Gene Therapy for Hereditary Angioedema (HAErmony-1 Trial)

Palo Alto (17 mi)

Age: 18+

Sex: Any

Travel: May be covered

Time Reimbursement: Varies

Trial Phase: Phase 1 & 2

Waitlist Available

Sponsor: BioMarin Pharmaceutical

No Placebo Group

Trial Summary

What is the purpose of this trial?This trial tests BMN 331, a gene therapy given through an IV, for people with hereditary angioedema (HAE) who lack a specific protein. The therapy uses a virus to deliver a healthy gene to liver cells, enabling them to produce the missing protein. The study aims to see if this treatment is safe and effective over several years. BMN 331 is a gene therapy that uses a virus to deliver a healthy gene to liver cells, a method that has shown promise in treating various genetic disorders.

Eligibility Criteria

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.

Inclusion Criteria

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

Exclusion Criteria

I cannot use steroids due to glaucoma or untreated osteoporosis.

I have received gene therapy before.

I have a serious health condition besides skin cancer that needs regular treatment.

I have a history or risk of serious blood clots.

I do not have any active infections or conditions that weaken my immune system.

I have a significant liver condition, like NASH, hepatitis B or C, or autoimmune hepatitis.

Treatment Details

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).

1Treatment groups

Experimental Treatment

Group I: BMN 331Experimental Treatment7 Interventions

AAV Gene Therapy Infusion