Clinical trials: Pick Disease of the Brain
Frontotemporal Dementia
Open Label Pilot Study of the Effects of Memantine on FDG-PET in Frontotemporal Dementia
Memantine has been approved for use in Alzheimer's disease. Its mechanism of action raises questions of whether it can also be effective for non-Alzheimer's dementias such as frontotemporal dementia (FTD), which currently has no…
- Trial name
- NCT00594737
- Testing
- memantine hydrochloride
- Where
- Canada
- Volunteers
- 17 people
- Sponsor
- Tiffany Chow, MD
Dementia, Mild Cognitive Impairment (MCI)
Comprehensive Assessment of Neurodegeneration and Dementia
This is a longitudinal observational study recruiting individuals between the ages of 50 and 90 with different types of dementia as well as a comparison group without cognitive deficits.
- Conditions
- Dementia, Mild Cognitive Impairment (MCI), Subjective Cognitive Impairment, Parkinson's Disease (PD)
- Trial name
- COMPASS-ND (NCT03402919)
- Where
- 19 sites in Canada
- Volunteers
- 1,573 people, healthy volunteers welcome
- Sponsor
- McGill University
Progressive Multifocal Leukoencephalopathy
Tysabri Observational Cohort Study - Multiple Sclerosis (MS) Registries
The primary purpose of this study is to estimate the incidence of progressive multifocal leukoencephalopathy (PML) among patients who switched to Tysabri from disease modifying therapies (DMTs), including newer DMTs (including…
- Trial name
- NCT03399981
- Testing
- Tysabri
- Where
- United States
- Volunteers
- 80,327 people
- Sponsor
- Biogen
Alzheimer's Disease
24 Months Safety and Efficacy Study of AADvac1 in Patients With Mild Alzheimer's Disease
This study evaluates the safety and efficacy of AADvac1 in the treatment of patients with mild Alzheimer's disease. 60% of participants will receive AADvac1 and 40% of participants will receive placebo.
- Trial name
- ADAMANT (NCT02579252)
- Testing
- AADvac1
- Where
- 42 sites in Austria, Czechia and 6 more
- Volunteers
- 208 people
- Sponsor
- Axon Neuroscience SE
Sphingomyelin Lipidosis
A Prospective and Retrospective Cohort Study in Patients With Chronic Forms of Acid Sphingomyelinase Deficiency (ASMD)
Primary Objective: To describe the clinical features and their severity at the time of diagnosis and their evolution over time in patients with confirmed chronic visceral and chronic neurovisceral forms of ASMD To describe…
- Trial name
- NCT04106544
- Testing
- Investigational Procedures
- Where
- 28 sites in United States, Argentina and 11 more
- Volunteers
- 84 people
- Sponsor
- Sanofi
Frontotemporal Dementia (FTD)
Low-Dose Lithium for the Treatment of Behavioral Symptoms in Frontotemporal Dementia
Frontotemporal dementia (FTD) is a progressive neurodegenerative illness that affects the frontal and anterior temporal lobes of the brain.
- Trial name
- Lithium (NCT02862210)
- Testing
- Lithium Carbonate
- Where
- United States
- Volunteers
- 17 people
- Sponsor
- Columbia University
Alzheimer Disease
A Phase I [18F]THK-5351 Positron Emission Tomography Study in Healthy Subjects and Alzheimer's Disease
This is a study to evaluate biodistribution, pharmacokinetics and safety of [18F]THK-5351 positron emission computed tomography in Cognitively Healthy Subjects and Patients with Alzheimer's Disease.
- Trial name
- NCT03112096
- Testing
- [18F]THK-5351
- Where
- South Korea
- Volunteers
- 12 people, healthy volunteers welcome
- Sponsor
- Asan Foundation
Frontotemporal Lobar Degeneration
Effects of Tolcapone on Frontotemporal Dementia
This study will test the effects of a medication called tolcapone on cognitive, behavioral, and language problems seen in patients with frontotemporal dementia (FTD).
- Trial name
- NCT00604591
- Testing
- Tolcapone
- Where
- United States
- Volunteers
- 28 people
- Sponsor
- Columbia University
Lysosomal Acid Lipase Deficiency, Wolman Disease
Safety, Tolerability, Efficacy, Pharmacokinetics, and Pharmacodynamics of Sebelipase Alfa in Children With Growth Failure Due to Lysosomal Acid Lipase Deficiency
This was an open-label, repeat-dose, intra-participant dose-escalation study of SBC-102 (sebelipase alfa) in children with growth failure due to lysosomal acid lipase (LAL) Deficiency.
- Trial name
- NCT01371825
- Testing
- Sebelipase alfa (SBC-102)
- Where
- 7 sites in United States, Egypt and 3 more
- Volunteers
- 9 people
- Sponsor
- Alexion Pharmaceuticals, Inc.
Alzheimer's Disease, Healthy Adult Participants
Ultra-high-performance Positron Emission Tomography (PET) of Synaptic Density, Reactive Astrogliosis and Tau in Alzheimer's Disease
AIM: to investigate the relationship between synaptic density, tau and reactive astrogliosis in Alzheimer's disease (AD) versus healthy controls and centenarians.
- Trial name
- NCT07906054
- Testing
- 18F-SMBT-1 PET, 18F-SynVesT-1 PET, 18F-MK-6240 PET
- Where
- Belgium
- Volunteers
- 70 people, healthy volunteers welcome
- Sponsor
- Universitaire Ziekenhuizen KU Leuven
Niemann-Pick Diseases
Health Insurance Literacy and Challenges in Accessing Health Services in Niemann-Pick
This study is a US based qualitative PRO research study to document the health insurance literacy as well as the patient experience in Niemann-Pick as it relates to accessing desired care, services and medications for patients.
- Trial name
- NCT04469894
- Where
- United States
- Volunteers
- 76 people
- Sponsor
- National Niemann-Pick Disease Foundation
Amyotrophic Lateral Sclerosis, Frontal Temporal Dementia
Therapy in Amyotrophic Lateral Sclerosis (TAME)
The purpose of this study is to determine if memantine at up to 20 mg twice a day when used in conjunction with riluzole, can slow down the disease progression of patients with ALS including potentially improving their…
- Trial name
- TAME (NCT02118727)
- Testing
- Memantine
- Where
- 13 sites in United States
- Volunteers
- 89 people
- Sponsor
- University of Kansas Medical Center
Fabry Disease, Gaucher Disease
Complement Activation in the Lysosomal Storage Disorders
The lysosomal storage disorders (LSDs) are monogenic disorders associated with inflammation affecting multiple organs, and early death.
- Conditions
- Fabry Disease, Gaucher Disease, Niemann-Pick Disease, Type C, Lysosomal Storage Diseases
- Trial name
- CATALYST (NCT04189601)
- Testing
- Complement measurements
- Where
- Australia
- Volunteers
- healthy volunteers welcome
- Sponsor
- Melbourne Health
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.
Glioblastoma, Astrocytoma
Stem Cell Transplant for High Risk Central Nervous System (CNS) Tumors
The primary goal of this study is to determine if a stem cell transplant in patients with newly diagnosed high risk CNS tumors (glioblastoma multiforme [GBM], high grade astrocytoma, pineoblastoma, rhabdoid tumor, supratentorial…
- Conditions
- Glioblastoma, Astrocytoma, Pineoblastoma, Rhabdoid Tumor
- Trial name
- NCT00179803
- Testing
- Stem Cell Transplant
- Where
- United States
- Volunteers
- 24 people
- Sponsor
- Ann & Robert H Lurie Children's Hospital of Chicago
Niemann-Pick Disease, Type C
Biomarker Validation for Niemann-Pick Disease, Type C: Safety and Efficacy of N-Acetyl Cysteine
Background: Niemann-Pick disease type C (NPC) is a genetic disorder that results in progressive loss of nervous system function by affecting the membranes of nerve cells. There is no known cure for NPC.
- Trial name
- NCT00975689
- Testing
- N-Acetyl Cysteine
- Where
- United States
- Volunteers
- 35 people
- Sponsor
- Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
Post-stroke Cognitive Impairment, Neuroinflammation
The Influence of [18F]PMPBB3 and [18F]THK5351 PET Distribution Patterns on Post-stroke Cognitive Impairment
Background and objects： Neuroinflammation is an active process detectable in the earliest stages of the neurodegeneration pathway.
- Trial name
- NCT04318626
- Testing
- PMPBB3, THK5351
- Where
- Taiwan
- Volunteers
- 80 people, healthy volunteers welcome
- Sponsor
- Chang Gung Memorial Hospital
Acid Sphingomyelinase Deficiency
A Study to Evaluate Safety and Tolerability of Olipudase Alfa in Pediatric and Adult Participants With Acid Sphingomyelinase Deficiency (ASMD) Who Completed the DFI12712 or the LTS13632 Study in France
This was an open-label study to evaluate safety and tolerability and provide enzyme replacement therapy (ERT) with olipudase alfa to patients with acid sphingomyelinase deficiency (ASMD) who completed the DFI12712 or the LTS13632…
- Trial name
- NCT06949358
- Testing
- Olipudase alfa
- Where
- 2 sites in France
- Volunteers
- 3 people
- Sponsor
- Sanofi
Granulin Mutation
Study to Assess the Safety, Tolerability, and Pharmacodynamic (PD) Effects of FRM-0334 in Subjects With Prodromal to Moderate Frontotemporal Dementia With Granulin Mutation
The purposes of this study are to investigate the safety, tolerability, and pharmacodynamics of FRM-0334 in subjects with prodromal to moderate frontotemporal dementia with granulin mutation.
- Trial name
- NCT02149160
- Testing
- FRM-0334
- Where
- 13 sites in United States, Belgium and 4 more
- Volunteers
- 30 people
- Sponsor
- FORUM Pharmaceuticals Inc
Niemann-Pick Disease, Type C
Study of IV VTS-270 for Infantile Liver Disease Associated With Niemann-Pick Disease, Type C
Niemann-Pick disease, type C (NPC) is a lethal, autosomal recessive, lysosomal storage disorder characterized by neurodegeneration in early childhood and death in adolescence.
- Trial name
- NCT03471143
- Testing
- VTS-270
- Where
- United States
- Volunteers
- 4 people
- Sponsor
- Washington University School of Medicine