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

NCT04108260 | Unknown Phase 4

• 1 other identifier: 201907004MIPB
• Study Type: interventional
• Target: 5
• Locations: 1 country
• Sites: 1

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.

Conditions

Hemophilia

Outcome Measures

Primary Outcomes (1)

• AsBR

  To evaluate AsBR of Idelvion in routine patient care

  12 months

Study Arms (1)

Single arm_Idelvion treated

EXPERIMENTAL

Drug: Albutrepenonacog Alfa 1 UNT [IDELVION]

Interventions

Albutrepenonacog Alfa 1 UNT [IDELVION] DRUG

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.

Single arm_Idelvion treated

Eligibility Criteria

• Age: 20 Years+
• Sex: male
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• National Taiwan University Hospital: lead

Study Sites (1)

National Taiwan University Hospital

Taipei, Taiwan

RECRUITING

MeSH Terms

Conditions

Hemophilia A

Interventions

albutrepenonacog alfa

Condition Hierarchy (Ancestors)

Blood Coagulation Disorders, Inherited; Blood Coagulation Disorders; Hematologic Diseases; Hemic and Lymphatic Diseases; Coagulation Protein Disorders; Hemorrhagic Disorders; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Central Study Contacts

SHENG-CHIEH DR. CHOU, MD/PHD

CONTACT

886223123456; potatoaureus@gmail.com

YI-LIN YEH, Master

CONTACT

886227576970; yeh.yi-lin@cslbehring.com

Study Design

• Study Type: interventional
• Phase: phase 4
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: September 26, 2019
• First Posted: September 30, 2019
• Study Start: April 21, 2020
• Primary Completion: November 1, 2021
• Study Completion: April 1, 2022
• Last Updated: October 30, 2020

Record last verified: 2020-10

Data Sharing

• IPD Sharing: Will not share

Locations

TW (1)