Clinical trials: Brain Disorder, Metabolic
Fabry Disease
Study to Compare the Efficacy and Safety of Oral AT1001 and Enzyme Replacement Therapy in Patients With Fabry Disease
Study to compare the efficacy and safety of migalastat and enzyme replacement therapy (ERT) in male and female participants with Fabry disease who are currently receiving ERT and who have an alpha galactosidase-A (α Gal-A)…
- Trial name
- NCT01218659
- Testing
- migalastat hydrochloride, agalsidase
- Where
- 25 sites in United States, Australia and 8 more
- Volunteers
- 68 people
- Sponsor
- Amicus Therapeutics
Obesity
Reduced Carbohydrate Versus Fat in Obese Subjects
Popular weight loss strategies often involve reducing an individual's consumption of carbohydrates or fat. However, no controlled study has been carried out to evaluate the effects of reducing carbohydrate versus fat consumption…
- Trial name
- NCT00846040
- Testing
- Reduced fat diet, Reduced carbohydrate diet, Drug: f-18 fallypride
- Where
- United States
- Volunteers
- 43 people
- Sponsor
- National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)
Idiopathic Intracranial Hypertension
Safety and Effectiveness of 11b-Hydroxysteroid Dehydrogenase Type 1 Inhibitor (AZD4017) to Treat Idiopathic Intracranial Hypertension.
Assessing the safety and effectiveness of a 11-βhydroxysteroid dehydrogenase type 1 inhibitor (AZD4017), in a placebo controlled trial, in acute idiopathic intracranial hypertension (IIH) IIH is a condition of young, overweight…
- Trial name
- IIH:DT (NCT02017444)
- Testing
- AZD4017
- Where
- United Kingdom
- Volunteers
- 31 people
- Sponsor
- University of Birmingham
Gliosis, Obesity
Does What You Eat Affect Your Brain
The purpose of this study is to use magnetic resonance imaging (MRI) to evaluate the human hypothalamus for signs of inflammation in response to specific diets.
- Conditions
- Gliosis, Obesity, Eating
- Trial name
- WYE (NCT05626907)
- Testing
- Diet modification #1, Diet modification #2, Diet modification #3
- Where
- United States
- Volunteers
- 36 people, healthy volunteers welcome
- Sponsor
- University of Washington
Fabry Disease
Evaluation of the Long-term Safety, Pharmacodynamics, and Exploratory Efficacy of GZ/SAR402671 in Treatment-Naïve Adult Male Patients With Fabry Disease
Primary Objective: To assess the long-term safety of GZ/SAR402671 in adult male participants with Fabry disease who previously completed study ACT13739 (NCT02489344).
- Trial name
- NCT02489344
- Testing
- GZ/SAR402671
- Where
- 7 sites in United States, France and 3 more
- Volunteers
- 8 people
- Sponsor
- Genzyme, a Sanofi Company
Fabry Disease
Safety and Efficacy Study of Several Replagal Dosing Regimens on Cardiac Function in Adults With Fabry Disease
The purpose of this study is to compare the safety and effectiveness of various doses of Replagal in patients with cardiomyopathy due to Fabry disease.
- Trial name
- NCT00864851
- Testing
- Replagal
- Where
- 12 sites in United States, Australia and 6 more
- Volunteers
- 44 people
- Sponsor
- Shire
Fabry Disease
A Study to Describe the Experience of Both Patients and Their Clinicians in the Treatment of Fabry Disease With Enzyme Replacement Therapy.
This is an international, non-interventional research study of adult patients with Fabry Disease and their caregivers. The study comprised a prospective time and motion evaluation and a cross-sectional evaluation of patient and…
- Trial name
- NCT04281537
- Testing
- Enzyme Replacement Therapy (ERT)
- Where
- 9 sites in United States, Brazil and 3 more
- Volunteers
- 82 people
- Sponsor
- Amicus Therapeutics
Fabry Disease
A Study of AT1001 (Migalastat Hydrochloride) in Participants With Fabry Disease
Study to evaluate the safety, tolerability, pharmacokinetics, and pharmacodynamics of migalastat hydrochloride (HCl) (migalastat) in participants with Fabry disease.
- Trial name
- NCT00214500
- Testing
- migalastat HCl
- Where
- 5 sites in United States
- Volunteers
- 9 people
- Sponsor
- Amicus Therapeutics
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
Amyotrophic Lateral Sclerosis
An In-home Study of Brain Computer Interfaces
The investigators are developing a tool to help people who are severely paralyzed. This tool is called a brain-computer interface (BCI). BCIs can connect to computers or other electronic devices.
- Trial name
- NCT01123200
- Testing
- Brain Computer Interface for Wheelchair Tilt Control
- Where
- United States
- Volunteers
- 1 people
- Sponsor
- University of Michigan
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.
Empaglifozin, Physiological Effects of Drugs
Sodium-glucose Co Transporter 2 (sGLT2) Inhibitor and Endogenous Ketone Production
Background: The drug empagliflozin treats diabetes. It lowers blood sugar by increasing glucose the kidneys excrete. This increases levels of ketones formed in the blood.
- Conditions
- Empaglifozin, Physiological Effects of Drugs, Hypoglycemic Agents, Sodium-Glucose Transporter 2 Inhibitors
- Trial name
- NCT03852901
- Testing
- empagliflozin (Jardiance) 25 mg
- Where
- United States
- Volunteers
- 21 people
- Sponsor
- National Institute on Aging (NIA)
Gaucher Disease
Plant Cell Expressed Recombinant Human Glucocerebrosidase Extension Trial
Gaucher disease, the most prevalent lysosomal storage disorder, is caused by mutations in the human glucocerebrosidase gene (GCD) leading to reduced activity of the lysosomal enzyme glucocerebrosidase and thereby to the…
- Trial name
- NCT00705939
- Testing
- Taliglucerase alfa
- Where
- 11 sites in United States, Australia and 6 more
- Volunteers
- 45 people
- Sponsor
- Pfizer
Idiopathic Parkinson's Disease
The Effect of Pramipexole on Metabolic Network Activity Compared With Levodopa in Early Parkinson's Disease
Levodopa and non-ergot dopaminergic agonists such as pramipexole are both recommended as the first-line symptomatic treatment for early untreated Parkinson's disease (PD), previous clinical trial indicated that initial…
- Trial name
- NCT01470859
- Testing
- pramipexole, Sinemet CR
- Where
- China
- Volunteers
- 30 people
- Sponsor
- Huashan Hospital
Gaucher Disease, Type 1
A Gene Therapy Study in Patients With Gaucher Disease Type 1
This study is a first-in-human, open-label, safety, tolerability, and efficacy study in adult patients with Gaucher disease Type 1.
- Trial name
- GALILEO-1 (NCT05324943)
- Testing
- FLT201
- Where
- 10 sites in United States, Brazil and 4 more
- Volunteers
- 10 people
- Sponsor
- Spur Therapeutics
Gaucher Disease, Type 1, Cerebroside Lipidosis Syndrome
Safety and Efficacy of Cerezyme® Infusions Every 4 Weeks Versus Every 2 Weeks in Type 1 Gaucher Disease
This is a multicenter, randomized trial to compare the safety and efficacy of two dosing frequencies of Cerezyme® in patients with Gaucher disease who are currently being treated with Cerezyme®.
- Conditions
- Gaucher Disease, Type 1, Cerebroside Lipidosis Syndrome, Glucocerebrosidase Deficiency Disease, Glucosylceramide Beta-Glucosidase Deficiency Disease
- Trial name
- NCT00364858
- Testing
- Cerezyme
- Where
- 26 sites in United States, Brazil and 5 more
- Volunteers
- 95 people
- Sponsor
- Genzyme, a Sanofi Company
Hepatic Encephalopathy
A Retrospective, Open-label, Uncontrolled Cohort Study to Assess the Effectiveness and Safety of Lactulose Retention Enema in the Treatment of Cirrhotic Patients With Grade 3 or 4 (West Haven Criteria) Hepatic Encephalopathy (HE) [The RETRO Study]
This is a retrospective study in adult patients with cirrhosis and grade 3 or 4 HE (West Haven Criteria) to evaluate the effectiveness and safety of lactulose retention enema in the treatment of deep grade HE (West Haven…
- Trial name
- NCT02377947
- Where
- 3 sites in India
- Volunteers
- 50 people
- Sponsor
- Abbott
GM1 Gangliosidoses, GM2 Gangliosidoses
Synergistic Enteral Regimen for Treatment of the Gangliosidoses
The investigators hypothesize that a combination therapy using miglustat and the ketogenic diet for infantile and juvenile patients with gangliosidoses will create a synergy that 1) improves overall survival for patients with…
- Conditions
- GM1 Gangliosidoses, GM2 Gangliosidoses, Tay-Sachs Disease, Sandhoff Disease
- Trial name
- Syner-G (NCT02030015)
- Testing
- miglustat, Ketogenic Diet
- Where
- United States
- Volunteers
- 16 people
- Sponsor
- University of Minnesota
Cushing Syndrome
A Study of the Efficacy and Safety of Relacorilant in Patients With Endogenous Cushing Syndrome
This is a Phase 3, double-blind, placebo-controlled, randomized-withdrawal study to assess the efficacy, safety and pharmacokinetics (PK) of relacorilant in patients with endogenous Cushing syndrome and concurrent type 2 diabetes…
- Trial name
- GRACE (NCT03697109)
- Testing
- Relacorilant
- Where
- 64 sites in United States, Austria and 9 more
- Volunteers
- 152 people
- Sponsor
- Corcept Therapeutics
Metachromatic Leukodystrophy (MLD)
Multicenter Study of HGT-1110 Administered Intrathecally in Children With Metachromatic Leukodystrophy (MLD)
The purpose of this study is to determine the safety of ascending doses of HGT-1110 administered by intrathecal (IT) injection for 38 weeks (20 injections) in children with metachromatic leukodystrophy (MLD).
- Trial name
- IDEAMLD (NCT01510028)
- Testing
- Recombinant human arylsulfatase A
- Where
- 5 sites in Australia, Denmark and 3 more
- Volunteers
- 24 people
- Sponsor
- Shire