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Answer ALS: Individualized Initiative for ALS Discovery (AnswerALS)

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
ClinicalTrials.gov Identifier: NCT02574390
Recruitment Status : Active, not recruiting
First Posted : October 12, 2015
Last Update Posted : February 5, 2019
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

Tracking Information
First Submitted Date October 9, 2015
First Posted Date October 12, 2015
Last Update Posted Date February 5, 2019
Study Start Date December 2015
Actual Primary Completion Date January 2019   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures
 (submitted: January 24, 2018)
  • 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.
  • 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.
  • 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.
  • 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.
Original Primary Outcome Measures
 (submitted: October 9, 2015)
  • 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
  • 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.
  • 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.
  • 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.
Change History Complete list of historical versions of study NCT02574390 on ClinicalTrials.gov Archive Site
Current Secondary Outcome Measures Not Provided
Original Secondary Outcome Measures Not Provided
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title Answer ALS: Individualized Initiative for ALS Discovery
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
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
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]
Study Design Observational Model: Case-Only
Time Perspective: Prospective
Target Follow-Up Duration 1 Year
Biospecimen Retention:   Samples With DNA
Description:
plasma, serum, DNA, Cerebrospinal fluid, induced pluripotent stem cells,
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
Condition
  • Amyotrophic Lateral Sclerosis
  • Primary Lateral Sclerosis
  • Flail Arm ALS
  • Progressive Muscular Atrophy
  • Monomelic Amyotrophy
  • Motor Neuron Disease
  • Asymptomatic ALS Gene Carriers
  • Healthy Controls
Intervention Not Provided
Study Groups/Cohorts Not Provided
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status Active, not recruiting
Estimated Enrollment
 (submitted: October 9, 2015)
1000
Original Estimated Enrollment Same as current
Estimated Study Completion Date January 2020
Actual Primary Completion Date January 2019   (Final data collection date for primary outcome measure)
Eligibility 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.
Sex/Gender
Sexes Eligible for Study: All
Ages 18 Years to 100 Years   (Adult, Older Adult)
Accepts Healthy Volunteers Yes
Contacts Contact information is only displayed when the study is recruiting subjects
Listed Location Countries United States
Removed Location Countries  
 
Administrative Information
NCT Number NCT02574390
Other Study ID Numbers IRB00082277
Has Data Monitoring Committee No
U.S. FDA-regulated Product Not Provided
IPD Sharing Statement Not Provided
Responsible Party Johns Hopkins University
Study Sponsor Johns Hopkins University
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
Investigators
Principal Investigator: Nicholas J Maragakis, MD Johns Hopkins University
Principal Investigator: James D Berry, MD Massachusetts Generel Hospital
PRS Account Johns Hopkins University
Verification Date February 2019