E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Factor X deficiency.
Deficiencia de Factor X |
|
MedDRA Classification |
E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 12.0 |
E.1.2 | Level | LLT |
E.1.2 | Classification code | 10052474 |
E.1.2 | Term | Factor X deficiency |
|
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 |
Evaluar la farmacocinética del Factor X tras una dosis única de 25 UI/kg.
To assess the pharmacokinetics of Factor X after a single dose of 25 IU/kg |
|
E.2.2 | Secondary objectives of the trial |
Evaluar la eficacia de Factor X en el tratamiento de episodios hemorrágicos durante al menos 6 meses. Evaluar la seguridad de Factor X en el tratamiento de episodios hemorrágicos durante al menos 6 meses.
To assess the efficacy of Factor X in the treatment of bleeding episodes over at least 6 months. To assess the safety of Factor X in the treatment of bleeding episodes over at least 6 months. |
|
E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
Consentimiento informado. Al menos 12 años de edad. Con deficiencia hereditaria grave o moderada de Factor X (< 5% de la actividad inicial en el diagnóstico). Tratados actualmente con plasma fresco congelado (PFC), concentrados de complejo de protrombina (CCP) o concentrado de factor IX/X. Haber sufrido al menos una hemorragia espontánea o menorrágica en los 12 últimos meses, que requiriese tratamiento con PFC, CCP o concentrado de factor IX/X. Haber pasado al menos 14 días desde una infusión al paciente de PFC, CCP o concentrado de factor IX/X en la Visita Inicial. Las mujeres en edad fértil no deben estar embarazadas al comienzo del ensayo y deben utilizar un método anticonceptivo durante todo el estudio.
Informed consent At least 12 years of age With hereditary severe or moderate factor X deficiency (<5% basal activity at diagnosis) Currently treated with fresh frozen plasma (FFP), prothrombin complex concentrates (PCC) or factor IX/X concentrate Have had a minimum of one spontaneous or menorrhagic bleed in the last 12 months which required treatment with either FFP, PCC or factor IX/X concentrate At least 14 days since an infusion of FFP, PCC or factor IX/X concentrate at the Baseline Visit Female subjects of childbearing potential must not be pregnant at entry to the study and must practice contraception for the duration of the study. |
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E.4 | Principal exclusion criteria |
Antecedentes de desarrollo de inhibidores a FX o un resultado positivo en la Visita de Selección. Hemorragia en la Visita Inicial. Sujetos con trombocitopenia, enfermedad renal clínicamente significativa o enfermedad hepática clínicamente significativa. Sujetos con otra coagulopatía o trombofilia. Hipersensibilidad comprobada o sospechada al producto en fase de investigación o sus excipientes. Conocimiento del abuso de sustancias químicas o drogas en los 12 últimos meses. Antecedentes de falta de fiabilidad o de colaboración. Participación en otro ensayo en los 30 últimos días, salvo el estudio de cirugía de FX de BPL (número de protocolo Ten03). En esos casos, los pacientes habrán realizado su Visita Fin de Estudio antes o el día de la Visita de Selección para este estudio. Mujeres embarazadas o en periodo de lactancia.
History of inhibitor development to FX, or a positive inhibitor result at the Screening Visit Bleeing at the Baseline Visit Subjects with thrombocytopenia, clinically significant renal disease or clinically significant liver disease Subjects with other coagulopathy or thrombophilia Known or suspected hypersensitivity to the investigational medicinal product or its excipients Known to have abused chemicals or drugs within the past 12 months History of unreliability or non-cooperation Participation in another clinical trial within the past 30 days, with the exception of BPL FX surgery study (protocol No Ten03). In such cases, subjects should have completed their End-of-Study Visit either before or on the day of the Screening Visit for this study Female subjects who are pregnant or lactating. |
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E.5 End points |
E.5.1 | Primary end point(s) |
Los parámetros FC recuperación incremental, vida media (no compartimental), vida media(bicompartimental, si procede), AUC (0-144h), AUC (0-infinito), volumen de distribución, Cmáx y Tmáx para FX:C en Visita Inicial y Visita del mes 6.
The PK parameters incremental recovery, half-life (non-compartmental), half-life (two compartmental, if appropriate), AUC (0-144h), AUC (0-infinity), volume of distribution, Cmax and Tmax for FX:C at Baseline Visit and 6 Month Visit. |
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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 | 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 | No |
E.7 | Trial type and phase |
E.7.1 | Human pharmacology (Phase I) | No |
E.7.1.1 | First administration to humans | Information not present in EudraCT |
E.7.1.2 | Bioequivalence study | Information not present in EudraCT |
E.7.1.3 | Other | Information not present in EudraCT |
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 | Information not present in EudraCT |
E.8.1.2 | Open | Information not present in EudraCT |
E.8.1.3 | Single blind | Information not present in EudraCT |
E.8.1.4 | Double blind | Information not present in EudraCT |
E.8.1.5 | Parallel group | Information not present in EudraCT |
E.8.1.6 | Cross over | Information not present in EudraCT |
E.8.1.7 | Other | Information not present in EudraCT |
E.8.2 | Comparator of controlled trial |
E.8.2.1 | Other medicinal product(s) | Information not present in EudraCT |
E.8.2.2 | Placebo | Information not present in EudraCT |
E.8.2.3 | Other | Information not present in EudraCT |
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 | 6 |
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 | Information not present in EudraCT |
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 |
|
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
|
See protocol Section A-4.4 |
|
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 | 0 |
E.8.9.1 | In the Member State concerned days | 0 |
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 |