Actively Recruiting
Testing Clenbuterol for Safety and Effectiveness in Adults With Spinal and Bulbar Muscular Atrophy
Led by Gianni Soraru · Updated on 2024-04-15
90
Participants Needed
1
Research Sites
26 weeks
Total Duration
AI-Summary
What this Trial Is About
Spinal and Bulbar Muscular Atrophy SBMA is a rare condition with no current cure to stop or slow its progression. Researchers are studying the potential of beta2-agonist drugs, specifically clenbuterol, to improve muscle function in people with SBMA. This phase II, multicenter, randomized, double-blind, placebo-controlled trial aims to evaluate the safety and effectiveness of clenbuterol for this condition, addressing concerns from earlier small-scale studies. In this trial, 90 participants with genetically confirmed SBMA will be randomly assigned to one of two groups. One group will receive clenbuterol tablets at a dose of 0.04 mg per day, while the other group will receive placebo tablets. Both groups will be treated for 48 weeks under a quadruple-blind design to compare the effects of clenbuterol against placebo. Participants will be involved for 48 weeks during which their motor function and quality of life will be closely monitored. Assessments include a 6-minute walk test, SBMA-FRS scale, Adult Myopathy Assessment Tool, lung function tests, serum creatinine levels, and neuromuscular quality of life questionnaires. Safety and efficacy data collected will help determine the potential benefits and risks of clenbuterol in SBMA patients.
CONDITIONS
Brief Title
A Placebo-controlled Study of Clenbuterol in Spinal and Bulbar Muscular Atrophy
Research Team
G
Gianni Sorarù, MD
E
Elisabetta Pupillo, PharmD
Not the Right Trial for You?
Explore thousands of other clinical trials that might be a better match.
Sign up to get personalized trial recommendations delivered to your inbox.
Already have an account? Log in here
