Phase 3 Trial Testing Ambroxol Tablets to Slow Parkinsons Disease Progression in Adults Aged 35 to 75

What this Trial Is About

Researchers are evaluating the drug ambroxol hydrochloride in patients with Parkinsons disease PD to see if it can slow disease progression and to assess its safety and tolerability. This trial is a Phase IIIa study conducted in the UK, involving patients diagnosed with PD within the past 7 years. Ambroxol hydrochloride is already licensed for respiratory conditions, and this study aims to explore its potential benefits in PD treatment. Participants will be randomly assigned to receive either ambroxol hydrochloride or a matching placebo in a double-blind manner for 104 weeks. The dosing starts at 420mg daily for the first 5 days, increases to 840mg daily for days 6-10, and then 1260mg daily for the remainder of the blinded treatment period. After this, all participants enter a 26-week open-label extension phase, during which they will receive ambroxol hydrochloride at the same dosing schedule. An optional sub-study involves lumbar punctures at two time points to measure drug levels and related biomarkers in the cerebrospinal fluid. Throughout the study, participants will attend regular visits for assessments including the Movement Disorder Society Unified Parkinsons Disease Rating Scale MDS-UPDRS and cognitive tests. Safety will be monitored through vital signs and blood tests at multiple time points. The primary measure is the change in MDS-UPDRS score from baseline to week 104. Secondary outcomes include motor and non-motor symptoms, quality of life, and symptom severity. The total participation lasts approximately 130 weeks, including the extension phase, with detailed monitoring and data collection.

Ambroxol to Slow Progression in Parkinson Disease