Clinical trials: Brain Diseases, Metabolic
PKU, Hyperphenylalaninemia
Study of a National Cohort of Adult Patients With Phenylketonuria
Phenylketonuria (PKU) is a metabolic disease of genetic origin. This is a rare disease (incidence 1 / 16000 births) which is the subject of a systematic neonatal screening in France, because it is treatable by a diet low in…
- Trial name
- ECOPHEN (NCT01619722)
- Where
- 16 sites in France
- Volunteers
- 220 people
- Sponsor
- University Hospital, Tours
Propionic/Methylmalonic Acidemias, Maple Syrup Urine Disease
Selective Screening of Children for Hereditary Metabolic Diseases by Tandem Mass Spectrometry in Kazakhstan
Inborn errors of metabolism (IEM) are not have specific clinical signs, they masquerade as other diseases, and are difficult to diagnose using only clinical manifestations or routine laboratory tests.
- Conditions
- Propionic/Methylmalonic Acidemias, Maple Syrup Urine Disease, Citrullinemia, Argininosuccinic Aciduria
- Trial name
- NCT05910151
- Testing
- Obtaining Dry Blood Spots From Healthy Newborns (Aged 1-7 Days), Obtaining Dry Blood Spots From Healthy Children Aged 8 Days - 7 Years, Obtaining Dry Blood Spots From Healthy Children Aged 8-18 Years
- Where
- Kazakhstan
- Volunteers
- 2,250 people, healthy volunteers welcome
- Sponsor
- West Kazakhstan Medical University
Vitamin D Deficiency, Carotid Artery Diseases
Vitamin D Deficiency as Independent Predictor for Plaque Vulnerability
Role of vitamin D in cardiovascular disease is controversial. Here the investigators aimed to investigate the association of vitamin D deficiency and carotid plaque morphology as well as outcome of participants with high-grade…
- Trial name
- NCT06473337
- Testing
- Blood sample
- Where
- Austria
- Volunteers
- 332 people
- Sponsor
- Medical University of Vienna
Overweight, Obesity
Transcutaneous Auricular Vagus Nerve Stimulation Combined With Lifestyle Intervention for Visceral Fat Reduction in Adults With Overweight or Obesity
This is a single-center, randomized, open-label controlled trial with blinded outcome assessment and data analysis. Fifty adults with overweight or obesity (body mass index [BMI] >= 24 kg/m2) and a sedentary lifestyle will be…
- Trial name
- taVNS (NCT07907029)
- Testing
- transcutaneous auricular vagus nerve stimulation (taVNS), Standardized lifestyle program (calorie-restricted diet plus aerobic exercise)
- Where
- China
- Volunteers
- 50 people
- Sponsor
- Guangdong Provincial Hospital of Traditional Chinese Medicine
Alzheimer's Disease
AC-1204 Long-term Efficacy Response Trial (ALERT Protocol)
The study will evaluate the safety \& efficacy of AC-1204, a ketogenic compound, administered orally on a daily basis for 6 months.
- Trial name
- ALERT (NCT01211782)
- Testing
- caprylic triglyceride, long-chain triglyceride
- Where
- United States
- Sponsor
- Cerecin
Inherited Metabolic Disorders (IMD)
MGTA-456 in Patients With Inherited Metabolic Disorders Undergoing Hematopoietic Stem Cell Transplantation (HSCT)
This study is designed to assess the safety and efficacy of using MGTA-456 in patients with Inherited Metabolic Disorders (IMD) undergoing stem cell transplantation.
- Trial name
- NCT03406962
- Testing
- MGTA-456
- Where
- 2 sites in United States
- Volunteers
- 8 people
- Sponsor
- Magenta Therapeutics, Inc.
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
Obesity
Involvement of the Gut Microbiota-brain Cross-talk in the Loss of Eating Control
Overweight and obesity are increasingly prevalent worldwide. These bodyweight disorders are closely related to deficiencies in the control of food intake.
- Trial name
- GMBTalk-Food (NCT05646901)
- Where
- Spain
- Volunteers
- 116 people, healthy volunteers welcome
- Sponsor
- Institut d'Investigació Biomèdica de Girona Dr. Josep Trueta
Brain and Central Nervous System Tumors, Chronic Myeloproliferative Disorders
Perceptions of Burden in Patients With Late-Stage Cancer and Their Caregivers
RATIONALE: Gathering information over time about patients' sense of being a burden on their caregiver, and caregivers' sense of burden on themselves, may help doctors learn more about the desire to die in patients with late-stage…
- Conditions
- Brain and Central Nervous System Tumors, Chronic Myeloproliferative Disorders, Depression, Leukemia
- Trial name
- NCT00770419
- Testing
- medical chart review, questionnaire administration, psychosocial assessment and care
- Sponsor
- Rutgers, The State University of New Jersey
Fabry Disease, Lysosomal Storage Diseases
A Long Term Follow-Up Study of Fabry Disease Subjects Treated With FLT190
Fabry disease is a rare, X-linked inborn error of glycosphingolipid metabolism caused by an abnormal gene encoding the α-galactosidase A (αGLA) enzyme.
- Trial name
- NCT04455230
- Testing
- FLT190
- Where
- 2 sites in Germany, United Kingdom
- Volunteers
- 3 people
- Sponsor
- Spur Therapeutics
Friedreich Ataxia
Efficacy Study of Epoetin Alfa in Friedreich Ataxia
Friedreich's ataxia (FRDA) is a rare genetic disorder characterised by severe neurological disability and cardiomyopathy. Friedreich's ataxia is the consequence of frataxin deficiency.
- Trial name
- FRIEMAX (NCT01493973)
- Testing
- Epoetin alfa
- Where
- 3 sites in Italy
- Volunteers
- 56 people
- Sponsor
- Federico II University
Frontotemporal Dementia
Serotonergic Function and Behavioural and Psychological Symptoms of Frontotemporal Dementia
Frontotemporal lobar degeneration(FTLD) is a common cause of early-onset dementia. FTLD is characterized multiple behavioral symptoms including mental rigidity, irritability, emotional blunting, disinhibition, apathy, and…
- Trial name
- NCT00376051
- Testing
- Citalopram
- Where
- 2 sites in Canada
- Volunteers
- 22 people
- Sponsor
- Sunnybrook Health Sciences Centre
Osteoporosis, Cerebral Palsy
Use of Oral Bisphosphonates in the Treatment of Osteoporosis of Non-walking Children With Cerebral Palsy
It is a double blind randomized study aiming at estimating the efficiency of oral bisphosphonates on the decrease of osteoporosis by comparing the evolution of densitometric values between two groups of children (treatment versus…
- Trial name
- NCT00822029
- Testing
- FOSAMAX
- Where
- France
- Volunteers
- 2 people
- Sponsor
- Department of Clinical Research and Innovation
Hepatic Encephalopathy
Deprescribing Proton Pump Inhibitors to Reduce Post-TIPS Hepatic Encephalopathy
A total of 40 patients taking proton pump inhibitors (PPIs) who undergo transjugular intrahepatic portosystemic shunt (TIPS) creation as part of routine clinical care will be randomized in 1:1 fashion to either continue or…
- Trial name
- NCT05070351
- Testing
- PPI deprescribing
- Where
- United States
- Volunteers
- 33 people
- Sponsor
- Duke University
Mindfulness Based Stress Reduction, Caregiver Burnout
Mindfulness-Based Stress Reduction in Dementia Caregivers
Aims: The study will contribute to our understanding of how the cultivation of caregiver mindfulness might improve their overall relational well-being (Primary Outcome), their psychological well-being (Secondary Outcome), and…
- Conditions
- Mindfulness Based Stress Reduction, Caregiver Burnout, Caregiver Stress Syndrome, Dementia
- Trial name
- MBSR (NCT04977245)
- Testing
- Mindfulness-Based Stress Reduction, Self-Guided Mindfulness eCourse
- Where
- United States
- Volunteers
- 40 people, healthy volunteers welcome
- Sponsor
- University of California, San Francisco
Lysosomal Storage Diseases, Inborn Errors of Metabolism
Fetal Umbilical Cord Blood (UCB) Transplant for Lysosomal Storage Diseases
The purpose of this study is to determine if it is safe to administer unrelated umbilical cord blood to pregnant women in their first trimester of pregnancy with a fetus that has a known diagnosis of certain lysosomal storage…
- Trial name
- IUHST-001 (NCT01003912)
- Testing
- ALD-601
- Where
- 2 sites in United States
- Sponsor
- Joanne Kurtzberg, MD
Arteriosclerosis, Atherosclerosis
Early Detection of Arteriosclerosis
Arteriosclerosis is a common chronic disease with well known risk factors like diabetes mellitus, hypertension, dyslipidemia, adipositas and smoking, leading to severe complications like myocardial infarction, stroke or…
- Conditions
- Arteriosclerosis, Atherosclerosis, Myocardial Infarction, Stroke
- Trial name
- NCT00863967
- Volunteers
- 269 people
- Sponsor
- Kantonsspital Baselland Bruderholz
Type 2 Diabetes
Chronotherapy with Low-dose Aspirin for Primary Prevention
Brief summary: Aspirin (ASA) has been shown to provide marked benefits in primary and secondary prevention of cardiovascular events.
- Trial name
- CARING (NCT00725127)
- Testing
- aspirin
- Where
- 19 sites in Spain
- Volunteers
- 3,200 people, healthy volunteers welcome
- Sponsor
- University of Vigo
Obesity, Gastrointestinal Microbiome
Study on the Mechanism of Regulating Gut Microbiome in Obese People by Regulating Brain-gut Axis With Fast Diet
1. Normal diet：Starting from May 20, 2018, the subjects will eat a normal diet for 4 days, with no restriction on calories and food types. The subjects are required to record their daily diet and dietary intake of calories. 2.
- Conditions
- Obesity, Gastrointestinal Microbiome, Magnetic Resonance Imaging
- Trial name
- NCT04691583
- Testing
- Fast diet
- Where
- China
- Volunteers
- 35 people
- Sponsor
- Henan Provincial People's Hospital
Leigh Syndrome (Maternally Inherited, MILS), Leigh Syndrome (AR, AD, XR)
Drug Repurposing for Mitochondrial Disorders Using iPSCs Derived Neural Cells
In this project, the investigators are using iPSC lines derived from patients with Leigh syndrome that carry mutations in the mitochondrial (mtDNA) and in the nuclear DNA (nDNA) to reprogram them into neural progenitor cells and…
- Trial name
- cureMILS (NCT06967831)
- Testing
- skin biopsy, generation of iPSCs, blood drawing
- Where
- 2 sites in Germany
- Volunteers
- 80 people
- Sponsor
- Charite University, Berlin, Germany
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