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Results for “Niemann-Pick Disease Type A”

38 trials

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Showing 20 of 38 results

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 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 completedNCT02435030
What this trial is testing

A Prospective Non-therapeutic Study in Patients Diagnosed With Niemann-Pick Disease Type C

Who this might be right for
Niemann-Pick Disease, Type C
ZevraDenmark 35
Large-scale testing (Phase 3)Looking for participantsNCT07082543
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 A.G. 75
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
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
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 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
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
Not applicableEnrolling By InvitationNCT03655223
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
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
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
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
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)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
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 A.G. 72
Testing effectiveness (Phase 2)Not Yet RecruitingNCT07399704
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 A.G. 21
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 applicableStudy completedNCT04845958
What this trial is testing

A Non-Interventional National Study in Pediatric Patients With Unexplained Enlarged Spleen

Who this might be right for
Gaucher Disease, SplenomegalyAcid SphingoMyelinase Deficiency
Sanofi 60
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
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