Estudio abierto para evaluar el tratamiento profiláctico y caracterizar la eficacia, seguridad y farmacocinética del Factor VIII recombinante de dominio B delecionado libre de albúmina (moroctocog alfa [AF-CC]) en niños con hemofilia A

CompletadoFase 3ClinicalTrials.gov

ID: NCT00543439Tipo: INTERVENTIONALInicio: 1 de dic de 2007Fin estimado: 1 de abr de 2018

Resumen

El propósito de este estudio de investigación es determinar la efectividad, seguridad y farmacocinética (FK) de moroctocog alfa (AF-CC) en sujetos previamente tratados, menores de 6 años de edad, con hemofilia A grave o moderadamente grave.

Elegibilidad

Edad mínima: 6 MonthsEdad máxima: 15 YearsSexo: MALE

Criterios de inclusión

Male subjects, aged less than 6 years, with moderately severe to severe hemophilia A.
A negative FVIII inhibitor titer at screening, and a medical history negative for a past FVIII inhibitor.
At least 20 exposure days to any FVIII replacement product.
Adequate hepatic and renal function
CD4 count \> 400 cells/uL, and if receiving antiviral therapy must be on a stable regimen
Additional criteria for subjects participating in the PK assessment:
Male subjects as described immediately above except they must have a FVIII Activity of less than or equal to 1% confirmed by the central laboratory screening test
Age \< 6 years at time of PK assessment.
The subject's size is sufficient to permit PK-related phlebotomy.
The subject is able to comply with the procedures conducted during the PK assessment, including a mandatory 72-hour washout period preceding the PK assessment.

Criterios de exclusión

A history of FVIII inhibitor.
Presence of a bleeding disorder in addition to hemophilia A.
Treatment with any investigational drug or device within 30 days before the time of signing the informed consent form.
Major or orthopedic surgery planned to occur during the course of the study.
Regular (e.g., daily, every other day) use of antifibrinolytic agents or medications known to influence platelet function such as aspirin or certain nonsteroidal anti-inflammatory drugs (NSAIDs), or regular, concomitant therapy with immunomodulating drugs (e.g., intravenous immunoglobulin \[IVIG\], routine systemic corticosteroids).
Known hypersensitivity to hamster protein.

Intervenciones

biological

Moroctocog alfa (AF-CC)

On-demand therapy for 6 months, followed by routine prophylaxis 25 IU/kg, administered every other day for 1 year.

biological

Moroctocog alfa (AF-CC)

Routine prophylaxis crossover: 45 IU/kg, administered 2 times a week for 1 year followed by 25 IU/kg administered every other day for 1 year, or, 25 IU/kg, administered every other day for 1 year, followed by 45 IU/kg, administered 2 times a week for 1 year.

Ubicaciones

Fundacion de la Hemofilia

Buenos Aires, Argentina

Patrocinadores

PrincipalPfizer

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Estudio abierto para evaluar el tratamiento profiláctico ... | EligiMed