Clinical Research Directory

Extension Study of Oral PHA-022121 for Acute Treatment of Angioedema Attacks in Patients With Hereditary Angioedema

This 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. The study will enroll participants from Study PHA022121-C201 (NCT04618211), Study PHA022121-C306 (NCT06343779) and deucrictibant treatment naïve HAE-nC1INH adult participants who elect to participate in this extension study and meet the eligibility requirements.

Real-life Ecological Momentary Assessment of Lived Burden in Hereditary AngioEdema

The aim of this study is to conduct an in-depth analysis of the Burden of Disease (BoD) perceived by patients with Hereditary Angioedema (HAE), through daily prospective observations based on Ecological Momentary Assessment (EMA) via digital surveys and standardised questionnaires. Participants will answer online survey questions about their perceived burden of disease for 8 consecutive weeks. The main hypothesis is that daily prospective observation (EMA) will reveal a higher and more fluctuating burden of disease compared to traditional retrospective scales, providing a more accurate representation of the impact of HAE on patients' daily lives.

Identification and Characterization of Genetic Variants in Hereditary Angioedema

This project aims to analyse in an unbiased way the existence of genetic variants that contribute to explaining and predicting the differences in clinical expression between patients with HAE.