Polysomnographic Titration of Non-invasive Ventilation in Motor Neurone Disease
Motor Neuron Disease / Amyotrophic Lateral SclerosisA two-arm, individual participant randomised controlled, assessor-blinded trial in 7 MND care centres across Australia will be undertaken.
Regulatory T Cells for Amyotrophic Lateral Sclerosis
Amyotrophic Lateral SclerosisPhase 1 Safety Run-in Study of 6 patients followed by Phase 1b Randomized, Double Blind, Placebo Control Trial of CK0803, neurotropic, allogeneic, umbilical cord blood derived T regulatory (Treg) cells in additional 60 patients with Amyotrophic Lateral Sclerosis.
Volume Mode Non-invasive Ventilation in Amyotrophic Lateral Sclerosis
Amyotrophic Lateral SclerosisRespiratory Failure7 moreThe purpose of this study is to assess the efficacy of using intelligent volume assured pressure support (iVAPS-AE) versus spontaneous timed (ST) modes of non-invasive ventilation (NIV) in patients diagnosed with amyotrophic lateral sclerosis (ALS). The investigators believe that the use of iVAPS-AE mode NIV over a 90 day period will produce NIV compliance data and health-related quality of life (HRQOL) scores that are equivalent or no worse compared to ST mode NIV.
Neurologic Stem Cell Treatment Study
Neurologic DisordersNervous System Diseases24 moreThis is a human clinical study involving the isolation of autologous bone marrow derived stem cells (BMSC) and transfer to the vascular system and inferior 1/3 of the nasal passages in order to determine if such a treatment will provide improvement in neurologic function for patients with certain neurologic conditions. http://mdstemcells.com/nest/
Platform Trial to Assess the Efficacy of Multiple Drugs in Amyotrophic Lateral Sclerosis (ALS)
Amyotrophic Lateral SclerosisThe objective of this phase III, placebo-controlled platform study is to investigate the efficacy of drugs for patients with ALS (Amyotrophic lateral sclerosis).
A Study of BIIB067 (Tofersen) Initiated in Clinically Presymptomatic Adults With a Confirmed Superoxide...
Amyotrophic Lateral Sclerosis Associated With a SOD1 Gene MutationThe primary objective of this study is to evaluate the efficacy of tofersen in presymptomatic adult carriers of a superoxide dismutase 1 (SOD1) mutation with elevated neurofilament (NF). The secondary objectives of this study are to evaluate the safety and tolerability tofersen and to evaluate the effect of tofersen on pharmacodynamics (PD)/treatment response biomarkers when initiated prior to versus at the time of emergence of clinically manifest amyotrophic lateral sclerosis (ALS).
Non-invasive BCI-controlled Assistive Devices
Motor DisordersHealthy6 moreA brain-computer interface (BCI) decodes users' behavioral intentions or mental states directly from their brain activity, thus allowing operation of devices without requiring any overt motor action. One major modality for BCI control is based on motor imagery (MI), which is the mental rehearsal of the kinesthetics of a movement without actually performing it. MI-based BCIs translate motor intents into control commands for external devices. A major challenge in such BCIs is differentiating MI patterns corresponding to fine hand movements of the same limb from non-invasive EEG recordings with low spatial resolution since the cortical sources responsible for these movements are overlapping. In this study, the investigators hypothesize that neuromuscular electrical stimulation (NMES) applied contingent to the voluntary activation of the primary motor cortex through MI can help differentiate patterns of activity associated with different hand movements of the same limb by consistently recruiting the separate neural pathways associated with each of the movements within a closed-loop BCI setup. This is expected to be associated with neuroplastic changes at the cortical or corticospinal levels.
Neurodegenerative Alzheimer's Disease and Amyotrophic Lateral Sclerosis (NADALS) Basket Trial
Amyotrophic Lateral SclerosisAlzheimer Disease1 moreThis is an open-label, biomarker-driven basket trial of baricitinib in people with subjective cognitive disorder, mild cognitive impairment, Alzheimer's disease (AD), Amyotrophic lateral sclerosis (ALS), or asymptomatic carriers of an ALS-related gene, such as a hexanucleotide expansion in the C9ORF72 gene, with evidence of abnormal inflammatory signaling in cerebrospinal fluid (CSF) at baseline. Each participant will be treated with baricitinib for 24 weeks; no placebo will be given. Participants will receive baricitinib 2 mg per day by mouth for the first 8 weeks and baricitinib 4 mg per day by mouth for the remaining 16 weeks. This proof of concept trial will ascertain whether baricitinib at 2 mg per day, 4 mg per day, or both reaches therapeutic levels in the CSF and suppresses inflammatory biomarkers associated with type I interferon signaling among the study participants.
Transcranial Static Magnetic Field Stimulation (tSMS) in Amyotrophic Lateral Sclerosis (ALS).
Amyotrophic Lateral SclerosisThis study aims to evaluate safety and efficacy of tSMS in ALS patients and to obtain preliminary data about the effects of tSMS on cortical excitability. To this purpose, 40 ALS patients will be recruited and randomized to real or sham tSMS. After at least 3 months follow-up, they will undergo tSMS, daily for 120 min, at home, for 6 consecutive months. Clinical status will be tested before, during and after the stimulation period. Moreover, cortical excitability will be tested by transcranial magnetic stimulation (TMS) before and after the stimulation period.
A Multicenter, Open-label Extension Study to Investigate the Long-term Safety of FAB122 in Patients...
Amyotrophic Lateral SclerosisA multicenter, open-label extension study to investigate the long-term safety of FAB122 in patients with Amyotrophic Lateral Sclerosis