper/lower respiratory tract, cardiovascular system, or a combination of target organs
6. Mean wheal diameter on SPT to peanut ≥ 8 mm greater than the negative saline control at screening
7. Serum IgE to peanut of ≥ 14 kUA/L at screening
8. For sexually active females of childbearing potential, use of a highly effective method of birth control, defined as one that results in a low failure rate (ie, < 1% per year) when used consistently and correctly |
1. Edad de 4 a 17 años, ambos inclusive, en la selección.
2. Consentimiento informado por escrito de los sujetos, según proceda conforme a los requisitos locales, y del tutor legal/progenitor (o de ambos progenitores cuando así lo exijan las autoridades locales) de los sujetos menores de edad.
3. Asentimiento por escrito de los sujetos menores de edad, según proceda conforme a los requisitos locales.
4. Consentimiento informado por escrito del progenitor/cuidador que cumplimentará los cuestionarios pertinentes durante el estudio.
5. Antecedentes de alergia a los cacahuetes mediada por la inmunoglobulina (Ig) E y diagnosticada por un médico, lo que incluye la aparición de signos y síntomas característicos de alergia en las 2 horas siguientes a la exposición oral conocida a cacahuetes o alimentos que contienen cacahuetes. En general, los signos y síntomas característicos de las reacciones alérgicas mediadas por la IgE son objetivos y afectan a los órganos diana de la piel, el tubo digestivo, las vías respiratorias superiores e inferiores, el aparato cardiovascular o una combinación de órganos diana.
6. Diámetro medio de la pápula en la PPC para el cacahuete ≥ 8 mm mayor que la del control negativo con solución salina en la selección.
7. IgE sérica para el cacahuete ≥ 14 kUA/l en la selección.
8. En las mujeres en edad fértil sexualmente activas, uso de un método anticonceptivo muy eficaz, que se define como un método que tiene un índice bajo de fallos (es decir, inferior al < 1% anual) cuando se utiliza de forma constante y correcta. |
|
| E.4 | Principal exclusion criteria |
1. Uncertain clinical diagnosis of peanut allergy
2. History of severe or life-threatening anaphylaxis or anaphylactic shock within 60 days before screening
3. History of eosinophilic esophagitis (EoE); other eosinophilic GI disease; chronic, recurrent, or severe gastroesophageal reflux disease (GERD); symptoms of dysphagia (eg, difficulty swallowing, food “getting stuck”); or recurrent GI symptoms of any etiology
4. History of a mast cell disorder including systemic mastocytosis, urticaria pigmentosa, chronic idiopathic or chronic physical urticaria beyond simple dermatographism (eg, cold urticaria, cholinergic urticaria), and hereditary or idiopathic angioedema
5. Severe persistent asthma
6. Mild or moderate asthma (criteria steps 1-4; NHLBI, 2007) that is uncontrolled or difficult to control
7. History of high-dose corticosteroid medication use (eg, > 3 days at 1-2 mg/kg of prednisone or equivalent)
8. History of chronic disease (except asthma, atopic dermatitis, or allergic rhinitis) that is or is at significant risk of becoming unstable or requiring a change in a chronic therapeutic regimen, including malignancies within 5 years before screening and autoimmune diseases
9. History of cardiovascular disease including uncontrolled or inadequately controlled hypertension
10. Use of beta-blockers (oral), angiotensin-converting enzyme inhibitors, angiotensin receptor blockers, calcium channel blockers, or tricyclic antidepressants
11. Unable to discontinue antihistamines 5 half-lives of the medication before the SPT, first day of dose escalation, and food challenges
12. Lack of an available palatable vehicle food to which the subject is not allergic
13. Allergy to oat
14. Use of any therapeutic antibody or any immunomodulatory therapy (including immunosuppressive medications) except aeroallergen or venom immunotherapy used in the maintenance phase within 6 months before screening |
1. Diagnóstico clínico incierto de alergia a los cacahuetes.
2. Antecedentes de anafilaxia o choque anafiláctico grave o potencialmente mortal en los 60 días previos a la selección.
3. Antecedentes de esofagitis eosinofílica (EEo); otras enfermedades gastrointestinales eosinofílicas; enfermedad por reflujo gastroesofágico (ERGE) crónica, recurrente o grave; síntomas de disfagia (p. ej., dificultad para tragar, “atragantamiento” con alimentos); o síntomas digestivos recurrentes de cualquier etiología.
4. Antecedentes de un trastorno relacionado con los mastocitos, como mastocitosis sistémica, urticaria pigmentaria, urticaria idiopática o física crónica más allá de un dermatografismo simple (por ejemplo, urticaria por frío, urticaria colinérgica) y angioedema hereditario o idiopático.
5. Asma persistente grave.
6. Asma leve o moderada (criterios 1-4 del NHLBI, 2007), no controlada o difícil de controlar.
7. Antecedentes de uso de corticosteroides en dosis altas (p. ej., > 3 días con 1 2 mg/kg de prednisona o equivalente.
8. Antecedentes de enfermedades crónicas (excepto asma, dermatitis atópica o rinitis alérgica) que tengan o presenten un riesgo significativo de inestabilidad o que precisen un cambio en un régimen terapéutico crónico, incluidas neoplasias malignas, en los 5 años previos a la selección y enfermedades autoinmunitarias.
9. Antecedentes de enfermedad cardiovascular, incluida la hipertensión no controlada o controlada insuficientemente.
10. Uso de beta bloqueantes (orales), inhibidores de la enzima de conversión de la angiotensina; antagonistas del receptor de la angiotensina, antagonistas del calcio o antidepresivos tricíclicos.
11. Imposibilidad de suspender la administración de antihistamínicos 5 semividas de la medicación antes de la PPC, el primer día de aumento escalonado de la dosis y las provocaciones alimentarias.
12. Falta de un alimento apetitoso que sirva como vehículo y al que el sujeto no sea alérgico.
13. Alergia a la avena.
14. Uso de cualquier anticuerpo terapéutico o tratamiento inmunomodulador (incluidos los inmunodepresores), excepto inmunoterapia con alérgenos aéreos o contra las picaduras o mordeduras de animales venenosos en la fase de mantenimiento en los 6 meses previos a la selección. |
|
| E.5 End points |
| E.5.1 | Primary end point(s) |
| To assess proxy- and self-reported disease-specific HRQOL of peanut‑allergic subjects aged 4 to 17 years, inclusive, receiving AR101 in combination with standard of care versus standard of care alone for approximately 18 months |
| Evaluar la CVRS específica de la enfermedad evaluada por el propio sujeto o por un allegado en sujetos alérgicos al cacahuete de 4 a 17 años de edad, ambos inclusive, que reciban AR101 en combinación con el tratamiento habitual o solo el tratamiento habitual durante aproximadamente 18 meses. |
|
| E.5.1.1 | Timepoint(s) of evaluation of this end point |
| Questionnaires are completed before randomization and approximately every 3 months thereafter. |
| Los cuestionarios se completan antes de la aleatorización y aproximadamente cada 3 meses a partir de entonces. |
|
| E.5.2 | Secondary end point(s) |
-Safety and tolerability of AR101
To characterize changes over the course of the study in the following:
-Disease-specific HRQOL of parents/caregivers
-Relationship between clinical efficacy of AR101 (change in level of sensitization to peanut allergen) and HRQOL of subjects and parents/caregivers |
- Seguridad y tolerabilidad del AR101
Caracterizar los cambios observados durante el estudio en los siguientes aspectos:
- CVRS de progenitores/cuidadores específica de la enfermedad.
- Relación entre la eficacia clínica de AR101 (variación del grado de sensibilización a los alérgenos del cacahuete) y la CVRS de los sujetos y de sus progenitores/cuidadores. |
|
| E.5.2.1 | Timepoint(s) of evaluation of this end point |
|
| E.6 and E.7 Scope of the trial |
| E.6 | Scope of the trial |
| E.6.1 | Diagnosis | No |
| E.6.2 | Prophylaxis | No |
| E.6.3 | Therapy | Yes |
| E.6.4 | Safety | Yes |
| E.6.5 | Efficacy | Yes |
| E.6.6 | Pharmacokinetic | No |
| E.6.7 | Pharmacodynamic | No |
| E.6.8 | Bioequivalence | No |
| E.6.9 | Dose response | No |
| E.6.10 | Pharmacogenetic | No |
| E.6.11 | Pharmacogenomic | No |
| E.6.12 | Pharmacoeconomic | Yes |
| E.6.13 | Others | Yes |
| E.6.13.1 | Other scope of the trial description |
| quality of life |
| Calidad de vida |
|
| E.7 | Trial type and phase |
| E.7.1 | Human pharmacology (Phase I) | No |
| E.7.1.1 | First administration to humans | No |
| E.7.1.2 | Bioequivalence study | No |
| E.7.1.3 | Other | No |
| E.7.1.3.1 | Other trial type description | |
| E.7.2 | Therapeutic exploratory (Phase II) | No |
| E.7.3 | Therapeutic confirmatory (Phase III) | Yes |
| E.7.4 | Therapeutic use (Phase IV) | No |
| E.8 Design of the trial |
| E.8.1 | Controlled | Yes |
| E.8.1.1 | Randomised | Yes |
| E.8.1.2 | Open | Yes |
| E.8.1.3 | Single blind | No |
| E.8.1.4 | Double blind | No |
| E.8.1.5 | Parallel group | No |
| E.8.1.6 | Cross over | No |
| E.8.1.7 | Other | No |
| E.8.2 | Comparator of controlled trial |
| E.8.2.1 | Other medicinal product(s) | No |
| E.8.2.2 | Placebo | No |
| E.8.2.3 | Other | Yes |
| E.8.2.3.1 | Comparator description |
| Tto habitual: evitar alimentos que contengan cacahuete, manejo de síntomas, medicacion de rescate |
| standard of care: avoid peanut(s) containing food, management of symptoms, rescue medication |
|
| E.8.2.4 | Number of treatment arms in the trial | 2 |
| E.8.3 |
The trial involves single site in the Member State concerned
| No |
| E.8.4 | The trial involves multiple sites in the Member State concerned | Yes |
| E.8.4.1 | Number of sites anticipated in Member State concerned | 14 |
| E.8.5 | The trial involves multiple Member States | Yes |
| E.8.5.1 | Number of sites anticipated in the EEA | 45 |
| E.8.6 Trial involving sites outside the EEA |
| E.8.6.1 | Trial being conducted both within and outside the EEA | No |
| E.8.6.2 | Trial being conducted completely outside of the EEA | No |
| E.8.7 | Trial has a data monitoring committee | No |
| E.8.8 |
Definition of the end of the trial and justification where it is not the last
visit of the last subject undergoing the trial
|
|
| E.8.9 Initial estimate of the duration of the trial |
| E.8.9.1 | In the Member State concerned years | 0 |
| E.8.9.1 | In the Member State concerned months | 18 |
| E.8.9.1 | In the Member State concerned days | 0 |
| E.8.9.2 | In all countries concerned by the trial years | 0 |
| E.8.9.2 | In all countries concerned by the trial months | 18 |
| E.8.9.2 | In all countries concerned by the trial days | 0 |
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