A Placebo-controlled Study of Clenbuterol in Spinal and Bulbar Muscular Atrophy — BetaSBMA  

Spinal and Bulbar Muscular Atrophy Clinical Trial

Official title: A Placebo-controlled Study to Evaluate the Efficacy and Safety of Clenbuterol in Patients With Spinal and Bulbar Muscular Atrophy (SBMA)

Status Recruiting

Clinical Trial 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.

Clinical Trial Description

n/a

Related Conditions & MeSH terms

• Atrophy
• Bulbo-Spinal Atrophy, X-Linked
• Muscular Atrophy
• Spinal and Bulbar Muscular Atrophy

• NCT number: NCT06169046
• Study type: Interventional
• Source: University Hospital Padova
• Contact: Gianni Sorarù, MD
• Phone: 0498213600
• Email: gianni.soraru@unipd.it
• Status: Recruiting
• Phase: Phase 2
• Start date: April 13, 2024
• Completion date: December 31, 2027