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.
Phase 2
Completed
- Conditions
- Christmas disease1006447710005330
- Registration Number
- NL-OMON54158
- Lead Sponsor
- CSL Behring LLC
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- Completed
- Sex
- Not specified
- Target Recruitment
- 6
Inclusion Criteria
1. Subjects with congenital hemophilia B who completed Study CT-AMT-060-01
2. Able to provide informed consent following receipt of verbal and written
information about the trial.
Exclusion Criteria
Enrolled subjects will have already been assessed based on the exclusion
criteria for Study CT-AMT-060-01.
Study & Design
- Study Type
- Observational invasive
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method <p>The primary endpoint is to demonstrate the long-term safety (6-10 years) after<br /><br>dosing of AAV5-hFIX.<br /><br>Primary safety endpoints include the following:<br /><br>- AEs possibly or probably related to previous AAV5-hFIX administration<br /><br>- ALT/aspartate aminotransferase (AST) levels<br /><br>- Liver pathology (assessed by ultrasound)<br /><br>- Alpha-fetoprotein (AFP)</p><br>
- Secondary Outcome Measures
Name Time Method <p>The secondary endpoints will focus on the long-term efficacy (6-10 years) after<br /><br>dosing of AAV5-hFIX on FIX activity, overall FIX utilization, bleeding events,<br /><br>any procedures and Quality of Life.<br /><br>Secondary efficacy endpoints include the following:<br /><br>- Endogenous FIX activity<br /><br>- Utilization of FIX-replacement therapy<br /><br>- Annualized bleeding rate; including the following:<br /><br>o All bleeds (treated and untreated)<br /><br>o Spontaneous bleeds<br /><br>o Traumatic bleeds<br /><br>o Joint bleeds<br /><br>- Procedures (including major and minor surgeries)<br /><br>- Short form (SF-36) and EuroQol-5 Dimensions-5 Levels (EQ-5D-5L) QoL scores<br /><br>- Hemophilia Joint Health Score (HJHS)</p><br>