A Longitudinal Study of Amyotrophic Lateral Sclerosis (ALS) Biomarkers

Completed

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

• Registration Number: NCT01495390
• Lead Sponsor: 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.

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 Timeline

• Study Start: 2011-11
• Study First Submitted: 2011-12-16
• Study First Submitted QC: 2011-12-19
• Study First Posted: 2011-12-20
• Primary Completion: 2016-12-31
• Study Completion: 2016-12-31
• Status Verified: 2017-05
• Last Update Submitted: 2017-05-24
• Last Update Posted: 2017-05-30

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 139

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 & Design

• Study Type: OBSERVATIONAL
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
ALS Functional Rating Scale	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)	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)	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	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	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)	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.

Trial Locations

Locations (6)

Massachusetts General Hospital; 🇺🇸 Boston, Massachusetts, United States

University of Massachusetts Medical Center; 🇺🇸 Worcester, Massachusetts, United States

University of Pittsburgh; 🇺🇸 Pittsburgh, Pennsylvania, United States

Barrow Neurological Institute; 🇺🇸 Phoenix, Arizona, United States

Mayo Clinic Jacksonville; 🇺🇸 Jacksonville, Florida, United States

Emory University; 🇺🇸 Atlanta, Georgia, United States