The Dutch Parkinson GBA Ambroxol Trial (DUPARG-AMBROXOL)
Status:
Recruiting
Trial end date:
2025-07-01
Target enrollment:
Participant gender:
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.