HEREDITARY ANGIOEDEMA

This early-stage study is testing a single injection of an experimental gene therapy called NTLA-2002 in adults with hereditary angioedema (HAE). The main goals are to see if the treatment is safe and if it can reduce the number of painful, unpredictable swelling attacks people e…

This study is testing the long-term safety of an oral medication called KVD900 for treating sudden swelling attacks in people with hereditary angioedema. The trial includes 145 patients aged 12 and older who have type I or II hereditary angioedema and have experienced at least tw…

This study is testing a daily oral medication called berotralstat to prevent painful and dangerous swelling attacks in children aged 2 to 12 with hereditary angioedema (HAE). Researchers are finding the right dose based on a child's weight and checking how safe the drug is for yo…

This study is testing the long-term safety and effectiveness of donidalorsen, a medication designed to prevent swelling attacks in people with hereditary angioedema (HAE). The research involves 154 participants who will receive the treatment for up to two years while researchers …

This study is testing whether a single infusion of an experimental gene therapy called NTLA-2002 can safely and effectively reduce the number of sudden, severe swelling attacks in people with hereditary angioedema (HAE). About 60 participants will be randomly assigned to receive …

This early-stage study is testing a new drug called ADX-324 to see if it's safe for people. First, healthy volunteers receive different doses. Then, a small group of patients with hereditary angioedema—a rare condition causing sudden, severe swelling—will try it. The main goal is…

This study is monitoring people who previously received an investigational gene therapy called NTLA-2002 for hereditary angioedema (HAE). The main goal is to track their long-term health, safety, and how well the treatment continues to control their HAE attacks over time. It is a…

This study provides continued access to the oral medication berotralstat for people with hereditary angioedema (HAE) who were in previous trials. It aims to monitor the long-term safety and tolerability of the drug for up to 10 years in adults and adolescents, and up to 5 years i…

This study is testing the long-term safety and effectiveness of an oral medication called deucrictibant for treating sudden swelling attacks in people with hereditary angioedema (HAE). It will enroll about 150 adults and adolescents who have HAE and have participated in related s…