Answer ALS: Individualized Initiative for ALS Discovery

Amyotrophic Lateral Sclerosis Clinical Trial

— AnswerALS  

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

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT02574390
• Other study ID #: IRB00082277
• Status: Completed
• Start date: December 2015
• Est. completion date: January 1, 2020

Study information

• Verified date: January 2020
• Source: Johns Hopkins University
• Contact: n/a
• Is FDA regulated: No
• Study type: Observational [Patient Registry]

Clinical Trial 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

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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 1049
• Est. completion date: January 1, 2020
• Est. primary completion date: December 31, 2019
• Accepts healthy volunteers: Accepts Healthy Volunteers
• Gender: All
• Age group: 18 Years to 100 Years

Eligibility

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.

Related Conditions & MeSH terms

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

Locations

Country	Name	City	State
United States	Emory University	Atlanta	Georgia
United States	Johns Hopkins University	Baltimore	Maryland
United States	Massachusetts General Hospital	Boston	Massachusetts
United States	Northwestern University Feinberg School of Medicine	Chicago	Illinois
United States	Ohio State University Wexner Medical Center	Columbus	Ohio
United States	Texas Neurology	Dallas	Texas
United States	Cedars-Sinai Medical Center	Los Angeles	California
United States	Washington University School of Medicine	Saint Louis	Missouri

Sponsors (12)

Lead Sponsor	Collaborator
Johns Hopkins University	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

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	ALS Functional Rating Scale-Revised (ALSFRS-R)	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.	once every 3 months for one year
Primary	ALS Cognitive Behavioral Scale (ALS-CBS)	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.	once every 3 months for one year
Primary	Slow Vital Capacity (SVC)	measurement of the maximum amount of air that can be exhaled following a deep breath.	once every 3 months for one year
Primary	Strength Testing with Hand Held Dynamometer (HHD)	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.	once every 3 months for one year