Study of AAV5-hFIX in Severe or Moderately Severe Haemophilia B

Study Overview
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Brief Summary
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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).

Official Title
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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

Conditions
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Hemophilia B

Intervention / Treatment
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* Genetic: AAV5-hFIX


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Subjects with congenital hemophilia B who completed Study CTAMT-060-01 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.

Ages Eligible for Study
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Minimum Age: 18 Years

Sexes Eligible for Study
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Male

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| CSL220<br>AAV5 containing a codon-optimized human factor IX gene | Genetic: AAV5-hFIX<br>* AAV5 containing a codon-optimized human factor IX gene<br>* Other names: CSL220;|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Number of Adverse events Possibly or probably related to previous AAV5-hFIX administration |  | 6-10 years after dosing in CT-AMT-060-01 study | 
| Neutralizing FIX antibodies (FIX inhibitors) |  | 6-10 years after dosing in CT-AMT-060-01 study | 
| ALT/AST levels |  | 6-10 years after dosing in CT-AMT-060-01 study | 
| Liver pathology score Assessed by ultrasound every 6 months |  | 6-10 years after dosing in CT-AMT-060-01 study | 
| Alfa fetoprotein levels |  | 6-10 years after dosing in CT-AMT-060-01 study | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Endogenous Percent FIX activity |  | 6-10 years after dosing in CT-AMT-060-01 study | 
| Total Consumption of FIX Replacement Therapy on-demand and prophylactic |  | 6-10 years after dosing in CT-AMT-060-01 study | 
| Annualized bleeding rate Including all bleeds (treated and untreated), spontaneous bleeds, traumatic bleeds and joint bleeds |  | 6-10 years after dosing in CT-AMT-060-01 study | 
| Number of Procedures (including major and minor surgeries) |  | 6-10 years after dosing in CT-AMT-060-01 study | 
| Quality of Life questionnaire SF-36 score |  | 6-10 years after dosing in CT-AMT-060-01 study | 
| Quality of Life questionnaire EQ-5D-5L score |  | 6-10 years after dosing in CT-AMT-060-01 study | 
| Hemophilia Joint Health Score |  | 6-10 years after dosing in CT-AMT-060-01 study |