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Trial + Data / Events	Status	Ph	N	Region	Interventions	Sponsor	Conditions	Primary compl	Study compl
Hastings, Caroline A
NCT03893071: Open-Label Study of Long-Term Safety and Efficacy of Intravenous Trappsol Cyclo (HPβCD) in Niemann-Pick Disease Type C	Active, not recruiting	1	12	US	Hydroxypropyl-β-cyclodextrin, Hydroxypropyl-β-cyclodextrin (HP-β-CD), Trappsol® Cyclo	Cyclo Therapeutics, Inc.	Niemann-Pick Disease, Type C1	03/22	03/22
OR-ARI-EAP-NPC, NCT04316637: Early Access Program With Arimoclomol in US Patients With NPC	Available	N/A		US	Arimoclomol	ZevraDenmark	Niemann-Pick Disease, Type C
NCT04041102: Natural History Study of Infantile and Juvenile GM1 Gangliosidosis (GM1) Patients	Recruiting	N/A	40	Europe, Canada, US, RoW		University of Pennsylvania, Passage Bio, Inc.	GM1 Gangliosidosis	06/26	12/26
Sato, Mariko
ACTION, NCT05580562: ONC201 in H3 K27M-mutant Diffuse Glioma Following Radiotherapy (the Study) Jan 2026 - Dec 2026: Final data from ACTION trial for newly diagnosed H3 K27M-mutant diffuse glioma Jan 2025 - Mar 2025: Interim data from ACTION trial for newly diagnosed H3 K27M-mutant diffuse glioma	Recruiting	3	450	Europe, Canada, Japan, US, RoW	Dordaviprone (ONC201), Dordaviprone (ONC201) + Placebo, Placebo	Chimerix	H3 K27M, Glioma	08/26	08/26
NCT06413706: A Study Comparing Abemaciclib Plus Temozolomide to Temozolomide Monotherapy in Children and Young Adults With High-grade Glioma Following Radiotherapy	Recruiting	2	45	Europe, Japan, US, RoW	Abemaciclib, LY2835219, Temozolomide	Eli Lilly and Company	Glioma	07/27	02/28
NCT04617002: Intermediate-size Expanded Access to ONC201 for Patients With H3 K27M-mutant and/or Midline Gliomas	Available	N/A		US	ONC201 (dordaviprone)	Chimerix	Glioma, H3 K27M