Factor IX Padua AAV gene therapy in adolescents with hemophilia B: a phase 1 trial.

What was studied

This early trial tested gene therapy for hemophilia B in adolescents. The therapy is BBM-H901, which carries the FIX Padua gene. This type had been shown to work and be safe in adults. The trial was multicenter and single-arm, in China. It enrolled 11 adolescents aged 12 to 18 years. They had severe or moderately severe disease. All got the gene therapy by infusion. They were followed for 52 weeks. The main aim was safety.

What they found

No dose-limiting toxicity was seen. The most common adverse events were higher white blood cell and neutrophil counts. Rash linked to corticosteroid use was also common. One serious adverse event occurred. One person had abnormal liver tests. These returned to normal 4 weeks after immunosuppression. At week 52, mean FIX activity was 41.8 IU per dl. The mean annualized bleeding rate fell from 13.9 to 0.5.

What it means for patients

The authors say the gene therapy was safe in adolescents. They say it gives first signs of efficacy.

Why it is not certain yet

Only 11 adolescents took part. There was no comparison group. Follow-up was 52 weeks. The abstract calls the efficacy insights initial. The spread in FIX activity was wide.

Source

Factor IX Padua AAV gene therapy in adolescents with hemophilia B: a phase 1 trial.

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