HAErmony-1 Study

The HAErmony-1 study is a clinical research study (also known as a clinical trial) for adults with hereditary angioedema (HAE). 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 see if the study drug (investigational gene therapy) is safe and 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*:

  • 18 years of age or older
  • Diagnosed with HAE due to C1-INH deficiency
  • Use preventive HAE medication to prevent attacks or on-demand medication during HAE attacks
  • Have at least 1 HAE attack per month on average

*Additional requirements apply.

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:

  • Review of medical history and current/previous medications
  • Study assessments such as physical exams, liver imaging, and blood tests
  • Single dose of the study drug as an intravenous (IV) infusion (may need to stay at the study clinic overnight)
  • Study visit attendance (at the study clinic and/or via home healthcare provider)
  • HAE attack reporting via electronic diary

Find a study clinic