Clinical Trials: Completed
Disclaimer: Information regarding clinical trials for NPC disease is obtained from the https://clinicaltrials.gov website and may not capture every trial. Please see links for additional information about specific clinical trials.
Longitudinal Study of Cognition With Niemann-Pick Disease, Type C (NPC)
NCT01899950
January 6, 2016
Mayo Clinic
Marc C. Patterson, M.D., Mayo Clinic (Responsible Party)
This study will examine and characterize the longitudinal progression of neurocognitive symptoms of NPC with the goal of identifying early markers of disease progression that may be utilized in later trials to evaluate treatment efficacy.
A Prospective Non-therapeutic Study in Patients Diagnosed With Niemann-Pick Disease Type C
ZevraDenmark
ZevraDenmark (Responsible Party)
The aim is to characterize the individual patient disease progression profile through the historical and 6 months prospective evaluation of clinical, imaging, biological(biomarkers) and quality of life data.
N-Acetyl-L-Leucine for Niemann-Pick Disease, Type C (NPC)
IntraBio Inc
IntraBio Inc (Responsible Party)
This is a multinational, multicenter, open-label, rater-blinded prospective Phase II study which will assess the safety and efficacy of N-Acetyl-L-Leucine (IB1001) for the treatment of Niemann-Pick type C disease (NPC).
Biomarker Validation for Niemann-Pick Disease, Type C: Safety and Efficacy of N-Acetyl Cysteine
Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
The goals of this protocol are:
- To validate the use of biomarkers in a therapeutic trial for NPC.
- To evaluate the safety of N-acetylcysteine in NPC patients.
- To evaluate the efficacy of N-acetylcysteine to improve biomarkers associated with increased oxidative stress in NPC patients.
Application of Miglustat in Patients With Niemann-Pick Type C
National Taiwan University Hospital
National Taiwan University Hospital (Responsible Party)
To evaluate the changes in cognitive function after miglustat treatment in Niemann-Pick type C patients.
VTS-270 to Treat Niemann-Pick Type C1 (NPC1) Disease
Mandos LLC
Mandos LLC (Responsible Party)
This study is to find out how safe and effective VTS-270 is for patients with Niemann-Pick Type C1 (NPC1) disease who have neurologic symptoms
Safety and Efficacy of Miglustat in Chinese NPC Patients
Actelion
Actelion (Responsible Party)
Saccadic Eye Movements in Patients With Niemann-Pick Type C Disease
National Eye Institute (NEI)
National Institutes of Health Clinical Center (CC)
This study is done in conjunction with a trial, conducted at Columbia University College of Physicians and Surgeons in New York and the Royal Manchester Children's Hospital in England, to examine the effectiveness of a new drug called OGT 918 for treating Niemann-Pick Type C (NPC) disease.
Safety and Efficacy of Intravenous Trappsol Cyclo (HPBCD) in Niemann-Pick Type C Patients
Cyclo Therapeutics, Inc.
Cyclo Therapeutics, Inc. (Responsible Party)
This is a Phase I/II Study to Evaluate the Safety and PK of iv Trappsol Cyclo (HP-β-CD) in Patients With NPC1 and the Pharmacodynamic Effects of Treatment Upon Markers of Cholesterol Metabolism and Clinical Outcomes.
Miglustat in Niemann-Pick Type C Disease
Actelion
Actelion
This is a phase II randomized controlled study of miglustat in adult and juvenile patients with Niemann-Pick Type C disease.
More information can be found on https://clinicaltrials.gov
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