Clinical trials: Brain Diseases, Metabolic
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
Cardiovascular, Diabetes
Home-Based Walking Study in Older Adults With Type 2 Diabetes
Older persons with diabetes have a harder time maintaining blood pressure when standing up. When blood pressure drops when standing up, fainting may occur.
- Conditions
- Cardiovascular, Diabetes, Orthostatic Hypotension, Type 2 Diabetes
- Trial name
- NCT00824330
- Testing
- Control Phase; Exercise Phase
- Where
- 2 sites in Canada
- Volunteers
- 13 people
- Sponsor
- University of British Columbia
Depression, Pain
Efficacy of Pain Treatment on Depression in Patients With Dementia
The purpose of this study is to determine whether pain treatment can reduce symptoms of depression in patients suffering from dementia and depression. Depression is commonly diagnosed in patients with dementia.
- Conditions
- Depression, Pain, Dementia
- Trial name
- DEP-PAIN-DEM (NCT02267057)
- Testing
- Paracetamol, Buprenorphine
- Where
- Norway
- Volunteers
- 163 people
- Sponsor
- University of Bergen
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
Gaucher Disease
Genzyme Osteopenia/Osteoporosis Study
Gaucher disease is a most common genetic metabolic disease characterized by low platelet number, liver and spleen enlargement and various forms of bone diseases including low bone mineral density leading to brittle bones.
- Trial name
- NCT02785744
- Testing
- Gaucher disease DNA mutation analysis
- Where
- United States
- Volunteers
- 76 people
- Sponsor
- NYU Langone Health
Urea Cycle Disorder, Ornithine Transcarbamylase Deficiency
Liver Disease in Urea Cycle Disorders
This is a multi-center, cross-sectional study to assess risk for liver fibrosis and hepatic injury in individuals with urea cycle disorders (UCDs) using serum biomarkers, Fibroscan, and MRE.
- Conditions
- Urea Cycle Disorder, Ornithine Transcarbamylase Deficiency, Citrullinemia 1, ARGI Deficiency
- Trial name
- NCT04612764
- Where
- 5 sites in United States
- Volunteers
- 62 people
- Sponsor
- Baylor College of Medicine
Amyotrophic Lateral Sclerosis
Restore Motor Function Through Robotic Arm Exoskeleton and Brain Computer Interface
The current project will aid patients with motor impairment to reduce the need for homecare. Specifically the aim is to develop and implement a robotic exoskeleton and brain computer interface to assist and eventually perform arm…
- Trial name
- REMAP (NCT03900247)
- Testing
- REMAP EEG based BCI and SEM-Glove BioServo
- Where
- Denmark
- Volunteers
- 15 people
- Sponsor
- University of Aarhus
Wilson's Disease, Myocardial Dysfunction
Subclinical Myocardial Dysfunction in Children With Wilson's Disease
Wilson's disease (WD) is one of the most common metabolic liver diseases in older children. The most frequent clinical presentation is liver disease. However, Wilson's disease (WD) is a multisystem disorder.
- Conditions
- Wilson's Disease, Myocardial Dysfunction, Children, Wilson Disease
- Trial name
- NCT07765472
- Testing
- Echocardiography, Electrocardiography, N-terminal pro b- type natriuretic peptide
- Where
- 2 sites in Egypt
- Volunteers
- 72 people, healthy volunteers welcome
- Sponsor
- Hebatullah Fawzy
Electrolyte Disturbances, Subarachnoid Hemorrhage
Electrolyte and Fluid Disturbances in Subarachnoid Hemorrhage and Traumatic Brain Injury
During the course of their acute illness patients with subarachnoid hemorrhage and severe traumatic brain injury often develop disturbances in their fluid balance and electrolyte homeostasis.
- Conditions
- Electrolyte Disturbances, Subarachnoid Hemorrhage, Traumatic Brain Injury, Natriuretic Peptides
- Trial name
- NCT01313975
- Where
- Switzerland
- Volunteers
- 85 people
- Sponsor
- Insel Gruppe AG, University Hospital Bern
Gaucher Disease, Splenomegaly
A National Study in Patients With Unexplained Splenomegaly
Primary Objective: To estimate the prevalence of Gaucher disease and of other etiologies, in patients of 15 years old or more presenting with unexplained splenomegaly after exclusion of first intention-diagnoses (e.g.
- Trial name
- SMS (NCT04430881)
- Where
- France
- Volunteers
- 506 people
- Sponsor
- Sanofi
Phenylketonuria
A Study to Evaluate Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of AG-181 in Subjects With Phenylketonuria
The primary purpose of this study is to assess the safety and tolerability of AG-181 in subjects with Phenylketonuria (PKU).
- Trial name
- NCT07241234
- Testing
- AG-181
- Where
- 5 sites in United States, Poland
- Volunteers
- 20 people
- Sponsor
- Agios Pharmaceuticals, Inc.
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
Hepatic Encephalopathy, Acute-On-Chronic Liver Failure
PEG3350 in ACLF With Hepatic Encephalopathy
it is a single blind randomised control study which aims to study the effect of PEG3350 in resolution of overt hepatic encephalopathy in patients of acute on chronic liver failure.
- Trial name
- NCT03987893
- Testing
- PEG-3350 with Electolytes, Lactulose
- Where
- India
- Volunteers
- 60 people
- Sponsor
- Post Graduate Institute of Medical Education and Research, Chandigarh
Adrenomyeloneuropathy, Adrenoleukodystrophy
Plasma Exchange With Albumin in AMN Patients
Adrenoleukodystrophy (X-ALD) is the most common genetic disorder of the brain white matter with an incidence of 1:14,700 births.
- Trial name
- NCT04303416
- Testing
- Albumin solution
- Where
- Spain
- Volunteers
- 5 people
- Sponsor
- Pujol, Aurora, M.D.
Esophageal Cancer
Outside the Cage (OTC) Robotic Esophagectomy
The goal of this clinical trial is to evaluate the feasibility and safety of a novel non-intercostal robotic approach for minimally invasive esophagectomy (OTC MIE) in the treatment of esophageal cancer.
- Trial name
- NCT06735638
- Testing
- OTC MIE Esophagectomy
- Where
- Canada
- Volunteers
- 31 people
- Sponsor
- Centre hospitalier de l'Université de Montréal (CHUM)
Tauopathies
Study of Biodistribution, Metabolism, Excretion and Brain Uptake11C-M503
The current protocol is to determine the biodistribution, metabolism, excretion and brain uptake of 11C-M503. The goal of this radiotracer is to quantify alpha-synuclein that is abnormally deposited in the brain of people with…
- Trial name
- NCT06303921
- Testing
- 11C-M503 PET, Brain MRI, Amyloid PET
- Where
- 3 sites in United States
- Volunteers
- 70 people, healthy volunteers welcome
- Sponsor
- University of Pennsylvania
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
Bardet Biedl Syndrome (BBS)
Bardet Beidle Syndrome in a Syrian Adolescent : a Rare Case Report
Bardet-Biedl Syndrome (BBS) is an uncommon genetic disorder that affects multiple organs. and presents with a variety of characteristics. It is caused by a dysfunction in the cilia.
- Trial name
- AlBaathU (NCT06615011)
- Volunteers
- 100 people
- Sponsor
- Al Baath University
Ischemic Stroke
hsCRP in Japan Statin Treatment Against Recurrent Stroke (J-STARS hsCRP)
Inhibition of 3-hydroxy-3-methylglutaryl-coenzyme A(HMG-CoA) reductase can play a role in preventing recurrent vascular events from ischemic heart disease patients, whose mechanism consists in not only the reduction of serum…
- Trial name
- NCT00361699
- Testing
- Statin
- Where
- 2 sites in Japan
- Volunteers
- 1,095 people
- Sponsor
- Translational Research Center for Medical Innovation, Kobe, Hyogo, Japan
Homocystinuria
Safety, Tolerability and Pharmacodynamics of SYNB1353 in Healthy Adult Volunteers
This is a Phase 1, double-blind (Sponsor-open), placebo-controlled, randomized, dose-escalation, inpatient study using a multiple-ascending dose (MAD) design to assess the safety, tolerability, and PD of SYNB1353 in HVs.
- Trial name
- HCU (NCT05462132)
- Testing
- SYNB1353
- Where
- United States
- Volunteers
- 31 people, healthy volunteers welcome
- Sponsor
- Synlogic