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NCT05121376 · ClinicalTrials.gov registry record · Phase 1
A Gene Therapy Study of BMN 331 in Subjects With Hereditary Angioedema
A Phase 1 study of Hereditary Angioedema and HAE, sponsored by BioMarin Pharmaceutical.
- Active
- Registry status
- Phase 1
- Development phase
- 44
- Enrollment target
- 16
- Study locations
NCT05121376 is a Phase 1 study of Hereditary Angioedema and HAE that is active but no longer recruiting, run by BioMarin Pharmaceutical. The registered enrollment target is 44 participants, below the 73-participant average among 14 other Hereditary Angioedema trials with a reported enrollment target (40% lower). The trial reports 16 study locations across 13 states.
The verdict
NCT05121376, a Phase 1 study of Hereditary Angioedema and HAE, is active but no longer recruiting, sponsored by BioMarin Pharmaceutical.
- ACTIVE NOT RECRUITING
- Registry status
- Phase 1
- Development phase
- 44 participants
- Enrollment target
- 16
- Study locations
Study Summary
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.
Conditions Studied
Interventions
- GENETIC 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
Study Locations (16)
Other
- Royal Prince Alfred Hospital, - Camperdown
- Hospital Universitario Vall d'Hebron - Barcelona
- Hospital Universitario La Paz - Madrid
Ohio
- University of Cincinnati (UC) Physicians Company, LLC - Cincinnati
- Optimed Research, LTD - Columbus
Alabama
- AllerVie Clinical Research - Birmingham
Arizona
- Medical Research of Arizona - Scottsdale
California
- University of California San Diego - San Diego
Colorado
- Asthma & Allergy Associates P.C. - Colorado Springs
Kansas
- Dr. Henry J. Kanarek Allergy, Asthma & Immunology - Overland Park
Maryland
- Institute For Asthma & Allergy - Chevy Chase
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 44 participants |
| Start Date | 2022-02-15 |
| Est. Completion | 2028-11 |
| Phase | Phase 1 |
Interested in This Trial?
Full Details on ClinicalTrials.gov ↗What the Registry Record Tells You About NCT05121376
The ClinicalTrials.gov registry entry for NCT05121376 describes a study currently listed as active not recruiting, categorized as Phase 1. The registered enrollment target is 44 participants, a figure that helps gauge the scale of data the investigators plan to collect, below the 73-participant average among 14 other Hereditary Angioedema trials with a reported enrollment target (40% lower). The listed sponsor is BioMarin Pharmaceutical, which has 80 total studies on file at ClinicalTrials.gov.
The record links to 2 conditions, with Hereditary Angioedema appearing as the primary indexed condition, and to 5 interventions - of which Dose 1 of BMN 331 is the first listed.
NCT05121376 reports 16 study locations spanning 13 distinct geographic areas - top geographies include Other, Ohio, Alabama.
Frequently Asked Questions
What is clinical trial NCT05121376 about?
NCT05121376 is a clinical study titled "A Gene Therapy Study of BMN 331 in Subjects With Hereditary Angioedema". 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 ge...
What is the current status of trial NCT05121376?
This trial is currently active not recruiting. It is a Phase 1 study. The enrollment target is 44 participants. The study started on 2022-02-15. Estimated completion is 2028-11.
What conditions does trial NCT05121376 study?
This clinical trial studies the following conditions: Hereditary Angioedema, HAE.
What interventions are being tested in trial NCT05121376?
The interventions under investigation include: 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).
Who is sponsoring clinical trial NCT05121376?
This trial is sponsored by BioMarin Pharmaceutical, which has 80 total clinical trials registered on ClinicalTrials.gov.
Where is trial NCT05121376 being conducted?
This trial has 16 study locations across Alabama, Arizona, California, Colorado, Kansas. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
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