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 haemophilia A |
Trastorno hemorrágico: Deficiencia del Factor 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 | 14.1 |
E.1.2 | Level | LLT |
E.1.2 | Classification code | 10018937 |
E.1.2 | Term | Haemophilia A |
E.1.2 | System Organ Class | 10010331 - Congenital, familial and genetic disorders |
|
E.1.3 | Condition being studied is a rare disease | No |
E.2 Objective of the trial |
E.2.1 | Main objective of the trial |
To evaluate safety of NNC 0155-0000-0004 in paediatric previously untreated patients (PUP) with haemophilia A |
Evaluar la seguridad de NNC 0155-0000-0004 en pacientes pediátricos con hemofilia A sin tratamiento previo. |
|
E.2.2 | Secondary objectives of the trial |
- To evaluate efficacy of NNC 0155-0000-0004 in treatment of bleeds in paediatric PUP with haemophilia A - To evaluate preventive effect of NNC 0155-0000-0004 on bleeds in paediatric PUP with haemophilia A |
- Evaluar la eficacia y la seguridad de NNC 0155-0000-0004 para el tratamiento de las hemorragias en pacientes pediátricos con hemofilia A previamente no tratados. - Evaluar el efecto preventivo de NNC 0155-0000-0004 para las hemorragias en pacientes pediátricos con hemofilia A sin tratamiento previo. |
|
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 procedure that would not have been performed during normal management of the patient) - Male patients with congenital severe haemophilia A (baseline level FVIII <=1%) - Age < 6 years - No prior use of purified clotting factor products (previous exposure to blood components is acceptable) |
- Consentimiento informado obtenido antes de cualquier actividad relacionada con el ensayo (son actividades relacionadas con el ensayo todas las que no se habrían realizado para el tratamiento normal del paciente). - Varones con hemofilia A congénita grave (valor basal del FVIII ? 1 %) - Edad < 6 años - Ausencia de tratamiento previo con productos purificados de factores de la coagulación (se acepta la exposición previa a hemoderivados) |
|
E.4 | Principal exclusion criteria |
- Known or suspected allergy to hamster protein or intolerance to trial product(s) or related products - Previous participation in this trial defined as withdrawal after administration of trial product - Congenital or acquired coagulation disorders other than haemophilia A - FVIII inhibitor (? 0.6 BU/mL) at screening - Ongoing treatment or planned treatment during the trial with immunomodulatory agents (e.g. intravenous immunoglobulin (IVIG)), routine systemic corticosteroids) - Platelet count <50,000 platelets/?L |
- Alergia confirmada o sospechada a las proteínas de hámster, o intolerancia a los productos del ensayo o productos afines. - Participación previa en este ensayo, definida como retirada tras la administración de producto del ensayo. - Trastornos de la coagulación congénitos o adquiridos distintos de la hemofilia A. - Presencia de inhibidores del FVIII (? 0,6 UB/ml) en el momento de la selección. - Tratamiento con inmunomoduladores (por ejemplo, inmunoglobulina intravenosa [IGIV]) y corticoesteroides sistémicos clásicos) - Recuento de plaquetas <50.000plaquetas/?l |
|
E.5 End points |
E.5.1 | Primary end point(s) |
Incidence rate of FVIII inhibitors (?0.6 BU/mL) |
Tasa de incidencia de inhibidores del FVIII (? 0,6 UB/ml). |
|
E.5.1.1 | Timepoint(s) of evaluation of this end point |
Will be evaluated from Visit 2 to End of Trial Visit/Visit 5 |
Serán evaluados desde Visita 2 a Visita final del estudio/Visita 5 |
|
E.5.2 | Secondary end point(s) |
- Haemostatic effect of NNC 0155-0000-0004 on treatment of bleeds assessed on a predefined four point scale: Excellent, Good, Moderate and None - Annualized bleeding rate |
- Efecto hemostático de NNC 0155-0000-0004 para el tratamiento de las hemorragias, evaluado en una escala de cuatro puntos predefinidos: excelente, bueno, moderado. - El índice anual de hemorragias |
|
E.5.2.1 | Timepoint(s) of evaluation of this end point |
Will be evaluated from Visit 2 to End of Trial Visit/Visit 5 |
Serán evaluados desde Visita 2 hasta la Visita final del estudio/ Visita 5. |
|
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 | 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) | 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 | No |
E.8.1.1 | Randomised | No |
E.8.1.2 | Open | No |
E.8.1.3 | Single blind | No |
E.8.1.4 | Double blind | No |
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 | No |
E.8.2.3 | Other | No |
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 | 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 |
Austria |
Brazil |
China |
Croatia |
Czech Republic |
Greece |
Hong Kong |
Japan |
Malaysia |
Russian Federation |
Serbia |
Spain |
Turkey |
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
|
LPLV |
Última visita del último paciente |
|
E.8.9 Initial estimate of the duration of the trial |
E.8.9.1 | In the Member State concerned years | 4 |
E.8.9.1 | In the Member State concerned months | 3 |
E.8.9.1 | In the Member State concerned days | 0 |
E.8.9.2 | In all countries concerned by the trial years | 4 |
E.8.9.2 | In all countries concerned by the trial months | 3 |
E.8.9.2 | In all countries concerned by the trial days | 0 |