Clinical trials: Brain Diseases, Metabolic
Fabry Disease
A 12-Week Safety and Pharmacodynamic Study of AT1001 (Migalastat Hydrochloride) in Participants With Fabry Disease
Study to evaluate the safety, tolerability, and pharmacodynamics of migalastat hydrochloride (HCl) (migalastat) and how migalastat works in participants with Fabry disease.
- Trial name
- NCT00283959
- Testing
- migalastat HCl
- Where
- 2 sites in Australia, Brazil
- Volunteers
- 4 people
- Sponsor
- Amicus Therapeutics
Infantile Neronal Ceroid Lipofuscinosis
Cystagon to Treat Infantile Neuronal Ceroid Lipofuscinosis
This study will examine the effectiveness of a drug called Cystagon in treating infantile neuronal ceroid lipofuscinosis (INCL), a progressive neurological disease affecting children.
- Trial name
- NCT00028262
- Testing
- Cystagon
- Where
- United States
- Volunteers
- 10 people
- Sponsor
- Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
Amyloidosis; Heart (Manifestation), Senile Cardiac Amyloidosis
Doxycycline and TUDCA in Patients With Transthyretin Amyloid Cardiomyopathy
The objective of the study is to determine whether the combination of the bile acid TUDCA, and doxycycline will slow the progression of familial and senile amyloidosis.
- Trial name
- NCT01855360
- Testing
- Tauroursodeoxycholic Acid and Doxycycline
- Where
- United States
- Volunteers
- 40 people
- Sponsor
- Brigham and Women's Hospital
Metabolism, Inborn Errors, Lipid Metabolism, Inborn Errors
Fat and Sugar Metabolism During Exercise in Patients With Metabolic Myopathy
This study aims to characterize the pathophysiological mechanisms of 21 different metabolic myopathies. The study will focus on exercise capacity and the metabolic derangement during exercise.
- Conditions
- Metabolism, Inborn Errors, Lipid Metabolism, Inborn Errors, Carbohydrate Metabolism, Inborn Errors, Long-Chain 3-Hydroxyacyl-CoA Dehydrogenase Deficiency
- Trial name
- NCT02635269
- Testing
- Sugar
- Where
- Denmark
- Volunteers
- 60 people, healthy volunteers welcome
- Sponsor
- Copenhagen University Hospital, Rigshospitalet
Pompe Disease
Newborn Screening Assay of Pompe's Disease
The purpose of this study is to test the feasibility of a newborn screen assay for Pompe disease
- Trial name
- NCT01758354
- Testing
- Pompe disease newborn screening
- Where
- Taiwan
- Volunteers
- 236,536 people, healthy volunteers welcome
- Sponsor
- National Taiwan University Hospital
Pompe Disease
High Risk Screen of Childhood Late-onset Pompe Disease in Pediatric Outpatient Clinics
Establish of high-risk screening criteria to earlier identify possible childhood LOPD for early treatment and better prognosis. Therefore, validation of the high-risk screening criteria for childhood LOPD will be critical for…
- Trial name
- NCT04981210
- Where
- Taiwan
- Volunteers
- 1,500 people
- Sponsor
- National Taiwan University Hospital
Brain Ischemia, Hyperglycemia
Glycemic Variability: Prognostic Impact on Acute Ischemic Stroke
This is a multicenter observational clinical study in patients with acute ischemic stroke. The main objective is to evaluate the impact of glycemic variability (GV) on stroke outcome (mortality, functional recovery) of patients…
- Conditions
- Brain Ischemia, Hyperglycemia, Glycemic Variability
- Trial name
- GLIAS-III (NCT04001049)
- Where
- Spain
- Volunteers
- 192 people
- Sponsor
- Instituto de Investigación Hospital Universitario La Paz
Niemann Pick Diseases
Saccadic Eye Movements in Patients With Niemann-Pick Type C Disease
This study is done in conjunction with a trial, conducted at Columbia University College of Physicians and Surgeons in New York and the Royal Manchester Children's Hospital in England, to examine the effectiveness of a new drug…
- Trial name
- NCT00316498
- Testing
- OGT918
- Where
- 3 sites in United States, United Kingdom
- Volunteers
- 30 people
- Sponsor
- National Eye Institute (NEI)
Prediabetes, Prediabetes (Insulin Resistance, Impaired Glucose Tolerance)
Peanuts for Cardiometabolic, Brain, and Intestinal Health
The overall objective of this 14-month randomized crossover study is to seek evidence demonstrating that daily consumption of peanuts and peanut products improve cardiometabolic, cognitive, and intestinal health in a racially…
- Conditions
- Prediabetes, Prediabetes (Insulin Resistance, Impaired Glucose Tolerance), Cognition, Microvascular Function
- Trial name
- NCT06867198
- Testing
- Peanuts
- Where
- United States
- Volunteers
- 72 people
- Sponsor
- Georgia State University
ALD (Adrenoleukodystrophy), Adrenoleukodystrophy
Adrenoleukodystrophy National Registry Study
The aim of this registry to understand the natural history and disease progression in ALD and potentially develop bio-markers using the biospecimens collected using this registry.
- Conditions
- ALD (Adrenoleukodystrophy), Adrenoleukodystrophy, Cerebral Adrenoleukodystrophy
- Trial name
- NCT03789721
- Testing
- Medical Record Abstraction, Biospecimen Sample Collection
- Where
- United States
- Volunteers
- 1,000 people
- Sponsor
- Masonic Cancer Center, University of Minnesota
Cardiovascular Diseases, Heart Diseases
Reducing Total Cardiovascular Risk in an Urban Community
PLEASE NOTE: THIS STUDY IS ONLY ENROLLING PATIENTS CURRENTLY BEING TREATED AT BELAIR-EDISON FAMILY HEALTH CENTER. The purpose of this study is to compare the clinical effectiveness and cost effectiveness of two cardiovascular…
- Conditions
- Cardiovascular Diseases, Heart Diseases, Coronary Disease, Diabetes Mellitus
- Trial name
- COACH (NCT00241904)
- Testing
- Lifestyle Changes, Antiplatelet Agents, Beta Blocker
- Where
- United States
- Volunteers
- 525 people
- Sponsor
- Johns Hopkins University
Cognitive Decline
Metabolic Syndrome, Inflammation, and Risk of Cognitive Decline
The purpose of this study is to determine if the metabolic syndrome is a risk factor for cognitive decline and if this association is modified by inflammation.
- Trial name
- NCT00099567
- Where
- 2 sites in United States
- Volunteers
- 2,632 people, healthy volunteers welcome
- Sponsor
- National Institute on Aging (NIA)
Phenylketonurias
Open-label, Randomized, 2-way Crossover, Monocentric, Controlled Study to Evaluate the Effect on Daily PHE Fluctuation of PKU GOLIKE Versus SoC in Patients With PKU.
This is an open-label, randomized, 2-way crossover, monocentric controlled study in patients (≥ 16 years old) with phenylketonuria (PKU).
- Trial name
- NCT05827536
- Testing
- PKU GOLIKE, Standard of Care
- Where
- Italy
- Volunteers
- 21 people
- Sponsor
- APR Applied Pharma Research s.a.
Coronary Artery Disease, Stroke
The SMART-ORACLE Study
After having had a first cardiovascular event, there is a considerable risk of developing a subsequent event. Only recently, a risk prediction model was developed for this group of patients.
- Conditions
- Coronary Artery Disease, Stroke, Peripheral Arterial Disease, Hypertension
- Trial name
- SMART-ORACLE (NCT01932671)
- Where
- Netherlands
- Volunteers
- 1,500 people
- Sponsor
- UMC Utrecht
Wilson Disease, Movement Disorders
Inhibitory rTMS in Dystonic Wilson Patients
Wilson disease is a genetic disorder resulting in copper accumulation in liver, brain and eye. The neurologic complications include dystonic syndrome, which is a prolonged and excessive muscle activation responsible for abnormal…
- Conditions
- Wilson Disease, Movement Disorders, Repetitive Transcranial Magnetic Stimulation
- Trial name
- WILSTIM (NCT01980433)
- Testing
- Repetitive Transcranial Magnetic Stimulation (rTMS), pre and post-rTMS electroencephalogram, WCRS
- Where
- France
- Volunteers
- 14 people
- Sponsor
- Assistance Publique - Hôpitaux de Paris
Brain and Central Nervous System Tumors, Chronic Myeloproliferative Disorders
Opioid Titration Order Sheet or Standard Care in Treating Patients With Cancer Pain
RATIONALE: An Opioid Titration Order Sheet that allows healthcare providers to adjust the dose and schedule of pain medication may help improve pain treatment for patients with cancer.
- Conditions
- Brain and Central Nervous System Tumors, Chronic Myeloproliferative Disorders, Leukemia, Lymphoma
- Trial name
- NCT00666211
- Testing
- educational intervention, Titrated pain management, questionnaire administration
- Where
- 11 sites in United States
- Volunteers
- 98 people
- Sponsor
- Vanderbilt-Ingram Cancer Center
Fabry Disease
Alternative Dosing and Regimen of Replagal to Treat Fabry Disease
The main goal of this study is to assess the pharmacodynamic effects of different or more frequent doses of Replagal compared to the standard dosing regimen.
- Trial name
- NCT00075244
- Testing
- Replagal
- Where
- United States
- Volunteers
- 25 people
- Sponsor
- National Institute of Neurological Disorders and Stroke (NINDS)
Primary Progressive Aphasia, Neurodegenerative Diseases
Home-Based Exercise in Primary Progressive Aphasia (HEPPA)
This pilot study aims to evaluate the feasibility of a novel home-based multicomponent exercise program in adults clinically diagnosed with Primary Progressive Aphasia
- Conditions
- Primary Progressive Aphasia, Neurodegenerative Diseases, Logopenic Progressive Aphasia, Semantic Dementia
- Trial name
- HEPPA (NCT06181500)
- Testing
- Home-based multicomponent exercise intervention program
- Where
- 2 sites in Canada
- Volunteers
- 13 people
- Sponsor
- Montreal Heart Institute
Growth Hormone Deficiency, Growth Hormone Treatment
Effects of Growth Hormone and IGF-1 on Anabolic Signals and Stem Cell Recruitment in Human Skeletal Muscle
12 adult hypopituitary patients with newly diagnosed Growth hormone (GH)-deficiency will be studied two times. The first examinations will be performed shortly after time of diagnose before initiation of exogenous GH treatment…
- Trial name
- NCT03878992
- Testing
- Genotropin miniquick 0.5 mg, injection
- Where
- Denmark
- Volunteers
- 12 people, healthy volunteers welcome
- Sponsor
- University of Aarhus
Cerebral Amyloid Angiopathy
Cortical Superficial Siderosis and Risk of Recurrent Intracerebral Hemorrhage in Cerebral Amyloid Angiopathy.
Cerebral amyloid angiopathy (CAA) is a major cause of lobar intracerebral hemorrhage (ICH) in the elderly with high risk of recurrence.
- Trial name
- CORELIA (NCT03464344)
- Testing
- neurological, neuropsychological and MRI evaluation
- Where
- 4 sites in France
- Volunteers
- 170 people
- Sponsor
- University Hospital, Toulouse
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