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Deep Phenotyping in Patients With ALS

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ClinicalTrials.gov Identifier: NCT02819765
Recruitment Status : Recruiting
First Posted : June 30, 2016
Last Update Posted : January 8, 2019
Sponsor:
Collaborator:
ALS Association
Information provided by (Responsible Party):
Barrow Neurological Institute

Brief Summary:
This study aims to establish a biorepository and phenotyping database to investigate longitudinal changes in ALS subjects. Blood, including DNA and RNA, cerebrospinal fluid (CSF) and electrophysiologic measures will be collected every 6 months over 1 and a half years. The database and specimen repository will be made available to ALS researchers on a merit basis.

Condition or disease
ALS Motor Neuron Disease

  Show Detailed Description

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Study Type : Observational
Estimated Enrollment : 50 participants
Observational Model: Case-Only
Time Perspective: Prospective
Official Title: Fluid Biomarkers With Deep Phenotyping in Patients With ALS
Actual Study Start Date : February 7, 2017
Estimated Primary Completion Date : July 2020
Estimated Study Completion Date : December 2020





Primary Outcome Measures :
  1. ALS Functional Rating Scale Revised (ALSFRS-R) [ Time Frame: Every 6 months through month 18 ]
    The ALSFRS-R is a quickly administered (5 minutes) instrument used to determine subjects' assessment of their capability and independence in 12 functional activities, each rated on an ordinal scale (ratings 0-4).

  2. Slow vital capacity (SVC) [ Time Frame: Every 6 months through month 18 ]
    Vital capacity will be measured as slow vital capacity (SVC) using standard technique

  3. Hand held dynamometry (HHD) [ Time Frame: Every 6 months through month 18 ]
    Hand held dynamometry (HHD) will be used as a quantitative measure of muscle strength for this study. 10 muscle groups will be examined bilaterally in both upper and lower extremities. Mean and standard deviation for each muscle group will be established from the initial values for each subject in this trial, so that strength determinations can be converted to Z scores and averaged to provide an HHD megascore for both upper and lower extremities.

  4. ALS cognitive behavioral screen (ALS-CBS) [ Time Frame: Every 6 months through month 18 ]
    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.

  5. Multipoint incremental motor unit number estimates (MIMUNE) [ Time Frame: Every 6 months through month 18 ]
    MIMUNE is a validated technique to estimate the number of motor units in specific muscles of patients. In this study it will be performed on one intrinsic hand muscle unilaterally throughout the study.

  6. Paired Pulse Stimulation Using Transcranial Magnetic Stimulation (TMS) [ Time Frame: Every 6 months through month 18 ]
    TMS is a technique that uses a focused magnetic field to depolarize neurons of interest. By depolarizing motor neurons and measuring the evoked response, an assessment of the neuronal excitability can be determined.

  7. Threshold tracking nerve conduction studies (ttNCS) [ Time Frame: Every 6 months through month 18 ]
    ttNCS is a technique that uses stimulation of peripheral motor nerves to measure a variety of variables to determine the excitability of the peripheral motor neurons.


Other Outcome Measures:
  1. MRI Cytography [ Time Frame: At baseline and at 6 months ]
    The MRI scan is a non-invasive procedure that uses a powerful magnetic field and radio waves to obtain images of internal organs and tissues.


Biospecimen Retention:   Samples With DNA
Blood Serum Cerebrospinal fluid RNA DNA


Information from the National Library of Medicine

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Ages Eligible for Study:   18 Years to 75 Years   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Sampling Method:   Non-Probability Sample
Study Population
ALS patient 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:

  • Participants with sporadic ALS diagnosed as possible, laboratory-supported probable, probable or definite according to the World Federation of Neurology El Escorial criteria.
  • Expected to survive >1 year (12 months) after enrollment.
  • Male or female, aged 18-75.
  • Ability to medically undergo lumbar puncture (LP) as determined by the investigator (i.e., no bleeding disorder, allergy to local anesthetics, prior lumbar surgery which might make LP difficult, 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.

Exclusion Criteria:

  • Any contraindications to having an LP, including but not limited to: Platelet count <100,000/µL.
  • History of bleeding disorder.
  • History of intolerance to the LP procedure.
  • Evidence of topical or other skin infection at the LP site.
  • History of allergy or other adverse reaction to local anesthetics used in the study.
  • History of traumatic central nervous system injury or stroke.
  • Other unspecified reasons that, in the opinion of the Investigator, make the subject unsuitable for enrollment.

Additional criteria for sites performing TMS:

  • Inability to perform either TMS or NCS studies due to insufficient motor evoked potential (MEP) or compound muscle action potential (CMAP) amplitude.
  • Contraindication to TMS studies including ferromagnetic metal in the head or neck (potentially found in aneurysm clips, implanted medication pumps, implanted brain stimulators, pacemakers, cochlear implants), or history of epilepsy. Dental fillings are permitted.

Additional criteria for sites performing MRI cytography:

  • Subjects who have a history of claustrophobia that cannot be adequately controlled.
  • Subjects who have a physical limitation related to fitting in the bore of the magnet.
  • Subjects who have a history of allergic reaction to contrast agents.
  • Subjects with a pacemaker, epicardia pacemaker wires, MRI-incompatible cardiac valve prostheses, MRI-incompatible vascular clips.
  • Subjects with MRI-incompatible cochlear implants.
  • Subjects with spinal nerve simulators.
  • Subjects with an infusion pump.
  • Subjects with metallic fragments in the eyes/orbits or in the vicinity of the brain or major neurovascular structures of the body, subjects with an employment history which involves exposure to welding, or subjects who have shrapnel any place in their body.
  • Subjects with acute kidney injury or renal insufficiency (eGFR of <20 ml/min/1.73 m^2) as they are at increased risk of Nephrogenic Systemic Fibrosis following administration of gadolinium-based MRI contrast agents.
  • Subjects unable to lay supine in the magnet because of orthopnea.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02819765


Contacts
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Contact: Kerisa Shelton, PhD 602-406-6598 Kerisa.Shelton@DignityHealth.org
Contact: Meghan Hall Meghan.Hall@DignityHealth.org

Locations
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United States, Arizona
Barrow Neurological Institute Recruiting
Phoenix, Arizona, United States, 85013
Contact: Nicole Turcotte, MPH    602-406-4775    nicole.turcotte@dignityhealth.org   
Principal Investigator: Shafeeq S Ladha, MD         
United States, California
University of California, Irvine, Dept. of Neurology Recruiting
Orange, California, United States, 92868
Contact: Ivonne M Turner    714-456-8323    imturner@uci.edu   
Principal Investigator: Namita Goyal, MD         
Sub-Investigator: Tahseen Mozaffar, MD         
Sub-Investigator: Tim Lai, MD         
Sub-Investigator: Monica Lavian, DO         
United States, Michigan
University of Michigan | Neurology - ALS Clinic Suspended
Ann Arbor, Michigan, United States, 48109
Spectrum Health Recruiting
Grand Rapids, Michigan, United States, 49503
Contact: Brandon James    616-486-2058    Brandon.James@spectrumhealth.org   
Principal Investigator: Paul T Twydell, DO         
United States, New York
Hospital for Special Surgery (HSS) Recruiting
New York, New York, United States, 10021
Contact: Mona Shahbazi    212-774-2361      
Principal Investigator: Dale Lange, MD         
United States, Oregon
Oregon Health & Science University Recruiting
Portland, Oregon, United States, 97239
Contact: Diana Dimitrova, PhD    503-494-7269    dimitrov@ohsu.edu   
Principal Investigator: Chafic Karam, MD         
United States, Pennsylvania
Temple University Recruiting
Philadelphia, Pennsylvania, United States, 19122
Contact: Kathleen Hatala, RN, BSN    215-707-4171    kathleen.hatala@tuhs.temple.edu   
Principal Investigator: Terry Heiman-Patterson, MD         
University of Pittsburgh Medical Center Recruiting
Pittsburgh, Pennsylvania, United States, 15213
Contact: Danielle Rowlands    412-864-2873    doerdx@upmc.edu   
Principal Investigator: David Lacomis, MD         
United States, South Carolina
Department of Neurosurgery & Neurology | Medical University of South Carolina Recruiting
Charleston, South Carolina, United States, 29425
Contact: Michelle DeCandio, RN, OCN    843-792-9016    decandio@musc.edu   
Contact: Katrina Madden    843-792-9186    maddenka@musc.edu   
Principal Investigator: I-Hweii Amy Chen, MD, PhD         
Sponsors and Collaborators
Barrow Neurological Institute
ALS Association
Investigators
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Principal Investigator: Shafeeq Ladha, MD Barrow Neurological Institute

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Responsible Party: Barrow Neurological Institute
ClinicalTrials.gov Identifier: NCT02819765     History of Changes
Other Study ID Numbers: ALSA-BIO3
PHX16BN016 ( Other Identifier: Barrow Neurological Institute )
First Posted: June 30, 2016    Key Record Dates
Last Update Posted: January 8, 2019
Last Verified: January 2019
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: Yes
Plan Description: The data will be part of the Northeast ALS consortium biorepository database.

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Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No

Additional relevant MeSH terms:
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Motor Neuron Disease
Amyotrophic Lateral Sclerosis
Neurodegenerative Diseases
Nervous System Diseases
Neuromuscular Diseases
Spinal Cord Diseases
Central Nervous System Diseases
TDP-43 Proteinopathies
Proteostasis Deficiencies
Metabolic Diseases