This website was developed and funded by BioMarin Pharmaceutical, Inc.

HAErmony-1 Study

The HAErmony-1 study is a hereditary angioedema clinical trial (also known as a research study) for adults suffering from frequent HAE attacks. This study is testing an investigational study drug called BMN 331. BMN 331 has not been approved for use or determined to be safe or effective; it is under investigation in clinical trials.

What is the purpose of the HAErmony-1 study?

The main purpose of the study is to understand the safety of the study drug (investigational gene therapy) and if it might help improve HAE symptoms. Investigational means that the study drug has not been approved for public use and can only be used in clinical research studies. What we learn from this study may help scientists and doctors develop the study drug as a possible HAE therapy.

Who can join the HAErmony-1 study?

You may qualify if you meet the following requirements*:

*Additional requirements apply.

Contact a study clinic for further information on required criteria.

Study Clinic Locations

What will happen during the study?

For those who qualify, participation lasts about 5 years after receiving the study drug. There are weekly and monthly visits during the first year. After the first year, the visits occur every 3 months or every 6 months. Participation includes:

1. Medical history

Review of medical history and current/previous medications

2. Assessments

Study assessments such as physical exams, liver imaging, and blood tests

3. Receive the study

Single dose of the study drug as an intravenous (IV) infusion (participants will be required at least one overnight stay at the study clinic)

4. Study visits

Attend study visits (at the study clinic and/or via home healthcare provider)

5. Reporting

HAE attack reporting via electronic diary