HomeTrialNCT05121376
🔬NCT05121376Phase 1Phase 2ACTIVE NOT RECRUITING

A Gene Therapy Study of BMN 331 in Subjects With Hereditary Angioedema

Study Summary

This is a Phase 1/2, single-arm, open-label, dose-escalation and dose-expansion study of BMN 331 for the treatment of hereditary angioedema (HAE) due to C1 Esterase Inhibitor (C1-INH) protein deficiency. The study drug BMN 331is identified as AAV5 hSERPING1, an adeno-associated virus (AAV5)-based gene therapy vector that expresses wild-type human C1 Esterase Inhibitor (hC1-INH), under the control of a liver-selective promoter, and is being developed for the treatment of HAE with C1-INH deficiency. The pharmaceutical form of BMN 331 is a solution for intravenous infusion.

View on ClinicalTrials.gov ↗
Study Details
Enrollment44 participants
Primary Completion
Lead SponsorBioMarin Pharmaceutical
Data Retrieved2026-04-11 02:55:23.531406+00:00
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