What type of study is this?

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

What question did this study set out to answer?

This research assesses the safety and preliminary efficacy of VSA012, a CFB-targeted siRNA, in PNH patients.

December 8, 2025BloodOpen Access

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Interim analysis shows VSA012 improves hemoglobin and reduces complement factor B in PNH patients, indicating favorable safety profile.

This research assesses the safety and preliminary efficacy of VSA012, a CFB-targeted siRNA, in PNH patients.
Ongoing multicenter, non-randomized, multiple-ascending-dose, open-label phase Ib clinical trial.
Enrolled Chinese PNH patients receiving 60 mg or 120 mg VSA012 via subcutaneous injection.
Phase included screening, treatment, and follow-up with safety, efficacy, pharmacokinetics, and pharmacodynamics assessments.
No serious adverse events were observed in participants.
In the 60 mg group, hemoglobin increased by 43.5% within 56 days; 75% of patients achieved an increase of 2 g/dL or more.
Complement factor B and other health markers showed significant reductions in the study.

Wang et al. (2025) studied this question.

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