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Results for “Neimann-Pick Disease”

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Showing 20 of 0 results

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
Large-scale testing (Phase 3)Ended earlyNCT03643562
What this trial is testing

Niemann-Pick Type C Treatment With Adrabetadex for Symptoms of Brain and Nervous System

Who this might be right for
Niemann-Pick Type C Disease
Mandos LLC 27
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
Not applicableNot Yet RecruitingNCT06985212
What this trial is testing

National Multicentre Study of the Natural History of Acid Sphingo-myelinase Deficiency in Adults and Children

Who this might be right for
Acid Sphingomyelinase Deficiency (ASMD)Niemann Pick Disease
Wladimir MAUHIN, Dr 200
Not applicableStudy completedNCT01899950
What this trial is testing

Longitudinal Study of Cognition With Niemann-Pick Disease, Type C

Who this might be right for
Niemann-Pick Disease, Type C
Mayo Clinic 70
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
Not applicableActive Not RecruitingNCT07011329
What this trial is testing

Brain Alterations Based on Multiplex MR Imaging in Nasopharyngeal Carcinoma Patients After Treatment, and Their Correlation With Clinical Cognitive Scores.

Who this might be right for
Nasopharyngeal Carcinoma (NPC)
Hubei Cancer Hospital 60
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
Not applicableStudy completedNCT03883750
What this trial is testing

Induced Pluripotent Stem Cells for Niemann Pick Disease

Who this might be right for
Niemann-Pick Diseases
CENTOGENE GmbH Rostock 40
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
Early research (Phase 1)Study completedNCT01722526
What this trial is testing

Tolerability and Safety Study of Recombinant Human Acid Sphingomyelinase in Acid Sphingomyelinase Deficiency Patients

Who this might be right for
Human Acid Sphingomyelinase Deficiency
Genzyme, a Sanofi Company 5
Not applicableLooking for participantsNCT05588167
What this trial is testing

Establishment of Genomic and Phenotypic Database for 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) 100
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
Large-scale testing (Phase 3)Ended earlyNCT00654433
What this trial is testing

ALD-101 Adjuvant Therapy of Unrelated Umbilical Cord Blood Transfusion (UCBT) in Patients With Inherited Metabolic Diseases

Who this might be right for
Inherited Metabolic DiseasesLysosomal Storage DisordersPeroxisomal Storage Diseases+2 more
Aldagen 40
Testing effectiveness (Phase 2)Ended earlyNCT03887533
What this trial is testing

Combined Intrathecal and Intravenous VTS-270 Therapy for Liver and Neurological Disease Associated With Niemann-Pick Disease, Type C1

Who this might be right for
Niemann-Pick Disease, Type C1
Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD) 2
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
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)Study completedNCT01760564
What this trial is testing

Application of Miglustat in Patients With Niemann-Pick Type C

Who this might be right for
Niemann-Pick Disease Type C
National Taiwan University Hospital 5
Not applicableUnknownNCT05904366
What this trial is testing

Magnetic Resonance Spectroscopy in Acid Sphingomyelinase Deficiency

Who this might be right for
Asmd, Visceral Type
Eline C. B. Eskes 34
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