There is no cure to arrest or delay SBMA progression. It is estimated that \~1000 individuals are affected by SBMA in Italy at any given time (prevalence: 1.5/100000) with an annual incidence of 0.19/100000 males. Here, we are going to test the potential of beta2-agonist stimulation on muscle as a therapeutic avenue for SBMA. We have provided pre-clinical evidence that β-agonist stimulation may be a therapeutic strategy for SBMA. Moreover, we have shown that beta2-agonists are effective in improving motor function without relevant adverse events in a small cohort of SBMA patients. To establish safety and efficacy of clenbuterol as a cure for SBMA, we are conducting a multicenter, phase II, randomized, double-blind, parallel-group, single dose, placebo-controlled trial. Indeed, based on our preliminary data, some concerns remain to be addressed.
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
QUADRUPLE
Enrollment
90
tablets
tablets
Azienda Ospedale Università di Padova
Padua, PD, Italy
RECRUITING6-minute-walk test
to assess the efficacy of chronic treatment with clenbuterol in patients with SBMA by the 6MW test
Time frame: 48 weeks
SBMA-FRS scale
SBMA-FRS total score during the 12-months treatment period (from V2 to V7) in the two treatment arms
Time frame: 48 weeks
Adult Myopathy Assessment Tool (AMAT)
AMAT total score during the 12-months treatment period (from V2 to V7) in the two treatment arms
Time frame: 48 weeks
FVC
FVC during the 12-months treatment period (from V2 to V7) in the two treatment arms
Time frame: 48 weeks
6K scale
6K total score during the 12-months treatment period (from V2 to V7) in the two treatment arms
Time frame: 48 weeks
Serum creatinine levels
serum creatinine levels during the 12-months treatment period (from V2 to V7) in the two treatment arms
Time frame: 48 weeks
ALSAQ-40
ALSAQ-40 total score during the 12-months treatment period (from V2 to V7) in the two treatment arms
Time frame: 48 weeks
individualized neuromuscular quality of life (INQoL) questionnaire
INQOL total score during the 12-months treatment period (from V2 to V7) in the two treatment arms
Time frame: 48 weeks
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