A Study of Lanadelumab in Teenagers and Adults With Hereditary Angioedema (HAE)
Hereditary Angioedema (HAE)The main aim of this study is to compare the HAE attack rate requiring on-demand treatment before and within 2 years after participants with HAE have been treated with lanadelumab. This study is conducted in the United Kingdom where participants were treated or about to be treated with landelumab according to their routine practice at hospitals. Data will be directly collected from participants via study diaries, questionnaires, their medical records, and study doctors treating them. Participants will be contacted every 3 months during study participation (via phone).
Global Registry to Gather Data on Natural History of Patients With Hereditary Angioedema Type I...
Hereditary Angioedema Type I and IIThe objective of this international hereditary angioedema (HAE) register is to collect homogeneous clinical and laboratory data on patients with HAE type I and II gathering better information on the natural course of the disease and detecting therapeutic options to manage it.
Firazyr® Patient Registry (Icatibant Outcome Survey - IOS)
Hereditary Angioedema (HAE)The Icatibant Outcome Survey (IOS) is a prospective, observational disease registry designed to document the routine clinical outcomes over time in participants with angioedema treated with Firazyr® (icatibant) and/or Cinryze® (C1 inhibitor [human]) in countries where it is currently approved. The data collected will be used to evaluate the safety of Firazyr (icatibant) and Cinryze (C1 inhibitor [human]) in routine clinical practice and as a data source for post-marketing investigations.
Lanadelumab in Bradykinin Angioedema
AngioedemaA multicenter observational study aiming to evaluate the efficacy of kallikrein inhibition by lanadelumab in patients with bradykinin- angioedema
Status of Dental Care Practices in Patients With Hereditary Angioedema
Angio Edema HereditaryHereditary angioedema (HAE) is a rare genetic disease, it can occur at any age and evolves in flare-ups. These are subcutaneous or submucosal edemas responsible for tightness and pain. All areas can be reached. In addition, all their life, patients have an unpredictable risk of death from asphyxiating angioedema (25% mortality in the absence of specific treatment). Surgical procedures, endoscopies and dental procedures can trigger laryngeal AE. Thus, the national reference center for angioedema (CREAK) and the latest international consensus1 recommend that all these procedures be preceded by short-term prophylaxis. The reference treatment is the C1 inhibitor 2 concentrate. But to date, there is no prospective study that has demonstrated the effectiveness of this short-term prophylaxis before dental treatment. Only a retrospective study has shown that its implementation made it possible to reduce the risk of crisis by 42% after tooth extraction 3 and that the risk of crisis was greatest within 8 hours following tooth extraction. The C1 inhibitor concentrate must be administered IVL by a nurse within 6 hours before the procedure and therefore implies, in addition to its high cost (1500 €), an organizational burden for the patient who must plan for the passage of an IDE, the availability of the product and this sometimes for a simple dental treatment. These constraints have two consequences for dental care: patients avoid going to the dentist to the detriment of their dental health or do not take prophylaxis with the risk of a fatal attack.
A Survey of Icatibant in Pediatric Participants With Hereditary Angioedema
Hereditary Angioedema (HAE)This study is a survey in Japan of Icatibant subcutaneous injection 30 mg syringe used to treat children or teenagers with acute attacks of hereditary angioedema (HAE). The study sponsor will not be involved in how the participants are treated but will provide instructions on how the clinics will record what happens during the study. The main aim of the study is to check for side effects related from Icatibant subcutaneous injection 30 mg syringe and to check if Icatibant subcutaneous injection 30 mg syringe improves acute attacks of HAE. During the study, pediatric participants with HAE will take Icatibant subcutaneous injection 30mg syringe according to their clinic's standard practice. The study doctors will check for side effects from Icatibant subcutaneous injection 30 mg syringe for 3 months.
A Survey of Lanadelumab in Participants With Hereditary Angioedema
Hereditary Angioedema (HAE)This study is a survey in Japan of Lanadelumab used to treat people with hereditary angioedema (HAE). The study sponsor will not be involved in how the participants are treated but will provide instructions on how the clinics will record what happens during the study. The main aim of the study is to check for side effects related from Lanadelumab and to check if Lanadelumab improves symptoms of HAE. During the study, participants with HAE will take Lanadelumab subcutaneous injection according to their clinic's standard practice. The study doctors will check for side effects from Lanadelumab for 12 months.
Open-label Berotralstat Access to HAE Patients Previously Enrolled in Berotralstat Studies
Hereditary AngioedemaHAEThis is a phase 3b open-label study providing access to berotralstat for HAE patients who were previously enrolled in berotralstat studies.
Dose-ranging Study of Oral PHA-022121 for Prophylaxis Against Angioedema Attacks in Patients With...
Hereditary AngioedemaHereditary Angioedema Type I8 moreThis study evaluates the safety and efficacy of PHA-022121 administered orally for prophylaxis against angioedema attacks in patients with hereditary angioedema (HAE). The study consists of 2 parts, with patients completing participation in Part 1 prior to initiation of treatment in Part 2. Part 1 of the study has 3 parallel arms and approximately 30 patients will be equally randomized to one of two dose regimens of PHA-022121 or matching placebo. Patients will continue to the single open-label arm in Part 2 of the study after completion of Part 1. The screening period is up to 8 weeks and the treatment periods are 12 weeks (Part 1) and 30 months (Part 2) in duration.
Long-term Safety and Efficacy of CSL312 (Garadacimab) in the Prophylactic Treatment of Hereditary...
Hereditary AngioedemaThis phase 3b study will evaluate long-term safety and efficacy of CSL312 (also known as garadacimab) when administered subcutaneously (SC)