Active clinical trials for "Motor Neuron Disease"

The involvement of Lymphocyte type B in Amyotrophic lateral sclerosis (ALS) patients will be compared to lymphocyte in healthy subjects.

The general purpose of this observational study is to examine biomarkers associated with the pathology of neurodegenerative diseases to potentially develop novel therapeutic approaches.

R(+) pramipexole dihydrochloride monohydrate [R(+)PPX], an experimental neuroprotective drug, is provided in this open label extension study to ALS patients who have participated in earlier clinical protocols.

This expanded access protocol is to provide access to the investigational product, SLS-005, to participants with ALS who are not eligible to participate in clinical trials.

This EAP will provide access to pridopidine for up to 200 patients with ALS who are ineligible for clinical trials. Pridopidine will be given at a dose of 45 mg twice daily p.o. (or via feeding tube). Each patient will be followed for 2 years with regularly scheduled visits. The screening and baseline visits will be performed in person; subsequent visits may occur in person or remotely. Recommended in-person visits will occur at Weeks 4, 12, 28, 52, 78, and end of treatment (Week 104 or early termination). If the patient is unable to complete the visits in person, these visits may also be completed remotely.

This is an open label expanded access protocol for the treatment of up to approximately 250 adult patients with amyotrophic lateral sclerosis (ALS) who have difficulty swallowing oral riluzole tablets and may be able to derive benefit from treatment with an alternative oral formulation of riluzole.

The objective of this early access program (EAP) is to provide access to tofersen to eligible participants with amyotrophic lateral sclerosis (ALS) associated with a mutation in the superoxide dismutase 1 (SOD1) gene prior to an alternative access mechanism in order to address a high unmet medical need in this population.

The specific aim of this study is to investigate rod, cone and melanopsin driven pupillary light response in individuals with progressive supranuclear palsy (PSP), age-matched healthy controls and individuals with other neurodegenerative diseases using chromatic pupillometry, with special interest in assessing melanopsin-driven post-illumination pupil response (PIPR) as an identifier for PSP. The study addresses the following hypotheses: Chromatic pupil responses, including rod/cone-driven rapid phase constriction and melanopsin-driven PIPR, are reduced in subjects with PSP compared to age-matched normal healthy control subjects, Pupil parameters of the melanopsin-driven PIPR are abnormal in PSP subjects without supranuclear palsy, which is indicative of a subclinical physiological deficit of the OPN in the early stages of PSP. If these hypotheses are upheld, chromatic pupillometry to measure the PIPR promises to be a reliable in vivo, non-invasive, convenient and inexpensive technique to detect asymptomatic pupillomotor impairment in advance of diagnostic oculomotor signs and deterioration of cognitive function.

The study team propose that a new, hand-held test device may be valuable in the management of breathing failure in patients with Motor Neurone Disease (MND). The study team need to validate this device against the current gold standard of blood gas analysis and determine whether people with MND can use it at home. The new device, called 'N-Tidal C™' measures the carbon dioxide (CO2) in expired breath. At the end of the breath (end tidal) the CO2 level gives an indication of the CO2 in the person's arterial blood. Ventilatory failure is diagnosed at present using the value of CO2 in the arterial blood, but usually this can only be measured in specialist clinics. The study will determine if the end tidal CO2 measured by the new device agrees with CO2 measured on a blood test in clinic and also whether or not the device is practical for home use. The team will analyse the output of the device during home monitoring to see if changes in the pattern of CO2 in the expired breath identify, or even predict, the development of breathing failure in the community. With the results of these measures and detailed information about the patients in Papworth's clinic, recruited to this study, collected over a year the team will design a follow on study to see if using the new device at home can improve survival and quality of life for people with MND.

The research is aimed to asses the validity of the communication system improving the quality of life and the degree of sanctification of clinical patients and their caregiver.