Clinical trials: FTD
Niemann-Pick Disease Type C, GM2 Gangliosidosis
Long-Term Safety and Tolerability of Nizubaglustat in Participants With Rare Lysosomal Disorders
This is an open-label extension trial to evaluate the long-term safety and tolerability of nizubaglustat (AZ-3102) in participants with rare lysosomal disorders who have participated in a previous nizubaglustat trial and wish to…
- Conditions
- Niemann-Pick Disease Type C, GM2 Gangliosidosis, GM1 Gangliosidosis, Gaucher Disease Type 3
- Trial name
- HARBOR (NCT07885917)
- Testing
- AZ-3102
- Volunteers
- 218 people
- Sponsor
- Azafaros B.V.
Niemann-Pick Disease, Type C
Safety and Efficacy of Miglustat in Chinese NPC Patients
This is a prospective, multi-center, open-label, non-randomized, single-arm Phase IV confirmatory study. Approximately 19 subjects with Niemann Pick Type C disease (NPC) will be enrolled in this study.
- Trial name
- NCT03910621
- Testing
- Miglustat
- Where
- 2 sites in China
- Volunteers
- 17 people
- Sponsor
- Actelion
Sphingomyelin Lipidosis
Safety, Tolerability, PK, and Efficacy Evaluation of Repeat Ascending Doses of Olipudase Alfa in Pediatric Patients <18 Years of Age With Acid Sphingomyelinase Deficiency
Primary Objective: To evaluate the safety and tolerability of olipudase alfa administered intravenously in pediatric participants every 2 weeks for 64 weeks.
- Trial name
- ASCEND-Peds (NCT02292654)
- Testing
- Olipudase alfa
- Where
- 6 sites in United States, Brazil and 4 more
- Volunteers
- 20 people
- Sponsor
- Genzyme, a Sanofi Company
Memory Disorders, Alzheimer Disease
Evaluation of a Handheld Event Related Potential (ERP)/Quantitative Electroencephalography (qEEG) System (COGNISION™) as a Useful Cognitive Biomarker for Alzheimer's Disease.
The proposed study is designed to evaluate the performance of the COGNISION™ System as a tool to assist physicians in diagnosing Alzheimer's Disease (AD) in real-world clinical settings.
- Conditions
- Memory Disorders, Alzheimer Disease, Dementia, Cognitive Impairment
- Trial name
- COGNISION™ (NCT00938665)
- Testing
- COGNISION™ System
- Where
- 7 sites in United States
- Volunteers
- 204 people, healthy volunteers welcome
- Sponsor
- Neuronetrix, Inc.
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
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
Niemann-Pick Disease, Type C
A Prospective Non-therapeutic Study in Patients Diagnosed With Niemann-Pick Disease Type C
This is a prospective non-therapeutic observational study in NP-C patients. The aim is to characterize the individual patient disease progression profile through the historical and 6 months prospective evaluation of clinical…
- Trial name
- NCT02435030
- Where
- 16 sites in Denmark, France and 6 more
- Volunteers
- 35 people
- Sponsor
- ZevraDenmark
Mild Cognitive Impairment, Dementia
COMPARATIVE EFFECTIVENESS OF MCI and DEMENTIA TREATMENTS IN A COMMUNITY-BASED DEMENTIA PRACTICE
This retrospective study is a more extensive, confirmatory analysis of the cognitive and functional outcomes initially seen in 2 groups of MCI/dementia patients in Springfield, MA and compares specialized dementia care and a…
- Conditions
- Mild Cognitive Impairment, Dementia, Hypoxia, Hyperhomocysteinemia
- Trial name
- NCT02860338
- Volunteers
- 900 people
- Sponsor
- Clionsky Neuro Systems Inc.
Advanced Solid Tumors
Study of the Mass Balance of Oral FTD and TPI as Components of TAS-102 in Patients With Advanced Solid Tumors
The purpose of this study is to evaluate, in patients with advanced solid tumors, the mass balance of FTD and TPI after a single dose of TAS-102 with a light tracer dose of [14C]FTD or [14C]TPI.
- Trial name
- NCT02031055
- Testing
- TAS-102 with a light tracer dose of [14C]FTD, TAS-102 with a light tracer dose of [14C] TPI, TAS-102 tablets
- Where
- United States
- Volunteers
- 8 people
- Sponsor
- Taiho Oncology, Inc.
Niemann-Pick Disease, Type C1
Open-Label Study of Long-Term Safety and Efficacy of Intravenous Trappsol Cyclo (HPβCD) in Niemann-Pick Disease Type C
The purpose of this study is to provide continued access to treatment for NPC-1 after participation and completion of the Phase I trial CTD-TCNPC-101, when administered at doses of 1500 mg/kg and 2500 mg/kg by slow IV infusion…
- Trial name
- NCT03893071
- Testing
- Hydroxypropyl-β-cyclodextrin
- Where
- United States
- Volunteers
- 12 people
- Sponsor
- Cyclo Therapeutics, 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
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
18-months Safety Follow-up Study of AADvac1, an Active Tau Vaccine for Alzheimer's Disease
This follow-up study continues to observe patients who have completed the phase 1 trial of AADvac1, for another 18 months. Long-term safety and behavior of the immune response to AADvac1 over time are the main points of interest.
- Trial name
- FUNDAMANT (NCT02031198)
- Testing
- AADvac1
- Where
- 4 sites in Austria
- Volunteers
- 25 people
- Sponsor
- Axon Neuroscience SE
Niemann-Pick Disease, Type C
A Pivotal Study of N-Acetyl-L-Leucine on Niemann-Pick Disease Type C
A pivotal, randomized, double-blind, placebo controlled, multi-center therapeutic study for patients age 4 and older with a confirmed diagnosis of Niemann Pick disease type C (NPC).
- Trial name
- NCT05163288
- Testing
- N-Acetyl-L-Leucine
- Where
- 14 sites in United States, Australia and 6 more
- Volunteers
- 53 people
- Sponsor
- IntraBio Inc
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.
Frontotemporal Dementia, FTD
A Study to Evaluate the Safety and Effect of AVB-101, a Gene Therapy Product, in Subjects With a Genetic Sub-type of Frontotemporal Dementia (FTD-GRN)
The goal of this clinical study is to learn about an investigational gene therapy product called AVB-101, which is designed to treat a disease called Frontotemporal Dementia with Progranulin Mutations (FTD-GRN).
- Conditions
- Frontotemporal Dementia, FTD, FTD-GRN, Dementia, Frontotemporal
- Trial name
- ASPIRE-FTD (NCT06064890)
- Testing
- Intrathalamic AAV.PGRN administration, Intrathalamic AVB-101
- Where
- 19 sites in United States, Belgium and 7 more
- Volunteers
- 18 people
- Sponsor
- AviadoBio Ltd
Dementia, Frontotemporal
A Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of VES001 in Healthy Participants
This is a Phase 1, randomized, placebo-controlled, double-blind study to evaluate the safety, tolerability, pharmacokinetics (PK), and pharmacodynamics (PD) of single and multiple doses of VES001 in a two part followed by a…
- Trial name
- SORT-IN-1 (NCT06226064)
- Testing
- VES001
- Where
- Netherlands
- Volunteers
- 78 people, healthy volunteers welcome
- Sponsor
- Vesper Biotechnologies ApS
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
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 Disease, Type C1
Safety and Efficacy of Intravenous Trappsol Cyclo (HPBCD) in Niemann-Pick Type C Patients
This research study is being conducted to find out whether Trappsol® Cyclo™, an experimental treatment for people with Niemann-Pick disease Type C1 (NPC-1) is safe at 3 different dose levels and what effects it has on people who…
- Trial name
- NCT02912793
- Testing
- Hydroxypropyl-beta-cyclodextrin
- Where
- 5 sites in Israel, Sweden and 1 more
- Volunteers
- 12 people
- Sponsor
- Cyclo Therapeutics, Inc.