HEREDITARY ANGIOEDEMA

This study tests a new drug called ADX-324 for hereditary angioedema (HAE), a rare genetic condition that causes sudden, severe swelling in the body. The trial has two parts: first, a small group of healthy volunteers will receive the drug to check its safety and how the body pro…

This phase 3 trial tests a one-time gene editing treatment called NTLA-2002 for people with hereditary angioedema (HAE), a rare genetic condition causing sudden, painful swelling attacks. About 80 participants will receive either the treatment or a placebo. The goal is to see if …

This study looks at whether it is safe for people aged 12 and older with hereditary angioedema (HAE) to switch from their current preventive medicine to a new monthly injection called garadacimab. Researchers will monitor for side effects over about 3 months. The goal is to see i…

This study tests a new treatment called NTLA-2002 for adults with hereditary angioedema (HAE), a condition that causes sudden, painful swelling attacks. The therapy uses gene editing to lower a protein that triggers these attacks. The goal is to see if it is safe and can reduce h…

This study tests a daily medication called berotralstat in 29 children aged 2 to under 12 who have hereditary angioedema (HAE), a condition that causes sudden, painful swelling attacks. The goal is to find the right dose based on weight and to check if it is safe and effective at…

This study gives people with hereditary angioedema (HAE) who were in earlier berotralstat trials continued access to the medication. About 139 participants will receive berotralstat and be monitored for side effects. The goal is to keep treating their condition while gathering mo…

This study tests the long-term safety and effectiveness of donidalorsen, a drug given as a shot under the skin, to prevent attacks of hereditary angioedema (HAE). HAE causes sudden, painful swelling in various body parts. The trial involves 154 participants who previously complet…

This study follows about 100 people with hereditary angioedema who previously received the gene-editing therapy NTLA-2002. Researchers will monitor side effects and how well the treatment controls swelling attacks over time. The goal is to understand if a single dose can provide …

This study tests a pill called deucrictibant for quickly treating swelling attacks in people with hereditary angioedema (HAE), including life-threatening throat swelling. About 150 adults who have had HAE attacks before will take the drug as needed and be monitored for safety and…