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

Study acronym: HAErmony-1
StatusActive, Not Recruiting
PhasePhase 1/2
Started
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Record History

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  1. Trial status: Recruiting to Active, Not Recruiting
    • Site details revised at 16 of 16 sites
    v17
  2. Enrollment target: 34 to 44v16 Show 4 more changesShow less
    • Study sites: 15 to 16
    • Interventions: Dose 1 of BMN 331 (Genetic), Dose 2 of BMN 331 (Genetic), Dose 3 of BMN 331 (Genetic), Dose 4 of BMN 331 (Genetic), Dose 5 of BMN 331… to Dose 1 of BMN 331 (Genetic), Dose 2 of BMN 331 (Genetic), Dose 3 of BMN 331 (Genetic), Dose 4 of BMN 331 (Genetic), Dose 5 of BMN 331…
    • Eligibility criteria expanded, about 24 words
    • Secondary endpoints: 7 to 8
  3. Study sites: 13 to 15v15
  4. Eligibility criteria expanded, about 20 wordsv14
  5. Study sites: 12 to 13v13
Show 11 earlier versionsHide 11 earlier versions
  1. Study sites: 11 to 12v12
  2. Site contacts updated at 1 of 11 sitesv11
  3. Study sites: 10 to 11v10
  4. Site contacts updated at 1 of 10 sitesv9
  5. Study sites: 9 to 10v8
  6. Study sites: 6 to 9v7
  7. Study sites: 5 to 6v6
  8. Study sites: 4 to 5v5
  9. Study sites: 2 to 4v4
  10. Study sites: 1 to 2
    • Start date changed from estimate of Nov 2021 to Feb 15, 2022
    v3
  11. Trial status: Not Yet Recruiting to Recruiting
    • Study sites: 0 to 1
    • Study title shortened, about 6 words
    v2
  1. Study First Submitted
  • Stopped: an ongoing trial was terminated, suspended or withdrawn.
  • Notable: the trial’s plan changed such as a big move in its timeline or size, a new primary endpoint, sponsor or phase, or recruiting opened or resumed.
  • Minor: routine progress and housekeeping, such as sites added or enrollment closed.
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.
Trial Details
NCT Number NCT05121376
Lead Sponsor BioMarin Pharmaceutical
Conditions
(from sponsor)		 Hereditary Angioedema, HAE
Related terms Angioedemas, Hereditary
Enrollment 44 participants
Start Date
Primary Completion (estimated)
Study Completion (estimated)
Updated on ClinicalTrials.gov