Hereditary Angioedema (HAE) Clinical Trials and Treatments

Hereditary Angioedema results from mutations in genes encoding C1-esterase inhibitor (C1-INH), Factor XII, or other components of contact system regulation, leading to dysregulation of bradykinin production. C1-INH deficiency accounts for approximately 85% of cases (types I and II), with type I involving reduced C1-INH production and type II involving dysfunctional C1-INH protein. Bradykinin mediates vascular permeability changes, leading to angioedema.

The most common symptoms of Hereditary Angioedema include Sudden onset of deep tissue swelling (angioedema) without hives, Facial swelling affecting lips, tongue, and throat, Gastrointestinal attacks with severe abdominal pain, nausea, and vomiting, Swelling of extremities lasting hours to days, Potentially life-threatening airway swelling, among others. Approximately 1 in 10,000 to 50,000 people; estimated 5,000-10,000 people in the United States. Symptoms can range from mild to severe and may vary between individuals.

Trial Friend lists all active clinical trials for Hereditary Angioedema from ClinicalTrials.gov, with eligibility criteria translated into plain English. Patients can filter by trial phase and recruitment status, search by location, and use the Match Me tool to score trials against their profile. Research in Hereditary Angioedema is transforming treatment with a diverse array of new approaches targeting different points in the disease pathway. Kallikrein inhibitors, Factor XII inhibitors, and bradykinin receptor antagonists represent multiple strategic approaches to preventing attacks and reducing their frequency and severity.

Yes. As of September 2026, Trial Friend lists 5 FDA-approved drugs used to treat Hereditary Angioedema: Takhzyro (lanadelumab), first approved in 2018; Orladeyo (berotralstat), first approved in 2020; Andembry (garadacimab), first approved in 2025; Ekterly (sebetralstat), first approved in 2025; and Dawnzera (donidalorsen), first approved in 2025.

Each has its own page with what the label covers, side effects and how it is given, and the Treatments section of this page adds any further approved drugs found in FDA labeling data.

The FDA is due to decide on Lonvo-z (lonvoguran ziclumeran) by March 10, 2027 and Deucrictibant by April 23, 2027; the FDA calendar page carries the outcome the day it lands.

Hereditary Angioedema is caused by variants in the SERPING1, F12 and PLG genes. SERPING1 variants cause types I and II (C1-INH deficiency); F12 causes most HAE with normal C1-INH, with PLG and a few other genes in a minority.

It is autosomal dominant, so each child of an affected parent has a 1 in 2 chance of inheriting it. Approximately 1 in 10,000 to 50,000 people; estimated 5,000-10,000 people in the United States. Genetic testing confirms the diagnosis.

Yes. Genetic testing for Hereditary Angioedema looks at the SERPING1, F12 and PLG genes. SERPING1 variants cause types I and II (C1-INH deficiency); F12 causes most HAE with normal C1-INH, with PLG and a few other genes in a minority.

It is autosomal dominant, so each child of an affected parent has a 1 in 2 chance of inheriting it.

A confirmed variant establishes the diagnosis and is often required to enroll in gene-specific trials. The labs currently offering the test are listed in the Genetic Testing section of this page, pulled live from the NIH Genetic Testing Registry. A genetic counselor can help interpret the result and decide which relatives should be tested.

Eligibility for Hereditary Angioedema clinical trials depends on factors like age, diagnosis, treatment history, and overall health. Each trial sets its own inclusion and exclusion criteria. Trial Friend translates these requirements from medical language into plain English so patients can review them more easily. The Match Me tool can score your profile against active trials to help identify studies that may be a fit.

Participating in a Hereditary Angioedema clinical trial typically involves scheduled visits, medical tests, and close monitoring by a research team. Depending on the study phase, you may receive an investigational treatment, a placebo, or the current standard of care. Trials may last from a few weeks to several years. Study participation is voluntary, and you can withdraw at any time.