Clinical trials: Primary Progressive Aphasia(PPA)
Rare Diseases
European Registry on Rare Neurological Diseases
The recent implementation of European Reference Networks for Rare Diseases (ERNs) is an unprecedented move to improve the care of patients suffering from rare health disorders by transnational collaboration.
- Trial name
- ERN-RND reg (NCT04319796)
- Testing
- Data set as defined by the ERN Research Workgroup of the European Commission
- Volunteers
- 5,000 people
- Sponsor
- University Hospital Tuebingen
Niemann-Pick Disease, Type C
Establishment of Genomic and Phenotypic Database for Niemann-Pick Disease, Type C
Background: Niemann-Pick type C (NPC) disease is a rare, progressive neurodegenerative disease that affects mainly the brain, liver, and spleen but also other parts of the body.
- Trial name
- NCT05588167
- Where
- United States
- Volunteers
- 100 people
- Sponsor
- Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
Niemann-Pick Disease, Type C
VTS-270 to Treat Niemann-Pick Type C1 (NPC1) Disease
Due to different study designs, the sponsor separated Part C into a separate registration (NCT04958642), leaving Parts A/B here in NCT02534844.
- Trial name
- NCT02534844
- Testing
- Parts A/B: Adrabetadex
- Where
- 29 sites in United States, Australia and 7 more
- Volunteers
- 56 people
- Sponsor
- Mandos LLC
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.
Rare Disorders, Undiagnosed Disorders
Rare Disease Patient Registry & Natural History Study - Coordination of Rare Diseases at Sanford
The Coordination of Rare Diseases at Sanford (CoRDS) is an international patient registry that serves as a centralized resource for rare disease research.
- Conditions
- Rare Disorders, Undiagnosed Disorders, Disorders of Unknown Prevalence, Cornelia De Lange Syndrome
- Trial name
- CoRDS (NCT01793168)
- Where
- United States
- Volunteers
- 20,000 people
- Sponsor
- Sanford Health
Primary Progressive Aphasia
Language in Primary Progressive Aphasia
The purpose of this study is to further define the neurological and linguistic deterioration in primary progressive aphasia.
- Trial name
- NCT00537004
- Where
- United States
- Volunteers
- 500 people
- Sponsor
- Northwestern University
Semantic Dementia, Alzheimer Disease
Metacognition in Semantic Dementia: Comparison With Alzheimer's Disease
This study aims at exploring patients' ability to monitor their own memory performance depending on their primary deficit and the type of memory involved in the criterion task.
- Conditions
- Semantic Dementia, Alzheimer Disease, Healthy
- Trial name
- META-DEM (NCT04597827)
- Testing
- Metacognition tests
- Where
- 7 sites in France, Switzerland
- Volunteers
- 40 people, healthy volunteers welcome
- Sponsor
- University Hospital, Grenoble
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
Anxiety, bvFTD
Auditory Beat Stimulation and Behavioural Variant of Frontotemporal Dementia
The goal of this study is to examine the effects of auditory beat stimulation on anxiety in patients diagnosed with bvFTD. Main aims are: to ascertain whether anxiety in bvFTD patients can be modulated using auditory beat…
- Trial name
- NCT05917886
- Testing
- Auditory beat stimulation: 8Hz monaural beat stimulation
- Where
- Germany
- Volunteers
- 11 people
- Sponsor
- University Hospital, Bonn
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
Combined Intrathecal and Intravenous VTS-270 Therapy for Liver and Neurological Disease Associated With Niemann-Pick Disease, Type C1
Background: For people who have Niemann-Pick disease, type C1 (NPC1), cholesterol and other fats have trouble moving out of liver and other tissue cells. This makes the cells sick.
- Trial name
- NCT03887533
- Testing
- VTS-270
- Where
- United States
- Volunteers
- 2 people
- Sponsor
- Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
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.
Amyotrophic Lateral Sclerosis, Frontotemporal Dementia
Phenotype, Genotype & Biomarkers in ALS and Related Disorders
The goals of this study are: (1) to better understand the relationship between the phenotype and genotype of amyotrophic lateral sclerosis (ALS) and related diseases, including primary lateral sclerosis (PLS), hereditary spastic…
- Conditions
- Amyotrophic Lateral Sclerosis, Frontotemporal Dementia, Primary Lateral Sclerosis, Hereditary Spastic Paraplegia
- Trial name
- NCT02327845
- Where
- 15 sites in United States, Germany and 1 more
- Volunteers
- 708 people, healthy volunteers welcome
- Sponsor
- University of Miami
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.
Alzheimer's Disease, Brain Neoplasm
PET Scan of Brain Metabolism in Relation to Age and Disease
The main source of energy for the brain comes from a combination of oxygen and glucose (sugar). For brain cells to function normally they must receive a constant supply of these nutrients.
- Conditions
- Alzheimer's Disease, Brain Neoplasm, Niemann Pick Disease
- Trial name
- NCT00001972
- Testing
- 15 O Water
- Where
- United States
- Volunteers
- 123 people, healthy volunteers welcome
- Sponsor
- National Institute of Neurological Disorders and Stroke (NINDS)
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