E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
|
E.1.1.1 | Medical condition in easily understood language |
Bleeding disorder, inherited deficiency in clotting factor VIII |
Disordine della coagulazione a carico del fattore VIII |
|
E.1.1.2 | Therapeutic area | Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] |
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 | 10018938 |
E.1.2 | Term | Haemophilia A (Factor VIII) |
E.1.2 | System Organ Class | 100000004850 |
|
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 investigate the safety of turoctocog alfa pegol during continuous use for prevention and treatment of bleeding episodes of previously turoctocog alfa pegol treated severe haemophilia A patients. |
Investigare la sicurezza di turoctocog alfa pegol durante l'uso regolare per la prevenzione e il trattamento degli episodi di sanguinamento in pazienti con emofilia A grave precedentemente trattati con turoctocog alfa pegol. |
|
E.2.2 | Secondary objectives of the trial |
To investigate the following in severe haemophilia A patients previously treated with turoctocog alfa pegol 1. Development of FVIII inhibitors 2. Efficacy of turoctocog alfa pegol prophylaxis 3. Haemostatic efficacy of turoctocog alfa pegol when used for treatment of bleeds |
Investigare i seguenti aspetti in pazienti con emofilia grave A precedentemente trattati con turoctocog alfa pegol 1. Sviluppo di inibitori del fattore VIII 2. Efficacia della profilassi con turoctocog alfa pegol 3. Efficacia emostatica di turoctocog alfa pegol quando utilizzato per il trattamento di sanguinamenti |
|
E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
1. Male patients of all ages with the diagnosis of severe congenital haemophilia A (FVIII activity <1%) based on medical records 2. On-going participation in NN7088-3859 (pathfinder2), or NN7088-3885 (pathfinder5) at the time of transfer |
1 -Pazienti di sesso maschile appartenenti a tutte le fasce di età con diagnosi di emofilia A congenita grave (attività del FVIII <1%), valutato sulla base delle cartelle cliniche 2 - Pazienti in corso di partecipazione agli studi clinici NN7088-3859 (pathfinder2) o a NN7088- 3885 (pathfinder5) al momento del trasferimento allo studio pathfinder8 |
|
E.4 | Principal exclusion criteria |
1. Known or suspected hypersensitivity to trial product including allergy to hamster protein or related products 2. Any disorder, except for conditions associated with haemophilia, which in the investigator’s opinion might jeopardise patient’s safety or compliance with the protocol 3. Current participation in any clinical trial (except NN7088-3859 (pathfinder2) or NN7088-3885 (pathfinder5)) of an approved or non-approved investigational medicinal product |
1. Ipersensibilità accertata o sospetta al prodotto sperimentale, compresa l’allergia alle proteine di origine animale (criceto) o ai prodotti correlati 2. Qualsiasi disturbo, ad eccezione delle condizioni associate all’emofilia, che, a giudizio dello sperimentatore, potrebbe mettere a rischio la sicurezza del paziente o l’aderenza al protocollo 3. Partecipazione in corso a qualsiasi altra sperimentazione clinica (tranne NN7088-3859 o NN7088-3885, rispettivamente pathfinder2 o pathfinder5) con un prodotto medicinale sperimentale approvato o non approvato |
|
E.5 End points |
E.5.1 | Primary end point(s) |
Number of adverse events reported |
Numero di eventi avversi riportati |
|
E.5.1.1 | Timepoint(s) of evaluation of this end point |
Week 0 to week 104 |
Dalla settimana 0 alla settimana 104 |
|
E.5.2 | Secondary end point(s) |
1. Incidence of FVIII inhibitors > or = 0.6 BU 2. Number of bleeding episodes on prophylaxis 3. Number of spontaneous bleeding episodes on prophylaxis 4. Haemostatic effect of turoctocog alfa pegol when used for treatment of bleeding episodes assessed as: Excellent, Good, Moderate, or None 5. Number of turoctocog alfa pegol injections required per bleeding episode |
1. Incidenza degli inibitori del FVIII > or = 0,6 BU 2. Numero di episodi di sanguinamento nel trattamento di profilassi 3. Numero di episodi di sanguinamento spontanei nel trattamento di profilassi 4. Effetto emostatico di turoctocog alfa pegol quando usato per il trattamento di episodi di sanguinamento valutati come: Eccellente, Buono, Moderato o Nessuno 5. Numero di infusioni di pegol di turoctocog alfa richieste per episodio di sanguinamento |
|
E.5.2.1 | Timepoint(s) of evaluation of this end point |
1- 5: After 104 weeks |
1-5: dopo 104 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 | Yes |
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 | Yes |
E.6.13.1 | Other scope of the trial description |
Immunogenicity |
immunogenicità |
|
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 | No |
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 | 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 | No |
E.8.2.3 | Other | Yes |
E.8.2.3.1 | Comparator description |
Dosaggio di profilassi una, due o tre volte alla settimana |
Dosing once, twice or three times weekly |
|
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 | 12 |
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 |
Australia |
Brazil |
Canada |
Israel |
Japan |
Korea, Republic of |
Malaysia |
Taiwan |
Turkey |
Ukraine |
United States |
Norway |
Switzerland |
European Union |
|
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 | 2 |
E.8.9.1 | In the Member State concerned months | 7 |
E.8.9.1 | In the Member State concerned days | 2 |
E.8.9.2 | In all countries concerned by the trial years | 2 |
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 | 2 |