Clinical trials: Primary Progressive Aphasia
Alzheimer's Disease, Behavioral Variant Frontotemporal Dementia
Open-Label Study of Leuco-methylthioninium Bis(Hydromethanesulfonate) (LMTM) in Subjects With Alzheimer's Disease or Behavioral Variant Frontotemporal Dementia (bvFTD)
The purpose of this study is to provide subjects who have completed participation in a Phase 2 or Phase 3 trial of LMTM continued access to therapy and to evaluate the long-term safety of LMTM.
- Trial name
- NCT02245568
- Testing
- LMTM
- Where
- 135 sites in United States, Australia and 15 more
- Volunteers
- 913 people
- Sponsor
- TauRx Therapeutics Ltd
Motor Neuron Disease, Amyotrophic Lateral Sclerosis
Clinical Characteristics, Natural History, Health Care Measures, and Genetic Screening in Patients With ALS
Patients with sporadic ALS (sALS), which refers to those without a family history of ALS, are typically not subjected to genetic investigations as part of their standard care. Therefore, their mutation status is often unknown.
- Trial name
- ID-ALS (NCT05852405)
- Where
- Germany
- Volunteers
- 2,000 people
- Sponsor
- Ambulanzpartner Soziotechnologie APST GmbH
Progressive Supranuclear Palsy (PSP)
Safety, Tolerability and Pharmacokinetics of Multiple Ascending Doses of NIO752 in Progressive Supranuclear Palsy
This is a phase 1, multi-center, double-blind, placebo-controlled, multiple dose escalation study with NIO752 in progressive supranuclear palsy (PSP) participants.
- Trial name
- NCT04539041
- Testing
- antisense oligonucleotide
- Where
- 12 sites in United States, Canada and 2 more
- Volunteers
- 59 people
- Sponsor
- Novartis Pharmaceuticals
Niemann-Pick Disease, Type C
Safety and Tolerability Study of VTS-270 in Pediatric Participants With Niemann-Pick Type C (NPC) Disease
This is a Phase-2, multicenter, multiple dose, open-label, 2-part evaluation study which will primarily assess the safety and tolerability of VTS-270 (2-hydroxypropyl beta-cyclodextrin [HP-β-CD]) in pediatric participants with…
- Trial name
- NCT03687476
- Testing
- VTS-270
- Sponsor
- Vtesse, LLC, a Mallinckrodt Pharmaceuticals Company
Alzheimer's Disease, Frontotemporal Dementia
Imaging Characteristics of Florbetapir 18F in Patients With Frontotemporal Dementia, Alzheimer's Disease and Normal Controls.
Study 18F-AV-45-010 is designed to evaluate the cerebral uptake of florbetapir 18F as measured by PET imaging in frontotemporal disorder (FTD) in comparison to cognitively normal volunteers and subjects with Alzheimer's disease…
- Trial name
- NCT01890343
- Testing
- florbetapir 18F, 18F-FDG
- Where
- United Kingdom
- Volunteers
- 34 people, healthy volunteers welcome
- Sponsor
- Avid Radiopharmaceuticals
Niemann-Pick Type C Disease
A Study to Evaluate the Safety and Efficacy of Oral Nizubaglustat (AZ-3102) in Late-infantile and Juvenile Forms of Niemann-Pick Type C Disease (NPC)
An 18-month double-blind, randomized, placebo-controlled, multicenter, Phase 3 study to evaluate the safety and efficacy of oral nizubaglustat (AZ-3102) in late-infantile and juvenile forms of Niemann-Pick type C disease
- Trial name
- NCT07082725
- Testing
- Nizubaglustat
- Where
- 34 sites in United States, Argentina and 13 more
- Volunteers
- 72 people
- Sponsor
- Azafaros B.V.
Niemann-Pick Disease, Type C1
Hydroxypropyl Beta Cyclodextrin for Niemann-Pick Type C1 Disease
Background: Hydroxypropyl beta cyclodextrin (HPBCD) is being tested for a disease called Niemann-Pick disease type C1 (NPC1). NPC1 is a genetic disorder that results in gradual loss of nervous system function.
- Trial name
- NCT01747135
- Testing
- VTS-270
- Where
- United States
- Volunteers
- 14 people
- Sponsor
- Vtesse, LLC, a Mallinckrodt Pharmaceuticals Company
Lysosomal Acid Lipase Deficiency
Clinical Study In Infants With Rapidly Progressive Lysosomal Acid Lipase Deficiency
This was an open-label, repeat-dose, study of sebelipase alfa in infants with rapidly progressive lysosomal acid lipase deficiency (LAL-D). Eligible participants received once-weekly infusions of sebelipase alfa for up to 3 years.
- Trial name
- NCT02193867
- Testing
- Sebelipase Alfa
- Where
- 5 sites in United States, Finland and 2 more
- Volunteers
- 10 people
- Sponsor
- Alexion Pharmaceuticals, Inc.
Frontotemporal Dementia, FTD
A Study of PBFT02 in Participants With FTD and Mutations in the Granulin Precursor (GRN) or C9ORF72 Genes
PBFT02 is a gene therapy for frontotemporal dementia intended to deliver a functional copy of the GRN gene to the brain. This study will assess the safety, tolerability and efficacy of this treatment in patients with…
- Conditions
- Frontotemporal Dementia, FTD, FTD-GRN, Dementia Frontotemporal
- Trial name
- upliFT-D (NCT04747431)
- Testing
- PBFT02
- Where
- 10 sites in United States, Australia and 3 more
- Volunteers
- 30 people
- Sponsor
- Passage Bio, Inc.
Alzheimer Disease
COGNIShunt® System for Alzheimer's Disease
This is a study of the effect on the progression of Alzheimer's Disease of a surgically implanted shunt (tube) to increase the flow of cerebrospinal fluid and improve the clearance of potential neurotoxins from the fluid bathing…
- Trial name
- NCT00056628
- Testing
- The COGNIShunt® System
- Volunteers
- 250 people
- Sponsor
- Eunoe
Dementia, Alzheimer Disease
Feasibility of Passive Data Collection in Dementia Subjects With Agitation
This is a multi-center, observational, feasibility study, to evaluate long term passive data collection, data quality, and user experience of HealthMode Agitation (Apps) to collect motion, location, physiological, and audio data…
- Conditions
- Dementia, Alzheimer Disease, Vascular Dementia, Frontotemporal Dementia
- Trial name
- NCT04413851
- Where
- 2 sites in United States
- Volunteers
- 1 people
- Sponsor
- HealthMode Inc.
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
Neurologic Disorders, Nervous System Diseases
Neurologic Stem Cell Treatment Study
This is a human clinical study involving the isolation of autologous bone marrow derived stem cells (BMSC) and transfer to the vascular system and inferior 1/3 of the nasal passages in order to determine if such a treatment will…
- Conditions
- Neurologic Disorders, Nervous System Diseases, Neurodegenerative Diseases, Neurological Disorders
- Trial name
- NEST (NCT02795052)
- Testing
- Intravenous and Intranasal BMSC
- Where
- 3 sites in United States, United Arab Emirates
- Volunteers
- 500 people
- Sponsor
- MD Stem Cells
Mild Alzheimer's Disease
Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of IONIS-MAPTRx in Patients With Mild Alzheimer's Disease
The purpose of this study was to assess the safety, tolerability, pharmacokinetics, and pharmacodynamics of IONIS-MAPTRx in patients with Mild Alzheimer's Disease.
- Trial name
- NCT03186989
- Testing
- IONIS MAPTRx
- Where
- 13 sites in Canada, Finland and 4 more
- Volunteers
- 46 people
- Sponsor
- Ionis Pharmaceuticals, Inc.
Alzheimer Disease, Mild Cognitive Impairment
Longitudinal Cognitive Assessment by BoCA
The Boston Cognitive Assessment (BoCA) is a self-administered online test intended for longitudinal cognitive monitoring. BoCA uses random not-repeating tasks to minimize learning effects.
- Conditions
- Alzheimer Disease, Mild Cognitive Impairment, Vascular Dementia, Frontotemporal Dementia
- Trial name
- BoCA (NCT04114994)
- Testing
- Boston Cognitive Assessment (BoCA)
- Where
- United States
- Volunteers
- 10,000 people, healthy volunteers welcome
- Sponsor
- Alzheimer's Light LLC
Frontotemporal Dementia, Pick Complex
A Pilot Study to Explore the Safety and Tolerability of Galantamine HBr in the Treatment of Pick Complex/Frontotemporal Dementia
The purpose of this study is to explore the safety and tolerability and the efficacy of galantamine treatment in subjects with Pick Complex/ Frontotemporal Dementia (PC/FTD).
- Trial name
- NCT00416169
- Testing
- galantamine hydrobromide
- Volunteers
- 41 people
- Sponsor
- Johnson & Johnson Pharmaceutical Research & Development, L.L.C.
Niemann-Pick Type C Disease
Niemann-Pick Type C Treatment With Adrabetadex for Symptoms of Brain and Nervous System
This study was amended from expanded access to a clinical trial. Information will be collected about long-term safety and effectiveness of adrabetadex shots in the spine every 2 weeks.
- Trial name
- NCT03643562
- Testing
- Adrabetadex
- Where
- 17 sites in United States
- Volunteers
- 27 people
- Sponsor
- Mandos LLC
Healthy Volunteers, Tauopathies
A First-in-Human Study to Assess Single Doses of APNmAb005 in Healthy Participants
This is a Phase 1, first-in-human (FIH), double-blinded, placebo-controlled study where healthy subjects are randomly allocated to receive APNmAb005 or placebo.
- Conditions
- Healthy Volunteers, Tauopathies, Alzheimer Disease
- Trial name
- NCT05344989
- Testing
- APNmAb005
- Where
- United States
- Volunteers
- 40 people, healthy volunteers welcome
- Sponsor
- APRINOIA Therapeutics, LLC
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
Frontotemporal Dementia (FTD), Amyotrophic Lateral Sclerosis (ALS)
[18F]ACI-19626 PET in TDP-43 Proteinopathies
The goal of this clinical trial is to test whether we can reliably and safely measure the accumulation of pathological protein TDP-43 [involved in rare forms of dementia such as frontotemporal dementia (FTD) and in amyotrophic…
- Conditions
- Frontotemporal Dementia (FTD), Amyotrophic Lateral Sclerosis (ALS), TDP-43 Proteinopathies, Suspected Limbic Predominant Age-related TDP-43 Encephalopathy (LATE)
- Trial name
- NCT06891716
- Testing
- [18F]ACI-19626
- Where
- Netherlands
- Volunteers
- 45 people, healthy volunteers welcome
- Sponsor
- AC Immune SA