Clinical trials: Pick Disease of the Brain
Niemann-Pick Diseases, Acid Sphingomyelinase Deficiency
A Real-world Long-term Safety and Immunogenicity Study of Olipudase Alfa Therapy in Pediatric Patients Less Than 2 Years of Age With Acid Sphingomyelinase Deficiency (ASMD)
US, multicenter, cohort, open label observational study with primary data collection. Ancillary protocol-specified procedures to address the study objectives (eg, assessment of ADA) may be considered outside the standard of care…
- Trial name
- NCT06192576
- Testing
- Olipudase alfa
- Where
- 6 sites in United States
- Volunteers
- 10 people
- Sponsor
- Sanofi
Fronto-temporal Dementia, Fronto-temporal Lobar Dementia
Vortioxetine for the Treatment of Mood and Cognitive Symptoms in Frontotemporal Dementia
The goal of this clinical trial is to learn if vortioxetine improves mood symptoms and cognition in patients with early-stage behavioral variant Frontotemporal Dementia (bvFTD).
- Conditions
- Fronto-temporal Dementia, Fronto-temporal Lobar Dementia, Frontotemporal Degeneration, Frontotemporal Dementia (FTD)
- Trial name
- NCT06604520
- Testing
- Vortioxetine
- Where
- United States
- Volunteers
- 50 people, healthy volunteers welcome
- Sponsor
- Johns Hopkins University
Stroke, Ischemic
Optimizing the Radiological Selection and Transfer Model in Stroke Patients Presenting 6-24h After Onset (VESTA Study)
We aim to optimize the radiological selection and the transfer model in patients with a 6-24h stroke for a fairer and broader access to a reperfusion treatment. The specific objectives are: 1.
- Trial name
- VESTA (NCT05299034)
- Testing
- Multiparametric imaging, Non contrast CT+CT angiography
- Where
- Spain
- Volunteers
- 2,173 people
- Sponsor
- Germans Trias i Pujol Hospital
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
Sphingomyelin Lipidosis
Compassionate Use Program for Olipudase Alfa Enzyme Replacement Therapy for Patients With Chronic Acid Sphingomyelinase Deficiency (ASMD)
The objective of this program is to provide access to enzyme replacement therapy (ERT) with olipudase alfa for certain patients with ASMD, a severe, life threatening disease, that could not participate in the olipudase clinical…
- Trial name
- NCT04877132
- Testing
- olipudase alfa (GZ402665)
- Sponsor
- Sanofi
Mild Cognitive Impairment
Lithium As a Treatment to Prevent Impairment of Cognition in Elders
Alzheimer's disease (AD) is the most common cause of dementia in adults 65 years and older. AD leads to a complete loss of memory and independent function, and presently there is no cure.
- Trial name
- LATTICE (NCT03185208)
- Testing
- Lithium Carbonate
- Where
- United States
- Volunteers
- 83 people
- Sponsor
- Ariel Gildengers, MD
Dementia
Efficacy and Tolerability of Memantine in Frontotemporal Dementia (FTD) Patients
The purpose of this trial is to assess the efficacy and tolerability of memantine (anti-excitotoxic, neuroprotective treatment currently used in Alzheimer's disease [AD]) in frontotemporal dementia patients after a one-year…
- Trial name
- NCT00200538
- Testing
- memantine
- Where
- France
- Volunteers
- 52 people
- Sponsor
- Nantes University Hospital
Alzheimer's Disease
Transcranial Electromagnetic Treatment (TEMT) Against Alzheimer's Disease
This is a second extension of EM 1000-1 wherein mild/moderate AD subjects who participated in the original study have completed participation in a first extension of 4-months.
- Trial name
- NCT04271163
- Testing
- MemorEM
- Where
- United States
- Volunteers
- 6 people
- Sponsor
- NeuroEM Therapeutics, Inc.
Brain and Central Nervous System Tumors
Improving the Selection of Patients With Glioblastoma Multiforme for Treatment With Epidermal Growth Factor Receptor Inhibitor Therapies
RATIONALE: Studying samples of tissue from patients with cancer in the laboratory may help doctors identify and learn more about biomarkers related to cancer.
- Trial name
- NCT00897663
- Testing
- gene expression analysis, microarray analysis, protein expression analysis
- Where
- 10 sites in United States
- Volunteers
- 56 people
- Sponsor
- Alliance for Clinical Trials in Oncology
Acute Ischemic Stroke
NoNO-42 Trial in Acute Ischemic Stroke Patients Selected for Thrombolysis With or Without Endovascular Thrombectomy
ACT-42 is a domain of the ACT-GLOBAL platform (NCT06352632). This trial is a Phase 2b, multicenter, prospective, randomized, open label, blinded-endpoint (PROBE) controlled single-dose adaptive trial.
- Trial name
- NCT06403267
- Testing
- NoNO-42
- Where
- 9 sites in Canada
- Volunteers
- 600 people
- Sponsor
- NoNO Inc.
Niemann-Pick Disease, Type C
Adrabetadex to Treat Niemann-Pick Type C1 (NPC1) Disease
Due to different study designs, the sponsor separated Part C into this separate registration (NCT04958642), leaving Parts A/B in NCT02534844.
- Trial name
- NCT04958642
- Testing
- Adrabetadex
- Where
- 30 sites in United States, Australia and 7 more
- Volunteers
- 66 people
- Sponsor
- Mandos LLC
Adrenoleukodystrophy, Batten Disease
UCB Transplant of Inherited Metabolic Diseases With Administration of Intrathecal UCB Derived Oligodendrocyte-Like Cells
The primary objective of the study is to determine the safety and feasibility of intrathecal administration of DUOC-01 in patients who are undergoing standard treatment with umbilical cord blood transplant (UCBT) for inborn…
- Conditions
- Adrenoleukodystrophy, Batten Disease, Mucopolysaccharidosis II, Leukodystrophy, Globoid Cell
- Trial name
- DUOC-01 (NCT02254863)
- Testing
- DUOC-01
- Where
- United States
- Volunteers
- 40 people
- Sponsor
- Joanne Kurtzberg, MD
GM2 Gangliosidosis, Niemann-Pick Type C Disease
A Study to Evaluate the Safety and Efficacy of Nizubaglustat (AZ-3102) in Patients With GM2 Gangliosidosis or Niemann-Pick Type C Disease
This open-label study aims to gather long-term safety, tolerability, PK, biomarker, and clinical efficacy data relating to daily administration of Nizubaglustat in participants previously enrolled in the Phase 2 RAINBOW study…
- Trial name
- PRISMA (NCT07399704)
- Testing
- AZ-3102
- Where
- 3 sites in Brazil
- Volunteers
- 21 people
- Sponsor
- Azafaros B.V.
Dementia, Frontotemporal Dementia
Study in Asymptomatic GRN-FTD Patients to Investigate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of VES001
This is an open-label, multiple dosing study in asymptomatic GRN-FTD carriers to investigate the safety, tolerability, PK, and PD of VES001.
- Conditions
- Dementia, Frontotemporal Dementia, Asymptomatic Condition
- Trial name
- SORT-IN-2 (NCT06705192)
- Testing
- VES001
- Where
- 2 sites in Netherlands, United Kingdom
- Volunteers
- 6 people
- Sponsor
- Mads Kjolby
Alzheimer Disease, Cognitive Impairment
The Clinical Impact of the Novel Alzheimer's Blood-based Biomarkers
The goal of this observational study is to determine whether the early adoption of blood-based biomarkers for Alzheimer's disease is associated with an impact on etiological diagnosis, patient's management, emotional impact…
- Conditions
- Alzheimer Disease, Cognitive Impairment, Dementia, Neurodegenerative Diseases
- Trial name
- PLASMAR (NCT06246019)
- Testing
- Early adoption of blood-based biomarkers for Alzheimer's disease
- Where
- Spain
- Volunteers
- 200 people
- Sponsor
- Hospital del Mar Research Institute (IMIM)
Alzheimer, Alzheimer Dementia (AD)
Restoring Cervical Lymphatic Drainage in Alzheimer's Disease
Alzheimer's disease (AD) is a progressive neurodegenerative disorder associated with the accumulation of abnormal proteins, including amyloid-beta and tau, in the brain.
- Trial name
- LymphDAD (NCT07854327)
- Testing
- Cervical lymphovenous anastomosis (LVA)
- Where
- Italy
- Volunteers
- 5 people
- Sponsor
- University of Siena
Alzheimer Disease
Alzheimer's PET Imaging in Racially/Ethnically Diverse Adults
The study employs tau positron emission tomography (PET) imaging in a well-characterized multi-racial/ethnic cohort to examine the extent to which tau pathology is associated with cognition, differences in tau pathology across…
- Trial name
- NCT03706261
- Testing
- 18F-MK-6240, 18F-Florbetaben
- Where
- United States
- Volunteers
- 145 people, healthy volunteers welcome
- Sponsor
- Adam Brickman
Primary Progressive Nonfluent Aphasia
A 24-month Phase 1 Pilot Study of AADvac1 in Patients With Non Fluent Primary Progressive Aphasia
This study is a pilot trial evaluating the safety and immunogenicity of AADvac1 in patients with the non-fluent variant of Primary Progressive Aphasia.
- Trial name
- AIDA (NCT03174886)
- Testing
- AADvac1 40 µg, AADvac1 160 µg
- Where
- 3 sites in Germany
- Volunteers
- 33 people
- Sponsor
- Axon Neuroscience SE
Brain Tumors, Radiotherapy
The Cognitive-Behavioral Dysfunctions and the Potential of Neuroplasticity in Brain Tumors Patients During Radiotherapy
Primary and secondary brain tumors are a constant challenge for the medicine. Tissue sensitivity to ionizing radiation differs and depends on numerous factors and the same dose of radiation may produce different effects in…
- Trial name
- REHABrain (NCT05192447)
- Testing
- Exercise treatment
- Where
- Poland
- Volunteers
- 150 people
- Sponsor
- The Greater Poland Cancer Centre
Mild Cognitive Impairment (MCI), Alzheimer's Disease (AD)
Alzheimer's Disease Neuroimaging Initiative 3
Since its launch in 2004, the overarching aim of the Alzheimer's Disease Neuroimaging Initiative (ADNI) has been realized in informing the design of therapeutic trials in AD.
- Trial name
- ADNI3 (NCT02854033)
- Where
- 59 sites in United States, Canada
- Volunteers
- 1,141 people, healthy volunteers welcome
- Sponsor
- University of Southern California