Answer ALS: Individualized Initiative for ALS Discovery (AnswerALS)

Johns Hopkins University

Status: Completed

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

Study type: Observational

Funder types: Other

Identifiers

NCT02574390

IRB00082277

Details and patient eligibility

About

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

Full 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.

Enrollment: 1,049 patients

Sex: All

Ages: 18 to 100 years old

Volunteers: Accepts Healthy Volunteers

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.