Clinical trials: Brain Diseases, Metabolic
Danon Disease, Fabry Disease
Precision Diagnosis and Risk Stratification of Rare Cardiomyopathies Based on Novel Cardiac Magnetic Resonance Techniques
What is this study about? This research is focused on improving the care for people with rare heart muscle diseases, known as rare cardiomyopathies.
- Conditions
- Danon Disease, Fabry Disease, Cardiac Amyloidosis, Noonan Syndrome
- Trial name
- NCT07336394
- Where
- China
- Volunteers
- 1,000 people, healthy volunteers welcome
- Sponsor
- Chinese Academy of Medical Sciences, Fuwai Hospital
Cerebral Palsy, Mastication Disorder
Growth, Dietary Intakes and Feeding Behaviors of Children With Cerebral Palsy Who Have Chewing Disorders
The nutritional status of CP children may be decreased when children have chewing disorders too due to insufficient solid food intake.
- Conditions
- Cerebral Palsy, Mastication Disorder, Malnutrition, Growth Delay
- Trial name
- NCT02777645
- Testing
- Growth, feeding evaluation
- Where
- Turkey (Türkiye)
- Volunteers
- 85 people
- Sponsor
- Hacettepe University
Urea Cycle Disorders, Inborn, Other Metabolic Diseases
Protein Sorbent Properties of Montmorillonite in Vitro and in Vivo Models
Montmorillonite (MONT) is a phyllosilicate layered mineral with unique physicochemical properties, such as swelling and cation exchange capability.
- Trial name
- NCT02124330
- Testing
- montmorillonite 5 g, montmorillonite 3 g, Montmorillonite 1 g
- Where
- Italy
- Volunteers
- 35 people, healthy volunteers welcome
- Sponsor
- Luca Ronfani
Coronary Artery Disease
Serial OCT Evaluation of Tissue Coverage in Patients Submitted to Inspiron Drug Eluting Stent Implantation
Prospective, multicentric, single-arm study to evaluate fast and effective tissue repair in patients undergone percutaneous coronary intervention with drug eluting stent Inspiron.
- Trial name
- REPAIR (NCT03269461)
- Testing
- Angiography and Optical Coherence Tomography evaluations
- Where
- 5 sites in Brazil
- Volunteers
- 60 people
- Sponsor
- Scitech Produtos Medicos SA
Pediatric Migraine, Migraine Disorders, Brain
Comorbidity-Based Propranolol Use in Pediatric Migraine
This observational retrospective cohort study aims to identify clinical and biochemical predictors of response to propranolol prophylaxis in pediatric migraine.
- Conditions
- Pediatric Migraine, Migraine Disorders, Brain, Vitamin D Deficiency
- Trial name
- NCT07103031
- Testing
- Behavioral therapy, Propranolol
- Where
- Turkey (Türkiye)
- Volunteers
- 488 people
- Sponsor
- Kayseri University
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
Hyponatremia, Syndrome of Inappropriate Antidiuresis
Protein Supplementation in Hyponatremia Due to the Syndrome of Inappropriate Antidiuresis
The goal of this study is to assess the impact of protein supplementation on hyponatremia caused by the syndrome of inappropriate antidiuresis during hospitalization.
- Trial name
- TREASUREx (NCT06821802)
- Testing
- Moltein PURE Protein Powder, Fluid restriction
- Where
- Switzerland
- Volunteers
- 70 people
- Sponsor
- University Hospital, Basel, Switzerland
Obesity
PINS Stimulator System for Deep Brain Stimulation to Treat Obesity
The purpose of this clinical study is to verify the long term effecacy and safety of a bilateral deep brain stimulation (DBS) produced by Beijing PINS Medical Co., Ltd. as a treatment option for patients with obesity.
- Trial name
- NCT02254395
- Testing
- Deep Brain Stimulation
- Volunteers
- 8 people, healthy volunteers welcome
- Sponsor
- Beijing Pins Medical Co., Ltd
Type 2 Diabetes Mellitus, Cognitive Impairment
Establish Diagnostic Models Based on Olfactory Function and Odor-induced Brain Activation for Diabetes-Related Cognitive Impairment
This is a cross-sectional and longitudinal study to establish diagnostic models based on olfactory function assessments and odor-induced brain activation for cognitive impairment in patients with type 2 diabetes mellitus.
- Trial name
- NCT05543967
- Testing
- Cognitive assessments, Olfactory function measurements, Functional magnetic resonance imaging
- Where
- China
- Volunteers
- 200 people, healthy volunteers welcome
- Sponsor
- The Affiliated Nanjing Drum Tower Hospital of Nanjing University Medical School
Alzheimer Disease, Parkinson Disease
hUC-MSC-sEV-001 Nasal Drops for Neurodegenerative Diseases
To evaluate the safety and preliminary efficacy of human umbilical cord mesenchymal stem cell-derived small extracellular vesicles hUC-MSC-sEV-001 nasal drops in multiple neurodegenerative diseases, including Alzheimer's disease…
- Conditions
- Alzheimer Disease, Parkinson Disease, Lewy Body Dementia, Multiple System Atrophy
- Trial name
- NCT06607900
- Testing
- hUC-MSC-sEV-001 nasal drops
- Where
- China
- Volunteers
- 120 people
- Sponsor
- Xuanwu Hospital, Beijing
Dementia, Frontotemporal Dementia
Study in Asymptomatic GRN-FTD Patients to Investigate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of VES001
This is an open-label, multiple dosing study in asymptomatic GRN-FTD carriers to investigate the safety, tolerability, PK, and PD of VES001.
- Conditions
- Dementia, Frontotemporal Dementia, Asymptomatic Condition
- Trial name
- SORT-IN-2 (NCT06705192)
- Testing
- VES001
- Where
- 2 sites in Netherlands, United Kingdom
- Volunteers
- 6 people
- Sponsor
- Mads Kjolby
Atherosclerotic Plaque, Diabetes Mellitus, Type 2
Pakistan Study of Premature Coronary Atherosclerosis in Young Adults
Coronary heart disease (CHD) is a major cause of morbidity, disability, mortality, and health expenditures worldwide. A wealth of studies has demonstrated that people of South Asian ancestry have a higher risk of CHD and…
- Conditions
- Atherosclerotic Plaque, Diabetes Mellitus, Type 2, Hypertension, Stroke
- Trial name
- PAKSEHAT (NCT05156736)
- Where
- Pakistan
- Volunteers
- 2,000 people, healthy volunteers welcome
- Sponsor
- Tabba Heart Institute
Stroke, Osteoporosis
Assessment and Comparison of Osteoporosis Risk Factors After Stroke
The aim of this study is to evaluate the effects of stroke and associated clinical, functional, nutritional, and muscular factors, including handgrip strength, on bone mineral density and osteoporosis by comparing patients with…
- Trial name
- NCT07228611
- Testing
- Bone Mineral Density
- Where
- Turkey (Türkiye)
- Volunteers
- 84 people, healthy volunteers welcome
- Sponsor
- Ankara Etlik City Hospital
Friedreich Ataxia, Healthy Volunteers
Determine Range of Tissue Frataxin Concentrations and Other Potential Biomarkers
To examine range of tissue frataxin (FXN) concentrations, specific ribonucleic acids, other proteins and specialized lipids in the buccal cells, blood, and skin cells of normal healthy volunteers without Friedreich's ataxia…
- Trial name
- NCT05028764
- Testing
- Buccal Swabs, Blood Draws and Skin Punch Biopsy
- Where
- United States
- Volunteers
- 60 people, healthy volunteers welcome
- Sponsor
- Larimar Therapeutics, Inc.
Primary Progressive Nonfluent Aphasia
A 24-month Phase 1 Pilot Study of AADvac1 in Patients With Non Fluent Primary Progressive Aphasia
This study is a pilot trial evaluating the safety and immunogenicity of AADvac1 in patients with the non-fluent variant of Primary Progressive Aphasia.
- Trial name
- AIDA (NCT03174886)
- Testing
- AADvac1 40 µg, AADvac1 160 µg
- Where
- 3 sites in Germany
- Volunteers
- 33 people
- Sponsor
- Axon Neuroscience SE
Obesity
Pupillography as Screening Tool for Brain Insulin Resistance
Cerebral insulin action is important in the regulation of whole body glucose metabolism. However, the cerebral insulin action differs between individuals - there is a so called cerebral insulin resistance.
- Trial name
- NCT03651661
- Testing
- human insulin, Pupillography
- Where
- Germany
- Volunteers
- 22 people, healthy volunteers welcome
- Sponsor
- University Hospital Tuebingen
Friedreich Ataxia
A Study to Assess Nomlabofusp in Adolescents and Children With Friedreich's Ataxia
The goal of this clinical trial is to evaluate the safety and tolerability of nomlabofusp (CTI-1601) in adolescents and children with Friedreich's ataxia (FRDA).
- Trial name
- NCT06681766
- Testing
- Nomlabofusp
- Where
- United States
- Volunteers
- 18 people
- Sponsor
- Larimar Therapeutics, Inc.
Mitochondrial Myopathy, MELAS Syndrome
Study of the Metabolism of Pyruvate and Related Problems in Patients With Lactic Acidemia
OBJECTIVES: I. Study the metabolism of pyruvate and related problems in patients with lactic acidemia. II. Define the nature of the metabolic defect.
- Conditions
- Mitochondrial Myopathy, MELAS Syndrome, Lactic Acidosis
- Trial name
- NCT00004353
- Where
- United States
- Volunteers
- 35 people
- Sponsor
- National Center for Research Resources (NCRR)
Hepatocellular Carcinoma (HCC)
Triple Recipient Strategy Using Split, Domino, and Auxiliary Techniques
The investigators report a case of the simultaneous use of split liver transplantation (SLT), domino liver transplantation (DLT) and auxiliary liver transplantation (ALT) to achieve "one donor for three recipients".
- Trial name
- NCT06683820
- Where
- China
- Volunteers
- 3 people
- Sponsor
- Second Affiliated Hospital, Zhejiang University, School of Medicine
Rare Diseases, Fabry Disease
MigALastat Therapy Adherence Among FABRY Patients: A Prospective Multicentral Observational Study
This study evaluates adherence to the oral chaperone therapy migalastat in patients with Fabry disease.
- Conditions
- Rare Diseases, Fabry Disease, Adherence, Medication, Quality of Life
- Trial name
- MALTA-FABRY (NCT03683966)
- Where
- Germany
- Volunteers
- 30 people
- Sponsor
- Wuerzburg University Hospital
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