Answer ALS: Individualized Initiative for ALS Discovery

Completed

• Conditions: Progressive Muscular Atrophy; Flail Arm ALS; Healthy Controls; Motor Neuron Disease; Amyotrophic Lateral Sclerosis; Monomelic Amyotrophy; Asymptomatic ALS Gene Carriers; Primary Lateral Sclerosis

• Registration Number: NCT02574390
• Lead Sponsor: 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

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 Timeline

• Study First Submitted: 2015-10-09
• Study First Submitted QC: 2015-10-09
• Study First Posted: 2015-10-12
• Study Start: 2015-12
• Primary Completion: 2019-12-31
• Status Verified: 2020-01
• Study Completion: 2020-01-01
• Last Update Submitted: 2020-01-09
• Last Update Posted: 2020-01-13

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 1049

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.

Study & Design

• Study Type: OBSERVATIONAL
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
ALS Functional Rating Scale-Revised (ALSFRS-R)	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)	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)	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)	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.

Trial Locations

Locations (8)

Texas Neurology; 🇺🇸 Dallas, Texas, United States

Cedars-Sinai Medical Center; 🇺🇸 Los Angeles, California, United States

Emory University; 🇺🇸 Atlanta, Georgia, United States

Northwestern University Feinberg School of Medicine; 🇺🇸 Chicago, Illinois, United States

Washington University School of Medicine; 🇺🇸 Saint Louis, Missouri, United States

Johns Hopkins University; 🇺🇸 Baltimore, Maryland, United States

Ohio State University Wexner Medical Center; 🇺🇸 Columbus, Ohio, United States

Massachusetts General Hospital; 🇺🇸 Boston, Massachusetts, United States