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Results for “Niemann-Pick Disease, Type C”

43 trials

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Showing 20 of 43 results

Not applicableLooking for participantsNCT04880356
What this trial is testing

Longitudinal Study of Ultra-rare Inherited Metabolic and Degenerative Neurological Diseases.

Who this might be right for
Inherited DiseaseRare DiseasesMetabolic Disease+3 more
Fondazione I.R.C.C.S. Istituto Neurologico Carlo Besta 100
Not applicableAvailableNCT04316637
What this trial is testing

Early Access Program With Arimoclomol in US Patients With NPC

Who this might be right for
Niemann-Pick Disease, Type C
ZevraDenmark
Testing effectiveness (Phase 2)Study completedNCT00975689
What this trial is testing

Biomarker Validation for Niemann-Pick Disease, Type C: Safety and Efficacy of N-Acetyl Cysteine

Who this might be right for
Niemann-Pick Disease, Type C
Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD) 35
Large-scale testing (Phase 3)Study completedNCT02612129
What this trial is testing

Arimoclomol Prospective Study in Participants Diagnosed With Niemann-Pick Disease Type C

Who this might be right for
Niemann-Pick Disease, Type C
ZevraDenmark 50
Testing effectiveness (Phase 2)Study completedNCT03759639
What this trial is testing

N-Acetyl-L-Leucine for Niemann-Pick Disease, Type C (NPC)

Who this might be right for
Niemann-Pick Disease, Type C
IntraBio Inc 33
Not applicableEnrolling By InvitationNCT05368038
What this trial is testing

ScreenPlus: A Comprehensive, Flexible, Multi-disorder Newborn Screening Program

Who this might be right for
Acid Sphingomyelinase DeficiencyCeroid Lipofuscinosis, Neuronal, 2Cerebrotendinous Xanthomatosis+11 more
Albert Einstein College of Medicine 100,000
Testing effectiveness (Phase 2)Study completedNCT02124083
What this trial is testing

Phase 1/2 Study of Vorinostat Therapy in Niemann-Pick Disease, Type C1

Who this might be right for
Neimann-Pick Disease
Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD) 12
Not applicableWithdrawnNCT04189601
What this trial is testing

Complement Activation in the Lysosomal Storage Disorders

Who this might be right for
Fabry DiseaseGaucher DiseaseNiemann-Pick Disease, Type C+1 more
Melbourne Health
Testing effectiveness (Phase 2)Ended earlyNCT00668564
What this trial is testing

Hematopoietic Stem Cell Transplantation (HCT) for Inborn Errors of Metabolism

Who this might be right for
Hurler's SyndromeMaroteaux-Lamy SyndromeSly Syndrome+8 more
Masonic Cancer Center, University of Minnesota 18
Large-scale testing (Phase 3)Looking for participantsNCT07054515
What this trial is testing

Evaluate the Safety and Efficacy of Oral Nizubaglustat (AZ-3102) in Late-infantile and Juvenile Forms of Niemann-Pick Type C Disease, GM1 Gangliosidosis or GM2 Gangliosidosis

Who this might be right for
Niemann-Pick Type C DiseaseGM1 GangliosidosisGM2 Gangliosidosis
Azafaros A.G. 147
Early research (Phase 1)UnknownNCT03893071
What this trial is testing

Open-Label Study of Long-Term Safety and Efficacy of Intravenous Trappsol Cyclo (HPβCD) in Niemann-Pick Disease Type C

Who this might be right for
Niemann-Pick Disease, Type C1
Cyclo Therapeutics, Inc. 12
Testing effectiveness (Phase 2)Ended earlyNCT02193867
What this trial is testing

Clinical Study In Infants With Rapidly Progressive Lysosomal Acid Lipase Deficiency

Who this might be right for
Lysosomal Acid Lipase Deficiency
Alexion Pharmaceuticals, Inc. 10
Large-scale testing (Phase 3)Active Not RecruitingNCT04860960
What this trial is testing

Phase 3 Study to Evaluate Intravenous Trappsol(R) Cyclo(TM) in Pediatric and Adult Patients With Niemann-Pick Disease Type C1

Who this might be right for
Niemann-Pick Disease, Type C1
Cyclo Therapeutics, Inc. 94
Testing effectiveness (Phase 2)Study completedNCT00517153
What this trial is testing

Miglustat in Niemann-Pick Type C Disease

Who this might be right for
Niemann-Pick Type C Disease
Actelion 29
Early research (Phase 1)Study completedNCT01747135
What this trial is testing

Hydroxypropyl Beta Cyclodextrin for Niemann-Pick Type C1 Disease

Who this might be right for
Niemann-Pick Disease, Type C1
Vtesse, LLC, a Mallinckrodt Pharmaceuticals Company 14
Testing effectiveness (Phase 2)Study completedNCT02912793
What this trial is testing

Safety and Efficacy of Intravenous Trappsol Cyclo (HPBCD) in Niemann-Pick Type C Patients

Who this might be right for
Niemann-Pick Disease, Type C1
Cyclo Therapeutics, Inc. 12
Not applicableWithdrawnNCT01306604
What this trial is testing

Biomarker for Niemann Pick Type C Disease (BioNPC)

Who this might be right for
Niemann-Pick DiseaseNiemann-Pick Disease, Type C
CENTOGENE GmbH Rostock
Large-scale testing (Phase 3)Study completedNCT01371825
What this trial is testing

Safety, Tolerability, Efficacy, Pharmacokinetics, and Pharmacodynamics of Sebelipase Alfa in Children With Growth Failure Due to Lysosomal Acid Lipase Deficiency

Who this might be right for
Lysosomal Acid Lipase DeficiencyWolman Disease
Alexion Pharmaceuticals, Inc. 9
Early research (Phase 1)Study completedNCT02939547
What this trial is testing

Study of the Pharmacokinetics of Trappsol and Effects on Potential Biomarkers of Niemann-Pick C1 (NPC1)

Who this might be right for
Niemann-Pick Disease, Type C1
Cyclo Therapeutics, Inc. 13
Large-scale testing (Phase 3)Ended earlyNCT04958642
What this trial is testing

Adrabetadex to Treat Niemann-Pick Type C1 (NPC1) Disease

Who this might be right for
Niemann-Pick Disease, Type C
Mandos LLC 66
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