Clinical trials: Pick Disease of the Brain
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
Progressive Multifocal Leukoencephalopathy
Tysabri Observational Cohort Study - Multiple Sclerosis (MS) Registries
The primary purpose of this study is to estimate the incidence of progressive multifocal leukoencephalopathy (PML) among patients who switched to Tysabri from disease modifying therapies (DMTs), including newer DMTs (including…
- Trial name
- NCT03399981
- Testing
- Tysabri
- Where
- United States
- Volunteers
- 80,327 people
- Sponsor
- Biogen
Sphingomyelin Lipidosis
A Long-Term Study of Olipudase Alfa in Patients With Acid Sphingomyelinase Deficiency
The primary objective of this study was to obtain data regarding the safety of olipudase alfa in participants with acid sphingomyelinase deficiency (ASMD) who were exposed to long term treatment with olipudase alfa.
- Trial name
- NCT02004704
- Testing
- GZ402665
- Where
- 9 sites in United States, Belgium and 5 more
- Volunteers
- 25 people
- Sponsor
- Genzyme, a Sanofi Company
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.
Progressive Supranuclear Palsy Richardson Syndrome (PSP-RS)
A Study to Evaluate the Efficacy of NIO752 in Participants With Progressive Supranuclear Palsy
This Phase III study is intended to evaluate the efficacy and safety of NIO752 in participants with Progressive Supranuclear Palsy (PSP).
- Trial name
- NCT07498426
- Testing
- NIO752
- Where
- 64 sites in United States, Australia and 10 more
- Volunteers
- 300 people
- Sponsor
- Novartis Pharmaceuticals
Alzheimer Disease
A First-In-Human Study of LY3954068 in Participants With Early Symptomatic Alzheimer's Disease
The main purpose of this study is to evaluate the safety of LY3954068 in participants with early symptomatic Alzheimer's Disease (AD).
- Trial name
- NCT06297590
- Testing
- LY3954068, Flortaucipir F18
- Where
- 10 sites in United States, Japan and 1 more
- Volunteers
- 56 people
- Sponsor
- Eli Lilly and Company
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.
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.
Alzheimer Disease, Mild Cognitive Impairment
Longitudinal Cognitive Assessment by BoCA
The Boston Cognitive Assessment (BoCA) is a self-administered online test intended for longitudinal cognitive monitoring. BoCA uses random not-repeating tasks to minimize learning effects.
- Conditions
- Alzheimer Disease, Mild Cognitive Impairment, Vascular Dementia, Frontotemporal Dementia
- Trial name
- BoCA (NCT04114994)
- Testing
- Boston Cognitive Assessment (BoCA)
- Where
- United States
- Volunteers
- 10,000 people, healthy volunteers welcome
- Sponsor
- Alzheimer's Light LLC
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.
Seizures, Epilepsies, Partial
Efficacy and Safety of Diazepam in the Management of Refractory Epilepsy in Selected Patients Who Require Intermittent Medical Intervention for Acute Repetitive Seizures.
To evaluate the efficacy and safety of diazepam in the management of refractory epilepsy in selected patients who require intermittent medical intervention for the control of episodes of acute repetitive seizures.
- Conditions
- Seizures, Epilepsies, Partial, Epilepsy, Complex Partial, Epilepsy, Generalized
- Trial name
- NCT00319501
- Testing
- Vanquix Auto-Injector (Diazepam Injection)
- Where
- 86 sites in United States
- Volunteers
- 234 people
- Sponsor
- Pfizer
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 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
Acute Ischemic Stroke
NoNO-42 Trial in Acute Ischemic Stroke Patients Selected for Thrombolysis With or Without Endovascular Thrombectomy
ACT-42 is a domain of the ACT-GLOBAL platform (NCT06352632). This trial is a Phase 2b, multicenter, prospective, randomized, open label, blinded-endpoint (PROBE) controlled single-dose adaptive trial.
- Trial name
- NCT06403267
- Testing
- NoNO-42
- Where
- 9 sites in Canada
- Volunteers
- 600 people
- Sponsor
- NoNO Inc.
Glioblastoma Multiforme, Glioblastoma
Efficacy and Safety of NaviFUS System add-on Bevacizumab (BEV) in Recurrent GBM Patients
This is a prospective, single-arm, two stages, open-label, pilot study to investigate the efficacy and safety of FUS add-on bevacizumab (BEV) in rGBM patients.
- Conditions
- Glioblastoma Multiforme, Glioblastoma, Glioma, Brain Tumor
- Trial name
- NCT04446416
- Testing
- NaviFUS System, Bevacizumab
- Where
- Taiwan
- Volunteers
- 6 people
- Sponsor
- NaviFUS Corporation
Granulin Mutation
Study to Assess the Safety, Tolerability, and Pharmacodynamic (PD) Effects of FRM-0334 in Subjects With Prodromal to Moderate Frontotemporal Dementia With Granulin Mutation
The purposes of this study are to investigate the safety, tolerability, and pharmacodynamics of FRM-0334 in subjects with prodromal to moderate frontotemporal dementia with granulin mutation.
- Trial name
- NCT02149160
- Testing
- FRM-0334
- Where
- 13 sites in United States, Belgium and 4 more
- Volunteers
- 30 people
- Sponsor
- FORUM Pharmaceuticals Inc
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
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
GM2 Gangliosidosis, Niemann-Pick Type C Disease
A Study to Evaluate the Safety and Efficacy of Nizubaglustat (AZ-3102) in Patients With GM2 Gangliosidosis or Niemann-Pick Type C Disease
This open-label study aims to gather long-term safety, tolerability, PK, biomarker, and clinical efficacy data relating to daily administration of Nizubaglustat in participants previously enrolled in the Phase 2 RAINBOW study…
- Trial name
- PRISMA (NCT07399704)
- Testing
- AZ-3102
- Where
- 3 sites in Brazil
- Volunteers
- 21 people
- Sponsor
- Azafaros B.V.
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