Clinical trials: Brain Diseases, Metabolic
Phenylketonuria, Galactosemia
The Early History of Universal Screening for Metabolic Disorders
We are doing this study to learn more about the early history of universal screening for metabolic disorders such as PKU and galactosemia.
- Conditions
- Phenylketonuria, Galactosemia, Inborn Errors of Metabolism
- Trial name
- NCT00309400
- Where
- United States
- Volunteers
- 10 people, healthy volunteers welcome
- Sponsor
- University of Miami
Obesity, Healthy Volunteers
Brain Dopamine Function in Human Obesity
Background: Dopamine is a natural chemical in the brain that may influence eating behavior and physical activity. Researchers want to measure the brain s dopamine activity and understand how it differs in people with obesity.
- Conditions
- Obesity, Healthy Volunteers, Overweight
- Trial name
- NCT03648892
- Testing
- [c11] raclopride, [18F]fallypride
- Where
- United States
- Volunteers
- 61 people, healthy volunteers welcome
- Sponsor
- National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)
Phenylketonuria
Safety, Tolerability, and Efficacy Study of rAvPAL-PEG Administered Daily in Subjects With Phenylketonuria (PKU)
The purpose of this study is to evaluate the effect of daily administration of rAvPAL-PEG on the reduction of blood Phe concentrations in subjects with PKU.
- Trial name
- NCT01212744
- Testing
- rAvPAL-PEG
- Where
- 9 sites in United States
- Volunteers
- 16 people
- Sponsor
- BioMarin Pharmaceutical
Hypercholesterolemia, Thrombosis
The Inhibition of Platelet Antiaggregating Activity of Clopidogrel by Atorvastatin Detected by Erythromycin Breath Test: a Metabolic Inhibition of Hepatic Cytochrome P450-3A
The objective of this study is to determine if the action of the drug called clopidogrel, that you will start taking, will be decreased by another drug called atorvastatin, that you will also start taking.
- Trial name
- NCT00004564
- Testing
- Clopidogrel, Atorvastatin
- Where
- United States
- Sponsor
- National Center for Research Resources (NCRR)
Pompe Disease
Frequency of Pompe Disease in Patients With Myalgia With or Without Hyper Ckemia - Data From the Reference Center (CERCA)
Pompe's disease is a lysosomal storage disease of autosomal recessive genetic transmission due to a deficiency in acid alpha glucosidase.
- Trial name
- POEM (NCT05092230)
- Where
- Martinique
- Volunteers
- 100 people
- Sponsor
- University Hospital Center of Martinique
Phenylketonuria
Phase 3 Open-label Study to Evaluate the Response and Safety of Kuvan® in Subjects With Phenylketonuria
This is an open-label, non-comparative, Phase 3 study to evaluate the degree, frequency of response and safety of Kuvan® (sapropterin dihydrochloride) in subjects aged 4 to 18 years who have phenylketonuria and with elevated…
- Trial name
- NCT01732471
- Testing
- Kuvan®
- Where
- Germany
- Volunteers
- 90 people
- Sponsor
- Merck KGaA, Darmstadt, Germany
Obesity, Self-Regulation
ABC Brain Games Self-Regulation Intervention
The goal of this project is to measure childhood self-regulation targets known to be associated with obesity risk and poor adherence to medical regimens and to assess whether intervening on these mechanisms can improve…
- Trial name
- NCT03060863
- Testing
- 2. Executive Functioning, 3. Food Bias, 4. Emotion Regulation
- Where
- United States
- Volunteers
- 246 people
- Sponsor
- University of Michigan
Healthy, Brain Insulin Sensitivity
Long-Term Effects of Walnut Consumption on Brain Function
Rationale: Healthy foods, including mixed nuts, may improve brain function, which is essential for cognitive and metabolic health, and may contribute to improved food intake regulation.
- Conditions
- Healthy, Brain Insulin Sensitivity, Cerebral Blood Flow, Brain Vascular Function
- Trial name
- WalBrain (NCT07422220)
- Testing
- Walnuts
- Where
- Netherlands
- Volunteers
- 55 people, healthy volunteers welcome
- Sponsor
- Maastricht University Medical Center
ALS (Amyotrophic Lateral Sclerosis), FTD
Disease Biosignatures in ALS/FTD Spectrum: New Impactful Biological Perspectives Beyond Clinical Approaches
Diagnosis of ALS/FTD disease spectrum is challenging because it largely relies on clinical symptoms. Identifying novel biomarkers is essential for a paradigm shift towards a more precise biological-based diagnosis.
- Conditions
- ALS (Amyotrophic Lateral Sclerosis), FTD, Neuropathic, Psychiatric Disorders
- Trial name
- SPECTRALS (NCT06856850)
- Where
- 4 sites in Italy
- Volunteers
- 230 people
- Sponsor
- Fondazione I.R.C.C.S. Istituto Neurologico Carlo Besta
Hereditary Tyrosinemia, Type I
Hereditary Hepatorenal Tyrosinemia Natural History in Egypt and the Arab World (Multicenter Clinical Study)
The purpose of the registry/repository is to understand the natural history of tyrosinemia in our region and to provide a mechanism to store data and specimens to support the conduct of future research about hereditary…
- Trial name
- NCT03446586
- Where
- Egypt
- Volunteers
- 50 people
- Sponsor
- Yassin Abdelghaffar Charity Center for Liver Disease and Research
Alzheimer s Disease
Terazosin And Metabolic Engagement in Alzheimer's Disease
The TAME-AD trial will be a randomized, double-blind, placebo-controlled Phase IIa clinical trial to evaluate the metabolic target engagement of terazosin for the treatment of Alzheimer's disease.
- Trial name
- TAME-AD (NCT07796308)
- Testing
- Terazosin (Hytrin)
- Where
- United States
- Volunteers
- 100 people
- Sponsor
- Qiang Zhang
Hepatic Encephalopathy
MNK-6105 for Patients With Cirrhosis and High Ammonia Levels Affecting Brain Function
This study is for patients with cirrhosis and hepatic encephalopathy who are in the hospital. This means they have a high ammonia level which is affecting their brain function.
- Trial name
- NCT04128462
- Testing
- MNK-6105, Standard of Care
- Sponsor
- Mallinckrodt
Parkinson Disease, Prodromal Stage
Slow-SPEED: Slowing Parkinson's Early Through Exercise Dosage
The goal of this clinical trial is two-fold. First to investigate the feasibility of whether a remotely administered smartphone app can increase the volume and intensity of physical activity in daily life in individuals with a…
- Conditions
- Parkinson Disease, Prodromal Stage, Neurodegenerative Diseases, Basal Ganglia Diseases
- Trial name
- Slow-SPEED (NCT06993142)
- Testing
- Increase of physical activity volume and intensity with the use of a motivational smartphone application
- Where
- 2 sites in United States, Netherlands
- Volunteers
- 600 people
- Sponsor
- Radboud University Medical Center
Friedreich Ataxia
A Single Ascending Dose Study of DT-216P2 in Normal Healthy Participants
This purpose of the study is to evaluate the safety, tolerability and pharmacokinetics of single ascending doses of DT-216P2 administered either subcutaneously (SC) and intravenously (IV) in normal healthy participants.
- Trial name
- NCT06772870
- Testing
- DT-216P2, Saline
- Where
- Australia
- Volunteers
- 36 people, healthy volunteers welcome
- Sponsor
- Design Therapeutics, Inc.
Ischemic Stroke, Hyperglycemia
Assessment of Markers of Stress in Acute Ischemic Stroke With Hyperglycemia
Hyperglycemia is present in 50 percent of patients with acute ischemic stroke. Patients with hyperglycemia have higher in hospital, 30 and 90 day mortality and morbidity.
- Trial name
- ASIIST (NCT03318679)
- Testing
- No intervention is planned.
- Where
- United States
- Volunteers
- 88 people
- Sponsor
- University of Minnesota
Ornithine Transcarbamylase Deficiency, Ornithine Transcarbamylase Deficiency Disease
An Open-label Study to Investigate ECUR-506 in Male Babies Less Than 9 Months of Age With Neonatal Onset OTC Deficiency
Ornithine Transcarbamylase (OTC) deficiency, the most common urea cycle disorder, is an inherited metabolic disorder caused by a genetic defect in a liver enzyme responsible for detoxifying of ammonia.
- Conditions
- Ornithine Transcarbamylase Deficiency, Ornithine Transcarbamylase Deficiency Disease, Ornithine Carbamoyltransferase Deficiency (Disorder), Urea Cycle Disorders, Inborn
- Trial name
- NCT06255782
- Testing
- ECUR-506
- Where
- 12 sites in United States, Australia and 2 more
- Volunteers
- 20 people
- Sponsor
- iECURE, Inc.
Alzheimer Disease
Does EVOO Induce Gene and Metabolic Changes in Healthy Subjects
Investigators' recent findings from the pilot clinical trial in MCI individuals demonstrated EVOO improved vascular function and memory.
- Trial name
- NCT05929924
- Testing
- Extra virgin olive oil
- Where
- United States
- Volunteers
- 39 people, healthy volunteers welcome
- Sponsor
- Augusta University
Urea Cycle Disorders
Efficacy and Safety of HPN-100 for the Treatment of Adults With Urea Cycle Disorders
This was a randomized, active-controlled, double-blind, cross-over study designed to enroll subjects with UCDs who are being treated with NaPBA.
- Trial name
- NCT00992459
- Testing
- HPN-100, Buphenyl (NaPBA)
- Where
- 22 sites in United States, Canada
- Volunteers
- 46 people
- Sponsor
- Amgen
Diabetes Mellitus, Type 1, Epilepsy
Impact of Parental Perfectionistic Cognitions Self-compassion Intervention Effects on Shame in Child Health Context
Parents of children with long-term health conditions (LTCs) can experience shame related to parenting. Whilst self-compassion interventions (SCIs) can reduce parental shame, this has not been studied with parents of children with…
- Conditions
- Diabetes Mellitus, Type 1, Epilepsy, Asthma, Shame
- Trial name
- NCT03504605
- Testing
- Online self-compassion intervention
- Where
- United Kingdom
- Volunteers
- 344 people, healthy volunteers welcome
- Sponsor
- University of Sheffield
Phenylketonuria
A Phase 3 Substudy to Evaluate Executive Function in Adults With PKU Who Are Participating in the Phase 3 Study, 165-302
A Phase 3 substudy to evaluate executive function in adults with phenylketonuria who are participating in the phase 3 Study, 165-302. Approximately 100 subjects, aged ≥ 18 to ≤ 70 years old, with PKU will be enrolled.
- Trial name
- PRISM303 (NCT02468570)
- Testing
- Administration of CANTAB and Subject Global Assessment
- Where
- 4 sites in United States
- Volunteers
- 9 people
- Sponsor
- BioMarin Pharmaceutical
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