Brief Title
The Effectiveness of Recombinant Coagulation Factor IX With Recombinant Albumin (rIX-FP) in Severe Hemophilia B Patients
Official Title
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
Brief Summary
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.
Study Phase
Phase 4
Study Type
Interventional
Primary Outcome
AsBR
Condition
Hemophilia
Intervention
Albutrepenonacog Alfa 1 UNT [IDELVION]
Study Arms / Comparison Groups
Single arm_Idelvion treated
Description:
Publications
* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.
Recruitment Information
Recruitment Status
Drug
Estimated Enrollment
5
Start Date
April 21, 2020
Completion Date
April 2022
Primary Completion Date
November 2021
Eligibility Criteria
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.
Gender
Male
Ages
20 Years - N/A
Accepts Healthy Volunteers
No
Contacts
, 886223123456, [email protected]
Location Countries
Taiwan
Location Countries
Taiwan
Administrative Informations
NCT ID
NCT04108260
Organization ID
201907004MIPB
Responsible Party
Sponsor
Study Sponsor
National Taiwan University Hospital
Study Sponsor
, ,
Verification Date
October 2020