Safety of 40K Pegylated Recombinant Factor IX in Non-Bleeding Patients With Haemophilia B

Study Overview
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Brief Summary
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This trial is conducted in Europe, Japan and the United States of America (USA). The aim of this clinical trial is to investigate the safety and pharmacokinetics (the determination of the concentration of the administered medication in blood over time) of Pegylated Recombinant Factor IX (nonacog beta pegol) in Non-Bleeding Patients with Haemophilia B.

Official Title
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A Multi-Centre, Multi-National, Open-Label, Dose Escalation Trial Evaluating Safety and Pharmacokinetics of Ascending Intravenous Doses of 40K Pegylated Recombinant FIX in Non-Bleeding Patients With Haemophilia B.

Conditions
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Congenital Bleeding Disorder, Haemophilia B

Intervention / Treatment
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* Drug: nonacog beta pegol
* Drug: nonacog beta pegol
* Drug: nonacog beta pegol


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Diagnosed with haemophilia B (baseline level of Factor IX less than or equal to 2%) History of at least 150 exposure days to any Factor IX products Body Mass Index (BMI) below 30.0 kg/m2 (inclusive) Exclusion Criteria: History of Factor IX inhibitors Platelet count less than 50,000 platelets/microlitre (assessed by laboratory) Kidney or liver dysfunction Scheduled surgery requiring Factor IX replacement therapy, during the trial period

Ages Eligible for Study
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Minimum Age: 18 Years
Maximum Age: 65 Years

Sexes Eligible for Study
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Male

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Allocation: Non-Randomized
Intervention Model: Single Group Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: 25U/kg<br> | Drug: nonacog beta pegol<br>* Cohort to receive a single dose of 25U/kg nonacog beta pegol administered intravenously (into the vein)<br>* Other names: 40K PEG-rFIX;|
| Experimental: 50U/kg<br> | Drug: nonacog beta pegol<br>* Cohort to receive a single dose of 50U/kg nonacog beta pegol administered intravenously (into the vein)<br>* Other names: 40K PEG-rFIX;|
| Experimental: 100U/kg<br> | Drug: nonacog beta pegol<br>* Cohort to receive a single dose of 100U/kg nonacog beta pegol administered intravenously (into the vein)<br>* Other names: 40K PEG-rFIX;|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Frequency of Adverse Events (AEs), Serious Adverse Events (SAEs) and Medical Events of Special Interests (MESIs) reported during the trial period |  | assessed up to five weeks after trial product administration | 
| Antibody formation against 40K PEG-rFIX and test for inhibitors (Bethesda) |  | assessed up to five weeks after trial product administration | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| AUC, CL, T½, Incremental recovery (first sample) from 0 to 48 hours after trial product administration |  | assessed up to five weeks after trial product administration | 
| AUC, CL, T½, Incremental recovery (first sample) from 0 to 168 hours after trial product administration |  | assessed up to five weeks after trial product administration |