Active clinical trials for "Motor Neuron Disease"

This is a multi-center, 20-week study of inosine treatment. Study Objectives and Endpoints The primary objective of the study is to determine the safety and tolerability of oral administration of inosine (administered daily) dosed to moderately elevate serum urate over 20 weeks. The primary outcome measures will be Safety, as measured by adverse events Tolerability, defined as the ability of subjects to complete the entire 20-week study. As an exploratory objective, we will test the feasibility and utility of a smartphone application for monitoring symptoms and disease progression in patients with amyotrophic lateral sclerosis (ALS).

This is a pilot, phase 2, prospective, open-label, single-arm study to evaluate disease progression, forced vital capacity, and the safety and tolerability of plasma exchange (PE) using Albutein® 5% in participants with amyotrophic lateral sclerosis (ALS).

This is a phase 2a open label, multicentre design study to investigate the safety of Triumeq in patients with ALS at 24 weeks post treatment. In this phase 2a study the investigators aim to determine whether a combination of anti-retroviral therapy, Triumeq (dolutegravir 50mg, abacavir 600mg, lamivudine 300mg) is tolerated and safe in patients with ALS. As secondary outcomes, ALSFRS-R, ALSQOL, physical examination, neurophysical parameters and respiratory and muscle function will be evaluated. Blood and urine samples will be stored for possible future analysis for viral activity. Subjects will be screened for the study after signing an approved Informed consent document.

The purpose of this study is to evaluate the safety of HLA-haplo matched Allogenic Bone Marrow Derived stem cells("HYNR-CS-Allo inj"), through intrathecal delivery for the treatment in patients with amyotrophic lateral sclerosis(ALS). This study is an open label, dose up and down study using the 3+3 design to assess the safety of HLA-haplo matched Allogenic Bone Marrow Derived stem cells("HYNR-CS-Allo inj")

ALS is a debilitating disease with varied etiology characterized by rapidly progressive weakness, muscle atrophy and fasciculations, muscle spasticity, difficulty speaking (dysarthria), difficulty swallowing (dysphagia), and difficulty breathing (dyspnea). ALS is the most common of the five motor neuron diseases.Riluzole (Rilutek) is the only treatment that has been found to improve survival but only to a modest extent. It lengthens survival by several months, and may have a greater survival benefit for those with a bulbar onset. It also extends the time before a person needs ventilation support.Stem cell transplantation is a new hopeful way to improve the patients conditions and reduce the period of disabilities.

In patients with amyotrophic lateral sclerosis (ALS), non-invasive mechanical ventilation (NIV) is usually initiated in an in-hospital regime. The investigators evaluated if NIV initiated in an outpatient setting can be as effective as regards patients' adherence. The investigators also evaluated factors predicting NIV adherence and disease progression.

GM604 is an endogenous human embryonic stage neural regulatory and signaling peptide that controls the development, monitoring and correction of the human nervous system. Neurological diseases are multisystem, multifactorial, and single target drugs are ineffective. Genervon's Master Regulators play a significant role in embryonic/fetal nervous system development and are potent disease modification drug candidates modulating many pathways including inflammation, apoptotic, and hypoxia. The study drug is an regulatory peptide with a sequence identical to one of the active sites of human Motoneuronotrophic Factor and is manufactured by solid phase synthesis. Pre-clinical research indicates it to be a neuro-protective agent in animal models of ALS, motorneuron diseases, PD, other neuro-degenerative diseases and stroke. GM604 controls and modulates over many known and significant ALS genes with positive effects interactively and dynamically through multiple pathways, and up to twenty-two biological processes, including neuro-protection, neurogenesis, neural development, neuronal signaling, neural transport, and other processes. GM6 is not a cocktail of drugs, but one master regulator peptide drug that functions through multiple pathways. Genervon hypothesized that studying the biomarkers of protein expressions of these ALS genes such as superoxide dismutase 1 (SOD1) and the protein expression of substances such as tau, neurofilament - heavy (NF-H), Cystatin C which were indications of degeneration of neuron in the CSF collected from ALS patients will provide information of the possible GM604's mechanisms of action in treating ALS. 1. This pilot trial is designed to test proof of principle, i.e. determine if a 2-week IV bolus treatment with this agent can (1) change ALS protein expression (target biomarkers and efficacy biomarkers) after treatment (2) have preliminary effects measures of ALS disease clinical progression. Study Objectives are: To test the safety and tolerability of GM604 in a population of ALS patients. To test for changes in ALS biomarkers before and after treatment. To determine preliminary effects of injections of GM604 on measures of ALS disease biomarkers and clinical progression

Autologous cell therapy in patients with ALS can stimulate neuroplasticity, modifying the neurodegenerative process and stops the clinical progression of disease.

In the double-blind, cross-over part of the study, ODM-109 capsules and placebo capsules for ODM-109 will be administered for 2 weeks separated by a 19-23 days wash-out period. During each treatment period of the double-blind cross-over part, there will be a baseline visit (day 1) and 2 visits (5 ± 2 and 14 ± 2 days) after the start of study treatment. After completing the 3rd treatment period, the subjects will continue in the open-label follow-up part for 6 months. During the open-label follow-up, visits will be at 1, 3 and 6 months. An end-of-study visit will take place 14-25 days after the last study treatment administration for each subject. The study duration will be about 13-14 weeks for the double-blind cross-over part, and about 9-10 months for the entire study including the 6 months open-label follow-up. The number of randomised study subjects is planned to be approximately 54 in cross-over comparison. The maximum number of subjects will not exceed 70. Primary objective is to investigate the efficacy of oral ODM-109 on respiratory function in patients with amyotrophic lateral sclerosis (ALS).

This open label trial is conducted to investigate the efficacy and safety of the combination therapy of allogeneic umbilical cord blood (UCB) and granulocyte-colony stimulating factor (G-CSF) for patients with brain injury or neurodegenerative disorders.