E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Refractory invasive pulmonary aspergillosis (IPA) |
Aspergillosi polmonare invasiva (IPA) refrattaria |
|
E.1.1.1 | Medical condition in easily understood language |
Fungal infection of the airway |
Infezione fungina delle vie respiratorie |
|
E.1.1.2 | Therapeutic area | Diseases [C] - Bacterial Infections and Mycoses [C01] |
MedDRA Classification |
E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 20.0 |
E.1.2 | Level | LLT |
E.1.2 | Classification code | 10059259 |
E.1.2 | Term | Pulmonary aspergillosis |
E.1.2 | System Organ Class | 100000004862 |
|
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 efficacy of nebulized PC945 in combination with systemic antifungal therapy for the treatment of refractory IPA |
Valutare l’efficacia di PC945 nebulizzato in associazione a una terapia antimicotica sistemica per il trattamento dell’IPA refrattaria. |
|
E.2.2 | Secondary objectives of the trial |
To assess the safety and tolerability of nebulized PC945 in combination with systemic antifungal therapy for the treatment of refractory IPA. |
Valutare la sicurezza e la tollerabilità di PC945 nebulizzato in associazione a una terapia antimicotica sistemica per il trattamento dell’IPA refrattaria. |
|
E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
1. Participant has proven or probable IPA according to the 2019 European Organization for Research and Treatment of Cancer/ Mycoses Study Group Education and Research Consortium (EORTC/MSGERC) consensus definitions or according to the 2010 International Society for Heart and Lung Transplantation (ISHLT) consensus statements for the definitions of infections in cardiothoracic transplant recipients 2. Participant's IPA has an unfavorable response to adequate antifungal therapy |
1. Soggetti con IPA certa o probabile secondo le definizioni della consensus della European Organization for Research and Treatment of Cancer/ Mycoses Study Group Education and Research Consortium (EORTC/MSGERC) del 2019 o secondo gli statement della consensus dell'International Society for Heart and Lung Transplantation (ISHLT) del 2010 prelativi alla definizione delle infezioni nei riceventi di trapianto cardiotoracico 2. 7. L’IPA del soggetto mostra una risposta non favorevole a terapia antimicotica adeguata |
|
E.4 | Principal exclusion criteria |
1. Participant with a known or suspected concomitant medical condition or post-surgery complication that, in the opinion of the Investigator, may jeopardize adherence to the protocol requirements or impede the accurate measurement of efficacy or may be an unacceptable additional risk to the Participant should he/she participate in the study 2. Participant who has previously received PC945 3. Participant with a known history of allergy, hypersensitivity, or any previous serious reaction to any component of the PC945 or placebo formulations 4. Participant who is participating, or who is due to participate at any time during the study, in a therapeutic or diagnostic clinical trial |
1. Soggetti con condizioni cliniche concomitanti note o sospette o complicanze post-chirurgiche che, secondo la valutazione dello Sperimentatore, sono tali da poter compromettere l’aderenza ai requisiti del protocollo o impedire un’accurata misurazione dell’efficacia o da poter comportare per tali soggetti un rischio aggiuntivo non accettabile in caso di partecipazione allo studio 2. Soggetti precedentemente trattati con PC945 3. Soggetti per i quali sia nota all'anamnesi allergia, ipersensibilità o qualunque reazione grave precedente a uno qualunque dei componenti della formulazione di PC945 o del placebo. 4. Soggetti che stanno prendendo parte o per i quali è prevista in qualunque momento dello studio la partecipazione a una sperimentazione clinica terapeutica o diagnostica |
|
E.5 End points |
E.5.1 | Primary end point(s) |
Number of Participants with Complete or Partial Overall Response |
Numero di Partecipanti con Risposta Globale Completa o Parziale |
|
E.5.1.1 | Timepoint(s) of evaluation of this end point |
Up to 12 weeks |
Fino a 12 settimane |
|
E.5.2 | Secondary end point(s) |
Time to Complete or Partial Overall Clinical Response |
Tempo di Risposta Clinica Globale Completa o Parziale |
|
E.5.2.1 | Timepoint(s) of evaluation of this end point |
Up to 12 weeks |
Fino a 12 settimane |
|
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 | Yes |
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 | 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
| 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 | 4 |
E.8.5 | The trial involves multiple Member States | Yes |
E.8.5.1 | Number of sites anticipated in the EEA | 25 |
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 | Information not present in EudraCT |
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 |
Argentina |
Brazil |
Canada |
Chile |
Colombia |
India |
Israel |
Korea, Republic of |
Taiwan |
United States |
Austria |
France |
Spain |
Germany |
Greece |
Italy |
Belgium |
United Kingdom |
|
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 | 7 |
E.8.9.1 | In the Member State concerned days | 15 |
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 | 7 |
E.8.9.2 | In all countries concerned by the trial days | 15 |