Clinical trials: Leukoencephalopathies
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
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
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, 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
Adrenoleukodystrophy, Metachromatic Leukodystrophy
HSCT for High Risk Inherited Inborn Errors
Hematopoietic stem cell transplantation has proven effective therapy for individuals with adrenoleukodystrophy (ALD), metachromatic leukodystrophy (MLD) or globoid cell leukodystrophy (GLD, or Krabbe disease).
- Conditions
- Adrenoleukodystrophy, Metachromatic Leukodystrophy, Globoid Cell Leukodystrophy, Tay Sachs Disease
- Trial name
- NCT00383448
- Testing
- Clofarabine, Total body Irradiation, Melphalan
- Where
- United States
- Volunteers
- 38 people
- Sponsor
- Masonic Cancer Center, University of Minnesota
Dementia, Dementia, Vascular
Palliative Care for Persons With Late-stage Alzheimer's and Related Dementias and Their Caregivers
Millions of Americans have late-stage Alzheimer's and related dementias (ADRD), causing suffering due to loss of awareness of self and family, progressive dependency, physical and neuropsychiatric symptoms, and physical…
- Conditions
- Dementia, Dementia, Vascular, Dementia With Lewy Bodies, Dementia Frontal
- Trial name
- ADRD-PC (NCT04948866)
- Testing
- ADRD-PC Program
- Where
- 5 sites in United States
- Volunteers
- 884 people
- Sponsor
- University of North Carolina, Chapel Hill
Alzheimer's Disease, Dementia
Optimizing Dementia Care
The purpose of this randomized controlled pilot study is to examine the preliminary effectiveness, feasibility, and potential treatment moderators (i.e., behavioral symptoms and spousal relationship status) of a newly developed…
- Conditions
- Alzheimer's Disease, Dementia, Dementia, Vascular, Caregivers
- Trial name
- ODeC (NCT02585232)
- Testing
- Counseling (C), Care Consultation (CC)
- Where
- United States
- Volunteers
- 86 people
- Sponsor
- VA Office of Research and Development
Depression, Stress, Psychological
Rural Dementia Caregiver Project
Rural caregivers face challenges of geographic isolation and lower health care access and quality. Many rural dementia caregivers experience serious health consequences due to caregiving responsibilities that can limit their…
- Conditions
- Depression, Stress, Psychological, Self Efficacy, Loneliness
- Trial name
- NCT04428112
- Testing
- Building Better Caregivers Workshop, Attention Control
- Where
- United States
- Volunteers
- 418 people, healthy volunteers welcome
- Sponsor
- University of California, San Francisco
Infantile Refsum's Disease, Zellweger Syndrome
Compassionate Treatment of Patients With Inborn Errors of Bile Acid Metabolism With Cholic Acid
OBJECTIVES: I. To Evaluate the therapeutic efficacy of cholic acid during provision of compassionate treatment to patients with identified inborn errors of bile acid synthesis and metabolism II.
- Conditions
- Infantile Refsum's Disease, Zellweger Syndrome, Adrenoleukodystrophy, Peroxisomal Disorders
- Trial name
- NCT00007020
- Testing
- Cholic Acids
- Where
- United States
- Volunteers
- 85 people
- Sponsor
- Mirum Pharmaceuticals, Inc.
Alzheimer's Disease, Vascular Dementia
Non-Interventional Study With Aricept® Evess
The Aricept® Evess study is a prospective, non-comparative, non-interventional study on use of Aricept® Evess in the treatment of out-patients with AD and Vascular Dementia.
- Trial name
- NCT00889603
- Testing
- Aricept® Evess
- Where
- 12 sites in Romania
- Volunteers
- 370 people
- Sponsor
- Pfizer
Alzheimer's Disease or Vascular Dementia
A Study to Evaluate Efficacy and Safety of BAC in Patient With Alzheimer's Disease or Vascular Dementia
A Randomized, Double-Blind, Vehicle-Controlled, Parallel, Phase II Study to Evaluate Efficacy and Safety of BAC in Patient with Alzheimer's Disease or Vascular Dementia
- Trial name
- NCT02886494
- Testing
- BAC treatment, Matched vehicle
- Where
- 10 sites in United States
- Volunteers
- 80 people
- Sponsor
- Charsire Biotechnology Corp.
Immune Reconstitution Inflammatory Syndrome, Leukoencephalopathy, Progressive Multifocal
Corticosteroids for Immune Reconstitution Inflammatory Syndrome (IRIS)
The objectives of this study are to explore the effects of administering high-dose corticosteroids to participants who developed progressive multifocal leukoencephalopathy (PML) while on natalizumab as measured by time-course…
- Trial name
- IRIS (NCT01211665)
- Testing
- Methylprednisolone, Prednisolone
- Where
- 3 sites in United States, Germany
- Volunteers
- 3 people
- Sponsor
- Biogen
Dementia, Alzheimer Disease
All About Me: An Intervention to Ease the Transition to Long Term Care
This Phase I study will involve initial development and evaluation of an innovative cross-platform software app called All About Me (AAM), which will consist of Serious Digital Health Games (SDHGs) for PWD.
- Conditions
- Dementia, Alzheimer Disease, Dementia, Vascular, Dementia, Mixed
- Trial name
- AAM (NCT05516134)
- Testing
- SAFE at Home
- Where
- United States
- Volunteers
- 51 people
- Sponsor
- Hopeful Aging
Late Infantile Metachromatic Leukodystrophy
Long-term Metazym Treatment of Patients With Late Infantile Metachromatic Leukodystrophy (MLD)
This is a single center, open-label study of patients with late infantile MLD. All patients were previous treated 26 weeks in the phase I trial (EudraCT number: 2006-005341-11, NCT00418561).
- Trial name
- NCT00633139
- Testing
- Recombinant human Arylsulfatase A (rhASA)
- Where
- Denmark
- Volunteers
- 13 people
- Sponsor
- Shire
Mild Cognitive Impairment
Rehabilitation of Attention in Patients With MCI and Brain Subcortical Vascular Changes Using the APT-II
Background: Subcortical Vascular Dementia (VaD), consequent to deep brain small vessel disease (SVD), is the most frequent form of VaD.
- Trial name
- RehAtt (NCT02033850)
- Testing
- Attention Process Training-II
- Where
- Italy
- Volunteers
- 46 people
- Sponsor
- Azienda Ospedaliero-Universitaria Careggi
Dementia, Vascular, Dementia, Mixed
Vaspect Study - An Open-Label Trial Of Donepezil in Vascular and Mixed Dementia
To document effectiveness, safety, and tolerability of donepezil in patients with mixed AD/VaD, and to further document the effectiveness, safety, and tolerability of donepezil in patients with VaD.
- Trial name
- NCT00174382
- Testing
- Donepezil
- Where
- 30 sites in Canada
- Volunteers
- 149 people
- Sponsor
- Pfizer
Hurler's Syndrome, Maroteaux-Lamy Syndrome
Hematopoietic Stem Cell Transplantation (HCT) for Inborn Errors of Metabolism
The primary objective of this clinical trial is to evaluate the ability to achieve and sustain donor engraftment in patients with lysosomal and peroxisomal inborn errors of metabolism undergoing hematopoietic stem cell…
- Conditions
- Hurler's Syndrome, Maroteaux-Lamy Syndrome, Sly Syndrome, Alpha Mannosidosis
- Trial name
- NCT00668564
- Testing
- Stem Cell Transplantation, Cyclophosphamide, Campath-1H
- Where
- United States
- Volunteers
- 18 people
- Sponsor
- Masonic Cancer Center, University of Minnesota
Leukodystrophy, White Matter Disease
LeukoSEQ: Whole Genome Sequencing as a First-Line Diagnostic Tool for Leukodystrophies
Leukodystrophies, and other heritable disorders of the white matter of the brain, were previously resistant to genetic characterization, largely due to the extreme genetic heterogeneity of molecular causes.
- Conditions
- Leukodystrophy, White Matter Disease, 4H Syndrome, Adrenoleukodystrophy
- Trial name
- NCT02699190
- Where
- United States
- Volunteers
- 236 people
- Sponsor
- Children's Hospital of Philadelphia
Adrenoleukodystrophy
Effect of Glycerol Trierucate on Clinical Course of Adrenoleukodystrophy
OBJECTIVES: I. Evaluate the clinical efficacy of combination glyceryl trierucate and glyceryl trioleate (Lorenzo's Oil) therapy in boys with X-linked adrenoleukodystrophy. II.
- Trial name
- NCT00004418
- Testing
- glyceryl trierucate/glyceryl trioleate
- Where
- 2 sites in United States
- Volunteers
- 126 people
- Sponsor
- Hugo W. Moser Research Institute at Kennedy Krieger, Inc.
Dementia, Vascular, Alzheimer Disease
LEAD IT! An App to Enable Persons With Early Stage Dementia to Lead Group Activities for Their Peers
The proposed Phase 2 project will involve the further development and evaluation of LEAD IT!, which is a tablet-based app designed to enable persons with dementia to lead activities for their peers (i.e., other persons with…
- Conditions
- Dementia, Vascular, Alzheimer Disease, Dementia, Mixed
- Trial name
- NCT05516342
- Testing
- LEAD IT! Programming
- Where
- United States
- Volunteers
- 182 people
- Sponsor
- Hopeful Aging