TOLERABILITY AND EFFICACY OF L-ACETYLCARNITINE IN PATIENTS WITH PERIPHERAL NEUROPATHIES - A SHORT-TERM, OPEN MULTICENTER STUDY

A total of 1097 patients, mean age 53.8 +/- 15 years, with peripheral mono-, multi- or polyneuropathy of various aetiologies, were enrolled in a multicentre, open-label, noncomparative, prospective clinical tri al to evaluate the short-term tolerability and efficacy of L-acetylcar nitine (LAG). The drug was administered intramuscularly at a dosage of 1000 mg/day for the first 10 days, then orally at a dosage of 2000 mg /day for a further 20 days. Standard laboratory tests were used to eva luate safety and tolerability. Treatment efficacy was assessed clinica lly in the whole population, and in a subgroup of patients with 'lower than normal' baseline nerve conduction velocities (CVs), neurophysiol ogical investigations were also performed at the end of the treatment period. After 30 days' therapy, there were no changes in vital signs o r in blood tests. Only 18 patients reported poor tolerability of the t reatment, mainly because of gastrointestinal events, and only 6 withdr ew from the study because of these adverse events. The general and loc al (i.e. injection site) tolerability of LAC was rated highly by both patients and investigators. Neurological examination revealed that a s ignificant percentage of patients with altered indices at baseline had normal indices by the end of the treatment period. The percentage of normalised patients varied from 11.9% for muscular trophism to 29.1% f or the topographic score according to the different parameters taken i nto account in the neurological examination. Disease was rated as impr oved by 83.1% of investigators and 84.2% of patients. In patients with reduced CVs, significant increments were also recorded for motor and sensory nerves. This study demonstrates that LAC is well tolerated whe n given either intramuscularly or orally. The beneficial effects of sh ort-term therapy in subjects with peripheral mono-, multi-or polyneuro pathy should be confirmed by longterm studies.