Clinical trials: Primary Progressive Aphasia(PPA)
Neurodegenerative Diseases, Alzheimer Disease
Sociodemographic Factors and Criminal Behaviour Preceding Neurodegenerative Disease - Retrospective Register Study
In this retrospective register study, clinically classified individuals with neurodegenerative disease from the years 2010-2021 will be verified from the clinical records from KUH and Oulu University Hospital (OUH).
- Conditions
- Neurodegenerative Diseases, Alzheimer Disease, Frontotemporal Dementia, Lewy Body Disease
- Trial name
- DEGERWD (NCT06209515)
- Where
- Finland
- Volunteers
- 61,292 people
- Sponsor
- Kuopio University Hospital
Sphingomyelin Lipidosis
Compassionate Use Program for Olipudase Alfa Enzyme Replacement Therapy for Patients With Chronic Acid Sphingomyelinase Deficiency (ASMD)
The objective of this program is to provide access to enzyme replacement therapy (ERT) with olipudase alfa for certain patients with ASMD, a severe, life threatening disease, that could not participate in the olipudase clinical…
- Trial name
- NCT04877132
- Testing
- olipudase alfa (GZ402665)
- Sponsor
- Sanofi
Niemann-Pick Disease, Type C1
Phase 3 Study to Evaluate Intravenous Trappsol(R) Cyclo(TM) in Pediatric and Adult Patients With Niemann-Pick Disease Type C1
A prospective, randomized, double-blind, placebo controlled, multi-center therapeutic study for patients age 3 and older with confirmed diagnosis of Niemann Pick disease type C1 (NPC1).
- Trial name
- TransportNPC (NCT04860960)
- Testing
- Hydroxypropyl-beta-cyclodextrin
- Where
- 35 sites in United States, Argentina and 11 more
- Volunteers
- 94 people
- Sponsor
- Cyclo Therapeutics, Inc.
Niemann-Pick Disease, Type C
Arimoclomol Prospective Study in Participants Diagnosed With Niemann-Pick Disease Type C
A prospective, randomized, double-blind, placebo controlled therapeutic study in participants with confirmed diagnosis of Niemann-Pick disease type C (NPC).
- Trial name
- NCT02612129
- Testing
- arimoclomol
- Where
- 14 sites in United States, Denmark and 7 more
- Volunteers
- 50 people
- Sponsor
- ZevraDenmark
Alzheimer, Alzheimer Dementia (AD)
Restoring Cervical Lymphatic Drainage in Alzheimer's Disease
Alzheimer's disease (AD) is a progressive neurodegenerative disorder associated with the accumulation of abnormal proteins, including amyloid-beta and tau, in the brain.
- Trial name
- LymphDAD (NCT07854327)
- Testing
- Cervical lymphovenous anastomosis (LVA)
- Where
- Italy
- Volunteers
- 5 people
- Sponsor
- University of Siena
Spinal Muscular Atrophy, Fragile X Syndrome
Early Check: Expanded Screening in Newborns
Early Check provides voluntary screening of newborns for a selected panel of conditions. The study has three main objectives: 1) develop and implement an approach to identify affected infants, 2) address the impact on infants and…
- Conditions
- Spinal Muscular Atrophy, Fragile X Syndrome, Fragile X - Premutation, Duchenne Muscular Dystrophy
- Trial name
- NCT03655223
- Testing
- Confirmatory Testing
- Where
- United States
- Volunteers
- 30,000 people, healthy volunteers welcome
- Sponsor
- RTI International
Sickle Cell Disease, Thalassemia
Unrelated Hematopoietic Stem Cell Transplantation(HSCT) for Genetic Diseases of Blood Cells
This is a clinical trial of bone marrow transplantation for patients with the diagnosis of a genetic disease of blood cells that do not have an HLA-matched sibling donor.
- Conditions
- Sickle Cell Disease, Thalassemia, Anemia, Granuloma
- Trial name
- NCT00730314
- Testing
- Hematopoietic stem cell transplantation
- Where
- United States
- Volunteers
- 25 people
- Sponsor
- Children's Hospital Los Angeles
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
Alzheimer Disease
Effect of Lithium and Divalproex in Alzheimer's Disease
This study will examine the effect of the drugs lithium and divalproex (Depakote) on tau proteins, a type of protein in the brain and spinal fluid that are altered in patients with Alzheimer's disease.
- Trial name
- NCT00088387
- Testing
- Divalproex, Lithium
- Where
- United States
- Volunteers
- 35 people
- Sponsor
- National Institute of Neurological Disorders and Stroke (NINDS)
Hurler's Syndrome, Maroteaux-Lamy Syndrome
Hematopoietic Stem Cell Transplantation (HCT) for Inborn Errors of Metabolism
The primary objective of this clinical trial is to evaluate the ability to achieve and sustain donor engraftment in patients with lysosomal and peroxisomal inborn errors of metabolism undergoing hematopoietic stem cell…
- Conditions
- Hurler's Syndrome, Maroteaux-Lamy Syndrome, Sly Syndrome, Alpha Mannosidosis
- Trial name
- NCT00668564
- Testing
- Stem Cell Transplantation, Cyclophosphamide, Campath-1H
- Where
- United States
- Volunteers
- 18 people
- Sponsor
- Masonic Cancer Center, University of Minnesota
Acid Sphingomyelinase Deficiency, Ceroid Lipofuscinosis, Neuronal, 2
ScreenPlus: A Comprehensive, Flexible, Multi-disorder Newborn Screening Program
ScreenPlus is a consented, multi-disorder pilot newborn screening program implemented in conjunction with the New York State Newborn Screening Program that provides families the option to have their newborn(s) screened for a…
- Conditions
- Acid Sphingomyelinase Deficiency, Ceroid Lipofuscinosis, Neuronal, 2, Cerebrotendinous Xanthomatosis, Fabry Disease
- Trial name
- ScreenPlus (NCT05368038)
- Testing
- Confirmatory Testing
- Where
- 9 sites in United States
- Volunteers
- 100,000 people, healthy volunteers welcome
- Sponsor
- Albert Einstein College of Medicine
Stuttering, Developmental, Aphasia, Primary Progressive
Investigating Speech Sequencing in Neurotypical Speakers and Persons With Disordered Speech
Persistent developmental stuttering affects more than three million people in the United States, and it can have profound adverse effects on quality of life.
- Trial name
- NCT05437159
- Testing
- Learning of non-native phoneme combinations: 6 training sessions, Learning of non-native phoneme combinations: 1 training session, Learning of novel multisyllabic nonwords
- Where
- 3 sites in United States
- Volunteers
- 278 people, healthy volunteers welcome
- Sponsor
- Boston University Charles River Campus
Primary Progressive Aphasia
Speech-Language Treatment With Remotely Supervised Transcranial Direct Current Stimulation in Primary Progressive Aphasia
Primary progressive aphasia (PPA) is a disorder characterized by gradual decline in speech-language ability caused by underlying neurodegenerative disease.
- Trial name
- NCT05901233
- Testing
- Remotely supervised Transcranial Direct Current Stimulation (RS-tDCS), Lexical Retrieval Training (LRT), Video Implemented Script Training in Aphasia (VISTA)
- Where
- 2 sites in United States
- Volunteers
- 10 people, healthy volunteers welcome
- Sponsor
- University of Texas at Austin
Niemann-Pick Disease, Type C
Longitudinal Study of Cognition With Niemann-Pick Disease, Type C
Niemann-Pick Disease, Type C (NPC) is a rare neurodegenerative disorder with a wide clinical spectrum and variable age of onset.
- Trial name
- NPC (NCT01899950)
- Where
- United States
- Volunteers
- 70 people
- Sponsor
- Mayo Clinic
Niemann-Pick Disease Type C
Application of Miglustat in Patients With Niemann-Pick Type C
To evaluate the changes in cognitive function after miglustat treatment in Niemann-Pick type C patients.
- Trial name
- NCT01760564
- Testing
- Miglustat
- Where
- Taiwan
- Volunteers
- 5 people
- Sponsor
- National Taiwan University Hospital
Fabry Disease, Gaucher Disease
Complement Activation in the Lysosomal Storage Disorders
The lysosomal storage disorders (LSDs) are monogenic disorders associated with inflammation affecting multiple organs, and early death.
- Conditions
- Fabry Disease, Gaucher Disease, Niemann-Pick Disease, Type C, Lysosomal Storage Diseases
- Trial name
- CATALYST (NCT04189601)
- Testing
- Complement measurements
- Where
- Australia
- Volunteers
- healthy volunteers welcome
- Sponsor
- Melbourne Health
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.
Aphasia, Acquired, Aphasia Primary Progressive
Spaced Retrieval as Treatment for Aphasia
Spaced Repetition training has been found to be more effective than massed repetition for individuals with aphasia. This study seeks to examine the relative efficiency of three kinds of spacing for picture-naming training…
- Trial name
- NCT04623125
- Testing
- Spaced Retrieval Practice
- Where
- Canada
- Volunteers
- 21 people
- Sponsor
- Rotman Research Institute at Baycrest
Frontotemporal Dementia, Family Members
Genetic Frontotemporal Dementia Initiative for Neurodevelopment
This is an international multi-centre cohort study of first and second degree family members of individuals who carry Frontotemporal Dementia (FTD) mutations in MAPT, GRN or C9ORF72 repeat expansions for youths between the ages…
- Trial name
- GENFI-NeuroDev (NCT05779813)
- Where
- Canada
- Volunteers
- 200 people, healthy volunteers welcome
- Sponsor
- Western University
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