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 April 2023).

Read more about these and other clinical trials at:

www.clinicaltrials.gov
www.clinicaltrialsregister.eu
https://trialsearch.who.int/

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, Long Term Safety and Efficacy Study of Donidalorsen in the Prophylactic Treatment of HAE – recruiting in Belgium, Bulgaria, Canada, Denmark, France, Germany, Israel, Italy, the Netherlands, Poland, Spain, Turkey, the United Kingdom, the United States
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, Bulgaria, Canada, France, Greece, Hungary, Israel, Italy, the Netherlands, New Zealand, North Macedonia, Poland, Puerto Rico, Spain, the United Kingdom, 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
A safety and pharmacokinetic study of oral berotralstat for HAE attacks in pediatric patients – recruiting in Austria, Canada, France, Germany, Hungary, Israel, Italy, North Macedonia, Poland, Romania, Spain, the United Kingdom
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 –recruiting in the United Kingdom
A Study of Lanadelumab (SHP643) in Chinese Participants with HAE – recruiting in China
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 STAR-0215 in Healthy Adult Participants – recruiting in 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 Assess the Long-Term Safety and Efficacy of Donidalorsen in the Prophylactic Treatment of HAE – recruiting in Bulgaria, Canada, Italy, the Netherlands, Spain, the United States
A study to assess whether different doses of KVD824 are effective in preventing attacks of HAE Type I or Type II – 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 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, Japan, the Netherlands, New Zealand, Poland, Portugal, 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 to Review the Treatment and Outcomes of Teenagers and Adults with Non-histaminergic Angioedema with Normal C1 Inhibitor in Canada – will be recruiting in Canada
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
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 – recruiting in France, the Netherlands, the United States
Berotralstat Treatment in Children with HAE – recruiting in Austria, Canada, France, Germany, Israel, Italy, 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
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, Spain, the United Kingdom, the United States
Extension Study of Oral PHA-022121 for Acute Treatment of Angioedema Attacks in Patients with HAE – recruiting in Spain, the United States
FIRAZYR General Drug Use-Results Survey (Japan) – recruiting in Japan
Firazyr® Patient Registry (Icatibant Outcome Survey – IOS) – recruiting in Australia, Austria, Brazil, 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
HAE multi-national survey study – recruiting in Argentina, Brazil, Colombia, Croatia, Denmark, Germany, Hungary, Ireland, Norway, Poland, Portugal, Romania, Sweden
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 hereditary angioedema 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, Denmark, France, Germany, Israel, Italy, Netherlands, Spain, Turkey, the United Kingdom, the United States
Pathophysiological study for autoimmune dysregulation of HAE – recruiting in Japan
Pharmacokinetics and Safety of Human Pasteurised C1-Inhibitor Concentrate (Berinert/CE1145) in Subjects with Congenital C1-INH Deficiency – recruiting in Italy
Phase 1/2 Study to Evaluate Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of NTLA-2002 in Adults with HAE – recruiting in Denmark, France, the Netherlands
PK Subtrial in Adolescent Patients with HAE Type I or II Participating in the KVD900-302 Trial – recruiting in France, the United States
Safety, Tolerability, PK, PD of ADX-324 in Healthy Volunteers and HAE Patients – recruiting in Australia
Status of Dental Care Practices in Patients with HAE – recruiting in France
Stopping androgen treatment in patients with HAE – characterization of reasons and protocols and development of advice for patients and physicians – recruiting in France, Germany, Hungary
The Role of the Coagulation Pathways in Recurrent Angioedema – recruiting in France