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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 : April 21, 2017
Information provided by (Responsible Party):

Study Description
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 Design

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

Groups and Cohorts

Outcome Measures

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

  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.

  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.

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

Eligibility Criteria

Information from the National Library of Medicine

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Ages Eligible for Study:   18 Years to 100 Years   (Adult, Senior)
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

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.
Contacts and Locations

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

Contact: Aiana Rodriguez, BS 443-287-0627 arodri47@jhmi.edu
Contact: Betsy Mosmiller, BA 410-502-0495 emosmil1@jhmi.edu

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: Meriada Polak, RN    404-779-3807    mpolak@emory.edu   
Contact: Anna Partlow, RN, MN    404-778-3181    anna.partlow@emory.edu   
Principal Investigator: Jonathan Glass, MD         
United States, Maryland
Johns Hopkins University Recruiting
Baltimore, Maryland, United States, 21205
Contact: Betsy Mosmiller    410-502-0495    emosmil1@jhmi.edu   
Contact: Aiana Rodriguez    443-287-0627    arodri47@jhmi.edu   
United States, Massachusetts
Massachusetts General Hospital Recruiting
Boston, Massachusetts, United States, 02114
Contact: Leah Miller    617-724-7398    LMILLER33@mgh.harvard.edu   
Contact: Maryangel Jeon    617-724-9196    mjeon2@partners.org   
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         
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
Principal Investigator: Nicholas J Maragakis, MD Johns Hopkins University
Principal Investigator: James D Berry, MD Massachusetts Generel Hospital
More Information

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: April 21, 2017
Last Verified: April 2017

Keywords provided by Johns Hopkins University:
stem cell
disease progression

Additional relevant MeSH terms:
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