Recent Updates
Recently added Catalysts

Dose 1 of BMN 331

Phase 1

Hereditary Angioedema | Gene therapy | Immunology |BioMarin Pharmaceutical Inc.|Last Updated: May 16, 2024

Success Probability
Subscribe to view
Market & Valuation
Subscribe to view
Trial Design
UNCONTROLLEDDMC
Total Trials1
Total Enrollment44
FDA Designations
No designations recorded
Clinical trial landscape

Dose 1 of BMN 331 · 1 trial · 2 indications

Phase 1 1
NCT05121376A Gene Therapy Study of BMN 331 in Subjects With Hereditary AngioedemaHereditary Angioedema
ACTIVE NOT_RECRUITING44 Analytics
PHASE1ACTIVE NOT_RECRUITING
A Gene Therapy Study of BMN 331 in Subjects With Hereditary Angioedema
Hereditary Angioedema
NCT0512137644
Unlock trial analytics
Study Endpoints
Primary Endpoints
Number of participants with treatment-emergent adverse events following a single IV administration of BMN 331
At 5 years
Secondary Endpoints
Time-normalized number of investigator-confirmed HAE attacks
At 5 years
Time-normalized number of investigator-confirmed HAE attacks by severity (mild, moderate, severe)
At 5 years
Time-normalized use of HAE-specific medication
At 5 years
Unlock Study Endpoints
Study Design & Arms
AllocationNA
MaskingNONE
ModelSEQUENTIAL
PurposeTREATMENT
Treatment Arms
ArmTypeDescription
BMN 331EXPERIMENTALAAV Gene Therapy Infusion
Interventions
NameTypeDescription
Dose 1 of BMN 331GENETICBMN 331 AAV Gene Therapy
Dose 2 of BMN 331GENETICBMN 331 AAV Gene Therapy
Dose 3 of BMN 331GENETICBMN 331 AAV Gene Therapy
Dose 4 of BMN 331GENETICBMN 331 AAV Gene Therapy
Dose 5 of BMN 331GENETICBMN 331 AAV Gene Therapy
Dose 6 of BMN 331GENETICBMN 331 AAV Gene Therapy
Dose 7 of BMN 331GENETICBMN 331 AAV Gene Therapy
Unlock Study Design Details
Eligibility Criteria
Age Range18 Years to N/A
SexALL
Healthy VolunteersNo
Study Sites16

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 SERPING...

Countries:United StatesAustraliaSpain
Unlock Eligibility Criteria
Competitive Landscape -Hereditary Angioedema 9 trials
CompanyTickerTrialsLead PhaseDrugs
Intellia Therapeutics, Inc.NTLA3PHASE3NTLA-2002, Normal Saline Administration
BioCryst Pharmaceuticals, Inc.BCRX2PHASE3Berotralstat
Ionis Pharmaceuticals, Inc.IONS1PHASE3Donidalorsen
Pharvaris N.V.PHVS1PHASE2deucrictibant
BioMarin Pharmaceutical Inc.BMRN1PHASE1Dose 1 of BMN 331
Astria Therapeutics, Inc.ATXS1PHASE2STAR-0215
Unlock Competitive Intelligence
Recent Changes (Last 90 Days)
LOWMay 26, 2026NCT05121376primaryCompletionDate: changed
LOWMay 24, 2026NCT05121376studyFirstPostDate: changed