E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Prevention of oesophageal strictures in adult patients after endoscopic submucosal dissection |
Prevenção de estenoses esofágicas em doentes adultos após disseção endoscópica da submucosa |
|
E.1.1.1 | Medical condition in easily understood language |
Prevention of strictures of the oesophagus in adult patients after removal of cancer tissue from the oesophagus using an endoscopic surgery technique |
Prevenção de estenoses do esofágo em doentes adultos após remoção de tecido cancerígeno do esófago utilizando uma técnica cirúrgica endoscópica |
|
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 | 21.0 |
E.1.2 | Level | LLT |
E.1.2 | Classification code | 10030186 |
E.1.2 | Term | Oesophageal squamous cell carcinoma NOS |
E.1.2 | System Organ Class | 10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps) |
|
E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 21.0 |
E.1.2 | Level | PT |
E.1.2 | Classification code | 10030137 |
E.1.2 | Term | Oesophageal adenocarcinoma |
E.1.2 | System Organ Class | 10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps) |
|
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 | 10004137 |
E.1.2 | Term | Barrett's oesophagus |
E.1.2 | System Organ Class | 10017947 - Gastrointestinal 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 the efficacy of eight weeks treatment with 2 x 1 mg/day or 2 x 2 mg/day budesonide orodispersible tablets vs. placebo for prevention of oesophageal strictures after endoscopic submucosal dissection |
• Avaliar a eficácia de oito semanas de tratamento com comprimidos orodispersíveis de budesonida 2 x 1 mg/dia ou 2 x 2 mg/dia vs. placebo para prevenção de estenoses esofágicas após dissecção endoscópica da submucosa. |
|
E.2.2 | Secondary objectives of the trial |
• To study safety and tolerability of budesonide orodispersible tablets vs. placebo by means of adverse events and laboratory parameters. • To assess patients’ quality of life.
|
• Estudar a segurança e a tolerabilidade de comprimidos orodispersíveis de budesonida vs. placebo por meio de acontecimentos 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, • Male or female patients, 18 to 85 years of age, • Estimated life expectancy of at least one year (not applicable in Portugal); • ECOG Performance Status of ≤ 2 at the randomisation visit (i.e. after the ESD-procedure), • a) Biopsy proven or endoscopically suspect oesophageal SCC and/or high grade dysplasia in a focal lesion of the squamous epithelium, treated with ESD; or b) Biopsy proven or endoscopically suspect BE-HGD or EAC, treated with ESD; • Mucosal defect after ESD of a) ≥ 50% oesophageal circumference in a patient with SCC, or b) ≥ 75% oesophageal circumference in a patient with BE-HGD or EAC; • Negative pregnancy test in females of childbearing potential at the screening visit.
|
|
E.4 | Principal exclusion criteria |
• Any prior or intended chemotherapy for oesophageal cancer, • Any prior ESD in the area where ESD will be done, • Any prior or intended oesophageal surgery or surgery for the mediastinum, endoscopic mucosal resection (EMR), or radio frequency ablation (RFA), in the area where ESD will be done, • Evidence of regional lymph node metastases or distant metastases prior to ESD, • Any prior or intended radiotherapy which involves or affects the area of ESD during the last 5 years; • Any prior endoscopic dilation for oesophageal stenosis which involves or affects the area of ESD during the last 5 years; • Any other concomitant oesophageal disease, • Any severe concomitant disease, which in the opinion of the investigator might have an influence on the patient’s compliance or the interpretation of the results, or any disorder which in the opinion of the investigator might affect the patient’s safety, • Any systemic therapy for any reason that may affect assessment of primary or secondary endpoints.
|
|
E.5 End points |
E.5.1 | Primary end point(s) |
Primary efficacy endpoint: • Percentage of patients free of strictures at visit week 8.
|
|
E.5.1.1 | Timepoint(s) of evaluation of this end point |
after 8 weeks of double-blind phase |
|
E.5.2 | Secondary end point(s) |
Secondary efficacy endpoints: • Number of endoscopic dilations per patient during the DB treatment phase. • Percentage of patients free of strictures until the FU visit.
|
|
E.5.2.1 | Timepoint(s) of evaluation of this end point |
Endpoint 'Number of endoscopic dilations per patient during the DB treatment phase': after 8 weeks of double-blind phase
Endpoint 'Percentage of patients free of strictures until the FU visit': at follow-up visit |
|
E.6 and E.7 Scope of the trial |
E.6 | Scope of the trial |
E.6.1 | Diagnosis | No |
E.6.2 | Prophylaxis | Yes |
E.6.3 | Therapy | No |
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) | Yes |
E.7.3 | Therapeutic confirmatory (Phase III) | No |
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) | No |
E.8.2.2 | Placebo | Yes |
E.8.2.3 | Other | No |
E.8.2.4 | Number of treatment arms in the trial | 3 |
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 | 3 |
E.8.5 | The trial involves multiple Member States | Yes |
E.8.5.1 | Number of sites anticipated in the EEA | 14 |
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 | Yes |
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 | 1 |
E.8.9.1 | In the Member State concerned months | 10 |
E.8.9.1 | In the Member State concerned days | 18 |
E.8.9.2 | In all countries concerned by the trial years | 1 |
E.8.9.2 | In all countries concerned by the trial months | 10 |
E.8.9.2 | In all countries concerned by the trial days | 18 |