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Trial + Data / Events	Status	Ph	N	Region	Interventions	Sponsor	Conditions	Primary compl	Study compl
RIZZO, WILLIAM
NCT03720990: Smith-Lemli-Opitz Syndrome and Cholic Acid	Completed	1/2	12	US	Cholic Acid, Cholbam	University of Nebraska, University of Colorado, Denver, Children's Hospital Medical Center, Cincinnati, University of Pittsburgh	Smith-Lemli-Opitz Syndrome	08/22	09/23
NCT05443685: ADX-629 Therapy for Sjogren-Larsson Syndrome	Recruiting	1/2	10	US	ADX-629, Open label	University of Nebraska	Sjogren-Larsson Syndrome	06/25	06/25
NCT05781399: First-in-Human, Multiple Part Clinical Study of JNT-517 in Healthy Participants and in Participants With Phenylketonuria	Recruiting	1	112	US, RoW	JNT-517 Suspension, Placebo Suspension, JNT-517 Tablet, Placebo Tablet	Jnana Therapeutics	Phenylketonuria	12/24	03/25
NCT05795361: Post-trial Access Program of Idursulfase-IT Along With Elaprase in Children With Hunter Syndrome	Available	N/A		Europe, US, RoW	Idursulfase-IT, HGT-2310, TAK-609	Takeda	Hunter Syndrome
NCT01971957: Sjogren-Larsson Syndrome: Natural History, Clinical Variation and Evaluation of Biochemical Markers	Completed	N/A	20	US		University of Nebraska	Sjogren-Larsson Syndrome (SLS)	08/23	09/23