Hereditary angioedema – European Clinical Trials Information Network

A study of navenibart for long-term safety in patients with hereditary angioedema

Thu, 30 Apr 2026 13:08:42 +0000

This study involves Hereditary Angioedema, which is a rare inherited condition that causes episodes of swelling in different parts of the body, including the hands, feet, face, intestinal tract, and airways. These swelling episodes, called attacks, can be painful and potentially dangerous, especially when they affect the throat or abdomen. The treatment being tested is called Navenibart, also known by its code name STAR-0215. This medication is designed to work against a specific protein in the blood called plasma kallikrein, which plays a role in causing the swelling attacks. Some participants will receive Navenibart while others will receive placebo. The medication is given as an injection under the skin.

The purpose of this study is to assess the long-term safety and tolerability of Navenibart in people with Hereditary Angioedema. The study will also look at how well the medication works in preventing swelling attacks over an extended period of time. Participants in this study will be people who previously took part in another study called STAR-0215-301 and either completed that study or received at least two doses of the medication and were followed for at least two months after the second dose.

During the study, participants will receive regular doses of the medication, with a maximum daily dose of 600 milligrams and a total maximum dose of 9600 milligrams over a treatment period of up to 48 weeks. The study will track any side effects or health problems that occur during treatment. Researchers will also count how many swelling attacks participants experience, including how many are moderate or severe, and how many require emergency treatment. The study will measure whether participants have fewer attacks compared to before treatment, including whether they achieve reductions of 50 percent, 70 percent, or 90 percent in their attack rate, or whether they have no attacks at all. Quality of life will be measured using a questionnaire specifically designed for people with swelling conditions.

A Study of ADX-324 to Prevent Attacks in Patients With Hereditary Angioedema

Thu, 30 Apr 2026 13:08:17 +0000

This study is looking at a condition called Hereditary Angioedema, which is a rare genetic disorder that causes repeated episodes of severe swelling in different parts of the body such as the hands, feet, face, intestinal tract, and airways. These swelling episodes, called attacks, can be painful and potentially dangerous, especially when they affect the throat and airways. The study will test a medication called ADX-324, which is given as an injection under the skin, against placebo to see if it can help prevent these swelling attacks from happening. The purpose of the study is to find out if ADX-324 works to prevent swelling attacks in people with this condition.

The study is designed so that participants will be randomly assigned to receive either ADX-324 or placebo. The study is double-blind, which means that neither the participants nor the doctors will know who is receiving the actual medication or placebo during the treatment period. Before starting the treatment, there will be a screening period lasting up to eight weeks to confirm that participants have the right type of condition and experience swelling attacks frequently enough to be part of the study. During this screening time, doctors will check medical history and confirm the diagnosis through specific blood tests that measure certain proteins and complement levels in the blood.

The treatment period will last for 25 weeks, during which participants will receive regular injections of either ADX-324 or sterile normal saline, which is the placebo. Throughout the study, participants will need to keep track of any swelling attacks they experience and will have access to rescue medication that they have used before to treat attacks when they occur. The study will measure how many swelling attacks occur during the treatment period, how many attacks require the use of rescue medication, and how many participants remain free from attacks. Doctors will also look at the severity of any attacks that do occur and compare the results between those receiving ADX-324 and those receiving placebo.

A study of donidalorsen for children with hereditary angioedema

Wed, 29 Apr 2026 15:09:11 +0000

This study focuses on Hereditary Angioedema, a rare condition that causes unexpected swelling in different parts of the body. This swelling can occur under the skin or in deeper tissues, such as the throat or digestive tract. The study aims to evaluate the safety and how the body processes donidalorsen, an experimental medication provided as a subcutaneous injection, which is an injection given just under the skin.

During the study, participants will receive the medication ISIS 721744. Researchers will monitor how the body handles the drug and look for any side effects. The study will also observe changes in plasma, which is the liquid part of the blood, and levels of plasma kallikrein, a specific protein involved in the swelling process. This investigation will take place over a period of 12 months to see how the treatment affects the frequency and severity of swelling episodes.

Study of Navenibart to prevent attacks in adults and adolescents with Hereditary Angioedema

Wed, 29 Apr 2026 15:07:29 +0000

This study focuses on people with Hereditary Angioedema (HAE), a rare genetic condition that causes unexpected episodes of severe swelling in various parts of the body. The research evaluates a new medication called Navenibart (also known as STAR-0215), which is being tested to see if it can prevent HAE attacks from occurring.

The study compares Navenibart against placebo to determine how effective it is in reducing the frequency of HAE attacks. During the study, participants will receive either Navenibart or placebo as a subcutaneous (under the skin) injection. The treatment period lasts for 6 months, during which participants will be monitored for any HAE attacks and their severity.

Throughout the study, researchers will track how many HAE attacks occur and how severe they are. They will also monitor the overall well-being of participants and any changes in their quality of life. The medication will be tested in both adults and adolescents with HAE to evaluate its safety and effectiveness in preventing attacks.

Safety Study of Switching from Current Treatment to Garadacimab in Patients Age 12 and Older with Hereditary Angioedema

Wed, 29 Apr 2026 15:06:31 +0000

This study focuses on Hereditary Angioedema (HAE), a rare genetic condition that causes sudden episodes of swelling in various parts of the body. The study will evaluate a medication called garadacimab (also known as CSL312), which is given as an injection under the skin. The research aims to assess the safety of switching to garadacimab from other currently used preventive treatments for HAE.

The medication will be administered as a subcutaneous injection with doses up to 400 milligrams per day. The study will involve people who are currently using other preventive treatments for HAE and will monitor how their body responds when switching to garadacimab. The treatment period will last for approximately 3 months.

During the study, participants will receive regular doses of garadacimab, and their health will be closely monitored for any side effects or reactions to the medication. The study will also measure how much of the medication remains in the blood and whether the body produces antibodies against it. Additionally, participants will have the opportunity to share their experience with the new medication compared to their previous treatment.

Study on the Effect and Safety of BW-20805 for Adults with Hereditary Angioedema

Wed, 29 Apr 2026 15:04:23 +0000

This clinical trial is focused on studying the effects and safety of a treatment called BW-20805 Injection for adults with Hereditary Angioedema. Hereditary Angioedema is a rare genetic condition that causes sudden swelling in various parts of the body, such as the face, hands, and feet. The treatment being tested, BW-20805, is a solution for injection that is administered under the skin.

The purpose of the study is to determine how well BW-20805 can prevent attacks of Hereditary Angioedema. Participants in the study will receive the BW-20805 Injection and will be monitored over a period of time to see if there is a reduction in the frequency of their swelling attacks. The study will also look at the safety of the treatment by observing any side effects that may occur during the trial.

Participants will be involved in the study for several months, during which they will receive regular injections and attend follow-up visits to track their progress. The study aims to provide valuable information on whether BW-20805 can be an effective and safe option for managing Hereditary Angioedema in adults.

Study on NTLA-2002 for Treating Hereditary Angioedema in Patients: Evaluating the Effects of Ziclumeran and Lonvoguran

Wed, 29 Apr 2026 15:03:56 +0000

This clinical trial is focused on studying Hereditary Angioedema (HAE), a rare genetic condition that causes sudden swelling in various parts of the body. The study will evaluate a new treatment called NTLA-2002, which is a special type of medicine known as an advanced therapy medical product. This treatment involves a dispersion for infusion, which means it is given through a drip directly into the bloodstream. The active substances in NTLA-2002 are ziclumeran and lonvoguran, which are designed to target specific genetic components involved in HAE.

The purpose of the study is to assess how effective NTLA-2002 is in reducing the number of HAE attacks compared to a placebo. Participants in the study will receive either NTLA-2002 or a placebo through an intravenous infusion. The study will monitor participants over several weeks to see how the treatment affects the frequency and severity of their HAE attacks. The trial will also look at the overall quality of life for participants during the study period.

Throughout the study, participants will be closely observed to ensure their safety and to gather information on how well the treatment works. The study aims to provide valuable insights into the potential benefits of NTLA-2002 for people living with hereditary angioedema, offering hope for a new treatment option for this challenging condition.

Study of Deucrictibant Extended-Release Tablets for Prevention of Hereditary Angioedema Attacks in Adults and Adolescents

Wed, 29 Apr 2026 15:03:47 +0000

This study focuses on people with Hereditary Angioedema, a rare genetic condition that causes unexpected episodes of severe swelling in various parts of the body. The research evaluates a new oral medication called deucrictibant, which is being tested as a preventive treatment to reduce the frequency of swelling attacks. The study will compare deucrictibant extended-release tablets to a placebo in adolescents and adults.

The study will test if taking a 40 mg tablet of deucrictibant daily can prevent or reduce the occurrence of swelling attacks compared to taking a placebo. For comparison purposes, some participants will also have access to icatibant, an already approved medication that is injected under the skin to treat acute attacks when they occur.

The treatment period will last 24 weeks, during which participants will take either deucrictibant or placebo tablets. Throughout the study, participants will need to keep track of any swelling attacks they experience and may use emergency treatment if needed. The study will monitor how well the medication works and check for any potential side effects through regular health assessments.

Study of deucrictibant tablets for prevention of hereditary angioedema attacks in adults and adolescents

Wed, 29 Apr 2026 15:03:18 +0000

This clinical study focuses on testing a medication called deucrictibant for people with Hereditary Angioedema (HAE), a rare genetic condition that causes unexpected episodes of severe swelling in various parts of the body. These swelling attacks can affect the skin, digestive tract, and airways, potentially causing significant discomfort and health risks.

The purpose of this research is to evaluate how safe and effective deucrictibant extended-release tablets are when taken regularly to prevent HAE attacks. The medication will be given as a 40 mg oral tablet that patients take to help reduce the frequency and severity of swelling episodes.

This is a long-term study where participants will receive the study medication and be monitored for any side effects and changes in their condition. During the study, patients will continue to have access to their usual emergency medications for treating sudden HAE attacks. Participants will need to keep track of their symptoms and complete various questionnaires about their quality of life and how well the treatment is working for them.

Long-Term Safety Study of Berotralstat for Patients with Hereditary Angioedema from Previous Trials

Wed, 29 Apr 2026 15:01:48 +0000

This clinical trial is focused on studying a condition known as Hereditary Angioedema (HAE). HAE is a rare genetic disorder that causes sudden swelling in various parts of the body, such as the face, hands, and feet. The treatment being studied in this trial is a medication called Berotralstat, which is taken orally in the form of a capsule. Berotralstat is designed to help prevent the swelling attacks associated with HAE.

The purpose of this study is to monitor the long-term safety of Berotralstat in individuals who have previously participated in Berotralstat studies. Participants in this trial will continue to take Berotralstat over an extended period, allowing researchers to gather important information about its safety and any potential side effects. The study will provide ongoing access to Berotralstat for those who have been part of earlier clinical trials, ensuring they can continue their treatment while contributing to valuable research.

Throughout the study, participants will be monitored for any treatment-related side effects or serious adverse events. The trial will continue until another way to access the medication becomes available, or until the sponsor decides to stop the global development and commercialization of Berotralstat. This study aims to ensure that Berotralstat remains a safe and effective option for managing Hereditary Angioedema in the long term.

Study on the Safety and Effects of Berotralstat for Children Aged 2 to 12 with Hereditary Angioedema (HAE)

Wed, 29 Apr 2026 14:59:14 +0000

This clinical trial is focused on studying a condition called Hereditary Angioedema (HAE), which is a rare genetic disorder that causes sudden swelling in various parts of the body. The study is testing a medication called Berotralstat, which is taken orally to help prevent these swelling attacks. The medication is available in two forms: hard capsules known as Orladeyo 150 mg and granules, both containing the active substance Berotralstat. The purpose of the study is to evaluate the safety and how the body processes this medication in children aged 2 to less than 12 years who have HAE.

Participants in the study will take Berotralstat over a period of time, and researchers will monitor them to see how the medication affects their bodies and if it helps reduce the frequency and severity of HAE attacks. The study will also look at any side effects that may occur. The medication is taken by mouth, and the study will last for a maximum of 144 weeks. During this time, researchers will collect information on the number of HAE attacks, how long they last, and any other related symptoms.

The study aims to provide valuable information on the use of Berotralstat in young children with HAE, helping to understand its potential benefits and any risks involved. This information could be important for developing better treatment options for children with this condition in the future.

Study on the Safety of Donidalorsen for Patients with Hereditary Angioedema

Wed, 29 Apr 2026 14:58:21 +0000

This clinical trial is focused on studying Hereditary Angioedema, a rare genetic condition that causes sudden swelling in various parts of the body, such as the face, hands, and feet. The treatment being tested in this study is called ISIS 721744, also known by its active substance name, Donidalorsen. This medication is administered as an injection and is a type of treatment known as an antisense oligonucleotide, which is designed to target specific genetic material in the body.

The purpose of this study is to evaluate the safety of using ISIS 721744 over an extended period and to explore different dosing schedules. Participants in this study will receive the medication through injections and will be monitored for any side effects or adverse reactions. The study will also look at how often participants experience attacks of swelling and how the treatment affects their quality of life. Additionally, the study will measure certain substances in the blood to understand how the medication works in the body.

This study is an open-label extension, meaning that all participants will receive the active medication rather than a placebo. The trial will last for approximately 64 weeks, during which participants will have regular check-ups and assessments to ensure their safety and to gather information about the treatment’s effects. The goal is to provide valuable insights into the long-term use of ISIS 721744 for managing Hereditary Angioedema.

Study on the Long-Term Safety of STAR-0215 for Adults with Hereditary Angioedema

Wed, 29 Apr 2026 14:57:45 +0000

This clinical trial is focused on studying hereditary angioedema, a rare genetic condition that causes sudden swelling in various parts of the body, such as the face, hands, and feet. The study will use a treatment called STAR-0215, which is administered as a sterile solution through subcutaneous use, meaning it is injected under the skin. The purpose of the study is to assess the long-term safety and tolerability of this treatment in adults with hereditary angioedema.

Participants in the trial will receive repeat doses of STAR-0215 over a period of time. The study will monitor the occurrence of any adverse events, which are unexpected medical problems that may happen during the trial. Additionally, the study will observe changes in the frequency and severity of hereditary angioedema attacks, as well as the duration of these attacks. The trial will also look at how long it takes for an attack to occur after each dose and the number of days participants remain free from attacks.

Throughout the study, researchers will measure the concentration of STAR-0215 in the body and any changes in plasma kallikrein activity, which is a protein involved in the swelling process. The formation of antibodies against the drug will also be monitored. This trial aims to provide valuable information on the effectiveness and safety of STAR-0215 for individuals living with hereditary angioedema.

Study on C1 Esterase Inhibitor for Treating and Preventing Hereditary Angioedema Attacks in Patients with C1-INH Deficiency

Wed, 29 Apr 2026 14:57:24 +0000

This clinical trial is focused on studying a condition known as hereditary angioedema, which is a rare genetic disorder that causes sudden swelling in various parts of the body, such as the face, hands, and feet. The study is testing a treatment called C1-esterase inhibitor, which is derived from human plasma and is designed to help prevent and treat these swelling attacks. The treatment is given through an intravenous injection, meaning it is administered directly into a vein.

The purpose of the study is to determine if the C1-esterase inhibitor is more effective than a placebo in relieving the symptoms of an acute attack in people with hereditary angioedema. Participants in the study will receive either the treatment or a placebo, and neither the participants nor the researchers will know which one is being administered, a method known as a double-blind study. The study will monitor how quickly participants experience relief from their symptoms after receiving the injection.

Participants will be involved in the study for a period of time during which they will receive the treatment or placebo and be observed for any changes in their condition. The study aims to provide valuable information on the safety and effectiveness of the C1-esterase inhibitor in managing hereditary angioedema attacks, potentially leading to improved treatment options for those affected by this condition.

Study on Oral PHA-022121 for Preventing Angioedema Attacks in Patients with Hereditary Angioedema

Wed, 29 Apr 2026 14:47:38 +0000

This clinical trial is focused on studying a condition known as hereditary angioedema (HAE), which is a rare genetic disorder that causes sudden swelling in various parts of the body, such as the face, hands, and feet. The swelling is due to a deficiency in a protein called C1-Inhibitor. The study is testing a new treatment called PHA-022121, which is taken as a soft capsule by mouth. This medication is designed to prevent the swelling attacks associated with HAE by blocking a specific receptor in the body known as the bradykinin B2 receptor.

The purpose of the study is to evaluate the safety and effectiveness of PHA-022121 in preventing HAE attacks. Participants in the study will be randomly assigned to receive either the new medication or a placebo, which looks like the medication but does not contain the active ingredient. The study is conducted in two parts. In the first part, different doses of the medication will be tested to find the most effective dose. In the second part, the long-term safety of the medication will be assessed. Participants will take the medication over a period of time and will be monitored for any changes in the frequency and severity of their HAE attacks.

Throughout the study, participants will have regular check-ups to ensure their safety and to track the effectiveness of the treatment. The study aims to provide valuable information on whether PHA-022121 can be a safe and effective option for people with hereditary angioedema, potentially offering a new way to manage this challenging condition.

Study on Long-Term Safety of Donidalorsen for Patients with Hereditary Angioedema

Wed, 29 Apr 2026 14:30:03 +0000

This clinical trial is focused on studying Hereditary Angioedema (HAE), a rare genetic condition that causes sudden swelling in various parts of the body, such as the face, hands, and feet. The study will use a treatment called Donidalorsen, which is an injection designed to help prevent these swelling attacks. Donidalorsen is a type of medication known as an antisense oligonucleotide, which works by targeting specific genetic material to reduce the frequency of HAE attacks.

The purpose of this study is to evaluate the long-term safety of using Donidalorsen in patients with HAE. Participants in the study will receive regular injections of Donidalorsen and will be monitored over an extended period to assess how well the treatment works and to identify any potential side effects. The study will also track the number of HAE attacks participants experience each month and how the treatment affects their quality of life.

Throughout the study, participants will have regular check-ups with healthcare professionals to ensure their safety and to gather information on the effectiveness of the treatment. The study aims to provide valuable insights into the long-term use of Donidalorsen for managing Hereditary Angioedema and to improve the overall understanding of this condition.

Long-term safety study of garadacimab (CSL312) for prevention of hereditary angioedema attacks

Wed, 29 Apr 2026 14:29:47 +0000

This clinical trial focuses on studying Hereditary Angioedema, a rare genetic condition that causes severe swelling attacks in different parts of the body. The study examines a new medication called garadacimab (also known as CSL312) that is given as an injection under the skin to prevent these swelling attacks. The purpose is to evaluate how safe and effective this medication is when used for a long time in people who experience regular swelling attacks.

The medication is given as a solution for injection that is administered under the skin (subcutaneous injection). This is a long-term study where participants receive regular treatments with garadacimab to prevent swelling attacks. The study looks at how well the medication works in reducing the number and severity of swelling attacks compared to before treatment.

During the study, researchers track how many swelling attacks occur and how severe they are. They also monitor any side effects that participants may experience while using the medication. The study focuses particularly on seeing if the treatment can significantly reduce the frequency of swelling attacks and improve the quality of life for people with this condition.

Study on NTLA-2002 for Adults with Hereditary Angioedema Using Ziclumeran and Lonvoguran

Wed, 29 Apr 2026 14:29:31 +0000

This clinical trial is focused on studying a condition called Hereditary Angioedema (HAE), which is a rare genetic disorder that causes sudden swelling in various parts of the body, such as the face, hands, and feet. The study is testing a new treatment called NTLA-2002, which is designed to help manage and reduce the frequency of these swelling attacks. NTLA-2002 is a special type of treatment that uses advanced technology involving nucleic acids, which are the building blocks of genetic material. This treatment is given through an infusion, which means it is delivered directly into the bloodstream through a vein.

The purpose of this study is to evaluate the safety and effectiveness of NTLA-2002 in adults with Hereditary Angioedema. The study is divided into different phases. In the first phase, the main goal is to determine the safety of NTLA-2002 and find the right dose for further testing. In the second phase, the focus is on understanding how well NTLA-2002 can reduce the number of HAE attacks. Some participants may receive a placebo, which is a substance with no active medication, to compare the effects of NTLA-2002. There is also a part of the study where participants who initially received a placebo or a lower dose of NTLA-2002 may receive a higher dose to further assess safety.

Participants in the study will receive the treatment through an intravenous infusion, which means it is administered directly into a vein. The study will monitor participants over a period of time to observe any changes in the frequency of HAE attacks and to ensure the treatment is safe. The study aims to provide valuable information that could lead to better management of Hereditary Angioedema in the future.

Study on the Safety and Effects of STAR-0215 for Adults with Hereditary Angioedema

Wed, 29 Apr 2026 14:26:38 +0000

This clinical trial is focused on studying a condition known as Hereditary Angioedema (HAE), which is a rare genetic disorder that causes sudden and severe swelling in various parts of the body. The study is investigating a new treatment called STAR-0215, which is administered as a sterile solution through a small injection under the skin, known as subcutaneous use. The main goal of the study is to evaluate the safety and tolerability of this treatment in adults with Type I or Type II HAE.

Participants in the trial will receive either a single dose or multiple doses of STAR-0215. Throughout the study, researchers will monitor participants for any side effects and changes in their health, such as vital signs and laboratory test results. The study will also look at how often HAE attacks occur, how severe they are, and how long they last. Additionally, the study will measure the levels of STAR-0215 in the blood and check for any immune response to the treatment.

The trial is expected to continue until October 2024, with recruitment having started in September 2023. This research aims to provide valuable information about the potential of STAR-0215 as a treatment option for people living with Hereditary Angioedema, potentially improving their quality of life by reducing the frequency and severity of swelling episodes.

Study on the Safety and Effects of Garadacimab for Preventing Hereditary Angioedema in Children Aged 2 to 11

Wed, 29 Apr 2026 14:26:30 +0000

This clinical trial is focused on studying Hereditary Angioedema (HAE), a rare genetic condition that causes sudden swelling in various parts of the body. The study is testing a treatment called garadacimab, also known by its code name CSL312. This treatment is given as a solution for injection under the skin, known as subcutaneous use. The purpose of the study is to evaluate the safety and how the body processes this medication in children aged 2 to 11 years who have HAE.

Participants in the study will receive regular injections of garadacimab over a period of up to 12 months. The study will monitor the number of HAE attacks the participants experience and any side effects that may occur. Some participants may receive a placebo, which is a substance with no active medication, to compare the effects of the treatment. The study aims to understand how well garadacimab works in preventing HAE attacks and to ensure it is safe for use in children.

Throughout the study, researchers will collect information on the frequency and severity of HAE attacks, as well as any adverse events or side effects. This information will help determine the effectiveness of garadacimab in reducing the number of HAE attacks and improving the quality of life for children with this condition. The study is expected to continue until mid-2026, providing valuable insights into the treatment of Hereditary Angioedema in young patients.

Long-Term Safety Study of NTLA-2002 for Patients with Hereditary Angioedema, Using Messenger RNA Encoding Cas9 and HU-G012267

Wed, 29 Apr 2026 14:26:19 +0000

This clinical trial is focused on the long-term follow-up of individuals who have been treated with a medication called NTLA-2002. The study is specifically looking at people with a condition known as Hereditary Angioedema, which is a rare genetic disorder that causes sudden and severe swelling in different parts of the body. The treatment being studied, NTLA-2002, is a solution for infusion that contains messenger RNA, a type of genetic material, which is designed to target the kallikrein B1 gene (KLKB1). This gene is involved in the process that leads to the symptoms of Hereditary Angioedema.

The purpose of this study is to evaluate the long-term safety of NTLA-2002 in individuals who have previously received this treatment. Participants in the study will be monitored over an extended period to observe any potential side effects or adverse events related to the treatment. The study will also track the frequency of Hereditary Angioedema attacks and any changes in the use of medications needed to manage these attacks. Additionally, the study will assess changes in the overall quality of life for participants, using specific tools designed to measure this aspect.

Throughout the study, participants will attend regular visits to ensure their health and safety while collecting important data about the treatment’s effects. The study aims to provide valuable information about the long-term impact of NTLA-2002 on individuals with Hereditary Angioedema, contributing to a better understanding of how this treatment can be used effectively and safely in the future.