Hereditary Angiodema (HAE)
Hereditary Angioedema HAE) also known as C1 Esterase Inhibitor Deficiency is a genetic condition that is very rare and can be life threatening. This condition is classified by swelling, or edema in different parts of the body. These can include the hands, feet, face, and airway (throat). Symptoms can also include abdominal pain, nausea, and vomiting. Swelling of the throat is one of the major concerns as this can cause asphyxiation, which can lead to death. This disease is hereditary, and those with the disease have a 50% chance of passing it on to their children. There are currently 7 FDA approved medications for the treatment of HAE, and 2 additional medications that are in the clinical trial process. At CRCA, we have multiple HAE studies that are available. With each study, medication and study-related care are provided at no cost, while those who qualify are provided compensation for your time and travel for each completed office visit.
Ruconest Prodrome Study
A prodrome is the medical term for clinical signs or symptoms that often proceed medical events. Many HAE patients report having prodromes prior to having an acute HAE attack. Ruconest (C1 INH-R) is currently FDA approved for the rescue of acute attacks. This study will investigate the effectiveness of using Ruconest at the start of the prodrome to see if it can prevent the symptoms from progressing to the development of an acute attack. Enrolling patients with HAE Type I & II
The purpose of this study is to evaluate the long-term safety and tolerability of daily dosing of oral BCX7353 in subjects with hereditary Angioedema (HAE) types I & II as well as to assess the effectiveness (HAE attack frequency, severity and disease activity over time) of BCX7353 during long-term administration.
IV and Injection Study
CSL Behring 312
The purpose of this study is to investigate a new type of medicine, know as a monoclonal antibody, as a preventative treatment for acute HAE attacks. CSL312 blocks the activity of a blood clotting factor called Factor XlIIa, and may be able to decrease the production of substances that make blood vessels release fluids into the surrounding tissue causing swelling. CSL312 is given by injection in a vein (IV) and under the skin, subcutaneously (SC). This study is open to patients with HAE types 1 & 2, and those patients with a laboratory confirmed Factor XIIa gene mutation.
The purpose of this study is to help answer the following question; How effective is lanadelumab (Takhzyro®) in preventing HAE attacks? What is the comparison on quality of life, work productivity and treatment satisfaction before and after lanadelumab treatment? Finally, what is the use of healthcare resources for HAE attacks before and after lanadelumab treatment? Individuals with Type I or Type II Hereditary Angioedema may qualify.