E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Haemophilia A |
Hemofilia A |
|
E.1.1.1 | Medical condition in easily understood language |
Bleeding disorder, inherited deficiency in clotting factor VIII |
Desorden de la coagulación, deficiencia heredada del factor VIII de la coagulación |
|
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. |
Investigar la seguridad del turoctocog alfa pegol durante su uso continuo para la prevención y el tratamiento de los episodios hemorrágicos de pacientes con hemofilia A grave tratados previamente 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 |
Investigar todo lo siguiente en pacientes con hemofilia A grave tratados previamente con turoctocog alfa pegol: 1. Desarrollo de inhibidores contra FVIII. 2. Eficacia de la profilaxis con turoctocog alfa pegol. 3. Eficacia hemostática de turoctocog alfa pegol cuando se utiliza como tratamiento de hemorragias. |
|
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. Varones de cualquier edad con el diagnóstico de hemofilia A congénita grave (actividad del FVIII < 1%) según la historia clínica. 2. Participación en curso en el estudio NN7088-3859 (pathfinder2) o NN7088-3885 (pathfinder5) en el momento de la transferencia. |
|
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. Hipersensibilidad confirmada o presunta al producto del ensayo, incluida la alergia a proteínas de hámster o a otros productos relacionados. 2. Cualquier trastorno, excepto los procesos asociados a la hemofilia, que en opinión del investigador podría poner en peligro la seguridad del paciente o el cumplimiento del protocolo. 3. Participación actual en cualquier ensayo clínico (excepto el NN7088-3859 (pathfinder2) o NN7088-3885 (pathfinder5)) de un medicamento en investigación aprobado o no aprobado. |
|
E.5 End points |
E.5.1 | Primary end point(s) |
Number of adverse events reported |
Número de acontecimientos adversos notificados |
|
E.5.1.1 | Timepoint(s) of evaluation of this end point |
Week 0 to week 104 |
Semana 0 a semana 104 |
|
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. Incidencia de inhibidores del FVIII ≥ 0,6 UB. 2. Número de episodios hemorrágicos durante la profilaxis. 3. Número de episodios hemorrágicos espontáneos durante la profilaxis. 4. Efecto hemostático del turoctocog alfa pegol cuando se utiliza como tratamiento de episodios hemorrágicos, evaluado como: excelente, bueno, moderado o nulo. 5. Número de inyecciones de turoctocog alfa pegol necesarias por episodio hemorrágico. |
|
E.5.2.1 | Timepoint(s) of evaluation of this end point |
1. - 5: After 104 weeks |
1. - 5: Después de 104 semanas |
|
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 |
Inmunogenicidad |
|
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 |
Administración de dosis una, dos o 3 veces por semana |
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 | 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
|
LVLS |
UVUS (Última Visita del Último Sujeto) |
|
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 | 6 |
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 |