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Results for “Sphingomyelin Lipidosis”

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Showing 20 of 0 results

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)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
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 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
Not applicableStudy completedNCT00001972
What this trial is testing

PET Scan of Brain Metabolism in Relation to Age and Disease

Who this might be right for
Alzheimer's DiseaseBrain NeoplasmNiemann Pick Disease
National Institute of Neurological Disorders and Stroke (NINDS) 123
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 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
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 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
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
Not applicableUnknownNCT02000310
What this trial is testing

Molecular and Cellular Mechanisms of Lysosomal Storage Diseases

Who this might be right for
Lysosomal Storage Disorders
O & O Alpan LLC 80
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
Early research (Phase 1)Ended earlyNCT00410566
What this trial is testing

Safety Study of rhASM Enzyme Replacement Therapy in Adults With Acid Sphingomyelinase Deficiency (Niemann-Pick Disease)

Who this might be right for
Acid Sphingomyelinase DeficiencyNiemann-Pick Disease
Genzyme, a Sanofi Company 11
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 B.V. 21
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
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
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
Testing effectiveness (Phase 2)Study completedNCT05758922
What this trial is testing

Phase 2 Study Evaluating the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Oral AZ-3102 in Patients With GM2 Gangliosidosis or Niemann-Pick Type C Disease

Who this might be right for
GM2 GangliosidosisNiemann-Pick Disease, Type C
Azafaros B.V. 13
Large-scale testing (Phase 3)Study completedNCT02004691
What this trial is testing

Efficacy, Safety, Pharmacodynamic, and Pharmacokinetics Study of Olipudase Alfa in Patients With Acid Sphingomyelinase Deficiency

Who this might be right for
Sphingomyelin Lipidosis
Genzyme, a Sanofi Company 36
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