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.

Haemophilia B

The primary endpoint is to demonstrate the long-term safety (6-10 years) after dosing of CSL220., Primary safety endpoints include the following: - AEs possibly or probably related to previous CSL220 administration, Neutralising FIX antibodies (FIX inhibitors), ALT/aspartate aminotransferase (AST) levels, Liver pathology (assessed by ultrasound), Alpha-fetoprotein (AFP)

The secondary endpoints will focus on the long-term efficacy (6-10 years) after dosing of CSL220 on FIX activity, overall FIX utilisation, bleeding events, any procedures, and QoL., Secondary efficacy endpoints include the following: - Endogenous FIX activity, Utilisation of FIX-replacement therapy, Annualized bleeding rate; including the following: o All bleeds (treated and untreated) o Spontaneous bleeds o Traumatic bleeds o Joint bleeds, Procedures (including major and minor surgeries), 36-Item Short Form Survey (SF-36) and EQ-5D-5L Quality of Life (QoL) scores, Hemophilia Joint Health Score (HJHS)

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Germany, Netherlands