ENABLE
Research type
Research Study
Full title
A three-year, non-interventional, prospective, multicenter study to evaluate the long-term effectiveness of lanadelumab in real-world clinical practice (ENABLE)
IRAS ID
275945
Contact name
Sorena Kiani-Alikhan
Contact email
Sponsor organisation
Takeda Development Center Americas, Inc.
Duration of Study in the UK
4 years, 4 months, 3 days
Research summary
Hereditary angioedema (HAE) is a rare disease caused by a gene mutation. Type 1 and type 2 HAE result in recurrent attacks of severe swelling. The swelling most commonly affects the arms, legs, face, intestinal tract, and airway. The most serious HAE attacks result in laryngeal oedema, which causes obstruction of the upper airways that may lead to death by asphyxiation if not timely and effectively treated. There is a significant burden associated with HAE, particularly due to the unpredictability of disabling HAE attacks.
Current therapy for HAE consists of treatment of acute attacks, as well as short-term and long-term treatment.
Lanadelumab was shown to significantly reduce the frequency and severity of HAE attacks. It also induced a clinically meaningful improvement in patient-reported quality of life while being generally well-tolerated. Lanadelumab has been approved by the European Commission for the treatment of recurrent attacks in patients with HAE.
Currently no information is available on outcomes and unmet needs in the management of patients treated with lanadelumab in real-world clinical practice. This study aims to evaluate the effectiveness of lanadelumab on the occurrence of HAEattacks for up to three years in patients with type 1 or type 2 HAE who initiate treatment with lanadelumab according to the current product labelling. This study will help in post-marketing monitoring of the long term side effects lanadelumab.
Patients will be enrolled over a period of approximately 18 months and will be followed for up to 36 months. Data from routine patient visits will be captured by study sites. For specific study outcomes, patients will complete diaries and standardised PRO questionnaires using a secure digital application installed on a smartphone around the same time points of the visits at sites (approximately every 6 months) and additionally at month 1, month 2, and month 3.
REC name
North of Scotland Research Ethics Committee 1
REC reference
20/NS/0020
Date of REC Opinion
3 Feb 2020
REC opinion
Favourable Opinion