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Gene Therapy Trial Report

Summary

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


NCTID NCT05121376 (View at clinicaltrials.gov)
Description
Development Status Inactive
Indication Hereditary Angioedema
Disease Ontology Term DOID:14735
Compound Name BMN 331
Sponsor BioMarin Pharmaceutical
Funder Type Industry
Recruitment Status
Active not recruiting
Enrollment Count 44 (ESTIMATED)
Results Posted Not Available

Therapy Information


Target Gene/Variant SERPING1
Therapy Type Gene transfer
Therapy Route In-vivo
Mechanism of Action Functional gene replacement
Route of Administration Intravenous
Drug Product Type Viral vector
Target Tissue/Cell Liver
Delivery System Viral transduction
Vector Type AAV5
Dose 1 6E13 vg/kg
Dose 2 Other unknown doses

Study Record Dates


Current Stage Phase1, Phase2
Submit Date 2021-10-28
Completion Date 2028-11
Last Update 2024-05-16

Participation Criteria


Eligible Age >=18 Years
Standard Ages Adult, Older adult
Sexes Eligible for Study ALL
Eligibility Criteria
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)
View Inclusion and Exclusion Criteria at ClinicalTrials.gov

Locations


No.of Trial Sites 16
Locations United States,Australia,Spain

Regulatory Information


Has US IND True
FDA Designations Orphan Drug Designation
Recent Updates Biomarin announced they were discontinuing this program April 2024

Resources/Links