Niemann-Pick Disease Type C1 - 25 Studies Found
Completed |
: Biomarker Validation for Niemann-Pick Disease, Type C: Safety and Efficacy of N-Acetyl Cysteine : Niemann-Pick Disease, Type C : 2009-09-10 : Drug: N-Acetyl Cysteine 900mg effervescent tablet; Dosed as 15 mg/kg/day (maximum dose 900 mg per day) f |
Completed |
: Longitudinal Study of Cognition With Niemann-Pick Disease, Type C : Niemann-Pick Disease, Type C : 2013-07-04 |
Completed |
: Hydroxypropyl Beta Cyclodextrin for Niemann-Pick Type C1 Disease : Niemann-Pick Disease, Type C1 : 2012-12-08 : Drug: VTS-270 Other Name: 2-hydroxypropyl-beta-cyclodextrin |
Terminated |
: Screening of Niemann-Pick Disease, Type C in a Psychiatric Population : Psychiatric Adults Patients : 2016-07-11 :
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Completed |
: Phase 1/2 Study of Vorinostat Therapy in Niemann-Pick Disease, Type C1 : Neimann-Pick Disease : 2014-04-25 : Drug: Vorinostat Histone deactylase inhibitor |
Active, not recruiting |
: Arimoclomol Prospective Study in Patients Diagnosed With NiemannPick Disease Type C : Niemann-Pick Disease, Type C : 2015-11-12 :
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Recruiting |
: Study of VTS-270 (2-hydroxypropyl-β-cyclodextrin) to Treat Niemann-Pick Type C1 (NPC1) Disease : Niemann-Pick Disease, Type C : 2015-08-18 :
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Completed |
: A Prospective Non-therapeutic Study in Patients Diagnosed With Niemann-Pick Disease Type C : Niemann-Pick Disease, Type C : 2015-04-22 |
Recruiting |
: Study of the Pharmacokinetics of Trappsol and Effects on Potential Biomarkers of Niemann-Pick C1 (NPC1) : Niemann-Pick Disease, Type C1 : 2016-09-21 : Drug: Hydroxypropyl-beta-cyclodextrin Used in the treatment of Niemann-Pick Disease C1 ( NPC1) |
Recruiting |
: Study of Pharmacokinetics and Preliminary Efficacy in Patients With Niemann-Pick C1 : Niemann-Pick Disease, Type C1 : 2016-08-19 : Drug: Hydroxypropyl-beta-cyclodextrin Used in the treatment of Niemann-Pick C1 |