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A Phase 1/2 Open-Label, Dose-Escalation Study to Determine the Safety, Tolerability & Efficacy of BMN 331 an AAV Vector-Mediated Gene Transfer of Human SERPING1 Gene in Subjects With HAE Due to Human C1-INH Deficiency

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Trial Profile

A Phase 1/2 Open-Label, Dose-Escalation Study to Determine the Safety, Tolerability & Efficacy of BMN 331 an AAV Vector-Mediated Gene Transfer of Human SERPING1 Gene in Subjects With HAE Due to Human C1-INH Deficiency

Status: Active, no longer recruiting
Phase of Trial: Phase I/II

Latest Information Update: 21 May 2024

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At a glance

  • Drugs BMN 331 (Primary)
  • Indications Hereditary angioedema
  • Focus Adverse reactions; First in man
  • Acronyms 331-201; HAErmony-1
  • Sponsors BioMarin Pharmaceutical
  • Most Recent Events

    • 15 May 2024 Status changed from recruiting to active, no longer recruiting.
    • 05 Dec 2023 Increase in patient no and no. of doses of drug in treatment regimen
    • 05 Dec 2023 Planned number of patients changed from 34 to 44.
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