Inhibitor Development in Previously Untreated Patients (PUPs) or Minimally Blood Component-Treated Patients (MBCTPs) When Exposed to Plasma-derived Von Willebrand Factor-Containing Factor VIII (VWF/FVIII) Concentrates and to Recombinant Factor VIII (rFVIII) Concentrates: An Independent, International, Multicentre, Prospective, Controlled, Randomised, Open Label, Clinical Trial

CompletadoFase 4ClinicalTrials.gov

ID: NCT01064284Tipo: INTERVENTIONALInicio: 1 de ene de 2010Fin estimado: 1 de may de 2015

Traducción no disponible, mostrando original

Resumen

The primary objective of the study is to assess the immunogenicity of VWF/FVIII and of rFVIII concentrates by determining the frequency of inhibitor development in previously untreated patients (PUPs) or minimally blood component-treated (MBCTPs) in the first 50 EDs or in the first 3 years from enrollment, whichever occurs first. .

Descripción detallada

Patients meeting the enrollment criteria will be consecutively enrolled at each participating centre, randomized to be treated exclusively with a single FVIII product either plasma-derived or recombinant, and followed up until inhibitor development or until 50 exposure days (EDs) or 3 years from enrolment have elapsed, whichever comes first. Study products, belonging to the class of rFVIII concentrates and to the class of plasma-derived VWF/FVIII concentrates, will be provided for free to the patients for all the duration of the study

Elegibilidad

Edad mínima: 1 MinuteEdad máxima: 6 YearsSexo: MALE

Criterios de inclusión

Male subjects
Any ethnicity
Age \<6 years
Severe haemophilia A (FVIII:C \<1%), as confirmed at enrolment by the central laboratory.
o Those patients diagnosed locally as severe but subsequently found to have FVIII levels \>= 1% on testing at the central laboratory will be separately recorded in the screening list.
Previously untreated (0 EDs to any FVIII concentrates or blood products) or minimally treated (\<5 EDs) with blood components, namely whole blood, fresh frozen plasma, packed red blood cells, platelets or cryoprecipitate.
o Patients not meeting these criteria will be separately recorded in the screening list.
Negative inhibitor measurement at both local and central laboratory at screening
Ability to comply with study requirements
Signed informed consent of legal tutors o Patients who will not accept to enter into the study or to be randomized will be separately recorded.

Criterios de exclusión

Previous history of FVIII inhibitor
Other congenital or acquired bleeding defects
Plasma FVIII level \>= 1%, as assayed at the central laboratory
o Those patients originally diagnosed locally as severe but subsequently found to have FVIII levels ranging from 1% to 2% on testing at the central laboratory will be separately recorded in the screening list.
Concomitant congenital or acquired immunodeficiency
Concomitant treatment with systemic immunosuppressive drugs
Concomitant treatment with any investigational drug

Intervenciones

drug

PLASMA DERIVED Factor VIII

Maximum dosage : 50IU per kilo. 2-3 times per week or on demand during acute episode of bleeding

drug

Recombinant FVIII

Maximum dosage : 50IU per kilo. 2-3 times per week or on demand during acute episode of bleeding

Ubicaciones

Hospital de Ninos Sor Maria Ludovica La Plata Servicio de Hematologia

La Plata, Buenos Aires, Argentina

Fundacion de la Hemofilia

Buenos Aires, Argentina

Patrocinadores

PrincipalFondazione Angelo Bianchi Bonomi

ColaboradorSintesi Research Srl

Aviso: La información de este ensayo proviene de fuentes públicas y tiene carácter exclusivamente informativo. No constituye asesoramiento médico, regulatorio ni de ningún otro tipo. Las traducciones son automatizadas y no poseen validez oficial. Consultá siempre con un profesional de la salud. Ver Términos de Uso.

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