E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Hemophilia A Severe hemophilia A |
Hemofilia A Hemofilia A Severa |
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E.1.1.1 | Medical condition in easily understood language |
Hemophilia A is a rare but serious bleeding disorder which affects males and is characterized by a deficiency in the plasma protein known as coagulation Factor VIII. |
Hemofilia A is una enfermedad rara pero seria de la sangre que afecta a varones y se caracteriza por una deficiencia en el plasma de una proteina conocida como Factor de coagulación VIII |
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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 | 17.0 |
E.1.2 | Level | LLT |
E.1.2 | Classification code | 10060613 |
E.1.2 | Term | Hemophilia A (Factor VIII) |
E.1.2 | System Organ Class | 100000004850 |
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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 |
The primary objective of this study is to evaluate the safety of long term use of rVIII-SingleChain |
El principal objetivo de este estudio es evaluar la seguridad del uso a largo plazo del factor r VIII de cadena sencilla. |
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E.2.2 | Secondary objectives of the trial |
The secondary objectives of the study are: ? To measure the incidence rate of inhibitor formation to FVIII after 10 EDs and after 50 EDs ? To collect and evaluate additional efficacy information on the prophylaxis and treatment of bleeding events ? To assess the hemostatic efficacy of rVIII-SingleChain for subjects who undergo surgery ? To characterize the safety profile of rVIII-SingleChain |
Los objetivos secundarios del estudio son: -Medir la tasa de incidencia de formación de inhibidores del factor VIII tras 10 DE y tras 50 DE. -Recoger y evaluar información adicional sobre la eficacia de la profilaxis y el tratamiento de los episodios hemorrágicos. -Evaluar la eficacia hemostática del factor rVIII - cadena sencilla en pacientes sometidos a cirugía. -Calificar el perfil de seguridad del factor rVIII - cadena sencilla. |
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E.2.3 | Trial contains a sub-study | Yes |
E.2.3.1 | Full title, date and version of each sub-study and their related objectives |
Subjects may participate in a surgical substudy in which rVIII-SingleChain may be administered before, during and after surgery. The sub-study objective is to assess the hemostatic efficacy of rVIII-SingleChain for subjects who undergo surgery. |
Los pacientes pueden participar en un subestudio quirúrgico en el que rVIII-cadena sencilla pueda ser administrado antes, durante y después de la cirugía. El objetivo del subestudio es evaluar la eficacia hemostática de rVIII -cadena sencilla para pacientes que experimentaron la cirugía |
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E.3 | Principal inclusion criteria |
? Males of any age who have been diagnosed with severe hemophilia A (FVIII activity levels < 1%) ? Participated in a previous CSL-sponsored clinical study with rVIII-SingleChain. " |
-Varones de cualquier edad que hayan sido diagnosticado de hemofilia A ( niveles de actividad FVIII<1%)
-Haber participado en un estudio de investigación previo con factor VIII recombinante de cadena simple patrocinado por CSL. |
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E.4 | Principal exclusion criteria |
? Known or suspected hypersensitivity to rVIII?SingleChain or to any excipients of rVIII?SingleChain or Chinese hamster ovary (CHO) proteins. ? Currently receiving a therapy not permitted during the study. |
-Hipersensibilidad conocida o presunta al factor rVIII - cadena sencilla o a cualquiera de los excipientes del factor rVIII -cadena sencilla o a las proteínas de ovario de hámster chino (OHC).
-Estar recibiendo actualmente algún tratamiento no permitido durante el estudio |
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E.5 End points |
E.5.1 | Primary end point(s) |
The primary outcome measure is the incidence rate of inhibitor formation to FVIII over 100 EDs. |
El criterio de valoración principal es la tasa de incidencia de formación de inhibidores del factor VIII tras 100 DE. |
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E.5.1.1 | Timepoint(s) of evaluation of this end point |
At the closest visit after 100 EDs (up to three years). |
En la visita más cercana después de 100 EDS (hasta tres años). |
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E.5.2 | Secondary end point(s) |
? Treatment success: Rate of treatment success where treatment success of a bleeding episode is defined as a rating of "excellent" or "good" on the investigator's clinical assessment of hemostatic efficacy 4-point scale "excellent, good, moderate or poor/no response". ? Annualized bleeding rate: The annualized bleeding rate for subjects taking prophylaxis and on-demand treatment regimens ? The proportion of bleeding episodes requiring 1, 2, 3, or > 3 infusions of rVIII-SingleChain to achieve hemostasis ? Consumption of rVIII-SingleChain - infusions: The number of rVIII-SingleChain infusions per month and per year ? Consumption of rVIII-SingleChain - IU/kg: rVIII-SingleChain consumption (IU/kg) per month and per year, and per event during prophylaxis, on-demand, and surgery. ? Hemostatic efficacy of rVIII-SingleChain for subjects who undergo surgery: The investigator will rate the efficacy of the rVIII-SingleChain treatment during surgery based on a hemostatic efficacy four point rating scale of "excellent, good, moderate or poor/no response". ? The incidence rate of inhibitor formation to FVIII |
-La evaluación clínica del investigador sobre la eficacia hemostática en el tratamiento de episodios hemorrágicos según una escala ordinal de 4 puntos (excelente, buena, moderada, escasa/ninguna respuesta) -Se resumirá la tasa anualizada de hemorragias (traumáticas y no traumáticas) durante el tratamiento profiláctico y a demanda. -Se tabulará la proporción de episodios hemorrágicos que precisen 1, 2, 3 o más de 3 infusiones del factor rVIII - cadena sencilla para conseguir la hemostasia. -Consumo de rVIII-cadena sencilla-infusión: número de infusiones de rVIII-cadena sencilla por mes y por año -Consumo de rVIII-cadena sencilla-IU/Kg: consumo rVIII-cadena sencilla (IU/kg) por mes y por año y por evento durante profilaxis, o en demanda, y cirugía -Eficacia hemostática de rVIII-cadena sencilla en pacientes sometidos a cirugía: el investigador tasará la eficacia del tratamiento de factor rVIII - cadena sencilla en pacientes sometidos a cirugía sobre la eficacia hemostática en el tratamiento de episodios hemorrágicos según una escala ordinal de 4 puntos (excelente, buena, moderada, escasa/ninguna respuesta). -Tasa de incidencia de la formación del inhibidor FVIII |
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E.5.2.1 | Timepoint(s) of evaluation of this end point |
For secondary end points 1 to 5: Up to three years For secondary end point 6: From the start of surgery through the post-operative recovery (generally up to 14 days after surgery) For secondary end point 7: After 10 and after 50 exposure days |
Criterios secundarios de valoración del 1-5: hasta 3 años Criterios secundarios de valoración 6: desde el comienzo de la cirugía hasta la recuperación postoperatorio (generalmente hasta 14 días después de la cirugía) Para el criterio secundario de valoración punto 7: después de 10 días y después de 50 días de exposición. |
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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 | 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 | 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 | Yes |
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) | 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
| 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 | 4 |
E.8.5 | The trial involves multiple Member States | Yes |
E.8.5.1 | Number of sites anticipated in the EEA | 55 |
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 |
Canada |
Chile |
European Union |
Georgia |
Japan |
Korea, Democratic People's Republic of |
Lebanon |
Malaysia |
Philippines |
Russian Federation |
South Africa |
Switzerland |
Thailand |
Turkey |
Ukraine |
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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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 |