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

Hemophilia Clinical Trial

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

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT04108260
• Other study ID #: 201907004MIPB
• Status: Recruiting
• Phase: Phase 4
• Start date: April 21, 2020
• Est. completion date: April 2022

Study information

• Verified date: October 2020
• Source: National Taiwan University Hospital
• Contact: SHENG-CHIEH DR. CHOU, MD/PHD
• Phone: 886223123456
• Email: potatoaureus[@]gmail.com
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 5
• Est. completion date: April 2022
• Est. primary completion date: November 2021
• Accepts healthy volunteers: No
• Gender: Male
• Age group: 20 Years and older

Eligibility

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.

Related Conditions & MeSH terms

• Hemophilia
• Hemophilia A
• Hemophilia B

Intervention

Drug:
• Albutrepenonacog Alfa 1 UNT [IDELVION]
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.

Locations

Country	Name	City	State
Taiwan	National Taiwan University Hospital	Taipei

Sponsors (1)

Lead Sponsor	Collaborator
National Taiwan University Hospital

Country where clinical trial is conducted

Taiwan, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	AsBR	To evaluate AsBR of Idelvion in routine patient care	12 months