E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Haemophilia A |
Hemofilija A |
|
E.1.1.1 | Medical condition in easily understood language |
Bleeding disorder, inherited deficiency in clotting factor VIII |
Poremećaj krvarenja, nasljedni nedostatak faktora 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. |
Procjena sigurnosti kontinuirane primjene lijeka turoktokog alfa pegol (N8-GP) u prevenciji i liječenju epizoda krvarenja u ispitanika s teškom hemofilijom A koji su prethodno liječeni s N8-GP. |
|
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 |
Istražiti sljedeće u bolesnika s teškom hemofilijom A, prethodno liječenih s lijekom turoktog alfa pegol 1. Istražiti razvoj FVIII inhibitora 2. Procjena učinkovitost N8-GP u prevenciji krvarenja 3. Procjena hemostatske učinkovitosti kada se N8-GP koristi u liječenju krvarenja
|
|
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. Muški ispitanici s teškom urođenom hemofilijom A (aktivnost FVIII <1%, prema medicinskim podacima) 2. Trenutno sudjelovanje u kliničkom ispitivanju NN7088-3859 (pahtfinder2) ili NN7088-3885 (pathfinder5) |
|
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. Poznata preosjetljivost ili sumnja na preosjetljivost na ispitivani lijek uključujući alergiju na protein hrčka ili srodne proizvode 2. Bilo koji poremećaj, osim stanja povezanih s hemofilijom, koji po mišljenju ispitivača može ugroziti sigurnost bolesnika ili pridržavanje protokola 3. Trenutačno sudjelovanje u bilo kojem kliničkom ispitivanju (osim NN7088-3859 (pathfinder2) ili NN7088-3885 (pathfinder5)) odobrenog ili neodobrenog ispitivanog lijeka
|
|
E.5 End points |
E.5.1 | Primary end point(s) |
Number of adverse events reported |
Broj prijavljenih štetnih događaja |
|
E.5.1.1 | Timepoint(s) of evaluation of this end point |
Week 0 to week 104 |
0-104 tjedna |
|
E.5.2 | Secondary end point(s) |
1. Incidence of FVIII inhibitors ≥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. Stopa incidencije FVIII inhibitora ≥0,6 BU 2. Godišnja stopa krvarenja u u profilaktičnom liječenju 3. Broj spontanih epizoda krvarenja u profilaktičkom liječenju 4. Hemostatsko djelovanje N8-GP pri liječenju krvarenja, procijenjeno kao izvrsno, dobro, umjereno i bez djelovanja 5. Broj injekcija N8-GP potrebnih po epizodi krvarenja
|
|
E.5.2.1 | Timepoint(s) of evaluation of this end point |
1. - 5: After 104 weeks |
1. - 5: Nakon 104 tjedna |
|
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 | Yes |
E.6.13.1 | Other scope of the trial description |
Immunogenicity |
Imunogeničnost |
|
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 |
Doziranje jedan, dva ili tri puta tjedno |
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
| Yes |
E.8.4 | The trial involves multiple sites in the Member State concerned | No |
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 | No |
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 |
European Union |
Israel |
Japan |
Korea, Republic of |
Malaysia |
Norway |
Switzerland |
Taiwan |
Turkey |
Ukraine |
United States |
|
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 |