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Trial + Data / Events	Status	Ph	N	Region	Interventions	Sponsor	Conditions	Primary compl	Study compl
Spencer-Manzon, Michele
NCT05222178: Safety and Efficacy of HMI-103 in Participants With Classical PKU Due to PAH Deficiency	Terminated	1	3	US	HMI-103	Homology Medicines, Inc	Phenylketonurias, PAH Deficiency, Phenylketonuria	09/23	09/23
Jasne, Michele
HERO, NCT04732429 / 2023-000785-33: Study of HST5040 in Subjects With Propionic or Methylmalonic Acidemia	Terminated	2	26	US, RoW	HST5040, Placebo	HemoShear Therapeutics, HemoShear Therapeutics	Methylmalonic Acidemia, Propionic Acidemia	10/23	10/23
HYP01, NCT06042257: Guanfacine for Hyperactivity in Children with Down Syndrome (HYPEbeGONE_DS)	Recruiting	2	60	US	Guanfacine Hydrochloride Immediate Release, Placebo	Rachel G. Greenberg, MD, MB, MHS, The Emmes Company, LLC, Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)	Hyperactivity in Children with Down Syndrome, Impulsivity in Children with Down Syndrome	11/25	02/26
NCT05222178: Safety and Efficacy of HMI-103 in Participants With Classical PKU Due to PAH Deficiency	Terminated	1	3	US	HMI-103	Homology Medicines, Inc	Phenylketonurias, PAH Deficiency, Phenylketonuria	09/23	09/23
NCT05238324: Safety and Efficacy of HMI-203 in ERT-Treated Adults With MPS II	Withdrawn	1	9	Canada, US	Genetic HMI-203	Homology Medicines, Inc	Mucopolysaccharidosis II	10/24	01/29
EMPOWER, NCT04598698: The Study: Endometriosis Diagnosis Using MicroRNA	Active, not recruiting	N/A	750	NA		Dot Laboratories, Inc.	Endometriosis	05/23	12/24