A Longitudinal Study of Amyotrophic Lateral Sclerosis (ALS) Biomarkers

This study is currently recruiting participants. (see Contacts and Locations)
Verified April 2015 by Massachusetts General Hospital
Sponsor:
Collaborator:
ALS Therapy Alliance
Information provided by (Responsible Party):
James D. Berry MD, Massachusetts General Hospital
ClinicalTrials.gov Identifier:
NCT01495390
First received: December 16, 2011
Last updated: April 15, 2015
Last verified: April 2015
  Purpose

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
Amyotrophic Lateral Sclerosis
Nervous System Diseases
Sclerosis
Motor Neuron Disease
Spinal Cord Diseases
Central Nervous System Diseases
Neurodegenerative Diseases
Neuromuscular Diseases
ALS

Study Type: Observational
Study Design: Observational Model: Case-Only
Time Perspective: Prospective
Official Title: A Multicenter Study for the Discovery and Validation of ALS Biomarkers

Resource links provided by NLM:


Further study details as provided by Massachusetts General Hospital:

Primary Outcome Measures:
  • ALS Functional Rating Scale [ Time Frame: Approximately every 4 months ] [ Designated as safety issue: No ]
    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.

  • Vital Capacity (VC) [ Time Frame: Approximately every 4 months ] [ Designated as safety issue: No ]
    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.

  • Hand Held Dynamometry (HHD) [ Time Frame: Approximately every 4 months ] [ Designated as safety issue: No ]
    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.

  • Ashworth Spasticity Scale [ Time Frame: Approximately every 4 months ] [ Designated as safety issue: No ]
    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.

  • Fronto-Temporal Dementia (FTD) Assessment [ Time Frame: Approximately every 4 months ] [ Designated as safety issue: No ]
    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.

  • ALS Cognitive Behavioral Screen (ALS CBS) [ Time Frame: Approximately every 4 months ] [ Designated as safety issue: No ]

    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.


Estimated Enrollment: 250
Study Start Date: November 2011
Estimated Study Completion Date: December 2015
Estimated Primary Completion Date: December 2015 (Final data collection date for primary outcome measure)
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.

  Eligibility

Ages Eligible for Study:   18 Years and older
Genders Eligible for Study:   Both
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

Criteria

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.
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT01495390

Contacts
Contact: Sylvia Baedorf Kassis, MPH 617-643-5582 sbaedorfkassis@partners.org

Locations
United States, Arizona
Barrow Neurological Institute Recruiting
Phoenix, Arizona, United States, 85013
Contact: Gale Kittle, RN, MPH    602-406-4792    Gale.Kittle@DignityHealth.org   
Principal Investigator: Shafeeq Ladha, MD         
United States, Florida
Mayo Clinic Jacksonville Recruiting
Jacksonville, Florida, United States, 32224
Contact: Karen Overstreet    904-953-6523    overstreet.karen@mayo.edu   
Principal Investigator: Kevin Boylan, MD         
United States, Georgia
Emory University Recruiting
Atlanta, Georgia, United States, 30322
Contact: Latoya Shaw    404-727-1673    Latoya.q.shaw@emory.edu   
Principal Investigator: Jonathan Glass, MD         
United States, Massachusetts
Massachusetts General Hospital Recruiting
Boston, Massachusetts, United States, 02114
Contact: Christina Dheel    617-643-6252    cdheel@partners.org   
Principal Investigator: James Berry, MD, MPH         
University of Massachusetts Medical Center Recruiting
Worcester, Massachusetts, United States, 01655
Contact: Diane McKenna-Yasek    508-856-4697    diane.mckenna-yasek@umassmed.edu   
Principal Investigator: Robert Brown, MD, DPhil         
United States, Pennsylvania
University of Pittsburgh Recruiting
Pittsburgh, Pennsylvania, United States, 15213
Contact: Danielle Rowlands, RN    412-648-9053    doerdx@UPMC.EDU   
Principal Investigator: David Lacomis, MD         
Sponsors and Collaborators
Massachusetts General Hospital
ALS Therapy Alliance
Investigators
Principal Investigator: James D. Berry, MD, MPH Massachusetts General Hospital
  More Information

Additional Information:
No publications provided

Responsible Party: James D. Berry MD, Principal Investigator, Massachusetts General Hospital
ClinicalTrials.gov Identifier: NCT01495390     History of Changes
Other Study ID Numbers: BIO-ALS-02
Study First Received: December 16, 2011
Last Updated: April 15, 2015
Health Authority: United States: Institutional Review Board

Keywords provided by Massachusetts General Hospital:
Biomarkers
Biological Markers
Surrogate Markers
Clinical Markers
Plasma
Cerebrospinal Fluid
DNA
Serum
RNA

Additional relevant MeSH terms:
Neurodegenerative Diseases
Amyotrophic Lateral Sclerosis
Central Nervous System Diseases
Motor Neuron Disease
Nervous System Diseases
Neuromuscular Diseases
Sclerosis
Spinal Cord Diseases
Metabolic Diseases
Pathologic Processes
Proteostasis Deficiencies
TDP-43 Proteinopathies

ClinicalTrials.gov processed this record on June 28, 2015