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Answer ALS: Individualized Initiative for ALS Discovery (AnswerALS)

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ClinicalTrials.gov Identifier: NCT02574390
Recruitment Status : Recruiting
First Posted : October 12, 2015
Last Update Posted : May 15, 2018
Sponsor:
Collaborators:
Massachusetts General Hospital
Emory University
Ohio State University
Washington University School of Medicine
Cedars-Sinai Medical Center
University of California, Irvine
Massachusetts Institute of Technology
New York Genome Center
Leandro P. Rizzuto Foundation
Texas Neurology
Northwestern University Les Turner ALS Center
Information provided by (Responsible Party):
Johns Hopkins University

Brief Summary:
Creation of a large repository of induced pluripotent stem cells (iPSC), bio-fluid samples (blood and spinal fluid (optional)), and cell lines for ALS gene identification. This will be combined carefully with collected measures of the pattern of the symptoms people with ALS have and how these change over time. People with other motor neuron diseases and healthy controls will be included as comparisons

Condition or disease
Amyotrophic Lateral Sclerosis Primary Lateral Sclerosis Flail Arm ALS Progressive Muscular Atrophy Monomelic Amyotrophy Motor Neuron Disease Asymptomatic ALS Gene Carriers Healthy Controls

Detailed Description:
Patients will have 5 study visits; screening, 3, 6, 9 and 12 months. There will be a one year post-participation follow-up period, during which they will receive an email or phone call interview once every 3 months. During the first year, samples will be collected, breathing, muscle strength, spasticity, general function and cognitive behavior will be assessed. Healthy controls will have 2 study visits during which blood samples will be collected and questionnaires given.

Study Type : Observational [Patient Registry]
Estimated Enrollment : 1000 participants
Observational Model: Case-Only
Time Perspective: Prospective
Target Follow-Up Duration: 1 Year
Official Title: Answer ALS -Creation of a Large Bio-repository of iPS Cells, Cell Lines, and Bio-fluid Samples, Combined With Clinical Information to Rapidly Advance Therapeutics That Could Treat ALS
Study Start Date : December 2015
Estimated Primary Completion Date : January 2019
Estimated Study Completion Date : July 2019





Primary Outcome Measures :
  1. ALS Functional Rating Scale-Revised (ALSFRS-R) [ Time Frame: once every 3 months for one year ]
    12 questions about patient's ability to function in certain activities of daily living. Each question is out of 4 with 4 being normal and 0 being completely impaired.

  2. ALS Cognitive Behavioral Scale (ALS-CBS) [ Time Frame: once every 3 months for one year ]
    short measure of cognition and behavior in patients with ALS. The cognitive portion consists of 8 tasks with a perfect score being 20. The behavioral portion measures changes in personality and behavior since the onset of ALS symptoms as well as mood, pseudobulbar affect and fatigue and is completed by a family member or caregiver. A normal score is 45.

  3. Slow Vital Capacity (SVC) [ Time Frame: once every 3 months for one year ]
    measurement of the maximum amount of air that can be exhaled following a deep breath.

  4. Strength Testing with Hand Held Dynamometer (HHD) [ Time Frame: once every 3 months for one year ]
    muscle strength testing performed on upper and lower limbs, ankles, wrists and fingers using a small hand held device. These measurements are followed over time and compared to measure decline.


Biospecimen Retention:   Samples With DNA
plasma, serum, DNA, Cerebrospinal fluid, induced pluripotent stem cells,


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Ages Eligible for Study:   18 Years to 100 Years   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   Yes
Sampling Method:   Non-Probability Sample
Study Population
patients with ALS, Primary Lateral Sclerosis Flail Arm ALS Progressive Muscular Atrophy Monomelic Amyotrophy Motor Neuron Disease Asymptomatic ALS Gene Carriers
Criteria

Inclusion Criteria:

  • Participants with familial or sporadic ALS diagnosed as possible, laboratory-supported probable, probable or definite according to the World Federation of Neurology (WFN) El Escorial criteria, Primary Lateral Sclerosis Flail Arm ALS, Progressive Muscular Atrophy, Monomelic Amyotrophy, Motor Neuron Disease, Asymptomatic ALS Gene Carriers
  • Participants who are ages 18-100, inclusive.

Exclusion Criteria:

  • Participants with Spinal-Bulbar Muscular Atrophy
  • Known diagnosis of HIV/AIDS, Hepatitis B, or Hepatitis C.

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): NCT02574390


Contacts
Contact: Betsy Mosmiller, BA 410-502-0495 emosmil1@jhmi.edu

Locations
United States, California
Cedars-Sinai Medical Center Recruiting
Los Angeles, California, United States, 90048
Contact: Peggy Allred    424-315-2694    Peggy.Allred@cshs.org   
Contact: Dana Fine    310-423-8497    dana.fine@cshs.org   
Principal Investigator: Robert H. Baloh, MD, PhD         
United States, Georgia
Emory University Recruiting
Atlanta, Georgia, United States, 30322
Contact: Arish Jamil    404-727-1273    arish.jamil@emory.edu   
Contact: Meraida Polack, RN    404-779-3807    mpolack@emory.edu   
Principal Investigator: Jonathan Glass, MD         
United States, Illinois
Northwestern University Feinberg School of Medicine Recruiting
Chicago, Illinois, United States, 60611
Contact: Benjamin Joslin    312-503-7504    ben.joslin@northwestern.edu   
Contact: Patricia Casey    312-503-7504    patricia-casey@northwestern.edu   
United States, Maryland
Johns Hopkins University Recruiting
Baltimore, Maryland, United States, 21205
Contact: Betsy Mosmiller    410-502-0495    emosmil1@jhmi.edu   
United States, Massachusetts
Massachusetts General Hospital Recruiting
Boston, Massachusetts, United States, 02114
Contact: Brixhilda Dedi    617-726-4284    bdedi@mgh.harvard.edu   
Contact: Jacqueline Andrews    617-726-0981    jandrews9@mgh.harvard.edu   
Principal Investigator: James D Berry, MD         
United States, Missouri
Washington University School of Medicine Recruiting
Saint Louis, Missouri, United States, 63110
Contact: Jesse Markway    314-747-7881    neuroclinicalstudies@neuro.wustl.edu   
Contact: Jennifer Jockel-Balsarotti    314-362-6159    neuroclinicalstudies@neuro.wustl.edu   
Principal Investigator: Timothy Miller, MD         
United States, Ohio
Ohio State University Wexner Medical Center Recruiting
Columbus, Ohio, United States, 43221
Contact: Ifeanyi Okoh    614-688-7837    ifeanyi.okoh@osumc.edu   
Contact: Sharon Chelnick, MHSA, CCRC    614-293-4973    sharon.chelnick@osumc.edu   
Principal Investigator: Stephen J Kolb, MD, PhD         
United States, Texas
Texas Neurology Recruiting
Dallas, Texas, United States, 75214
Contact: Todd Morgan    214-827-3610 ext 228    tmorgan@texasneurology.com   
Principal Investigator: Daragh Heitzman, MD         
Sponsors and Collaborators
Johns Hopkins University
Massachusetts General Hospital
Emory University
Ohio State University
Washington University School of Medicine
Cedars-Sinai Medical Center
University of California, Irvine
Massachusetts Institute of Technology
New York Genome Center
Leandro P. Rizzuto Foundation
Texas Neurology
Northwestern University Les Turner ALS Center
Investigators
Principal Investigator: Nicholas J Maragakis, MD Johns Hopkins University
Principal Investigator: James D Berry, MD Massachusetts Generel Hospital

Responsible Party: Johns Hopkins University
ClinicalTrials.gov Identifier: NCT02574390     History of Changes
Other Study ID Numbers: IRB00082277
First Posted: October 12, 2015    Key Record Dates
Last Update Posted: May 15, 2018
Last Verified: May 2018

Keywords provided by Johns Hopkins University:
ALS
stem cell
biomarker
disease progression

Additional relevant MeSH terms:
Sclerosis
Atrophy
Motor Neuron Disease
Amyotrophic Lateral Sclerosis
Muscular Atrophy
Muscular Atrophy, Spinal
Pathologic Processes
Pathological Conditions, Anatomical
Neurodegenerative Diseases
Nervous System Diseases
Neuromuscular Diseases
Spinal Cord Diseases
Central Nervous System Diseases
TDP-43 Proteinopathies
Proteostasis Deficiencies
Metabolic Diseases
Neuromuscular Manifestations
Neurologic Manifestations
Signs and Symptoms