E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Haemophilia A with inhibitors Haemophilia B with inhibitors |
Emofilia A con inibitori Emofilia B con inibitori |
|
E.1.1.1 | Medical condition in easily understood language |
Bleeding disorder, inherited deficiency in clotting factor VIII or factor IX with antibodies to clotting factor replacement therapy |
Disturbo della coagulazione, deficenza congenita del fattore VIII o IX della coagulazione con sviluppo di anticorpi contro la terapia sostitutiva del fattore |
|
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 | 10053752 |
E.1.2 | Term | Hemophilia B with anti factor IX |
E.1.2 | System Organ Class | 100000004850 |
|
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 | 10053751 |
E.1.2 | Term | Hemophilia A with anti 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 assess the efficacy of concizumab administered s.c. once daily in preventing bleeding episodes in haemophilia A and B patients with inhibitors |
Valutare l'efficacia di concizumab in somministrazione giornaliera sottocutanea nella prevenzione degli episodi emorragici nei pazienti affetti da Emofilia A e B con inibitori. |
|
E.2.2 | Secondary objectives of the trial |
1. To assess the longer-term efficacy of concizumab in haemophilia patients with inhibitors 2. To assess the safety of concizumab in haemophilia patients with inhibitors 3. To assess the safety of administering recombinant factor VIIa (rFVIIa) to haemophilia patients with inhibitors that are exposed to concizumab 4. To assess the immunogenicity of concizumab in haemophilia patients with inhibitors |
1) Valutare l¿efficacia a lungo termine di concizumab nei pazienti affetti da Emofilia con inibitori. 2) Valutare la sicurezza di concizumab nei pazienti affetti da Emofilia con inibitori 3) Valutare la sicurezza della somministrazione del fattore ricombinante VIIa ai pazienti affetti da emofilia con inibitori esposti a concizumab. 4) Valutare l' immunogenicità di concizumab nei pazienti affetti da emofilia con inibitori. |
|
E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
- Informed consent obtained before any trial related activities. Trial related activities are any procedures that are carried out as part of the trial, including activities to determine the suitability for the trial - Male haemophilia A or B patients with inhibitors aged 18 years or older at the time of signing informed consent - Patients currently in need of treatment with bypassing agents |
-Modulo di consenso informato ottenuto prima di qualsiasi attività correlata allo studio. Per attività correlata allo studio si intende qualunque procedura svolta nell’ ambito della sperimentazione, incluse le attività richieste per stabilire l’idoneità alla partecipazione dei soggetti. -Soggetti di età > oppure = 18 anni di sesso maschile con emofilia A o B con inibitori, al momento della firma del modulo di consenso informato. -Soggetti che al momento dello screening necessitano di un trattamento con agenti bypassanti |
|
E.4 | Principal exclusion criteria |
- Known or suspected hypersensitivity to trial product(s) or related products - Known inherited or acquired bleeding disorder other than haemophilia - Ongoing or planned immune tolerance induction therapy or prophylaxis with FVIII or FIX |
- Ipersensibilità accertata o sospetta al/ai prodotto/i sperimentale/i o ai prodotti correlati. -Accertati disturbi emorragici congeniti o acquisiti diversi dall’emofilia. - Terapia di induzione della tolleranza immunologica in corso o programmata oppure profilassi con FVIII o FIX. |
|
E.5 End points |
E.5.1 | Primary end point(s) |
The number of bleeding episodes during at least 24 weeks from treatment onset. |
Numero di episodi emorragici nelle 24 settimane dall’inizio del trattamento. |
|
E.5.1.1 | Timepoint(s) of evaluation of this end point |
During at least 24 weeks from treatment onset |
Durante almeno 24 settimane dall'inizio del trattamento |
|
E.5.2 | Secondary end point(s) |
- The number of spontaneous bleeding episodes during at least 24 weeks from treatment onset
- Number of treatment emergent adverse events (TEAEs) during at least 24 weeks from treatment onset |
-Il numero di episodi emorragici spontanei nelle 24 settimane dall¿inizio del trattamento
- Il numero di eventi avversi emersi a causa del trattamento (Treatment Emergent Adverse Event, TEAE) nelle 24 settimane dall¿inizio del trattamento |
|
E.5.2.1 | Timepoint(s) of evaluation of this end point |
During at least 24 weeks from treatment onset
All endpoints referring to the time frame of at least 24 weeks will be evaluated in the main part of the trial, defined to end when the last patient has completed a minimum of 24 weeks of dosing treatment with trial product (or has withdrawn). In addition, number of bleeding episodes during 76 weeks of treatment with prophylactic concizumab will be analysed.
|
Durante almeno 24 settimane dall'inizio del trattamento
Tutti gli endpoint in riferimento al periodo di almeno 24 settimane dall'inizio del trattamento saranno valutati nella fase principale dello studio, il cui termine ¿ definito quando l'ultimo paziente ha completato un minimo di 24 settimane di trattamento. |
|
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 | 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 | Yes |
E.8.1.7.1 | Other trial design description |
Con potenziale aumento della dose |
With potential dose escalation |
|
E.8.2 | Comparator of controlled trial |
E.8.2.1 | Other medicinal product(s) | Yes |
E.8.2.2 | Placebo | No |
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 | 2 |
E.8.5 | The trial involves multiple Member States | Yes |
E.8.5.1 | Number of sites anticipated in the EEA | 15 |
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 |
Canada |
Israel |
Japan |
Malaysia |
Ukraine |
United States |
Austria |
Croatia |
Denmark |
Greece |
Italy |
Spain |
Sweden |
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 | 2 |
E.8.9.1 | In the Member State concerned months | 2 |
E.8.9.1 | In the Member State concerned days | 7 |
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 | 2 |
E.8.9.2 | In all countries concerned by the trial days | 7 |