Clinical trials: Frontotemporal Dementia
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.
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
Sphingomyelin Lipidosis
Efficacy, Safety, Pharmacodynamic, and Pharmacokinetics Study of Olipudase Alfa in Patients With Acid Sphingomyelinase Deficiency
Primary Objective: The primary objective of this phase 2/3 study was to evaluate the efficacy of olipudase alfa (recombinant human acid sphingomyelinase) administered intravenously once every 2 weeks for 52 weeks in adult…
- Trial name
- ASCEND (NCT02004691)
- Testing
- Olipudase alfa
- Where
- 24 sites in United States, Argentina and 15 more
- Volunteers
- 36 people, healthy volunteers welcome
- Sponsor
- Genzyme, a Sanofi Company
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
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
Dementia, Alzheimer Disease
Efficacy Study of Kinto Care Coaching for Dementia Family Caregivers
The goal of this clinical trial is to evaluate Kinto's Care Coaching intervention for dementia caregivers. The main questions it aims to answer are: Does the intervention help caregivers to address their general caregiving goals…
- Conditions
- Dementia, Alzheimer Disease, Lewy Body Disease, Vascular Dementia
- Trial name
- NCT05916664
- Testing
- Kinto Care Coaching
- Where
- United States
- Volunteers
- 495 people, healthy volunteers welcome
- Sponsor
- Kinto
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.
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
Niemann-Pick Disease, Type C
N-Acetyl-L-Leucine for Niemann-Pick Disease, Type C (NPC)
This is a multinational, multicenter, open-label, rater-blinded prospective Phase II study which will assess the safety and efficacy of N-Acetyl-L-Leucine (IB1001) for the treatment of Niemann-Pick type C disease (NPC).
- Trial name
- NCT03759639
- Testing
- IB1001
- Where
- 9 sites in United States, Germany and 3 more
- Volunteers
- 33 people
- Sponsor
- IntraBio Inc
Semantic Dementia
Revealing Engagement Dynamics Among Semantic Dementia Patients
The study intends to investigate the personal experiences of semantic dementia patients who take part in a separate clinical study including a specific medication intervention.
- Trial name
- NCT06218732
- Where
- United States
- Volunteers
- 500 people
- Sponsor
- Power Life Sciences 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
Alzheimer Disease, Mild Cognitive Impairment
Dementia Signal Development Study of Nautilus NeuroWave TM for the Detection of Dementia
This study is being performed to generate data regarding brain vibration /oscillation differences between individuals with dementia and normal controls.
- Conditions
- Alzheimer Disease, Mild Cognitive Impairment, Frontotemporal Lobar Degeneration, Memory Disorders
- Trial name
- NCT02333942
- Testing
- Nautilus NeuroWaveTM System
- Where
- United States
- Volunteers
- 41 people, healthy volunteers welcome
- Sponsor
- Jan Medical, Inc.
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
A Phase 3 Study to Evaluate Efficacy and Safety of AL001 in Frontotemporal Dementia (INFRONT-3)
A phase 3 double blind, placebo controlled study evaluating the efficacy and safety of AL001 in participants at risk for or with frontotemporal dementia due to heterozygous mutations in the progranulin gene.
- Trial name
- NCT04374136
- Testing
- Part 1 Blinded - AL001, Part 2 (OLE Treatment) - AL001
- Where
- 44 sites in United States, Argentina and 14 more
- Volunteers
- 119 people
- Sponsor
- Alector Inc.
Niemann-Pick Disease, Type C
Early Access Program With Arimoclomol in US Patients With NPC
NPC is a rare, relentlessly progressive, neurological disease and associated with serious morbidity and shortened life expectancy.
- Trial name
- NCT04316637
- Testing
- Arimoclomol
- Where
- 13 sites in United States
- Sponsor
- ZevraDenmark
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
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
GM2 Gangliosidosis, Niemann-Pick Disease, Type C
Phase 2 Study Evaluating the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Oral AZ-3102 in Patients With GM2 Gangliosidosis or Niemann-Pick Type C Disease
This phase 2 is a randomized, double-blind, placebo controlled, 12 weeks study with daily oral administration of AZ-3102 aiming to evaluate the safety and pharmacokinetic (PK) profile in GM2 Gangliosidosis and Niemann-Pick type C…
- Trial name
- RAINBOW (NCT05758922)
- Testing
- AZ-3102 (Dose 1), AZ-3102 (Dose 2)
- Where
- 3 sites in Brazil
- Volunteers
- 13 people
- Sponsor
- Azafaros B.V.
Niemann-Pick Diseases
Induced Pluripotent Stem Cells for Niemann Pick Disease
Establishment of individualized human cellular disease models based on induced pluripotent stem cells that reflect the broad heterogeneous phenotypic spectrum of Niemann Pick disease
- Trial name
- IPSNPABC (NCT03883750)
- Where
- Pakistan
- Volunteers
- 40 people
- Sponsor
- CENTOGENE GmbH Rostock
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.