In recent years, several multicenter, randomized, placebo-controlled, DFG- and BMBF-funded clinical trials have been conducted at the initiative of centers of the German ALS/MND Network.
Investigator Initiated Trials (IITs)
- LIPCAL-ALS (high-calorie high-fat dietary supplementation vs placebo): 12 sites, enrollment of 207 patients in 14 months; NCT02306590. The study found evidence of a beneficial effect of high-calorie, high-fat supplementation on survival, motor function, body weight, and neurofilaments, especially in rapidly progressive cases.
Ludolph AC, Dorst J, Dreyhaupt J, et al. Effect of high-caloric nutrition on survival in amyotrophic lateral sclerosis. Annals of neurology 2019; 17(10): 25661.
Dorst J, Schuster J, Dreyhaupt J, et al. Effect of high-caloric nutrition on serum neurofilament light chain levels in amyotrophic lateral sclerosis. Journal of neurology, neurosurgery, and psychiatry 2020; 91(9): 1007-9.
- RAS-ALS (rasagiline vs placebo) : 15 sites, enrollment of 252 patients in 15 months; NCT01879241. Post-hoc analyses of the study found evidence of prolonged survival and slowed loss of motor function in rapidly progressive patients on rasagiline.
Ludolph AC, Schuster J, Dorst J, et al. Safety and efficacy of rasagiline as an add-on therapy to riluzole in patients with amyotrophic lateral sclerosis: a randomised, double-blind, parallel-group, placebo-controlled, phase 2 trial. Lancet neurology 2018; 18(18): 30176-5.
- GERP-ALS (pioglitazone vs placebo): 15 sites, enrollment of 211 patients in 9 months; NCT00690118. This study found no evidence of efficacy of pioglitazone in ALS. There was evidence of altered metabolism and hypothalamic affection in ALS patients.
Dupuis L, Dengler R, Heneka MT, et al. A randomized, double blind, placebo-controlled trial of pioglitazone in combination with riluzole in amyotrophic lateral sclerosis. PloS one 2012; 7(6): e37885
- ROCK-ALS (fasudil vs placebo): 15 sites, enrollment of 120 patients; NCT03792490. Enrollment for this study is complete and results are highly anticipated.
Lingor P, Weber M, Camu W, et al. ROCK-ALS: Protocol for a Randomized, Placebo-Controlled, Double-Blind Phase IIa Trial of Safety, Tolerability and Efficacy of the Rho Kinase (ROCK) Inhibitor Fasudil in Amyotrophic Lateral Sclerosis.Front Neurol. 2019 Mar 27;10:293. doi: 10.3389/fneur.2019.00293.
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