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)

ClinicalTrials.gov ID
NCT01899950
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Last Update Posted
January 6, 2016
Sponsor
Mayo Clinic
Information provided by
Marc C. Patterson, M.D., Mayo Clinic (Responsible Party)
Study Description:

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

ClinicalTrials.gov ID
NCT02435030
Last Update Posted
May 18, 2017
Sponsor
ZevraDenmark
Information provided by
ZevraDenmark (Responsible Party)
Study Description:

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)

ClinicalTrials.gov ID
NCT03759639
Last Update Posted
November 28, 2023
Sponsor
IntraBio Inc
Information provided by
IntraBio Inc (Responsible Party)
Study Description:

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

ClinicalTrials.gov ID
NCT00975689
Last Update Posted
May 6, 2013
Sponsor
Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
Information provided by
Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
Study Description:

The goals of this protocol are:

  1. To validate the use of biomarkers in a therapeutic trial for NPC.
  2. To evaluate the safety of N-acetylcysteine in NPC patients.
  3. 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

ClinicalTrials.gov ID
NCT01760564
Last Update Posted
January 4, 2013
Sponsor
National Taiwan University Hospital
Information provided by
National Taiwan University Hospital (Responsible Party)
Study Description:

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

ClinicalTrials.gov ID
NCT02534844
Last Update Posted
February 22, 2023
Sponsor
Mandos LLC
Information provided by
Mandos LLC (Responsible Party)
Study Description:

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

ClinicalTrials.gov ID
NCT03910621
Last Update Posted
May 25, 2022
Sponsor
Actelion
Information provided by
Actelion (Responsible Party)

Saccadic Eye Movements in Patients With Niemann-Pick Type C Disease

ClinicalTrials.gov ID
NCT00316498
Last Update Posted
July 2, 2017
Sponsor
National Eye Institute (NEI)
Information provided by
National Institutes of Health Clinical Center (CC)
Study Description:

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

ClinicalTrials.gov ID
NCT02912793
Last Update Posted
August 10, 2021
Sponsor
Cyclo Therapeutics, Inc.
Information provided by
Cyclo Therapeutics, Inc. (Responsible Party)
Study Description:

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

ClinicalTrials.gov ID
NCT00517153
Last Update Posted
March 26, 2010
Sponsor
Actelion
Information provided by
Actelion
Study Description:

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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