Resumen

Objetivo primario: - Evaluar la seguridad a largo plazo de BIVV001 en sujetos previamente tratados con hemofilia A. Objetivos secundarios: - Evaluar la eficacia de BIVV001 como tratamiento profiláctico. - Evaluar la eficacia de BIVV001 en el tratamiento de episodios de sangrado. - Evaluar el consumo de BIVV001 para la prevención y el tratamiento de episodios de sangrado. - Evaluar el efecto de la profilaxis con BIVV001 sobre los resultados de salud articular. - Evaluar el efecto de la profilaxis con BIVV001 sobre los resultados de calidad de vida (CdV). - Evaluar la seguridad y tolerabilidad del tratamiento con BIVV001. - Evaluar la farmacocinética (FK) de BIVV001 basada en el tiempo de tromboplastina parcial activada (aPTT) de una etapa y los ensayos de actividad cromogénica de FVIII de dos etapas (aplicable únicamente al grupo B). - Evaluar la eficacia de BIVV001 para el manejo perioperatorio.

Descripción detallada

Los participantes recibirán BIVV001 una vez por semana durante un total de al menos 100 días de exposición a BIVV001 (incluida la exposición durante un estudio original con BIVV001, si corresponde). Los participantes tendrán la oportunidad de continuar en este estudio durante hasta 4 años, a menos que BIVV001 esté disponible comercialmente en su país de participación.

Elegibilidad

Sexo: ALL

Criterios de inclusión

For participants rolling over into Arm A
Participants who have completed the studies EFC16923, EFC16925, Arm B or Arm C of the current study, or any other potential BIVV001 study.
Male or Female
For participants new to BIVV001 (Arm B and C)
Participants who have severe hemophilia A, defined as \<1 IU/dL (\<1%) endogenous FVIII activity as documented either by central laboratory testing at screening or in historical medical records from a clinical laboratory demonstrating \<1% FVIII coagulant activity (FVIII:C) or a documented genotype known to produce severe hemophilia A.
Previous treatment for hemophilia A (prophylaxis or on-demand) with any recombinant and/or plasma-derived FVIII, or cryoprecipitate for at least 150 EDs or 50 EDs for participants aged \<6 years.
Platelet count ≥100 000 cells/μL at screening.
A participant known to be human immunodeficiency virus (HIV) antibody positive, either previously documented or identified from screening assessments, must have the following results prior to enrollment: CD4 lymphocyte count \>200 cells/mm³ and viral load of \<400 000 copies/mL
Male
Only for Arm B: Chinese participants
Only for Arm C: planned major surgery within 6 months after Day 1.

Criterios de exclusión

For participants rolling over into Arm A
Positive inhibitor result, defined as ≥0.6 Bethesda units (BU)/mL.
Participation in another study.
For participants new to BIVV001 (Arm B and Arm C)
Any concurrent clinically significant liver disease that, in the opinion of the Investigator, would make the participant unsuitable for enrollment. This may include, but is not limited to cirrhosis, portal hypertension, and acute hepatitis.
Serious active bacterial, fungal, or viral infection (other than chronic hepatitis or HIV) present within 30 days of screening.
Other known coagulation disorder(s) in addition to hemophilia A.
History of hypersensitivity or anaphylaxis associated with any FVIII product.
History of a positive inhibitor (to FVIII) test defined as ≥0.6 BU/mL, or any value greater than or equal to the lower sensitivity cut-off for laboratories with cut-offs for inhibitor detection between 0.7 and 1.0 BU/mL, or clinical signs or symptoms of decreased response to FVIII administrations. Family history of inhibitors will not exclude the participant.
Positive inhibitor test (FVIII) result, defined as ≥0.6 BU/mL at screening.
Treatment with acetylsalicylic acid (ASA) or antiplatelet agents that are not nonsteroidal anti-inflammatory drugs (NSAIDs) within 2 weeks prior to screening.
Treatment with NSAIDs greater than the maximum dose specified in the regional prescribing information within 2 weeks prior to screening.
Systemic treatment within 12 weeks prior to Screening with chemotherapy and/or other immunosuppressive drugs (except for the treatment of hepatitis C virus \[HCV\] or HIV).
Emicizumab use within the 20 weeks prior to screening.
Major surgery within 8 weeks prior to screening.
The above information is not intended to contain all considerations relevant to a patient's potential participation in a clinical trial.

Estudio de fase 3, abierto, multicéntrico, para evaluar la seguridad y eficacia a largo plazo de la proteína de fusión intravenosa recombinante factor de coagulación VIII Fc-factor de von Willebrand-XTEN (rFVIIIFc-VWF-XTEN; BIVV001) en pacientes con hemofilia A grave previamente tratados

Resumen

Descripción detallada

Elegibilidad

Criterios de inclusión

Criterios de exclusión

Intervenciones

efanesoctocog alfa (BIVV001)

Ubicaciones

Investigational Site Number : 0320001

Investigational Site Number : 0320002

Investigational Site Number : 0320003

Patrocinadores