E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Hemophilia A or B with FVIII or FIX inhibitors |
Hemofilia A o B con inhibidores de FVIII o FIX |
|
E.1.1.1 | Medical condition in easily understood language |
Hemophilia A or B with FVIII or FIX inhibitors |
Hemofilia A o B con inhibidores de FVIII o FIX |
|
E.1.1.2 | Therapeutic area | Diseases [C] - Blood and lymphatic diseases [C15] |
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 | 10053751 |
E.1.2 | Term | Hemophilia A with anti factor VIII |
E.1.2 | System Organ Class | 100000004850 |
|
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 | 10053752 |
E.1.2 | Term | Hemophilia B with anti factor IX |
E.1.2 | System Organ Class | 100000004850 |
|
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 the safety and efficacy of rFVIIa BI in the treatment of acute bleeding episodes per an on demand regimen in hemophilia A or B patients with inhibitors |
Evaluar la seguridad y la eficacia de rFVIIa BI en el tratamiento de los episodios hemorrágicos agudos mediante un régimen a demanda en pacientes con hemofilia A o B con inhibidores |
|
E.2.2 | Secondary objectives of the trial |
To determine or evaluate: ? Treatment response based on a four-point scale ? Total percentage of subjects with sustained bleeding control for all acute bleeding episodes at 24 hours after infusion ? Safety and tolerability of treatment regimens ? Inhibitor development to FVII ? Treatment success rate of joint vs. non joint bleeding episodes ? Treatment success rates of severe vs. mild/moderate bleeds |
Determinar o evaluar: - La respuesta al tratamiento basada en una escala de cuatro puntos - El porcentaje total de sujetos con mantenimiento del control de la hemorragia en todos los episodios hemorrágicos agudos a las 24 horas de la infusión - La seguridad y la tolerabilidad de los regímenes de tratamiento - El desarrollo de inhibidores ante el FVII - La tasa de éxito del tratamiento de los episodios hemorrágicos articulares frente a los no articulares - Las tasas de éxito del tratamiento de las hemorragias graves frente a las leves o moderadas. |
|
E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
? Subject is male with hemophilia A or B with inhibitors, with a high titer (?5 BU) or a historical high anamnestic response. ? Subject is 12 to 65 years old at the time of screening. ? Subject is currently using or has used bypassing agents for treatment of bleeding episodes. |
- El sujeto es un varón que padece hemofilia A o B con inhibidores, con un valor elevado (=>5 UB) o una elevada respuesta anamnésica en sus antecedentes. - El sujeto tiene entre 12 y 65 años de edad en el momento de la selección. - El sujeto se encuentra utilizando actualmente o ha utilizado agentes de derivación para el tratamiento de los episodios hemorrágicos. |
|
E.4 | Principal exclusion criteria |
? Subject has a known hypersensitivity to rFVIIa, hamster or murine proteins, or Tween 80. ? Subject has a prior history of thromboembolic event ? Subject is positive for a FVII inhibitor at screening |
- El sujeto que posee hipersensibilidad conocida al rFVIIa, a las proteínas murinas o del hámster o al Tween 80. - El sujeto que posee antecedentes de un episodio tromboembólico. - El sujeto es positivo a un inhibidor del FVII en el momento de la selección. |
|
E.5 End points |
E.5.1 | Primary end point(s) |
The primary outcome measure is no additional hemostatic product required within 12 hours of the first dose of IP, other than the prescribed dosing regimen (up to 3 × 90 µg/kg doses or 1 × 270 µg/kg dose). |
El éxito del tratamiento de los episodios hemorrágicos: se define como la no necesidad de producto hemostático adicional dentro de las 12 horas siguientes a la primera dosis otro que no sea el régimen de administración prescrito (hasta 3 × 90 ?g/kg o 1 × 270 ?g/kg). |
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E.5.1.1 | Timepoint(s) of evaluation of this end point |
The endpoint is evaluated 12 hours after first dose. |
La variable se evalua 12 horas después de la primera dosis. |
|
E.5.2 | Secondary end point(s) |
? Treatment response based on a four-point scale ? Total percentage of clinical responders (sustained bleeding control) for all acute bleeding episodes at 24 hours after infusion ? Safety and tolerability of treatment regimens by clinical assessment of related AEs ? Inhibitor development to FVII |
-La respuesta al tratamiento basada en una escala de cuatro puntos - El porcentaje total de sujetos que responden al tratamiento (con mantenimiento del control de la hemorragia) en todos los episodios hemorrágicos agudos a las 24 horas de la infusión. - Seguridad y tolerabilidad de los regímenes de tratamiento mediante evaluación clínica de los acontecimientos adversos relacionados - El desarrollo de inhibidores frente al FVII. |
|
E.5.2.1 | Timepoint(s) of evaluation of this end point |
Treatment response at the end of treatment and 24 hours Safety endpoint continuously |
La respuesta al tratamiento al final del tratamiento y a las 24 horas. La variable de seguridad de forma continua. |
|
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 | 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 | 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 | 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 | 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 |
se comparan 2 regímenes de dosificación |
2 dosage regimens are compared |
|
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 | 10 |
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 |
Belgium |
Bulgaria |
India |
Japan |
Poland |
Romania |
Russian Federation |
Spain |
Ukraine |
United States |
|
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
|
LVLS |
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 | 1 |
E.8.9.1 | In the Member State concerned months | 5 |
E.8.9.1 | In the Member State concerned days | 13 |
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 | 0 |
E.8.9.2 | In all countries concerned by the trial days | 0 |