Clinical trials: Primary Progressive Aphasia (PPA)
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
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
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
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
Mild Alzheimer's Disease
Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of IONIS-MAPTRx in Patients With Mild Alzheimer's Disease
The purpose of this study was to assess the safety, tolerability, pharmacokinetics, and pharmacodynamics of IONIS-MAPTRx in patients with Mild Alzheimer's Disease.
- Trial name
- NCT03186989
- Testing
- IONIS MAPTRx
- Where
- 13 sites in Canada, Finland and 4 more
- Volunteers
- 46 people
- Sponsor
- Ionis Pharmaceuticals, Inc.
Frontotemporal Dementia
A Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of DNL593 in Healthy Participants and Participants With Frontotemporal Dementia (FTD-GRN)
This is a Phase 1/2, multicenter, randomized, placebo-controlled, double-blind study to evaluate the safety, tolerability, pharmacokinetics (PK), and pharmacodynamics (PD) of single and multiple doses of DNL593 in two parts…
- Trial name
- NCT05262023
- Testing
- DNL593
- Where
- 26 sites in United States, Belgium and 10 more
- Volunteers
- 85 people, healthy volunteers welcome
- Sponsor
- Denali Therapeutics Inc.
Alzheimer's Disease
Transcranial Electromagnetic Treatment (TEMT) Against Alzheimer's Disease
This is a second extension of EM 1000-1 wherein mild/moderate AD subjects who participated in the original study have completed participation in a first extension of 4-months.
- Trial name
- NCT04271163
- Testing
- MemorEM
- Where
- United States
- Volunteers
- 6 people
- Sponsor
- NeuroEM Therapeutics, Inc.
Frontotemporal Dementia (FTD), Amyotrophic Lateral Sclerosis (ALS)
[18F]ACI-19626 PET in TDP-43 Proteinopathies
The goal of this clinical trial is to test whether we can reliably and safely measure the accumulation of pathological protein TDP-43 [involved in rare forms of dementia such as frontotemporal dementia (FTD) and in amyotrophic…
- Conditions
- Frontotemporal Dementia (FTD), Amyotrophic Lateral Sclerosis (ALS), TDP-43 Proteinopathies, Suspected Limbic Predominant Age-related TDP-43 Encephalopathy (LATE)
- Trial name
- NCT06891716
- Testing
- [18F]ACI-19626
- Where
- Netherlands
- Volunteers
- 45 people, healthy volunteers welcome
- Sponsor
- AC Immune SA
Alzheimer's Disease, Mild Cognitive Impairment
Performance of AclarusDx™, a Blood-Based Transcriptomic Test for AD, in US Patients Newly Referred to a Memory Center
The purpose of this study is to assess the performance of AclarusDx™, an investigational blood test detecting gene expression information, and intended to help physicians in making an Alzheimer's Disease diagnosis in patients…
- Conditions
- Alzheimer's Disease, Mild Cognitive Impairment, Vascular Dementia, Fronto-temporal Dementia
- Trial name
- NCT01465360
- Where
- 3 sites in United States
- Volunteers
- 160 people
- Sponsor
- Exonhit
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
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 Lobar Degeneration (FTLD), Frontotemporal Dementia (FTD)
A Wearable Sensor Platform for Remote Monitoring of Individuals on the Frontotemporal Dementia Spectrum
The primary objective of this clinical study is to provide the initial validation for monitoring biomarkers of symptoms and functioning for individuals with frontotemporal lobar degeneration (FTLD) syndromes.
- Conditions
- Frontotemporal Lobar Degeneration (FTLD), Frontotemporal Dementia (FTD), Corticobasal Degeneration, Corticobasal Syndrome (CBS)
- Trial name
- ReMoTe (NCT07569367)
- Where
- 2 sites in United States
- Volunteers
- 60 people
- Sponsor
- BioSensics
Dementia, Frontotemporal Dementia
Study in Asymptomatic GRN-FTD Patients to Investigate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of VES001
This is an open-label, multiple dosing study in asymptomatic GRN-FTD carriers to investigate the safety, tolerability, PK, and PD of VES001.
- Conditions
- Dementia, Frontotemporal Dementia, Asymptomatic Condition
- Trial name
- SORT-IN-2 (NCT06705192)
- Testing
- VES001
- Where
- 2 sites in Netherlands, United Kingdom
- Volunteers
- 6 people
- Sponsor
- Mads Kjolby
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
Hurler Syndrome (MPS I), Hurler-Scheie Syndrome
Phase I/II Pilot Study of Mixed Chimerism to Treat Inherited Metabolic Disorders
The goal of this research study is to establish chimerism and avoid graft-versus-host-disease (GVHD) in patients with inherited metabolic disorders.
- Conditions
- Hurler Syndrome (MPS I), Hurler-Scheie Syndrome, Hunter Syndrome (MPS II), Sanfilippo Syndrome (MPS III)
- Trial name
- NCT01372228
- Testing
- hematopoietic stem cell infusion
- Where
- United States
- Volunteers
- 3 people
- Sponsor
- Talaris Therapeutics Inc.
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
Niemann-Pick Disease, Type C1
Hydroxypropyl Beta Cyclodextrin for Niemann-Pick Type C1 Disease
Background: Hydroxypropyl beta cyclodextrin (HPBCD) is being tested for a disease called Niemann-Pick disease type C1 (NPC1). NPC1 is a genetic disorder that results in gradual loss of nervous system function.
- Trial name
- NCT01747135
- Testing
- VTS-270
- Where
- United States
- Volunteers
- 14 people
- Sponsor
- Vtesse, LLC, a Mallinckrodt Pharmaceuticals Company
Frontotemporal Lobar Degeneration (FTLD), Frontotemporal Dementia (FTD)
Digital Measurements of Motor and Voice Functions in FTD
The primary objective of this clinical study is to provide the initial validation for monitoring biomarkers of symptoms and functioning for individuals with FTLD syndromes.
- Conditions
- Frontotemporal Lobar Degeneration (FTLD), Frontotemporal Dementia (FTD), Corticobasal Syndrome, Corticobasal Syndrome(CBS)
- Trial name
- NCT07333898
- Volunteers
- 20 people
- Sponsor
- BioSensics
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
Sphingomyelin Lipidosis
A Prospective and Retrospective Cohort Study in Patients With Chronic Forms of Acid Sphingomyelinase Deficiency (ASMD)
Primary Objective: To describe the clinical features and their severity at the time of diagnosis and their evolution over time in patients with confirmed chronic visceral and chronic neurovisceral forms of ASMD To describe…
- Trial name
- NCT04106544
- Testing
- Investigational Procedures
- Where
- 28 sites in United States, Argentina and 11 more
- Volunteers
- 84 people
- Sponsor
- Sanofi