A Gene Therapy Study of BMN 331 in Subjects With Hereditary Angioedema
Public ClinicalTrials.gov record NCT05121376. Field values are reproduced from the official study page; the official ClinicalTrials.gov record remains the source of truth for eligibility, enrollment, and contact information.
Data is sourced from official ClinicalTrials.gov public API records. Always review the official ClinicalTrials.gov record for the latest information.
Official 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
Brief summary
Reproduced verbatim from the official ClinicalTrials.gov record. Not medical advice.
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.
Study identification
- NCT ID
- NCT05121376
- Recruitment status
- Active, not recruiting
- Study type
- Interventional
- Phase
- Phase 1, Phase 2
- Enrollment
- 44 participants
Conditions and interventions
Conditions
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 Genetic
- Dose 6 of BMN 331 Genetic
- Dose 7 of BMN 331 Genetic
Genetic
Eligibility (public fields only)
- Age range
- 18 Years and older
- Sex
- All
- Healthy volunteers
- Healthy volunteers not accepted
This page does not interpret eligibility. Detailed inclusion and exclusion criteria are on the official ClinicalTrials.gov record.
Study timeline
- Start date
- Feb 15, 2022 (Actual)
- Primary completion
- Nov 2028 (Estimated)
- Completion
- Nov 2028 (Estimated)
- Last update posted
- May 16, 2024
2022 – 2028
United States locations
- U.S. sites
- 13
- U.S. states
- 12
- U.S. cities
- 13
| Facility | City | State | ZIP | Site status |
|---|---|---|---|---|
| AllerVie Clinical Research | Birmingham | Alabama | 35209 | — |
| Medical Research of Arizona | Scottsdale | Arizona | 85251 | — |
| University of California San Diego | San Diego | California | 92122 | — |
| Asthma & Allergy Associates P.C. | Colorado Springs | Colorado | 80907 | — |
| Dr. Henry J. Kanarek Allergy, Asthma & Immunology | Overland Park | Kansas | 66211 | — |
| Institute For Asthma & Allergy | Chevy Chase | Maryland | 20815 | — |
| Mississippi Center for Advanced Medicine | Madison | Mississippi | 39110 | — |
| Washington University School of Medicine | St Louis | Missouri | 63141 | — |
| Duke Health | Durham | North Carolina | 27705 | — |
| University of Cincinnati (UC) Physicians Company, LLC | Cincinnati | Ohio | 45267 | — |
| Optimed Research, LTD | Columbus | Ohio | 43235 | — |
| The Pennsylvania State University (Penn State) Milton S. Hershey Medical Center | Hershey | Pennsylvania | 16802 | — |
| AARA Research Center | Dallas | Texas | 75231 | — |
Site contact phone numbers, emails, and investigator names are intentionally not displayed here. Open the official ClinicalTrials.gov record for site contact information.
Non-U.S. locations
This page focuses on the U.S. directory. The official record also lists 3 non-U.S. sites.
About this trial record page
- What this page shows
- Public field values for ClinicalTrials.gov record NCT05121376, including study identification, conditions, interventions, eligibility (age, sex, healthy volunteer), timeline, and U.S. site list.
- What this page does not do
- No medical advice, eligibility judgments, treatment recommendations, study quality scoring, or AI-generated medical summaries. No site contact phone numbers, emails, or investigator names.
- Where the data comes from
- Sourced from the official ClinicalTrials.gov public API. The official record is the source of truth.
- Last refresh
- Last update posted May 16, 2024 · Synced Oct 24, 2026
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Open the official record
The complete public registry record, listed eligibility criteria, and available contact information for NCT05121376 live on ClinicalTrials.gov.