Recombinant Factor IX (BAX 326): A Phase 1/3, Prospective, Controlled, Multicenter Study Evaluating Pharmacokinetics, Efficacy, Safety and Immunogenicity in Previously Treated Patients With Severe or Moderately Severe Hemophilia B

CompletadoFase 3ClinicalTrials.gov

ID: NCT01174446Tipo: INTERVENTIONALInicio: 29 de jul de 2010Fin estimado: 3 de may de 2012

Traducción no disponible, mostrando original

Resumen

The purpose of this pivotal Phase 1/3 study is to determine the pharmacokinetic (PK) parameters, the hemostatic efficacy, and the safety of BAX 326, a recombinant factor IX, in previously treated patients (PTPs) with severe and moderately severe hemophilia B.

Elegibilidad

Edad mínima: 12 YearsEdad máxima: 65 YearsSexo: ALL

Criterios de inclusión

Participant is 12 to 65 years old at the time of screening
Participant and/or legal representative has/have provided signed informed consent
Participant has severe (factor IX (FIX) level \< 1%) or moderately severe (FIX level 1-2%) hemophilia B (based on the one stage activated partial thromboplastin time (aPTT) assay), as tested at screening at the central laboratory
Participant is previously treated with plasma-derived or recombinant FIX concentrate(s) for a minimum of 150 exposure days (EDs) (based on the participant's medical records); if a verifiable, documented history is unavailable, the participant can be enrolled if s/he has 100-150 EDs to any FIX product that are not fully documented and has participated in Study 050901 for at least 50 EDs to Immunine prior to enrollment (not valid for US and Japan).
Participant has no evidence of a history of FIX inhibitors
If the participant is to receive prophylactic treatment, the participant is willing to receive prophylactic treatment over a period of 6 months.
If the participant is to receive on-demand treatment, the participant has ≥12 documented bleeding episodes requiring treatment within 12 months prior to enrollment and is willing to receive on-demand treatment for the duration of this study.

Criterios de exclusión

The participant has a detectable FIX inhibitor at screening, with a titer ≥0.6 BU as determined by the Nijmegen modification of the Bethesda assay in the central laboratory
The participant has a history of FIX inhibitors with a titer ≥0.6 Bethesda Units (BU) (as determined by the Nijmegen modification of the Bethesda assay or the assay employed in the respective local laboratory) at any time prior to screening
The participant's weight is \< 35 kg or \> 120 kg
The participant has a history of allergic reaction, eg, anaphylaxis, following exposure to FIX concentrate(s)
The participant has a known hypersensitivity to hamster proteins or recombinant furin (rFurin)
The participant has ongoing or recent evidence of a thrombotic disease, fibrinolysis or disseminated intravascular coagulation (DIC)

Intervenciones

biological

BAX 326

* Study Part 1: Pharmacokinetic (PK) Crossover with BAX326 and BeneFIX * Study Part 2: Open-label evaluation of prophylaxis and on-demand BAX326 only * Study Part 3: Open-label repeat of PK evaluation (repeat Study Part 1) with BAX326 only and same study participants as Study Part 1

biological

BeneFIX

* Study Part 1: Pharmacokinetic (PK) Crossover with BAX326 and BeneFIX. * BeneFIX only used in Part 1 of this study. * Study Part 2 and 3 only utilized BAX326

Ubicaciones

Rosario, Argentina

Patrocinadores

PrincipalBaxalta now part of Shire

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