title: Pilot trial of clenbuterol in spinal and bulbar muscular atrophy.
abstract: 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.
  
  
  METHODS
  This study provides Class IV evidence that clenbuterol is effective in improving motor function in SBMA.
authors: Neuromuscular Center, Department of Neurosciences, University of Padova, Italy.
authors: Giorgia
authors: G
authors: Querin
authors: Carla
authors: C
authors: D'Ascenzo
authors: Enrico
authors: E
authors: Peterle
authors: Mario
authors: M
authors: Ermani
authors: Luca
authors: L
authors: Bello
authors: Paola
authors: P
authors: Melacini
authors: Lucia
authors: L
authors: Morandi
authors: Letizia
authors: L
authors: Mazzini
authors: Vincenzo
authors: V
authors: Silani
authors: Monika
authors: M
authors: Raimondi
authors: Jessica
authors: J
authors: Mandrioli
authors: Silvia
authors: S
authors: Romito
authors: Corrado
authors: C
authors: Angelini
authors: Elena
authors: E
authors: Pegoraro
authors: Gianni
authors: G
authors: Sorarù
mesh_terms: D000203:Activities of Daily Living; D000318:Adrenergic beta-Agonists / Q000008:administration & dosage / Q000009:adverse effects / Q000494:pharmacology*; D000328:Adult; D000368:Aged; D055534:Bulbo-Spinal Atrophy, X-Linked / Q000188:drug therapy*; D002976:Clenbuterol / Q000008:administration & dosage / Q000009:adverse effects / Q000494:pharmacology*; D005080:Exercise Test / Q000379:methods; D005260:Female; D005500:Follow-Up Studies; D006801:Humans; D008297:Male; D008875:Middle Aged; D053580:Muscle Strength; D010865:Pilot Projects; D012720:Severity of Illness Index; D016896:Treatment Outcome; D016138:Walking / Q000502:physiology
chemical_list: D000318:Adrenergic beta-Agonists; D002976:Clenbuterol