What type of study is this?

This is a Human Clinical Trial study (also classified as: Phase 3, Pre-Registered Trial).

What question did this study set out to answer?

To evaluate the safety and efficacy of AAV-FIX Padua gene therapy in patients with hemophilia B.

November 20, 2025Nature MedicineOpen Access

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Single-arm trials show improved efficacy and safety of AAV gene therapy in hemophilia B, suggesting significant benefits for treatment.

To evaluate the safety and efficacy of AAV-FIX Padua gene therapy in patients with hemophilia B.
Conducted a single-arm dose-escalation phase 1/2 trial with 6 male participants.
Conducted a multicentre phase 3 trial with 26 male participants.
Assessed primary endpoint of adverse events and annualized bleeding rate.
No dose-limiting toxicities observed in phase 1/2 trial.
Annualized bleeding rate decreased to 0.60 within 52 weeks post-infusion in phase 3 trial.
80.8% of participants experienced zero bleeds during the 52-week follow-up.

Xue et al. (2025) studied this question.

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