Clinical trials: White Matter Disease
Inherited Metabolic Disorders (IMD)
MGTA-456 in Patients With Inherited Metabolic Disorders Undergoing Hematopoietic Stem Cell Transplantation (HSCT)
This study is designed to assess the safety and efficacy of using MGTA-456 in patients with Inherited Metabolic Disorders (IMD) undergoing stem cell transplantation.
- Trial name
- NCT03406962
- Testing
- MGTA-456
- Where
- 2 sites in United States
- Volunteers
- 8 people
- Sponsor
- Magenta Therapeutics, Inc.
Multiple Sclerosis
Anakinra for the Treatment of Chronically Inflamed White Matter Lesions in Multiple Sclerosis
Background: Multiple sclerosis (MS) is a disease of the central nervous system (CNS). People who have MS may have lesions that form on parts of the CNS, such as the brain. Some of these lesions may be inflamed for a long time.
- Trial name
- NCT04025554
- Testing
- Anakinra
- Where
- United States
- Volunteers
- 8 people
- Sponsor
- National Institute of Neurological Disorders and Stroke (NINDS)
Metachromatic Leukodystrophy (MLD)
Multicenter Study of HGT-1110 Administered Intrathecally in Children With Metachromatic Leukodystrophy (MLD)
The purpose of this study is to determine the safety of ascending doses of HGT-1110 administered by intrathecal (IT) injection for 38 weeks (20 injections) in children with metachromatic leukodystrophy (MLD).
- Trial name
- IDEAMLD (NCT01510028)
- Testing
- Recombinant human arylsulfatase A
- Where
- 5 sites in Australia, Denmark and 3 more
- Volunteers
- 24 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-related Psychosis
Relapse Prevention Study of Pimavanserin in Dementia-related Psychosis
The purpose of this study is to evaluate the efficacy of pimavanserin compared to placebo in preventing relapse of psychotic symptoms in subjects with dementia-related psychosis who responded to 12 weeks of open label…
- Trial name
- NCT03325556
- Testing
- Pimavanserin 34 mg, Pimavanserin 20 mg
- Where
- 83 sites in United States, Bulgaria and 11 more
- Volunteers
- 392 people
- Sponsor
- ACADIA Pharmaceuticals Inc.
Dementia, Dementia, Vascular
The BRAIN App: Building Relationships Using Artificial Intelligence and Nostalgia
This study will involve testing of an Alpha version of an app called "Building Relationships using Artificial Intelligence and Nostalgia" or BRAIN.
- Conditions
- Dementia, Dementia, Vascular, Alzheimer Disease, Dementia, Mixed
- Trial name
- BRAIN (NCT05515679)
- Testing
- The BRAIN App
- Where
- United States
- Volunteers
- 45 people
- Sponsor
- Hopeful Aging
Bone Marrow Failure Syndrome, Thalassemia
Conditioning Regimen for Allogeneic Hematopoietic Stem-Cell Transplantation
In this study, the investigators test 2 dose levels of thiotepa (5 mg/kg and 10 mg/kg) added to the backbone of targeted reduced dose IV busulfan, fludarabine and rabbit anti-thymocyte globulin (rATG) to determine the minimum…
- Conditions
- Bone Marrow Failure Syndrome, Thalassemia, Sickle Cell Disease, Diamond Blackfan Anemia
- Trial name
- NCT03513328
- Testing
- Thiotepa--single daily dose, Thiotepa--escalated dose
- Where
- United States
- Volunteers
- 6 people
- Sponsor
- University of Florida
Mitochondrial Diseases, Drug Resistant Epilepsy
A Study to Evaluate Efficacy and Safety of Vatiquinone for Treating Mitochondrial Disease in Participants With Refractory Epilepsy
This is a parallel-arm, double-blind, placebo-controlled study with a screening phase that includes a 28-day run-in phase to establish baseline seizure frequency, followed by a 24-week, randomized, placebo-controlled phase.
- Conditions
- Mitochondrial Diseases, Drug Resistant Epilepsy, Leigh Disease, Leigh Syndrome
- Trial name
- MIT-E (NCT04378075)
- Testing
- Vatiquinone
- Where
- 27 sites in United States, Canada and 7 more
- Volunteers
- 68 people
- Sponsor
- PTC Therapeutics
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
Metachromatic Leukodystrophy (MLD)
Metazym for the Treatment of Patients With Late Infantile Metachromatic Leukodystrophy (MLD)
Objectives: The overall objective is to evaluate the safety, efficacy and pharmacokinetics (PK) of rhASA treatment in patients with late infantile MLD.
- Trial name
- NCT00418561
- Testing
- rhASA - Dose Level 1, rhASA - Dose Level 2, rhASA - Dose Level 3
- Where
- Denmark
- Volunteers
- 13 people
- Sponsor
- Shire
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
Late Infantile Metachromatic Leukodystrophy
Open-Label Extension Study of Recombinant Human Arylsulfatase A (HGT-1111) in Late Infantile MLD
This is a multi-center, open-label, extension study of patients with late infantile MLD who have previously completed clinical study HGT-MLD-048 (NCT00633139), defined as the completion of all Week 52 procedures.
- Trial name
- NCT00681811
- Testing
- HGT-1111
- Where
- Denmark
- Volunteers
- 11 people
- Sponsor
- Shire
Lysosomal Storage Disease, Peroxisomal Disorder
Reduced-Intensity Hematopoietic Stem Cell Transplant for High Risk Lysosomal and Peroxisomal Disorders
This study is designed to test the ability to achieve donor hematopoietic engraftment while maintaining low rates of transplant-related mortality (TRM) in patients with high-risk lysosomal and peroxisomal disorders using a novel…
- Trial name
- NCT01626092
- Testing
- Campath-1H, Clofarabine, Melphalan
- Where
- United States
- Volunteers
- 3 people
- Sponsor
- Masonic Cancer Center, University of Minnesota
Cerebral Adrenoleukodystrophy (CALD)
A Clinical Study to Assess the Efficacy and Safety of Gene Therapy for the Treatment of Cerebral Adrenoleukodystrophy (CALD)
The purpose of this study is to evaluate the efficacy and safety of Lenti-D Drug Product (also known as elivaldogene autotemcel or Skysona, hereafter referred to as eli-cel) after myeloablative conditioning with busulfan and…
- Trial name
- NCT03852498
- Testing
- Lenti-D
- Where
- 8 sites in United States, France and 4 more
- Volunteers
- 35 people
- Sponsor
- Genetix Biotherapeutics Inc.
Dementia, Alzheimer Disease
Zolpidem CR and Hospitalized Patients With Dementia
The purpose of this research study is to compare the effectiveness of Zolpidem CR to that of placebo in improving sleep efficiency in people with dementia admitted to the hospital because of their symptoms.
- Conditions
- Dementia, Alzheimer Disease, Dementia, Vascular, Sleep Disorders
- Trial name
- NCT00814502
- Testing
- Zolpidem CR
- Where
- United States
- Volunteers
- 20 people
- Sponsor
- Massachusetts General Hospital
Depression
Emotional and Cognitive Control in Late-Onset Depression
This study may help identify how abnormalities in brain systems that control the ability to ignore irrelevant information may contribute to the development of depression in older adults.
- Trial name
- NCT01728194
- Testing
- Escitalopram, Magnetic Resonance Imaging
- Where
- 2 sites in United States
- Volunteers
- 121 people, healthy volunteers welcome
- Sponsor
- Weill Medical College of Cornell University
Dementia, Alzheimer Disease
The Healthy Patterns Sleep Study
The Healthy Patterns Study intervention is a home-based activity intervention designed to improve symptoms of circadian rhythm disorders (CRD) and quality of life (QOL) in home-dwelling persons with dementia.
- Conditions
- Dementia, Alzheimer Disease, Circadian Rhythm Disorders, Circadian Rhythm Sleep Disorder
- Trial name
- NCT03682185
- Testing
- Attention-Control Condition, Timed Activity Intervention
- Where
- United States
- Volunteers
- 421 people, healthy volunteers welcome
- Sponsor
- University of Pennsylvania
RVCL - Retinal Vasculopathy Cerebral Leukoencephalopathy
Crizanlizumab for Treatment of Retinal Vasculopathy With Cerebral Leukoencephalopathy (RVCL)
This is a Phase 2 trial that will test the efficacy and safety of crizanlizumab for the treatment of retinal vasculopathy with cerebral leukoencephalopathy (RVCL), a very rare and uniformly fatal genetic condition that affects…
- Trial name
- NCT04611880
- Testing
- Crizanlizumab
- Where
- 2 sites in United States
- Volunteers
- 18 people
- Sponsor
- Washington University School of Medicine
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.
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