Clinical trials: Dementia, Frontotemporal
Acid Sphingomyelinase Deficiency (ASMD), Niemann Pick Disease
National Multicentre Study of the Natural History of Acid Sphingo-myelinase Deficiency in Adults and Children
The goal of this study is to describe the natural history of ASMD in adult and paediatric patients with or without specific treatment in order to assess the impact of the disease on their daily lives and quality of life.
- Trial name
- FASMD (NCT06985212)
- Where
- France
- Volunteers
- 200 people
- Sponsor
- Wladimir MAUHIN, Dr
Lysosomal Storage Disorders
Molecular and Cellular Mechanisms of Lysosomal Storage Diseases
The lysosome is a specialized part of the cell that functions to degrade metabolic wastes in the cell. Defects in the functioning of the lysosome result in accumulation and subsequent storage of such metabolic wastes.
- Trial name
- NCT02000310
- Where
- United States
- Volunteers
- 80 people, healthy volunteers welcome
- Sponsor
- O & O Alpan LLC
Primary Progressive Aphasia, Neurodegenerative Diseases
Home-Based Exercise in Primary Progressive Aphasia (HEPPA)
This pilot study aims to evaluate the feasibility of a novel home-based multicomponent exercise program in adults clinically diagnosed with Primary Progressive Aphasia
- Conditions
- Primary Progressive Aphasia, Neurodegenerative Diseases, Logopenic Progressive Aphasia, Semantic Dementia
- Trial name
- HEPPA (NCT06181500)
- Testing
- Home-based multicomponent exercise intervention program
- Where
- 2 sites in Canada
- Volunteers
- 13 people
- Sponsor
- Montreal Heart Institute
Alzheimer Disease, Dementia With Lewy Bodies
Cerebro Spinal Fluid Collection (CSF)
Cognitive neurodegenerative diseases are a major public health issue. At present, the diagnosis of certainty is still based on anatomopathological analyses.
- Conditions
- Alzheimer Disease, Dementia With Lewy Bodies, Frontotemporal Dementia, Parkinson's Disease Dementia
- Trial name
- Analzheimer (NCT04844060)
- Testing
- Lumbar punction
- Where
- France
- Volunteers
- 10,000 people
- Sponsor
- University Hospital, Strasbourg, France
Niemann-Pick Disease, Type C1
Study of Lithium Carbonate to Treat Niemann-Pick Type C1 Disease
This study is planned to study whether lithium carbonate has protective effect on the brain of Niemann-Pick disease type C1.
- Trial name
- NCT03201627
- Testing
- Lithium Carbonate
- Where
- China
- Volunteers
- 18 people
- Sponsor
- Xinhua Hospital, Shanghai Jiao Tong University School of Medicine
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
Niemann-Pick Disease, Niemann-Pick Disease, Type C
Biomarker for Niemann Pick Type C Disease (BioNPC)
Development of a new MS-based biomarker for the early and sensitive diagnosis of Niemann Pick Type C disease from Blood (plasma)
- Trial name
- BioNPC (NCT01306604)
- Where
- 4 sites in Germany, India and 1 more
- Sponsor
- CENTOGENE GmbH Rostock
Insomnia, Dementia
Lemborexant for Insomnia in a Patient With Dementia: An N-of-1 Trial
Insomnia is a highly common, chronic disorder that is distressful for the patient but also for caregivers and can give rise to a heavy burden on the healthcare team.
- Conditions
- Insomnia, Dementia, Frontotemporal Dementia
- Trial name
- NCT06093126
- Testing
- Lemborexant 5 MG
- Where
- Canada
- Volunteers
- 1 people
- Sponsor
- Nova Scotia Health Authority
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