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Clinical Trials

Clinical trials are health-related studies in people that are closely supervised and carefully follow a pre-defined protocol. Each study answers scientific questions and tries to find better ways to prevent, screen for, diagnose or treat a disease. Clinical trials may be done to see how a new treatment compares with the standard  Dr. Khan, Meraida Polak, Questcor_Acthar kickoff meetingtreatment or to see if a new treatment is safe and effective for a certain condition or disease. Participation in this type of research involves treatment with an experimental treatment or medical device, which may or may not provide a direct benefit to the individual. A clinical trial must be conducted for all new treatments before the FDA will approve the treatment for the public. The Emory ALS Center is actively engaged in research projects involving clinical trials of new medications to slow the progression of ALS. Search for clinical trials

ALS patients have never had as many opportunities to participate in research projects as they have today.

Open and recruiting:

Multiple Dose Study of BIIB067 (Isis-SOD1Rx) in Adults with ALS who have SOD1 mutation, sponsored by Biogen.  See NCT02623699 Contact Anna Partlow (404-778-3181), 

Multiple-Ascending-Dose Study of GDC-0134 to Determine Initial Safety, Tolerability, and Pharmacokinetic Parameters in Patients with ALS.  See NCT02655614 Contact Jane Bordeau (404-727-1679), 


  • Probably in early 2017, is a study with Amylyx pharmaceuticals.  More info can be found HERE.  
  • Also in mid to late 2017 we expect to have several other new drug studies hoping to change the course of ALS.
  • We are also working with industry on a new medication for cramps.

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Stem Cell Trial: 

We are in between the completion of Phase II and the next phase of the trial. The next phase will be directed at evaluating the ability of stem cell transplantation to slow the course of ALS. Trial updates will be posted to the Emory ALS Center website.  We are not currently recruiting, nor do we know all of the eligibility criteria.