Hereditary angioedema with C1Inh deficiency

This phase 3 study tests an intravenous C1 esterase inhibitor (C1-INH) in people with hereditary angioedema (HAE) to treat acute attacks and prevent attacks before medical procedures. About 124 participants, aged 2 and older, will receive either the drug or a placebo. The main go…

This study tests a drug called donidalorsen in 20 children aged 2 to 12 with hereditary angioedema (HAE), a condition that causes sudden, painful swelling. The goal is to see if the drug is safe and how it works in young bodies. Researchers will monitor side effects and measure h…

This phase 3 trial is testing an experimental drug called ADX-324 in 90 adults with hereditary angioedema (HAE) types 1 or 2. HAE causes sudden, painful swelling in the body. ADX-324 uses a technology called siRNA to lower levels of a protein involved in triggering these attacks.…

This study is for people with hereditary angioedema (HAE) who have already completed a previous trial of the drug ADX-324. ADX-324 is an RNA-based medicine designed to lower a protein involved in swelling attacks. The goal is to see if long-term use is safe and can reduce the num…

This study looks at whether measuring just one protein function (C1-inhibitor) can accurately diagnose hereditary angioedema (HAE), a rare condition causing sudden swelling. About 514 people in Algeria who are suspected of having HAE or have family members with it will take part.…

This study looks back at medical records of 50 people in Saudi Arabia with hereditary angioedema (HAE) who took Takhzyro (lanadelumab) to prevent attacks. Researchers want to see if the drug reduces HAE activity and improves quality of life after 6 to 12 months. No new treatments…

This study uses smartphone surveys to track the real-life burden of hereditary angioedema (HAE) in 30 adults. Participants answer quick questions about their symptoms, mood, and daily activities every other day for 8 weeks. The goal is to capture day-to-day changes that standard …