Trials / Recruiting
RecruitingNCT06008938
An Observational Cohort Study to Characterize the Effectiveness and Safety of HEMGENIX® in Patients With Hemophilia B
An Observational Post-authorization Long-term Follow-up Study to Characterize the Effectiveness and Safety of HEMGENIX® (Etranacogene Dezaparvovec) in Patients With Hemophilia B
- Status
- Recruiting
- Phase
- —
- Study type
- Observational
- Enrollment
- 500 (estimated)
- Sponsor
- CSL Behring · Industry
- Sex
- All
- Age
- 18 Years
- Healthy volunteers
- Not accepted
Summary
This observational, post-authorization, long-term follow-up study aims to investigate the short and long-term effectiveness and safety of HEMGENIX in patients with hemophilia B. The study will also include a cohort of patients with hemophilia B treated with FIX prophylaxis to enable interpretation of relevant efficacy and safety findings of HEMGENIX.
Conditions
Interventions
| Type | Name | Description |
|---|---|---|
| GENETIC | HEMGENIX | HEMGENIX is a gene therapy medicinal product that aims to deliver a factor IX (FIX) gene expression cassette to the liver of patients with hemophilia B. |
| BIOLOGICAL | Factor IX (FIX) | FIX prophylaxis therapy |
Timeline
- Start date
- 2023-06-15
- Primary completion
- 2043-08-01
- Completion
- 2043-08-01
- First posted
- 2023-08-24
- Last updated
- 2026-02-19
Locations
12 sites across 7 countries: United States, Austria, Denmark, France, Germany, Spain, Switzerland
Source: ClinicalTrials.gov record NCT06008938. Inclusion in this directory is not an endorsement.