Rio Pieoras, Puerto Rico
Technical and Clinical Validation of the WPM-SEMG Prototype
The study consists of two parts: - the first part concerns the technical validation of the prototype. It will enrol 10 healthy volunteers (Technical Validation Group). - the second part concerns the clinical validation of the prototype in comparison with the gold standard, that is the needle-EMG. It will enrol 50 healthy volunteers (Clinical Validation Group), including also the 10 subjects of the Technical Validation Group, and 20 patients Measurements will be done on 4 muscles with the WPM-SEMG device prototype using a rigid electrode matrix. For each muscle, the participant will execute some spontaneous motor activities and measurements will be taken on the muscle under tension (low, medium, and high) for a total of 12 measurements. Measurements will be repeated two times after a pause for a total of 24 acquisitions. For the group with myopathies an additional fifth muscle, not foreseen by the protocol, can be done if considered the clinical most affected muscle followingthe neurological examination. A control intervention is done on the same subjects and consists in a needle-EMG which will be applied as for routine clinical practice on the same muscles used for the WPM-SEMG device prototype. Measurements will be taken for each muscles as for the rigid matrix.
Phase
N/ASpan
432 weeksSponsor
Alain KaelinRecruiting
Healthy Volunteers
Evaluating Verbal Communication in Structured Interactions: Theoretical and Clinical Implications
PALS and age-matched adults will participate in one solo speech production task (clear speech) and three interactive tasks (structured communicative interaction, unstructured communicative interaction, and clear speech structured communicative interaction) in which they work with an unfamiliar, naive interlocutor. This study is designed to examine the differences in speech produced in the four tasks. Comparisons of speech produced by PALS and age-matched adults will clarify whether differences in speech observed across the four tasks are a function of the speech difficulties experiences by PALS. Plans for Assignment - This is a single group study in which all participants will engage in the same tasks. Delivery of Intervention: Using tablets and audio recording devices provided to them, participants will complete this task in the comfort of their home. Study protocols will be explained via videoconferencing by experimenters. Produced speech will be recorded using solid-state audio recorders as well as remotely through the video conferencing software. Adequacy of Sample size. Assuming medium effect sizes (Cohen's f = 0.3) based on our pilot data, for 80% power at an alpha of .05, the investigators will require 76 speakers. The investigators propose n = 100 PALS in order to account for speech variability that is common for PALS. The investigators plan to recruit 50 age-matched speakers. The investigators anticipate that this sample size will be sufficient to make appropriate comparison to the PALS group because there will be considerably less variability in these speakers. Adequacy of Analyses. The proposed statistical analyses (Generalized mixed effects regressions) are standard and will be used to analyze the effect of the intervention on the outcome measures described below. Severity of condition (for PALS) will be included in the analyses and by-subject slopes and intercepts will be used to account for variability across participants.
Phase
N/ASpan
230 weeksSponsor
Penn State UniversityRecruiting
Healthy Volunteers
Efficiency of Spatially Distributed Sequential Stimulation (Sdss) for Functional Electrical Stimulation (FES) of Upper Motor Neuron Syndrome (UMR) Patients
Design: SCED (single case experimental design) prospective, monocentric, comparative interventional study (SDSS versus SES) Population: patients with motor deficit due to upper motor neuron syndrome Setting: Neurologic Rehabilitation Unit Interventions: 3 FES cycling sessions separated by 48h of rest. Each session is comprised of 2 phases separated by 20 minutes of rest. Each phase is comprised of a 3 minutes passive cycling warm-up, followed by 3 minutes of electrically stimulated cycling. Participants will be evaluated before and during the training.
Phase
N/ASpan
33 weeksSponsor
UGECAM Rhône-AlpesRecruiting
An Open Label Extension Study of Monepantel in Individuals With Motor Neurone Disease
ALS/MND is a progressive, fatal neurodegenerative disease; characterized by motor neuron loss resulting in muscle weakness and atrophy, disability, and eventually death from failure of the ventilatory muscles. The median age of onset is 55 years and average survival is 3-5 years after onset of the first symptoms. The only FDA-approved disease modifying medications confer only a modest survival benefit. Given the poor prognosis and dearth of effective treatments, clinical studies are of primary importance for people with ALS/MND. Abnormal protein accumulation within motor neurons of the brain associates with the cause of ALS/MND. Inhibition of the mTOR signaling pathway slows disease progression in certain preclinical models of ALS/MND and is suggested to provide synergy with the ALS/MND standard-of-care drug, riluzole. PharmAust has shown that MPL and its major metabolite MPL sulfone (MPLS) have activity against mTOR signaling pathways in humans; based on published data, the inhibition of mTOR may be relevant to the treatment of ALS/MND. This Phase I Open Label Extension will further test the hypotheses that MPL administration to individuals living with ALS/MND will safely reduce disease associated protein accumulation in motor neurons and provide therapeutic benefit. The safety and tolerability of oral monepantel administration and markers of efficacy will continue to be tested in the same participants that completed the Phase I Study (MON-2021-001). A daily dose of 10 mg/kg monepantel (QD) will be studied in the Open Label Extension Study (MON-2023-001) to further evaluate long-term safety and efficacy in participants with MND/ALS that completed the Phase I Study (MON-2021-001). Based on the previous pre-clinical efficacy data and clinical safety data, a dose of 10 mg/kg QD is estimated to produce the most robust mTOR inhibition while still being well tolerated. The 10 mg/kg QD dose was the maximum dose evaluated in the Phase 1 Study.
Phase
1Span
59 weeksSponsor
Neurizon Therapeutics LimitedRecruiting
Creation of a Clinical Database for the Study of Phenotypic Variability in Motor Neuron Diseases
Phase
N/ASpan
196 weeksSponsor
Istituto Auxologico ItalianoRecruiting
Open Label Extension of TUDCA-ALS Study
The TUDCA-ALS Open label extension study is designed to investigate long term safety, tolerability and efficacy of tauroursodeoxycholic acid in patients with ALS who completed the TUDCA-ALS study
Phase
3Span
179 weeksSponsor
Humanitas Mirasole SpARecruiting
Longitudinal Assessment of Autonomic and Sensory Nervous System in ALS
Phase
N/ASpan
190 weeksSponsor
Istituti Clinici Scientifici Maugeri SpARecruiting
Healthy Volunteers
A Mindful Community for People With ALS and Their Primary Caregivers
Phase
N/ASpan
42 weeksSponsor
Harvard UniversityRecruiting
Satisfaction of Patients With Amyotrophic Lateral Sclerosis Regarding Home Assisted Teleconsultation
Amyotrophic Lateral Sclerosis (ALS) is a progressive and rapidly progressing neurodegenerative disease. Depending on the degree of muscle damage, patients may suffer from reduced mobility, difficulty swallowing, speech difficulties and finally respiratory weakness. Death occurs within an average of 2-3 years and is usually due to respiratory failure. Currently there is no cure for ALS, so palliative care and symptomatic treatments are essential for the management of these patients. The gold standard treatment for respiratory impairment is non-invasive ventilation (NIV). The aim of this mask ventilation is to improve gas exchange by compensating for the weakness of the respiratory muscles. The progressive nature of ALS requires regular assessment of the patient's clinical condition and evaluation of NIV parameters. Regular medical appointments are therefore essential to ensure optimal ventilation and close surveillance of the patient. A day hospitalization or a consultation is organized every 3 or 4 months. These visits can cause significant fatigue, not only because of the difficulty patients have to move around but also because of the time spent waiting in hospital. ALS is characterised by its severity and it requires our society to think about and implement new ways of managing the disease. Thus, e-health innovations could be an interesting potential in the remote follow-up of these patients, to reduce the burden of hospital consultations. The hypothesis is that the patient will be more satisfied with a home assisted teleconsultation follow-up, while keeping the same quality of non-invasive nocturnal ventilatory assistance.
Phase
N/ASpan
103 weeksSponsor
AGIR à DomRecruiting
Clinical Study to Evaluate the Efficacy and Safety of FB1006 in the Treatment of ALS Patients
This is a randomized double-blind controlled exploratory clinical study to evaluate the efficacy and safety of FB1006 in the treatment of amyotrophic lateral sclerosis (ALS) patients. The study has two phases: the first phase is 24 weeks, using a randomized double-blind placebo-controlled design; the second phase is 24 weeks, using an open-label study design. FB1006 was approved by the National Medical Products Administration(NMPA)on June 21, 2021.
Phase
4Span
246 weeksSponsor
Peking University Third HospitalRecruiting