GRoningen Early-PD Ambroxol Treatment

Summary

The most common genetic risk factor for Parkinson's Disease is a heterozygous mutation of the GBA1 gene, encoding the lysosomal enzyme glucocerebrosidase (GCase). Reduced GCase activity is associated with aggregation of the protein alpha synucleine (aSyn) in the central nervous system, which is related to the pathological cause of PD. Ambroxol is a mucolytic expectorant that appears to facilitate the refolding of the misfolded GBA protein thats acts as a chaperone for GCase. This randomized placebo-controlled trial aims to investigate the disease-modifying properties of ambroxol in PD patients with a GBA1-mutation. Patients will undergo motor and cognitive tests, as well as imaging and blood tests.

Timeline

Start

2023-05

Primary completion

2025-07

Completion

2025-07

Publications

• Moes HR, Buskens E, van Laar T. Continuous subcutaneous levodopa-carbidopa infusion for Parkinson's disease. Lancet Neurol. 2024 Sep;23(9):856-857. doi: 10.1016/S1474-4422(24)00272-2. No abstract available.
• Siemeling O, Slingerland S, van der Zee S, van Laar T. Study protocol of the GRoningen early-PD Ambroxol treatment (GREAT) trial: a randomized, double-blind, placebo-controlled, single center trial with ambroxol in Parkinson patients with a GBA mutation. BMC Neurol. 2024 May 1;24(1):146. doi: 10.1186/s12883-024-03629-9.

Drugs

Indications