Clinical Trial

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

Study acronym: HAErmony-1
Active, Not Recruiting Phase 1/2
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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.
Protocol Amendment History 16 amendments
This ClinicalTrials.gov record has been amended 16 times since 2021-11-04; most recent amendment 2024-05-15.
Status change: Recruiting → Active, Not Recruiting 2024-05-15
Status change: Not Yet Recruiting → Recruiting 2021-11-18
Trial Details
NCT Number NCT05121376
Lead Sponsor BioMarin Pharmaceutical
Conditions Hereditary Angioedema, HAE
Enrollment 44 participants
Start Date 2022-02-15
Primary Completion 2028-11 (estimated)
Study Completion 2028-11 (estimated)
Updated on ClinicalTrials.gov 2024-05-16