Clinical trials: Primary Progressive Aphasia(PPA)
Niemann-Pick Disease, Type C
Open-label Study of VTS-270 in Participants With Neurologic Manifestations of Niemann-Pick Type C1
This is a multicenter, multinational, open-label study of VTS-270 to evaluate the long-term safety and tolerability of VTS-270 (2-hydroxypropyl-β-cyclodextrin) in participants transitioning from Study VTS301 (Parts A/B…
- Trial name
- NCT03879655
- Testing
- VTS-270
- Where
- Costa Rica
- Volunteers
- 2 people
- Sponsor
- Mandos LLC
Healthy, Frontotemporal Dementia
A First in Human Study in Healthy Volunteers and in Participants With Frontotemporal Dementia With Granulin (GRN) Mutation
A first in human phase 1 study in healthy volunteers and participants with Granulin (GRN) mutation causative of frontotemporal dementia (FTD) to assess AL001 safety, tolerability, pharmacokinetics, and pharmacodynamics
- Trial name
- NCT03636204
- Testing
- AL001
- Where
- 8 sites in United States, Canada and 1 more
- Volunteers
- 64 people, healthy volunteers welcome
- Sponsor
- Alector Inc.
Niemann-Pick Type C Disease, GM1 Gangliosidosis
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, GM1 Gangliosidosis or GM2 Gangliosidosis
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 and in…
- Conditions
- Niemann-Pick Type C Disease, GM1 Gangliosidosis, GM2 Gangliosidosis
- Trial name
- NCT07054515
- Testing
- AZ-3102
- Where
- 39 sites in United States, Argentina and 15 more
- Volunteers
- 147 people
- Sponsor
- Azafaros B.V.
Alzheimer's Disease (AD), Neurodegenerative Disease
A Biomarker-chip Assay in Preserved Red Blood Cells for the Diagnosis of Alzheimer's Disease
The ADCHIP study (ST0067) is a non-interventional, monocentric, prospective study conducted by Amoneta Diagnostics and the Leenaards Memory Center (Lausanne, Switzerland).
- Conditions
- Alzheimer's Disease (AD), Neurodegenerative Disease, Dementia, Frontotemporal Dementia (FTD)
- Trial name
- ADCHIP (NCT07307872)
- Where
- Switzerland
- Volunteers
- 150 people, healthy volunteers welcome
- Sponsor
- Amoneta Diagnostics SAS
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
Amyotrophic Lateral Sclerosis, Frontotemporal Dementia
Open-label Extension (OLE) Study of WVE-004 in Patients With C9orf72-associated Amyotrophic Lateral Sclerosis (ALS) and/or Frontotemporal Dementia (FTD)
This is an OLE study conducted to evaluate the safety, tolerability, pharmacokinetics (PK), pharmacodynamics (PD), and clinical effects of WVE-004 in adult participants with ALS, FTD, or mixed ALS/FTD phenotype with a documented…
- Trial name
- NCT05683860
- Testing
- WVE-004 10 mg Q12W
- Where
- 3 sites in Netherlands, United Kingdom
- Volunteers
- 8 people
- Sponsor
- Wave Life Sciences USA, Inc.
Inherited Metabolic Diseases, Lysosomal Storage Disorders
ALD-101 Adjuvant Therapy of Unrelated Umbilical Cord Blood Transfusion (UCBT) in Patients With Inherited Metabolic Diseases
Eligible research subjects will receive an unrelated umbilical cord blood transfusion as a possible cure for their inherited metabolic disease.
- Conditions
- Inherited Metabolic Diseases, Lysosomal Storage Disorders, Peroxisomal Storage Diseases, Inborn Errors of Metabolism
- Trial name
- UCBT-002 (NCT00654433)
- Testing
- ALD-101
- Where
- 3 sites in United States
- Volunteers
- 40 people
- Sponsor
- Aldagen
Frontotemporal Dementia
Epidemiological Study in FRONtoTemporal Dementia
An international, multicenter, epidemiological observational study aims to investigate the prevalence of genetic etiologies in patients diagnosed with FTD or clinically suspected for FTD.
- Trial name
- EFRONT (NCT05075187)
- Testing
- Genetic Screening
- Where
- 37 sites in Belgium, Germany and 5 more
- Volunteers
- 2,287 people, healthy volunteers welcome
- Sponsor
- CENTOGENE GmbH Rostock
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.
Neurodegenerative Diseases
Continuation Study for Latozinemab
Continuation study to provide continued access to latozinemab for participants who have previously participated in a latozinemab study
- Trial name
- NCT06111014
- Testing
- Latozinemab
- Where
- 18 sites in United States, Belgium and 8 more
- Volunteers
- 17 people
- Sponsor
- Alector Inc.
Frontotemporal Dementia
Flortaucipir PET Imaging in Subjects With FTD
This study is designed to assess the usefulness of flortaucipir in Positron Emission Tomography (PET) imaging for subjects diagnosed with Frontotemporal Dementia (FTD).
- Trial name
- NCT03040713
- Testing
- Flortaucipir F18, Brain PET scan
- Where
- 3 sites in United States
- Volunteers
- 16 people
- Sponsor
- Avid Radiopharmaceuticals
Progressive Supranuclear Palsy, Corticobasal Degeneration
A Study to Evaluate the Safety, Tolerability and Pharmacodynamics of NIO752 in Primary Tauopathies
This is a phase II study intended to evaluate the safety, tolerability and pharmacodynamics of NIO752 in participants with primary tauopathies.
- Conditions
- Progressive Supranuclear Palsy, Corticobasal Degeneration, Frontotemporal Lobar Degeneration, Tauopathies
- Trial name
- NCT07868328
- Testing
- NIO752
- Volunteers
- 48 people
- Sponsor
- Novartis Pharmaceuticals
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
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'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
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
Frontotemporal Dementia
Phase 1/2 Clinical Trial of LY3884963 in Patients With Frontotemporal Dementia With Progranulin Mutations (FTD-GRN)
Study J4B-MC-OKAA is a Phase 1/2, multi-center, open-label ascending dose, first-in-human study that will evaluate the safety and effect of intra-cisternal LY3884963 administration on progranulin protein (PGRN) levels in patients…
- Trial name
- PROCLAIM (NCT04408625)
- Testing
- LY3884963, Methylprednisolone, Optional Sirolimus
- Where
- 12 sites in United States, Australia and 4 more
- Volunteers
- 35 people
- Sponsor
- Prevail Therapeutics
Nonfluent Variant Primary Progressive Aphasia (nfvPPA)
Clinical Study of Neflamapimod in Patients With Primary Progressive Aphasia
The goal of this exploratory study is to evaluate the effect of neflamapimod in participants with nonfluent variant primary progressive aphasia (nfvPPA).
- Trial name
- NCT07033481
- Testing
- Neflamapimod
- Where
- 7 sites in United States, United Kingdom
- Volunteers
- 20 people
- Sponsor
- EIP Pharma Inc
Alzheimer Disease
Safety Study of AADvac1, a Tau Peptide-KLH-Conjugate Active Vaccine to Treat Alzheimer's Disease
This first-time-in-man study is mainly designed to assess the safety and tolerability of AADvac1 in the treatment of Alzheimer's disease.
- Trial name
- NCT01850238
- Testing
- AADvac1
- Where
- 3 sites in Austria
- Volunteers
- 30 people
- Sponsor
- Axon Neuroscience SE
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