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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  clinical trialstreatment 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.

Clinical Trial Opportunities: (treatment with an experimental drug)

Acthar: ENROLLING--This phase 2 placebo-controlled trial sponsored by Mallinckrodt will test the safety, tolerability, and pharmacokinetics of an investigational injectable drug in patients with ALS. Participants will have approximately 12 study visits (participation in the open label extension will add an additional 5 visits) and a site phone call.  For information about this study, contact Meraida Polak at 404-778-3807 or

Biogen SOD1 study:  LIMITED ENROLLMENT--The VALOR Study is evaluating the safety and potential efficacy of an investigational drug for people living with ALS caused by a SOD1 gene mutation.  All participants will receive either the investigational drug or placebo (inactive drug), and all study related visits and care, at no cost.  Assistance with travel, accommodations, and reimbursement for study related expenses may be available.  For more information contact Meraida Polak at 404-778-3807 or

OrionPharma Trial of Levosimendan in ALS (RefALS): ENROLLING -- OrionPharma is sponsoring a Phase 3 study of the drug levosimendan in people with ALS. Levosimendan is an FDA-approved medication for heart failure that acts by causing the heart muscle to beat more strongly. The hope is that the drug, when given to people with diaphragmatic weakness secondary to ALS, will strengthen the action of the diaphragm and result in improvement in breathing. Eligibility criteria include having had symptoms of ALS for between 12-48 months, ability to swallow study drug, and vital capacity between 60-90%. Use of Trilogy is not permitted, nor is edaravone and certain other drugs. People with certain cardiac diseases and/or COPD may not participate.  The trial will run over 48 weeks and require 8 visits to the site plus follow-up telephone calls. For further information please contact Jane Bordeau at 404-727-1679 or

Genentech GDC-0134:  NO LONGER ENROLLING--This is a first-in-human study to examine whether the drug GDC-0134 is safe and tolerable in people with ALS.  Subject groups will start at a low dose of drug and subsequent groups will be given higher doses of drug if safety of the prior dose is established.  There is a placebo group.  Participants may only participate in a single dosing period of 28 days, then come in for 2 additional follow-up visits over 1 month. For information contact Jane Bordeau, RN at 404-727-1679 or

Genentech GDC-0134 Open-Label Extension—NO LONGER ENROLLING—Genentech will be offering an open-label extension of the GDC-0134 study detailed above. This study will be dosing with the same drug, but there will be no placebo group: all participants will receive active drug. The study will require screening and will employ the same eligibility criteria as the main study.  The study period will be approximately 12 months, including a 4-week follow-up period. Participants from previous phases of the study are eligible as well as new participants.  For further information please contact Jane Bordeau at 404-727-1679 or

Emory is also enrolling for a gene therapy study designed to treat patients with mutations in the C9orf72 gene.  For more information contact Meraida Polak at 404-778-3807 or