Clinical trials: Brain Diseases, Metabolic
Fabry Disease
Global Burden and Treatment Trajectiories in Italian Patients With Fabry Disease
GROUND study is an Italian, multicenter retrospective longitudinal cohort study with a cross-sectional phase with the aim to quantify the severe clinical burden in terms of severe and fatal outcomes and extension of clinical…
- Trial name
- GROUND (NCT04916977)
- Testing
- FD patients
- Where
- 7 sites in Italy
- Volunteers
- 200 people
- Sponsor
- Chiesi Italia
Hereditary Tyrosinemia, Type I
Long Term Safety Study of Orfadin Treatment in HT-1 Patients in Standard Clinical Care
The purpose of this study is to look at the long term safety profile of Orfadin treatment in patients suffering from hereditary tyrosinemia type 1 (HT-1).
- Trial name
- OPAL (NCT02320084)
- Testing
- Nitisinone
- Where
- 77 sites in Austria, Belgium and 15 more
- Volunteers
- 315 people
- Sponsor
- Swedish Orphan Biovitrum
Mesenteric Ischemia
Microcirculation During Extracorporeal Circulation
Digestive tract is supposed to be the motor of multiple organ failure. In ICU patients, mesenteric ischemia appears even when macrocirculatory parameters are controlled especially in our experience in patients coming in ICU after…
- Trial name
- CITRON4 (NCT01389947)
- Where
- France
- Volunteers
- 66 people
- Sponsor
- Centre Hospitalier Universitaire de Besancon
Phenylketonurias
A Phase 3, Multicenter, Open-Label Extension Study of Phenoptin in Subjects With PKU Who Have Elevated Phenylalanine Levels
The primary objective of this trial is to evaluate the safety and tolerability of long-term Phenoptin treatment in subjects with PKU.
- Trial name
- NCT00225615
- Testing
- sapropterin dihydrochloride
- Where
- 14 sites in United States
- Volunteers
- 100 people
- Sponsor
- BioMarin Pharmaceutical
Parkinson's Disease, Gaucher's Disease
Intravenous N-acetylcysteine for the Treatment of Gaucher's Disease and Parkinson's Disease
The investigators are interested in determining if the investigators are able to detect changes in brain chemistry using Magnetic Resonance Spectroscopy (MRS) in individuals with Parkinson's disease (PD), those with Gaucher's…
- Trial name
- NCT01427517
- Testing
- N-acetylcysteine
- Where
- United States
- Volunteers
- 9 people, healthy volunteers welcome
- Sponsor
- University of Minnesota
Amyloid, Amyloid Cardiomyopathy
18F-florbetaben PET-CT to Non-invasively Diagnose Cardiac AL Amyloidosis
Amyloidoses are systemic or acquired disorders characterized by the deposition in the extracellular spaces of amyloid fibers formed by proteins codified by mutated genes or non-mutated but misfolded proteins.
- Trial name
- PETAL (NCT06048601)
- Testing
- 18F-florbetaben PET/CT
- Where
- Italy
- Volunteers
- 150 people
- Sponsor
- Fondazione Toscana Gabriele Monasterio
Alzheimer Disease, Alzheimer Dementia
Multimodal Ocular Imaging in Neurodegeneration
Alzheimer's disease (AD) and frontotemporal dementia (FTD) are two of the most common types of age-related neurodegenerative disorders.
- Conditions
- Alzheimer Disease, Alzheimer Dementia, Frontotemporal Dementia
- Trial name
- NCT03699644
- Testing
- Spectral-Domain Optical Coherence Tomography (SD-OCT), Magnetic Resonance Imaging (MRI), Positron Emission Tomography (PET)
- Where
- United States
- Volunteers
- 16 people, healthy volunteers welcome
- Sponsor
- University of Michigan
Bipolar I Disorder, Psychosis
Ketogenic and Nutritional Interventions for First Episode Bipolar Disorder
This is a randomized, controlled clinical trial to assess the effects of the ketogenic diet in combination with treatment as usual on brain energy metabolism and psychiatric symptoms in individuals with first episode bipolar…
- Conditions
- Bipolar I Disorder, Psychosis, Schizoaffective Disorder
- Trial name
- NCT06221852
- Testing
- Ketogenic diet, Dietary Guidelines for Americans
- Where
- United States
- Volunteers
- 50 people
- Sponsor
- Mclean Hospital
Sjogren-Larsson Syndrome (SLS)
Sjogren-Larsson Syndrome: Natural History, Clinical Variation and Evaluation of Biochemical Markers
Sjogren-Larsson syndrome (SLS) is a rare genetic disease in which patients typically exhibit ichthyosis (dry, scaly skin), intellectual disability, spasticity, seizures and a distinctive maculopathy.
- Trial name
- SLS (NCT01971957)
- Where
- 2 sites in United States
- Volunteers
- 20 people
- Sponsor
- University of Nebraska
Anovulation, FHA
FHA: Characterization of Metabolic Status, Brain Circuitry, and Stress-Reactivity
Functional Hypothalamic Amenorrhea (FHA), the spontaneous cessation of the menstrual cycle for at least 6 months after menstrual cyclicity has been established, is a common and reversible form of anovulation not due to…
- Conditions
- Anovulation, FHA, Eumenorrhea, Polycystic Ovary Syndrome
- Trial name
- NCT00453219
- Testing
- Screening evaluation, Meal Response, Blood Collection
- Where
- United States
- Volunteers
- healthy volunteers welcome
- Sponsor
- Emory University
Friedreich Ataxia
Safety and Efficacy of Etravirine in Friedreich Ataxia Patients
A drug repositioning effort provided evidence supporting the possible use of Etravirine, a drug approved for the treatment of HIV infections in patients starting from 2 years of age, as a treatment for FA.
- Trial name
- FAEST1 (NCT04273165)
- Testing
- Etravirine Tablets
- Where
- Italy
- Volunteers
- 30 people
- Sponsor
- IRCCS Eugenio Medea
Hereditary Cystatin C Amyloid Angiopathy (HCCAA), Hemorrhage Brain
Safety, Tolerability and Efficacy of NPI-001 in Patients With Hereditary Cystatin C Amyloid Angiopathy (HCCAA)
The study aims to measure safety, tolerability and biomarker-based efficacy of NPI-001 (AT-001) in Subjects with Hereditary Cystatin C Amyloid Angiopathy (HCCAA).
- Conditions
- Hereditary Cystatin C Amyloid Angiopathy (HCCAA), Hemorrhage Brain, Hemorrhage Cerebral
- Trial name
- HCCAA (NCT07716865)
- Testing
- NACA
- Where
- Iceland
- Volunteers
- 15 people
- Sponsor
- Arctic Therapeutics
Gaucher Disease
A Study of Eliglustat Tartrate (Genz-112638) in Patients With Gaucher Disease to Evaluate Once Daily Versus Twice Daily Dosing (EDGE)
The primary objective of this study was to evaluate the efficacy and safety of once daily (QD) versus twice daily (BID) dosing of eliglustat tartrate (Genz-112638) in participants with Gaucher disease type 1 who had demonstrated…
- Trial name
- NCT01074944
- Testing
- Eliglustat tartrate
- Where
- 46 sites in United States, Australia and 15 more
- Volunteers
- 170 people
- Sponsor
- Genzyme, a Sanofi Company
Phenylketonurias, Nutritional and Metabolic Diseases
Comparison of Atherogenic Risk Factors and Efficacy of Nutritional Treatment Among Adult Phenylketonuria Patients
Phenylketonuria is a rare metabolic disease that results from the absence or near-absence activity of the enzyme phenylalanine hydroxylase, which metabolizes the amino acid phenylalanine to tyrosine in the body.
- Trial name
- NCT04969809
- Testing
- Ultrasound of the jugular veins, addition of phenylalanine-free protein substitutes
- Volunteers
- 50 people
- Sponsor
- University Medical Centre Ljubljana
Phenylketonurias
Study to Evaluate the Effect on Nitrogen Retention of Two Different Posology Schemes of PKU GOLIKE PLUS 3-16 and Free AAs in Patients With Phenylketonuria
This is an open-label, randomized, 4-way crossover, monocentric, controlled study in patients (3 ≤ years of age ≤ 20 )) with phenylketonuria (PKU).
- Trial name
- NCT05229549
- Testing
- GOLIKE PLUS 3-16, Free AAs
- Where
- Italy
- Volunteers
- 5 people
- Sponsor
- APR Applied Pharma Research s.a.
Obesity
fMRI and Appetite-Related Hormones Pre and Post Obesity Surgery
The investigators are looking at the differences in appetite and obesity between weight loss participants and patients undergoing bariatric surgery.
- Trial name
- NCT01583725
- Testing
- Liquid meal responses, fMRI responses to food-cues
- Where
- United States
- Volunteers
- 120 people, healthy volunteers welcome
- Sponsor
- New York Obesity and Nutrition Research Center
Overweight and Obesity, Metabolic Syndrome
Changes in the Hypotalamic-pituitary Region of Patients With Metabolic Syndrome and Obesity
This study aims to identify new morphological and quantitative magnetic imaging parameters of pituitary gland and sellar region in overweight and obese patient at baseline and after 3 years, dividing patients in 3 groups (weight…
- Trial name
- CHIASM (NCT03458533)
- Testing
- Bariatric surgery, Diet and lifestyle changes
- Where
- Italy
- Volunteers
- 69 people
- Sponsor
- University of Roma La Sapienza
Glycogen Storage Disease Type II (GSD-II), Pompe Disease (Late-Onset)
Alglucosidase Alfa Temporary Access Program
Pompe disease (also known as glycogen storage disease Type II) is caused by a deficiency of a critical enzyme in the body called acid alpha-glucosidase (GAA).
- Conditions
- Glycogen Storage Disease Type II (GSD-II), Pompe Disease (Late-Onset), Acid Maltase Deficiency Disease, Glycogenosis 2
- Trial name
- ATAP (NCT00520143)
- Testing
- alglucosidase alfa (recombinant human acid alpha-glucosidase [rhGAA])
- Where
- 74 sites in United States
- Sponsor
- Genzyme, a Sanofi Company
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
X-Linked Adrenoleukodystrophy (X-ALD), Cerebral Adrenoleukodystrophy (CALD)
Observational Study to Evaluate Allogeneic HSCT Outcomes for Cerebral Adrenoleukodystrophy (CALD)
Study ALD-103 will be a multi-site, global, prospective and retrospective data collection study that is designed to evaluate outcomes of allo-HSCT in male subjects with CALD ≤17 years of age.
- Conditions
- X-Linked Adrenoleukodystrophy (X-ALD), Cerebral Adrenoleukodystrophy (CALD), Adrenoleukodystrophy (ALD)
- Trial name
- NCT02204904
- Testing
- Allo-HSCT
- Where
- 13 sites in United States, Argentina and 5 more
- Volunteers
- 59 people
- Sponsor
- Genetix Biotherapeutics Inc.
The first 1,020 results can be paged through. Add a filter or change the sort order to reach the rest.