A Longitudinal Study of Amyotrophic Lateral Sclerosis (ALS) Biomarkers

NCT01495390 | Completed DMC

• 1 other identifier: BIO-ALS-02
• Study Type: observational
• Target: 139
• Locations: 1 country
• Sites: 6

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.

Conditions

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

Keywords

Biomarkers; Biological Markers; Surrogate Markers; Clinical Markers; Plasma; Cerebrospinal Fluid; DNA; Serum; RNA

Outcome Measures

Primary Outcomes (6)

• 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

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)
• 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

You may qualify if:

• 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

You may not qualify if:

• Abnormal CSF pressure or intracranial/intraspinal tumors
• Use of anticoagulant medication that cannot be safely withheld
• Bleeding disorders
• This is a partial listing.

Sponsors & Collaborators

• Massachusetts General Hospital: lead
• ALS Association: collaborator
• ALS Finding a Cure: collaborator

Study Sites (6)

Barrow Neurological Institute

Phoenix, Arizona, 85013, United States

Location

Mayo Clinic Jacksonville

Jacksonville, Florida, 32224, United States

Location

Emory University

Atlanta, Georgia, 30322, United States

Location

Massachusetts General Hospital

Boston, Massachusetts, 02114, United States

Location

University of Massachusetts Medical Center

Worcester, Massachusetts, 01655, United States

Location

University of Pittsburgh

Pittsburgh, Pennsylvania, 15213, United States

Location

Related Links

• Northeast ALS Consortium Website

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.

MeSH Terms

Conditions

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

Condition Hierarchy (Ancestors)

TDP-43 Proteinopathies; Proteostasis Deficiencies; Metabolic Diseases; Nutritional and Metabolic Diseases; Pathologic Processes; Pathological Conditions, Signs and Symptoms

Study Officials

• James D. Berry, MD, MPH

  Massachusetts General Hospital

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: observational
• Observational Model: CASE ONLY
• Time Perspective: PROSPECTIVE
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Principal Investigator

Study Record Dates

• First Submitted: December 16, 2011
• First Posted: December 20, 2011
• Study Start: November 1, 2011
• Primary Completion: December 31, 2016
• Study Completion: December 31, 2016
• Last Updated: May 30, 2017

Record last verified: 2017-05

Locations

US (6)