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 for squamous cell carcinoma |
Zapobieganie powstawania zwężeń przełyku u pacjentów dorosłych po endoskopowej dyssekcji podśluzówkowej wykonanej z powodu raka płaskonabłonkowego |
|
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 |
Zapobieganie powstawania zwężeń przełyku u pacjentów dorosłych po usunięciu tkanki nowotworowej z przełyku przy użyciu techniki chirurgii endoskopowej |
|
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 | 100000004864 |
|
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. |
• Ocena skuteczności trwającego osiem tygodni leczenia budezonidem w postaci tabletek ulegających rozpadowi w jamie ustnej w dawce 2 x 1 mg/dobę lub 2 x 2 mg/dobę w porównaniu z placebo w celu zapobiegania zwężeniom przełyku po endoskopowej dyssekcji podśluzówkowej. |
|
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.
|
• Określenie bezpieczeństwa stosowania i tolerancji budezonidu w postaci tabletek ulegających rozpadowi w jamie ustnej w porównaniu z placebo na podstawie występowania zdarzeń niepożądanych i oceny parametrów laboratoryjnych. • Ocena jakości życia pacjentów. |
|
E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
• Signed informed consent, • Male or female patients, 18 to 80 years of age, • Estimated life expectancy of at least three years, • ECOG Performance Status of ≤ 2 at the randomisation visit (i.e. after the ESD-procedure), • Biopsy proven or endoscopically suspect oesophageal SCC and/or high grade dysplasia in a focal lesion of the squamous epithelium, treated with ESD, • Mucosal defect ≥ 75% oesophageal circumference after ESD, or patient with a complete circumferential ESD, • 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, • Any prior endoscopic dilation for oesophageal stenosis, • 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 | 2 |
E.8.5 | The trial involves multiple Member States | Yes |
E.8.5.1 | Number of sites anticipated in the EEA | 13 |
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 | 14 |
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 | 9 |
E.8.9.2 | In all countries concerned by the trial days | 6 |