A Longitudinal Study of Amyotrophic Lateral Sclerosis (ALS) Biomarkers - Full Text View

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:

Genetics Home Reference related topics: amyotrophic lateral sclerosis

MedlinePlus related topics: Amyotrophic Lateral Sclerosis Neurologic Diseases Neuromuscular Disorders Spinal Cord Diseases

Genetic and Rare Diseases Information Center resources: Amyotrophic Lateral Sclerosis

U.S. FDA Resources

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 2016
Estimated Primary Completion Date:	December 2016 (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 (Adult, Senior)
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: Daniela Grasso	617-726-0842	dgrasso@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: Amelia Robertson 904-953-9498 robertson.amelia@mayo.edu
Principal Investigator: Kevin Boylan, MD
United States, Georgia
Emory University	Recruiting
Atlanta, Georgia, United States, 30322
Contact: Meraida Polak 404-778-3807 mpolak@emory.edu
Principal Investigator: Jonathan Glass, MD
United States, Massachusetts
Massachusetts General Hospital	Recruiting
Boston, Massachusetts, United States, 02114
Contact: Leah Miller 617-724-7398 lmiller33@mgh.harvard.edu
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 Association

ALS Finding a Cure

Investigators


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

More Information

Additional Information:

Northeast ALS Consortium Website This link exits the ClinicalTrials.gov site


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:	June 2, 2016
Health Authority:	United States: Institutional Review Board
Individual Participant Data
Plan to Share IPD:	Undecided

Keywords provided by Massachusetts General Hospital:


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

Additional relevant MeSH terms:


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

ClinicalTrials.gov processed this record on September 29, 2016