Clinical Trials

According to clinicaltrials.gov under the U.S. National Institutes of Health, the EU Clinical Trials Register, and the International Clinical Trials Registry Platform under World Health Organization (WHO) the following trials should be recruiting at this moment (updated October 2022):

A Gene Therapy Study of BMN 331 in Subjects with HAE – recruiting in the United States
An extension phase 2/3 study to test the safety of long-term administration of oral PHA-022121 for acute treatment of angioedema attacks in patients with HAE – recruiting in Bulgaria, Canada, Czech Republic, France, Germany, Hungary, Israel, Italy, Poland, Spain, the United Kingdom, the United States
An Open-label Extension Trial to Evaluate the Long- term Safety of KVD900 for On-Demand Treatment of Angioedema Attacks in Adolescent and Adult Patients with HAE – will be recruiting
A Phase III, Crossover Trial Evaluating the Efficacy and Safety of KVD900 for On-Demand Treatment of Angioedema Attacks in Adolescent and Adult Patients with HAE – recruiting in Australia, Canada, the Netherlands, North Macedonia, Puerto Rico, Spain, the United States
A Phase 3, Multicenter, Randomized, Single-Blind, Dose-Ranging, Crossover Study to Evaluate the Safety and Efficacy of Intravenous Administration of CINRYZE® (C1 Esterase Inhibitor [Human]) for the Prevention of Angioedema Attacks in Children 6 to 11 Years of Age with HAE – recruiting in Argentina, Germany, Italy, Mexico, Romania, the United Kingdom, the United States
A randomized, placebo-controlled, double-blind Phase III study of the efficacy and safety of recombinant human C1 inhibitor for the treatment of acute attacks in patients with HAE – recruiting in Italy
Assessment of the State of Health, Quality of Life and Expectations of Patients with HAE – recruiting in France
A Study in Adults with HAE Who Currently Receive Icatibant at Home – will be recruiting in the United Kingdom
A Study of Lanadelumab in Persons with HAE Type I or II – recruiting in Austria, Germany, Israel, Italy, Kuwait, Portugal, Spain, Switzerland
A Study of Lanadelumab in Teenagers and Adults with HAE – will be recruiting in the United Kingdom
A Study of Lanadelumab in Teenagers and Adults with HAE in Argentina – recruiting in Argentina
A Study of Lanadelumab (SHP643) in Chinese Participants with HAE – recruiting in China
A Study of STAR-0215 in Healthy Adult Participants – recruiting in the United States
A Study of the Burden of Illness and Treatment Patterns in Teenagers and Adults with HAE – recruiting in Canada, Croatia, Czech Republic, Estonia, Germany, Greece, Hungary, Ireland, Latvia, North Macedonia, Poland, Portugal, Romania, Serbia, Slovakia, Spain
A Study to Assess the Long-Term Safety and Efficacy of Donidalorsen in the Prophylactic Treatment of HAE – recruiting in the United States
A study to assess whether different doses of KVD824 are effective in preventing attacks of HAE – recruiting in Australia, Bulgaria, Canada, Czech Republic, France, Germany, Hungary, Italy, New Zealand, North Macedonia, Romania, the United Kingdom, the United States
A study to a) evaluate the tolerability and blood levels of KVD900 when given as a single dose to patients and b) to assess whether KVD900 is effective in treating attacks of swelling in patients with the genetic disease HAE – recruiting in Austria, Germany, Hungary, the Netherlands, North Macedonia, the United Kingdom, the United States
A study to evaluate if different doses of KVD900 are safe and effective in treating attacks in patients with HAE – recruiting in Australia, Bulgaria, Canada, France, Germany, Greece, Hungary, Israel, Italy, the Netherlands, New Zealand, North Macedonia, Poland, Romania, Spain, the United Kingdom, the United States
A Study to Evaluate NTLA-2002 in adults with HAE – recruiting in Australia, France, Germany, the Netherlands, New Zealand, the United Kingdom, the United States
A Study with Lanadelumab in Persons with HAE in Poland – recruiting in Poland
A Survey of Icatibant in Pediatric Participants with HAE – recruiting in Japan
A Survey of Lanadelumab in Participants with HAE – recruiting in Japan
A Trial to Evaluate the Efficacy and Safety of Different Doses of KVD824 for Prophylactic Treatment of HAE Type I or II – recruiting in Australia, Bulgaria, Canada, Czech Republic, France, Germany, Hungary, Italy, New Zealand, North Macedonia, Puerto Rico, the United Kingdom, the United States
Berotralstat Treatment in Children with HAE – will be recruiting in Austria, Canada, France, Germany, Israel, Italy, North Macedonia, Poland, Romania, Spain, the United Kingdom
Characterization of Rhythmicity Profiles of Bradykinin-mediated Angioedema Attacks Using a Tracking Smartphone Application – will be recruiting
CLOUD-R HAE REGISTRY – recruiting in France
C1 Inhibitor Registry in the Treatment of HAE Attacks – recruiting in Bulgaria, Croatia, Czech Republic, France, Germany, Hungary, Italy, the Netherlands, North Macedonia, Norway, Poland, Slovakia, Slovenia, Sweden
Contrast-Enhanced Ultrasound for the Evaluation of Changes in Tumor Blood Flow Surrounding HAE – recruiting in the United States
CSL312 (garadacimab) in the prevention of HAE attacks – recruiting in Canada, Germany, Hungary, Israel, Italy, the Netherlands, Spain, the United States
Dose-ranging Study of Oral PHA-022121 for Prophylaxis Against Angioedema Attacks in Patients with HAE Type I or Type II – recruiting in Bulgaria, Canada, Germany, Israel, Poland, the United Kingdom, the United States
Efficacy and Safety of Lanadelumab (SHP643) in Japanese Subjects with HAE – recruiting in Japan
FIRAZYR General Drug Use-Results Survey (Japan) – recruiting in Japan
Firazyr® Patient Registry (Icatibant Outcome Survey – IOS) – recruiting in Australia, Austria, Brazil, Czech Republic, Denmark, France, Germany, Greece, Ireland, Israel, Italy, Spain, Sweden, the United Kingdom
Global Registry to Gather Data on Natural History of Patients with HAE Type I and II – recruiting in Italy
Involvement of Monocytic B1 and B2 Receptors in Inflammation and Chronic Vascular Disease in Patients with Hereditary Bradykinetic Angioedema – recruiting in France
Lanadelumab tested in patients suffering from HAE with normal C1-Inhibitor – recruiting in Germany
Long term access to berotralstat for HAE subjects from previous clinical trials – recruiting in Czech Republic, France, North Macedonia, Slovakia, South Africa, South Korea
Long-term safety and efficacy of CSL312 (garadacimab) in the prophylactic treatment of HAE attacks – recruiting in Australia, Canada, Czech Republic, Germany, Hong Kong, Hungary, Israel, Italy, Japan, the Netherlands, New Zealand, Russia, Spain, Taiwan, the United Kingdom, the United States
NTLA-2002 in Adults with HAE – recruiting in the Netherlands, New Zealand, the United Kingdom
OASIS-HAE: A Study to Evaluate the Safety and Efficacy of Donidalorsen (ISIS 721744 or IONIS-PKK- LRx) in Participants with HAE – recruiting in Belgium, Bulgaria, Canada, France, Germany, Italy, Spain, the United Kingdom, the United States
Pathophysiological study for autoimmune dysregulation of HAE – recruiting in Japan
Patient Registry to Evaluate the Real-world Safety of Ruconest® – recruiting in the United States
Pharmacokinetics and Safety of Human Pasteurised C1-Inhibitor Concentrate (Berinert/CE1145) in Subjects with Congenital C1-INH Deficiency – recruiting in Italy
PK Subtrial in Adolescent Patients with HAE Type I or II Participating in the KVD900-302 Trial – will be recruiting
Study to Assess the Tolerability and Safety of Ecallantide in Children and Adolescents with HAE – recruiting in the United States
Study to Evaluate the Real-World Long-Term Effectiveness of Lanadelumab in Participants with HAE – recruiting in Austria, Germany, Israel, Switzerland, the United Kingdom
The influence of stress and lifestyle in HAE – recruiting in New Zealand
The Role of the Coagulation Pathways in Recurrent Angioedema – recruiting in France