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Gene Therapy Vector
Gene Therapy for Hereditary Angioedema (HAErmony-1 Trial)
Phase 1 & 2
Recruiting
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 belowHAErmony-1 Trial Timeline
Screening ~ 3 weeks3 visits
Treatment ~ Varies
Follow Up ~ at 5 years
Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~at 5 years
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 moreHAErmony-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
Eligibility Criteria:
This trial includes the following eligibility criteria:- I am 18 years old or older.I cannot use steroids due to glaucoma or untreated osteoporosis.I have used high-dose testosterone treatments in the past year.I have received gene therapy before.I can manage my sudden health attacks at home.I have a serious health condition besides skin cancer that needs regular treatment.I have been on long-term HAE medication for 6 months or used on-demand medication for frequent attacks.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.
Research Study Groups:
This trial has the following groups:- Group 1: BMN 331
Awards:
This trial has 1 awards, including:- No Placebo-Only Group - All patients enrolled in this study will receive some form of active treatment.
Timeline:
This trial has the following timeline:- Screening: It may take up to 3 Weeks to process to see if you qualify in this trial.
- Treatment: The duration you will receive the treatment varies.
- Follow Ups: You may be asked to continue sharing information regarding the trial for 6 Months after you stop receiving the treatment.
Frequently Asked Questions
These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.
How many people are participating in this clinical trial?
"That is correct, the online information notes that this study is looking for participants. The trial was created on February 15th, 2022 and updated November 8th of the same year. A total of 34 individuals are needed from 10 different sites."
Answered by AI
Could you please tell me how many different hospitals are conducting this trial at the moment?
"This trial is being conducted out of various locations including Washington University School of Medicine, AARA Research Center as well as 10 other centres."
Answered by AI
Are new participants currently being enrolled in this experiment?
"The latest information on clinicaltrials.gov suggests that this trial is currently recruiting patients. The original posting date was February 15th, 2022 and the page has been updated as recently as November 8th, 2022."
Answered by AI
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