A Gene Therapy Study of BMN 331 in Subjects With Hereditary Angioedema (NCT05121376)
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
🩺 Plain-English Summary (8th-Grade Level)
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.
Who can join: Review the eligibility criteria below.
Location: AllerVie Clinical Research — Birmingham, Alabama
Age range: 18 Years
🧪 Interventions in This Study
- 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)
📋 "Do I Qualify?" — 1-Minute Self Screener
Instant ChecklistFull eligibility criteria are available on the official NIH protocol. View the criteria ↗
📍 All Participating Trial Locations (16 Sites)
- AllerVie Clinical Research — Birmingham, Alabama
- Medical Research of Arizona — Scottsdale, Arizona
- University of California San Diego — San Diego, California
- Asthma & Allergy Associates P.C. — Colorado Springs, Colorado
- Dr. Henry J. Kanarek Allergy, Asthma & Immunology — Overland Park, Kansas
- Institute For Asthma & Allergy — Chevy Chase, Maryland
- Mississippi Center for Advanced Medicine — Madison, Mississippi
- Washington University School of Medicine — St Louis, Missouri
- Duke Health — Durham, North Carolina
- University of Cincinnati (UC) Physicians Company, LLC — Cincinnati, Ohio
- Optimed Research, LTD — Columbus, Ohio
- The Pennsylvania State University (Penn State) Milton S. Hershey Medical Center — Hershey, Pennsylvania
- AARA Research Center — Dallas, Texas
- Royal Prince Alfred Hospital, — Camperdown,
- Hospital Universitario Vall d'Hebron — Barcelona,
- Hospital Universitario La Paz — Madrid,
💰 Cost, Insurance & Patient Rights
- 100% Free Items: The investigational treatment and protocol-required procedures are supplied at zero cost by the sponsor.
- Routine Patient Care: Under federal law (ACA § 2709) and many state statutes, routine care costs are covered during participation.
- Voluntary Participation: You may withdraw at any time without affecting your standard medical care.
More studies in Hereditary Angioedema
- A Study to Assess the Long-Term Safety and Efficacy of Donidalorsen in the Prophylactic Treatment of Hereditary Angioedema (HAE) — Paradise Valley, Arizona
- HAELO: A Phase 3 Study to Evaluate NTLA-2002 in Participants With Hereditary Angioedema (HAE) — Scottsdale, Arizona
- A Long-term Study of STAR-0215 in Participants With Hereditary Angioedema — Birmingham, Alabama
- Berotralstat Treatment in Children With Hereditary Angioedema — Vienna, Ontario
- Safety and Effectiveness of Sebetralstat (KVD900) for Short-Term Prophylaxis Before Procedures in People With Hereditary Angioedema (KONTROL) — Colorado Springs, Colorado
Explore by condition: All Hereditary Angioedema clinical trials
💡 How to Participate
- Check the checklist above to see if you may qualify.
- Review the official NIH record below to find the coordinating site.
- Ask about the visit schedule, what is covered at no cost, and any travel support.
- Confirm with your own doctor before making a decision.