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Results for “Niemann-Pick Disease Type A”

37 trials

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Showing 20 of 37 results

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)Looking for participantsNCT05163288
What this trial is testing

A Pivotal Study of N-Acetyl-L-Leucine on Niemann-Pick Disease Type C

Who this might be right for
Niemann-Pick Disease, Type C
IntraBio Inc 53
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
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 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
Early research (Phase 1)Study completedNCT01586455
What this trial is testing

Human Placental-Derived Stem Cell Transplantation

Who this might be right for
Mucopolysaccharidosis IMucopolysaccharidosis VIAdrenoleukodystrophy+13 more
New York Medical College 43
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)Looking for participantsNCT07082725
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 (NPC)

Who this might be right for
Niemann-Pick Type C Disease
Azafaros B.V. 72
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
Testing effectiveness (Phase 2)Study completedNCT00730314
What this trial is testing

Unrelated Hematopoietic Stem Cell Transplantation(HSCT) for Genetic Diseases of Blood Cells

Who this might be right for
Sickle Cell DiseaseThalassemiaAnemia+9 more
Children's Hospital Los Angeles 25
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
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
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 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 applicableEnrolling By InvitationNCT05368038
What this trial is testing

ScreenPlus: A Comprehensive, Flexible, Multi-disorder Newborn Screening Program

Who this might be right for
Acid Sphingomyelinase DeficiencyCeroid Lipofuscinosis, Neuronal, 2Cerebrotendinous Xanthomatosis+11 more
Albert Einstein College of Medicine 100,000
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
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
Testing effectiveness (Phase 2)Study completedNCT03759639
What this trial is testing

N-Acetyl-L-Leucine for Niemann-Pick Disease, Type C (NPC)

Who this might be right for
Niemann-Pick Disease, Type C
IntraBio Inc 33
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
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
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