A Placebo-controlled Study to Evaluate the Efficacy and Safety of Clenbuterol in Patients With Spinal and Bulbar Muscular Atrophy (SBMA)
试验速览
- 阶段
- 2 期
- 状态
- 招募中
- 发起方
- 入组人数
- 90
- 试验地点
- 1
- 主要终点
- 6-minute-walk test
研究概览
简要总结
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.
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Parallel
- 主要目的
- Treatment
- 盲法
- Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
入排标准
- 年龄范围
- 18 Years 至 75 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •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.
排除标准
- •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.
研究组 & 干预措施
clenbuterole
45 patients will receive Clenbuterol at a final dosage of 0.04 mg/day, treated for a total of 48 weeks
干预措施: Clenbuterol (Drug)
placebo
45 patients will receive placebo, treated for a total of 48 weeks
干预措施: Placebo (Drug)
结局指标
主要结局
6-minute-walk test
时间窗: 48 weeks
to assess the efficacy of chronic treatment with clenbuterol in patients with SBMA by the 6MW test
次要结局
- FVC(48 weeks)
- SBMA-FRS scale(48 weeks)
- Adult Myopathy Assessment Tool (AMAT)(48 weeks)
- 6K scale(48 weeks)
- Serum creatinine levels(48 weeks)
- ALSAQ-40(48 weeks)
- individualized neuromuscular quality of life (INQoL) questionnaire(48 weeks)
研究者
Gianni Soraru
Professor
University Hospital Padova