Clinical trials: Brain Disorder, Metabolic
Niemann-Pick Disease, Type C
Arimoclomol Prospective Study in Participants Diagnosed With Niemann-Pick Disease Type C
A prospective, randomized, double-blind, placebo controlled therapeutic study in participants with confirmed diagnosis of Niemann-Pick disease type C (NPC).
- Trial name
- NCT02612129
- Testing
- arimoclomol
- Where
- 14 sites in United States, Denmark and 7 more
- Volunteers
- 50 people
- Sponsor
- ZevraDenmark
Diabetic Ketoacidosis
Cerebral Edema in Pediatric Diabetic Ketoacidosis
The purpose of this trial is to compare two different rates of fluid administration during diabetic ketoacidosis (DKA) treatment in children to determine which fluid administration rate is more beneficial for brain metabolism and…
- Trial name
- NCT00629707
- Testing
- intravenous fluid treatment
- Where
- United States
- Volunteers
- 20 people
- Sponsor
- University of California, Davis
Wilson's Disease
A Phase I/II Study of VTX-801 in Adult Patients With Wilson's Disease
The objectives of this clinical trial are to assess, for up to 5 years, the safety, tolerability and pharmacological activity of a single ascending doses of VTX-801, a gene therapy, administered intravenously (IV) to adult…
- Trial name
- GATEWAY (NCT04537377)
- Testing
- VTX-801
- Where
- 10 sites in United States, Denmark and 2 more
- Volunteers
- 4 people
- Sponsor
- Vivet Therapeutics SAS
Gaucher Disease
Open-Label Extension Study Evaluating Long Term Safety in Patients With Type 1 Gaucher Disease Receiving DRX008A (ERT)
Gaucher disease is a rare lysosomal storage disorder caused by the deficiency of the enzyme glucocerebrosidase (GCB). Due to the deficiency of functional GCB, glucocerebroside accumulates within macrophages leading to cellular…
- Trial name
- NCT00391625
- Testing
- GA-GCB
- Where
- 3 sites in Israel, Romania and 1 more
- Volunteers
- 10 people
- Sponsor
- Shire
Brain Diseases, Metabolic, Inborn, Urea Cycle Disorder
Neurologic Injuries in Adults With Urea Cycle Disorders
Urea cycle disorders (UCDs) are a group of rare inherited metabolism disorders. The purpose of this study is to evaluate how UCD-related neurologic injuries affect adults with one of the most common types of UCD.
- Conditions
- Brain Diseases, Metabolic, Inborn, Urea Cycle Disorder, Ornithine Transcarbamylase Deficiency
- Trial name
- NCT00472732
- Where
- 2 sites in United States
- Volunteers
- 46 people, healthy volunteers welcome
- Sponsor
- Andrea Gropman
Glucose Metabolism Disorders
Glucose and Non-Invasive Brain Stimulation
Purpose: In this study, the investigators will delineate how brain network dynamics are modulated by experimentally induced elevated blood glucose levels and examine how glucose levels gate neuronal excitability measured by the…
- Trial name
- NCT04031404
- Testing
- Single-pulse TMS
- Where
- United States
- Volunteers
- 23 people, healthy volunteers welcome
- Sponsor
- University of North Carolina, Chapel Hill
Friedreich Ataxia
An Objective Double-blind Evaluation of Bupropion and Citalopram in an Individual With Friedreich Ataxia
The purpose of this study is to document the clinical effects of bupropion and citalopram in a single subject with Friedreich Ataxia.
- Trial name
- NCT01716221
- Testing
- bupropion & Citalopram
- Volunteers
- 1 people
- Sponsor
- University of Colorado, Denver
Gaucher Disease, Type 1
Phase III Study of ISU302 in Patients With Type 1 Gaucher Disease
The purpose of this study is to evaluate the safety and efficacy of ISU302 in patients with Type 1 Gaucher disease.
- Trial name
- NCT02770625
- Testing
- ISU302
- Volunteers
- 8 people
- Sponsor
- ISU Abxis Co., Ltd.
Phenylketonurias
Study to Evaluate the Safety and Efficacy of Phenoptin™ in Subjects With Phenylketonuria Who Have Elevated Phenylalanine Levels
The primary objective of this study is to evaluate the efficacy of Phenoptin™ (sapropterin dihydrochloride) in reducing blood phenylalanine (Phe) levels in subjects with phenylketonuria.
- Trial name
- NCT00104247
- Testing
- sapropterin dihydrochloride, 6R-BH4, tetrahydrobiopterin
- Where
- 13 sites in United States
- Volunteers
- 89 people
- Sponsor
- BioMarin Pharmaceutical
Dementia, Alzheimer Disease
The Healthy Patterns Sleep Study
The Healthy Patterns Study intervention is a home-based activity intervention designed to improve symptoms of circadian rhythm disorders (CRD) and quality of life (QOL) in home-dwelling persons with dementia.
- Conditions
- Dementia, Alzheimer Disease, Circadian Rhythm Disorders, Circadian Rhythm Sleep Disorder
- Trial name
- NCT03682185
- Testing
- Attention-Control Condition, Timed Activity Intervention
- Where
- United States
- Volunteers
- 421 people, healthy volunteers welcome
- Sponsor
- University of Pennsylvania
Stroke, Acute, Hyperglycemia
Pioglitazone Treatment for Hyperglycemic Acute Ischemic Stroke
Study objective is to determine whether Pioglitazone (PGZ) can improve clinical outcomes in hyperglycemic acute ischemic stroke (IS).
- Conditions
- Stroke, Acute, Hyperglycemia, Diabetes
- Trial name
- NCT04123067
- Testing
- Pioglitazone 45 mg
- Where
- United States
- Volunteers
- 1 people
- Sponsor
- Milton S. Hershey Medical Center
Phenylketonuria (PKU)
Study to Evaluate the Safety and Efficacy of Pegvaliase in Adolescents (Ages 12-17) With Phenylketonuria
This is a Phase 3 open-label randomized controlled study enrolling approximately 54 adolescents with PKU. The study is designed to assess the safety and efficacy of pegvaliase injections.
- Trial name
- PEGASUS (NCT05270837)
- Testing
- Pegvaliase, Diet Only
- Where
- 16 sites in United States, Germany
- Volunteers
- 55 people
- Sponsor
- BioMarin Pharmaceutical
Gaucher Disease
A Study of Eliglustat Tartrate (Genz-112638) in Patients With Gaucher Disease to Evaluate Once Daily Versus Twice Daily Dosing (EDGE)
The primary objective of this study was to evaluate the efficacy and safety of once daily (QD) versus twice daily (BID) dosing of eliglustat tartrate (Genz-112638) in participants with Gaucher disease type 1 who had demonstrated…
- Trial name
- NCT01074944
- Testing
- Eliglustat tartrate
- Where
- 46 sites in United States, Australia and 15 more
- Volunteers
- 170 people
- Sponsor
- Genzyme, a Sanofi Company
Peroxisome Biogenesis Disorders
Betaine and Peroxisome Biogenesis Disorders
The PBD are a rare group of inherited disorders due to the failure to form functional cellular peroxisomes. Most patients have progressive hearing and visual loss, leading to deafness and blindness, as well as neurological…
- Trial name
- NCT01838941
- Testing
- Betaine
- Where
- Canada
- Volunteers
- 12 people
- Sponsor
- McGill University Health Centre/Research Institute of the McGill University Health Centre
Schizophrenia
Impact of Aripiprazole Once Monthly Medications on Changes in Brain Structure and Metabolism
This is a Phase 4, randomized, open-label, parallel group study designed to assess the clinical and biological effects of 12 months of treatment with long acting intramuscular (IM) aripiprazole (Abilify®) as measured by clinical…
- Trial name
- NCT02237417
- Testing
- Standard of Care Oral antipsychotics, IM aripiprazole once monthly, No Treatment
- Where
- United States
- Volunteers
- 47 people, healthy volunteers welcome
- Sponsor
- University of Utah
Parkinson Disease, Lewy Body Dementia
Brain Small Chain Fatty Acid Metabolism in Parkinson Disease: Ketones
Small exploratory open-label pilot study to assess supplementation of a ketone ester (KetoneAid) as a potential therapy for persons with Parkinson disease (PD), Parkinson Disease Dementia/Lewy Body Dementia (PDD/LBD), and healthy…
- Conditions
- Parkinson Disease, Lewy Body Dementia, Parkinson Disease Dementia
- Trial name
- NCT05778695
- Testing
- Ketone Ester (R)-3-Hydroxybutyl (R)-3-Hydroxybutyrate
- Where
- 2 sites in United States
- Volunteers
- 16 people, healthy volunteers welcome
- Sponsor
- University of Michigan
Idiopathic Parkinson's Disease
The Effect of Pramipexole on Metabolic Network Activity Compared With Levodopa in Early Parkinson's Disease
Levodopa and non-ergot dopaminergic agonists such as pramipexole are both recommended as the first-line symptomatic treatment for early untreated Parkinson's disease (PD), previous clinical trial indicated that initial…
- Trial name
- NCT01470859
- Testing
- pramipexole, Sinemet CR
- Where
- China
- Volunteers
- 30 people
- Sponsor
- Huashan Hospital
Fabry Disease
Extension Study of PRX-102 for up to 60 Months
To evaluate the ongoing safety, tolerability, and efficacy parameters of PRX-102 in adult Fabry patients who have successfully completed treatment with PRX-102 in studies PB-102-F01 and PB-102-F02.
- Trial name
- NCT01981720
- Testing
- PRX-102 (pegunigalsidase alfa)
- Where
- 8 sites in United States, Paraguay and 2 more
- Volunteers
- 15 people
- Sponsor
- Protalix
Hyperglycemia, Stroke, Acute
Insulin for Hyperglycemia in Stroke Trial
Introduction: Glycemic control in acutely ill stroke patients with hyperglycemia is vital. Although insulin is the choice of anti-diabetic agent during acute stage, it is not clear which insulin regimen is better in terms of…
- Trial name
- NCT04834362
- Testing
- Analog Insulin, Human insulin
- Where
- Bangladesh
- Volunteers
- 452 people
- Sponsor
- National Institute of Neurosciences and Hospital, Dhaka
Cirrhosis, Ascites
The BRAINFOOD Trial to Prevent Recurrent Hepatic Encephalopathy.
This study is being completed for patients with cirrhosis, including patients with a prior history of hepatic encephalopathy (HE) to evaluate the feasibility and benefits of medically-tailored meals as an intervention.
- Conditions
- Cirrhosis, Ascites, Liver Diseases, Frailty
- Trial name
- NCT04675775
- Testing
- Medically-tailored meals (MTM), Protein supplements, Nutrition education handout
- Where
- United States
- Volunteers
- 17 people
- Sponsor
- University of Michigan