Clinical Trials: Recruiting
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.
Establishment of Genomic and Phenotypic Database for Niemann-Pick Disease, Type C
Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
National Institutes of Health Clinical Center (CC) (Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)) (Responsible Party)
The primary objective of this protocol is to investigate the phenotypic heterogeneity of NPC by using clinical and genomic data, and to establish a comprehensive database to facilitate future investigations.
Study of IV VTS-270 for Infantile Liver Disease Associated With Niemann-Pick Disease, Type C
Washington University School of Medicine
Washington University School of Medicine (Responsible Party)
This Phase 1/2a, open-label, multiple ascending dose trial will evaluate whether adrabetadex (VTS-270) administered intravenously is effective in treating acute liver disease in NPC1 infants.
Evaluation of Biochemical Markers and Clinical Investigation of Niemann-Pick Disease, Type C
Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
National Institutes of Health Clinical Center (CC) (Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)) (Responsible Party)
The study will: 1) look for a clinical or biochemical marker that can be used as a measure of response to treatment, and 2) define the rate of progression of biochemical marker abnormalities in a group of NPC patients who will later be invited to enroll in a treatment trial.
Phase 3 Study to Evaluate Intravenous Trappsol(R) Cyclo(TM) in Pediatric and Adult Patients With Niemann-Pick Disease Type C1 (TransportNPC)
NCT04860960
December 12, 2023
Cyclo Therapeutics, Inc.
Cyclo Therapeutics, Inc. (Responsible Party)
The objective of this study is to evaluate the safety, tolerability and efficacy of 2000 mg/kg dose of Trappsol Cyclo (hydroxypropyl betacyclodextrin) administered intravenously compared to standard of care.
Study of the Pharmacokinetics of Trappsol and Effects on Potential Biomarkers of Niemann-Pick C1 (NPC1)
Cyclo Therapeutics, Inc.
Cyclo Therapeutics, Inc. (Responsible Party)
This research study is being conducted to find out whether Trappsol® Cyclo™, an experimental treatment for people with Niemann Pick disease Type C (NPC-1) is safe at 2 different dose levels and what effects it has on people who have this condition.
More information can be found on https://clinicaltrials.gov
Share This Content