Clinical trials: Leukoencephalopathies
Adrenoleukodystrophy
Newborn Screening for Adrenoleukodystrophy
To test if the routine newborn screening dried blood spots can be used to test if elevation of C26:0 lysophosphatidylcholine (C26:0-lyso-PC), a status indicating adrenoleukodystrophy (ALD)
- Trial name
- NCT02952482
- Testing
- newborns testing for ALD
- Where
- Taiwan
- Volunteers
- 45,796 people, healthy volunteers welcome
- Sponsor
- National Taiwan University Hospital
Inherited Mitochondrial Disease
A Safety Study for Previously Treated Vatiquinone (PTC743) Participants With Inherited Mitochondrial Disease
The primary objective of this study is to assess the safety of vatiquinone in participants with inherited mitochondrial disease who had prior exposure to vatiquinone in a PTC/BioElectron sponsored (previously Edison) clinical…
- Trial name
- NCT05218655
- Testing
- Vatiquinone
- Where
- 26 sites in United States, France and 5 more
- Volunteers
- 101 people
- Sponsor
- PTC Therapeutics
Posterior Reversible Encephalopathy Syndrome, Inflammatory Biomarkers
Inflammatory Biomarkers for the Diagnosis of oPRES
The purpose of this study is to explore the inflammatory biomarkers for the diagnosis of obstetric posterior reversible encephalopathy syndrome.
- Conditions
- Posterior Reversible Encephalopathy Syndrome, Inflammatory Biomarkers, Neutrophil-lymphocyte Ratio
- Trial name
- NCT05433870
- Testing
- Whether the PE or E patient combined with PRES
- Where
- China
- Volunteers
- 621 people
- Sponsor
- Dunjin Chen
Dementia, Neurodegenerative Diseases
Retinal Hyperspectral Imaging in Neurodegenerative Diseases
Hyperspectral retinal imaging is a non-invasive imaging modality in which a series of images of the retina are captured using light of different wavelengths.
- Conditions
- Dementia, Neurodegenerative Diseases, Alzheimer Disease, Parkinson Disease
- Trial name
- NCT07545473
- Testing
- Hyperspectral camera
- Where
- Australia
- Volunteers
- 930 people, healthy volunteers welcome
- Sponsor
- Center for Eye Research Australia
X-Linked Adrenoleukodystrophy
Safety, Pharmacokinetics and Pharmacodynamics of NV1205 in Pediatric Male Subjects With Adrenoleukodystrophy
This is a phase I/II, open label dose escalation study of multiple dose levels of NV1205 with a long-term treatment phase.
- Trial name
- NCT03196765
- Testing
- Sobetirome (NV1205)
- Where
- 13 sites in Argentina, Australia and 6 more
- Sponsor
- NeuroVia, Inc.
Hereditary Spastic Paraplegia, Adrenomyeloneuropathy
Repetitive Transcranial Magnetic Stimulation as Therapy in Hereditary Spastic Paraplegia and Adrenomyeloneuropathy
Hereditary spastic paraplegia (HSP) is the group of inherited disorders, characterized by progressive gait disturbance. There is no established therapy. Adrenoleukodystrophy (AMN) is an x-linked hereditary disease.
- Trial name
- NCT03627416
- Testing
- rTMS
- Where
- Poland
- Volunteers
- 15 people
- Sponsor
- Jakub Antczak
Multiple Sclerosis, Neuromyelitis Optica
Longitudinal Cortical Demyelination in Multiple Sclerosis and Related Disorders
In this protocol, a combination of MRI, blood, and cerebrospinal fluid (CSF) analysis will be used to understand the natural history, underlying immunologic mechanisms, and clinical implications of central nervous system (CNS)…
- Conditions
- Multiple Sclerosis, Neuromyelitis Optica, Demyelinating Autoimmune Diseases, CNS
- Trial name
- NCT05982925
- Where
- United States
- Volunteers
- 170 people, healthy volunteers welcome
- Sponsor
- Icahn School of Medicine at Mount Sinai
Adrenoleukodystrophy
Randomized Study of Beta Interferon and Thalidomide in Patients With Adrenoleukodystrophy
OBJECTIVES: I. Evaluate the efficacy of interferon beta and thalidomide in male patients with adrenoleukodystrophy who show evidence of brain inflammatory response and are receiving concurrent glyceryl trierucate and glyceryl…
- Trial name
- NCT00004450
- Testing
- glyceryl trierucate, glyceryl trioleate, interferon beta
- Volunteers
- 60 people
- Sponsor
- FDA Office of Orphan Products Development
Irregular Sleep Wake Rhythm Disorder
A Study to Determine the Signs and Symptoms That Impact Daily Life of Participants With Irregular Sleep-Wake Rhythm Disorder
The primary purpose of this study is to determine the signs and symptoms of irregular sleep-wake rhythm disorder (ISWRD) that are important to patients and caregivers of patients with Alzheimer's disease dementia (AD-D), AD-D…
- Trial name
- NCT04300569
- Testing
- Non-interventional
- Where
- 6 sites in United States
- Volunteers
- 37 people
- Sponsor
- Eisai Inc.
X-linked Adrenoleukodystrophy
Exercise Study of Function and Pathology for Women With X-linked Adrenoleukodystrophy
The purpose is to see how X-linked adrenoleukodystrophy (X-ALD) is associated with strength and sensation using MRI, in women with X-ALD.
- Trial name
- NCT01594853
- Testing
- exercise training
- Where
- United States
- Volunteers
- 31 people, healthy volunteers welcome
- Sponsor
- Hugo W. Moser Research Institute at Kennedy Krieger, Inc.
Dementia Alzheimers, Dementia
Detecting Dementia in the Retina Using Optical Coherence Tomography
This retrospective case control explores the retinal features of dementia associated with neurodegenerative diseases, particularly Alzheimer's disease.
- Conditions
- Dementia Alzheimers, Dementia, Dementia, Vascular, Dementia Senile
- Trial name
- NCT03631069
- Where
- United Kingdom
- Volunteers
- 280,000 people, healthy volunteers welcome
- Sponsor
- Moorfields Eye Hospital NHS Foundation Trust
Multiple Sclerosis, Primary Progressive Multiple Sclerosis
Metformin Add-on Clinical Study in Multiple Sclerosis to Evaluate Brain Remyelination And Neurodegeneration
This clinical trial aims to demonstrate that metformin can prevent clinical disability in patients with progressive MS by stopping or slowing down neurodegeneration by enhancing endogenous remyelination.
- Conditions
- Multiple Sclerosis, Primary Progressive Multiple Sclerosis, Secondary-progressive Multiple Sclerosis
- Trial name
- NCT05893225
- Testing
- Metformin Hydrochloride 850 mg Oral Tablet
- Where
- 5 sites in Belgium
- Volunteers
- 120 people
- Sponsor
- University Hospital, Antwerp
Hurler Syndrome (MPS I), Hurler-Scheie Syndrome
Phase I/II Pilot Study of Mixed Chimerism to Treat Inherited Metabolic Disorders
The goal of this research study is to establish chimerism and avoid graft-versus-host-disease (GVHD) in patients with inherited metabolic disorders.
- Conditions
- Hurler Syndrome (MPS I), Hurler-Scheie Syndrome, Hunter Syndrome (MPS II), Sanfilippo Syndrome (MPS III)
- Trial name
- NCT01372228
- Testing
- hematopoietic stem cell infusion
- Where
- United States
- Volunteers
- 3 people
- Sponsor
- Talaris Therapeutics Inc.
Neurological Impairments, Leukoencephalopathies
Hyperbaric Oxygen Treatment in Patients With White Matter Hyperintensities
The primary purpose of this study is to assess whether hyperbaric oxygen treatment improves neurological function in patients who exhibit white matter hyperintensities on MRI examination.
- Trial name
- NCT00497432
- Where
- 2 sites in United States
- Volunteers
- 20 people
- Sponsor
- St. Luke's Hospital, Chesterfield, Missouri
Dementia, Alzheimer Disease
Efficacy Study of Kinto Care Coaching for Dementia Family Caregivers
The goal of this clinical trial is to evaluate Kinto's Care Coaching intervention for dementia caregivers. The main questions it aims to answer are: Does the intervention help caregivers to address their general caregiving goals…
- Conditions
- Dementia, Alzheimer Disease, Lewy Body Disease, Vascular Dementia
- Trial name
- NCT05916664
- Testing
- Kinto Care Coaching
- Where
- United States
- Volunteers
- 495 people, healthy volunteers welcome
- Sponsor
- Kinto
Lysosomal Storage Disorders, Sandhoff Disease
Safety, Tolerability, Pharmacokinetics, and Preliminary Efficacy of PLX-200 in Pediatric Patients (Master Protocol)
The purpose of this study is to evaluate the safety, tolerability and clinical activity of PLX-200 in pediatric patients with lysosomal storage disorders.
- Conditions
- Lysosomal Storage Disorders, Sandhoff Disease, Krabbe Disease, CLN2
- Trial name
- NCT07740512
- Testing
- PLX-200
- Volunteers
- 24 people
- Sponsor
- Polaryx Therapeutics, Inc.
Alzheimer's Disease, Dementia With Lewy Bodies
Imaging of Brain Amyloid Plaques in the Aging Population
This is a prospective, open label, non-therapeutic, diagnostic imaging study. The purpose of this study is to utilize Pittsburgh Compound B positron emission imaging (PiB PET) to ascertain the relationship between change in…
- Conditions
- Alzheimer's Disease, Dementia With Lewy Bodies, Frontotemporal Dementia, Vascular Dementia
- Trial name
- NCT00950430
- Testing
- Pittsburgh Compound B (C-11 PiB), F-18 FDG, Tau (18-F-AV-1451)
- Where
- United States
- Volunteers
- 8,000 people, healthy volunteers welcome
- Sponsor
- Mayo Clinic
Thalassemia, Sickle Cell Disease
Cerebrovascular Reserve and White Matter Disease in Patients with Chronic Anemia
This is primarily an observational trial in patients with chronic anemia syndromes (sickle cell disease and thalassemia) and control subjects.
- Conditions
- Thalassemia, Sickle Cell Disease, Healthy Controls
- Trial name
- CVR (NCT03715972)
- Testing
- Hydroxyurea, Acetazolamide
- Where
- United States
- Volunteers
- 165 people, healthy volunteers welcome
- Sponsor
- Children's Hospital Los Angeles
Multiple Sclerosis, Pathologic Processes
Ukulele Playing to Improve Cognition in People with Multiple Sclerosis: a Feasibility Study
Over the past 10 years, the rates of multiple sclerosis (MS) have nearly doubled in the United States. This chronic, neuroinflammatory, and neurodegenerative disease is most often diagnosed between the ages of 20-40.
- Conditions
- Multiple Sclerosis, Pathologic Processes, Demyelinating Autoimmune Diseases, CNS, Autoimmune Diseases of the Nervous System
- Trial name
- NCT05792176
- Testing
- Music Training Intervention, Music Listening
- Where
- United States
- Volunteers
- 29 people
- Sponsor
- University of Texas at Austin
Multiple Sclerosis, NMO Spectrum Disorder
China National Registry of Neuro-Inflammatory Diseases
Central nervous system (CNS) idiopathic inflammatory demyelinating diseases (IDD) are mainly diseases caused by autoimmune factors that result in CNS demyelination damage and loss.
- Conditions
- Multiple Sclerosis, NMO Spectrum Disorder, Clinically Isolated Syndrome, CNS Demyelinating Autoimmune Diseases
- Trial name
- CNRID (NCT05154370)
- Testing
- Intravenous steroid
- Where
- China
- Volunteers
- 10,000 people
- Sponsor
- Beijing Tiantan Hospital