Table 1.

Disease-modifying therapies in active GBA-PD trials.

Targeting mechanism	Mechanism of action	Drug	Target population (n. of cases)	Status	Primary outcomes	Enrolling countries	ClinicalTrials. gov/Netherlands Trial Register ID	Sponsor
Increase in GCase activity	GCase activation	Ambroxol	GBA-PD (n.10) PD (n.10)	Phase II, Not placebo controlled	Safety, Tolerability PK/PD	UK	NCT02941822	UCL and Cure Parkinson’s Trust
Increase in GCase activity	GCase activation	Ambroxol	PD-D (n.75)	Phase II, Placebo controlled	Safety, Tolerability PK/PD	Canada	NCT02914366	Lawson Health Research Institute and Weston Foundation
Increase in GCase activity	GCase activation	LTI-291	GBA-PD (n.15) PD (n.15)	Phase I, Placebo controlled	Safety, Tolerability PK/PD	Netherlands	NL7061	LTI, Allergan
Reduction of GCase-related glycosphingolipids	Glucosylcer-amide synthase inhibitor	Venglustat	GBA-PD (n.270)	Phase II, Placebo controlled	Change in MDS-UPDRS parts II & III	Austria Canada, France Germany Israel Italy, Japan Norway Portugal Singapore Spain Sweden Taiwan UK USA	NCT02906020	Sanofi-Genzyme

PD, Parkinson Disease; PDD, PD dementia; GBA-PD, GBA1-associated PD; PK/PD, Pharmacokinetics, and Pharmacodynamics; MDS-UPDRS, Movement Disorder Society-Unified Parkinson’s Disease Rating Scale.