Clinical trials: Brain Diseases, Metabolic
Pelizaeus-Merzbacher Disease
Rocket Study: A Study to Characterize Biomarkers and Disease Progression in Participants With Pelizaeus-Merzbacher Disease
The purpose of the study is to prospectively assess longitudinal changes in proteolipid protein 1 (PLP1) protein, disease-related biomarkers in cerebral spinal fluid (CSF) and blood, neuroimaging parameters relevant to…
- Trial name
- NCT05659901
- Where
- 10 sites in United States, France and 5 more
- Volunteers
- 32 people
- Sponsor
- Ionis Pharmaceuticals, Inc.
Growth Hormone Deficiency
304-C03/L3301n: Effects of Growth Hormone on Glucose and Protein Metabolism in Children With Growth Hormone Deficiency
The purpose of the proposed study is to investigate the effects of rhGH treatment on glucose, protein and fat metabolism in GHD children.
- Trial name
- NCT00362063
- Testing
- growth hormone (Nutropin)
- Where
- United States
- Volunteers
- 20 people, healthy volunteers welcome
- Sponsor
- Baylor College of Medicine
Obesity
Effects of Macronutrient Diet on Brain Activity
This study will evaluate the effect of macronutrient diets on brain activity in homeostatic and mesolimbic reward regions.
- Trial name
- NCT02300857
- Testing
- Low carbohydrate diet, Moderate carbohydrate diet, High carbohydrate diet
- Volunteers
- 72 people, healthy volunteers welcome
- Sponsor
- Brigham and Women's Hospital
Fabry Disease
Open-label Long-term Safety Study of AT1001 (Migalastat Hydrochloride) in Participants With Fabry Disease Who Have Completed a Previous AT1001 Study
Study to evaluate the long-term safety, tolerability, and pharmacodynamics (PD) of migalastat hydrochloride (HCl) (migalastat) in participants with Fabry disease
- Trial name
- NCT00526071
- Testing
- migalastat HCl
- Where
- 8 sites in United States, Australia and 3 more
- Volunteers
- 23 people
- Sponsor
- Amicus Therapeutics
Ataxia Telangiectasia, ATM Gene Mutation
Effects of Vitamin B3 in Patients With Ataxia Telangiectasia
This clinical trial investigates the effects of nicotinamide riboside (vitamin B3) on the disease course of patients with ataxia telangiectasia.
- Trial name
- NCT03962114
- Testing
- Vitamin B3
- Where
- Netherlands
- Volunteers
- 24 people
- Sponsor
- Radboud University Medical Center
Fabry Disease
A Study of the Safety and Efficacy of Fabrazyme in Patients With Fabry Disease
People with Fabry disease have an alteration in their genetic material (DNA) which causes a deficiency of the a-galactosidase A enzyme.
- Trial name
- NCT00074971
- Testing
- Fabrazyme (agalsidase beta)
- Where
- 20 sites in United States, France and 3 more
- Volunteers
- 58 people
- Sponsor
- Genzyme, a Sanofi Company
Hepatic Encephalopathy, Cirrhosis
The Safety/Efficacy of Rifaximin With/Without Lactulose in Participants With A History of Recurrent Hepatic Encephalopathy
The purpose of the study is to evaluate if rifaximin alone or rifaximin plus lactulose delays the onset of hepatic encephalopathy (HE) in participants with cirrhosis who have had a previous episode of HE.
- Trial name
- NCT01842581
- Testing
- Rifaximin, Lactulose
- Where
- 43 sites in United States
- Volunteers
- 222 people
- Sponsor
- Bausch Health Americas, Inc.
Celiac Disease, Cerebellar Ataxia
Gluten-Free Diet in Patients With Gluten Sensitivity and Cerebellar Ataxia
This study will screen patients with cerebellar ataxia to check for antibodies that indicate allergy to gluten (wheat protein) and will study the effect of a gluten-free diet in patients with these antibodies.
- Conditions
- Celiac Disease, Cerebellar Ataxia, Healthy
- Trial name
- NCT00006492
- Where
- United States
- Volunteers
- 150 people, healthy volunteers welcome
- Sponsor
- National Institute of Neurological Disorders and Stroke (NINDS)
Cardiovascular Diseases, Myocardial Ischemia
Cohort Study of Heart Rate Variability
To examine factors affecting heart rate variability (HRV) and the role of HRV in heart disease. Specifically, to examine the role of HRV: as a predictor of fatal and nonfatal ischemic heart disease over a six year follow-up of…
- Conditions
- Cardiovascular Diseases, Myocardial Ischemia, Carotid Stenosis, Hypertension
- Trial name
- NCT00005399
- Testing
- No interventions
- Volunteers
- 111,654 people
- Sponsor
- University of North Carolina, Chapel Hill
Mitochondrial Encephalomyopathies, Hereditary Spastic Paraplegia
Natural History Study of Patients with HPDL Mutations
This study uses medical records that allow retrospective data extraction of clinical manifestation to assess the natural history of HPDL mutations
- Conditions
- Mitochondrial Encephalomyopathies, Hereditary Spastic Paraplegia, Spastic Paraplegia, White Matter Disease
- Trial name
- NCT05848271
- Testing
- Patient Registry, Dry blood spots sampling
- Where
- United States
- Volunteers
- 50 people
- Sponsor
- University of California, San Diego
Uveitis, Posterior
Role of OCT & OCT Angiography in Patients With Posterior at the Uveitis Clinic of Assiut University Hospital.
OCT angiography is a recent evaluation of OCT technology which combines the structural assessment of ocular tissues obtained by OCT image with visualization of blood flow within the vessels in imaged area.
- Trial name
- NCT06743906
- Volunteers
- 25 people
- Sponsor
- Assiut University
Phenylketonuria, Hyperphenylalaninaemia
PKUDOS: Phenylketonuria (PKU) Demographic, Outcomes, and Safety Registry
The objective of this study is to evaluate the safety of long-term treatment with Kuvan.
- Trial name
- PKUDOS (NCT00778206)
- Testing
- Kuvan
- Where
- 45 sites in United States
- Volunteers
- 1,887 people
- Sponsor
- BioMarin Pharmaceutical
Diabetes Mellitus, Type 2, Diabetes Mellitus Type 2 in Obese
From Skin Fibroblasts to Neural Stem Cells to Investigate in Vitro the Impact of Diabetes on Adult Neurogenesis
Obesity and glucose intolerance or overt diabetes are increasing at an alarming rate in the population, and are bound to become a public health issue and a major cause of disability, loss of independence and high social costs in…
- Conditions
- Diabetes Mellitus, Type 2, Diabetes Mellitus Type 2 in Obese, Diabetes Mellitus Type 2 in Nonobese, Obesity
- Trial name
- NCT05755321
- Testing
- Skin biopsy
- Where
- Italy
- Volunteers
- 40 people, healthy volunteers welcome
- Sponsor
- Fondazione Policlinico Universitario Agostino Gemelli IRCCS
Hereditary Tyrosinemia, Type I
Efficacy and Safety of Once Daily Dosing Compared to Twice Daily Dosing of Nitisinone in HT-1
The purpose of this study is to look at the steady-state serum concentrations of nitisinone when switching from twice daily and once daily dosing.
- Trial name
- HT-1 (NCT02323529)
- Testing
- Nitisinone
- Where
- 6 sites in Belgium, Denmark and 3 more
- Volunteers
- 18 people
- Sponsor
- Swedish Orphan Biovitrum
Liver Cirrhosis
Fructans, Lactitol and Lactose in Minimal Hepatic Encephalopathy
As an alternative for the treatment of Minimal Hepatic Encephalopathy (MHE) Agave fructans have shown prebiotic effects, and have shown to improve function of the digestive system, control and induce glycemic effect satiety.
- Trial name
- NCT02022137
- Testing
- Red cereal bar, Green cereal bar, White cereal bar
- Where
- Mexico
- Volunteers
- 30 people
- Sponsor
- Instituto Nacional de Ciencias Medicas y Nutricion Salvador Zubiran
Phenylketonurias, Non Alcoholic Fatty Liver
Walking Program in Fatty Liver Children With Phenylketonuria
phenylketonuria (commonly known as PKU) is an inherited disorder that increases the levels of a substance called phenylalanine in the blood.
- Trial name
- NCT06718842
- Testing
- walking free online supervised program
- Where
- Egypt
- Volunteers
- 40 people
- Sponsor
- Cairo University
Amyotrophic Lateral Sclerosis
Compassionate Treatment : An Exploratory Clinical Trial to Assess Treatment of Amyotrophic Lateral Sclerosis
The purpose of the study is to determine the safety and possible effectiveness of Autologous Adipose-Tissue Derived Stem Cells treatment in a Amyotrophic Lateral Sclerosis(ALS) patient.
- Trial name
- NCT02383654
- Testing
- Autologous Adipose-Tissue Derived Stem Cells
- Where
- Taiwan
- Volunteers
- 1 people
- Sponsor
- China Medical University Hospital
Parkinson Disease, Multiple System Atrophy
Treatment of Parkinson Disease and Multiple System Atrophy Using Intranasal Insulin.
Parkinson disease (PD) and multiple system atrophy (MSA) are progressive neurodegenerative disorders characterized by abnormal accumulation of α-synuclein.
- Trial name
- NCT02064166
- Testing
- Intranasal Insulin
- Where
- United States
- Volunteers
- 15 people
- Sponsor
- Peter Novak
Niemann-Pick Diseases
Health Insurance Literacy and Challenges in Accessing Health Services in Niemann-Pick
This study is a US based qualitative PRO research study to document the health insurance literacy as well as the patient experience in Niemann-Pick as it relates to accessing desired care, services and medications for patients.
- Trial name
- NCT04469894
- Where
- United States
- Volunteers
- 76 people
- Sponsor
- National Niemann-Pick Disease Foundation
Epilepsy
PET/MRI/EEG Imaging Study in Epilepsy
This study aims to better understand how epilepsy alters brain function through neuroimaging of healthy people and people with epilepsy.
- Trial name
- NCT06974305
- Testing
- FDG-PET/fMRI/EEG imaging
- Where
- United States
- Volunteers
- 30 people, healthy volunteers welcome
- Sponsor
- Massachusetts General Hospital
The first 1,020 results can be paged through. Add a filter or change the sort order to reach the rest.