A Placebo-controlled Study of Clenbuterol in Spinal and Bulbar Muscular Atrophy
Status:
Recruiting
Trial end date:
2027-12-31
Target enrollment:
Participant gender:
Summary
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.
Phase:
Phase 2
Details
Lead Sponsor:
Gianni Soraru
Collaborator:
Mario Negri Institute for Pharmacological Research