Ambroxol as a Disease-modifying Treatment in GBA-PD
Status:
Recruiting
Trial end date:
2024-06-15
Target enrollment:
Participant gender:
Summary
The present multicenter, randomized, double-blind, placebo-controlled clinical trial will
investigate whether the prolonged administration of high-dose oral Ambroxol over 52 weeks is
safe, tolerable, able to change Glucocerebrosidase enzyme activity and alpha-synuclein levels
in the central nervous system and, ultimately, to reduce the progression of cognitive decline
and motor disability in 60 individuals with Parkinson's disease with mutations of the
glucocerebrosidase gene (GBA1; OMIM 606463).
Participants will undergo clinical, biomarker blood and cerebrospinal fluid analysis,
neuropsychological, neuroimaging assessment throughout the course of the study.
Phase:
Phase 2
Details
Lead Sponsor:
Fondazione I.R.C.C.S. Istituto Neurologico Carlo Besta
Collaborators:
IRCCS National Neurological Institute "C. Mondino" Foundation University of Campania "Luigi Vanvitelli"