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 end of September 2023).

Read more about these and other clinical trials at:

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

A 3-Part Study to Evaluate the Pharmacokinetics, Safety, and Efficacy of Subcutaneous Ecallantide in Prepubertal Paediatric Patients Experiencing Acute Attacks of Hereditary Angioedema – recruiting in Germany

A Gene Therapy Study of BMN 331 in Subjects with HAE – recruiting in Spain, the United States

A long-term study to evaluate if KVD900 is safe and effective in treating attacks in patients with hereditary angioedema – recruiting in Australia, Austria, Bulgaria, Canada, France, Germany, Greece, Hungary, Israel, Italy, Japan, the Netherlands, New Zealand, Poland, Portugal, Romania, Saudi Arabia, Slovakia, South Africa, Spain, the United Kingdom, United States

A Phase 1/2, Open-Label, Dose-Escalation Study to Determine the Safety, Tolerability, and Efficacy of BMN 331, an Adeno-Associated Virus (AAV) Vector-Mediated Gene Transfer of Human SERPING1, in Subjects with Hereditary Angioedema (HAE) due to Human C1 Esterase Inhibitor (C1-INH) Deficiency – recruiting in Spain

A Randomized, Double-Blind, Placebo-Controlled, Phase 3, Three-way Crossover Trial to Evaluate the Efficacy and Safety of Two Dose Levels of KVD900, an Oral Plasma Kallikrein Inhibitor, for On-Demand Treatment of Angioedema Attacks in Adolescent and Adult Patients with Hereditary Angioedema Type I or II – recruiting in United States, Japan, Australia, Bulgaria, Canada, France, Germany, Greece, Hungary, Israel, Italy, the Netherlands, New Zealand, North Macedonia, Poland, Puerto Rico, Romania, Spain, the United Kingdom, United States

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

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 Hereditary Angioedema (HAE) – recruiting in China

A Study of Lanadelumab in Teenagers and Adults with HAE – 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 Participants with Hereditary Angioedema – recruiting in the United States

A Study to Assess the Long-Term Safety and Efficacy of Donidalorsen in the Prophylactic Treatment of HAE – recruiting in Belgium, Bulgaria, Canada, France, Germany, Israel, Italy, the Netherlands, Poland, Puerto Rico, Spain, Turkey, the United Kingdom, 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 NTLA-2002 in adults with HAE – recruiting in Australia, France, Germany, the Netherlands, New Zealand, the United Kingdom, the United States

A Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of BW-20805 in Healthy Subjects – recruiting in Australia

A Study to Review the Treatment and Outcomes of Teenagers and Adults with Non-histaminergic Angioedema with Normal C1 Inhibitor in Canada – 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 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 Study of ISIS 721744 in Patients with Hereditary Angioedema – recruiting in the Netherlands

An Open-label Extension Trial to Evaluate the Long-term Safety of KVD900, an Oral Plasma Kallikrein Inhibitor, for On-demand Treatment of Angioedema Attacks in Adolescent and Adult Patients with Hereditary Angioedema Type I or II – recruiting in Australia, Austria, Spain, Bulgaria, France, Germany, Greece, Hungary, Israel, Italy, Japan, the Netherlands, North Macedonia, Poland, Italy, Germany, Portugal, Slovakia, South Africa, Spain, the United Kingdom, 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 – recruiting in France, the Netherlands, the United States, Austria, Bulgaria, Germany, Greece, Hungary, Israel, Italy, Japan, North Macedonia, Poland, Slovakia, South Africa, Spain, United Kingdom

An Open-label Study to Evaluate the Long-term Safety and Efficacy of CSL312 (Garadacimab) in the Prophylactic Treatment of Hereditary Angioedema – recruiting in Germany, Czechia, Hungary, the Netherlands, Italy

An open-label study to provide berotralstat access to subjects with type 1 and 2 hereditary angioedema who were previously enrolled in berotralstat studies – recruiting in France, Czechia, Slovakia, Poland

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

Armenian NAtionwide REGistry of Systemic Autoimmune and Autoinflammatory Disease – recruiting in Armenia

Berotralstat Treatment in Children with HAE – recruiting in Austria, Canada, France, Germany, Israel, Italy, Poland, Romania, Spain, the United Kingdom

C1 Inhibitor Registry in the Treatment of Hereditary Angioedema (HAE) Attacks – recruiting in Bulgaria, Croatia, Czechia, France, Germany, Hungary, Italy, North Macedonia, Norway, Poland, Slovakia, Slovenia, Sweden, Bulgaria, Croatia, Czechia, France, Germany, Hungary, Italy, North Macedonia, Norway, Poland, Slovakia, Slovenia, Sweden, the Netherlands

Characterization of Rhythmicity Profiles of Bradykinin-mediated Angioedema Attacks Using a Tracking Smartphone Application – not yet recruiting

CLOUD-R HAE REGISTRY – recruiting in France

CSL312_3003 Safety and Pharmacokinetic Study in Subjects 2 to 11 Years of Age with Hereditary Angioedema – recruiting in Israel

Deployment to the Multidisciplinary Prospective Cohort (IMMINeNT) – recruiting in France

Digital Biomarkers Study in Patients with Hereditary Angioedema in Japan: Exploratory Longitudinal Study about Trigger Factors of HAE Attacks – recruiting in Japan

Extension Study of Oral PHA-022121 for Acute Treatment of Angioedema Attacks in Patients with HAE – recruiting in Bulgaria, Canada, Czechia, France, Germany, Hungary, Israel, Poland, 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

Identification and Characterization of Genetic Variants in Hereditary Angioedema – recruiting in Spain

Involvement of Monocytic B1 and B2 Receptors in Inflammation and Chronic Vascular Disease in Patients with Hereditary Bradykinetic Angioedema – recruiting in France

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 and Frequent Hereditary Angioedema (HAE) Attacks – recruiting in Italy

Phase 1/2 Study to Evaluate Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of NTLA-2002 in Adults with HAE – recruiting in France, Germany, the Netherlands, New Zealand, the United Kingdom

PK Subtrial in Adolescent Patients with HAE Type I or II Participating in the KVD900-302 Trial – recruiting in Australia, Bulgaria, France, Germany, Greece, Israel, Japan, Netherlands, Slovakia, Spain, the United Kingdom, 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 Brazil, China, Germany, France, Hungary, India, South Korea, Oman, Peru, Portugal, Russia, Thailand, South Africa

The Role of the Coagulation Pathways in Recurrent Angioedema – recruiting in France

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