A Clinical Trial of Creatine in ALS
Mitochondrial dysfunction occurs early in the course of ALS, and the mitochondria may be an important site for therapeutic intervention. Creatine stabilizes the mitochondrial transition pore, and is important in mitochondrial ATP production. In a transgenic mouse model of ALS, administration of creatine prolongs survival and preserves motor function and motor neurons.
Disease:Amyotrophic Lateral Sclerosis (ALS), Familial ALS, Sporadic ALS
Study Type:Interventional Trial
Study Category:Drug Trial
Study Status:Not enrolling
Study Chair(s)/Principal Investigator(s):
Jeremy Shefner (SUNY)
Clinicaltrials.gov ID (11 digit #):
Coordinating Center Contact Information
Full Study Summary:
Estimated Study Start Date:09/01/2000
Estimated Study Completion Date:09/30/2002
Posting Last Modified Date:05/09/2019
Date Study Added to alsconsortium.org:05/09/2019
Gender:Neals Affiliated, Diseases, Study Type, Study Category, Study Status, Phase
Time since Symptom Onset:
Time since Diagnosis:
Can participants use Riluzole?
- Site Contact Information
Creatine was tolerated well, but no benefit of creatine could be demonstrated in any outcome measure. CI analysis showed that the study, although powered to detect a 50% or greater change in rate of decline of muscle strength, actually made an effect size of greater than 23% unlikely. It was also demonstrated that motor unit number estimation was performed with acceptable reproducibility and tolerability, and may be a useful outcome measure in future clinical trials.
Any beneficial effect of creatine at 5 g per day in ALS must be small. Other agents should be considered in future studies of therapeutic agents to address mitochondrial dysfunction in ALS. In addition, motor unit number estimation may be a useful outcome measure for future clinical trials in ALS.