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Posted by on May 22, 2009 in FDA, Neurology, Pharma

Actively Recruiting US Interventional Trials in ALS

Actively Recruiting US Interventional Trials in ALS

To provide potentially constructive direction to those interested in the clinical investigation of ALS, here are some novel (and not so novel) treatments that are currently being (or will be) assessed in US-based trials, according to the NIH database.

  • Arimoclomol: A small molecule that upregulates cellular “molecular chaperones,” which are believed to enhance responses to cellular stress. Positive preclinical data in ALS rodent models have been reported.
  • E0302 (mecobalamin): A form of vitamin B12.
  • GSK1223249: A humanized monoclonal antibody against Nogo-A, a neurite-outgrowth inhibitor.
  • SB509: A zinc finger DNA-binding protein transcription factor that is designed to upregulate the expression of VEGF.

Actively recruiting (or not-yet-recruiting) US-based trials in ALS:

Trial Phase


Company Sponsor


E0302 IM (mecobalamin) vs placebo



Arimoclomol PO vs placebo



Lithium + riluzole vs placebo + riluzole



SB509 IM (open label)

Sangamo Biosciences

Not yet recruiting


Arimoclomol PO vs placebo



GSK1223249 IV vs placebo


N.B. Contact information for trial enrollment is available through the links.

ALS = amyotrophic lateral sclerosis; SOD1 = superoxide dismutase 1; VEGF = vascular endothelial cell growth factor.

* Patients with SOD1-positive, familial ALS.

bmartin (1127 Posts)

A native East Tennessean, Barbara Martin is a formerly practicing, board-certified neurologist who received her BS (psychology, summa cum laude) and MD from Duke University before completing her postgraduate training (internship, residency, fellowship) at the Hospital of the University of Pennsylvania in Philadelphia. She has worked in academia, private practice, medical publishing, drug market research, and continuing medical education (CME). For the last 3 years, she has worked in a freelance capacity as a medical writer, analyst, and consultant. Follow Dr. Barbara Martin on and Twitter.

1 Comment

  1. Constructive indeed