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

Phase 4

• Conditions: Hemophilia
• Interventions: Drug: Albutrepenonacog Alfa 1 UNT [IDELVION]

• Registration Number: NCT04108260
• Lead Sponsor: National Taiwan University Hospital

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.

Detailed Description

Not available

Study Timeline

• Study First Submitted: 2019-09-26
• Study First Submitted QC: 2019-09-26
• Study First Posted: 2019-09-30
• Study Start: 2020-04-21
• Status Verified: 2020-10
• Last Update Submitted: 2020-10-28
• Last Update Posted: 2020-10-30
• Primary Completion: 2021-11
• Study Completion: 2022-04

Recruitment & Eligibility

• Status: UNKNOWN
• Sex: Male
• Target Recruitment: 5

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.

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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.

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Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
Single arm_Idelvion treated	Albutrepenonacog Alfa 1 UNT [IDELVION]	-

Primary Outcome Measures

Name	Time	Method
AsBR	12 months	To evaluate AsBR of Idelvion in routine patient care

Trial Locations

Locations (1)

National Taiwan University Hospital; 🇨🇳 Taipei, Taiwan