Clinical trials: Pick Disease of the Brain
Niemann-Pick Type C Disease
Miglustat in Niemann-Pick Type C Disease
This is a phase II randomized controlled study of miglustat in adult and juvenile patients with Niemann-Pick Type C disease. Up to 42 patients will be randomised in a 2:1 ratio to either treatment with miglustat or to a…
- Trial name
- NCT00517153
- Testing
- miglustat
- Volunteers
- 29 people
- Sponsor
- Actelion
Alzheimer's Disease, Brain Diseases
CNS Tau Kinetics in Healthy Aging and Alzheimer's Disease
Alzheimer's disease (AD) is the most common cause of dementia and currently has no disease modifying treatments or simple accurate diagnostic tests.
- Conditions
- Alzheimer's Disease, Brain Diseases, Central Nervous System Diseases, Delirium, Dementia, Amnestic, Cognitive Disorders
- Trial name
- NCT03938870
- Testing
- L-Leucine 13C6
- Where
- United States
- Volunteers
- 96 people, healthy volunteers welcome
- Sponsor
- Washington University School of Medicine
Dementia
Double Blind Trial of DC Polarization in FTD
This project is designed to test how direct current (DC) electrical polarization of the brain affects language and behavior in patients with frontotemporal dementia (FTD).
- Trial name
- NCT00117858
- Testing
- Anodal DC polarization of left prefrontal cortex
- Where
- United States
- Volunteers
- 20 people
- Sponsor
- National Institute of Neurological Disorders and Stroke (NINDS)
Alzheimer Disease, Frontotemporal Dementia
First-in-Human Evaluation of an Astrocytic Glutamate Transporter (EAAT2) PET Tracer in Dementia
This is a first in human study that will assess the safety and diagnostic performance of [18F]RP-115 (fluorine-18 labeled RP115), a positron emission tomography (PET) agent.
- Conditions
- Alzheimer Disease, Frontotemporal Dementia, Dementia
- Trial name
- NCT05374278
- Testing
- [18F]RP-115 PET/MRI or PET/CT and MRI
- Where
- United States
- Volunteers
- 102 people, healthy volunteers welcome
- Sponsor
- David Wilson
Sphingomyelin Lipidosis
Efficacy, Safety, Pharmacodynamic, and Pharmacokinetics Study of Olipudase Alfa in Patients With Acid Sphingomyelinase Deficiency
Primary Objective: The primary objective of this phase 2/3 study was to evaluate the efficacy of olipudase alfa (recombinant human acid sphingomyelinase) administered intravenously once every 2 weeks for 52 weeks in adult…
- Trial name
- ASCEND (NCT02004691)
- Testing
- Olipudase alfa
- Where
- 24 sites in United States, Argentina and 15 more
- Volunteers
- 36 people, healthy volunteers welcome
- Sponsor
- Genzyme, a Sanofi Company
Progressive Supranuclear Palsy, Corticobasal Degeneration
A Pilot Trial of Lithium in Subjects With Progressive Supranuclear Palsy or Corticobasal Degeneration
The goal of this trial is to evaluate the safety and tolerability of lithium in people with progressive supranuclear palsy or corticobasal degeneration.
- Trial name
- NCT00703677
- Testing
- Lithium
- Where
- 9 sites in United States, United Kingdom
- Volunteers
- 17 people
- Sponsor
- Westat
Opioid-Related Disorders
Tau PET Imaging in Opioid Use Disorder
The investigators plan to enroll up to 60 adult subjects in this study. There will be three groups of up to 20 subjects each in this study.
- Trial name
- NCT05651516
- Testing
- 18F-PI-2620 PET/CT
- Where
- United States
- Volunteers
- 12 people, healthy volunteers welcome
- Sponsor
- University of Pennsylvania
Niemann-Pick Disease, Niemann-Pick Disease, Type C
Biomarker for Niemann Pick Type C Disease (BioNPC)
Development of a new MS-based biomarker for the early and sensitive diagnosis of Niemann Pick Type C disease from Blood (plasma)
- Trial name
- BioNPC (NCT01306604)
- Where
- 4 sites in Germany, India and 1 more
- Sponsor
- CENTOGENE GmbH Rostock
Parkinson's Disease, Parkinsonism
Modifiable Variables in Parkinsonism (MVP)
We are trying to identify factors associated with improved quality of life and fewer PD symptoms. We are attempting to identify practices, beliefs, and therapies used by individuals who report excellent quality of life, few PD…
- Conditions
- Parkinson's Disease, Parkinsonism, MSA - Multiple System Atrophy, Progressive Supranuclear Palsy
- Trial name
- NCT02194816
- Where
- United States
- Volunteers
- 2,000 people
- Sponsor
- Bastyr University
Dementia of Alzheimer Type, Dementia Frontotemporal
Multifactorial Approach to Dementia, Multicentric Study
The goal of this observational study is to assess the role of narcisistic personality disorder and life stressful events in conversion rate to dementia, using a three tier approach along three research lines employing subjects…
- Conditions
- Dementia of Alzheimer Type, Dementia Frontotemporal, Narcissistic Personality Disorder, Depression
- Trial name
- NCT05923307
- Where
- Argentina
- Volunteers
- 450 people, healthy volunteers welcome
- Sponsor
- Daniel Serrani Azcurra
Meningococcal Disease, Meningococcal Meningitis
A Phase 3b, Randomized, Open-Label Study to Evaluate the Safety and Immunogenicity of Select Travel Vaccines When Administered Concomitantly With MenACWY in Adults
This study compares the safety and immunogenicity profile of several travel vaccines given alone or concomitantly with MenACWY-CRM to healthy adults.
- Conditions
- Meningococcal Disease, Meningococcal Meningitis, Typhoid, Yellow Fever
- Trial name
- NCT01466387
- Testing
- Typhoid Vi Polysaccharide Vaccine, Yellow Fever Vaccine, Japanese Encephalitis Vaccine
- Where
- 5 sites in Czechia, Germany
- Volunteers
- 552 people, healthy volunteers welcome
- Sponsor
- Novartis
Bipolar Disorder, Frontotemporal Dementia, Behavioral Variant
A Study of the Behavioral Variant of Frontotemporal Dementia and Bipolar Disorder: a Neuroimaging and Epigenetics Integrated Approach
The aim of this study is to investigate the selective epigenetic modifications and their effect on brain's morphology and functionality in the frontotemporal dementia behavioral variant and bipolar disorder.
- Trial name
- DISBAND (NCT06706687)
- Testing
- DISBAND protocol
- Where
- Italy
- Volunteers
- 210 people, healthy volunteers welcome
- Sponsor
- Fondazione IRCCS Ca' Granda, Ospedale Maggiore Policlinico
Frontotemporal Lobar Degeneration
The Skin as a Window to the Central Nervous System in Frontotempolar Lombar Degeneration
Frontotemporal lobar degeneration (FTLD) is a clinically heterogeneous syndrome, characterized by progressive decline in behaviour and/or language.
- Trial name
- PROTEINOSKIN (NCT06490822)
- Testing
- Skin biopsy
- Where
- France
- Volunteers
- 80 people, healthy volunteers welcome
- Sponsor
- Nantes University Hospital
Niemann-Pick Disease, Type C1
Phase 3 Study to Evaluate Intravenous Trappsol(R) Cyclo(TM) in Pediatric and Adult Patients With Niemann-Pick Disease Type C1
A prospective, randomized, double-blind, placebo controlled, multi-center therapeutic study for patients age 3 and older with confirmed diagnosis of Niemann Pick disease type C1 (NPC1).
- Trial name
- TransportNPC (NCT04860960)
- Testing
- Hydroxypropyl-beta-cyclodextrin
- Where
- 35 sites in United States, Argentina and 11 more
- Volunteers
- 94 people
- Sponsor
- Cyclo Therapeutics, Inc.
Frontotemporal Dementia
MSC-Exosome Therapy for Frontotemporal Dementia
This study is testing a new treatment for Frontotemporal Dementia (FTD) - a progressive brain disease that affects personality, behavior, and language.
- Trial name
- NCT07638813
- Testing
- Umbilical Cord Mesenchymal Stem Cell-Derived Exosomes （Low-Dose）, Umbilical Cord Mesenchymal Stem Cell-Derived Exosomes （High-Dose）
- Volunteers
- 33 people
- Sponsor
- Ruijin Hospital
Alzheimer Disease
University of Washington Alzheimer's Disease Research Center (UW ADRC) Imaging & Biomarker Core
This is a cross-sectional, observational study that characterizes research participants with Alzheimer's disease (AD) for their patterns of brain degeneration with the investigational tau positron emission tomography (PET)…
- Trial name
- NCT04437290
- Testing
- investigational radiotracer [18F]MK6240
- Volunteers
- 300 people
- Sponsor
- University of Washington
Early Onset Alzheimer Disease, Alzheimer Disease
Longitudinal Early-onset Alzheimer's Disease Study Protocol
The Longitudinal Early-onset Alzheimer's Disease Study (LEADS) is a non-randomized, natural history, non-treatment study designed to look at disease progression in individuals with early onset cognitive impairment.
- Conditions
- Early Onset Alzheimer Disease, Alzheimer Disease, Mild Cognitive Impairment
- Trial name
- LEADS (NCT03507257)
- Testing
- Flortaucipir, Florbetaben, Fluorodeoxyglucose
- Where
- 23 sites in United States, Argentina and 4 more
- Volunteers
- 850 people, healthy volunteers welcome
- Sponsor
- Indiana University
Dementia, Alzheimer Disease
Incremental Diagnostic Value of Tau-PET With [18F]RO948 vs Amyloid-PET in Patients With Cognitive Impairment
The objective of the study is to investigate the clinical validity of tau-PET with [18F]RO948 vs. amyloid-PET in patients with Mild Cognitive Impairment (MCI) or mild dementia
- Trial name
- IDV of tau-PET (NCT06618872)
- Testing
- PET/CT with RO958 (experimental)
- Where
- 2 sites in Switzerland
- Volunteers
- 120 people
- Sponsor
- University Hospital, Geneva
Sickle Cell Disease, Thalassemia
Unrelated Hematopoietic Stem Cell Transplantation(HSCT) for Genetic Diseases of Blood Cells
This is a clinical trial of bone marrow transplantation for patients with the diagnosis of a genetic disease of blood cells that do not have an HLA-matched sibling donor.
- Conditions
- Sickle Cell Disease, Thalassemia, Anemia, Granuloma
- Trial name
- NCT00730314
- Testing
- Hematopoietic stem cell transplantation
- Where
- United States
- Volunteers
- 25 people
- Sponsor
- Children's Hospital Los Angeles
Apraxia of Speech, Non-fluent Aphasia
The Neurobiology of Two Distinct Types of Progressive Apraxia of Speech
The purpose of this study is to identify and distinguish two different types of Progressive Apraxia of Speech through clinical imaging and testing.
- Conditions
- Apraxia of Speech, Non-fluent Aphasia, Primary Progressive Aphasia, Primary Progressive Nonfluent Aphasia
- Trial name
- SLD4T (NCT03313011)
- Testing
- Testing protocol for the study
- Where
- United States
- Volunteers
- 47 people
- Sponsor
- Mayo Clinic