Hereditary angioedema

This program provides access to an experimental oral medication called deucrictibant for people with hereditary angioedema (HAE) who have no satisfactory alternative treatments for acute attacks. Eligible participants, aged 12 to 75, take the capsule as needed to manage swelling …

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 program provides access to an investigational drug called donidalorsen for people with hereditary angioedema (HAE), a genetic condition that causes sudden, painful swelling in various body parts. The drug is given as a monthly or every-other-month injection under the skin. I…

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 program provides early access to an investigational drug called sebetralstat for children aged 2 to 11 with hereditary angioedema (HAE), a condition causing sudden, painful swelling. The drug is taken at the start of an attack to reduce symptoms. It is available to children …

This study compares two standard treatments for bleeding internal hemorrhoids: hemorrhoidal artery embolization (HAE) and rubber band ligation (RBL). HAE uses a catheter to block blood flow to the hemorrhoid with tiny particles or coils, while RBL uses rubber bands to cut off cir…

This Phase 3 trial tests an oral drug called deucrictibant in adults with acquired angioedema due to C1 inhibitor deficiency, a rare condition causing sudden, painful swelling attacks. The study has three parts: one to see if a daily extended-release tablet can prevent attacks, a…

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 follows 200 people with hereditary angioedema (HAE) who are starting garadacimab in routine medical care. Researchers will track how well the drug prevents swelling attacks, its safety, and its impact on quality of life over 48 months. The goal is to see if results fro…

This Phase 3 study tests a once-daily pill called deucrictibant to prevent swelling attacks in people aged 12 and older with hereditary angioedema (HAE). The trial will enroll 170 participants and monitor safety and attack rates over a long period. Unlike a cure, patients would n…

This study tests whether a single injection of Berinert, a protein that reduces inflammation, into a donor kidney just before transplant can improve kidney function in the first year. Researchers will compare this to a placebo in 180 high-risk patients receiving deceased-donor ki…

This study offers children aged 2 to 11 with hereditary angioedema (HAE) continued access to garadacimab, a lab-made antibody that helps prevent sudden, painful swelling attacks. Participants must have completed a prior study and shown good response with few or no attacks. The go…

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 tests a procedure called hemorrhoidal artery embolization (HAE) for people with internal hemorrhoids who have not had success with other treatments or cannot have surgery. The procedure blocks certain arteries to reduce blood flow to the hemorrhoid tissue, aiming to im…

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 …

This study is creating a large database to collect information on people with rare diseases like amyloidosis, sarcoidosis, and Gaucher disease. Researchers will track patients' health over time, including their symptoms, treatments, and outcomes. The goal is to improve diagnosis …