Clinical trials on Hereditary Angioedema

Understanding Hereditary Angioedema

Hereditary Angioedema (HAE) is a rare, genetic disorder characterized by unexpected and recurrent episodes of severe swelling. The swelling can occur in various parts of the body, including the extremities, face, gastrointestinal tract, and airways. This condition is caused by a deficiency or dysfunction of the C1 inhibitor protein, which plays a crucial role in regulating the inflammatory pathways in the body. There are three main types of HAE, categorized based on the underlying genetic cause and the levels or functionality of the C1 inhibitor.

The symptoms of HAE can be life-threatening, especially when the swelling affects the airways, leading to difficulty breathing. Swelling in the gastrointestinal tract can cause severe abdominal pain, nausea, and vomiting, often leading to misdiagnosis as a common abdominal ailment. The unpredictability of the attacks and the potential for severe complications necessitate a comprehensive management plan. Treatment options for HAE include on-demand treatment to manage acute attacks and prophylactic treatments to prevent the occurrence of attacks. Advances in medical research have led to the development of several effective therapies that target the underlying mechanisms of the disease, significantly improving the quality of life for individuals with HAE.

Diagnosis of HAE involves a combination of clinical evaluation and laboratory testing to measure the levels and functionality of the C1 inhibitor, as well as genetic testing to identify mutations in the genes responsible for the condition. Early diagnosis and appropriate management are crucial to prevent complications and improve outcomes for patients with HAE. Despite its challenges, with proper treatment and care, individuals with HAE can lead full and active lives.

Prognosis for Hereditary Angioedema

Hereditary Angioedema is a rare genetic disorder characterized by unexpected and recurrent episodes of severe swelling. The long-term prospects for individuals with Hereditary Angioedema have improved significantly with advances in disease awareness and diagnostic methods. While the condition is chronic and currently has no cure, early diagnosis and modern management strategies can help control symptoms and reduce the frequency and severity of attacks. Those affected by Hereditary Angioedema can lead full and active lives, although ongoing treatment may be required to prevent and manage acute episodes. Regular monitoring and personalized care plans are essential for maintaining a good quality of life. With appropriate medical support, it is possible to manage the condition effectively and minimize its impact on daily activities.

Complications in Hereditary Angioedema

Hereditary Angioedema (HAE) can lead to unexpected swelling in various parts of the body, including the limbs, face, intestinal tract, and airways. Swelling in the limbs and face may cause discomfort and disfigurement, affecting self-esteem and daily activities. When the intestines are involved, it can result in severe abdominal pain, nausea, and vomiting, which can be mistaken for other medical conditions and may interfere with normal eating or drinking. The most serious complication arises when swelling occurs in the airways, which can lead to difficulty breathing and, if not managed promptly, may become life-threatening. These episodes can be unpredictable and have a profound impact on quality of life, causing anxiety and limiting the ability to work, attend school, or participate in social activities.

Treatment Methods for Hereditary Angioedema

For the management of Hereditary Angioedema, several non-clinical trial treatments are recommended:

  • Dietary adjustments can play a role; avoidance of foods that trigger symptoms is advised.
  • Regular physical activity may contribute to maintaining overall health, but consultation with a healthcare provider is important for tailored exercise guidance.

Pharmacotherapy options include:

  • Medications that assist in preventing attacks or treating symptoms. These may be prescribed by healthcare professionals and tailored to the needs of the individual.

Modern technology offers tools for tracking symptoms and triggers, facilitating improved management of the condition. Applications and devices can assist in monitoring health and effective communication with the healthcare team.

Incorporation of these methods into daily routines can contribute to the management of Hereditary Angioedema, alongside any treatments under the guidance of healthcare providers. Adherence to a personalized plan that considers individual triggers and symptoms is essential.

  • CT-EU-00057347

    Long-term study on donidalorsen in hereditary angioedema

    This is a research assessing the long-term safety and effectiveness of donidalorsen in the prophylactic treatment of hereditary angioedema (HAE-1 and HAE-2). The study includes two groups of participants: 1) individuals transitioning from another study with donidalorsen, and 2) new participants not transitioning from another donidalorsen study, who were previously on prophylactic therapy with lanadelumab, berotralstat, or a C1-esterase inhibitor (C1-INH). The duration of participation in the study is 70 or 76 weeks.

    • Donidalorsen
  • Long-term safety study of sebetralstat in treating hereditary angioedema

    This trial is about a medicine sebetralstat also known as KVD900, which is being tested as a treatment to quickly stop swelling attacks in teenagers and adults who have a condition called hereditary angioedema (HAE). HAE is a rare, inherited disorder that causes episodes of swelling in different parts of the body. The study aims to find out how safe this medicine is if used for a long time. People who are 12 years and older and who have HAE type I or II can participate in this trial. One of the things the researchers will look at is how fast symptoms start to get better after taking this new medication.

    • Sebetralstat/KVD900
  • Berotralstat medication safety study in kids with hereditary angioedema

    This trial is all about a medicine called berotralstat and its use in little ones who have a condition called hereditary angioedema (HAE), which may cause sudden and serious swelling in different parts of the body. If aged between 2 and 12 years old, individuals may be eligible for participation in this study. The primary objective is to assess the safety of administering this medicine to children and evaluate the efficiency of their bodies in processing it. The dosage of the medication may vary based on the individual’s weight. The study comprises two main phases. Firstly, there is a 12-week period of standard care, followed by up to 144 weeks of treatment with berotralstat. Throughout this duration, continuous monitoring of participants’ well-being will be conducted, along with tracking the concentration of the medication in their system. These assessments aim to provide insights into the safety and effectiveness of berotralstat in pediatric patients.

    • Berotralstat
  • Testing the effect of sebetralstat treatment in hereditary angioedema

    This study focuses on adolescents with hereditary angioedema (HAE) type I or II. It aims to evaluate the effectiveness of Sebetralstat (KVD900), a drug intended to treat this disease. Teenagers taking part in this study will be given KVD900 to see how it affects their symptoms. The study is needed to understand how this drug may help treat HAE in younger patients, offering a potential new treatment option. Throughout the study, participants’ health and response to the drug will be closely monitored, with their safety and well-being being a priority. This research could significantly improve the treatment of hereditary angioedema in adolescents.

    • Sebetralstat/KVD900