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Clinical Trials About "Study of the Pharmacokinetics of Trappsol and Effects on Potential Biomarkers of Niemann-Pick C1 (NPC1)" RSS

13:36 EST 15th November 2019 | BioPortfolio

We list hundreds of Clinical Trials about "Study of the Pharmacokinetics of Trappsol and Effects on Potential Biomarkers of Niemann-Pick C1 (NPC1)" on BioPortfolio. We draw our references from global clinical trials data listed on ClinicalTrials.gov and refresh our database daily.

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Showing "Study Pharmacokinetics Trappsol Effects Potential Biomarkers Niemann Pick" Clinical Trials 1–25 of 35,000+

Extremely Relevant

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 have this condition. NPC-1 is caused by a defect in the protein which is important for the transport of fatty substances like cholesterol out of cells. Without this protein, fats build up in the cells ultimately leading to organ damage. The way in whi...


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 over a period of 8 to 9 hours every two weeks.

Study of Pharmacokinetics and Preliminary Efficacy in Patients With Niemann-Pick C1

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 have this condition. NPC-1 is caused by a defect in a protein which is important for the transport of fatty substances like cholesterol out of cells. Without this protein, fats build up in the cells ultimately leading to organ damage. The way in whic...


Miglustat in Niemann-Pick Type C Disease

This is a phase II randomized controlled study of miglustat in adult and juvenile patients with Niemann-Pick Type C disease. Up to 42 patients will be randomised in a 2:1 ratio to either treatment with miglustat or to a non-treatment group. Both groups will follow an identical visit schedule.

Induced Pluripotent Stem Cells for Niemann Pick Disease

Establishment of individualized human cellular disease models based on induced pluripotent stem cells that reflect the broad heterogeneous phenotypic spectrum of Niemann Pick disease

N-Acetyl-L-Leucine for Niemann-Pick Disease, Type C (NPC)

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).

Open-label Study of VTS-270 in Participants With Neurologic Manifestations of Niemann-Pick Type C1

This is a multicenter, multinational, open-label study of to evaluate the long-term safety, tolerability, and efficacy of VTS-270 (2-hydroxypropyl-β-cyclodextrin) in participants transitioning from Study VTS301 in participants with Neurologic Manifestations of Niemann-Pick Type C1 Disease.

A Prospective Non-therapeutic Study in Patients Diagnosed With Niemann-Pick Disease Type C

This is a prospective non-therapeutic observational study in NP-C patients. 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. Patients will be offered enrollment into a Phase II/III study on arimoclomol at the end of the study.

Study of 2-hydroxypropyl-β-cyclodextrin (HP-β-CD) to Treat Niemann-Pick Type C1 (NPC1) Disease

This study evaluates the efficacy and safety of 2-hydroxypropyl-β-cyclodextrin (HP-β-CD) in patients with neurologic manifestations of Niemann-Pick Type C1 (NPC1) Disease. Approximately two-thirds of patients will receive the study drug, 2-hydroxypropyl-β-cyclodextrin (HP-β-CD), while the remaining study participants will receive sham control.

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.

Safety and Efficacy of Miglustat in Chinese NPC Patients

This is a prospective, multi-center, open-label, non-randomized, single-arm Phase IV confirmatory study. Approximately 19 subjects with Niemann Pick Type C disease (NPC) will be enrolled in this study. The study will be conducted at 2 sites in China.

Safety and Tolerability Study of VTS-270 in Pediatric Participants With Niemann-Pick Type C (NPC) Disease

This is a Phase-2, multicenter, multiple dose, open-label, 2-part evaluation study which will primarily assess the safety and tolerability of VTS-270 (2-hydroxypropyl beta-cyclodextrin [HP-β-CD]) in pediatric participants with age

Screening of Niemann-Pick Disease, Type C in a Psychiatric Population

Niemann-Pick disease, Type C is a rare genetic disorder characterized by a failing in intracellular cholesterol transport, inducing an accumulation of sphingolipids in the brain. Neurological signs are at the forefront of the disease. There are also psychiatric signs of psychotic kind among 28 to 45 % of patients according to studies, and a thirty cases were published. These signs can be concomitant with neurological signs or precede them. Is is likely that psychotic disorder...

Study of IV VTS-270 for Infantile Liver Disease Associated With Niemann-Pick Disease, Type C

Niemann-Pick disease, type C (NPC) is a lethal, autosomal recessive, lysosomal storage disorder characterized by neurodegeneration in early childhood and death in adolescence. NPC results from mutation of either the Niemann-Pick C1 disease (NPC1) (~95% of cases) or NPC2 genes. NPC is characterized by the endolysosomal storage of unesterified cholesterol and lipids in both the central nervous system and peripheral tissues such as the liver. Individuals with NPC demonstrate progr...

An Open-label Expanded Access Treatment Protocol for VTS-270 in Patients With Neurologic Manifestations of NPC

Prospective, open-label, treatment protocol to provide expanded access to VTS-270 to eligible Niemann-Pick type C disease (NPC) patient

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. Researchers want to find out if a drug called VTS-270 can help. Objective: To test if VTS-270 is safe and effective in treating chronic liver disease associated with NPC1. Eligibility: People ages 4 60 with NPC1 Design: Participants may be screened by phone or und...

Expanded Access With Trappsol(R) Cyclo (TM) for an Individual Patient With Late Onset Alzheimer's Disease

To afford urgent access to a potential-disease modifying treatment, Dr. Diana Kerwin, in partnership with CTD Holdings, the manufacturer of Trappsol (R) Cyclo(TM), will administer the product to a patient with Alzheimer's Disease who has no other disease-modifying treatment options.

Relevant

Safety Study of rhASM Enzyme Replacement Therapy in Adults With Acid Sphingomyelinase Deficiency (Niemann-Pick Disease)

The purpose of this study is to determine the safe range of single doses of rhASM administered to adults with ASM deficiency.

Far Infrared Irradiation for the Management, Control and Treatment of Frontotemporal Dementia

Pick's disease, also known as Pick disease,or FTD is a rare fronto-temporal neurodegenerative disease. This study will investigate the use of far infrared radiation for the control, management and treatment of Pick's disease.

Arimoclomol Prospective Study in Patients Diagnosed With NiemannPick Disease Type C

A prospective, randomised, double-blind, placebo controlled therapeutic study in patients with confirmed diagnosis of NiemannPick disease type C (NPC). The purpose of this study is to assess the efficacy and safety of arimoclomol (compared to placebo) when it is administered as an add-on therapy to the patient's current prescribed best standard of care; patient's standard of care may, or may not, include miglustat.

Benefits of an Oocyte Pickup Simulation Training Program

The primary objective of this study was to evaluate oocyte pick up simulation training program for teaching residents. The secondary objectives were to evaluate resident satisfaction and the overall current state of oocyte pick up training in France.

Prognostic Value of Progesterone on Oocyte Retrieval Day for In Vitro Fertilization Outcome

This observational study aims the potential role of serum progesterone levels on the day of oocyte pick-up on the probability of pregnancy, in patients undergoing antagonist IVF cycles. There has been evidence, that increased serum levels of progesterone on triggering day are associated with a decreased probability of pregnancy after IVF. There is no study that investigate assocation between serum progesterone levels on the day of oocyte pick-up and IVF outcomes. This s...

Improving Clinic Attendance for Medication Collection Among HIV Positive Individuals in Nepal

Antiretroviral therapy (ART) has been a game changer in the context of HIV-epidemic. From 2005 to 2015, HIV-related deaths have fallen by 45% thanks to ART. However, ART's success heavily depends on HIV-positive individuals' high adherence to it. This includes clinic attendance for various purposes. It is necessary among HIV-positive individuals for their antiretroviral (ARV) pills pick up, monitoring of their treatment outcomes, and treatment of their opportunistic infections....

Evaluation of Biochemical Markers and Clinical Investigation of Niemann-Pick Disease, Type C

This study will evaluate clinical and laboratory tests that might be useful in determining if an investigational drug can slow the progression of Niemann-Pick Disease, Type C (NPC), a genetic disorder that results in progressive loss of nervous system function. 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 wh...

Characterization of Potential Biomarkers of Eye Disease and Vision

Characterize potential biomarkers of sight-threatening eye diseases based on the information acquired in clinical practice at CEC, AIBILI


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