A Placebo-controlled Study of Clenbuterol in Spinal and Bulbar Muscular Atrophy
- Conditions
- Spinal and Bulbar Muscular Atrophy
- Interventions
- Drug: Placebo
- Registration Number
- NCT06169046
- Lead Sponsor
- Gianni Soraru
- Brief 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.
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- RECRUITING
- Sex
- All
- Target Recruitment
- 90
- males who have received a genetically confirmed diagnosis of SBMA (AR CAG repeat number >= 38);
- aged between 18 and 75 (+364 days) years;
- displaying one or more of the following clinical symptoms: muscle atrophy, limb weakness, bulbar palsy;
- able to walk independently with or without a cane or other supporting device (all supporting devices are acceptable except on wheelchair);
- providing a written informed consent.
- a documented cardiovascular disease precluding the use of beta2 agonists (in the judgment of the investigators);
- glaucoma, severe prostatic hypertrophy, hyperthyroidism, pheochromocytoma, and other medical conditions that, in the judgment of the investigators, would expose the patient to undue risk of harm or prevent the patient from completing the study;
- concomitant treatment with either beta-blockers or sympathomimetic drugs (If a beta-blockers concomitant medication is ongoing before the study inclusion, the patient can be enrolled if the beta-blocker is discontinued for 3 weeks prior to randomization visit);
- inability to walk or walking only with the support of a caregiver;
- use of beta2 agonists in the preceding 6 months;
- participation to an interventional trial in the preceding 3 months;
- neuromuscular disease other than SBMA.
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- PARALLEL
- Arm && Interventions
Group Intervention Description clenbuterole Clenbuterol 45 patients will receive Clenbuterol at a final dosage of 0.04 mg/day, treated for a total of 48 weeks placebo Placebo 45 patients will receive placebo, treated for a total of 48 weeks
- Primary Outcome Measures
Name Time Method 6-minute-walk test 48 weeks to assess the efficacy of chronic treatment with clenbuterol in patients with SBMA by the 6MW test
- Secondary Outcome Measures
Name Time Method FVC 48 weeks FVC during the 12-months treatment period (from V2 to V7) in the two treatment arms
SBMA-FRS scale 48 weeks SBMA-FRS total score during the 12-months treatment period (from V2 to V7) in the two treatment arms
Adult Myopathy Assessment Tool (AMAT) 48 weeks AMAT total score during the 12-months treatment period (from V2 to V7) in the two treatment arms
6K scale 48 weeks 6K total score during the 12-months treatment period (from V2 to V7) in the two treatment arms
Serum creatinine levels 48 weeks serum creatinine levels during the 12-months treatment period (from V2 to V7) in the two treatment arms
ALSAQ-40 48 weeks ALSAQ-40 total score during the 12-months treatment period (from V2 to V7) in the two treatment arms
individualized neuromuscular quality of life (INQoL) questionnaire 48 weeks INQOL total score during the 12-months treatment period (from V2 to V7) in the two treatment arms
Trial Locations
- Locations (1)
Azienda Ospedale Università di Padova
🇮🇹Padova, PD, Italy