Clinical trials: Brain Diseases, Metabolic
Wilson Disease
Evaluation of Cognitive Dysfunction and Psychiatric Comorbidities
Primary objective Collection of patients with wilson disease either presented with neurological or hepatic symptoms Assessment of psychiatric and cognitive disorders in both groups by using specific scales Secondary objective…
- Trial name
- NCT06179394
- Volunteers
- 40 people
- Sponsor
- Assiut University
Neurologic Disorder, Neurologic Diseases
Functional Outcomes and Control Using Synchron BCI - Australia
Functional Outcomes and Control Using Synchron BCI - Australia (FOCUS-AUS)
- Conditions
- Neurologic Disorder, Neurologic Diseases, MND (Motor Neurone DIsease), Motor Neuron Disease
- Trial name
- FOCUS-AUS (NCT07533903)
- Testing
- Stentrode
- Where
- Australia
- Volunteers
- 10 people
- Sponsor
- Synchron, Inc.
Phenylketonuria
Efficacy and Safety of Orally Administered Engineered Probiotics (CBT102-A) for the Treatment of Children With Phenylketonuria
This is a randomized, double-blind, placebo-controlled, parallel-group study. A total of 15 children with phenylketonuria(PKU) age 3 to 17 years will be randomized to two groups.
- Trial name
- NCT05948020
- Testing
- CBT102-A capsule
- Where
- China
- Volunteers
- 15 people
- Sponsor
- Children's Hospital of Fudan University
Friedreich Ataxia
A Study to Assess Variation in Potential Biomarkers in Friedreich Ataxia
To test the variability of specific ribonucleic acid (RNA) and proteins as well as frataxin levels in samples of blood and buccal cells taken directly from patients with Friedreich's ataxia (FRDA) in order to confirm potential…
- Trial name
- NCT04255680
- Testing
- Buccal Swabs and Blood Draws
- Where
- United States
- Volunteers
- 20 people
- Sponsor
- Larimar Therapeutics, Inc.
Friedreich Ataxia
Efficacy of Stabilometric Platform to Improve Standing Balance in Patients With Friedreich's Ataxia
The primary objective is to evaluate the potential effectiveness of an individualized intensive rehabilitation intervention using the "Prokin 252" stabilometric platform in the treatment of adolescent and adult patients with…
- Trial name
- NCT06692296
- Testing
- Balance training over stabilometric platform, Balance training by means of conventional rehabilitation
- Where
- Italy
- Volunteers
- 24 people
- Sponsor
- IRCCS Eugenio Medea
Cerebral Adrenoleukodystrophy (CALD), Adrenoleukodystrophy (ALD)
Long-term Follow-up of Participants With Cerebral Adrenoleukodystrophy Who Were Treated With Lenti-D Drug Product
This is a multi-center, long-term safety and efficacy follow-up study for participants with cerebral adrenoleukodystrophy (CALD) who have received Lenti-D Drug Product (eli-cel) in a parent clinical study (Study ALD-102 or Study…
- Conditions
- Cerebral Adrenoleukodystrophy (CALD), Adrenoleukodystrophy (ALD), X-Linked Adrenoleukodystrophy (X-ALD)
- Trial name
- NCT02698579
- Testing
- No interventional drug product utilized in this follow-up study
- Where
- 13 sites in United States, Argentina and 7 more
- Volunteers
- 64 people
- Sponsor
- Genetix Biotherapeutics Inc.
Cardiovascular Diseases, Peripheral Vascular Diseases
Homocysteine and Progression of Atherosclerosis
In the first phase, to establish the relationship of progression of peripheral vascular disease (PVD) to plasma homocysteine. In the second phase, to conduct a randomized, controlled trial of folic acid treatment of plasma…
- Conditions
- Cardiovascular Diseases, Peripheral Vascular Diseases, Atherosclerosis, Cerebrovascular Disorders
- Trial name
- NCT00005338
- Sponsor
- National Heart, Lung, and Blood Institute (NHLBI)
Liver Cirrhosis, Hepatic Encephalopathy
Diagnosis of Covert/Minimal Hepatic Encephalopathy by Means of Continuous Reaction Time Measurement
The investigators wish to investigate how the Continuous Reaction Time (CRT) method can be used in the diagnosis and monitoring of covert hepatic encephalopathy (cHE)in patients with cirrhosis of the liver.
- Trial name
- NCT01773538
- Testing
- Lactulose and rifaximin, Branched Chain amino acids
- Where
- 2 sites in Denmark
- Volunteers
- 136 people, healthy volunteers welcome
- Sponsor
- Mette Munk Lauridsen
Homocystinuria
Homocystinuria: Treatment With N-Acetylcysteine
The purpose of this study is determine if oral N-acetylcysteine is effective in lowering homocysteine in individuals with homocystinuria.
- Trial name
- NCT00483314
- Testing
- N-acetylcysteine
- Where
- 2 sites in Canada
- Volunteers
- 5 people
- Sponsor
- McGill University Health Centre/Research Institute of the McGill University Health Centre
Wilson's Disease
International Wilson's Disease Patient Registry (iWilson Registry)
Longitudinal, observational, non-interventional, standard of care Registry. Data will be collected from the routinely scheduled WD clinic visits at approximately 6-12 month intervals.
- Trial name
- NCT05239858
- Where
- 16 sites in Belgium, France and 5 more
- Volunteers
- 500 people
- Sponsor
- Orphalan
Type 2 Diabetes, Prediabetes
Sweet Sensing in Type 2 Diabetes
Diabetes is a global challenge and the number of people affected by diabetes is expected to rise to 5.5 million by 2030, of which 90% are type 2 diabetes (T2D).
- Trial name
- NCT06804187
- Where
- United Kingdom
- Volunteers
- 505 people, healthy volunteers welcome
- Sponsor
- University of Nottingham
Parkinson Disease
Lipoprotein Metabolism and Bacterial Lipopolysaccharide in Parkinson's Disease
Patients with Parkinson's disease (PD) present an impaired intestinal permeability with consequent lipopolysaccharide (LPS) translocation in the systemic circulation.
- Trial name
- LiMBaLiP (NCT03937284)
- Testing
- Patients not treated
- Where
- Italy
- Volunteers
- 45 people, healthy volunteers welcome
- Sponsor
- University of Milan
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
Mitochondrial Diseases
EPI-743 for Mitochondrial Respiratory Chain Diseases
This study will evaluate the safety and efficacy of EPI-743 in participants with severe mitochondrial respiratory chain diseases who are considered to be within 90 days of end-of-life care.
- Trial name
- NCT01370447
- Testing
- EPI-743
- Where
- 16 sites in United States
- Sponsor
- PTC Therapeutics
Friedreich's Ataxia
Safety and Pharmacology Study of VP 20629 in Adults With Friedreich's Ataxia
The objectives of the study are: To evaluate the safety and tolerability of single and multiple oral doses of VP 20629 in subjects with Friedreich's ataxia (FA).
- Trial name
- NCT01898884
- Testing
- VP 20629
- Where
- 5 sites in United States
- Volunteers
- 46 people
- Sponsor
- Shire
Hydrocephalus, Normal Pressure, Biochemical Lesions Head Region
Role of Brain Specific Biomarkers in Hydrocephalus
Normal pressure hydrocephalus (NPH) is a neurodegenerative disease of unclear etiology characterized by a clinical trias named after the neurosurgeon Hakim.
- Conditions
- Hydrocephalus, Normal Pressure, Biochemical Lesions Head Region, Brain Damage
- Trial name
- NCT06083233
- Testing
- Lumbar puncture, External lumbar drainage, Ventriculo-peritoneal shunt placement
- Where
- Czechia
- Volunteers
- 32 people
- Sponsor
- University Hospital Hradec Kralove
PKU, Tyrosinemias
GMP Case Studies of Tolerance, Safety and Acceptability in PKU and TYR
Three new protein substitutes have been developed to help with compliance in PKU and TYR patients, which are powdered protein substitutes, low in Phe and, low in Phe and Tyr respectively, with a mixed balance of glycomacropeptide…
- Trial name
- NCT05062226
- Testing
- PKU/TYR GMP Protein Substitute
- Where
- United Kingdom
- Volunteers
- 45 people
- Sponsor
- Nutricia UK Ltd
Acute Ischemic Stroke, Type 2 Diabetes
Efficacy and Safety of Urinary Kallidinogenase in the Treatment of Acute Ischemic Stroke Combined With Type 2 Diabetes Mellitus
This study is a multicenter, randomized, double-blind, placebo parallel control study, aim to evaluate the efficacy and safety of human urinary kallidinogenase in the treatment of acute ischemic stroke with type 2 diabetes.
- Trial name
- TK-SEEK (NCT06085378)
- Testing
- Urinary Kallidinogenase for injection
- Where
- China
- Volunteers
- 630 people
- Sponsor
- First Affiliated Hospital, Sun Yat-Sen University
Friedreich's Ataxia (FA)
A Study of SGT-212 Gene Therapy in Friedreich's Ataxia
This is a phase 1b, first in-human, open-label, dose-finding study investigating the safety and tolerability of SGT-212 in participants with Friedreich's ataxia (FA).
- Trial name
- FALCON (NCT07180355)
- Testing
- SGT-212
- Where
- 3 sites in United States
- Volunteers
- 10 people
- Sponsor
- Solid Biosciences Inc.
Parkinson Disease
The Effect of Intranasal Insulin on Motor and Non-motor Symptoms in Parkinson's Disease Patients
This study will evaluate the effect of intranasal insulin on motor and non-motor symptoms of patients with Parkinson's disease over a 12-week period.
- Trial name
- NCT04687878
- Testing
- Insulin, Normal saline
- Where
- Iran
- Volunteers
- 28 people
- Sponsor
- dr.dargahi
The first 1,020 results can be paged through. Add a filter or change the sort order to reach the rest.