Hemophilia
Conditions
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.
Interventions
DRUGAlbutrepenonacog 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.
Sponsors
National Taiwan University Hospital
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
MALE
Age
20 Years to No maximum
Healthy volunteers
No
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.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| AsBR | 12 months | To evaluate AsBR of Idelvion in routine patient care |
Countries
Taiwan
Contacts
Primary ContactSHENG-CHIEH DR. CHOU, MD/PHD
Backup ContactYI-LIN YEH, Master
Outcome results
None listed