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Results for “Niemann Pick Diseases”

77 trials

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Showing 20 of 77 results

Not applicableNot Yet RecruitingNCT06258577
What this trial is testing

Screening for Gaucher Disease and Acid Sphingomyelinase Deficiency

Who this might be right for
Gaucher Disease
Chung-Hsing Wang 50
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
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
Not applicableEnded earlyNCT02841358
What this trial is testing

Screening of Niemann-Pick Disease, Type C in a Psychiatric Population

Who this might be right for
Psychiatric Adults Patients
University Hospital, Grenoble 22
Not applicableStudy completedNCT05642221
What this trial is testing

Functional Near-Infrared Spectroscopy (fNIRS) Combined With Diffuse Correlation Spectroscopy (DCS) in Neurocognitive Disease as Compared to Healthy Neurotypical Controls

Who this might be right for
Juvenile Neuronal Ceroid LipofuscinosisSmith-Lemli-Opitz SyndromeCreatine Transporter Deficiency+1 more
Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD) 73
Testing effectiveness (Phase 2)Study completedNCT02004704
What this trial is testing

A Long-Term Study of Olipudase Alfa in Patients With Acid Sphingomyelinase Deficiency

Who this might be right for
Sphingomyelin Lipidosis
Genzyme, a Sanofi Company 25
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
Testing effectiveness (Phase 2)Looking for participantsNCT07399704
What this trial is testing

Evaluate the Safety and Efficacy of Nizubaglustat (AZ-3102) in Patients With GM2 Gangliosidosis or Niemann-Pick Type C Disease

Who this might be right for
GM2 GangliosidosisNiemann-Pick Type C Disease
Azafaros A.G. 21
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
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
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
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 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
Testing effectiveness (Phase 2)Study completedNCT02292654
What this trial is testing

Safety, Tolerability, PK, and Efficacy Evaluation of Repeat Ascending Doses of Olipudase Alfa in Pediatric Patients <18 Years of Age With Acid Sphingomyelinase Deficiency

Who this might be right for
Sphingomyelin Lipidosis
Genzyme, a Sanofi Company 20
Not applicableActive Not RecruitingNCT07274826
What this trial is testing

Diagnostic Creteria of Acid Sphingomyelinase Deficiency (ASMD)

Who this might be right for
Acid Sphingomyelinase Deficiency (ASMD)
Sohag University 7
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
Not applicableApproved For MarketingNCT04877132
What this trial is testing

Compassionate Use Program for Olipudase Alfa Enzyme Replacement Therapy for Patients With Chronic Acid Sphingomyelinase Deficiency (ASMD)

Who this might be right for
Sphingomyelin Lipidosis
Sanofi
Testing effectiveness (Phase 2)Active Not RecruitingNCT02171104
What this trial is testing

MT2013-31: Allo HCT for Metabolic Disorders and Severe Osteopetrosis

Who this might be right for
Mucopolysaccharidosis DisordersHurler SyndromeHunter Syndrome+26 more
Masonic Cancer Center, University of Minnesota 149
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 applicableStudy completedNCT05641103
What this trial is testing

PREDIGA 2: Spanish Acronym of "Educational and Diagnostic Project for Gaucher and ASMD"

Who this might be right for
Gaucher DiseaseAcid SphingoMyelinase DeficiencyASMD+2 more
Fundación Española de Hematología y Hemoterapía 122
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