A Longitudinal Study of Amyotrophic Lateral Sclerosis (ALS) Biomarkers

• ClinicalTrials.gov Identifier: NCT01495390
• Recruitment Status: Completed
• First Posted: December 20, 2011
• Last Update Posted: May 30, 2017
• Sponsor: Massachusetts General Hospital
• Collaborators: ALS Association; ALS Finding a Cure
• Information provided by (Responsible Party): James D. Berry MD, Massachusetts General Hospital

Tracking Information

• First Submitted Date: December 16, 2011
• First Posted Date: December 20, 2011
• Last Update Posted Date: May 30, 2017
• Study Start Date: November 2011
• Actual Primary Completion Date: December 31, 2016 (Final data collection date for primary outcome measure)

Current Primary Outcome Measures (submitted: April 14, 2015)

• 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.
• 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.
• 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.
• 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.
• 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.
• 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.

• Original Primary Outcome Measures: Not Provided
• Current Secondary Outcome Measures: Not Provided
• Original Secondary Outcome Measures: Not Provided
• Current Other Pre-specified Outcome Measures: Not Provided
• Original Other Pre-specified Outcome Measures: Not Provided

Descriptive Information

• Brief Title: A Longitudinal Study of Amyotrophic Lateral Sclerosis (ALS) Biomarkers
• Official Title: A Multicenter Study for the Discovery and Validation of ALS Biomarkers
• 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.
• 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
• Study Design: Observational Model: Case-Only; Time Perspective: Prospective
• Target Follow-Up Duration: Not Provided

Biospecimen

Retention:   Samples With DNA

Description:

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.

• 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

Condition

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

• Intervention: Not Provided
• Study Groups/Cohorts: Not Provided
• Publications *: Not Provided

Recruitment Information

• Recruitment Status: Completed
• Actual Enrollment (submitted: May 24, 2017): 139
• Original Estimated Enrollment (submitted: December 19, 2011): 250
• Actual Study Completion Date: December 31, 2016
• Actual Primary Completion Date: December 31, 2016 (Final data collection date for primary outcome measure)

Eligibility 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.

Sex/Gender

• Sexes Eligible for Study: All

• Ages: 18 Years and older (Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Contacts: Contact information is only displayed when the study is recruiting subjects
• Listed Location Countries: United States

Administrative Information

• NCT Number: NCT01495390
• Other Study ID Numbers: BIO-ALS-02
• Has Data Monitoring Committee: Yes
• U.S. FDA-regulated Product: Not Provided

IPD Sharing Statement

• Plan to Share IPD: Undecided

• Current Responsible Party: James D. Berry MD, Massachusetts General Hospital
• Original Responsible Party: Merit E. Cudkowicz, MD, Massachusetts General Hospital, Principal Investigator
• Current Study Sponsor: Massachusetts General Hospital
• Original Study Sponsor: Same as current

Collaborators

• ALS Association
• ALS Finding a Cure

Investigators

• Principal Investigator: James D. Berry, MD, MPH; Massachusetts General Hospital

• PRS Account: Massachusetts General Hospital
• Verification Date: May 2017