An Italian trial to test the effects of lithium carbonate in amyotrophic lateral sclerosis (ALS) has ended before its originally scheduled completion date because of an unusually high number of dropouts, lack of demonstrated benefit, and concerns about the drug's possible toxicity.
The interruption of this trial was announced this week by the ALS MND Alliance, a coalition of ALS/motor neuron disease associations throughout the world of which MDA is a member.
An interim analysis of the trial, conducted last month (October 2009), found that 103 (60 percent) of the original 171 trial participants did not complete the trial. Thirty-seven did not complete the study because of death or tracheostomy insertion; 35 because of severe adverse events; 29 because of perceived lack of efficacy; and two for failure to comply with the requirements of the trial.
The Alliance said this dropout rate is about 2.5 times higher than that observed in any other ALS trial. That, and the possible connection between lithium and adverse events (particularly in patients with respiratory compromise), and the fact that there was no survival benefit detected, prompted the trial's steering committee to stop the study earlier than planned.
In September, investigators on a study of lithium in ALS in the United States halted their trial early, after an interim analysis showed the drug was not beneficial. (See ALS NIH Lithium Trial.)
For now, an MDA-supported study of lithium in ALS remains ongoing at the Forbes Norris MDA/ALS Research Center in San Francosci, although it is closed to new participants. (See Lithium Open-Label Study.)