Clinical Research Directory

A Study of Navenibart in Participants With Hereditary Angioedema

This is a Phase 3 multicenter, randomized, double-blind, placebo-controlled clinical trial evaluating the safety and efficacy of subcutaneous administration of navenibart in adult and adolescent participants with type 1 or type 2 hereditary angioedema (HAE). The goal of this clinical trial is to evaluate the efficacy and safety of navenibart compared to placebo in preventing HAE attacks in participants with HAE.

Donidalorsen Treatment in Children With Hereditary Angioedema

The purpose of this study is to evaluate the safety, tolerability and pharmacokinetics of donidalorsen in pediatric participants with hereditary angioedema (HAE) Type I (HAE-1) or Type II (HAE-2).

A Study to Learn About the C1-Inhibitor Function as Diagnosis for Hereditary Angioedema

Hereditary angioedema (HAE) is a rare condition. It causes sudden swelling under the skin and inside the body, like in the belly, throat, or genitals. This swelling happens because of a temporary leak in blood vessels but does not cause itching or hives. HAE is classified based on the amount of a protein in the blood called C1-inhibitor (C1INH): HAE with normal C1INH levels and function (HAE-nC1INH) and HAE with deficiency in C1INH levels (HAE-C1INH-Type1) or dysfunction (HAE-C1INH-Type2). This study will focus on the practical use and accuracy of measuring the C1INH function alone to diagnose HAE-C1INH-Type1 and HAE-C1INH-Type2 compared to the tests used in normal clinical practice in Algeria. The main goal of the study is to see how well a test focusing on the C1INH function alone works to diagnose HAE-C1INH as compared to the tests used in normal clinical practice (standard of care or SoC) in Algeria. Another aim is to determine a reference value (helps in determining the accuracy) of the C1INH function test. This study will also help to find out how many people who are thought to have HAE or who have family members with HAE actually get diagnosed and to gather participants' health background information, such as their age when they were diagnosed, what signs and symptoms they had, how long it took to get diagnosed, and how they were sent to the doctors or specialists who treated them. During the study all participants will undergo two different methods of HAE testing: the test focusing on the C1INH function alone and the SoC tests. Test results will be confirmed via a second test run for newly index cases or for discordant results, but participants with a test result of "no HAE" and positive cases recruited through family screening will not undergo a second confirmatory test. In case of discordant test results in the second round, participants will undergo a third confirmatory test round. Participants can visit the clinic up to three times during the study. No further follow up is planned for participants, even for those who are diagnosed with HAE.

Long-Term, Open-label Study of Oral Deucrictibant Extended-Release Tablet for Prophylaxis Against Angioedema Attacks in Adolescents and Adults With HAE

This is a Phase 3, multicenter, long-term, open-label study to evaluate the safety and efficacy of once-daily orally administered deucrictibant extended-release tablet for prophylaxis to prevent angioedema attacks in participants aged ≥12 years with Hereditary Angioedema

Extension Study of Oral PHA-022121 for Acute Treatment of Angioedema Attacks in Patients With Hereditary Angioedema

This study evaluates the safety and efficacy of long-term on-demand treatment with orally administered deucrictibant for acute hereditary angioedema (HAE) attacks, including laryngeal attacks. The study will enroll participants from Study PHA022121-C201 (NCT04618211), Study PHA022121-C306 (NCT06343779) and deucrictibant treatment naïve HAE-nC1INH adult participants who elect to participate in this extension study and meet the eligibility requirements.

A Study of Lanadelumab in Teenagers and Adults With Hereditary Angioedema (HAE) in the Kingdom of Saudi Arabia

Hereditary angioedema (HAE) is a rare condition. It causes sudden swelling under the skin and inside the body, like in the belly, throat, or genitals. This swelling happens because of a temporary leak in blood vessels but does not cause itching or hives. HAE is classified based on the amount of a protein in the blood called C1 inhibitor (C1INH): HAE with normal C1INH levels (HAE-nC1INH) and HAE with limited or insufficient C1INH levels (HAE-C1INH); HAE-C1INH can be divided into Type 1, with low levels of C1INH, and Type 2, in which the protein is there, but does not work properly. This study will concentrate on people with HAE-C1INH Type 1 or 2 who have received Takhzyro® (lanadelumab) as prophylactic treatment for at least half a year (6 months). Prophylactic means that treatment is given to prevent the happening of HAE attacks. The main goal of the study is to see how well Takhzyro® works in everyday life to reduce the condition's activity after 6 months of treatment, or 12 months (if data is available). This will be measured by checking the change of the HAE activity from before treatment to after 6 months of treatment. The study design will permit a study follow-up of up to 12 months following the index event (i.e. date of first dose administration of Takhzyro®) unless the patient discontinues the index treatment, dies or is lost to follow-up within this timeframe. Chart abstractions will only occur once patients have at least 6 months' duration between the index event date and the date of chart abstraction initiation. Other goals are to find out how a person's quality of life changes after using Takhzyro® for 6 months, how often they had attacks before and after treatment and to learn which factors may have an impact on the treatment. The study will only look at data already existing in the participants' medical records. No treatment will be given as part of the study.

A Study About the Diagnosis and Management of Hereditary Angioedema (HAE) in Egypt

Hereditary angioedema (HAE) is a rare condition. It causes sudden swelling under the skin and inside the body, like in the belly, throat, or genitals. This swelling happens because of a temporary leak in blood vessels. HAE is classified based on the amount of a protein in the blood called C1 inhibitor (C1-INH): HAE with normal C1-INH levels and HAE with limited or insufficient C1-INH levels (C1-INH deficiency); HAE with C1-INH deficiency can be divided into Type1, with low levels of C1-INH, and Type 2, in which the protein is there, but does not work properly. This study will look at people with HAE Type1 or Type2. The main aim of this study is to describe the diagnosis and management of people with HAE Type1 or HAE Type2 in Egypt. Other aims are to learn more about people with HAE Type1 or Type2, including, but not limited to, other conditions they may have, family history, impact of HAE on daily life, if treatment is stopped and the reasons. During the study, information from already existing data in a participant's medical record will be reviewed and new data will be collected during routine visits of a participant to the study clinic.

A Study of Takhzyro in Teenagers and Adults With Hereditary Angioedema (HAE) in South Korea

Takhzyro is an approved treatment for hereditary angioedema (HAE) in South Korea. HAE is a rare condition which causes sudden swelling under the skin and inside the body, like in the belly, throat, or genitals. The main aim of this study is to check how safe Takhzyro is in teenagers and adults with HAE in everyday medical care in South Korea. Another aim is to see how well Takhzyro works in everyday medical care. The treatment of participants and any check-ups will be determined by the treating doctors according to their normal practice. Participants may visit the study clinic several times during the study.

A Phase 2 in Adult Subjects With Hereditary Angioedema

A Phase 2 in Adult Subjects with Hereditary Angioedema

Phase 3 Extension Study of ADX-324 in Participants With Hereditary Angioedema (HAE)

Study ADX-324-302 is an extension study for participants who complete the Phase 3 ADX-324-301 trial. The extension study will provide information about the safety and efficacy of additional dosing of ADX-324 in participants with Type I and Type II hereditary angioedema (HAE). The study will also include pharmacodynamic (PD), pharmacokinetic (PK), and health-related quality of life (HRQoL) measurements.

A Study of Lanadelumab in Children With Hereditary Angioedema (HAE) in Multiple Countries

HAE is a rare condition. It causes sudden swelling under the skin and inside the body, like in the belly, throat, or genitals. This swelling happens because of a temporary leak in blood vessels but does not cause itching or hives. HAE is classified based on the amount of a protein in the blood called C1 inhibitor (C1-INH): HAE with normal C1-INH levels and HAE with limited or insufficient C1-INH levels (C1-INH deficiency). This study will concentrate on children with HAE C1-INH deficiency who have received Takhzyro (Lanadelumab) as prophylactic treatment. The main goal of the study is to assess how well lanadelumab works in children with HAE-C1INH deficiency in everyday life. This will be measured by checking how long children who receive lanadelumab will be free of HAE attacks. Other goals are to understand how children with HAE-C1INH deficiency are being treated with lanadelumab, how well the treatment works for them, how safe it is and how often these children need to use healthcare services (like doctor visits, hospital stays, etc.) because of their condition. The study will only look at data already existing in the participants' medical records. No treatment will be given as part of the study.

Study of Oral Deucrictibant Extended-Release Tablet for Prophylaxis Against Angioedema Attacks in Adolescents and Adults With HAE

This is a Phase 3, multicenter, randomized, double-blind, placebo-controlled study to evaluate the efficacy and safety of once-daily orally administered deucrictibant extended-release tablet compared to placebo for prophylaxis to prevent angioedema attacks in participants aged ≥ 12 years with hereditary angioedema.

A Study of Lanadelumab in Teenagers and Adults With Hereditary Angioedema (HAE)

The main aim of this study is to compare the HAE attacks (both total and those requiring on-demand treatment) in the 12 months before and up to 24 months after initiation of treatment with lanadelumab. This study is conducted in the United Kingdom where participants were treated or about to be treated with landelumab according to their routine practice at hospitals. Data will be directly collected from participants via study diaries, questionnaires, their medical records, and study doctors treating them. Participants will be contacted every 3 months during study participation (via phone).

A Long-Term Study of Navenibart in Participants With Hereditary Angioedema

This is a Phase 3 multicenter trial in 2 parts to evaluate the long-term safety and efficacy of navenibart in adult and adolescent participants with hereditary angioedema (HAE) who participated in STAR-0215-301 (NCT06842823; ALPHA-ORBIT). Part 1 provides all participants with navenibart in a dose-controlled fashion; Part 2 introduces a personalized dosing option (PDO) for participants based on individual needs.

Oral Deucrictibant for Prophylactic and Acute Treatment in Hereditary Angioedema Patients

To assess the efficacy of prophylactic treatment with deucrictibant extended release (XR) tablet versus placebo in preventing angioedema attacks, and to also assess the efficacy of deucrictibant soft capsules as on-demand treatment versus placebo in achieving angioedema symptom relief during acute attacks.

HAE Burden and Crisis Management

The study has been designed as a French, multicentric survey study to obtain data to describe the burden of on-demand treatment for patients with hereditary angioedema (HAE). The online survey assesses various aspects of HAE and in particular the burden of HAE from the patients' and caregivers' perspectives.

Suicide Ideation in Hereditary Angioedema

This study focuses on patients with Hereditary Angioedema (HAE) to better understand how mental health affects overall well-being. Through a questionnaire, the investigators will assess the presence of suicidal thoughts, anxiety, and depression, as well as cardiovascular risk markers. By analyzing these factors together, the investigators aim to identify possible links between mental health and heart health in people with HAE. This research will help improve care strategies and highlight the importance of mental well-being in managing HAE.