A UK real-world study of lanadelumab in patients with HAE
Research type
Research Study
Full title
Demographic and clinical characteristics, treatment patterns, and real-world effectiveness of lanadelumab in patients with hereditary angioedema type I or II in the United Kingdom: an ambispective cohort study
IRAS ID
309513
Contact name
Sorena Kiani-Alikhan
Contact email
Sponsor organisation
Takeda UK Ltd
Duration of Study in the UK
3 years, 3 months, 1 days
Research summary
Hereditary angioedema is a disorder characterized by recurrent episodes of severe swelling underneath the skin. The swelling episodes are debilitating and unpredictable and when left untreated can worsen over 12-36 hours. Lanadelumab is a first in class fully human monoclonal antibody therapy which has demonstrated significant benefit in the prevention of HAE attacks clinical trials. This observed benefit was sustained with long-term treatment.The therapy is approved in several regions including Australia, Canada, USA, European Union, UK and Switzerland.
The purpose of this non-interventional study is to evaluate how effective lanadelumab is for treating patients with hereditary angioedema (HAE). There is currently a lack of UK research studies investigating the effectiveness of lanadelumab use in real-world clinical practice.
The primary objective of this study is to estimate and compare the incidence of HAE attacks requiring on-demand treatment before and after initiation of lanadelumab. Patient reported attack diaries and patient-reported outcomes (PROs) as well as physician assessments at standard of care visits will be used to describe HAE attack rates (frequency and severity), treatment patterns, healthcare utilization and participant health related quality of life.
Approximately 50 patients will be recruited across 4-12 selected NHS secondary/tertiary care sites and followed-up for up to 24 months after lanadelumab initiation. These patients will be above 12 years and have a confirmed diagnosis of HAE type I or II.
REC name
Wales REC 5
REC reference
22/WA/0241
Date of REC Opinion
4 Aug 2022
REC opinion
Favourable Opinion