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 treatment 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.
Clinical Trials Currently Enrolling
Expanded Access Trial of RAPA-501: CURRENTLY ENROLLING
This expanded-access study is intended to allow patients ineligible for regular clinical trials to receive investigational (not FDA-approved) therapy with RAPA-501, which is currently in a standard phase 2/3 clinical trial. Breathing test must be less than 50%. All participants will receive experimental treatment. Participants will have immune cells collected at the second visit, which will be sent to the sponsor to be manipulated with RAPA-501. Cells are then re-infused via an intravenous catheter in four subsequent visits using a procedure similar to blood transfusion. Duration of the study is 8 months, with approximately 7-8 on-site visits plus 6 remote follow-up visits. Please contact Anna Partlow at anna.partlow@emory.edu for further information.
Uniqure EPISOD1 Trial of AMT-162: CURRENTLY ENROLLING
This is a phase 1/2 clinical trial for patients with the SOD1 mutation (familial ALS). Although we now have an FDA-approved drug for SOD1 ALS, it requires monthly injections of drug into the cerebrospinal fluid (CSF), which may not be practical for some people. The AMT-162 treatment is a single injection into the CSF which uses a virus to deliver AMT-162 gene therapy. The trial will involve a 2-day stay in the Emory University Hospital. Participants will come to the study site for approximately 13 visits over the first 6 months, and then have every 3-to-6 month follow-up safety visits for an additional 5 years. Please contact Wanda Sanchez at wanda.sanchez@emory.edu for further information.
Novartis ASTRALS Trial of VHB937: CURRENTLY ENROLLING
This is a Phase II study to evaluate the efficacy and safety of the study drug VHB937, administered intravenously, in participants with early-stage ALS. The goal is to determine whether VHB937 can slow ALS disease progression. The randomized-controlled portion of the study is 40 weeks or approximately 11 visits. All participants will be invited to continue in the open-label phase for an additional 72 weeks with study visits once per month. If you are interested in learning more, please contact Karon Simmons at kmsimm2@emory.edu.