Crick
HomeTrialsGenesDrugs23andMeGraphBlogContact
  • Home
  • Trials
  • Genes
  • Drugs
  • 23andMe
  • Graph
  • Blog
  • Contact
Crick

An open-source clinical intelligence platform. Explore clinical trials, gene networks, and molecular structures using public data sources.

Data Sources

  • ClinicalTrials.gov
  • OpenTargets
  • ClinVar
  • PubChem

Links

  • Contact
  • Privacy

© 2026 Crick. All rights reserved.

Crick is for educational purposes only. Not medical advice.

Results for “Niemann-Pick Disease, Type C”

Filters

Phase
Early Phase 1
Phase 1
Phase 2
Phase 3
Phase 4
Status
Recruiting
Active, Not Recruiting
Not Yet Recruiting
Completed
Enrolling by Invitation

Showing 20 of 0 results

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
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 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 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
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
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
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
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
Testing effectiveness (Phase 2)Active Not RecruitingNCT02171104
What this trial is testing

MT2013-31: Allo HCT for Metabolic Disorders and Severe Osteopetrosis

Who this might be right for
Mucopolysaccharidosis DisordersHurler SyndromeHunter Syndrome+26 more
Masonic Cancer Center, University of Minnesota 149
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 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
Testing effectiveness (Phase 2)Ended earlyNCT00668564
What this trial is testing

Hematopoietic Stem Cell Transplantation (HCT) for Inborn Errors of Metabolism

Who this might be right for
Hurler's SyndromeMaroteaux-Lamy SyndromeSly Syndrome+8 more
Masonic Cancer Center, University of Minnesota 18
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
Large-scale testing (Phase 3)Ended earlyNCT04958642
What this trial is testing

Adrabetadex to Treat Niemann-Pick Type C1 (NPC1) Disease

Who this might be right for
Niemann-Pick Disease, Type C
Mandos LLC 66
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
Next Page