Clinical trial NCT02574390: 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
Study type: OBSERVATIONAL
Overall status: COMPLETED
Conditions: Amyotrophic Lateral Sclerosis
Primary Lateral Sclerosis
Flail Arm ALS
Progressive Muscular Atrophy
Monomelic Amyotrophy
Motor Neuron Disease
Asymptomatic ALS Gene Carriers
Healthy Controls
Keywords: ALS
stem cell
biomarker
disease progression
Lead sponsor: Johns Hopkins University
Sponsor class: OTHER
Collaborators: name: Massachusetts General Hospital
name: Emory University
name: Ohio State University
name: Washington University School of Medicine
name: Cedars-Sinai Medical Center
name: University of California, Irvine
name: Massachusetts Institute of Technology
name: New York Genome Center
name: Leandro P. Rizzuto Foundation
name: Texas Neurology
name: Northwestern University Les Turner ALS Center
First posted: 2015-10-12
Enrollment: 1049
Sex: ALL
Minimum age (years): 18
Maximum age (years): 100
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: yes
Observational model: CASE_ONLY
Time perspective: PROSPECTIVE
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

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.

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.

Primary outcomes:
measure: ALS Functional Rating Scale-Revised (ALSFRS-R)
timeFrame: once every 3 months for one year
measure: ALS Cognitive Behavioral Scale (ALS-CBS)
timeFrame: once every 3 months for one year
measure: Slow Vital Capacity (SVC)
timeFrame: once every 3 months for one year
measure: Strength Testing with Hand Held Dynamometer (HHD)
timeFrame: once every 3 months for one year
Secondary outcomes:
Other outcomes:
Locations:
city: Los Angeles
country: United States
facility: Cedars-Sinai Medical Center
state: California
city: Atlanta
country: United States
facility: Emory University
state: Georgia
city: Chicago
country: United States
facility: Northwestern University Feinberg School of Medicine
state: Illinois
city: Baltimore
country: United States
facility: Johns Hopkins University
state: Maryland
city: Boston
country: United States
facility: Massachusetts General Hospital
state: Massachusetts
city: Saint Louis
country: United States
facility: Washington University School of Medicine
state: Missouri
city: Columbus
country: United States
facility: Ohio State University Wexner Medical Center
state: Ohio
city: Dallas
country: United States
facility: Texas Neurology
state: Texas
Study officials:
affiliation: Johns Hopkins University
name: Nicholas J Maragakis, MD
role: PRINCIPAL_INVESTIGATOR
affiliation: Massachusetts Generel Hospital
name: James D Berry, MD
role: PRINCIPAL_INVESTIGATOR
References: