An observational study in Motor Neuron Disease and Primary Lateral Sclerosis, sponsored by National Institute of Neurological Disorders and Stroke (NINDS). Completed at 1 site in United States. Open to participants aged 20 Years and older. Per ClinicalTrials.gov, last updated 2019-11-05.
Sponsored by National Institute of Neurological Disorders and Stroke (NINDS) · Observational
Background:
- Primary lateral sclerosis (PLS) is a disorder in which nerve cells in the brain that control movement degenerate. The cause of PLS is not known, but some research has suggested that environmental factors that produce oxidative stress trigger PLS in people who carry certain genes. Oxidative stress is caused when the body makes chemicals called "free radicals" faster than its natural systems can break them down. Oxidative stress can be triggered by exposures to chemicals related to the bodily effects of lead, smoking, alcohol consumption, physical activity, and psychological stress. Chemicals produced by the body during oxidative stress can be measured in the blood and urine. Researchers are interested in studying the physical, neurological, and chemical effects of PLS to better understand the effects of oxidative stress on the disorder.
Objectives:
- To study the relation of oxidative stress to the diagnosis and progression of motor neuron disease.
Eligibility:
- Individuals 20 years of age or older who have been diagnosed with PLS, and have had symptoms of PLS for at least 5 but not more than 8 years and been previously enrolled in 01-N-0145 Screening: Neurologic Disorders with Muscle Stiffness
Design:
Objective
Primary lateral sclerosis (PLS) and amyotrophic lateral sclerosis (ALS) are motor neuron disorders with different phenotypes that progress at very different rates. ALS is a rapidly progressive disease with a median survival less than 5 years. Patients with PLS have a slowly progressive course with a normal lifespan. One hypothesis is that oxidative stress affects the way in which different motor neuron disorders progress. To test this hypothesis, exposures to putative triggers of oxidative stress and biomarkers that may reflect oxidative stress will be assessed in patients with motor neuron disorders. A multicenter effort (the COSMOS study) has been initiated to accumulate sufficient numbers of ALS patients to address this hypothesis. As an add-on study, PLS patients will also be assessed in the multicenter effort. The objective of this protocol is to enroll PLS patients in this multicenter effort. The goal is to assess environmental factors and markers of oxidative stress in patients with established PLS.
Study Population
15 adult patients with PLS who have symptoms of pure upper motor neuron dysfunction for at least 5 but not more than 15 years.
Design
Patients will undergo a standard battery of clinical, physiological, and cognitive screening tests at enrollment, with scheduled follow-up evaluation visits every 12 months for 36 months. Blood and urine samples will be sent to collaborators at Columbia University for analysis of markers of oxidative injury and genetic risk factors. Patients will complete a self-administered nutritional survey and will be interviewed by phone by Columbia University investigators using questionnaires to assess environmental, occupational, lifestyle and psychosocial factors thought to be triggers of oxidative stress.
Outcome Measures
The Columbia University collaborators will combine data from several centers in a regression model correlating the slope of decline of the ALS-FRS score with an index of oxidative stress.
717 studies on the registry are indexed under Motor Neuron Disease; 135 are open to participants now.
This study's enrollment of 10 is below the median of 107 across 216 observational studies indexed under Motor Neuron Disease.
Browse Motor Neuron Disease studies →National Institute of Neurological Disorders and Stroke (NINDS) is the lead sponsor of 601 studies on the registry; 56 are open to participants now.
Of its 18 completed or terminated interventional studies of FDA-regulated products, 8 (44%) have results posted.
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Patients will be included if they:
Are 20 years or older
Have PLS, that is pure UMN dysfunction (spasticity, pathological hyperreflexia, pathological reflexes with or without motor weakness) of undetermined etiology
Have been evaluated at NIH and are being willing to return for active follow-up for 3 years
Had PLS symptom onset at least 5 years prior to the study enrollment but not more than 15 years
Have normal nerve conduction studies and normal needle electrode examination performed within 12 months of the time of enrollment in this study
Have no other definable diseases causing spasticity such as structural brain or spinal cord disease, metabolic diseases, paraneoplastic syndromes, hereditary diseases, infectious diseases, or other significant neurological abnormalities
Have a reliable family caregiver who can assist in providing responses on telephone interviews and questionnaires if the proband has problems with speaking or writing
Are fluent in English
Ability to provide his/her own informed consent
EXCLUSION CRITERIA:
Patients will be excluded if they:
Have EMG evidence of active denervation or fasciculations in more than a few muscles with chronic neurogenic motor unit potentials at the time of enrollment
Have only lower extremity involvement
Have major medical diseases (e.g. active cancer, dialysis) that have required active medical treatment within the past 6 months
Are participating in clinical treatment trials at the time of enrollment and acquisition of baseline biological samples (participation in clinical trials after the baseline visit is permitted)
Are unwilling or unable to return for follow-up visits
Have pacemakers or other implanted electrical devices, which might make TMS unsafe will be excluded from TMS testing
Decline in ALSFRS score
This study is completed, as verified in Aug 2015. You cannot join it, but the record below documents what was studied.
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