Found 6,914 clinical trials
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). The objective of this study is to evaluate the safety, tolerability and efficacy of N-acetyl-L-leucine (IB1001) compared to standard of care.Following this randomized, double-blind, …
Safety and Tolerability Study of CTx1000 In Participants With Amyotrophic Lateral Sclerosis
This clinical study is in participants with Amyotrophic Lateral Sclerosis and is designed to evaluate the safety and tolerability of the gene therapy CTx1000.
A Dual Orexin Receptor Antagonist to Reduce Biomarkers of Neurodegeneration in Adults With Insomnia.
The goal of this clinical trial is to explore the potential neuroprotective benefits of a dual orexin receptor antagonist (DORA) in adults with insomnia. The main questions it aims to answer are:Does the DORA reduce blood-based phosphorylated TAU181, in adults with insomnia, when compared to placeboDoes the DORA reduce other …
Effects of Transcranial Direct Current Stimulation (tDCS) on Inhibition and Brain Function in Primary Progressive Aphasia
Primary progressive aphasia (PPA) is a focal dementia characterized by primary impairment of language abilities and functional disturbances associated with language.Although PPA is a progressive disorder, new techniques are being proposed to try to activate parts of the brain previously thought to be potentially inactive, due to the possibility of …
Augmented Reality BCI Longitudinal Study for Persons With Late Stage ALS
The goal of this study is refine the usability of a BCI based communication platform.The study will take place in the greater Los Angeles area and will enroll up to 10 participants with late stage ALS. Each subject will receive a Cognixion Axon-R augmented reality brain computer interface and associated …
The Importance of Positive Expiratory Pressure Associated With the In-exsufflator in ALS Patients
Amyotrophic Lateral Sclerosis (ALS) is a fatal neurodegenerative disorder that impairs motor neurons, with a life expectancy of 2 to 7 years after diagnosis. ALS manifests as 'spinal' when it primarily affects limbs, or 'bulbar' when it impairs speech and swallowing. The disease progressively weakens all skeletal muscles, causing respiratory …
A Study of the Behavioral Variant of Frontotemporal Dementia and Bipolar Disorder: a Neuroimaging and Epigenetics Integrated Approach
The aim of this study is to investigate the selective epigenetic modifications and their effect on brain's morphology and functionality in the frontotemporal dementia behavioral variant and bipolar disorder. The open-label, multicentric, interventional case-control study involves the analysis of 3 separate cohorts of patients, partly selected over the course of …
Safety and Efficacy Evaluation of GC101 Gene Therapy Via Intrathecal (IT) Injectionin the Treatment of Patients With Type 2 Spinal Muscular Atrophy (SMA) - Phase III
This trial employs a multicenter, randomized, open-label, standard-of-care-controlled design and plans to enroll 50 patients with Type 2 SMA aged 2 to 12 years who have previously received nusinersen. The primary objective of the trial is to evaluate the efficacy of GC101 in treating Type 2 SMA. The secondary objectives …
Wideband Tympanometry as a Non-behavioral Test of the Speech-weighted Middle Ear Transfer Function.
There is a need for a non-behavioral clinical test that provides an assessment of how speech is conducted through the middle ear for the optimization of middle ear implants. These implants utilize the functionality of the human middle ear and require adequate speech conduction through the middle ear chain. Consequently, …
Evaluation of Safety and Efficacy of Gene Therapy Drug in the Treatment of Spinal Muscular Atrophy (SMA) Type 3 Patients
The study will evaluate safety and efficacy of intrathecal delivery of GC101 gene therapy drug as a treatment of spinal muscular atrophy Type 3 (SMA 3) patients.
