E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Primary Graft Dysfunction (PGD) after lung transplantation |
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E.1.1.1 | Medical condition in easily understood language |
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E.1.1.2 | Therapeutic area | Diseases [C] - Respiratory Tract Diseases [C08] |
MedDRA Classification |
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 assess the clinical effect of orally inhaled AP301 on treatment of primary graft dysfunction (PGD) according to ISHLT (International Society for Heart and Lung Transplantation) criteria in patients after primary LuTX in comparison to placebo. Secondary Objectives
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E.2.2 | Secondary objectives of the trial |
To assess: • recipient mortality at 30 days after transplantation (%), • duration of intubation • requirement of mechanical ventilation, • length of ICU stay, • incidence of acute rejections, • freedom of chronic lung allograft dysfunction (CLAD), • graft survival within 30 days, • local and systemic safety and tolerability of AP301. |
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
Being a male or female recipient on the AKH`s waiting list for primary single or double LuTX PGD score ≥ 1 within 72 hours after LuTX Informed consent is available |
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E.4 | Principal exclusion criteria |
History of clinically relevant allergies or idiosyncrasies to AP301 or any other inactive ingredient(s) of the investigational product Postoperative ECMO support Paediatric /adolescent recipients (< 18 years) Lobar transplantation Retransplantation Combined solid organ transplants Participation in other drug trials
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E.5 End points |
E.5.1 | Primary end point(s) |
The primary efficacy variable will be the changes of the P/F ratio from T0 (time of PGD diagnosis) to day 7 of the treatment or to extubation of the patient. |
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E.5.1.1 | Timepoint(s) of evaluation of this end point |
72 hours after diagnosis of PGD |
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E.5.2 | Secondary end point(s) |
1. Oxygenation index until day 7 of treatment or until extubation. 2. EVWL (measured with PiCCO® technique) until day 7 of treatment 3. Changes in ΔpO2 during treatment 4. recipient mortality at 30 days after transplantation (%), 5. duration of intubation 6. requirement of mechanical ventilation, 7. length of ICU stay, 8. incidence of acute rejections, 9. freedom of chronic lung allograft dysfunction (CLAD) 10. graft survival within 30 days 11. overall survival within 30 days
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E.5.2.1 | Timepoint(s) of evaluation of this end point |
up to 7 days after diagnosis |
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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 | No |
E.6.5 | Efficacy | No |
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 | 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 | Yes |
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 | No |
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
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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 | 3 |
E.8.9.1 | In the Member State concerned days | |