Clinical trials: Brain Diseases, Metabolic
Wilson Disease
Study of Tetrathiomolybdate in Patients With Wilson Disease
OBJECTIVES: Evaluate the safety and efficacy of ammonium tetrathiomolybdate alone and compared with trientine therapy as initial treatment in patients with Wilson disease presenting neurologically.
- Trial name
- NCT00004339
- Testing
- tetrathiomolybdate, trientine
- Where
- United States
- Volunteers
- 90 people
- Sponsor
- National Center for Research Resources (NCRR)
Hypopituitarism, Pituitary Tumors
Effect of 5 Years of GH Replacement on Atherosclerosis
Adult patients with hypopituitarism under adequate conventional hormone replacement therapy have reduced life expectancy due to excess vascular events (1-4).
- Conditions
- Hypopituitarism, Pituitary Tumors, Growth Hormone Deficiency
- Trial name
- 5yrGH (NCT00462475)
- Testing
- Recombinant Growth Hormone, Genotropin (Pfizer)
- Where
- Italy
- Volunteers
- 20 people, healthy volunteers welcome
- Sponsor
- Federico II University
Ataxia Telangiectasia
NAD Supplementation to Prevent Progressive Neurological Disease in Ataxia Telangiectasia
The study investigates the effect of dietary supplementation of nicotinamide ribonucleoside (NR) in children with ataxia telangiectasia (AT), with main focus on neurological symptoms.
- Trial name
- NCT04870866
- Testing
- Nicotinamide ribonucleoside
- Where
- 2 sites in Norway
- Volunteers
- 13 people
- Sponsor
- University Hospital, Akershus
Urea Cycle Disorder, Crigler-Najjar Syndrome
HepaStem Long-Term Safety Registry
All patients having received at least one infusion of the Investigational Medicinal Product (IMP) HepaStem HHALPC during a previous interventional clinical study conducted by Promethera Biosciences
- Conditions
- Urea Cycle Disorder, Crigler-Najjar Syndrome, Acute on Chronic Liver Failure
- Trial name
- PROLONGSTEM (NCT03343756)
- Where
- 29 sites in Belgium, Bulgaria and 3 more
- Volunteers
- 44 people
- Sponsor
- Cellaion SA
Coronary Heart Disease, Stroke
Heart Disease on the Mend
The primary objective of this study was to evaluate the effectiveness of a multifactor cardiovascular disease (CVD) risk reduction program using team case management in high risk patients who have low family incomes and limited…
- Conditions
- Coronary Heart Disease, Stroke, Hyperlipidemia, Hypertension
- Trial name
- NCT00127751
- Testing
- Enhance health lifestyle changes and medication compliance
- Where
- United States
- Volunteers
- 150 people
- Sponsor
- Stanford University
Alzheimer Disease, Mild Cognitive Impairment
Brain Energy Metabolism and Sleep in Adults
The three primary goals of this pilot will be followed by a secondary goal to test if dental intervention improves brain health in terms of sleep and cognition. The primary and secondary goals are 1.
- Conditions
- Alzheimer Disease, Mild Cognitive Impairment, Cognitive Change, Energy Metabolism
- Trial name
- NCT03929302
- Testing
- MyTAP oral appliance plus mouth shield
- Where
- United States
- Volunteers
- 41 people
- Sponsor
- The University of Texas at Dallas
Cerebrovascular Accident, Diabetes
Impact of Diabetes and Glucose Control During Rehabilitation After Stroke
To assess whether patients with diabetes have less clinical improvement during inpatient rehabilitation than those without diabetes and whether hyperglycaemia during rehabilitation is an adverse prognostic indicator.
- Conditions
- Cerebrovascular Accident, Diabetes, Hyperglycemia
- Trial name
- NCT00241956
- Where
- Australia
- Volunteers
- 20 people
- Sponsor
- Melbourne Health
Phenylketonurias, PKU
Evaluation of the Express Plus Range
A prospective, open label, acceptability study to evaluate PKU, MSUD, HCU, TYR and GA express plus in the dietary management of 40 patients with IEM.
- Conditions
- Phenylketonurias, PKU, Homocystinuria, Maple Syrup Urine Disease
- Trial name
- express plus (NCT05051657)
- Testing
- PKU express plus
- Where
- 6 sites in United Kingdom
- Volunteers
- 28 people
- Sponsor
- Vitaflo International, Ltd
Gaucher Disease, Type 1
Long Term Impact of Rapid Intravenous Infusion of Velaglucerase Alfa (VPRIV)
Background: In order to allow our satisfied patients, who have successfully completed 24 months of rapid intravenous infusion of Velaglucerase alfa (VPRIV), to continue with the 10 minutes IV therapy, the clinical trial framework…
- Trial name
- NCT04120506
- Testing
- VPRIV
- Volunteers
- 15 people
- Sponsor
- Shaare Zedek Medical Center
Multiple System Atrophy (MSA)
Oligomeric Alpha-synuclein in Multiple System Atrophy
The main objectives are to determine on one hand whether oligomeric alpha-synuclein levels are increased in MSA patients compared to controls and on other hand whether there is a good agreement between cerebrospinal fluid (CSF)…
- Trial name
- BIOAMS (NCT01485549)
- Where
- France
- Volunteers
- 48 people
- Sponsor
- University Hospital, Bordeaux
Syndrome of Inappropriate Antidiuretic Hormone Secretion
A Multicenter Trial to Investigate the Efficacy and Safety of Tolvaptan in Patients With Hyponatremia in SIADH
The objective of this study is to investigate the efficacy and safety of tolvaptan based on the change in serum sodium concentration following administration of tolvaptan oral tablets at 7.5 to 60 mg/day for up to 30 days in…
- Trial name
- NCT03048747
- Testing
- Tolvaptan Oral Tablet
- Where
- 6 sites in Japan
- Volunteers
- 16 people
- Sponsor
- Otsuka Pharmaceutical Co., Ltd.
Syndrome of Inappropriate Antidiuretic Hormone Secretion (SIADH)
A PK Study of 3 Dosages of Tolvaptan in Patients With Syndrome of Inappropriate Antidiuretic Hormone Secretion (SIADH)
This is a study to evaluate how the body handles and metabolizes (PK) the various doses of the drug Tolvaptan, and what the effect (PD) of the various doses of Tolvaptan are on the content of "salt" in blood and urine
- Trial name
- NCT02009878
- Testing
- tolvaptan
- Where
- 14 sites in Czechia, Denmark and 5 more
- Volunteers
- 30 people
- Sponsor
- Otsuka Pharmaceutical Development & Commercialization, Inc.
Hepatic Encephalopathy, Minimal Brain Dysfunction
Minimal Hepatic Encephalopathy in Hereditary Hemorrhagic Telangiectasian
HHT or Rendu-Osler-Weber disease is a genetic disease with an autosomal dominant inheritance pattern, characterized by widespread telangiectases that can involve several organs including the intestinal tract and the liver.
- Trial name
- mHE-HHT (NCT03586115)
- Testing
- Critical flicker frequency assessment
- Where
- Italy
- Volunteers
- 50 people
- Sponsor
- University of Bari
Diabetes Mellitus
Sex Differences in the Risk of Cardiovascular Diseases Associated With Diabetes
To evaluate the risk of cardiovascular events associated with medication use for controlling blood glucose, blood pressure and cholesterol levels in men and women with diabetes treated in primary care
- Trial name
- NCT02542774
- Testing
- No intervention is given.
- Where
- United Kingdom
- Volunteers
- 80,000 people, healthy volunteers welcome
- Sponsor
- University College, London
Pelizaeus-Merzbacher Disease
Orbit Study: A Study to Evaluate the Safety, Pharmacokinetics, and Pharmacodynamics of Intrathecally Administered ION356 in Participants With Pelizaeus Merzbacher Disease (PMD)
The primary purpose of this study is to evaluate the safety and tolerability of ION356.
- Trial name
- NCT06150716
- Testing
- ION356
- Where
- 7 sites in United States, France and 3 more
- Volunteers
- 24 people
- Sponsor
- Ionis Pharmaceuticals, Inc.
Wilson Disease
French Wilson Disease Registry
This registry concerns adults and children with Wilson's disease. The collection of a large amount of data will allow a better understanding of the epidemiology of this rare disease, in particular the age of onset according to…
- Trial name
- WIL-FR (NCT05231876)
- Testing
- Recording of pathology-related information on the Wilson Register
- Where
- France
- Volunteers
- 1,000 people
- Sponsor
- Fondation Ophtalmologique Adolphe de Rothschild
Hepatic Encephalopathy, HE
Hepatic Encephalopathy: Lactulose or Polyethylene Glycol (H.E.L.P.)
The purpose of this study is to evaluate whether the use of polyethylene glycol is superior and more safe in treating hepatic encephalopathy compared to lactulose and also to determine if treatment with polyethylene glycol will…
- Conditions
- Hepatic Encephalopathy, HE, Cirrhosis, Altered Mental Status
- Trial name
- HELP (NCT01923376)
- Testing
- Lactulose, Polyethylene Glycol 3350
- Where
- United States
- Sponsor
- New York Presbyterian Brooklyn Methodist Hospital
Fabry Disease
A Qualitative Study on the Pain and Quality of Life of Patients With Fabry Disease
Background It is necessary to find out the difficulties experienced by patients with Fabry disease and their families, and to seek solutions for them.
- Trial name
- NCT05679076
- Where
- South Korea
- Volunteers
- 10 people
- Sponsor
- Yonsei University
POCD - Postoperative Cognitive Dysfunction, Mechanical Bowel Preparation
Investigation of the Effect of Preoperative Bowel Cleansing on Postoperative Cognitive Impairment
Cognitive impairment has been reported up to 54 % after colorectal surgeries. Mechanical bowel cleansing means that a system called the second brain in the body, which includes a wide variety of genetic and neuronal structures…
- Trial name
- NCT05942404
- Testing
- mechanical bowel preparation
- Where
- Turkey (Türkiye)
- Volunteers
- 84 people
- Sponsor
- TC Erciyes University
Diabetes Mellitus, Type 2, Arterial Hypertension
Antidiuretic Function Before and During Treatment With SGLT2 Inhibitors
Subjects treated with Canagliflozin, Dapagliflozin and Empagliflozin obtained improvement on blood pressure values, body weight and cardiovascular mortality but pathophysiological explanations of these effects are not yet known.
- Conditions
- Diabetes Mellitus, Type 2, Arterial Hypertension, Body Weight Changes
- Trial name
- GliRACo1 (NCT03917758)
- Testing
- SGLT2 inhibitor
- Where
- Italy
- Volunteers
- 30 people
- Sponsor
- University of Turin, Italy
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