A Registry-Based Clinical Trial of Pimozide in Patients With Neuromuscular Junction Transmission...
Amyotrophic Lateral Sclerosis (ALS)Amyotrophic lateral sclerosis (ALS) is a neuromuscular disease that results in rapid decline in normal muscle function and tone leading to difficulties with mobility, eating, drinking, breathing, sleeping, and communicating. The disease is progressive and no cure currently exists. Most people diagnosed with ALS succumb within 3 to 5 years. The only approved treatment to slow the progression of ALS is called Rilutek® (riluzole) which has only a modest effect and has been shown to increase survival by a few months. Muscular dysfunction present in people with ALS is caused by nerve breakdown and a dysfunction in the communication between the muscles and the nerves. The area where these communications occur is called the neuromuscular junction. Some recent studies have focused on using different medications to enhance communication at the neuromuscular junction with the goal of improving muscle function as a result. This approach is unproven but may help to slow the progression of the disease. Pimozide is a medication that has been demonstrated to enhance communication at the neuromuscular junction in fish and mice. This study will look at whether Pimozide may help to slow the progression of ALS and how much medication needs to be taken to have an effect.
Biological Treatment of Amyotrophic Lateral Sclerosis
Amyotrophic Lateral SclerosisThe purpose of this study is to test the safety and effectiveness of an autologous bone marrow-derived stem/progenitor cells infusion in the subjects with diagnosed amyotrophic lateral sclerosis.
Autologous Bone Marrow-Derived Stem Cell Therapy for Motor Neuron Disease
Motor Neuron DiseaseAmyotrophic Lateral Sclerosis3 moreHerein, the investigators study the safety and efficacy of transplanting purified autologous bone marrow-derived stem cells transplanted via the intrathecal route by interventional radiology and the intravenous route.
ALS Study Determining Various Biomarkers and Strength Comparison After Exercise
Amyotrophic Lateral SclerosisThe purpose of this study is to determine the muscle strength of a muscle in the thigh after 12 weeks of home exercise.
Nocturnal PtcCO2 Monitoring in Patients With Amyotrophic Lateral Sclerosis (ALS)
Amyotrophic Lateral SclerosisAmyotrophic lateral sclerosis (ALS) is a neurodegenerative disease affecting motoneurons, with a prevalence around 5/100.000. Respiratory muscle involvement is a major feature in ALS and remains the main prognostic factor. Timing and rate of progression of this respiratory muscle involvement is also highly variable among individuals. Respiratory manifestations justify a careful follow up including clinical evaluation, pulmonary function tests and blood gases. Prognostic value of respiratory muscle assessment has been clearly demonstrated in ALS, although several cut off values have been published. The clinical benefit of non invasive ventilation (NIV) is well established in ALS, but the optimal criteria for its initiation remain debated . The 1999 consensus for NIV selected classical criteria to consider NIV in patients with respiratory symptoms suggesting hypoventilation: daytime hypercapnia (PaCO2 > 45 mmHg), nocturnal SaO2 < 89 % more than 5 consecutive minutes and for progressive neuromuscular disorders (NMD) (mainly ALS), a vital capacity (VC) < 50 % pred or a PImax < 60 cmH2O. Besides daytime clinical and PFT assessment, nocturnal evaluation is essential in ALS. The prevalence of sleep apnea ranges from 16 % to 76 %. Transcutaneous PCO2 (tcPCO2) is an attractive technique to evaluate non invasively nocturnal hypoventilation. The technique is well validated in different settings. Its use in neuromuscular disorders (NMD) is recent. In particular one study has demonstrated a high predictive value of tcPCO2 for the development of daytime hypoventilation within 1 year. To our knowledge, this technique has not been specifically assessed in ALS. There is a potential role for nocturnal PtcCO2 monitoring in the close follow up of ALS patients. Indeed, a close respiratory follow up of ALS patients is essential to determine the optimal timing of NIV, avoiding the occurence of unexpected acute respiratory failure.
Digital Smartwatch Measurements as Potential Biomarkers for Remote Disease Tracking in ALS
Amyotrophic Lateral SclerosisPrimary Lateral Sclerosis4 moreThis observational study will use new smartwatch technology to continuously and remotely monitor the health of ALS patients and healthy controls over time. This information will be used to develop digital biomarkers for ALS.
Evaluation of Usability and Human Factors in the Novus System
Drop FootMuscle Weakness2 moreThe Novus system intended to provide ankle dorsiflexion and knee flexion or extension in individuals with foot drop and thigh muscle weakness, following an upper motor neuron injury or disease. The primary objective of the study is to evaluate the usability of the Novus system among these patients and to gain subjects' feedback regarding the device when used on a daily basis.The secondary objective is to obtain information about the performance of the system as a gait assistive device and to evaluate the subject's Quality of Life while using the system.
PET Imaging in ALS Patients
Amyotrophic Lateral Sclerosis (ALS)This is a pilot study to evaluate a potential imaging biomarker for aiding diagnosis and monitoring progression of ALS, based on a well established basic science pathway, published human autopsy data, preliminary data in ALS mutant mice, and our recently published data using brain PET scans to image the metabotropic glutamate receptor type 5 (mGluR5) in healthy human volunteers.
Supported Treadmill Ambulation Training (STAT) for Patients Diagnosed With Amyotrophic Lateral Aclerosis...
Amyotrophic Lateral SclerosisMotor Neuron Disease1 moreThe purpose of this study is to find out if supervised exercise training using a treadmill with partial weight support is safe and has an impact on gait and function of persons with Amyotrophic lateral sclerosis.
Developing a Discrimination Model to Diagnose ALS
Amyotrophic Lateral SclerosisTo combine several brain imaging techniques to develop a new diagnostic test to help with earlier diagnosis of amyotrophic lateral sclerosis.