Focal Accumulation of Iron in Cerebral Regions in Early ALS (Amyotrophic Lateral Sclerosis) Patients...
ALS (Amyotrophic Lateral Sclerosis)Iron OverloadThe FAIR-ALS study is to investigate the safety and efficacy of a scavenger treatment of iron deferiprone, which would reduce the brain iron to limit the development of amyotrophic lateral sclerosis. It has been shown an excess of iron in the central nervous system carrying a sporadic ALS patients. Iron overload associated with a loss of motor neurons may explain the signs of the disease (atrophy). The investigators discuss the hypothesis that reducing excess iron, the investigators can reduce the loss of neurons and thus the progression of signs of the disease.
Assessment of the Effects of Sheffield Support Snood in MND Patients
Motor Neurone DiseaseThe aim of this study is to evaluate the effects on MND patients of a new collar specifically designed for people affected by neck weakness: the Sheffield Support Snood.
Dexpramipexole Renal PK Study
Amyotrophic Lateral SclerosisRenal InsufficiencyThis is a multicenter, open-label, single-dose, PK and safety study in subjects with various stages of renal impairment.
Trial of Ascertaining Individual Preferences for Loved One's Role in End-of-Life
Amyotrophic Lateral SclerosisGI Cancer1 moreSpecific Aims and Hypotheses: Aim 1: To test the effect of the "Trial of Ascertaining Individual preferences for Loved Ones' Role in End-of-life Decisions" (TAILORED) Intervention on family decision-making self-efficacy at 8 weeks both with respect to the patient's present situation and in a hypothetical situation in which the patient lacks decision-making capacity. Hypotheses 1a: Family decision-making self-efficacy will be greater at 8 weeks in pairs that have undergone the TAILORED Intervention than in pairs receiving the standard information on advance directives in the patient's present situation. Hypotheses 1b: Family decision-making self-efficacy will be greater at 8 weeks in pairs that have undergone the TAILORED Intervention than in pairs receiving the standard information on advance directives in the hypothetical situation in which the patient lacks decision making capacity. Aim 2: To test the effect of the TAILORED Intervention on family psychological outcomes (depression, caregiver burden, decision making distress). Hypotheses 2a: Depression will be less at 8 weeks in family members who have undergone the TAILORED Intervention than in family members who have received the standard information on advance directives. Hypotheses 2b: Caregiver burden will be less at 8 weeks in family members who have undergone the TAILORED Intervention than in family members who have received the standard information on advance directives. Hypotheses 2c: Decision-making distress will be less at 8 weeks in family members who have undergone the TAILORED Intervention than in family members who have received the standard information on advance directives. Aim 3: To test the effect of the TAILORED Intervention on patient and family satisfaction with family decision-making involvement. Hypothesis 3a: Patient satisfaction with family decision involvement will be greater at 8 weeks in patients who have undergone the TAILORED Intervention than in patients receiving the standard information on advance directives. Hypothesis 3b: Family member satisfaction with decision involvement will be greater at 8 weeks in family members who have undergone the TAILORED Intervention than in family members receiving the standard information on advance directives. Aim 4: To explore family decision-making self-efficacy and perceptions of the TAILORED Intervention.
R(+)PPX High Dose Treatment of ALS
Amyotrophic Lateral SclerosisR(+)pramipexole is administered in escalating doses to patients with early ALS. Plasma and spinal fluid levels of R(+)PPX are monitored, in addition to biochemical markers of oxidative stress.
Safety and Efficacy of TRO19622 as add-on Therapy to Riluzole Versus Placebo in Treatment of Patients...
Amyotrophic Lateral SclerosisThe purpose of the assay is to assess the safety and the efficacy of TRO19622 330 mg QD as add-on therapy to riluzole 50 mg bid in the treatment of patients suffering from ALS, as compared to placebo, assessed by the 18-month survival rate.
Trial of Amivita in Amyotrophic Lateral Sclerosis
Amyotrophic Lateral SclerosisThe primary objectives of this study are to determine the safety and efficacy of Amivita, a compound of amino acids and vitamines in patients with Amyotrophic lateral sclerosis (ALS)ALS. The secondary objectives are to measure quality of life before and during intervention. This is a self-controlled clinical trial. Twenty patients in our ALS center who are already receiving riluzole or other treatments but the condition is worsening will receive treatment for 1o months. The evaluating investigators will be blinded to treatment assignment. Primary outcome measures will be adverse events, the ALS Functional Rating Scale-Revised (ALSFRS-R), and survival. Subjects will also be assessed at enrollment and at study end for weight loss, forced vital capacity (FVC), quality of life and grip strength.
Autologous Bone Marrow Mesenchymal Stem Cells in the Treatment of Patients With Amyotrophic Lateral...
Amyotrophic Lateral SclerosisThe goal of this study is to investigate the safety and tolerability of autologous bone marrow-derived mesenchymal stem cells administration in the individuals with diagnosed amyotrophic lateral sclerosis.
Treatment Effect of Edaravone in Patients With Amyotrophic Lateral Sclerosis (ALS)
Neuromuscular DiseasesAmyotrophic Lateral Sclerosis (ALS) is a neurodegenerative disease that causes the death of 30,000 affected individual every year. Complex nature and unknown pathogenesis of this disease are 2 major reasons for failure of therapeutic interventions. Edaravone is a free radical scavenger that slows down functional decline and prevents from disease progression in ALS patients. FDA newly approved this drug in these patients (2017/5/5). In this study, investigators aimed to assess the treatment effect of this newly approved drug in patients with ALS in a representative Iranian population.
Blood-Brain Barrier Opening Using MR-Guided Focused Ultrasound in Patients With Amyotrophic Lateral...
Amyotrophic Lateral SclerosisThe purpose of this study is to evaluate the safety, tolerability, and feasibility of Blood-Brain Barrier (BBB) opening using transcranial MRI-guided focused ultrasound in conjunction with an intravenous ultrasound contrast agent in patients with Amyotrophic Lateral Sclerosis (ALS).