Clinical Trial: Deep Phenotyping in Patients With ALS

Study Status: Recruiting
Recruit Status: Recruiting
Study Type: Observational

Official Title: Fluid Biomarkers With Deep Phenotyping in Patients With ALS

Brief Summary: This study aims to establish a biorepository and phenotyping database to investigate longitudinal changes in ALS subjects. Blood, including DNA and RNA, cerebrospinal fluid (CSF) and electrophysiologic measures will be collected every 3 months over 1 year. The database and specimen repository will be made available to ALS researchers on a merit basis.

Detailed Summary:

Rationale for the Study

The Northeast ALS Consortium (NEALS) biorepository is an existing resource which has provided scientists with a wide range of samples associated with clinical information. It is primarily cross sectional in nature; longitudinal collections have been associated with a limited set of clinical and functional assessments. The investigators goal is to continue to build this repository with the collection protocol proposed here, to associate biofluid collection with specific measures of upper and lower motor neuron function. This project will compliment others to upgrade the NEALS biorepository infrastructure, including updating the computer systems, expanding the number of sample freezers and establishing a repository in the Western United States, and strengthening the standard operating procedures for the repository. The project also supports existing efforts to expand the biorepository by collecting new blood and spinal fluid samples from people with ALS.

Study Design This is a multicenter, non interventional, longitudinal study in patients with ALS. There will be four (4) subject visits in this study: Baseline, month 6, month 12, and month 18. At each visit, subjects will have biofluids collected, and be evaluated with assessment tools that focus on upper and lower motor neuron burden as well as cognitive function.

Study Objectives and Endpoints The primary objective of the study is to obtain deep phenotyping information on patients studied at regular intervals over 18 months, in conjunction with biofluid collection over that same time period.

The secondary objective of the study is to integrate data from this study to other ongoing projects via the NEALS biorepository, and future collections now being
Sponsor: Barrow Neurological Institute

Current Primary Outcome:

  • ALS Functional Rating Scale Revised (ALSFRS-R) [ Time Frame: Every 6 months through month 18 ]
    The ALSFRS-R is a quickly administered (5 minutes) instrument used to determine subjects' assessment of their capability and independence in 12 functional activities, each rated on an ordinal scale (ratings 0-4).
  • Slow vital capacity (SVC) [ Time Frame: Every 6 months through month 18 ]
    Vital capacity will be measured as slow vital capacity (SVC) using standard technique
  • Hand held dynamometry (HHD) [ Time Frame: Every 6 months through month 18 ]
    Hand held dynamometry (HHD) will be used as a quantitative measure of muscle strength for this study. 10 muscle groups will be examined bilaterally in both upper and lower extremities. Mean and standard deviation for each muscle group will be established from the initial values for each subject in this trial, so that strength determinations can be converted to Z scores and averaged to provide an HHD megascore for both upper and lower extremities.
  • ALS cognitive behavioral screen (ALS-CBS) [ Time Frame: Every 6 months through month 18 ]
    The ALS Cognitive Behavioral Screen (ALS-CBS™) is a short measure of cognition and behavior in patients with Amyotrophic Lateral Sclerosis (ALS). The cognitive section includes commonly used elements of standard testing batteries, consisting of 8 tasks, with a possible total score of 20. It can be administered by a physician or other clinical care staff and takes approximately 5 minutes to complete. The behavioral section (ALS Caregiver Behavioral Qu

    Original Primary Outcome:

    • ALS Functional Rating Scale Revised (ALSFRS-R) [ Time Frame: Every 6 months through month 18 ]
      The ALSFRS-R is a quickly administered (5 minutes) instrument used to determine subjects' assessment of their capability and independence in 12 functional activities, each rated on an ordinal scale (ratings 0-4).
    • Slow vital capacity (SVC) [ Time Frame: Every 6 months through month 18 ]
      Vital capacity will be measured as slow vital capacity (SVC) using standard technique
    • Hand held dynamometry (HHD) [ Time Frame: Every 6 months through month 18 ]
      Hand held dynamometry (HHD) will be used as a quantitative measure of muscle strength for this study. 9 muscle groups will be examined bilaterally in both upper and lower extremities. Mean and standard deviation for each muscle group will be established from the initial values for each subject in this trial, so that strength determinations can be converted to Z scores and averaged to provide an HHD megascore for both upper and lower extremities.
    • ALS cognitive behavioral screen (ALS-CBS) [ Time Frame: Every 6 months through month 18 ]
      The ALS Cognitive Behavioral Screen (ALS-CBS™) is a short measure of cognition and behavior in patients with Amyotrophic Lateral Sclerosis (ALS). The cognitive section includes commonly used elements of standard testing batteries, consisting of 8 tasks, with a possible total score of 20. It can be administered by a physician or other clinical care staff and takes approximately 5 minutes to complete. The behavioral section (ALS Caregiver Behavioral Que

      Current Secondary Outcome:

      Original Secondary Outcome:

      Information By: Barrow Neurological Institute

      Dates:
      Date Received: June 7, 2016
      Date Started: February 7, 2017
      Date Completion: July 2018
      Last Updated: February 9, 2017
      Last Verified: February 2017