Active	Status	Sponsor type	Region	Phase	Filter
9 Active 2 Inactive 11 All	Not yet recruiting Recruiting Enrolling by invitation Active, not recruiting Available No longer recruiting	Industry lead sponsor Industry collaborator Coop Other	US Canada Japan Europe RoW	N/A 1 2 3 4	Highlight changes:- Show only changed items Enrollment:

Advanced Filters

5 Trials

Active Trials   Industry lead sponsorIndustry collaborator

 

Export

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
Dean, Joy
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
Cheung, Veronica
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