This clinical trial is focused on studying Parkinson’s disease, a condition that affects movement and can cause tremors, stiffness, and difficulty with balance and coordination. The trial is investigating the effects of a medication called ambroxol, which is being tested for its potential to modify the progression of the disease in patients who have a specific genetic mutation known as the GBA1-mutation. The purpose of the study is to explore whether ambroxol can have a positive impact on the disease’s progression in these patients.

Participants in the study will be given either ambroxol or a placebo, which is a substance with no active medication. The study will last for a period of 60 weeks, during which participants will take the medication orally. Throughout the study, various assessments will be conducted to monitor the effects of the treatment. These assessments include evaluating motor skills, which are movements controlled by the brain, and checking for any side effects or adverse events that may occur.

Additional tests will be performed to gather more information about the participants’ health and the impact of the treatment. These tests include MRI scans, which use magnetic fields to create detailed images of the brain, and F-DOPA PET scans, which help visualize brain activity. Cognitive assessments, such as the Montreal Cognitive Assessment (MoCA), will also be conducted to evaluate memory and thinking skills. The study aims to provide valuable insights into the potential benefits of ambroxol for individuals with Parkinson’s disease and the GBA1-mutation.