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news.Phase 2 trial of dexpramipexole, led by director of the Massachusetts General Hospital (MGH) Neurology Clinical Trials Unit and ALS Center, Merit Cudkowicz, has shown slow symptom progression in the neurodegenerative disease amyotrophic lateral sclerosis (ALS).
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Dexpramipexole, initially developed by Knopp Biosciences, is a novel drug which prevents dysfunction of mitochondria, the subcellular structures that provide most of a cell’s energy.
The researchers devised a two-stage study.
In the first, 102 patients who had recently been diagnosed with ALS were randomized into four groups, receiving oral tablets of either a placebo or dexpramipexole at total daily dosages of either 50, 150 or 300 mg for 12 weeks.
On completion of first stage, participants continuing in the trial received placebo only for four weeks and then were re-randomized into two different groups, receiving daily dosages of either 50 or 300 mg of the study drug for 24 weeks.
Knopp Biosciences collaborated with Merit Cudkowicz and the Northeast ALS Consortium to sponsor the study.