Clinical trials: Leukoencephalopathies
- Condition Leukoencephalopathies
- Funder type Industry (pharma, biotech, devices)
- Has posted results yes
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.
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
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
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
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
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.
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
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.
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.
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
Alzheimer Disease, Dementia
SAFE at Home: A Service to Provide Social Engagement to Community-Dwelling Persons With Dementia
The proposed Phase 1 study will involve initial development and evaluation of a new service called Social Activities For Engagement at Home or SAFE at Home (SaH).
- Conditions
- Alzheimer Disease, Dementia, Dementia, Vascular, Dementia, Mixed
- Trial name
- NCT05516147
- Testing
- SAFE at Home
- Where
- United States
- Volunteers
- 12 people
- Sponsor
- Hopeful Aging
Alzheimer's Disease With Vascular Pathology
A Study of the Effects of CY6463 in Participants With Alzheimer's Disease With Vascular Pathology
This study is being conducted to test the safety, tolerability, and pharmacokinetics of the investigational drug CY6463 compared with placebo in individuals who are aged 60 years or older and have Alzheimer's disease (AD) along…
- Trial name
- NCT04798989
- Testing
- CY6463
- Where
- 5 sites in United States
- Volunteers
- 12 people
- Sponsor
- Tisento Therapeutics
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
Relapsing-remitting Multiple Sclerosis
Safety, Tolerability, Efficacy and Optimal Dose Finding Study of BAF312 in Patients With Relapsing-remitting Multiple Sclerosis
The purpose of this study was to determine the dose-response curve for the MRI-based efficacy of BAF312 compared with placebo in patients with Relapsing-Remitting Multiple Sclerosis (RRMS), and to characterize its safety and…
- Trial name
- NCT00879658
- Testing
- BAF312
- Where
- 72 sites in United States, Canada and 10 more
- Volunteers
- 297 people
- Sponsor
- Novartis Pharmaceuticals
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
Multiple Sclerosis
Retrospective Cohort Study of Rebif® Use in Pediatric Multiple Sclerosis (MS) Subjects (REPLAY)
The aim of this retrospective study is to review and describe safety, tolerability and efficacy of Rebif® (subcutaneous interferon [IFN]-beta-1a) in children and adolescents, using information already recorded in medical records.
- Trial name
- REPLAY (NCT01207648)
- Testing
- Rebif®
- Where
- 18 sites in United States, Argentina and 6 more
- Volunteers
- 307 people
- Sponsor
- EMD Serono
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