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Results for “Niemann-Pick Disease, Type C”

43 trials

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Showing 20 of 43 results

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
Large-scale testing (Phase 3)Ended earlyNCT03879655
What this trial is testing

Open-label Study of VTS-270 in Participants With Neurologic Manifestations of Niemann-Pick Type C1

Who this might be right for
Niemann-Pick Disease, Type C
Mandos LLC 2
Post-approval studies (Phase 4)Study completedNCT03910621
What this trial is testing

Safety and Efficacy of Miglustat in Chinese NPC Patients

Who this might be right for
Niemann-Pick Disease, Type C
Actelion 17
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
Large-scale testing (Phase 3)Active Not RecruitingNCT07082543
What this trial is testing

Evaluate the Safety and Efficacy of Oral Nizubaglustat (AZ-3102) in Late-infantile and Juvenile Forms of GM1 Gangliosidosis or GM2 Gangliosidosis

Who this might be right for
Gangliosidoses, GM2Gangliosidosis, GM1
Azafaros B.V. 75
Testing effectiveness (Phase 2)Study completedNCT03471143
What this trial is testing

Study of IV VTS-270 for Infantile Liver Disease Associated With Niemann-Pick Disease, Type C

Who this might be right for
Niemann-Pick Disease, Type C
Washington University School of Medicine 4
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 B.V. 147
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)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
Very early researchUnknownNCT03201627
What this trial is testing

Study of Lithium Carbonate to Treat Niemann-Pick Type C1 Disease

Who this might be right for
Niemann-Pick Disease, Type C1
Xinhua Hospital, Shanghai Jiao Tong University School of Medicine 18
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)Study completedNCT02534844
What this trial is testing

VTS-270 to Treat Niemann-Pick Type C1 (NPC1) Disease

Who this might be right for
Niemann-Pick Disease, Type C
Mandos LLC 56
Large-scale testing (Phase 3)Looking for participantsNCT07082725
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 (NPC)

Who this might be right for
Niemann-Pick Type C Disease
Azafaros B.V. 72
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
Not applicableActive Not RecruitingNCT03655223
What this trial is testing

Early Check: Expanded Screening in Newborns

Who this might be right for
Spinal Muscular AtrophyFragile X SyndromeFragile X - Premutation+182 more
RTI International 30,000
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
Early research (Phase 1)Study completedNCT00316498
What this trial is testing

Saccadic Eye Movements in Patients With Niemann-Pick Type C Disease

Who this might be right for
Niemann Pick Diseases
National Eye Institute (NEI) 30
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)UnknownNCT04860960
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
Not applicableLooking for participantsNCT00344331
What this trial is testing

Evaluation of Biochemical Markers and Clinical Investigation of Niemann-Pick Disease, Type C

Who this might be right for
Niemann-Pick Disease, Type C
Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD) 900
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