Study of AAV5-hFIX in Severe or Moderately Severe Haemophilia B
Active, not recruiting
- Conditions
- Hemophilia B
- Registration Number
- NCT05360706
- Lead Sponsor
- CSL Behring
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- Active, not recruiting
- Sex
- Male
- Target Recruitment
- 9
Inclusion Criteria
Inclusion Criteria:<br><br> - Subjects with congenital hemophilia B who completed Study CTAMT-060-01<br><br> - Able to provide informed consent following receipt of verbal and written information<br> about the trial.<br><br>Exclusion Criteria:<br><br> - Enrolled subjects will have already been assessed based on the exclusion criteria<br> for Study CT-AMT-060-01.
Exclusion Criteria
Not provided
Study & Design
- Study Type
- Observational
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method Number of Adverse events Possibly or probably related to previous AAV5-hFIX administration;Neutralizing FIX antibodies (FIX inhibitors);ALT/AST levels;Liver pathology score Assessed by ultrasound every 6 months;Alfa fetoprotein levels
- Secondary Outcome Measures
Name Time Method Endogenous Percent FIX activity;Total Consumption of FIX Replacement Therapy on-demand and prophylactic;Annualized bleeding rate Including all bleeds (treated and untreated), spontaneous bleeds, traumatic bleeds and joint bleeds;Number of Procedures (including major and minor surgeries);Quality of Life questionnaire SF-36 score;Quality of Life questionnaire EQ-5D-5L score;Hemophilia Joint Health Score