A Phase I/IIb extension study assessing the long-term safety and efficacy of a gene therapy (AAV5-hFIX) previously administered to adult patients with severe or moderately severe haemophilia B during the CT-AMT-060-01 Phase I/II study.

Brief summary (registry, in English)

To assess the long-term safety (6-10 years after dosing, inclusive) of a systemic administration of CSL220, an AAV vector containing a codon-optimized human coagulation hFIX gene, to adult subjects with severe or moderately severe haemophilia B.

What you commit to

• Study type: Interventional · Phase 1/2
• Participants enrolled: 9
• 6 sites in 2 countries
• Visits and tests: ask the trial team, the registry does not list them

Built from the registry fields of EU CTIS (euclinicaltrials.eu). It describes the study, not your situation.

Who cannot take part

Copied verbatim from EU CTIS (euclinicaltrials.eu) and grouped by topic. Only the trial team decides who can take part.

Who the study is looking for

Age in the registry: 18 years to - · Sex: Male · Does not accept healthy volunteers

Full criteria on EU CTIS (euclinicaltrials.eu)

Sites (6)

Sites in registry order. Pick your city on the disease page to sort them by distance.