NCT06169046

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

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

• Status: Recruiting
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 90 (estimated)
• Sponsor: Gianni Soraru · Academic / Other
• Sex: All
• Age: 18 Years – 75 Years
• Healthy volunteers: Not accepted

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.

Conditions

• Spinal and Bulbar Muscular Atrophy

Interventions

Type	Name	Description
DRUG	Clenbuterol	tablets
DRUG	Placebo	tablets

Timeline

• Start date: 2024-04-13
• Primary completion: 2027-06-30
• Completion: 2027-12-31
• First posted: 2023-12-13
• Last updated: 2024-04-15

Locations

1 site across 1 country: Italy