P 81 ELEGANCE – A prospective non-interventional study of the long-term effectiveness and safety of levodopa–entacapone–carbidopa intestinal gel (LECIG) in patients with advanced Parkinson's disease in routine care

Abstract

Background: Levodopa–entacapone–carbidopa intestinal gel (LECIG) is a device-aided therapy option introduced in 2021 that has received marketing authorisation in several European countries for the treatment of patients with advanced Parkinson’s disease (PD) who have motor complications that cannot be adequately controlled by optimised oral/transdermal PD medication. The presence of entacapone in LECIG increases the bioavailability of levodopa compared with standard levodopa–carbidopa intestinal gel, allowing a reduced levodopa dose (by 20–35%) to be given to achieve the same effective plasma levodopa levels. The ELEGANCE study has been established to collect real-world data on the efficacy and safety of LECIG in clinical practice use. Study design: ELEGANCE is an international, prospective, non-interventional, observational study that will be undertaken in ∼16 countries, including Germany and Austria, once marketing authorisation for LECIG is received. Study centres will offer participation in the ELEGANCE study to all adult patients with advanced PD who have severe motor fluctuations and hyperkinesia or dyskinesia despite taking optimised or transdermal PD therapy and who have been prescribed treatment with LECIG as part of routine clinical practice. Subjects can be de novo patients or those who switch from another infusion therapy. The planned total number of patients to be recruited to the study is ∼300 across all countries. In Germany and Austria, it is planned that around 15 centres will participate with the aim of recruiting ∼75 patients. Patients will be followed for 24 months or until study discontinuation. Data collection commenced July 2021, and recruitment is planned until in July 2022 with study completion in Q2 2024. An interim analysis will be performed. Study endpoints: Primary objectives are to evaluate the long-term efficacy and safety of LECIG over 24 months. Efficacy evaluations will include effect on motor symptoms (change from baseline to 24 months in daily OFF time [MDS-UPDRS IV] and motor aspects of experiences of daily living [MDS-UPDRS II] scores), required levodopa dose, use of other PD medication, Clinical and Patient Global Impression, and satisfaction with treatment. Adverse events will be monitored for evaluation of safety. Secondary objectives are to investigate patients‘ non-motor symptoms (change from baseline in Non-Motor Symptom Scale Score, Parkinson's disease sleep scale-2, and MDS-UPDRS lb scores), quality of life (change from baseline in Parkinson's Disease Questionnaire total score [PDQ-8 or PDQ-39]), and healthcare resource utilisation (complications of PD leading to hospitalisation, problems with medication or device). Conclusions: The data generated from ELEGANCE will provide valuable real-world information on the efficacy and safety of LECIG, as well as on patient perspectives of this treatment and its impact on healthcare utilisation that can help inform clinical decisions and policy making.