The Effectiveness of Recombinant Coagulation Factor IX With Recombinant Albumin (rIX-FP) in Severe Hemophilia B Patients

Study Overview
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Brief Summary
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The pivotal clinical data support prolonged dosing intervals of up to 14 days for routine prophylaxis in hemophilia B patients. The majority of adult and pediatric patients using Idelvion for routine prophylaxis had a median annualized spontaneous bleeding rate (AsBR) of 0.00. The majority (99%) of bleeding episodes were managed successfully with one or two infusions, and 94% of bleeds were controlled with only one infusion regardless of the cause or location. The rationale for this study is to collect the effectiveness and safety data of severe hemophilia B patients treated with Idelvion following institutional standard of care. It is the aim of this study to extend the results of the clinical trial program to a broader hemophilia B population, and to compare with current alternative factor IX treatments in Taiwan.

Official Title
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The Effectiveness of Recombinant Fusion Protein Linking Coagulation Factor IX With Recombinant Albumin (rIX-FP) in Severe Hemophilia B Patients Switching From Previous Factor IX Treatment

Conditions
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Hemophilia

Intervention / Treatment
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* Drug: Albutrepenonacog Alfa 1 UNT [IDELVION]


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Male patients with age ≥ 20 years old Endogenous FIX activity ≤1 IU/dL, At least 50 exposure days (EDs) with FIX products, No detectable inhibitor to FIX or inhibitor history, Had a minimum of 2 nontrauma-induced bleeding episodes (any type or location) treated in the 6 months preceding study entry, The patient or patient's parents or legal authorized representative, as applicable, are capable to understand the study objectives and procedure, and sign the written consent, Accept that the supply of Idelvion might be stopped once the study is completed, Able to complete a diary during 12 months or 50 EDs, whichever comes first. Exclusion Criteria: Currently participating in an interventional clinical trial, Known hypersensitivity to any FIX product or hamster protein, Known inhibitor to FIX or inhibitor history, With other comorbidities which are not suitable for this study, at investigator's discretion, Not able to compliant with the prophylactic treatment, Lacking previous treatment and bleeding records.

Ages Eligible for Study
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Minimum Age: 20 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
Intervention Model: Single Group Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: Single arm_Idelvion treated<br> | Drug: Albutrepenonacog Alfa 1 UNT [IDELVION]<br>* Clinical data will be collected from patients who suffered from hemophilia B, and switch from other FIX products to Idelvion as prophylaxis, prevention of bleeding (e.g. surgery) or treatment of bleeding.<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| AsBR | To evaluate AsBR of Idelvion in routine patient care | 12 months |