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GRoningen Early-PD Ambroxol Treatment — GREAT  

Parkinson Disease Clinical Trial

Official title:
GRoningen Early-PD Ambroxol Treatment (GREAT) Trial: A Randomised, Double-blind, Placebo-controlled, Singlecenter Trial With Ambroxol in Parkinson Patients With a GBA Mutation

Clinical Trial 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.

Clinical Trial Description

n/a

Study Design

Related Conditions & MeSH terms

Clinical Trial Details
NCT number NCT05830396
Study type Interventional
Source University Medical Center Groningen
Contact Olav Siemeling
Phone 0031 (0)50 3615639
Email o.siemeling@umcg.nl
Status Recruiting
Phase Phase 2/Phase 3
Start date May 2023
Completion date July 2025
View Details
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