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Results for “Niemann-Pick Diseases”

78 trials

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Showing 20 of 78 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
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
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
Not applicableUnknownNCT02120235
What this trial is testing

Investigating Lysosomal Storage Diseases in Minority Groups

Who this might be right for
Lysosomal Storage DisordersGaucher DiseaseFabry Disease+2 more
O & O Alpan LLC 20,000
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
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
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
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)WithdrawnNCT03687476
What this trial is testing

Safety and Tolerability Study of VTS-270 in Pediatric Participants With Niemann-Pick Type C (NPC) Disease

Who this might be right for
Niemann-Pick Disease, Type C
Vtesse, LLC, a Mallinckrodt Pharmaceuticals Company
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 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
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
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 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
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
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 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
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
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 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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