Dettagli per singolo studio

DEC-NET Serial number ES472

Published online	15/09/2005 13.47.00
Last updated	02/05/2006 11.49.02

Other protocol ID number	3090A1-301-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 Treatment regimen (dosage and duration) N/A
Gender	Both
Age (range)	0-4 years old
Eligibility criteria
Inclusion criteria
Patients younger than 4 (with the aim of completing the treatment before they are 6 years old), with severe hemophilia B (level of activity of FIX in plasma [FIX:C] < or = 1%); able to follow-up the proceedings of the study (in the investigator's opinion, and also patient's and/or legal tutor's opinion); the parents or a legal tutor have to sign the informed consent before the screening procedures.
Exclusion criteria
Some level of FIX inhibitor (or history of), defined as > 0,6 UB; abnormal liver function (defined as levels > 2,5 the normal upper range of SGPT, SGOT and/or total bilirubin, excepting a documented hyperbilirubinemia of the newborn), abnormal renal function, low level of albumin, platelet <100.000 mm3, abnormal prothrombin time (>1,25 the upper normal range), known hypersensitivity to drugs composed mainly by proteins or to agents related with the sudied agent, i.e. proteins of hamster; HIV infection; active hepatitis; other genetic blood disorders; treatment with any investigational drug during the previous 30 days; and other.
Trial design/methodology
Phase	3
Kind of study	Efficacy Prophylaxis Safety Pharmacokinetics
Design
Purpose of study
The main objective is to assess the safety and efficacy of rFIX in children younger than 6 with severe hemophilia B: acute haemorrhages, prophilaxis and surgery. Secondary objectives: 1) To assess the incremental recovery of rFIX after an infusion bolus of 75 IU/kg; 2) To describe the immunogenicity/neoantigenicity of rFIX, independently of the previous treatment, by means of a the assessment of the possible development of inhibitor (Bethesda Inhibitor Assay [BIA]) during the trial; 3) To follow-up other interesting events: thrombogenicity, haemorrhage or no effect, allergic symptoms and agglutiation of red blood cells.
Primary outcomes
Efficacy will be evaluated by the dose of rFIX (IU/kg) used in total and in on-demand regime, profilactic or for surgery. Security will be evaluated by the presentation of adverse reactions in relation to the number of infusions given.
Secondary outcomes
Security: development of inhibitor anti-FIX (BIA, development of anti-FIX antibodies (ELISA) trombogenicity (TAT, D-dimer and protrombine fragments 1+2) allergic reactions hematies aglutination; seroconvertion to hepatitis A, B, C and HIV 1 and 2. Efficacy: Use of rFIX (dose in IU/kg and number of infusions per hemorrhagic episode). On the on-demand treatment: evaluation of the response to every infusion en a scale of 4 values(by the patient and/or the responsible of its care or the investigator). In profilaxis: dose, number of infusions, number of episodes of intercurrent hemorrhage (spontaneous or traumatic). In surgery: dose and response in the pre, intra and post surgical period (hemostasia, hemorrhagia, transfusions). Global evaluation by the researcher of the efficacy (5 categories).
Summary of study design, objectives, and ongoing research findings
This open multicenter study will include at least 20 patient younger than 6, independently of previous treatments with other FIX. They will receive FIX on-demand during acute haemorrhages and/or as intermittent prophylaxis or during surgical procedures. The treatment can be given at home or at the hospital, depending on the circumstance of each patient. The patient will receive the first dose of rFIX after a wash-out period of 4 days. They will receive the treatment on-deman or as prophylaxis depending on the investigator's criterion. Patients will be on study for 6 months; if they have been exposed for less than 30 days during this period, they will be on study for 6 additional months.