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OpenTrials
Completed

NCT Number: NCT02574390

Answer ALS: Individualized Initiative for ALS Discovery

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

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Key information

Conditions

Amyotrophic Lateral Sclerosis Amyotrophy, monomelic Asymptomatic ALS Gene Carriers Central Nervous System Diseases Disease Attributes Disease Progression Flail Arm ALS Healthy Controls Metabolic Diseases Monomelic Amyotrophy Motor Neuron Disease Muscular Atrophy, Spinal Nervous System Diseases Neurodegenerative Diseases Neuromuscular Diseases Nutritional and Metabolic Diseases Pathologic Processes Pathological Conditions, Signs and Symptoms Primary Lateral Sclerosis Progressive Muscular Atrophy Proteostasis Deficiencies Spinal Cord Diseases TDP-43 Proteinopathies

Age range

18 year–100 year

Sex eligibility

All sexes

Study type

Observational

Primary location

Cedars-Sinai Medical Center, Los Angeles, California, United States

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About this study

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.

Who can participate

Healthy volunteers accepted: Yes

Only the study team can determine whether someone qualifies for participation.

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.

Treatment and study plan

Primary outcomes

  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.

Sponsors and collaborators

Lead sponsor

Johns Hopkins University

Other

Collaborators

  • Cedars-Sinai Medical Center
  • Emory University
  • Leandro P. Rizzuto Foundation
  • Massachusetts General Hospital
  • Massachusetts Institute of Technology
  • New York Genome Center
  • Northwestern University Les Turner ALS Center
  • Ohio State University
  • Texas Neurology
  • University of California, Irvine
  • Washington University School of Medicine

Registry information

Official study 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

Acronym: AnswerALS

Important dates

Study start
2015
Primary completion
2019
Study completion
2020
First posted
Oct 12, 2015
Registry last updated
Jan 13, 2020

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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