Clinical trials: Niemann-Pick Disease, Type C1
Juvenile Neuronal Ceroid Lipofuscinosis, Smith-Lemli-Opitz Syndrome
Functional Near-Infrared Spectroscopy (fNIRS) Combined With Diffuse Correlation Spectroscopy (DCS) in Neurocognitive Disease as Compared to Healthy Neurotypical Controls
Background: Neurocognitive disorders affect how the brain uses oxygen. They may affect mental development in children. These disorders can be studied with imaging scans that use radiation; however, these methods are not ideal for…
- Conditions
- Juvenile Neuronal Ceroid Lipofuscinosis, Smith-Lemli-Opitz Syndrome, Creatine Transporter Deficiency, Niemann-Pick Disease Type C1
- Trial name
- NCT05642221
- Where
- United States
- Volunteers
- 73 people
- Sponsor
- Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
Spinal Muscular Atrophy, Fragile X Syndrome
Early Check: Expanded Screening in Newborns
Early Check provides voluntary screening of newborns for a selected panel of conditions. The study has three main objectives: 1) develop and implement an approach to identify affected infants, 2) address the impact on infants and…
- Conditions
- Spinal Muscular Atrophy, Fragile X Syndrome, Fragile X - Premutation, Duchenne Muscular Dystrophy
- Trial name
- NCT03655223
- Testing
- Confirmatory Testing
- Where
- United States
- Volunteers
- 30,000 people, healthy volunteers welcome
- Sponsor
- RTI International
Niemann-Pick Disease, Type C1
Phase 3 Study to Evaluate Intravenous Trappsol(R) Cyclo(TM) in Pediatric and Adult Patients With Niemann-Pick Disease Type C1
A prospective, randomized, double-blind, placebo controlled, multi-center therapeutic study for patients age 3 and older with confirmed diagnosis of Niemann Pick disease type C1 (NPC1).
- Trial name
- TransportNPC (NCT04860960)
- Testing
- Hydroxypropyl-beta-cyclodextrin
- Where
- 35 sites in United States, Argentina and 11 more
- Volunteers
- 94 people
- Sponsor
- Cyclo Therapeutics, Inc.
Niemann-Pick Disease, Type C1
Combined Intrathecal and Intravenous VTS-270 Therapy for Liver and Neurological Disease Associated With Niemann-Pick Disease, Type C1
Background: For people who have Niemann-Pick disease, type C1 (NPC1), cholesterol and other fats have trouble moving out of liver and other tissue cells. This makes the cells sick.
- Trial name
- NCT03887533
- Testing
- VTS-270
- Where
- United States
- Volunteers
- 2 people
- Sponsor
- Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
Niemann-Pick Disease, Type C1
Safety and Efficacy of Intravenous Trappsol Cyclo (HPBCD) in Niemann-Pick Type C Patients
This research study is being conducted to find out whether Trappsol® Cyclo™, an experimental treatment for people with Niemann-Pick disease Type C1 (NPC-1) is safe at 3 different dose levels and what effects it has on people who…
- Trial name
- NCT02912793
- Testing
- Hydroxypropyl-beta-cyclodextrin
- Where
- 5 sites in Israel, Sweden and 1 more
- Volunteers
- 12 people
- Sponsor
- Cyclo Therapeutics, Inc.
Niemann-Pick Disease, Type C1
Hydroxypropyl Beta Cyclodextrin for Niemann-Pick Type C1 Disease
Background: Hydroxypropyl beta cyclodextrin (HPBCD) is being tested for a disease called Niemann-Pick disease type C1 (NPC1). NPC1 is a genetic disorder that results in gradual loss of nervous system function.
- Trial name
- NCT01747135
- Testing
- VTS-270
- Where
- United States
- Volunteers
- 14 people
- Sponsor
- Vtesse, LLC, a Mallinckrodt Pharmaceuticals Company
Niemann-Pick Disease, Type C1
Open-Label Study of Long-Term Safety and Efficacy of Intravenous Trappsol Cyclo (HPβCD) in Niemann-Pick Disease Type C
The purpose of this study is to provide continued access to treatment for NPC-1 after participation and completion of the Phase I trial CTD-TCNPC-101, when administered at doses of 1500 mg/kg and 2500 mg/kg by slow IV infusion…
- Trial name
- NCT03893071
- Testing
- Hydroxypropyl-β-cyclodextrin
- Where
- United States
- Volunteers
- 12 people
- Sponsor
- Cyclo Therapeutics, Inc.
Niemann-Pick Disease, Type C1
Study of the Pharmacokinetics of Trappsol and Effects on Potential Biomarkers of Niemann-Pick C1 (NPC1)
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…
- Trial name
- NCT02939547
- Testing
- Hydroxypropyl-beta-cyclodextrin
- Where
- 2 sites in United States
- Volunteers
- 13 people
- Sponsor
- Cyclo Therapeutics, Inc.
Niemann-Pick Disease, Type C1
Study of Lithium Carbonate to Treat Niemann-Pick Type C1 Disease
This study is planned to study whether lithium carbonate has protective effect on the brain of Niemann-Pick disease type C1.
- Trial name
- NCT03201627
- Testing
- Lithium Carbonate
- Where
- China
- Volunteers
- 18 people
- Sponsor
- Xinhua Hospital, Shanghai Jiao Tong University School of Medicine