Study of Ambroxol Treatment for Early and Prodromal Dementia With Lewy Bodies Evaluating Cognitive, Functional, and Neuropsychiatric Effects in Patients Aged 50 to 85

What this Trial Is About

Researchers are evaluating the effects of ambroxol, a drug that enhances Glucocerebrosidase GCase, on cognition, functional decline, and neuropsychiatric symptoms in people diagnosed with prodromal and early dementia with Lewy bodies DLB. This phase IIa study aims to confirm how ambroxol influences these areas compared to a placebo, with attention to disease progression and biomarkers related to DLB. Participants will be randomly assigned to take either oral ambroxol or a matching placebo. The medication dose escalates over the first month, starting at 60 mg three times daily and increasing to 420 mg three times daily, continuing up to 550 days. The study includes a blinded phase lasting 18 months, followed by an open extension offering ambroxol to all participants for an additional year. Throughout the study, participants will attend eight hospital visits and receive sixteen telephone calls to monitor safety, side effects, and treatment adherence. Various assessments will be conducted, including cognitive tests, neuropsychiatric evaluations, blood tests, MRI, DaTSCAN imaging, ECG, EEG, and lumbar punctures. The primary outcomes focus on cognitive changes, global function, disease stage, and neuropsychiatric symptoms, with secondary and exploratory outcomes addressing sleep, motor symptoms, falls, and biomarker impacts.

Ambroxol in New and Early DLB, A Phase IIa Multicentre Randomized Controlled Double Blind Clinical Trial