Hemophilic bleeding Bleeder's disease
Conditions
Interventions
Etranacogene dezaparvovec, gene therapy treatment.
Sponsors
CSL Behring
Eligibility
Age
18 Years to 99 Years
Inclusion criteria
Inclusion criteria: A patient must meet all of the following criteria to be eligible for participation in the study: • Male, aged =18 years, with hemophilia B • Completed 60 months of post-treatment follow up in HOPE-B clinical trial • Resident of one of the countries in which the study is conducted. • Fluent in the language of the country where the interview will be conducted. • Is willing and able to participate in a one-time 60-minute one-on-one telephone or video-conference interview with ICON staff or ICON subcontractor • Is willing to share his contact details with ICON staff or ICON subcontractor
Exclusion criteria
Exclusion criteria: A patient who meets any of the following criteria is not eligible for participation in the study: • Has decreased mental function due to a psychiatric or medical (neurocognitive) disease rendering him unable to understand the nature and scope of the study. • Is unwilling or unable to comply with study procedures. • The patient is enrolled in a clinical trial, other than HOPE-B or CSL2223003, at the time of enrollment in the study, in which treatment for hemophilia B is managed through a protocol.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| The primary study objectives are: • To investigate changes in the health-related quality of life (HRQOL) of patients since receiving an infusion of a hemophilia gene therapy to treat their hemophilia B • To explore treatment satisfaction, hopes, and concerns related to receiving hemophilia gene therapy | — |
Secondary
| Measure | Time frame |
|---|---|
| The secondary study objectives are: • To understand the decision-making process for receiving hemophilia gene therapy • To understand, from the patient perspective, what are the most important aspects of hemophilia gene therapy that other patients and healthcare professionals (HCPs) should know | — |
Countries
Belgium, Netherlands, United States
Contacts
Public ContactB. Joseph
CSL Behring
Outcome results
None listed