RT Journal Article
SR Electronic
T1 Pilot trial of clenbuterol in spinal and bulbar muscular atrophy
JF Neurology
JO Neurology
FD Lippincott Williams & Wilkins
SP 2095
OP 2098
DO 10.1212/WNL.0b013e318295d766
VO 80
IS 23
A1 Querin, Giorgia
A1 D’Ascenzo, Carla
A1 Peterle, Enrico
A1 Ermani, Mario
A1 Bello, Luca
A1 Melacini, Paola
A1 Morandi, Lucia
A1 Mazzini, Letizia
A1 Silani, Vincenzo
A1 Raimondi, Monika
A1 Mandrioli, Jessica
A1 Romito, Silvia
A1 Angelini, Corrado
A1 Pegoraro, Elena
A1 Sorarù, Gianni
YR 2013
UL http://n.neurology.org/content/80/23/2095.abstract
AB Objective: To test the efficacy and tolerability of clenbuterol in patients with spinal and bulbar muscular atrophy (SBMA).Methods: Twenty patients with a diagnosis of SBMA were given oral clenbuterol (0.04 mg/d) for 12 months. The primary efficacy end point was the change from baseline of the walking distance covered in 6 minutes at 12 months. Secondary end points included the change over time in muscle strength assessed with the Medical Research Council scale, the revised Amyotrophic Lateral Sclerosis Functional Rating Scale (ALSFRS-R), and forced vital capacity values. Safety was assessed by a series of laboratory and instrumental tests, as well as reporting of adverse events.Results: Sixteen patients completed the study. There was a significant and sustained increase in walking distance covered in 6 minutes and forced vital capacity between the baseline and the 12-month assessments (p < 0.001). No differences were recorded in Medical Research Council or ALSFRS-R scores between baseline and follow-up assessments. Serious side effects, including those on heart function, were absent. A significant increase in serum creatine kinase levels was observed.Conclusions: Our findings suggest a positive effect of clenbuterol on SBMA disease progression.Classification of evidence: This study provides Class IV evidence that clenbuterol is effective in improving motor function in SBMA.ALSFRS-R=Amyotrophic Lateral Sclerosis Functional Rating Scale–revised; AR=androgen receptor; CK=creatine kinase; fVC=forced vital capacity; ITT=intention to treat; MRC=Medical Research Council; SBMA=spinal and bulbar muscular atrophy; 6MWD=6-minute walking distance; 6MWT=6-Minute Walk Test