Safety, Tolerability, PK, PD of ADX-324 in Healthy Volunteers and Hereditary Angioedema Patients...
Hereditary AngioedemaThe first-in-human Phase 1 study will evaluate the safety, tolerability, pharmacokinetics (PK) and pharmacodynamics (PD) of ADX-324 in healthy volunteers (HV) and in patients with Hereditary Angioedema (HAE).
NTLA-2002 in Adults With Hereditary Angioedema (HAE)
Hereditary AngioedemaThis study will be conducted to evaluate the safety, tolerability, activity, pharmacokinetics, and pharmacodynamics of NTLA-2002 in adults with Hereditary Angioedema (HAE).
A Gene Therapy Study of BMN 331 in Subjects With Hereditary Angioedema
Hereditary AngioedemaHAEThis is a Phase 1/2, single-arm, open-label, dose-escalation and dose-expansion study of BMN 331 for the treatment of hereditary angioedema (HAE) due to C1 Esterase Inhibitor (C1-INH) protein deficiency. The study drug BMN 331is identified as AAV5 hSERPING1, an adeno-associated virus (AAV5)-based gene therapy vector that expresses wild-type human C1 Esterase Inhibitor (hC1-INH), under the control of a liver-selective promoter, and is being developed for the treatment of HAE with C1-INH deficiency. The pharmaceutical form of BMN 331 is a solution for intravenous infusion.
Berotralstat Treatment in Children With Hereditary Angioedema
Hereditary AngioedemaPediatricThe purpose of this study is to evaluate the pharmacokinetics (PK) and safety of berotralstat to determine the appropriate weight-based dose for pediatric participants 2 to < 12 years old for prophylactic treatment to prevent attacks of hereditary angioedema (HAE).
A Study of Lanadelumab (SHP643) in Chinese Participants With Hereditary Angioedema (HAE)
Hereditary Angioedema (HAE)The main aim of this study is to evaluate the safety of lanadelumab in Chinese participants with HAE. Participants will be treated with lanadelumab for 26 weeks.
A Study of STAR-0215 in Participants With Hereditary Angioedema
Hereditary AngioedemaThe goal of this clinical trial is to test the drug STAR-0215 in participants with hereditary angioedema (HAE). One group of participants will get 1 dose of STAR-0215, and two other groups will get 2 doses of STAR-0215. Researchers will study the effects of STAR-0215 in participants with HAE as this is the first time that the drug has been given to participants with HAE.
CSL312_3003 Safety and Pharmacokinetic Study in Subjects 2 to 11 Years of Age With Hereditary Angioedema...
Hereditary Angioedema (HAE)The purpose of this study is to investigate the safety, PK / PD, and efficacy of SC CSL312 for prophylactic treatment of pediatric subjects with HAE.
An Open-label Extension Trial to Evaluate the Long-term Safety of KVD900 for On-Demand Treatment...
Hereditary AngioedemaThis is an open-label, multicenter extension trial to evaluate the long-term safety of KVD900 in patients who are 12 years or older with HAE type I or II.
Extension Study of Oral PHA-022121 for Acute Treatment of Angioedema Attacks in Patients With Hereditary...
Hereditary AngioedemaHereditary Angioedema Type I11 moreThis study evaluates the safety and efficacy of long-term on-demand treatment with orally administered deucrictibant for acute hereditary angioedema (HAE) attacks, including laryngeal attacks, in patients with HAE due to C1-esterase inhibitor (C1-INH) deficiency (type I/II). The study will enroll patients from Study PHA022121-C201 (NCT04618211) who elect to participate in this extension study and meet the eligibility requirements.
A Study to Assess the Long-Term Safety and Efficacy of Donidalorsen in the Prophylactic Treatment...
Hereditary AngioedemaThe purpose of this study is to evaluate the long-term safety and efficacy of donidalorsen in people with HAE and the effects of donidalorsen on the number of HAE attacks and their impact on quality of life (QoL).