. 2020 Jan 23;267(3):860–869. doi: 10.1007/s00415-020-09705-7

Table 2.

GBA-targeting treatments for PD in the clinical phase aiming at modulation of glycosphingolipid turnover and restoration of enzyme function

GBA	MOVES-PD study Part 1	MOVES-PD study Part 2	AiM-PD
Compound	Venglustat (GZ/SAR402671)		Ambroxol	RTB101	LTI-291	PR001
Administration	Oral		Oral	Oral	Oral	Injections
Sponsor	Sanofi		UCL and Cure PD Trust	Restorbio	LTI/Allergan	Prevail
RCT No	NCT02906020		NCT02941822		(NL7061; NTR7299)^a
Mechanism	Glucosylceramide synthase inhibiton; reduction of GBA-related GSLs		GCase activation	TORC1 inhibition	GCase activation	Gene therapy, AAV-based
Status	Completed	Recruiting, estimated primary completion 2021	Estimated completion 04-2018	Ongoing; data expected 2020	Recruiting in Leiden (NL)	Clinical centers initiated
Phase	2		2a	1b/2a	1b	1b
Design	Multicenter, randomized, double-blind, placebo–controlled, sequential cohort	Prospective, single-centre, open label	Multicenter 2:1 randomized double-blind, placebo-controlled	Multicenter, 2:1 randomized, double-blind, placebo-controlled	Randomized, placebo-controlled, double-blind, parallel study	Randomized, double-blind, sham procedure-controlled, ascending dose study
Total N of part	17		10 + 10	45	Apprx. 40	30/16
GBA-PD	$\sqrt$		$\sqrt$	$\sqrt$	$\sqrt$	$\sqrt$
Idiopathic PD	No		$\sqrt$	$\sqrt$	No	$\sqrt$
Age	18–80 yrs (mean 58 yrs)		40–80 yrs		18 years or older
Duration	36 weeks	52 weeks + 104 weeks extension	6 months	4 weeks	28 days
Doses tested	3 escalating doses	1 dose	5 escalating doses	300 mg; ± sirolimus	10 or 60 mg once daily	Two escalating dose cohorts