Answer ALS: Individualized Initiative for ALS Discovery

Status:	Active, not recruiting
Conditions:	Healthy Studies, Neurology
Therapuetic Areas:	Neurology, Other
Healthy:	No
Age Range:	18 - 100
Updated:	2/7/2019
Start Date:	December 2015
End Date:	January 2020

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

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

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.

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.

We found this trial at

sites

Massachusetts General Hospital

185 Cambridge Street
Boston, Massachusetts 02114

617-724-5200