Severe or Moderately Severe Hemophilia B Patients MedDRA version: 9.1 Level: LLT Classification code 10016077 Term: Factor IX deficiency
Conditions
Interventions
Product Name: Nanofiltered Factor IX
Pharmaceutical Form: Powder and solvent for solution for infusion
INN or Proposed INN: Coagulation factor IX
Concentration unit: IU international unit(s)
Concentra
Sponsors
KEDRION
Eligibility
Sex/Gender
Male
Inclusion criteria
Inclusion criteria: 1. Age ≥ 12 years. 2. Weight > 35 kg 3. Subjects should have severe or moderately severe haemophilia B as defined by a baseline factor IX level 2%, documented at pre-enrolment screening (see Section 7.1) on the basis of anamnesis data (i.e., at haemophilia diagnosis). 4. Prior to study entry, subjects should have a history of at least 150 exposure days for all types of factor IX containing products, including fresh frozen plasma (FFP). This minimum exposure rate shall be estimated by the haemophilia treating physician. 5. If subjects are HIV-1 seropositive they should have a CD4+ lymphocyte count ≥ 400/l documented on 2 consecutive occasions over a 12 months period prior to study entry. The latest CD4+ lymphocyte count qualifying the patient for enrolment (i.e. ≥ 400/l) should be within 6 months before enrolment in the study. 6. Subjects, or their legally authorised representative in the case of study participants ≥ 12 and =65 years) yes F.1.3.1 Number of subjects for this age range
Exclusion criteria
Exclusion criteria: The following subjects will be ineligible for enrolment: 1. Subjects with a detectable inhibitor to factor IX at the time of enrolment or a history of inhibitor to factor IX (> 0.6 BU). 2. Subjects with clinical or laboratory evidence of portal vein hypertension, such as an international normalized ratio (INR) > 1.4, the presence of splenomegaly and/or spider angiomata on physical examination and/or a history of oesophageal haemorrhage or documented oesophageal varices. 3. Subjects HIV positive who are treated with highly active anti-retroviral therapy (HAART) regimen. 4. Subjects with any condition which, in the opinion of the Investigator, might interfere with the evaluation of the study objectives, or participation in this trial. 5. Subjects unwilling to give written informed consent to participation, or who have participated in another clinical trial within 1 month before study initiation, i.e. they have received any test drug within 30 days prior the study. The Investigator will make sure that the patients are not planning to leave the area of the study site before the end of the study period.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: Pharmacokinetics (PK) evaluation in vivo incremental recovery; area under the curve (AUC);area under the moment curve (AUMC); plasma half-life (tœ),determination of the time necessary to reach the peak plasma concentration (Tmax),clearance (CL), mean permanence time (MRT),distribution volume at steady state (Vdss),peak plasma concentration (Cmax) Efficacy and Safety evaluation Efficacy: 1) Evaluation of the haemostatic efficacy of Kedrion human plasma-derived double virus inactivated and nanofiltered factor IX (henceforth: Investigational Medicinal Product- IMP) in the management of acute bleeding events. Safety: 1) Assessment of the risk of inhibitor (neutralizing anti factor IX antibodies) development in conjunction with infusion of the IMP throughout the study period 2) Assessment of thrombogenicity of the IMP 3) Assessment of the short and medium term safety of the IMP, when administered to severe/moderately severe Previously Treated Patients;Secondary Objective: see above;Primary end point(s): Assessment of pharmacokinetics, efficacy and safety of IMP | — |
Countries
Italy
Outcome results
None listed