Clinical trials: Brain Diseases, Metabolic
Phenylketonurias, Hyperphenylalaninaemia
PKU Low Calorie Drink Study
This study will evaluate the compliance, acceptability, gastrointestinal (GI) tolerance and safety of a lower calorie amino acid based liquid protein substitute for patients with Phenylketonuria (PKU) or hyperphenylalaninemia…
- Trial name
- NCT04272736
- Testing
- Low calorie protein substitute
- Sponsor
- Nutricia UK Ltd
Breast Cancer, Colo-rectal Cancer
The French E3N Prospective Cohort Study
The French E3N cohort was initiated in 1990 to investigate the risk factors associated with cancer and other major non-communicable diseases in women.
- Conditions
- Breast Cancer, Colo-rectal Cancer, Parkinson Disease, Asthma
- Trial name
- E3N (NCT03285230)
- Where
- France
- Volunteers
- 100,000 people, healthy volunteers welcome
- Sponsor
- Institut National de la Santé Et de la Recherche Médicale, France
Pompe Disease
Phrenic Nerve and Diaphragm Electrophysiology in Pompe Disease
Pompe disease is traditionally considered a lysosomal myopathy. However, increasing experimental and clinical evidence suggests involvement of the entire motor unit, including motor neurons, peripheral nerves, neuromuscular…
- Trial name
- NCT07664930
- Where
- Italy
- Volunteers
- 20 people
- Sponsor
- IRCCS National Neurological Institute "C. Mondino" Foundation
Gaucher Disease
Cellular and Molecular Mechanisms Governing Bone Marrow Stem Cells in Gaucher Disease
Gaucher disease is an inherited autosomal recessive lysosomal storage disorder caused by the defective activity of the glucocerebrosidase, leading to accumulation of glucocerebroside particularly in cells of the macrophage…
- Trial name
- MGMC (NCT01439607)
- Testing
- Bone marrow and blood sampling
- Where
- France
- Volunteers
- 30 people
- Sponsor
- Assistance Publique - Hôpitaux de Paris
Infantile Canavan Disease, Deficiency Disease, Aspartoacylase
GTA-Glyceryltriacetate for Canavan Disease
The purpose of this study is to determine whether oral supplementation of glyceryl triacetate improves the clinical prognosis of Canavan Disease.
- Trial name
- NCT00278707
- Testing
- GTA: Glyceryltriacetate
- Where
- Israel
- Volunteers
- 5 people
- Sponsor
- Sheba Medical Center
Epilepsy, Child, Only
The Effect of Levetiracetam on Lipid Profile in Children
Levetiracetam is a widely prescribed antiseizure medication in epileptic children due to an estimated better safety profile and easy accessibility.
- Conditions
- Epilepsy, Child, Only, Thyroid, Lipid Disorder
- Trial name
- NCT05276271
- Testing
- the blood sample test results
- Where
- Turkey (Türkiye)
- Volunteers
- 91 people
- Sponsor
- Ege University
Frontotemporal Dementia, Amyotrophic Lateral Sclerosis
Investigating Complex Neurodegenerative Disorders Related to Amyotrophic Lateral Sclerosis and Frontotemporal Dementia
Background: Neurodegenerative disorders can lead to problems in movement or memory. Some can cause abnormal proteins to build up in brain cells.
- Conditions
- Frontotemporal Dementia, Amyotrophic Lateral Sclerosis, Progressive Supranuclear Palsy
- Trial name
- NCT03225144
- Where
- United States
- Volunteers
- 360 people
- Sponsor
- National Institute of Neurological Disorders and Stroke (NINDS)
Gaucher Disease, Type 1
A Study of Eliglustat Tartrate (Genz-112638) in Patients With Gaucher Disease Who Have Reached Therapeutic Goals With Enzyme Replacement Therapy (ENCORE)
This Phase 3 study was designed to confirm the efficacy and safety of eliglustat tartrate (Genz-112638) in participants with Gaucher disease type 1 who had reached therapeutic goals with enzyme replacement therapy (ERT).
- Trial name
- ENCORE (NCT00943111)
- Testing
- Eliglustat tartrate, Imiglucerase
- Where
- 34 sites in United States, Argentina and 10 more
- Volunteers
- 160 people
- Sponsor
- Genzyme, a Sanofi Company
AMN, AMN Gene Mutation
A Study to Evaluate Administration of SBT101 Gene Therapy in Adult Patients With Adrenomyeloneuropathy (AMN)
This is a Phase 1/2 randomized, blinded, dose-escalation study to evaluate the safety and efficacy of intrathecal (IT) administration of SBT101, a recombinant adeno-associated virus serotype 9 (AAV9) containing a functional copy…
- Conditions
- AMN, AMN Gene Mutation, X-ALD
- Trial name
- PROPEL (NCT05394064)
- Testing
- SBT101 Treatment
- Where
- 2 sites in United States, Netherlands
- Volunteers
- 9 people
- Sponsor
- SwanBio Therapeutics, Inc.
Urea Cycle Disorders
Study to Evaluate the Efficacy of HepaStem in Urea Cycle Disorders Paediatric Patients (HEP002)
The aim of the study is to assess the efficacy of HepaStem treatment in paediatric patients suffering from urea cycle disorders.
- Trial name
- NCT02489292
- Testing
- HepaStem
- Where
- 6 sites in Belgium, France and 2 more
- Volunteers
- 5 people
- Sponsor
- Cellaion SA
Hepatic Encephalopathy
Rifaximin Plus Lactulose Versus Lactulose Alone for the Treatment of Hepatic Encephalopathy: a Double Blind Randomized Trial
The study will be double blind with respect to rifaximin, and randomization will be performed using tables of computer-generated random numbers.
- Trial name
- NCT01218568
- Testing
- Rifaximin plus lactulose, Lactulose
- Where
- India
- Volunteers
- 120 people
- Sponsor
- Institute of Liver and Biliary Sciences, India
Dementia, Abuse Client
Partnership in Safeguarding People With Dementia From Primary and Social Care Perspectives
"Adult safeguarding is everyone's responsibility". The Care Act 2014 makes safeguarding a legal duty. It provides local authorities and other agencies in England with a responsibility to protect adults with care and support needs…
- Conditions
- Dementia, Abuse Client, Neglect; Care, Malnutrition
- Trial name
- NCT05883527
- Testing
- Not applicable as not an intervention - PLwD and family/carers, Not applicable as not an intervention - HCP's
- Where
- 5 sites in United Kingdom
- Volunteers
- 37 people, healthy volunteers welcome
- Sponsor
- University of Manchester
Parkinson Disease, Ketosis
Effect of Ketone Esters in Parkinson's Disease
Ketones esters have shown to improve mitochondrial function and are currently use to enhanced functional performance. As Mitochondrial dysfunction is one of the proposed mechanism of neuronal injury in Parkinson's disease, the…
- Trial name
- NCT04477161
- Testing
- Ketone Ester Elite endurance Nutrition Drink, Stool Sample
- Where
- United States
- Volunteers
- 10 people
- Sponsor
- University of Florida
Parkinson
Impact of Exercise on Muscle Metabolism and Function for Persons With Parkinsons
This investigation assesses the impact of a strengthening program on muscle metabolism in the gastrocnemius muscle (GM) and is an essential step in the establishment of future clinical intervention for the GM in people with…
- Trial name
- NCT07720713
- Testing
- Home Exercise Program
- Where
- United States
- Volunteers
- 19 people, healthy volunteers welcome
- Sponsor
- University of Texas Southwestern Medical Center
Phenylketonuria
Study of a Phenylalanine Restricted Diet During Pregnancy to Prevent Symptoms in Offspring of Patients With Phenylketonuria
OBJECTIVES: I. Assess the impact of a phenylalanine restricted diet during pregnancy on symptoms in offspring of patients with phenylketonuria.
- Trial name
- NCT00006142
- Testing
- phenylalanine restricted diet
- Sponsor
- National Center for Research Resources (NCRR)
Obesity
Involvement of the Gut Microbiota-brain Cross-talk in the Loss of Eating Control
Overweight and obesity are increasingly prevalent worldwide. These bodyweight disorders are closely related to deficiencies in the control of food intake.
- Trial name
- GMBTalk-Food (NCT05646901)
- Where
- Spain
- Volunteers
- 116 people, healthy volunteers welcome
- Sponsor
- Institut d'Investigació Biomèdica de Girona Dr. Josep Trueta
Obesity
Activation of Brain Centers by Short-term Walnut Consumption in Obesity
The purposes of the study are: Study Aim 1. To assess the mechanisms underlying the biological effects of short-term walnut consumption on appetite with functional magnetic resonance imaging (fMRI) and assessing neurocognitive…
- Trial name
- NCT02673281
- Testing
- Walnut shake
- Where
- United States
- Volunteers
- 10 people, healthy volunteers welcome
- Sponsor
- Beth Israel Deaconess Medical Center
Alcohol Withdrawal, Lactic Acidosis
Alcohol: Thiamine and or Magnesium 1
Patients who suffer Alcohol Use Disorder (AUD) have a 30-80% incidence of thiamine deficiency causing Wernicke's Encephalopathy (WE).
- Conditions
- Alcohol Withdrawal, Lactic Acidosis, Vitamin B1 Deficiency, Magnesium Deficiency
- Trial name
- AToM1 (NCT03466528)
- Testing
- Magnesium Sulfate, Pabrinex
- Where
- United Kingdom
- Volunteers
- 127 people
- Sponsor
- Glasgow Royal Infirmary
Pompe Disease, Glycogen Storage Disease Type II
An Exploratory Study of the Safety and Efficacy of Prophylactic Immunomodulatory Treatment in Myozyme-naive Cross-Reacting Immunologic Material (CRIM[-]) Patients With Infantile-Onset Pompe Disease
The purpose of this study was to evaluate the efficacy, clinical benefits and safety of a prophylactic immunomodulatory regimen given prior to first treatment with alglucosidase alfa (Myozyme®) in patients with infantile-onset…
- Trial name
- NCT00701129
- Testing
- Alglucosidase Alfa, Methotrexate, Rituximab
- Where
- 2 sites in United States
- Volunteers
- 4 people
- Sponsor
- Genzyme, a Sanofi Company
Hepatic Encephalopathy, Urea Cycle Disorders
A Study of Glyceryl Tri-(4-phenylbutyrate) Administered Orally as a Single Dose, and Twice Daily for Seven Consecutive Days to Subjects With Hepatic Impairment With Cirrhosis and to a Control Group
The purpose of this study is to determine the safety and tolerability of GT4P administered orally as a single dose, and twice daily for 7 consecutive days, to subjects with hepatic impairment with cirrhosis (Child-Pugh scores of…
- Trial name
- NCT00986895
- Testing
- HPN-100
- Where
- 2 sites in Ukraine
- Volunteers
- 32 people
- Sponsor
- Amgen
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