A Gene Therapy Study of BMN 331 in Subjects With Hereditary Angioedema
NCT ID: NCT05121376
Last Updated: 2024-05-16
Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
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ACTIVE_NOT_RECRUITING
PHASE1/PHASE2
44 participants
INTERVENTIONAL
2022-02-15
2028-11-30
Brief Summary
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Detailed Description
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Study 331-201 is a two-part (part A and part B), first-in-human, Phase 1/2 study designed to assess the safety and efficacy of BMN 331 in patients with HAE. Subjects will be followed for 5 years following BMN 331 infusion. Part A of the study is a dose escalation phase designed to assess the preliminary safety of a single IV administration of BMN 331 and to determine whether there is a dose-dependent increase in C1-INH protein expression following administration of BMN 331. Part B is a dose expansion phase designed to demonstrate that up to three safe doses of BMN 331 (as determined in Part A) sustains a clinically meaningful increase in C1-INH levels.
Conditions
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Study Design
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NA
SEQUENTIAL
TREATMENT
NONE
Study Groups
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BMN 331
AAV Gene Therapy Infusion
Dose 1 of BMN 331
BMN 331 AAV Gene Therapy
Dose 2 of BMN 331
BMN 331 AAV Gene Therapy
Dose 3 of BMN 331
BMN 331 AAV Gene Therapy
Dose 4 of BMN 331
BMN 331 AAV Gene Therapy
Dose 5 of BMN 331
BMN 331 AAV Gene Therapy
Dose 6 of BMN 331
BMN 331 AAV Gene Therapy
Dose 7 of BMN 331
BMN 331 AAV Gene Therapy
Interventions
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Dose 1 of BMN 331
BMN 331 AAV Gene Therapy
Dose 2 of BMN 331
BMN 331 AAV Gene Therapy
Dose 3 of BMN 331
BMN 331 AAV Gene Therapy
Dose 4 of BMN 331
BMN 331 AAV Gene Therapy
Dose 5 of BMN 331
BMN 331 AAV Gene Therapy
Dose 6 of BMN 331
BMN 331 AAV Gene Therapy
Dose 7 of BMN 331
BMN 331 AAV Gene Therapy
Eligibility Criteria
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Inclusion Criteria
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
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)
18 Years
ALL
No
Sponsors
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BioMarin Pharmaceutical
INDUSTRY
Responsible Party
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Principal Investigators
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MD Medical Director
Role: STUDY_DIRECTOR
BioMarin Pharmaceutical
Locations
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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
Countries
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Other Identifiers
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331-201
Identifier Type: -
Identifier Source: org_study_id
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