Smavollan

Researchers are investigating the effects of ambroxol, a Glucocerebrosidase (GCase) enhancing chaperone, on cognition, functional decline, and neuropsychiatric symptoms in individuals diagnosed with prodromal and early dementia with Lewy bodies (DLB). This Phase IIa clinical trial aims to confirm the impact of ambroxol on these outcomes compared to placebo, focusing on patients with mild cognitive impairment or early dementia stages of DLB. Participants will be randomly assigned to receive either oral ambroxol or a matching placebo. The dosing starts at 60 mg three times daily for the first week and gradually increases through five intra-participant dose escalations up to 420 mg three times daily, continuing for up to 18 months during the blinded phase. After completing the blinded phase, all participants will be offered an open-label extension of ambroxol treatment for an additional 12 months. The study includes regular hospital visits and telephone contacts to monitor treatment effects and safety. During the study, participants will undergo clinical and laboratory assessments, including cognitive tests, blood analyses, electrocardiograms, lumbar punctures, MRI, DaTSCAN, and EEG scans. The research team will monitor adverse events and treatment tolerability throughout 18 months with scheduled visits at screening, baseline, weeks 4, 8, 24, 36, 52, and months 15 and 18, along with frequent phone calls to record any side effects. Primary outcomes include changes in cognition, global function, disease progression, and neuropsychiatric symptoms. Secondary and exploratory outcomes will assess sleep disturbances, falls, parkinsonism, and potential biomarkers related to the drug effects.