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Gene Therapy Vector

Gene Therapy for Hereditary Angioedema (HAErmony-1 Trial)

Phase 1 & 2
Waitlist Available
Research Sponsored by BioMarin Pharmaceutical
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
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
Be older than 18 years old
Timeline
Screening 3 weeks
Treatment Varies
Follow Up at 5 years
Awards & highlights

HAErmony-1 Trial Summary

This trial is testing a gene therapy, BMN 331, for the treatment of hereditary angioedema (HAE) due to C1 Esterase Inhibitor (C1-INH) protein deficiency.

Who is the study for?
Adults diagnosed with Hereditary Angioedema (HAE) due to C1-INH deficiency, who are on a stable HAE medication regimen or have had frequent attacks. Participants must be able to manage acute attacks at home, avoid alcohol for 52 weeks post-treatment, and use effective contraception. Excluded are those with recent high-dose androgen use, active infections including COVID-19, certain medical conditions like untreated osteoporosis or significant liver disease, prior gene therapy treatment, or risk of thrombosis.Check my eligibility
What is being tested?
The trial is testing BMN 331 gene therapy as a potential treatment for HAE. It involves different doses of the drug given through an IV infusion using an AAV5 virus vector designed to express normal human C1 Esterase Inhibitor in the liver. This Phase 1/2 study will gradually increase doses (dose-escalation) and then expand the number of participants at selected dose levels (dose-expansion).See study design
What are the potential side effects?
Potential side effects may include reactions related to immune response against the viral vector or infused protein such as fatigue, headache, muscle pain; possible liver inflammation; increased risk of blood clots; and complications from long-term glucocorticosteroid use if required.

HAErmony-1 Trial Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

HAErmony-1 Trial Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~at 5 years
This trial's timeline: 3 weeks for screening, Varies for treatment, and at 5 years for reporting.

Treatment Details

Study Objectives

Outcome measures can provide a clearer picture of what you can expect from a treatment.
Primary outcome measures
Number of participants with treatment-emergent adverse events following a single IV administration of BMN 331
Secondary outcome measures
Detection of neutralizing antibodies against C1-INH following BMN 331 infusion
Detection of total antibodies against AAV5 capsid following BMN 331 infusion
Detection of total antibodies against C1-INH following BMN 331 infusion
+5 more

HAErmony-1 Trial Design

1Treatment groups
Experimental Treatment
Group I: BMN 331Experimental Treatment7 Interventions
AAV Gene Therapy Infusion

Find a Location

Who is running the clinical trial?

BioMarin PharmaceuticalLead Sponsor
157 Previous Clinical Trials
189,967 Total Patients Enrolled
MD Medical DirectorStudy DirectorBioMarin Pharmaceutical

Media Library

BMN 331 (Gene Therapy Vector) Clinical Trial Eligibility Overview. Trial Name: NCT05121376 — Phase 1 & 2
Hereditary Angioedema Research Study Groups: BMN 331
Hereditary Angioedema Clinical Trial 2023: BMN 331 Highlights & Side Effects. Trial Name: NCT05121376 — Phase 1 & 2
BMN 331 (Gene Therapy Vector) 2023 Treatment Timeline for Medical Study. Trial Name: NCT05121376 — Phase 1 & 2
~29 spots leftby Nov 2028
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