Clinical Trials

The purpose of this study is so that blood (plasma and blood cells) and/or extra spinal fluid (which was taken for clinical purposes) can be obtained and stored for later research on a large cohort of patients with progressive motor neuronopathies and neuropathies, many of which will be determined to suffer from Amyotrophic Lateral Sclerosis (ALS). 

The purpose of this study is to evaluate and rank-order a set of new outcome measures administered to patients with amyotrophic lateral sclerosis (ALS), in order to identify measures that are more sensitive to disease progression than the current Amyotrophic Lateral Sclerosis Functional Rating Scale-Revised (ALSFRS-R).

The clinical development program has demonstrated that Edaravone is a well-tolerated treatment that slows the loss of physical function in ALS.
However, as observed in prior clinical trials, ALS presentation and progression is extremely heterogeneous among patients. There is therefore an imperious need for biomarkers, which could act as reliable indicators for Edaravone’s effect on disease progression and help better elucidate Edaravone’s mechanism of action. Biomarkers can help explain empirical results of clinical outcomes by helping us understand the mechanistic molecular and cellular pathways to clinical response. Future applications of biomarker identification will include determining the feasibility and face validity of these biomarkers in a patient registry study conducted in patients going on Edaravone as part of their clinical treatment plan, incorporating identified biomarkers in a large controlled post-marketing commitment study required by FDA studying higher and more frequent dosing of intravenous Edaravone in ALS compared to usual dosing, and optimizing development and dosing scheme for non-intravenous Edaravone formulation.
The present protocol is designed to investigate a selected panel of biomarkers in patients with ALS, treated with Edaravone. Biomarkers of oxidative stress, inflammation and neurodegeneration will be explored. Epigenetic and protein biomarkers will also be investigated.

This study proposes to identify the molecular defect in families with a clinical phenotype of “probable Inclusion Body Myositis” (“probable IBM”) but affecting  more than one family members, with or without family history of ALS.

This study is intended to obtain clinical information and establish a repository of DNA, RNA, peripheral blood monocyte, lymphocyte and skin tissue samples from people with ALS and related neurodegenerative motor neuron diseases, people with a family history of these conditions, and healthy people with no family history of these disorders. The samples will be used in future research to learn about how these disorders affect people, what causes these conditions, and how the investigators can tell when someone has this kind of disease. Future research may also include the generation of stem cells from stored blood cell and skin cell samples. Participants will not be paid for taking part in this study.

The purpose of this study is to collect clinical data, serial blood samples for plasma, serum, DNA and RNA extraction, and a single time blood sample for peripheral blood monocytes for the preparation of stem cell lines and lymphocytes for the preparation of lymphoblastoid cell lines. There will also be a single time skin biopsy sample for tissue examination and preparation of skin fibroblasts from patients who have familial or sporadic ALS, ALS-FTD or similar neurodegenerative motor neuron disorders, and are participating in NINDS funded research projects in the Mayo Clinic ALS Center.

This study is being done to collect skin samples from people with and without neurodegenerative and vascular disorders including Parkinson’s disease (PD), Alzheimer’s disease (AD), amyotrophic lateral sclerosis (ALS), stroke and many others. We will use these skin samples to make and bank (store) a group of cells (cell line) called inducible pluripotent stem (iPS) cells.

The purpose of this study is to evaluate the clinical efficacy of nL-CHCHD-001 in motor function and survival in a single ALS participant., and to evaluate further the effects of nL-CHCHD-001 in slowing, halting, or reversing the rate of decline of clinical functioning and improvement in biomarkers of disease severity in a single ALS participant.

The purposes of this study are to measure the impact of two doses of ALZT-OP1a (cromolyn) on neuro-inflammation by measuring plasma neuroinflammatory biomarkers over a 12-week treatment period, to evaluate the effects of ALZT-OP1a (cromolyn) on functional changes in subjects with mild-moderate stage ALS by using the ALS Functional Rating Scale-Revised (ALSFRS-R) to measure changes in function over a 12-week treatment period, to evaluate the safety of the ALZT-OP1a (cromolyn), and to determine the optimal dose selection of ALZT-OP1a (cromolyn) out of the two doses used in the study.

The purpose of this study is to develop a natural history dataset and biorepository of early Primary Lateral Sclerosis (PLS) and well-established PLS cases for future clinical trials.