Clinical trials: Brain Disorder, Metabolic
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
Homocystinuria
Effects of Exercise on Metabolic Parameters in Classical Homocystinuria
The aim of this research project is to compare the effect of an aerobic exercise session in two different populations. Sampling biological material and collecting health-related personal data entails minimal risks and burdens.
- Trial name
- NCT04021732
- Where
- 2 sites in Switzerland
- Volunteers
- 12 people, healthy volunteers welcome
- Sponsor
- University of Lausanne
Hepatic Encephalopathy, Liver Cirrhosis
The Effect of Induced Hyperammonaemia on Sleep and Melanopsin-mediated Pupillary Light Response in Patients With Liver Cirrhosis
Sleep disturbances are common among patients with liver cirrhosis, but the reasons are not well understood. In this project the investigators evaluated whether an increase in blood ammonia in patients with cirrhosis had an impact…
- Conditions
- Hepatic Encephalopathy, Liver Cirrhosis, Sleep Disturbance
- Trial name
- NCT04771104
- Testing
- Amino Acid Challenge (AAC)
- Where
- Denmark
- Volunteers
- 9 people
- Sponsor
- Copenhagen University Hospital, Rigshospitalet
Type 2 Diabetes Mellitus
Effects of Tirzepatide and Insulin Glargine on Glucolipid Metabolism and Brain Function in Patients With Type 2 Diabetes
The effects of Tirzepatide and Insulin Glargine on glucose and lipid metabolism and inflammation in patients with type 2 diabetes mellitus.
- Trial name
- NCT05553093
- Testing
- Tirzepatide, Insulin Glargine
- Where
- China
- Volunteers
- 150 people
- Sponsor
- Nanjing First Hospital, Nanjing Medical University
Stress Urinary Incontinence (SUI)
Mesh-Free Versus Mesh-Based Surgery for Female Stress Urinary Incontinence: A Prospective Comparison of Pubo-Urethral Ligament Plication and Transobturator Tape
This prospective clinical study aims to compare the short-term effectiveness, safety, and patient-reported outcomes of two surgical techniques used for the treatment of female stress urinary incontinence (SUI): Pubo-Urethral…
- Trial name
- PLP-TOT (NCT07290114)
- Testing
- Pubo-Urethral Ligament Plication (PLP), Transobturator Tape (TOT)
- Where
- Turkey (Türkiye)
- Volunteers
- 147 people
- Sponsor
- Mehmet Incebıyik
Fabry Disease
Safety, PK/PD, and Exploratory Efficacy Study of AMT-191 in Classic Fabry Disease
The main goals of this clinical study are to characterize safety and PK/PD of AMT-191 i.e. if drug doses used in the study are safe and tolerable and to understand how it acts in the body of people with Fabry disease.
- Trial name
- NCT06270316
- Testing
- AMT-191
- Where
- 10 sites in United States
- Volunteers
- 20 people
- Sponsor
- UniQure Biopharma B.V.
Alzheimer's Disease, Dementia With Lewy Bodies
Imaging of Brain Amyloid Plaques in the Aging Population
This is a prospective, open label, non-therapeutic, diagnostic imaging study. The purpose of this study is to utilize Pittsburgh Compound B positron emission imaging (PiB PET) to ascertain the relationship between change in…
- Conditions
- Alzheimer's Disease, Dementia With Lewy Bodies, Frontotemporal Dementia, Vascular Dementia
- Trial name
- NCT00950430
- Testing
- Pittsburgh Compound B (C-11 PiB), F-18 FDG, Tau (18-F-AV-1451)
- Where
- United States
- Volunteers
- 8,000 people, healthy volunteers welcome
- Sponsor
- Mayo Clinic
Inborn Errors of Metabolism, Epilepsy (Treatment Refractory)
Metabolites in Blood and Breath in Metabolic Conditions
In patients with inborn errors of metabolism (IEM) and in those requiring a ketogenic diet for the treatment of medical conditions, relevant biochemical analytes are routinely and regularly measured in venous or capillary blood…
- Trial name
- NCT07704606
- Testing
- Observational Study
- Volunteers
- 100 people, healthy volunteers welcome
- Sponsor
- University Children's Hospital, Zurich
GM2 Gangliosidosis, Niemann-Pick Type C Disease
A Study to Evaluate the Safety and Efficacy of Nizubaglustat (AZ-3102) in Patients With GM2 Gangliosidosis or Niemann-Pick Type C Disease
This open-label study aims to gather long-term safety, tolerability, PK, biomarker, and clinical efficacy data relating to daily administration of Nizubaglustat in participants previously enrolled in the Phase 2 RAINBOW study…
- Trial name
- PRISMA (NCT07399704)
- Testing
- AZ-3102
- Where
- 3 sites in Brazil
- Volunteers
- 21 people
- Sponsor
- Azafaros B.V.
Clostridium Difficile Infection, Multi Drug Resistant Organisms
Establishment of the Human Intestinal and Salivary Microbiota Biobank - Gastrointestinal Diseases
This is a prospective, clinical, multicentre study aimed to collect biological samples and study microbiota from subjects with Clostridium Difficile (CDI), subjects affected by Multi Drug Resistant Organisms (MDRO) infection…
- Conditions
- Clostridium Difficile Infection, Multi Drug Resistant Organisms, Chronic Inflammatory Bowel Disease, Irritable Bowel Syndrome
- Trial name
- BIOMIS-Gastr (NCT04698148)
- Testing
- Biological sample collection, Questionnaire, Medical examination
- Where
- 3 sites in Italy
- Volunteers
- 225 people, healthy volunteers welcome
- Sponsor
- Azienda Ospedaliero-Universitaria Consorziale Policlinico di Bari
Type 2 Diabetes, Coronary Heart Disease
NMR Metabolomics-Based Risk Assessment and Stratified Digital Health Interventions in Middle-Aged Adults (PREMAP)
PREMAP invites residents of the Eloisa region (South Savo, Finland) turning 45, 50, or 55 years of age in 2026 to undergo a blood-based NMR metabolomics risk assessment for five interlinked cardio-renal-metabolic conditions (type…
- Conditions
- Type 2 Diabetes, Coronary Heart Disease, Myocardial Infarction, Cerebrovascular Disease
- Trial name
- PREMAP (NCT07820800)
- Testing
- Digital health interventions
- Where
- Finland
- Volunteers
- 1,400 people, healthy volunteers welcome
- Sponsor
- University of Eastern Finland
Diabetes, Type 1 Diabetes
The Role of South Asian vs European Origins on Circulating Regenerative Cell Exhaustion
ORIGINS-RCE is an observational, cross-sectional, two-arm study aimed at determining if an individual's ethnic origin influences the number of blood vessel-forming stem cells in the bloodstream.
- Conditions
- Diabetes, Type 1 Diabetes, Type 2 Diabetes, Ischemic Heart Disease
- Trial name
- ORIGINS-RCE (NCT05253521)
- Where
- 3 sites in Canada
- Volunteers
- 120 people
- Sponsor
- Canadian Medical and Surgical Knowledge Translation Research Group
Tetrahydrobiopterin Deficiencies, Hyperphenylalaninemia, Non-Phenylketonuric
Safety and Efficacy Study of Phenoptin in Subjects With Hyperphenylalaninemia Due to BH4 Deficiency
The purpose of this study is to evaluate the ability of Phenoptin to control blood phenylalanine levels in subjects who have hyperphenylalaninemia due to a primary BH4 deficiency and to evaluate the safety of Phenoptin in this…
- Trial name
- NCT00355264
- Testing
- Phenoptin
- Where
- 10 sites in United States, Germany
- Volunteers
- 12 people
- Sponsor
- BioMarin Pharmaceutical
Rare Diseases, Fabry Disease
Evaluation of HEArt invoLvement in Patients With FABRY Disease
This study evaluates predictors for the incidence of arrhythmias and sudden cardiac death as well as terminal heart failure in patients with Fabry disease.
- Conditions
- Rare Diseases, Fabry Disease, Fabry Disease, Cardiac Variant, Hypertrophic Cardiomyopathy
- Trial name
- HEAL-FABRY (NCT03362164)
- Where
- Germany
- Volunteers
- 650 people
- Sponsor
- Wuerzburg University Hospital
Cerebral Edema, Diabetic Ketoacidosis
Randomized Control Trial of Fluid Therapy for Pediatric Diabetic Ketoacidosis
The investigators will conduct a randomized controlled trial comparing four different intravenous (IV) fluid treatment protocols for pediatric diabetic ketoacidosis (DKA).
- Trial name
- NCT01365793
- Testing
- 0.45% saline replacement fluid, 0.9% saline replacement fluid, 0.45% saline intravenous fluid
- Where
- 13 sites in United States
- Volunteers
- 1,389 people
- Sponsor
- University of California, Davis
Nutritional Deficiencies, Phenylketonuria
Follow-up of Adult Phenylketonuria (PKU) Patients
Adult patients with phenylketonuria (PKU) at the age around 40 years belong to the first patients generation with early treatment of the disease.
- Trial name
- NCT01096758
- Where
- Germany
- Volunteers
- 40 people, healthy volunteers welcome
- Sponsor
- Ludwig-Maximilians - University of Munich
Obesity, Metabolic Syndrome
The Effect of Combined Aerobic Exercise and Calorie Restriction on Mood, Cognition, and Motor Behavior in Overweight and Obese Women
The benefits of weight-loss programs on mood state and cognitive and motor behavior remain unclear and are largely limited to those of calorie restriction (CR) or physical exercise alone.
- Trial name
- NCT04016909
- Testing
- aerobic exercise plus calorie restriction
- Where
- Lithuania
- Volunteers
- 26 people, healthy volunteers welcome
- Sponsor
- Lithuanian Sports University
Dementia, Depression
Vegetarian Diet and Chronic Degenerative Diseases
To investigate the prospective association between a vegetarian diet and chronic degenerative diseases in two cohorts of Taiwanese Buddhists
- Conditions
- Dementia, Depression, Cardiovascular Diseases, Stroke
- Trial name
- NCT03470584
- Testing
- Diet Exposure
- Where
- Taiwan
- Volunteers
- 18,064 people, healthy volunteers welcome
- Sponsor
- Dalin Tzu Chi General Hospital
Coronary Disease, Stroke
Assessing Outcomes of Enhanced Chronic Disease Care Through Patient Education and a Value-based Formulary Study
The purpose of this study is to determine the effect of two novel interventions; (1) a value-based formulary which eliminates copayment for selected high-value medications (proven to prevent heart attacks, stroke, and…
- Conditions
- Coronary Disease, Stroke, Chronic Kidney Disease, Heart Failure
- Trial name
- ACCESS (NCT02579655)
- Testing
- Copayment Elimination, Personalized Education
- Where
- Canada
- Volunteers
- 4,764 people
- Sponsor
- University of Calgary
Acid Sphingomyelinase Deficiency (ASMD)
Diagnostic Creteria of Acid Sphingomyelinase Deficiency (ASMD)
Acid sphingomyelinase Deficiency known as Neiman _PICK disease is a group of rare genetic diseases. This study includes analysis of clinical manifestations in patients with ASMD and investigations done for diagnosis of these…
- Trial name
- NCT07274826
- Where
- Egypt
- Volunteers
- 7 people
- Sponsor
- Sohag University