The ANeED Study: Ambroxol in New and Early Dementia with Lewy bodies

Abstract

AbstractBackgroundNo disease modifying treatment is available for dementia with Lewy bodies (DLB). DLB patients have a rapid disease progression to the end stage of severe dementia, nursing home admission and death as compared to persons living with AD (1). Reported findings suggests reduced glucocerebrosidase (GCase) activity may be involved in pathomechanisms of synucleinopathies. Ambroxol is a well‐known drugs able to enhance GCase activity and thus increases synuclein clearance in CNS (2,3).MethodThe ANeED study is a national, multicentre, phase IIa RCT, placebo controlled clinical intervention trial including patients with prodromal and mild DLB with MMSE >14 (NCT04588285). The drug intervention is 1260 mg/d and treatment duration is 18 month. We additionally stratify participants based on genotypes for APOE and the CSF biomarker amyloid‐beta. Allocation ratio 1:1 for ambroxol and placebo.ResultThe study started in May 2021. As of February 2023, six out of seven sites have started recruitment. Of 47 included patients 38 started treatment with compliance >90%. Five patients completed the blind phase after 18 months and continue in the extended open label phase with ABX for 12 months. Adverse events reported are falls, nausea and sleep disturbances, but the relationship with ambroxol is uncertain. The goal for inclusions is 180 participants. Demographical and clinical data we present in table.ConclusionThe ANeED study continue to recruit patients as planned. We did not register any serious adverse events related to the study medication. The compliance is high and the tolerability and safety of study medication is good.