Fluid Biomarkers with Deep Phenotyping in Patients with ALS (ALSA-BIO3)
The goal of the study is to continue to build the Northeast ALS Consortium (NEALS) biorepository with this collection protocol to associate biofluid collection with specific measures of upper and lower motor neuron function.
Disease:Amyotrophic Lateral Sclerosis (ALS), Sporadic ALS
Study Type:Observational Study
Study Category:biological samples
Study Chair(s)/Principal Investigator(s):
Shafeeq Ladha, Barrow Neurological Institute
Clinicaltrials.gov ID (11 digit #):NCT02819765
Coordinating Center Contact InformationBarrow Neurological Institute
Full Study Summary:
The Northeast ALS Consortium (NEALS) biorepository is an existing resource which has provided scientists with a wide range of samples associated with clinical information. Our goal is to continue to build this repository with this collection protocol to associate biofluid collection with specific measures of upper and lower motor neuron function. This project will compliment others to upgrade the NEALS biorepository infrastructure, including updating the computer systems, expanding the number of sample freezers and establishing a repository in the Western United States, and strengthening the standard operating procedures for the repository. The project also supports existing efforts to expand the biorepository by collecting new blood and spinal fluid samples from people with ALS. This is a multicenter, non-interventional, longitudinal study in patients with ALS. There will be four (4) subject visits in this study: Baseline, month 6, month 12, and month 18. At each visit, subjects will have biofluids collected, and be evaluated with assessment tools that focus on upper and lower motor neuron burden as well as cognitive function.
The primary objective of the study is to obtain information on patients studied at regular intervals over 18 months, in conjunction with biofluid collection over that same time period. The secondary objective of the study is to integrate data from this study to other ongoing projects via the NEALS biorepository, and future collections now being planned. The outcome measures of this study are as follows:
• Motor unit number estimation will be performed on bilateral upper extremity muscles using the multipoint incremental technique (MIMNUE)
• Vital capacity, measured using slow vital capacity (SVC)
• Lower motor neuron excitability will be assessed with threshold tracking nerve conduction studies (ttNCS) in the least affected intrinsic hand muscle
• Cognitive abnormalities will be assessed using the ALS Cognitive Behavior Screen (ALS-CBS™)
• Upper motor neuron burden will be assessed using transcranial magnetic stimulation (TMS), employing a paired pulse protocol and recording from the least affected upper extremity intrinsic hand muscle
• Hand held dynamometry (HHD) will be performed on 9 muscle groups tested bilaterally
• Global function (ALSFRS-R)
At each visit, blood will be collected and banked for biomarker discovery; CSF will be collected at baseline, month 6, and at month 18.
Study Sponsor:The ALS Association and Fulton Family Foundation
4 visits over 18 months
Estimated Study Start Date:02/07/2017
Estimated Study Completion Date:07/01/2018
Posting Last Modified Date:01/14/2019
Date Study Added to alsconsortium.org:06/06/2016
In this informational webinar, Dr. Shafeeq Ladha reviews the BIO3 study design and answers questions about the study: https://www.neals.org/for-people-with-als-caregivers/educational-webinars/alsa-bio3-building-future-success-through-biomarkers
Time since Symptom Onset:
Time since Diagnosis:<24 months
Can participants use Riluzole?Yes
Expected to survive >1 year (12 months) after enrollment
Ability to medically undergo lumbar puncture (LP) as determined by the investigator (i.e., no bleeding disorder, allergy to local anesthetics, a skin infection at or near the LP site, or evidence of high intracranial pressure).
Willingness and medical ability to comply with scheduled visits, LP for CSF collection, laboratory tests, and other study procedures.
Ability to understand the purpose and risks of the study and provide signed and dated informed consent and authorization to use protected health information (PHI) in accordance with national and local subject privacy regulations.
Geographic accessibility to the study site.
Site Contact Information
Barrow Neurological Institute
Phoenix, Arizona 85013
University of California, Irvine
Dept. of Neurology
Orange, California 92868
Grand Rapids, Michigan 49503
Hospital for Special Surgery (HSS)
Mona Shahbazi / 212-774-2361
New York, New York 10021
Oregon Health & Science University
Portland, Oregon 97239
University of Pittsburgh Medical Center
Pittsburgh, Pennsylvania 15213
Philadelphia, Pennsylvania 19122
Medical University of South Carolina
Department of Neurosurgery & Neurology
Charleston, South Carolina 29425