Establishment of a Tissue Bank (Blood, CSF) for the Understanding of Motor Neuron Disease (MND) (WBC)

Establishment of a Tissue Bank (Blood, CSF) for the Understanding of the Disease Progression and Early Diagnosis of Motor Neuron Disease (MND).

Biomarkers are essential for the identification of disease states. There are no early diagnostic or prognostic markers for ALS. The purpose of this study is to identify a panel of biomarkers from blood or spinal fluid of ALS patients and to collect data to better understand disease progression.

Study Overview

• Status: Terminated
• Conditions: Amyotrophic Lateral Sclerosis

Detailed Description

Amyotrophic lateral sclerosis (ALS) is a motor neuron disease characterized by progressive degeneration of motor neurons, muscle atrophy and paralysis. There is no reliable early diagnostic test for ALS making identification of the disease difficult at its earliest stages. Early detection is critical to the initiation of early neuroprotective therapy. By the time a reliable diagnosis can be made, substantial damage to motor neurons and muscle has already occurred. The purpose of the current project is to establish a bank of blood samples (serum and protein/RNA/DNA from blood cells) and CSF for use in the development of an early diagnostic test for ALS and to better understand the progression of this disease.

Samples from patients that have a confirmed or unknown diagnosis of motor neuron disease will be examined. ALS and suspected neuromuscular disease (control) samples will be collected for comparison. Investigators will examine various biochemical, metabolic and genetic markers from these samples in hopes of finding differences in the expression between control subjects and ALS patients and how these biomarkers vary during disease progression. Participants will be asked to complete an optional questionnaire to collect data including medication and vitamin use and medical and disease history. This data will be linked to the patient's samples; however, all samples will be deidentified and coded to avoid the possibility of linking results to the patient. Results will not be stored in the patient's medical record.

• Study Type: Observational
• Enrollment (Actual): 766

Contacts and Locations

Study Locations

• United States
  • North Carolina
    • Charlotte, North Carolina, United States, 28207
      • Neurosciences Institute, Neurology - Charlotte

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years and older (Adult, Older Adult)
• Accepts Healthy Volunteers: Yes
• Genders Eligible for Study: All

• Sampling Method: Non-Probability Sample

Study Population

ALS patients in the Charlotte, NC, area.

Description

Inclusion Criteria:

• 18 years old or older

Exclusion Criteria:

• less than 18 years old

Study Plan

How is the study designed?

Number of groups / cohorts

2

Cohorts and Interventions

Group / Cohort
subjects w/ non-motor neurodegenerative disease; subjects with ALS or with non-motor neurodegenerative disease
subjects w/out motorneuron degenerative dis; subjects without motorneuron degenerative disease

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Levels of ALS biomarkers in blood	Blood samples will be processed to obtain serum samples and to isolate peripheral blood mononuclear cells (PBMC's). Serum samples will be analyzed for biochemical and metabolic markers of interest and future cell culture as needed. PBMCs will be processed for RNA, DNA or protein isolation. Aliquots of all samples will be stored for future study.	After blood is collected from study subjects. Data will be analyzed at one year.
Levels of ALS biomarkers in CSF	CSF samples will be processed and assayed for biomarkers of interest. An aliquot of CSF sample will be stored for future study.	After CSF is collected from study subject. Data will be analyzed at one year.

Collaborators and Investigators

• Sponsor: Wake Forest University Health Sciences
• Investigators: Principal Investigator: Benjamin R Brooks, MD, Neurosciences Institute, Neurology - Charlotte

Study record dates

Study Major Dates

• Study Start (Actual): March 29, 2004
• Primary Completion (Actual): December 31, 2017
• Study Completion (Actual): October 29, 2020

Study Registration Dates

• First Submitted: March 30, 2013
• First Submitted That Met QC Criteria: September 23, 2013
• First Posted (Estimate): September 26, 2013

Study Record Updates

• Last Update Posted (Actual): July 11, 2022
• Last Update Submitted That Met QC Criteria: July 8, 2022
• Last Verified: July 1, 2022

More Information

Terms related to this study

• Keywords: biomarkers; motor neuron disease; Amyotrophic lateral sclerosis; biological markers
• Additional Relevant MeSH Terms: Pathologic Processes; Metabolic Diseases; Central Nervous System Diseases; Nervous System Diseases; Neuromuscular Diseases; Neurodegenerative Diseases; Spinal Cord Diseases; TDP-43 Proteinopathies; Proteostasis Deficiencies; Sclerosis; Motor Neuron Disease; Amyotrophic Lateral Sclerosis
• Other Study ID Numbers: CHS-Neurology-WBC Tissue Bank