A Longitudinal Study of Amyotrophic Lateral Sclerosis (ALS) Biomarkers

A Multicenter Study for the Discovery and Validation of ALS Biomarkers

The purpose of this study is to collect biofluid samples for the banking and usage in ALS research. Through comparison of these samples, the researchers hope to learn more about the underlying cause of ALS, as well as find unique biological markers, which could be used to develop new therapies.

Study Overview

• Status: Completed
• Conditions: Central Nervous System Diseases; Nervous System Diseases; Sclerosis; Neuromuscular Diseases; Neurodegenerative Diseases; Motor Neuron Disease; Amyotrophic Lateral Sclerosis; Spinal Cord Diseases; ALS

Detailed Description

The purpose of the research study is to collect blood samples and cerebrospinal fluid (CSF) from people with amyotrophic lateral sclerosis (ALS). These samples will be collected approximately every 4 months.

• Study Type: Observational
• Enrollment (Actual): 139

Contacts and Locations

Study Locations

• United States
  • Arizona
    • Phoenix, Arizona, United States, 85013
      • Barrow Neurological Institute
  • Florida
    • Jacksonville, Florida, United States, 32224
      • Mayo Clinic Jacksonville
  • Georgia
    • Atlanta, Georgia, United States, 30322
      • Emory University
  • Massachusetts
    • Boston, Massachusetts, United States, 02114
      • Massachusetts General Hospital
    • Worcester, Massachusetts, United States, 01655
      • University of Massachusetts Medical Center
  • Pennsylvania
    • Pittsburgh, Pennsylvania, United States, 15213
      • University of Pittsburgh

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years and older (Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

• Sampling Method: Non-Probability Sample

Study Population

Volunteers will be invited to participate in this study by their neurologists either in clinic or at a regular scheduled appointment visit

Description

Inclusion Criteria:

• Age 18 or older
• Diagnosis of suspected, possible, probable or definite ALS according to El Escorial Criteria
• Vital capacity (VC) at least 50 percent predicted
• Able to undergo multiple lumbar punctures

Exclusion Criteria:

• Abnormal CSF pressure or intracranial/intraspinal tumors
• Use of anticoagulant medication that cannot be safely withheld

• Bleeding disorders

  • This is a partial listing.

Study Plan

How is the study designed?

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
ALS Functional Rating Scale	The ALSFRS-R is a quickly administered (5 min) ordinal rating scale used to determine a subject's assessment of their capability and independence in 12 functional activities. There are 12 questions, graded by the subject 0-4 (4 is normal). Score of 0 (worst) to 48 (best). Reflects speech and swallowing, fine motor skills, large motor skills, and breathing.	Approximately every 4 months
Vital Capacity (VC)	The vital capacity (VC) (percent of predicted normal) was determined using the slow VC method. Vital Capacity is the maximum amount of air a person can expel from the lungs after a maximum inhalation. A subject's VC depends on their age, sex and height. The value is recorded as a percent of predicted normal.	Approximately every 4 months
Hand Held Dynamometry (HHD)	Hand Held Dynamometry (HHD) will be used as a quantitative measure of muscle strength for this study. Six proximal muscle groups will be examined bilaterally in both upper and lower extremities (shoulder flexion, elbow flexion, elbow extension, hip flexion, knee flexion, and knee extension), all of which have been validated against maximum voluntary isometric contraction (MVIC) testing.	Approximately every 4 months
Ashworth Spasticity Scale	This is a standard measure for spasticity, has been used in a previous ALS clinical trials, and is applied in the current trial to evaluate the progression of spasticity due to upper motor neuron dysfunction in ALS.	Approximately every 4 months
Fronto-Temporal Dementia (FTD) Assessment	The FTD Screening Assessment is a quickly administered scale used to evaluate memory, executive functions, and language. It is aimed at determining the presence of subtle dysfunction of these domains of cognition and behavior that may portend the onset of FTD or FTD-like symptoms.	Approximately every 4 months
ALS Cognitive Behavioral Screen (ALS CBS)	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 Questionnaire) is composed of questions sensitive to organic brain changes. It consists of a set of questions that compare changes in personality and behavior since the onset of ALS, as well as yes/no questions about mood, pseudobulbar affect, and fatigue. It is completed by a caregiver, family member or other informant during the same time that the patient completes the cognitive portion. The questionnaire typically takes about 2 minutes to complete.	Approximately every 4 months

Collaborators and Investigators

• Sponsor: Massachusetts General Hospital
• Collaborators: ALS Association; ALS Finding a Cure
• Investigators: Principal Investigator: James D. Berry, MD, MPH, Massachusetts General Hospital

Publications and helpful links

Helpful Links

• Northeast ALS Consortium Website

Study record dates

Study Major Dates

• Study Start: November 1, 2011
• Primary Completion (Actual): December 31, 2016
• Study Completion (Actual): December 31, 2016

Study Registration Dates

• First Submitted: December 16, 2011
• First Submitted That Met QC Criteria: December 19, 2011
• First Posted (Estimate): December 20, 2011

Study Record Updates

• Last Update Posted (Actual): May 30, 2017
• Last Update Submitted That Met QC Criteria: May 24, 2017
• Last Verified: May 1, 2017

More Information

Terms related to this study

• Keywords: DNA; Serum; Biomarkers; Plasma; Biological Markers; RNA; Cerebrospinal Fluid; Surrogate Markers; Clinical Markers
• Additional Relevant MeSH Terms: Pathologic Processes; Metabolic Diseases; TDP-43 Proteinopathies; Proteostasis Deficiencies; Sclerosis; Motor Neuron Disease; Amyotrophic Lateral Sclerosis; Nervous System Diseases; Neurodegenerative Diseases; Spinal Cord Diseases; Central Nervous System Diseases; Neuromuscular Diseases
• Other Study ID Numbers: BIO-ALS-02

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: UNDECIDED