E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
eosinophilic esophagitis |
esofagite eosinofílica |
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E.1.1.1 | Medical condition in easily understood language |
allergy-like, chronic inflammation of the esophagus |
inflamação crónica e alérgica do esófago |
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E.1.1.2 | Therapeutic area | Diseases [C] - Digestive System Diseases [C06] |
MedDRA Classification |
E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 20.1 |
E.1.2 | Level | PT |
E.1.2 | Classification code | 10064212 |
E.1.2 | Term | Eosinophilic oesophagitis |
E.1.2 | System Organ Class | 10017947 - Gastrointestinal disorders |
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E.1.3 | Condition being studied is a rare disease | Yes |
E.2 Objective of the trial |
E.2.1 | Main objective of the trial |
To prove the non-inferiority of 1 x 2 mg/d vs. 2 x 1 mg/d budesonide orodispersible tablets for the induction of histological remission in adult patients with active eosinophilic esophagitis (EoE) |
Provar a não-inferioridade de 1 x 2 mg/d vs. 2 x 1 mg/d comprimidos orodispersíveis de budesonida para a indução da remissão histológica em doentes adultos com esofagite eosinófila activa (EoE) |
|
E.2.2 | Secondary objectives of the trial |
- To further assess EoE-associated clinical, endoscopic, and histological findings after 6 weeks treatment with budesonide orodispersible tablets, - To study safety and tolerability as assessed by adverse events and laboratory parameters, - To assess patients’ quality of life |
- Avaliar melhor os resultados clínicos, endoscópicos e histológicos associados à EoE após 6 semanas de tratamento com comprimidos orodispersíveis de budesonida, - Estudar a segurança e tolerabilidade avaliada por eventos adversos e parâmetros laboratoriais, - Avaliar a qualidade de vida dos doentes |
|
E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
- Signed informed consent, - Man or woman 18 to 75 years of age, - Confirmed diagnosis of eosinophilic esophagitis (EoE) - Active symptomatic and histological EoE - At least 4 points in Patient’s Global Assessment (PatGA) concerning the severity of EoE symptoms at baseline visit, |
- Consentimento informado assinado, - Homem ou mulher dos 18 aos 75 anos de idade, - Diagnóstico confirmado de esofagite eosinófila (EoE) - EoE activo sintomático e histológico - Pelo menos 4 pontos na Avaliação Global do Paciente (PatGA) relativamente à gravidade dos sintomas da EoE na visita de base |
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E.4 | Principal exclusion criteria |
- Gastroesophageal reflux disease (GERD), - Achalasia, scleroderma esophagus, or systemic sclerosis, - Clinically evident causes for esophageal eosinophilia other than EoE - Any concomitant esophageal disease and relevant active gastro-intestinal disease - Abnormal laboratory values, presence of or suspected relevant concomitant disease(s), that could affect study-specific assessments and/or their evaluation, or might compromise patient's safety and/or compliance (e.g. severe organic or psychiatric diseases/disorders, infectious diseases associated with clinical signs, liver cirrhosis, portal hypertension, cardiovascular disease, diabetes mellitus, osteoporosis, active peptic ulcer disease - History of cancer, gastrointestinal bleeding, esophageal surgery, esophageal dilation procedures or need for an immediate endoscopic intervention due to a stricture - Diagnosis of chickenpox, herpes zoster, or measles within the last 3 months prior to baseline, - Treatment with medication, that could compromise/influence the effects of the study treatment, assessment of the endpoints and/or patients' safety, during or within too narrow timeframe of the clinical trial (e.g. immunosuppressants, CYP3A4 inhibitors, live vaccination, non-stable treatment with proton pump inhibitors) - Exiting or intended pregnancy or breast-feeding |
- Doença do refluxo gastroesofágico (DRGE), - Achalasia, esclerodermia esofágica, ou esclerose sistémica, - Causas clinicamente evidentes para a eosinofilia esofágica que não a EoE - Qualquer doença concomitante do esófago e doença gastro-intestinal activa relevante - Valores laboratoriais anormais, presença ou suspeita de doença(s) concomitante(s) relevante(s), que possam afectar avaliações específicas do estudo e/ou a sua avaliação, ou que possam comprometer a segurança e/ou a conformidade do doente (por exemplo, doenças/perturbações orgânicas ou psiquiátricas graves, doenças infecciosas associadas a sinais clínicos, cirrose hepática, hipertensão portal, doença cardiovascular, diabetes mellitus, osteoporose, úlcera péptica activa - História de cancro, hemorragia gastrointestinal, cirurgia do esófago, procedimentos de dilatação esofágica ou necessidade de uma intervenção endoscópica imediata devido a uma estricção - Diagnóstico de varicela, herpes zoster, ou sarampo nos últimos 3 meses antes da linha de base, - Tratamento com medicamentos, que poderia comprometer/influenciar os efeitos do tratamento em estudo, avaliação dos pontos finais e/ou segurança dos pacientes, durante ou dentro de um prazo demasiado curto do ensaio clínico (por exemplo, imunossupressores, inibidores de CYP3A4, vacina viva, tratamento não estável com inibidores de bomba de protões) - Gravidez ou amamentação em curso ou prevista
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E.5 End points |
E.5.1 | Primary end point(s) |
Proportion of patients with histological remission |
Proporção de pacientes com remissão histológica |
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E.5.1.1 | Timepoint(s) of evaluation of this end point |
|
E.5.2 | Secondary end point(s) |
1. Change in the peak eos/mm2 hpf from baseline to week 6 (LOCF),
2. Change in the severity of dysphagia
3. Change in the severity of odynophagia
4. Change of overall severity of EoE symptoms
5. Change in the total weekly Eosinophilic Esophagitis Activity Index |
1. Alteração do pico eos/mm2 hpf da linha de base para a semana 6 (LOCF),
2. Alteração da gravidade da disfagia
3. Mudança na severidade da odinofagia
4. Alteração da gravidade geral dos sintomas da EoE
5. Mudança no índice semanal total de actividade de esofagite eosinófila |
|
E.5.2.1 | Timepoint(s) of evaluation of this end point |
week 6 (for secondary endpoints 1 - 5) |
semana 6 (para os pontos finais secundários 1 - 5) |
|
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 | No |
E.6.13 | Others | No |
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 | No |
E.8.1.3 | Single blind | No |
E.8.1.4 | Double blind | Yes |
E.8.1.5 | Parallel group | Yes |
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) | Yes |
E.8.2.2 | Placebo | No |
E.8.2.3 | Other | No |
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
| Yes |
E.8.4 | The trial involves multiple sites in the Member State concerned | No |
E.8.5 | The trial involves multiple Member States | Yes |
E.8.5.1 | Number of sites anticipated in the EEA | 24 |
E.8.6 Trial involving sites outside the EEA |
E.8.6.1 | Trial being conducted both within and outside the EEA | Yes |
E.8.6.2 | Trial being conducted completely outside of the EEA | No |
E.8.6.3 | If E.8.6.1 or E.8.6.2 are Yes, specify the regions in which trial sites are planned |
Switzerland |
Canada |
Russian Federation |
Austria |
Germany |
Portugal |
Spain |
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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 | 19 |
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 | 19 |
E.8.9.2 | In all countries concerned by the trial days | 0 |