Clinical trials: Primary Progressive Aphasia (PPA)
Lysosomal Acid Lipase Deficiency, Wolman Disease
Safety, Tolerability, Efficacy, Pharmacokinetics, and Pharmacodynamics of Sebelipase Alfa in Children With Growth Failure Due to Lysosomal Acid Lipase Deficiency
This was an open-label, repeat-dose, intra-participant dose-escalation study of SBC-102 (sebelipase alfa) in children with growth failure due to lysosomal acid lipase (LAL) Deficiency.
- Trial name
- NCT01371825
- Testing
- Sebelipase alfa (SBC-102)
- Where
- 7 sites in United States, Egypt and 3 more
- Volunteers
- 9 people
- Sponsor
- Alexion Pharmaceuticals, Inc.
Niemann-Pick Disease, Type C
Open-label Study of VTS-270 in Participants With Neurologic Manifestations of Niemann-Pick Type C1
This is a multicenter, multinational, open-label study of VTS-270 to evaluate the long-term safety and tolerability of VTS-270 (2-hydroxypropyl-β-cyclodextrin) in participants transitioning from Study VTS301 (Parts A/B…
- Trial name
- NCT03879655
- Testing
- VTS-270
- Where
- Costa Rica
- Volunteers
- 2 people
- Sponsor
- Mandos LLC
Apraxia Speech
Transcranial Magnetic Stimulation (TMS) for Primary Progressive Apraxia of Speech (PPAOS)
The purpose of this study is to assess the influence of transcranial magnetic stimulation (TMS) on speech performance in individuals with primary progressive apraxia of speech.
- Trial name
- NCT03028324
- Testing
- Transcranial Magnetic Stimulation (TMS)
- Where
- United States
- Volunteers
- 4 people
- Sponsor
- University of Miami
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.
Neimann-Pick Disease
Phase 1/2 Study of Vorinostat Therapy in Niemann-Pick Disease, Type C1
Niemann-Pick disease type C (NPC) is a lethal, autosomal recessive, lysosomal storage disorder characterized by neurodegeneration in early childhood and death in adolescence.
- Trial name
- NCT02124083
- Testing
- Vorinostat
- Where
- United States
- Volunteers
- 12 people
- Sponsor
- Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
Frontotemporal Dementia
A Phase 2 Study to Evaluate Safety of Long-term AL001 Dosing in Frontotemporal Dementia (FTD) Patients (INFRONT-2)
A Phase 2 open label study evaluating the safety, tolerability, pharmacokinetics (PK) and pharmacodynamics (PD) of AL001 in participants with a Granulin mutation or C9orf72 mutation causative of frontotemporal dementia.
- Trial name
- NCT03987295
- Testing
- AL001
- Where
- 12 sites in United States, Canada and 4 more
- Volunteers
- 33 people
- Sponsor
- Alector Inc.
Frontotemporal Lobar Degeneration
Effects of Tolcapone on Frontotemporal Dementia
This study will test the effects of a medication called tolcapone on cognitive, behavioral, and language problems seen in patients with frontotemporal dementia (FTD).
- Trial name
- NCT00604591
- Testing
- Tolcapone
- Where
- United States
- Volunteers
- 28 people
- Sponsor
- Columbia University
Frontal Lobe Dementia, Frontotemporal Lobe Dementia
Memantine (10mg BID) for the Frontal and Temporal Subtypes of Frontotemporal Dementia
The primary objective of the study is to determine whether memantine is effective in slowing the rate of behavioral decline in frontotemporal dementia.
- Conditions
- Frontal Lobe Dementia, Frontotemporal Lobe Dementia, Semantic Dementia
- Trial name
- NCT00545974
- Testing
- memantine
- Where
- 9 sites in United States
- Volunteers
- 81 people
- Sponsor
- University of California, San Francisco
Frontotemporal Dementia, Behavioral Variant
Intranasal Insulin in Frontotemporal Dementia (FTD)
This project will study intranasal (IN) insulin in Frontotemporal dementia (FTD) in 12 patients. Study Investigators aim to evaluate the feasibility of the EXAMINER cognitive battery as a cognitive outcome measure in FTD, the…
- Trial name
- NCT04115384
- Testing
- Novolin-R insulin
- Where
- United States
- Volunteers
- 3 people, healthy volunteers welcome
- Sponsor
- HealthPartners Institute
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)
Mild Cognitive Impairment, Primary Progressive Aphasia
Home-based tDCS in Frontotemporal Dementia or Alzheimer's Disease
The primary objective of this research is to evaluate the effects of non-invasive brain stimulation and computerized cognitive training on executive functioning in individuals with Primary Progressive Aphasia (PPA), mild…
- Conditions
- Mild Cognitive Impairment, Primary Progressive Aphasia, Dementia
- Trial name
- NCT05978804
- Testing
- Computerized Cognitive Training, Active tDCS (tDCS) on DLPFC
- Where
- United States
- Volunteers
- 5 people
- Sponsor
- Johns Hopkins University
Dementia, Cognitive Dysfunction
A Relational Research Recruitment and Engagement Intervention for Cognitive Aging Research
Despite well-documented disparities in Alzheimer's disease and related dementia (AD) prevalence, incidence, treatment, and mortality, individuals from disadvantaged backgrounds (e.g.
- Conditions
- Dementia, Cognitive Dysfunction, Alzheimer Disease, Lewy Body Disease
- Trial name
- NCT05444244
- Testing
- Relational Research Recruitment and Engagement Intervention
- Where
- United States
- Volunteers
- 182 people, healthy volunteers welcome
- Sponsor
- University of Wisconsin, Madison
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
Primary Progressive Aphasia
Remotely Supervised Transcranial Direct Current Stimulation (tDCS) for Primary Progressive Aphasia (PPA)
The purpose of this study is to establish the feasibility of a program of remotely supervised transcranial direct current stimulation (RS-tDCS) paired with language skills practice for people living with the semantic or logopenic…
- Trial name
- NCT05615922
- Testing
- Transcranial Direct Current Stimulation (tDCS), Word-Naming Activity
- Where
- United States
- Volunteers
- 11 people
- Sponsor
- NYU Langone Health
Primary Progressive Aphasia, MCI
tDCS Intervention in Primary Progressive Aphasia
Primary progressive aphasia (PPA) is a neurodegenerative disease that affects first and foremost language abilities. Mild cognitive impairment (MCI) is slowly progressive decline in a single domain of cognition (e.g.
- Conditions
- Primary Progressive Aphasia, MCI, FTD
- Trial name
- NCT02606422
- Testing
- Active HD-tDCS plus Speech-Language Therapy
- Where
- United States
- Volunteers
- 61 people, healthy volunteers welcome
- Sponsor
- Johns Hopkins University
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
Alzheimer Disease
A Study of LY3372689 to Assess the Safety, Tolerability, and Efficacy in Participants With Alzheimer's Disease
The purpose of this study is to assess the safety, tolerability and effect of study drug LY3372689 in participants with early symptomatic Alzheimer's Disease
- Trial name
- NCT05063539
- Testing
- LY3372689
- Where
- 69 sites in United States, Australia and 3 more
- Volunteers
- 327 people
- Sponsor
- Eli Lilly and Company
Dementia-related Psychosis
Relapse Prevention Study of Pimavanserin in Dementia-related Psychosis
The purpose of this study is to evaluate the efficacy of pimavanserin compared to placebo in preventing relapse of psychotic symptoms in subjects with dementia-related psychosis who responded to 12 weeks of open label…
- Trial name
- NCT03325556
- Testing
- Pimavanserin 34 mg, Pimavanserin 20 mg
- Where
- 83 sites in United States, Bulgaria and 11 more
- Volunteers
- 392 people
- Sponsor
- ACADIA Pharmaceuticals Inc.
Chronic Traumatic Encephalopathy
Tau Imaging of Chronic Traumatic Encephalopathy
Chronic traumatic encephalopathy (CTE) is a progressive degenerative brain disease with symptoms that include memory loss, problems with impulse control, and depression that can lead to suicide.
- Trial name
- NCT02191267
- Testing
- [F18]-T807, [F18]-Florbetapir, MRI/MRS
- Where
- 2 sites in United States
- Volunteers
- 30 people
- Sponsor
- Brigham and Women's Hospital
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