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Clinical trial

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

NCT05121376 is registered in ClinicalTrials.gov with BioMarin Pharmaceutical as lead sponsor.

NCT NCT05121376
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Registry record

As filed in ClinicalTrials.gov.

Title
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
Lead sponsor
BioMarin Pharmaceutical
Phase
Phase 1/2
Overall status
Active, not recruiting
Start date
February 15, 2022
Completion date
November 30, 2028
Enrollment
44 (estimated)
Sites
16

Conditions

As the registry lists them; a condition with its own page is linked.

hae, hereditary angioedema