View clinical trials related to Amyotrophic Lateral Sclerosis.
Filter by:A Phase II, double-blind, randomized, placebo-controlled ascending dose titration study to evaluate the safety, tolerability, pharmacokinetics and pharmacodynamic effects of multiple ascending doses of CK-2017357 to an individual patient maximum tolerated dose (MTD), using a within-patient twice daily (BID) dose-titration regimen in ALS patients on 50 mg riluzole once daily (QD).
The investigators laboratory has been studying families with a history of ALS for more than 30 years and is continuing to use new ways to understand how genes may play a role in ALS, motor neuron disease and other neuromuscular disorders. The purpose of this study is to identify additional genes that may cause or put a person at risk for either familial ALS (meaning 2 or more people in a family who have had ALS), sporadic ALS, or other forms of motor neuron disease in the hopes of improving diagnosis and treatment. As new genes are found that may be linked to ALS in families or individuals, the investigators can then further study how that gene may be contributing to the disease by studying it down to the protein and molecular level. This includes all forms of ALS, motor neuron disease and ALS with fronto-temporal dementia(ALS/FTD). We also continue to study other forms of neuromuscular disease such as Miyoshi myopathy, FSH dystrophy and other forms of muscular dystrophy by looking at the genes that may be associated with them. There have been a number of genes identified that are associated with both familial and sporadic ALS, with the SOD1, C9orf72, and FUS genes explaining the majority of the cases. However, for about 25% of families with FALS, the gene(s) are still unknown. The investigators also will continue to work with families already identified to carry one of the known genes associated with ALS.
This Phase 1 study will explore the safety, tolerability, and pharmacokinetics of single doses ranging from 300 to 600 mg and multiple daily doses ranging from 225 mg to 300 mg BID dexpramipexole in healthy volunteers.
This is a multicenter, open-label, single-dose, PK and safety study in subjects with various stages of renal impairment.
This is a single and multiple dose, open-label study to evaluate the pharmacokinetics (PK), safety, and tolerability of dexpramipexole administered orally to adult Japanese and Caucasian healthy subjects.
This is an open label safety and tolerability continuation study of intracerebroventricular administration of sNN0029, containing the growth factor VEGF165, in patients with amyotrophic lateral sclerosis that have previously participated in study sNN0029-001. The intention of the study is to investigate safety and tolerability of intracerebroventricular administration of sNN0029 and whether it can improve motor function and prolong survival in patients with ALS.
The study will generate data on safety, tolerability and pharmacokinetics after multiple daily doses of CK-2017357 in patients with ALS. Patients will be randomized into one of four different treatment groups, receiving daily oral doses of either placebo, 125 mg, 250 mg, or 375 mg of CK-2017357 for 14 days.
G72 gene is located on the common linkage locus in bipolar disorder and schizophrenia, and it encodes D-amino acid oxidase activator (DAOA). There are evidences that elucidated G72 and D-amino acid oxidase(DAO) together playing a critical role in the pathophysiology of schizophrenia. Recently, reports discovered missense mutations in the DAO (R199W DAO and R38H DAO) are associated with familial amyotrophic lateral sclerosis (FALS), and our preliminary data showed that the level of G72 autoantibody decreases in patients with ALS compared with normal control. Thus, we want to find out whether G72 plays a role in ALS and neurodegenerative diseases including Alzheimer disease and Parkinson's disease. First, we detect G72 protein and its autoantibody in sera of neurodegenerative diseases patients using ELISA and Western blotting, and the data are compared with normal control. We hypothesize the levels of G72 protein and its autoantibody in neurodegenerative diseases are less than those in normal control. Then, we extract genomic DNA of neurodegenerative diseases patients, and use polymerase chain reaction(PCR) to detect single nucleotide polymorphism (SNP) of G72. We aim to detect G72 missense SNP variants presented in ALS, AD and PD.
Background: -Spinal and bulbar muscular atrophy (SBMA) is an inherited disorder that affects men. People with SBMA often have weakness throughout the body, including the muscles they use for swallowing, breathing, and speaking. We do not know if exercise helps or harms people with SBMA. Objective: -To see if a 12-week program of either functional exercise or stretching exercises will improve strength, function, or quality of life in people with SBMA Eligibility: - Participants will be men 18 years of age or older who have genetic confirmation of SBMA. - They must be able to walk at least 50 feet with or without an assistive device such as a cane or a walker and stand for 10 minutes without using an assistive device. - They must have access to a computer with an Internet connection. Design: - At the first visit to NIH (2 days), participants will have a medical history taken and undergo a physical exam. They will also have blood tests and an EKG, and complete questionnaires about mood, health, and exercise. Tests of muscle strength, balance, and endurance will also be done. - Participants who qualify for the study will receive instruction about either strengthening or stretching exercises. They will do these exercises at home one to three times a week for 12 weeks. - They will wear a small activity monitor while they exercise and record their exercise in a diary. - At the end of 12 weeks, participants will return to the NIH for 2 days. They will undergo the same tests as they had on the first visit. - Participants will receive follow-up phone calls and e-mails during the study and for 4 weeks after the last visit....
PBA is a neurologic condition that is estimated to impact over a million patients and their families in the United States. PBA occurs secondary to an otherwise unrelated neurologic disease or injury, and manifests as involuntary, frequent, and disruptive outbursts of crying and/or laughing. Progress has been made in better understanding this debilitating condition, but much more needs to be done. That's why a new PBA patient registry, PRISM (Pseudobulbar Affect RegIstry Series), has been initiated. The goal of PRISM is to establish the prevalence and quality of life (QOL) impact of PBA in patients with underlying neurologic conditions including - Alzheimer's disease - Amyotrophic lateral sclerosis - Multiple sclerosis - Parkinson's disease - Stroke - Traumatic brain injury Because this is an observational registry, it doesn't require you to intervene with any specific treatment or procedure. Your participation allows the PRISM registry to collect and analyze data from your site and also compare it to national numbers captured in the PRISM registry about PBA across all of the major at-risk neurologic populations.