Clinical trials: Brain Disorder, Metabolic
Niemann-Pick Disease, Type C
Study of IV VTS-270 for Infantile Liver Disease Associated With Niemann-Pick Disease, Type C
Niemann-Pick disease, type C (NPC) is a lethal, autosomal recessive, lysosomal storage disorder characterized by neurodegeneration in early childhood and death in adolescence.
- Trial name
- NCT03471143
- Testing
- VTS-270
- Where
- United States
- Volunteers
- 4 people
- Sponsor
- Washington University School of Medicine
Diabetes, Effects of Liraglutide Administration on Brain Activity
Liraglutide in Obesity and Diabetes: Identification of CNS Targets Using fMRI
The main purpose of this study is to help us understand the effects of diabetes medication Liraglutide on weight loss and hunger.
- Conditions
- Diabetes, Effects of Liraglutide Administration on Brain Activity, Weight Loss, Hunger
- Trial name
- NCT01562678
- Testing
- Liraglutide
- Where
- United States
- Volunteers
- 28 people
- Sponsor
- Beth Israel Deaconess Medical Center
Gaucher Disease, Type 3
Efficacy and Safety Study of Velaglucerase Alfa in Children and Adolescents With Type 3 Gaucher Disease
Gaucher disease is a rare lysosomal storage disorder caused by the deficiency of the enzyme glucocerebrosidase (GCB). Gaucher disease has been classified into 3 clinical subtypes based on the presence or absence of neurological…
- Trial name
- NCT01685216
- Testing
- velaglucerase alfa
- Where
- 5 sites in Egypt, India and 1 more
- Volunteers
- 7 people
- Sponsor
- Shire
Abdominal Obesity, Growth Hormone Deficiency
Effectiveness of Growth Hormone Releasing Hormone in Reducing Abdominal Fat in People Who Are Obese
Obesity, a condition that occurs when a person has too much body fat, affects about 31% of people in the United States. It is associated with increased risk of diabetes, high blood pressure, high cholesterol, and cardiovascular…
- Trial name
- NCT00675506
- Testing
- Growth hormone releasing hormone (GHRH) 1-44
- Where
- United States
- Volunteers
- 60 people
- Sponsor
- Massachusetts General Hospital
Niemann-Pick Disease, Type C
VTS-270 to Treat Niemann-Pick Type C1 (NPC1) Disease
Due to different study designs, the sponsor separated Part C into a separate registration (NCT04958642), leaving Parts A/B here in NCT02534844.
- Trial name
- NCT02534844
- Testing
- Parts A/B: Adrabetadex
- Where
- 29 sites in United States, Australia and 7 more
- Volunteers
- 56 people
- Sponsor
- Mandos LLC
Atrial Fibrillation, Obesity
Effectiveness And Safety Of Oral Anticoagulants Among Obese Patients With Non-Valvular A-Fib In VA Patients With Medicare
The overall objective of this analysis is to understand patient characteristics, the use of treatment, and clinical outcomes among obese (overweight) and severely obese patients with non-valvular atrial fibrillation (NVAF) who…
- Conditions
- Atrial Fibrillation, Obesity, Anticoagulants
- Trial name
- NCT04681482
- Testing
- Apixaban
- Where
- United States
- Volunteers
- 107,383 people
- Sponsor
- Pfizer
Friedreich Ataxia
A Study to Learn About the Effects and Safety of RTA 408 (Omaveloxolone) in People Aged 16 to 40 With Friedreich's Ataxia
In this study, researchers are learning more about RTA 408, also known as omaveloxolone, BIIB141, or SKYCLARYS®. The main goal of this study is to learn more about the safety of RTA 408 and how it affects physical effort…
- Trial name
- NCT02255435
- Testing
- Omaveloxolone Capsules, 2.5 mg, Omaveloxolone Capsules, 5 mg, Omaveloxolone Capsules, 10 mg
- Where
- 11 sites in United States, Australia and 3 more
- Volunteers
- 172 people
- Sponsor
- Biogen
Fabry Disease
Study of the Safety and Efficacy of PRX-102 Compared to Agalsidase Beta on Renal Function
This was a randomized, double-blind, active control study of the enzyme replacement therapy (ERT) drug PRX-102 (pegunigalsidase alfa) in Fabry disease patients with impaired renal function.
- Trial name
- BALANCE (NCT02795676)
- Testing
- PRX-102 (pegunigalsidase alfa), agalsidase beta
- Where
- 29 sites in United States, Czechia and 10 more
- Volunteers
- 78 people
- Sponsor
- Protalix
Wilson Disease
Study of ALXN1840 on the Metabolism of a CYP2B6 Substrate in Healthy Participants
This is a Phase 1, randomized, 2-period, 2-sequence, cross-over study designed to determine the effect of ALXN1840 on the metabolism of bupropion, a sensitive cytochrome P450 2B6 (CYP2B6) substrate, in healthy male and female…
- Trial name
- NCT04526210
- Testing
- ALXN1840, Bupropion Hydrochloride
- Where
- United States
- Volunteers
- 54 people, healthy volunteers welcome
- Sponsor
- Alexion Pharmaceuticals, Inc.
Parkinson Disease
Safety and Efficacy of Liraglutide in Parkinson's Disease
The purpose of this study is to test the efficacy and safety of liraglutide in the treatment of patients with idiopathic Parkinson's disease (PD).
- Trial name
- NCT02953665
- Testing
- Liraglutide
- Where
- United States
- Volunteers
- 63 people
- Sponsor
- Cedars-Sinai Medical Center
Dementia, Cognitive Dysfunction
A Relational Research Recruitment and Engagement Intervention for Cognitive Aging Research
Despite well-documented disparities in Alzheimer's disease and related dementia (AD) prevalence, incidence, treatment, and mortality, individuals from disadvantaged backgrounds (e.g.
- Conditions
- Dementia, Cognitive Dysfunction, Alzheimer Disease, Lewy Body Disease
- Trial name
- NCT05444244
- Testing
- Relational Research Recruitment and Engagement Intervention
- Where
- United States
- Volunteers
- 182 people, healthy volunteers welcome
- Sponsor
- University of Wisconsin, Madison
Subarachnoid Hemorrhage
Statins and Cerebral Blood Flow in Subarachnoid Hemorrhage (SAH)
The primary objective of this project is to investigate the effect of statin therapy on cerebral blood flow in patients with aneurysmal SAH who are randomized to receive or not receive statins in a blinded design.
- Trial name
- NCT00795288
- Testing
- Simvastatin, 80 mg/day for 21 days
- Where
- United States
- Volunteers
- 42 people
- Sponsor
- Washington University School of Medicine
Cushing's Disease, Acromegaly
Study of Management of Pasireotide-induced Hyperglycemia in Adult Patients With Cushing's Disease or Acromegaly
The study was designed to investigate the optimal management of hyperglycemia developed during pasireotide treatment in participants with Cushing's disease or Acromegaly, which was not manageable with metformin.
- Trial name
- NCT02060383
- Testing
- Pasireotide s.c., Sitagliptin, Liraglutide
- Where
- 43 sites in United States, Belgium and 10 more
- Volunteers
- 249 people
- Sponsor
- Novartis Pharmaceuticals
Argininosuccinic Aciduria, Amino Acid Metabolism, Inborn Errors
Arginine and Buphenyl in Patients With Argininosuccinic Aciduria (ASA), a Urea Cycle Disorder
Urea cycle disorders are inherited illnesses in which the body does not produce enough of the chemicals that remove ammonia, a byproduct of protein metabolism, from the blood stream.
- Conditions
- Argininosuccinic Aciduria, Amino Acid Metabolism, Inborn Errors, Urea Cycle Disorders
- Trial name
- NCT00345605
- Testing
- Sodium Phenylbutyrate, Arginine
- Where
- United States
- Volunteers
- 12 people
- Sponsor
- Brendan Lee
Pain, Neuropathic, Epilepsy
Cancer in Patients With Gabapentin (GPRD)
High doses of gabapentin are associated with pancreatic acinar cell tumors in rats, but there has been no post marketing pancreatic carcinogenicity signal with gabapentin as reported by spontaneous reports in AERS or in the…
- Conditions
- Pain, Neuropathic, Epilepsy, Renal Pelvis Cancer, Pancreatic Cancer
- Trial name
- NCT01236053
- Testing
- Gabapentin prescriptions
- Volunteers
- 2,323,608 people
- Sponsor
- GlaxoSmithKline
Sphingomyelin Lipidosis
Safety, Tolerability, PK, and Efficacy Evaluation of Repeat Ascending Doses of Olipudase Alfa in Pediatric Patients <18 Years of Age With Acid Sphingomyelinase Deficiency
Primary Objective: To evaluate the safety and tolerability of olipudase alfa administered intravenously in pediatric participants every 2 weeks for 64 weeks.
- Trial name
- ASCEND-Peds (NCT02292654)
- Testing
- Olipudase alfa
- Where
- 6 sites in United States, Brazil and 4 more
- Volunteers
- 20 people
- Sponsor
- Genzyme, a Sanofi Company
Obesity, Healthy Volunteers
Brain Dopamine Function in Human Obesity
Background: Dopamine is a natural chemical in the brain that may influence eating behavior and physical activity. Researchers want to measure the brain s dopamine activity and understand how it differs in people with obesity.
- Conditions
- Obesity, Healthy Volunteers, Overweight
- Trial name
- NCT03648892
- Testing
- [c11] raclopride, [18F]fallypride
- Where
- United States
- Volunteers
- 61 people, healthy volunteers welcome
- Sponsor
- National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)
Urea Cycle Disorders
Study of the Safety and Tolerability of HPN-100 Compared to Sodium Phenylbutyrate in Children With Urea Cycle Disorders
Protocol HPN-100-005 was the first study of HPN-100 in pediatric subjects with urea cycle disorders (UCDs) and was a fixed-sequence, open-label, switch over study of HPN-100 with a long-term (12 month) safety extension designed…
- Trial name
- NCT00947544
- Testing
- HPN-100, NaPBA
- Where
- 8 sites in United States, Canada
- Volunteers
- 17 people
- Sponsor
- Amgen
Insulin Resistance, Obesity
Metformin Effect on Brain Function in Insulin Resistant Elderly People
Alzheimer's disease (AD) and other forms of dementia are rapidly increasing with the aging of the population, and show a clear preponderance among people with insulin resistance.
- Conditions
- Insulin Resistance, Obesity, Obesity, Abdominal
- Trial name
- NCT03733132
- Testing
- Metformin Hydrochloride
- Where
- United States
- Volunteers
- 40 people, healthy volunteers welcome
- Sponsor
- Mayo Clinic
Cirrhosis
A Trial to Improve Quality of Life in People With Cirrhosis
This trial is being completed to evaluate whether a crystallized form of lactulose (Kristalose) will improve quality of life, sleep and cognitive function in patients with cirrhosis that have not been diagnosed with Hepatic…
- Trial name
- NCT05061992
- Testing
- Lactulose
- Where
- United States
- Volunteers
- 56 people
- Sponsor
- University of Michigan