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
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.
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.
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.
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.
AMN, AMN Gene Mutation
Study of Disease Progression in Adults With Inherited Forms of Spastic Paraplegia
The course of AMN-related disabilities over time is poorly or incompletely understood due to a limited number of patients and lack of treatments.
- Conditions
- AMN, AMN Gene Mutation, X-ALD
- Trial name
- CYGNET (NCT05008874)
- Testing
- Natural History Observation
- Where
- 6 sites in United States, Germany and 1 more
- Volunteers
- 65 people
- Sponsor
- SwanBio Therapeutics, Inc.
Infantile Globoid Cell Leukodystrophy
The Natural History of Infantile Globoid Cell Leukodystrophy
The purpose of this natural history study is to understand more about the progression of infantile Krabbe disease, a very rare genetic disease. There is very little published longitudinal data with only anecdotal cases.
- Trial name
- NCT00983879
- Where
- United States
- Volunteers
- 6 people
- Sponsor
- Zymenex A/S
Dementia, Alzheimer Disease
A Study of the Effectiveness and Safety of Risperidone in the Treatment of Behavioral Disturbances in Patients With Dementia
The purpose of the study is to compare the effectiveness of an oral formulation of risperidone (an antipsychotic medication) to that of placebo for treating behavioral and psychological signs and symptoms in dementia (BPSSD)…
- Conditions
- Dementia, Alzheimer Disease, Vascular Dementia
- Trial name
- NCT00249158
- Testing
- Risperidone
- Volunteers
- 344 people
- Sponsor
- Janssen-Cilag Pty Ltd
Dementia, Alzheimer Disease
A Study of the Effectiveness and Safety of Risperidone in the Treatment of Behavioral Disturbances in Patients With Dementia
The purpose of the study is to compare the safety and efficacy of risperidone (an antipsychotic medication) to that of placebo in the treatment of behavioral disturbances associated with dementia.
- Conditions
- Dementia, Alzheimer Disease, Dementia, Vascular
- Trial name
- NCT00249145
- Testing
- risperidone
- Volunteers
- 349 people
- Sponsor
- Janssen Pharmaceutica N.V., Belgium
Lysosomal Storage Disease, Metachromatic Leukodystrophy
A Safety and Efficacy Study of Cryopreserved OTL-200 for Treatment of Metachromatic Leukodystrophy (MLD)
OTL-200 is autologous CD34+ cells transduced with lentiviral vector containing human arylsulfatase A (ARSA) complementary deoxyribonucleic acid (cDNA) used for the treatment of MLD.
- Trial name
- NCT03392987
- Testing
- OTL-200
- Where
- Italy
- Volunteers
- 10 people
- Sponsor
- Orchard Therapeutics
ALD (Adrenoleukodystrophy)
Study to Assess PXL065 in Subjects With Adrenomyeloneuropathy (AMN) Form of X-linked Adrenoleukodystrophy (X-ALD or ALD)
A randomized open-label Phase 2a study to assess the pharmacokinetics and pharmacodynamic parameters of PXL065 after 12 weeks of treatment in male subjects with adrenomyeloneuropathy (AMN).
- Trial name
- NCT05200104
- Testing
- PXL065
- Sponsor
- Poxel SA
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
Metachromatic Leukodystrophy
Allogeneic Stem Cell Transplantation for the Treatment of Multiple Sclerosis (Compassionate Use)
A subject was treated under compassionate use provisions under this study with facilitating cell therapy (FCRx) product manufactured using the CliniMACS (Miltenyi Biotec) device, rather than the Max Sep (Baxter) device.
- Trial name
- NCT02084121
- Testing
- Enriched Hematopoetic Stem Cell Infusion
- Where
- United States
- Sponsor
- Talaris Therapeutics Inc.
Dementia, Mild Cognitive Impairment
HOme-based Brain Monitoring With a GARment-EEG to Study Cognitive Decline in the Aging Population
This study will investigate the validity of the HOGAR EEG/PSG monitoring kit designed by Bitbrain as a tool for characterizing and assessing cognitive function in older adults, as well as for detecting and predicting cognitive…
- Conditions
- Dementia, Mild Cognitive Impairment, Dementia Alzheimers, Dementia, Mixed
- Trial name
- Hogar (NCT06993207)
- Testing
- Home Lab
- Where
- Spain
- Volunteers
- 500 people, healthy volunteers welcome
- Sponsor
- Bitbrain
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
Vanishing White Matter Disease
An Open-Label Exploratory Study of Fosigotifator in Participants With Vanishing White Matter Disease
Fosigotifator is an investigational drug being researched for the treatment of Vanishing White Matter disease in adult, pediatric and infant participants.
- Trial name
- NCT05757141
- Testing
- Fosigotifator
- Where
- 5 sites in United States, Canada and 1 more
- Volunteers
- 50 people
- Sponsor
- Calico Life Sciences LLC