E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Haemophilia A |
Hemofilia A |
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E.1.1.1 | Medical condition in easily understood language |
Bleeding disorder, inherited deficiency in clotting factor VIII |
Trastorno hemorrágico congénico por deficiencia de factor VIII de coagulación |
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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 | 19.0 |
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 evaluate and compare the single-dose pharmacokinetic of turoctocog alfa pegol from the pivotal process with turoctocog alfa pegol from the commercial process, each given as intravenous administrations of 50 U/kg to patients with severe haemophilia A |
Evaluar y comparar la farmacocinética de dosis única de turoctocog alfa pegol a partir del proceso pivotal y de turoctocog alfa pegol a partir del proceso comercial, cada uno administrado por vía intravenosa en una dosis de 50 U/kg a pacientes con hemofilia A grave |
|
E.2.2 | Secondary objectives of the trial |
To assess the safety of turoctocog alfa pegol from the pivotal process and turoctocog alfa pegol from the commercial process after single intravenous doses of 50 U/kg in patients with severe haemophilia A |
Evaluar la seguridad de turoctocog alfa pegol a partir del proceso pivotal y turoctocog alfa pegol a partir del proceso comercial tras dosis intravenosas únicas de 50 U/kg en pacientes con hemofilia A grave |
|
E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
1. 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 suitability for the trial. 2. Ongoing participation in pathfinder™2 (NN7088-3859) 3. Male, age ≥ 12 years at the time of signing informed consent (in certain countries the lower age limit will be 18 years, according to local requirements) |
1.Obtención del consentimiento informado antes de realizar ninguna actividad relacionada con el ensayo. Se consideran actividades relacionadas con el ensayo todos los procedimientos que se lleven a cabo como parte del ensayo, incluidas las actividades para determinar la idoneidad para participar en él. 2.Participación actual en pathfinder™2 3.Varones ≥ 12 años de edad en el momento de la firma del consentimiento informado (en algunos países, el límite de edad inferior será de 18 años, de conformidad con las normas locales) |
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E.4 | Principal exclusion criteria |
1. FVIII inhibitors (≥0.6 BU) at last visit in pathfinder™2 prior to entry in pathfinder™7 2. Planned surgery during the trial 3. Major surgery performed within 4 weeks prior to screening 4. Previous participation in this trial. Participation is defined as signed informed consent 5. Any disorder, except for conditions associated with haemophilia A, which in the investigator’s opinion might jeopardise patient’s safety or compliance with the protocol |
1.Inhibidores del FVIII (≥ 0,6 UB) en la última visita de pathfinder™2 antes de incorporarse a pathfinder™7 2.Intervención quirúrgica programada durante el ensayo 3.Intervención de cirugía mayor realizada en las 4 semanas previas a la selección 4.Participación previa en este ensayo. La participación se define como la firma del consentimiento informado. 5.Cualquier trastorno, salvo las afecciones asociadas a la hemofilia A, que en opinión del investigador pueda poner en peligro la seguridad del paciente o el cumplimiento del protocolo 6.Participación en cualquier ensayo clínico (excepto pathfinder™2) de un producto en investigación aprobado o no aprobado |
|
E.5 End points |
E.5.1 | Primary end point(s) |
Area under the FVIII activity-time curve - dose normalised to 50 U/kg (AUC0-96h, norm) |
Área bajo la curva de actividad del FVIII-tiempo entre 0 y 96 horas después de la inyección - con normalización de la dosis a 50 U/kg (AUC0-96 h, norm) |
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E.5.1.1 | Timepoint(s) of evaluation of this end point |
From 0 to 96 h post injection |
De 0 a 96 horas después de la inyección. |
|
E.5.2 | Secondary end point(s) |
The key secondary pharmacokinetic endpoints are for the first and second pharmacokinetic periods, separately.
The following pharmacokinetic endpoints will be derived based on plasma FVIII activity measured:
1. FVIII activity 30 min post administration - dose normalised to 50 U/kg 2. Area under the FVIII activity-time curve from 0 to infinity 3. Clearance 4. Incremental recovery 5. Terminal half-life
All blood samples for the pharmacokinetic assessment will be analysed using both chromogenic and one-stage clotting assays. |
Los criterios de valoración farmacocinéticos secundarios más importantes son para los periodos farmacocinéticos primero y segundo por separado. Se obtendrán los siguientes criterios de valoración farmacocinéticos basándose en la actividad plasmática del FVIII, medida desde el momento de la administración del producto del ensayo hasta 96 horas después de la dosis: •Actividad del FVIII 30 minutos después de la administración - con normalización de la dosis a 50 U/kg •Área bajo la curva de actividad del FVIII-tiempo entre 0 e infinito •Aclaramiento •Recuperación incremental •Semivida terminal Todas las muestras de sangre para la evaluación farmacocinética se analizarán mediante análisis de coagulación monofásico y cromogénico. |
|
E.5.2.1 | Timepoint(s) of evaluation of this end point |
From time of trial product administration to 96 hours post-dose |
Desde el momento de la administración del producto en investigación hasta 96 horas después de la inyección. |
|
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 | No |
E.6.6 | Pharmacokinetic | Yes |
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 |
Comparison between N8-GP from the pivotal and commercial process |
Comparación entre el N8-GP obtenido mediante el proceso pivotal y el N8-GP obtenido mediante el proceso comercial |
|
E.7 | Trial type and phase |
E.7.1 | Human pharmacology (Phase I) | Yes |
E.7.1.1 | First administration to humans | No |
E.7.1.2 | Bioequivalence study | No |
E.7.1.3 | Other | Yes |
E.7.1.3.1 | Other trial type description |
Pharmacokinetic |
Farmacocinética |
|
E.7.2 | Therapeutic exploratory (Phase II) | No |
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 | No |
E.8.1.3 | Single blind | No |
E.8.1.4 | Double blind | Yes |
E.8.1.5 | Parallel group | No |
E.8.1.6 | Cross over | Yes |
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 |
N8-GP obtenido a partir de otro proceso de producción |
N8-GP from another production process |
|
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
| 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 | 7 |
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 |
European Union |
United States |
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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 | 0 |
E.8.9.1 | In the Member State concerned months | 6 |
E.8.9.1 | In the Member State concerned days | 7 |
E.8.9.2 | In all countries concerned by the trial years | 0 |
E.8.9.2 | In all countries concerned by the trial months | 6 |
E.8.9.2 | In all countries concerned by the trial days | 7 |