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A Longitudinal Study of Amyotrophic Lateral Sclerosis (ALS) Biomarkers

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
ClinicalTrials.gov Identifier: NCT01495390
Recruitment Status : Completed
First Posted : December 20, 2011
Last Update Posted : May 30, 2017
ALS Association
ALS Finding a Cure
Information provided by (Responsible Party):
James D. Berry MD, Massachusetts General Hospital

Brief Summary:
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.

Condition or disease
Amyotrophic Lateral Sclerosis Nervous System Diseases Sclerosis Motor Neuron Disease Spinal Cord Diseases Central Nervous System Diseases Neurodegenerative Diseases Neuromuscular 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.

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Study Type : Observational
Actual Enrollment : 139 participants
Observational Model: Case-Only
Time Perspective: Prospective
Official Title: A Multicenter Study for the Discovery and Validation of ALS Biomarkers
Study Start Date : November 2011
Actual Primary Completion Date : December 31, 2016
Actual Study Completion Date : December 31, 2016

Primary Outcome Measures :
  1. ALS Functional Rating Scale [ Time Frame: Approximately every 4 months ]
    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.

  2. Vital Capacity (VC) [ Time Frame: Approximately every 4 months ]
    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.

  3. Hand Held Dynamometry (HHD) [ Time Frame: Approximately every 4 months ]
    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.

  4. Ashworth Spasticity Scale [ Time Frame: Approximately every 4 months ]
    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.

  5. Fronto-Temporal Dementia (FTD) Assessment [ Time Frame: Approximately every 4 months ]
    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.

  6. ALS Cognitive Behavioral Screen (ALS CBS) [ Time Frame: Approximately every 4 months ]

    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.

Biospecimen Retention:   Samples With DNA
Biological samples will be collected from participants with ALS over time. These biological samples include blood (plasma, serum, and RNA) and cerebrospinal fluid (CSF). DNA will be collected at one time point.

Information from the National Library of Medicine

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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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

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.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT01495390

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United States, Arizona
Barrow Neurological Institute
Phoenix, Arizona, United States, 85013
United States, Florida
Mayo Clinic Jacksonville
Jacksonville, Florida, United States, 32224
United States, Georgia
Emory University
Atlanta, Georgia, United States, 30322
United States, Massachusetts
Massachusetts General Hospital
Boston, Massachusetts, United States, 02114
University of Massachusetts Medical Center
Worcester, Massachusetts, United States, 01655
United States, Pennsylvania
University of Pittsburgh
Pittsburgh, Pennsylvania, United States, 15213
Sponsors and Collaborators
Massachusetts General Hospital
ALS Association
ALS Finding a Cure
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Principal Investigator: James D. Berry, MD, MPH Massachusetts General Hospital
Additional Information:
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Responsible Party: James D. Berry MD, Principal Investigator, Massachusetts General Hospital
ClinicalTrials.gov Identifier: NCT01495390    
Other Study ID Numbers: BIO-ALS-02
First Posted: December 20, 2011    Key Record Dates
Last Update Posted: May 30, 2017
Last Verified: May 2017
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: Undecided
Keywords provided by James D. Berry MD, Massachusetts General Hospital:
Biological Markers
Surrogate Markers
Clinical Markers
Cerebrospinal Fluid
Additional relevant MeSH terms:
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Motor Neuron Disease
Amyotrophic Lateral Sclerosis
Nervous System Diseases
Neurodegenerative Diseases
Neuromuscular Diseases
Central Nervous System Diseases
Spinal Cord Diseases
Pathologic Processes
TDP-43 Proteinopathies
Proteostasis Deficiencies
Metabolic Diseases