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Results for “Sphingomyelin Lipidosis”

76 trials

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Showing 20 of 76 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
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
Not applicableLooking for participantsNCT06192576
What this trial is testing

A Real-world Long-term Safety and Immunogenicity Study of Olipudase Alfa Therapy in Pediatric Patients Less Than 2 Years of Age With Acid Sphingomyelinase Deficiency (ASMD)

Who this might be right for
Niemann-Pick DiseasesAcid Sphingomyelinase Deficiency
Sanofi 10
Not applicableLooking for participantsNCT05992532
What this trial is testing

GammaGA: Prevalence of Acid Sphingomyelinase Deficiency Disease (ASMD) and Gaucher Disease in Patients With Monoclonal Gammopathies and/or Multiple Myeloma

Who this might be right for
Gaucher DiseaseAcid SphingoMyelinase Deficiency
Fundación Española de Hematología y Hemoterapía 210
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 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
Not applicableNot Yet RecruitingNCT06869499
What this trial is testing

Study of the Prevalence of Acid Sphingomyelinase Deficiency/Niemann Pick AB and B Disease in Patients With Diffuse Interstitial Lung Disease

Who this might be right for
SplenomegalySplenectomyThrombopenia+2 more
Wladimir MAUHIN, Dr 200
Testing effectiveness (Phase 2)Study completedNCT06949358
What this trial is testing

Evaluate Safety and Tolerability of Olipudase Alfa in Pediatric and Adult Participants With Acid Sphingomyelinase Deficiency (ASMD) Who Completed the DFI12712 or the LTS13632 Study in France

Who this might be right for
Acid Sphingomyelinase Deficiency
Sanofi 3
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
Large-scale testing (Phase 3)Study completedNCT00176904
What this trial is testing

Stem Cell Transplant for Inborn Errors of Metabolism

Who this might be right for
AdrenoleukodystrophyMetachromatic LeukodystrophyGloboid Cell Leukodystrophy+8 more
Masonic Cancer Center, University of Minnesota 135
Not applicableLooking for participantsNCT07545473
What this trial is testing

Retinal Hyperspectral Imaging in Neurodegenerative Diseases

Who this might be right for
DementiaNeurodegenerative DiseasesAlzheimer Disease+5 more
Center for Eye Research Australia 930
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
Not applicableStudy completedNCT04106544
What this trial is testing

A Prospective and Retrospective Cohort Study in Patients With Chronic Forms of Acid Sphingomyelinase Deficiency (ASMD)

Who this might be right for
Sphingomyelin Lipidosis
Sanofi 84
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
Early research (Phase 1)Looking for participantsNCT02254863
What this trial is testing

UCB Transplant of Inherited Metabolic Diseases With Administration of Intrathecal UCB Derived Oligodendrocyte-Like Cells

Who this might be right for
AdrenoleukodystrophyBatten DiseaseMucopolysaccharidosis II+9 more
Joanne Kurtzberg, MD 40
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