Pre-fALS

Full Title: The Pre-Symptomatic Familial ALS (Pre-fALS) Study
Status: Currently Recruiting

This is a longitudinal observational study of people who are at risk for developing fALS. We identify people as being ‘at risk’ if they have at least two family members with ALS and have an identifiable mutation (genetic change) in a gene that is known to cause ALS (SOD1, C9ORF72, TDP-43, FUS, etc.). By studying these individuals, we hope to better understand the early pre-clinical stage of the disease, learn more about environmental risk factors for ALS, develop biomarkers of the early stages of the disease, and work towards one day preventing the disease.

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CReATe PGB

Full Name: CReATe Study #8001: Phenotype, Genotype, & Biomarkers in ALS and Related Disorders (PGB)
Status: Currently Recruiting

The multi-center Clinical Research in ALS and Related Disorders for Therapy Development (CReATe) Consortium, initiated and led by Dr. Michael Benatar, is part of the NIH Rare Diseases Clinical Research Network (RDCRN).  CReATe is designed with the goals of increasing our understanding of the relationship between the genetic and physical characteristics of ALS and related disorders; developing biomarkers that might be useful in the development of new therapies for these disorders; providing training to young investigators in the ALS and related disorders research field; and annually funding pilot biomarker discovery projects.  The PGB study (with special focus on the phenotype-genotype correlation and biomarkers) is the first study initiated by the CReATe Consortium, and is currently enrolling at the University of Miami and many sites across the U.S.

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PRESS-ALS

Full Title: The ‘PRE-Symptomatic Studies in ALS’ (PRESS-ALS) Study
Status: Currently Recruiting

ALS typically begins focally (e.g. in one arm or leg) and, over time, the disease spreads to involve other body regions (e.g. other limbs or muscles that control talking, chewing, swallowing and breathing). Studying the unaffected regions of people who have been diagnosed with ALS provides an opportunity to understand the early manifestations of disease. PRESS-ALS is complementary to our Pre-fALS study, in which we evaluate people who are unaffected but at genetic risk for developing ALS. Together, these studies will help us elucidate the pre-symptomatic stage of disease, to develop biomarkers of pre-symptomatic ALS, and to work towards our goal of an early therapeutic or even a disease prevention trial.

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CRiALS Biomarker

Full Name: Clinical Research in ALS (CRiALS) Biomarker Study
Status: Currently Recruiting

The CRiALS Biomarker study is an observational study targeted towards identifying biomarkers of ALS and related neurological disorders. Biomarkers are indicators of disease that can be easily measured. Their discovery is essential for the development of new therapies, as well as for detecting disease in its earlier stages. This study is attempting to discover both wet (blood, urine, cerebrospinal fluid) and dry (imaging, muscle and respiratory assessment, neurological exam) biomarkers.

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CRiALS Genetics

Full Name: Clinical Research in ALS (CRiALS) Genetics Study

Status: Currently Recruiting

One goal of the Clinical Research in ALS (CRiALS) research program is to further our understanding of the genetic causes of ALS and related neurological disorders. The CRiALS Genetics study is designed to meet this aim by looking for mutations in known genes associated with disease, as well as looking for mutations in new genes.

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Neuraltus NP001

Full Title: Phase II Randomized, Double-Blind, Placebo-Controlled, Multicenter Study of NP001 in Subjects With Amyotrophic Lateral Sclerosis (ALS) and Evidence of Elevated Systemic Inflammation.
Status: Currently recruiting

This is a 6 month randomized, double-blind, placebo-controlled study of NP001 in subjects with ALS and evidence of elevated systemic inflammation. Subjects will be allocated (1:1) to NP001 and placebo. Drug or placebo will be given by intravenous administration for 5 consecutive days in Month 1 and for 3 consecutive days in Months 2 through 6.

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Skulpt Inc. EIM Study

Full Name: Noninvasive assessment of neuromuscular disease using electrical impedance (Skulpt Inc. EIM)
Status: Completed

This is a multicenter study that is being done to further develop and refine a new handheld medical device called Electrical Impedance Myography (EIM) developed by Skulpt Inc. for the purpose of assessing amyotrophic lateral sclerosis. New methods for evaluating patients with ALS are needed to help assess the potential benefit of new therapies. The study will include the testing of muscles using a number of non-invasive techniques including EIM.

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EIM

Full Title: Electrical Impedance Myography as an Outcome Measure in ALS Clinical Trials (EIM)
Status: Completed

Electrical impedance myography (EIM) is a novel technique that quantifies the physical characteristics of muscle. The goal of this study is to evaluate the utility of EIM as an outcome measure for use in ALS clinical trials. EIM is an entirely painless and non-invasive test. The study involves regular visits to a study center over a period of about 12 months. This study is being lead by Dr. Seward Rutkove at the Beth Israel Deaconess Medical Center in Boston.

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MRI (MRS & DTI)

Full Title: Magnetic Resonance Imaging Biomarkers in ALS (MRI)
Status: Completed

The goal of this study is to evaluate magnetic resonance spectroscopy (MRS) and diffusion tensor imaging (DTI) of the brain and spinal cord as potential biomarkers of disease progression. MRS and DTI are two types of MRI scan. This is a longitudinal study in which participants will return every 3-6 months. At each study visit they will undergo an hour-long MRI scan. This study is open to patients with either sporadic or familial ALS. The study also enrolls healthy subjects to serve as a control population.

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Arimoclomol

Full Title: Phase II/III Randomized, Placebo-Controlled Trial of Arimoclomol in SOD1 Positive Familial ALS (Arimoclomol)
Status: Completed

This is a randomized controlled clinical trial. People who are affected with rapidly progressive ALS that is caused by specific mutations in the SOD1 gene are eligible to participate. Study participants are randomized 1:1 to receive Arimoclomol or placebo for a period of 12 months. The trial requires two visits to a study center; all other study procedures are completed in participants’ homes.

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IBMPFD-ALS

Full Title: Motor Neuron Genotype-Phenotype Correlation (IBMPFD)
Status: Completed

IBMPFD is a degenerative disease characterized by muscle weakness, frontotemporal dementia and Paget’s disease of bone. The goal of this study is to better understand the cause of muscle weakness and the extent to which this results from motor neuron disease rather than primary muscle pathology

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Georgia ALS Registry

Full Title: Georgia ALS Registry: A Feasibility Study
Status: Completed

Efforts are underway to establish a national ALS registry. In preparation for this National Registry, we conducted a pilot study in Georgia to examine the feasibility of using existing data sources for correctly identifying cases of ALS or related motor neuron diseases.

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Cochrane Collaboration

Full Title: Cochrane Collaboration
Status: Completed

The Cochrane Collaboration develops systematic reviews and meta-analyses of important medical topics. In 2009 Benatar et al published a review of treatments for familial ALS/motor neuron disease, which is currently in the process of being updated with new data from more recently published randomized controlled trials. We are also in the midst of preparing a review on the subject of enteral feeding in patients with ALS.

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Cutometry

Full Title: Visco-elastic Properties of Skin in People at Risk for Familial ALS (Cutometry)
Status: Active, not recruiting

Cutometry is a non-invasive technique that measures the elastic properties of skin. Prior studies have suggested that the skin of patients with ALS may differ from that of people without ALS. The goal of this study is to determine whether the elastic properties of skin change prior to the onset of ALS and how these changes compare to those observed in people who have already developed ALS. Both people at risk for fALS and people with fALS may participate.

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Stem Cells

Full Title: Generation and Characterization of Amyotrophic Lateral Sclerosis iPS Cells (Stem Cells)
Status: Completed

The goal of this study is to generate and characterized induced pleuripotent stem (iPS) cells from fibroblast cultures of people who harbor a mutation in the SOD1 gene. Participants undergo a 3mm punch skin biopsy, from which fibroblasts and iPS cells are generated. The study is being led by Dr. Jeffrey Rothstein and Dr. Nicholas Maragakis at Johns Hopkins University in Baltimore.

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Reference Electrodiagnostic Data Study (Normative Data)

Full Title: Reference Electrodiagnostic Data Study (Normative Data)
Status: Completed

The interpretation of nerve conduction study data requires an appreciation of the range of values that might be encountered in a normal healthy population. In this study, commonly used nerve conduction studies were performed in a large group of healthy volunteers. The data were analyzed using a technique known as quantile regression to develop reference data (sometimes called normative data) for the nerve conduction studies commonly used in routine clinical practice.

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HIV Neuropathy

Full Title: Characteristics, Risk Factors and Prevention of HIV Polyneuropathy in South Africa (HIV Neuropathy)
Status: Completed

This is a prospective observational study of risk factors for anti-retroviral toxic neuropathy in an HIV positive population that is being conducted in Cape Town, South Africa. The goals are to evaluate the importance of risk factors such as prior tuberculosis and anti-tuberculosis therapy, malnutrition and alcohol consumption with respect to the risk of stavudine-induced polyneuropathy.

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Synaptic Activity Following Nerve Injury (SAFNI)

Full Title: Synaptic Activity Following Nerve Injury (SAFNI)
Status: Completed

Animal studies have suggested that nerve regeneration following injury is not always accompanied by the re-establishment of complex synaptic circuitry. For this study we are recruiting patients with a history of Bell’s palsy or the Guillain-Barre syndrome. The study involves electrical testing of nerves on at least two occasions to learn more about how nerves recover following injury.

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