Trials / Active Not Recruiting
Active Not RecruitingNCT05360706
Study of AAV5-hFIX in Severe or Moderately Severe Haemophilia B
A Phase I/IIb Extension Study Assessing the Long-term Safety and Efficacy of an Adeno-associated Viral Vector Containing a Codon-optimized Human Factor IX Gene (AAV5-hFIX) Previously Administered to Adult Patients With Severe or Moderately Severe Haemophilia B During the CT-AMT-060-01 Phase I/II Study
- Status
- Active Not Recruiting
- Phase
- —
- Study type
- Observational
- Enrollment
- 9 (actual)
- Sponsor
- CSL Behring · Industry
- Sex
- Male
- Age
- 18 Years
- Healthy volunteers
- Not accepted
Summary
This is an open-label, extension study enrolling patients who have successfully completed all assessments in Study CT-AMT-060-01 (Years 1-5). Assessment phase will begin at Visit 36 (the first clinical visit in this extension study, approximately 5.5 years after the initial dosing visit Study CT-AMT-060-01) and go to Visit 45 (10-years post-dosing in Study CT-AMT-060-01).
Conditions
Interventions
| Type | Name | Description |
|---|---|---|
| GENETIC | AAV5-hFIX | AAV5 containing a codon-optimized human factor IX gene |
Timeline
- Start date
- 2021-03-18
- Primary completion
- 2026-05-01
- Completion
- 2026-05-01
- First posted
- 2022-05-04
- Last updated
- 2025-08-26
Locations
6 sites across 2 countries: Germany, Netherlands
Regulatory
- FDA-regulated drug study
Source: ClinicalTrials.gov record NCT05360706. Inclusion in this directory is not an endorsement.