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Trial + Data / Events	Status	Ph	N	Region	Interventions	Sponsor	Conditions	Primary compl	Study compl
Crowther, Laura
NCT04899310 / 2020-004980-24: A Study to Assess Safety, Pharmacokinetics, and Pharmacodynamics of mRNA-3705 in Participants With Isolated Methylmalonic Acidemia	Recruiting	1/2	63	Europe, Canada, US, RoW	mRNA-3705, modified mRNA encoding human, methylmalonyl-coenzyme A mutase	ModernaTX, Inc.	Methylmalonic Acidemia	08/26	08/28
PROVIDE, NCT04411654: Phase 1/2 Clinical Trial of PR001 in Infants With Type 2 Gaucher Disease	Active, not recruiting	1/2	15	Europe, US	LY3884961, Methylprednisolone, Sirolimus, Prednisone	Prevail Therapeutics, Eli Lilly and Company	Gaucher Disease, Type 2	05/28	05/28