EICHORCondition: Hereditary Angioedema · HAE · Sponsor: BioMarin Pharmaceutical
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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.
This description comes directly from the study's public registry record.
No contact information is available for this study as per the public registry record.
Always discuss trial participation with your own doctor first.
Inclusion Criteria: 1. Female or male adults ( ≥ 18 years old) 2. 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 3. 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 4. Trained in self-administering acute attack treatment and is able to adequately manage acute attacks in a home setting 5. Willingness to abstain from consumption of alcohol for at least 52 weeks post BMN 331 infusion and to use highly effective contraception Exclusion Criteria: 1. Evidence of active or chronic infection, including severe acute respiratory syndrome coronavirus 2 (SARS-CoV-2), or any immunosuppressive disorder 2. Contraindication to using glucocorticosteroids GCS, including a diagnosis of glaucoma or untreated osteoporosis 3. 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 4. Prior gene therapy treatment 5. Prior use of high-dose attenuated androgens in the last 1 year prior to the study 6. History or current clinically relevant liver disease (eg, nonalcoholic steatohepatitis \[NASH\], or chronic viral hepatitis B or C \[HBV or HCV\] or autoimmune hepatitis) 7. Have a history or are at risk for clinically significant thromboembolic events (TEE) , or known underlying risk factor for thrombosis including thrombotic microangiopathy (TMA)
Reproduced word-for-word from the public registry record — the study team can answer questions about it.
| 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 | — |
+ 4 more locations — full list on the registry record.
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Source record: clinicaltrials.gov/study/NCT05121376