Dettagli per singolo studio

DEC-NET Serial number ES464

Published online	12/09/2005 10.19.00
Last updated	24/04/2006 8.51.54

Other protocol ID number	3090A1-304-WW
Current trial status	Complete(closed to recruitment of participants: follow-up complete)
Major Disease (ICD9 class)	CONGENITAL FACTOR IX DISORDER
Experimental drug
recombinant factor IX (reformuled: rFIX.-R) Treatment regimen (dosage and duration) N/A
Gender	Both
Age (range)	> or = 12 years old
Eligibility criteria
Inclusion criteria
12 years old patients or older, with moderate-severe hemophilia B (level of activity of factor IX in plasma [FIX:C] < or = 2%) previously treated with FIX for 150 days (at least), with appropriate hepatic, renal and medular function, lymphocyte CD4 >400/mcl and treated with the last dose of FIX at least 5 days before the first dose.
Exclusion criteria
Patients with some level of factor IX inhibitor (> or = 0,6 Bethesda units), history of presence of factor IX inhibitor, other clotting disorders, active hepatitis, planned surgery during the first part of the study, and patients treated with immunomodulators or, cronically, with systemic corticosteroids; and other.
Trial design/methodology
Phase	3
Kind of study	Efficacy Prophylaxis Safety Pharmacokinetics
Design	Controlled Randomised Blinded Cross-over Después hay una segunda fase abierta en que los pacientes recibirán r-FIX-R a demanda si se presenta una hemorragia, como tratamiento profiláctico y según precisen en caso de intervención quirúrgica.
Purpose of study
Main objective: to stablish the bioequivalence of two formulations of recombinant factor IX, r-FIX and r-FIX-R, in patients with moderate or severe hemophilia B previously treated.
Primary outcomes
Pharmacokinetics (area under the curve until infinite and AUC from 0 to 72 hours)
Secondary outcomes
-Another pharmacokinetic parameters · maximal concentration · clearance · half life elimination -Efficacy parameters ·number of infusion bolus and dose per hemorrhagic episode ·appreciation of the response (by the patient and the investigator) of the on-demand treatment ·response in the per operatory period (dose, hemorrhage, hemostasia and transfusions) ·global evaluation of the efficacy by the investigator -Toxicity parameters ·clinical and laboratory adverse reactions ·development of anti-FIX antibodies ·thrombogenesis ·allergic manifestations ·aglutination of hematies ·seroconversion to hepatitis virus A, B, C and HIV.
Summary of study design, objectives, and ongoing research findings
The first part of this study is a double-blind, randomized and cross-over study to assess the bioequivalence of rFIX and rFIX-R at a dosage of 75 UI/kg. This dose has been selected based on the Note for Guidance on the Clinical Investigation of Recombinant Factor VIII and IX products of the CPMP. Patients will be randomized to receive rFIX or 1 of 3 set of rFIX-R as a first treatment. Several samples will be obtained to asses the activity of factor IX in plasma before and after the injection, in several specific points during a 72 hours period after the infusion. After a washout period of at least 5 days (including the 72 hours period, when samples are obtained), patients will receive the alternative treatment at the same dose. The main objective is to stablish the bioequivalence of two formulations of recombinant factor IX, r-FIX and r-FIX-R, in patients with hemophilia B moderate-severe previously treated. Secondary objectives: To assess the efficacy and safety of r-FIX-R in the treatment of acute haemorrhagic episodes during an on-demand treatment (second part), during a routine prophylaxis (second part), during the control of haemorrhages intra and postoperative, if it is appropriate (second part) and to compare the pharmacokinetics of baseline rFIX and 6 months later,