Clinical trials: Brain Diseases, Metabolic
Friedreich Ataxia, Friedreich's Ataxia
A Study to Evaluate the Efficacy and Safety of Subcutaneous Nomlabofusp in Subjects With Friedreich's Ataxia
To evaluate the efficacy and safety of subcutaneous nomlabofusp in adult and pediatric subjects with Friedreich's ataxia
- Trial name
- FORWARD-FA (NCT07778836)
- Testing
- Nomlabofusp
- Where
- 4 sites in United States
- Volunteers
- 150 people
- Sponsor
- Larimar Therapeutics, Inc.
Acute Ischemic Stroke, Diabetes (DM)
CGM in Acute Ischemic Stroke
The goal of this randomized clinical trial is to evaluate whether continuous glucose monitoring (CGM) can be used to guide glucose management in patients with type 2 diabetes who are admitted with an acute ischemic stroke and…
- Conditions
- Acute Ischemic Stroke, Diabetes (DM), Diabetes Type 2, Hyperglycemia
- Trial name
- SENSTROKE (NCT07510919)
- Testing
- Continuous Glucose Monitoring, Point-of-Care Testing
- Volunteers
- 82 people
- Sponsor
- Isala
Fabry Disease
Aortic Dimensions in Patients With Fabry Disease
We will conduct a retrospective study aiming to define the characteristics, determinants, and progression of ascending aortic dilatation in adults with Fabry disease, using echocardiography and/or cardiac MRI
- Trial name
- DATA-Fab (NCT06956573)
- Testing
- aorta imaging
- Volunteers
- 200 people
- Sponsor
- University Hospital, Caen
Cardiovascular Disease, Diabetes
Meta-Analyses of Low-risk Lifestyle Behaviours and Patient Important Outcomes
Public health policy is universal in recommending the adoption of low risk low-risk lifestyle behaviors for health promotion and prevention of chronic or non-communicable diseases (NCDs).These behaviors generally include…
- Conditions
- Cardiovascular Disease, Diabetes, Cancer, Mortality
- Trial name
- NCT03234101
- Testing
- Low-risk lifestyle behaviours
- Where
- Canada
- Volunteers
- 1 people, healthy volunteers welcome
- Sponsor
- University of Toronto
Hepatic Encephalopathy
Vegetable Proteins Versus Mixed Animal Proteins on the Outcome of Hepatic Encephalopathy
Vegetables are thought to be beneficial not only because of their high content of fiber, which promotes bacterial fermentation and decreases colonic transit time, decreasing ammonia absorption from the gut, but also because of…
- Trial name
- NCT06075875
- Testing
- mixed animal and vegetable proteins, pure vegetable proteins
- Where
- Egypt
- Volunteers
- 196 people
- Sponsor
- National Hepatology & Tropical Medicine Research Institute
Gaucher Disease, Type 3
A Multicenter, Safety and Efficacy Study of Taliglucerase Alfa in Subjects With Type 3 Gaucher Disease
This is a multicenter study to assess the safety and efficacy of taliglucerase alfa (60 units/kg) in previously untreated subjects of any age with Type 3 GD.
- Trial name
- NCT04002830
- Testing
- Elelyso
- Where
- 3 sites in India, Israel and 1 more
- Volunteers
- 14 people
- Sponsor
- Ari Zimran
Pompe Disease (Late-onset)
A Study to Assess the Long-term Safety and Efficacy of ATB200/AT2221 in Adult Subjects With Late-Onset Pompe Disease (LOPD)
This is a multicenter, international open-label extension study of ATB200/AT2221 in adult subjects with late-onset Pompe disease (LOPD) who completed Study ATB200-03.
- Trial name
- NCT04138277
- Testing
- AT2221, ATB200
- Where
- 60 sites in United States, Argentina and 21 more
- Volunteers
- 119 people
- Sponsor
- Amicus Therapeutics
Premature Infants, Cerebral Palsy
Interrater Reliability of Infant Motor Profile
The aim of this study is to assess risky infants (or diagnosed infants) with Infant Motor Profile, and analyze interrater and intrarater reliability of this test.
- Conditions
- Premature Infants, Cerebral Palsy, Down Syndrome, Metabolic Disorders
- Trial name
- IMP (NCT03188107)
- Testing
- Video based assessment
- Volunteers
- 50 people, healthy volunteers welcome
- Sponsor
- Abant Izzet Baysal University
Stroke Acute, Encephalitis
Inflammatory and Metabolic Prognostic Assessment in Critically Ill Neurological Patients
The study aims to develop and validate a prognostic prediction model for adverse outcomes in neurocritical patients receiving enteral nutrition based on key inflammatory and metabolic markers.
- Conditions
- Stroke Acute, Encephalitis, Non-traumatic Brain Injury
- Trial name
- IMPACT-NEURO (NCT06849011)
- Where
- 19 sites in China
- Volunteers
- 1,185 people
- Sponsor
- Yan Zhang
Diabetes, Hypertension
Electronic-Nutrition-Optimizer for Personalized Prevention
This study aims to develop and validate a tool for immediate nutrition assessment and to test its user feasibility in routine clinical practice for health promotion.
- Conditions
- Diabetes, Hypertension, Frailty, Dementia
- Trial name
- eNO (NCT04866264)
- Testing
- eNutrition Optimizer
- Where
- Switzerland
- Volunteers
- 88 people, healthy volunteers welcome
- Sponsor
- Heike Bischoff-Ferrari
Gaucher Disease
A Study of the Effects of Hepatic Impairment on the Pharmacokinetics and Tolerability of Eliglustat Tartrate
Primary Objective: To study the effect of mild and moderate hepatic impairment on the pharmacokinetics (PK) of eliglustat. Secondary Objective: To assess the tolerability of eliglustat tartrate given as a single dose in subjects…
- Trial name
- NCT02536911
- Testing
- eliglustat
- Where
- 2 sites in United States
- Volunteers
- 24 people, healthy volunteers welcome
- Sponsor
- Genzyme, a Sanofi Company
Phenylketonuria
The Brain, Neurological Features and Neuropsychological Functioning in Adults With Phenylketonuria: A Pilot Study
Newborn screening and early treatment prevent the most severe manifestations of phenylketonuria (PKU). However, executive functioning deficits, attention deficit disorder, slow processing speed, and visual-motor problems commonly…
- Trial name
- NCT01917344
- Testing
- MRI
- Where
- United States
- Volunteers
- 10 people
- Sponsor
- Boston Children's Hospital
Hypercholesterolemia
Evaluation of the Safety & Efficacy of a Combination of Niacin ER & Simvastatin in Patients With Dyslipidemia (OCEANS)
The purpose of this 24 week study is to compare the effectiveness and safety of different doses of Niacin ER/Simvastatin (NS) in subjects with elevated fat levels in their blood (dyslipidemia).
- Trial name
- NCT00080275
- Testing
- Niacin Extended-Release and simvastatin Tablets
- Volunteers
- 600 people
- Sponsor
- Kos Pharmaceuticals
Phenylketonurias
Study of Phenoptin to Increase Phenylalanine Tolerance in Phenylketonuric Children on a Phenylalanine-restricted Diet
The primary objective of this trial is to evaluate the ability of Phenoptin to increase phenylalanine (phe) tolerance in children with phenylketonuria who are following a phe-restricted diet.
- Trial name
- NCT00272792
- Testing
- Sapropterin Dihydrochloride
- Where
- 9 sites in United States
- Volunteers
- 45 people
- Sponsor
- BioMarin Pharmaceutical
Diabetes, Senile Dementia, Alzheimer Type
A Study of Mobile Medical Supportive Care Program for Family Caregivers With Dementia Combined With Diabetes
The purpose of this project is to evaluate the effectiveness of the clinical application of a mHealth supportive care program for family caregivers with dementia combined with diabetes based on the supportive care framework.
- Trial name
- NCT06785857
- Testing
- mHealth Supportive Care Program, Offline supportive care
- Where
- China
- Volunteers
- 60 people, healthy volunteers welcome
- Sponsor
- Yuanjiao Yan
Phenylketonuria
Response to Kuvan® in Subjects With Phenylketonuria (PKU) in a 4 Weeks Testing Period
The primary objective of the study is to evaluate the proportion of responders (that is, greater than or equal to [>=] 30 percent reduction from Baseline in blood phenylalanine [Phe] level) to treatment with Kuvan® (sapropterin…
- Trial name
- ENDURE (NCT01082328)
- Testing
- Kuvan®
- Where
- Norway
- Volunteers
- 59 people
- Sponsor
- Merck KGaA, Darmstadt, Germany
PSP - Progressive Supranuclear Palsy
The Progressive Supranuclear Palsy Clinical Trial Platform - Regimen B: LM11A-31
The Progressive Supranuclear Palsy Clinical Trial Platform (PTP) is a multi-center, multi-regimen clinical trial evaluating the safety and efficacy of investigational products for the treatment of PSP.
- Trial name
- NCT07264283
- Testing
- LM11A-31
- Where
- United States
- Volunteers
- 147 people
- Sponsor
- Adam Boxer
Pompe Disease
Pompe Disease RWE Long-term Monitoring Program
Evaluate short- and long-term biomarker, motor function, respiratory function and safety with avalglucosidase alfa in IOPD and LOPD patients who switched from other ERT.
- Trial name
- NCT07834320
- Where
- Taiwan
- Volunteers
- 40 people
- Sponsor
- National Taiwan University Hospital
Lysosomal Storage Disease, Metachromatic Leukodystrophy
Gene Therapy for Metachromatic Leukodystrophy (MLD)
This Phase I/II clinical trial consists of the application of lentiviral vector-based gene therapy to patients affected by Metachromatic Leukodystrophy (MLD), a rare inherited Lysosomal Storage Disorder (LSD) resulting from…
- Trial name
- NCT01560182
- Testing
- OTL-200 Gene Therapy
- Where
- Italy
- Volunteers
- 20 people
- Sponsor
- Orchard Therapeutics
Arginase I Deficiency, Hyperargininemia
Efficacy and Safety of Pegzilarginase in Patients With Arginase 1 Deficiency
CAEB1102-300A is a multi-center randomized, double-blind, placebo-controlled study to evaluate the safety and efficacy of pegzilarginase in patients with ARG1-D.
- Trial name
- NCT03921541
- Testing
- Pegzilarginase
- Where
- 32 sites in United States, Austria and 5 more
- Volunteers
- 32 people
- Sponsor
- Immedica Pharma AB
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