Double-blind, crossover, placebo-controlled clinical trial with L-acetylcarnitine in patients with degenerative cerebellar ataxia

Clin Neuropharmacol. Mar-Apr 2000;23(2):114-8. doi: 10.1097/00002826-200003000-00010.


Despite the different genetic defects underlying degenerative ataxias, it has been suggested that mitochondrial energy production and antioxidative metabolism dysfunction may be common biochemical alterations related to these diseases. Acetylcarnitine, a cholinomimetic substance, is involved in oxidative metabolism and is a potential source of acetyl groups for the synthesis of acetylcholine in the mammalian brain. To determine whether treatment with L-acetylcarnitine may improve some clinical conditions of patients with ataxia, a double-blind crossover study with L-acetylcarnitine was performed in 24 patients with degenerative cerebellar diseases. Patients were selected from an ongoing prospective follow-up study at the Department of Neurology at the University of Florence, Italy. Each treatment phase with L-acetylcarnitine or placebo lasted 6 months, after which patients were crossed over to the other treatment phase. Ataxia was documented and quantified with use of a clinical score. After the trial, we observed a statistically significant improvement of some symptoms and a slow progression of the disease in both groups of patients.

Publication types

  • Clinical Trial
  • Randomized Controlled Trial

MeSH terms

  • Acetylcarnitine / therapeutic use*
  • Adolescent
  • Adult
  • Cerebellar Ataxia / drug therapy*
  • Cerebellar Ataxia / genetics
  • Cross-Over Studies
  • Double-Blind Method
  • Female
  • Humans
  • Male
  • Middle Aged
  • Neuropsychological Tests
  • Nootropic Agents / therapeutic use*


  • Nootropic Agents
  • Acetylcarnitine