Relationship between coagulation factor IX (FIX) genotype and plasma persistance (pharmacokinetics) of recombaint FIX (Nonacog alfa), administered for replacement therapy, in hemophilia B patients

F9 Genotype and PK Hemophilia B International Study - GePKHIS

Status

Active, not recruiting

Phases

Phase 4

Study type

Interventional

Source

EU CTR

Registry ID

EUCTR2017-003902-42-IT

Enrollment

Registered

2017-12-01

Start date

2018-03-08

Completion date

Unknown

Last updated

2022-03-14

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Deficiency of coagulation factor IX (FIX

Interventions

Trade Name: BeneFIX 250 UI powder and solvent for solution for injection Product Name: Nonacog alfa Pharmaceutical Form: Powder and solvent for solution for injection Trade Name: BeneFIX 500 UI powd

Eligibility

Sex/Gender

Male

Inclusion criteria

Inclusion criteria: - Severe or moderately severe haemophilia B patients (FIX 150 exposure days (1 exposure day = 1 day of treatment with one or more infusions of Nonacog alfa) following a once-a-week infusion schedule (usually on Monday); - 5-7 days wash out from the previous Nonacog alfa administration; - Signed Informed consent. Are the trial subjects under 18? no Number of subjects for this age range: F.1.2 Adults (18-64 years) yes F.1.2.1 Number of subjects for this age range 50 F.1.3 Elderly (>=65 years) yes F.1.3.1 Number of subjects for this age range 10

Exclusion criteria

Exclusion criteria: - Any kind of bleeding, in the last week before the PK; - Sever hepatic disease, PT<70%; - Ongoing HIV treatment (HAART); - Previous or current FIX inhibitor; - Previous treatment with rFIX EHL concentrates.

Design outcomes

Primary

Measure	Time frame
Main Objective: 1. Correlation between FIX (F9) genotype in a large cohort of hemophilia B (HB) patients and outcomes of a single dose PK of Nanocog alfa. 2. Recombinant expression of F9 mutation of each patient to evaluate the residual FIX activity and protein (FIX:C, FIX:Ag and FIX:Ag truncated molecules), to contribute elements to interpret the outcomes of PK. ;Secondary Objective: 1. Comprehensive and accurate analysis of Nonacog alfa pharmacokinetics (PK) parameters, according to a blood sampling design up to 96 hrs; 2. Definition of the best model fitting the data of PK; 3. Impact of co-variateson the population PK by means of Non Linear Mixwd Effects (Pharsight); 4. Better definition of the genotype-phenotype relation in Hemophilia B. ;Primary end point(s): 1. Expression levels of recombinant proteins bearing patients' mutations. 2. Dose/kg, Cmax, In Vivo Recovery, Area under the curve, Terminal Half life (non compartment analysis), Alfa Half life, Beta half life, Clearance, Distribution volume, Mean Residence Time, K 1-0, K1-2, K2-1.;Timepoint(s) of evaluation of this end point: 1. six months. 2. nine months.	—

Measure

Time frame

Main Objective: 1. Correlation between FIX (F9) genotype in a large cohort of hemophilia B (HB) patients and outcomes of a single dose PK of Nanocog alfa. 2. Recombinant expression of F9 mutation of each patient to evaluate the residual FIX activity and protein (FIX:C, FIX:Ag and FIX:Ag truncated molecules), to contribute elements to interpret the outcomes of PK. ;Secondary Objective: 1. Comprehensive and accurate analysis of Nonacog alfa pharmacokinetics (PK) parameters, according to a blood sampling design up to 96 hrs; 2. Definition of the best model fitting the data of PK; 3. Impact of co-variateson the population PK by means of Non Linear Mixwd Effects (Pharsight); 4. Better definition of the genotype-phenotype relation in Hemophilia B. ;Primary end point(s): 1. Expression levels of recombinant proteins bearing patients' mutations. 2. Dose/kg, Cmax, In Vivo Recovery, Area under the curve, Terminal Half life (non compartment analysis), Alfa Half life, Beta half life, Clearance, Distribution volume, Mean Residence Time, K 1-0, K1-2, K2-1.;Timepoint(s) of evaluation of this end point: 1. six months. 2. nine months.

—

Secondary

Measure	Time frame
Secondary end point(s): 1. Expression vectors bearing the specific mutation of each patient. 2. Cell cultures with patient-specific vectors.;Timepoint(s) of evaluation of this end point: 1. Four months. 2. Six months.	—

Countries

Italy

Contacts

Public ContactBlocco B - 1° Piano

Consorzio Futuro in Ricerca (CFR)

cfr@unife.it00390532762404

Outcome results

None listed

Source: EU CTR (via WHO ICTRP) · Data processed: Feb 4, 2026