Phase II/III Trial of Arimoclomol in SOD1-Positive Familial ALS
Opens for Enrollment

Updated November 2009

Status: Enrolling

A clinical trial of arimoclomol in patients with rapidly progressive forms of familial ALS caused by mutations in the superoxide dismutase (SOD1) gene has begun enrollment at Emory University in Atlanta, GA.

Arimcolomol acts by upregulating heat shock proteins.  It is effective in the SOD1 mouse model of ALS even when initiated after the onset of symptoms, and was found to be safe and well tolerated in a previous phase II clinical trial that included patients with sporadic ALS. 

Previously, medications that have been found to be effective in the mouse model of ALS have not shown benefit when brought to human clinical trials. Investigators at Emory University in Atlanta and the Massachusetts General Hospital (MGH) in Boston, led by Drs. Michael Benatar and Merit Cudkowicz, believe that the SOD1 mouse model of ALS most closely resembles SOD1-positive familial ALS in human and hence this is the population most likely to benefit from arimcolomol.

The target population for this double-blind, placebo-controlled, seamless adaptive design phase II/III clinical trial is patients with rapidly progressive ALS due to select mutations in the SOD1 gene.  The trial aims initially to recruit 30 ALS patients in order to evaluate drug safety and tolerability. Thereafter, approximately 50 more patients will be recruited.  All study participants will be treated for a total of 12 months. Study visits will occur monthly for the first six months and every other month thereafter. Participants in the trial will travel (at the study’s expense) either to Emory or MGH for the initial and month‑2 study visits. All other study visits will take place in individual patients’ homes to alleviate their burden of travel. especially as the disease progresses.

Patients with familial ALS who do not know if they carry a mutation in the SOD1 gene,  as well as patients with known abnormalities in the SOD1 gene, are encouraged to contact Dr. Benatar or Dr. Cudkowicz to learn more about this trial. Additional information is also available at the clinicaltrials.gov website.

The study is funded through TREAT ALS pilot clinical studies and will use the TREAT ALS/NEALS clinical trial website.