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

Running, not enrolling · Phase 1/Phase 2

Conditions studied: Hereditary Angioedema, HAE

In brief

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.

Key facts

Study ID

NCT05121376

Run by

BioMarin Pharmaceutical

People needed

44

Starts

2022-02-15

Expected to finish

2028-11-01

Last updated by the study team

2024-05-16

Who can join

Age: 18 and older. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• Female or male adults ( ≥ 18 years old)
• Part A only: Confirmed diagnosis of Type I HAE due to C1-INH deficiency confirmed by genotyping of the SERPING1 gene Part B only: Confirmed diagnosis of Type I or II HAE due to C1-INH deficiency confirmed by genotyping of the SERPING1 gene
• Currently using an HAE medication regimen that consists of a routine long-term prophylactic treatment for at least 6 months prior to enrollment or an on-demand therapy regimen for a documented attack frequency of at least 4 attacks within the last 12 months prior to enrollment or at least 2 attacks within the last 6 months prior to enrollment
• Trained in self-administering acute attack treatment and is able to adequately manage acute attacks in a home setting
• Willingness to abstain from consumption of alcohol for at least 52 weeks post BMN 331 infusion and to use highly effective contraception

You may not qualify if…

• Evidence of active or chronic infection, including severe acute respiratory syndrome coronavirus 2 (SARS-CoV-2), or any immunosuppressive disorder
• Contraindication to using glucocorticosteroids GCS, including a diagnosis of glaucoma or untreated osteoporosis
• Active malignancy (except non-melanoma skin cancer) autoimmune, metabolic (i.e., diabetes), hematologic, cardiac, or renal disease that is of clinical significance defined as requiring regular medical attention and treatment
• Prior gene therapy treatment
• Prior use of high-dose attenuated androgens in the last 1 year prior to the study
• History or current clinically relevant liver disease (eg, nonalcoholic steatohepatitis [NASH], or chronic viral hepatitis B or C [HBV or HCV] or autoimmune hepatitis)
• Have a history or are at risk for clinically significant thromboembolic events (TEE) , or known underlying risk factor for thrombosis including thrombotic microangiopathy (TMA)

Where it is running

• AllerVie Clinical Research — Birmingham, Alabama, United States
• Medical Research of Arizona — Scottsdale, Arizona, United States
• University of California San Diego — San Diego, California, United States
• Asthma & Allergy Associates P.C. — Colorado Springs, Colorado, United States
• Dr. Henry J. Kanarek Allergy, Asthma & Immunology — Overland Park, Kansas, United States
• Institute For Asthma & Allergy — Chevy Chase, Maryland, United States
• Mississippi Center for Advanced Medicine — Madison, Mississippi, United States
• Washington University School of Medicine — St Louis, Missouri, United States
• Duke Health — Durham, North Carolina, United States
• University of Cincinnati (UC) Physicians Company, LLC — Cincinnati, Ohio, United States
• Optimed Research, LTD — Columbus, Ohio, United States
• The Pennsylvania State University (Penn State) Milton S. Hershey Medical Center — Hershey, Pennsylvania, United States
• AARA Research Center — Dallas, Texas, United States
• Royal Prince Alfred Hospital, — Camperdown, Australia
• Hospital Universitario Vall d'Hebron — Barcelona, Spain
• Hospital Universitario La Paz — Madrid, Spain

Full record on ClinicalTrials.gov

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