Clinical trials: Brain Diseases, Metabolic
Pompe Disease (Late-onset)
ZIP Study-OL Study of Safety, PK, Efficacy, PD, Immunogenicity of ATB200/AT2221 in Pediatrics Aged 0 to < 18 y.o. w/LOPD
This is a Phase 3, open-label, multicenter study to evaluate the safety, PK, efficacy, PD, and immunogenicity of Cipaglucosidase Alfa/Miglustat treatment in enzyme replacement therapy (ERT)-experienced and ERT-naïve pediatric…
- Trial name
- NCT03911505
- Testing
- Cipaglucosidase Alfa, Miglustat
- Where
- 17 sites in United States, Australia and 4 more
- Volunteers
- 21 people
- Sponsor
- Amicus Therapeutics
Gaucher Disease
Plant Cell Expressed Recombinant Human Glucocerebrosidase Extension Trial
Gaucher disease, the most prevalent lysosomal storage disorder, is caused by mutations in the human glucocerebrosidase gene (GCD) leading to reduced activity of the lysosomal enzyme glucocerebrosidase and thereby to the…
- Trial name
- NCT00705939
- Testing
- Taliglucerase alfa
- Where
- 11 sites in United States, Australia and 6 more
- Volunteers
- 45 people
- Sponsor
- Pfizer
Zellweger Syndrome, Peroxisome Biogenesis Disorders
Hydroxychloroquine Administration for Reduction of Pexophagy
A series of N-of-1, crossover, randomized, placebo-controlled, double-blinded trial. Hydroxychloroquine (HCQ) and a crossover to placebo (order is randomized and blinded) will be administered in liquid suspension for 84 days (12…
- Trial name
- HARP (NCT03856866)
- Testing
- Hydroxychloroquine
- Where
- Canada
- Volunteers
- 3 people
- Sponsor
- The Hospital for Sick Children
Alzheimer's Disease (AD), Neurodegenerative Disease
A Biomarker-chip Assay in Preserved Red Blood Cells for the Diagnosis of Alzheimer's Disease
The ADCHIP study (ST0067) is a non-interventional, monocentric, prospective study conducted by Amoneta Diagnostics and the Leenaards Memory Center (Lausanne, Switzerland).
- Conditions
- Alzheimer's Disease (AD), Neurodegenerative Disease, Dementia, Frontotemporal Dementia (FTD)
- Trial name
- ADCHIP (NCT07307872)
- Where
- Switzerland
- Volunteers
- 150 people, healthy volunteers welcome
- Sponsor
- Amoneta Diagnostics SAS
Pompe Disease
Muscle Response to Enzyme Replacement Therapy in Pompe Disease
This study is to study the response of muscle cells from Pompe disease after enzyme replacement therapy
- Trial name
- NCT00713453
- Where
- Taiwan
- Volunteers
- 16 people, healthy volunteers welcome
- Sponsor
- National Taiwan University Hospital
Gaucher Disease, Type 1
A Gene Therapy Study in Patients With Gaucher Disease Type 1
This study is a first-in-human, open-label, safety, tolerability, and efficacy study in adult patients with Gaucher disease Type 1.
- Trial name
- GALILEO-1 (NCT05324943)
- Testing
- FLT201
- Where
- 10 sites in United States, Brazil and 4 more
- Volunteers
- 10 people
- Sponsor
- Spur Therapeutics
Diabetes, Diabetes Mellitus Type 2
Cognitive Screening and Multidomain Intervention Targeting Cognitive Decline in Type 2 Diabetes (The Brain Care Study)
The purpose of The Brain Care Study is to investigate the prevalence of cognitive impairment in older adults with type 2 diabetes (T2D) and to evaluate the effectiveness of a multidomain intervention designed to prevent cognitive…
- Conditions
- Diabetes, Diabetes Mellitus Type 2, Type 2 Diabetes, Type 2 Diabetes Mellitus (T2DM)
- Trial name
- NCT07250789
- Testing
- Multidomain Intervention Targeting Cognitive Decline
- Where
- 3 sites in Denmark
- Volunteers
- 420 people
- Sponsor
- Steno Diabetes Center Copenhagen
Sleep Apnea, Obstructive
Prescision Medicine in Obstructive Sleep Apnea, Cardiovascular and Cerebrovascular Disease: AI Electrocardiogram Patch Does it All
Obstructive Sleep apnea(OSA) is a disease with multiple causes, and treatments are very diverse. Patients and doctors have a lot of choices.
- Trial name
- NCT05566860
- Testing
- Positional Therapy, Intraoral Negative Pressure Therapy, Oral Appliances
- Where
- Taiwan
- Volunteers
- 80 people, healthy volunteers welcome
- Sponsor
- Shin Kong Wu Ho-Su Memorial Hospital
Hepatic Cirrhosis, Hepatic Encephalopathy (HE)
MNK6106 for Liver Disease (Hepatic Cirrhosis) That in the Past Has Affected the Brain (Hepatic Encephalopathy)
The main reason for this study is to see how the study drug interacts with the body. It will compare different doses of the study drug with a drug already in use.
- Trial name
- NCT03712280
- Testing
- MNK6106, Rifaximin
- Where
- 5 sites in United States, Puerto Rico
- Volunteers
- 50 people
- Sponsor
- Mallinckrodt
Cardiovascular Diseases, Heart Diseases
The Jackson Heart Study of Cardiovascular Disease Among African Americans
This is a prospective study of the environmental and genetic factors that influence the development of cardiovascular disease (CVD) in African American adults.
- Conditions
- Cardiovascular Diseases, Heart Diseases, Atherosclerosis, Coronary Disease
- Trial name
- JHS (NCT00005485)
- Where
- United States
- Volunteers
- 5,306 people, healthy volunteers welcome
- Sponsor
- National Heart, Lung, and Blood Institute (NHLBI)
Type 1 Diabetes, Hypoglycemia
Investigation Into the Effects Upon Brain Response to Change in Circulating Glucose Levels in Diabetes Mellitus
This study is designed to investigate the effects of diabetes mellitus and its treatment upon the body's responses to low blood glucose (blood sugar) levels.
- Trial name
- NCT00580710
- Where
- United States
- Volunteers
- 164 people, healthy volunteers welcome
- Sponsor
- Yale University
Lysosomal Storage Disorders
Molecular and Cellular Mechanisms of Lysosomal Storage Diseases
The lysosome is a specialized part of the cell that functions to degrade metabolic wastes in the cell. Defects in the functioning of the lysosome result in accumulation and subsequent storage of such metabolic wastes.
- Trial name
- NCT02000310
- Where
- United States
- Volunteers
- 80 people, healthy volunteers welcome
- Sponsor
- O & O Alpan LLC
Epilepsy
Use of the Atkins Diet for Children With Intractable Epilepsy: A Comparison of Daily Carbohydrate Limits
This study is designed to further investigate the use of a modified Atkins diet for children with intractable, daily seizures. It is a follow-up to a study recently completed by our institution.
- Trial name
- NCT00181090
- Testing
- Modified Atkins diet
- Where
- United States
- Volunteers
- 30 people
- Sponsor
- Johns Hopkins University
Migraine Disorders
Nutrition for Migraine Prevention
Migraine is a widespread, debilitating, chronic pain disorder and a major public health challenge. Most conventional, pharmaceutical treatments fail to give satisfactory long-term relief and their repeated use can have important…
- Trial name
- NCT02012790
- Testing
- Diet
- Where
- United States
- Volunteers
- 182 people
- Sponsor
- University of North Carolina, Chapel Hill
Alzheimer Disease, Parkinson Disease
P-glycoprotein Function in Brain Diseases
This study will measure the function of a protein called P-glycoprotein (P-gp), which is found at the blood-brain barrier, a membrane that normally prevents toxic material from entering the brain.
- Conditions
- Alzheimer Disease, Parkinson Disease, Frontotemporal Lobar Degeneration
- Trial name
- NCT00677885
- Where
- United States
- Volunteers
- 20 people
- Sponsor
- National Institute of Mental Health (NIMH)
Amyotrophic Lateral Sclerosis
Multimodal Brain Imaging Study
To explain the key brain network nodes and their brain mechanisms of ALS language cognitive impairment and decline, reveal the neural mechanism of the association between ALS language cognitive impairment and motor executive…
- Trial name
- NCT04960540
- Testing
- MRI,
- Where
- China
- Volunteers
- 40 people
- Sponsor
- Peking University Third Hospital
Gaucher's Disease
Effects of Enzyme Replacement in Gaucher's Disease
Gaucher disease is a lysosomal storage disease resulting from glycocerebroside accumulation in macrophages due to a genetic deficiency of the enzyme glucocerebrosidase.
- Trial name
- NCT00001289
- Where
- United States
- Volunteers
- 70 people
- Sponsor
- National Institute of Neurological Disorders and Stroke (NINDS)
Pompe Disease (Late-onset)
A Study About Antibody Levels and Biomarkers in the Blood in People With Late-onset Pompe Disease
Pompe disease is a genetic condition which causes muscle weakness over time. People with Pompe disease have a faulty gene that makes an enzyme called acid alpha-glucosidase (or GAA).
- Trial name
- NCT06150820
- Testing
- No Intervention
- Where
- 53 sites in United States, Australia and 9 more
- Volunteers
- 119 people
- Sponsor
- Astellas Gene Therapies
All Cause Mortality, Cardiovascular Diseases
Advice of Moderate Drinking Pattern Versus Advice on Abstention on Major Disease and Mortality
The goal of this clinical trial is to test two advices on alcohol drinking in more than 10.000 Spanish adult drinkers (men of 50 or more years and women of 55 or more years).
- Conditions
- All Cause Mortality, Cardiovascular Diseases, Invasive Cancer, Liver Cirrhosis
- Trial name
- UNATI (NCT06338215)
- Testing
- Moderate Alcohol Drinking Pattern, Abstention
- Where
- Spain
- Volunteers
- 10,000 people, healthy volunteers welcome
- Sponsor
- Clinica Universidad de Navarra, Universidad de Navarra
Mild Cognitive Impairment
Metformin in Amnestic Mild Cognitive Impairment
Hyperinsulinemia and type 2 diabetes (T2D) are important potential risk factors for cognitive decline and Alzheimer's disease (AD).
- Trial name
- MCI (NCT00620191)
- Testing
- Metformin
- Where
- United States
- Volunteers
- 80 people
- Sponsor
- Columbia University
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