E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
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E.1.1.1 | Medical condition in easily understood language |
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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 | 10053753 |
E.1.2 | Term | Hemophilia A without inhibitors |
E.1.2 | System Organ Class | 10010331 - Congenital, familial and genetic disorders |
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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 | 10060612 |
E.1.2 | Term | Hemophilia A |
E.1.2 | System Organ Class | 10010331 - Congenital, familial and genetic disorders |
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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 |
The primary objective of the study is to evaluate the long-term safety of rFVIIIFc in subjects with hemophilia A. |
El objetivo principal del estudio es evaluar la seguridad a largo plazo de rFVIIIFc en sujetos con hemofilia A. |
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E.2.2 | Secondary objectives of the trial |
The secondary objective of the study is to evaluate the efficacy of rFVIIIFc in the prevention and treatment of bleeding episodes in subjects with hemophilia A. |
El objetivo secundario del estudio es evaluar la eficacia de rFVIIIFc en la prevención y el tratamiento de episodios de sangrado en sujetos con hemofilia A. |
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
To be eligible to participate in this study, candidates must meet the following eligibility criteria at the time of signing the informed consent at Visit 1 of the study: 1. Ability to understand the purpose and risks of the study and provide signed and dated informed consent and authorization to use protected health information (PHI) in accordance with national and local subject privacy regulations. Parental or guardian consent is required for subjects who are less than 18 years of age or unable to give consent, or as applicable per local laws. Subjects who are less than 18 years of age may provide assent in addition to the parental/guardian consent, if appropriate. 2. Subjects who have completed the studies 997HA301, 8HA02PED, or other Phase 3 studies with rFVIIIFc. |
Para poder participar en este estudio, los candidatos deben reunir los siguientes criterios de participación en el momento de firmar el consentimiento informado en la visita 1 del estudio: 1.Capacidad de comprender el objetivo y los riesgos del estudio y de dar el consentimiento informado firmado y fechado y la autorización para usar información de salud protegida (Protected Health Information, PHI) de acuerdo con la normativa nacional y local sobre la privacidad de los sujetos. El consentimiento del padre, la madre o el tutor es obligatorio para los sujetos menores de 18 años o que no pueden otorgar su consentimiento, o según proceda de acuerdo con las leyes locales. Los sujetos menores de 18 años podrán otorgar su asentimiento además del consentimiento del progenitor/tutor, si es pertinente. 2.Los sujetos que hayan completado los estudios 997HA301, 8HA02PED u otros estudios de fase 3 con rFVIIIFc. |
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E.4 | Principal exclusion criteria |
Candidates will be excluded from study entry if any of the following exclusion criteria exist at the time of signing the informed consent at Visit 1 of the study: 1. Confirmed positive high-titer inhibitor test (?5.00 BU/mL) 2. Medical or other reasons that, in the opinion of the Investigator, make the subject unsuitable for enrollment. |
Los candidatos quedarán excluidos de la participación en el estudio si presentan alguno de los siguientes criterios de exclusión en el momento de firmar el consentimiento informado en la visita 1 del estudio: 1.Prueba de alta concentración de inhibidores con resultado positivo confirmado (? 5,00 UB/ml) 2.Motivos médicos o de otro tipo que, en opinión del investigador, hacen que el sujeto no sea adecuado para la inscripción. |
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E.5 End points |
E.5.1 | Primary end point(s) |
The frequency of inhibitor development |
La frecuencia del desarrollo de inhibidores |
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E.5.1.1 | Timepoint(s) of evaluation of this end point |
On every scheduled clinic visit by Nijmegen-modified Bethesda Assay. |
En cada visita del estudio mediante el método modificado de Nijmegen del ensayo Bethesda. |
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E.5.2 | Secondary end point(s) |
The number of annualized bleeding episodes (spontaneous and traumatic) per subject The number of annualized spontaneous joint bleeding episodes per subject The total number of days of exposure per subject per year The mean dose of rFVIIIFc per kg per subject per year per treatment regimen Physician?s global assessment of response to treatment using a 4-point scale Subject?s assessment of response to treatment using a 4-point scale The incidence of adverse events (AEs) and serious adverse events (SAEs) |
El número de episodios de sangrado (espontáneos y traumáticos) anualizado por sujeto. El número de episodios de sangrado articular espontáneos anualizado por sujeto. El número total de días de exposición por sujeto por año. La dosis media de rFVIIIFc por kg por sujeto por año por pauta posológica. Evaluación global del médico de la respuesta al tratamiento utilizando una escala de 4 puntos. Evaluación del sujeto de la respuesta al tratamiento utilizando una escala de 4 puntos. La incidencia de acontecimientos adversos (AA) y acontecimientos adversos graves (AAG). |
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E.5.2.1 | Timepoint(s) of evaluation of this end point |
The bleeding episodes, the number of treatment days and the mean dose of rFVIIIFc per kg per subject will be monitored by the physician on an ongoing basis, by reviewing patient eDiary information. The physician and subject assessment of response will be taken at every clinic visit. AE and SAE information will be collected at every clinic visit and in the infrequent visit period follow up calls to the patient every 2 months. |
Los episodios de sangrado, el número de días de tratamiento y la dosis media de rFVIIIFc por kilo por sujeto serán monitorizados por el médico del estudio de forma continua mediante la revisión del diario del paciente. La evaluación de la respuesta del médico y del sujeto se harán en cada visita. Se recogerá la información de los AA y AAG en cada visita y en el periodo de seguimiento por teléfono cada dos meses. |
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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 | Yes |
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 | Yes |
E.6.13 | Others | Yes |
E.6.13.1 | Other scope of the trial description |
To evaluate the following treatment regimens: On demand Tailored, Weekly Dosing (modified) and Personalized Prophylaxis and Rehabilitation Period following Surgery |
Para evaluar los siguientes regímenes de tratamiento: A demanda Profilaxis ajustada, semanal (modificada) y personalizada y Periodo de rehabilitación tras una cirugía. |
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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 | 44 |
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 |
Austria |
Belgium |
Brazil |
Canada |
Chile |
China |
France |
Germany |
Hong Kong |
India |
Israel |
Italy |
Japan |
New Zealand |
Poland |
Portugal |
Russian Federation |
South Africa |
Spain |
Sweden |
Switzerland |
United Kingdom |
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
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Last visit last subject |
Última visita del último sujeto. |
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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 | 0 |
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 | 0 |
E.8.9.2 | In all countries concerned by the trial days | 0 |