PDF(Pubmed)
Abstract：
UNASSIGNED: No treatments are approved for Becker muscular dystrophy (BMD). This study investigated the efficacy and safety of givinostat, a histone deacetylase pan-inhibitor, in adults with BMD.
UNASSIGNED: Males aged 18-65 years with a diagnosis of BMD confirmed by genetic testing were randomized 2:1 to 12 months treatment with givinostat or placebo. The primary objective was to demonstrate statistical superiority of givinostat over placebo for mean change from baseline in total fibrosis after 12 months. Secondary efficacy endpoints included other histological parameters, magnetic resonance imaging and spectroscopy (MRI and MRS) measures, and functional evaluations.
UNASSIGNED: Of 51 patients enrolled, 44 completed treatment. At baseline, there was greater disease involvement in the placebo group than givinostat, based on total fibrosis (mean 30.8 vs. 22.8%) and functional endpoints. Mean total fibrosis did not change from baseline in either group, and the two groups did not differ at Month 12 (least squares mean [LSM] difference 1.04%; p = 0.8282). Secondary histology parameters, MRS, and functional evaluations were consistent with the primary. MRI fat fraction in whole thigh and quadriceps did not change from baseline in the givinostat group, but values increased with placebo, with LSM givinostat-placebo differences at Month 12 of -1.35% (p = 0.0149) and -1.96% (p = 0.0022), respectively. Adverse events, most mild or moderate, were reported by 88.2% and 52.9% patients receiving givinostat and placebo.
UNASSIGNED: The study failed to achieve the primary endpoint. However, there was a potential signal from the MRI assessments suggesting givinostat could prevent (or slow down) BMD disease progression.
UNASSIGNED: Males aged 18-65 years with a diagnosis of BMD confirmed by genetic testing were randomized 2:1 to 12 months treatment with givinostat or placebo. The primary objective was to demonstrate statistical superiority of givinostat over placebo for mean change from baseline in total fibrosis after 12 months. Secondary efficacy endpoints included other histological parameters, magnetic resonance imaging and spectroscopy (MRI and MRS) measures, and functional evaluations.
UNASSIGNED: Of 51 patients enrolled, 44 completed treatment. At baseline, there was greater disease involvement in the placebo group than givinostat, based on total fibrosis (mean 30.8 vs. 22.8%) and functional endpoints. Mean total fibrosis did not change from baseline in either group, and the two groups did not differ at Month 12 (least squares mean [LSM] difference 1.04%; p = 0.8282). Secondary histology parameters, MRS, and functional evaluations were consistent with the primary. MRI fat fraction in whole thigh and quadriceps did not change from baseline in the givinostat group, but values increased with placebo, with LSM givinostat-placebo differences at Month 12 of -1.35% (p = 0.0149) and -1.96% (p = 0.0022), respectively. Adverse events, most mild or moderate, were reported by 88.2% and 52.9% patients receiving givinostat and placebo.
UNASSIGNED: The study failed to achieve the primary endpoint. However, there was a potential signal from the MRI assessments suggesting givinostat could prevent (or slow down) BMD disease progression.
摘要：
未经批准：没有批准治疗Becker肌营养不良（BMD）。这项研究调查了givinostat的疗效和安全性，组蛋白去乙酰化酶泛抑制剂，成人BMD
UNASSIGNED：年龄在18-65岁之间并经基因检测证实为BMD的男性随机接受2：1至12个月的吉维诺他或安慰剂治疗。主要目的是证明givinostat在12个月后总纤维化从基线的平均变化方面优于安慰剂。次要疗效终点包括其他组织学参数，磁共振成像和光谱学（MRI和MRS）测量，和功能评估。
未经批准：在51名患者中，44完成治疗。在基线,安慰剂组比givinostat有更多的疾病参与，基于总纤维化(平均30.8vs.22.8%)和功能终点。两组中平均总纤维化与基线相比均无变化，两组在第12个月没有差异（最小二乘均值[LSM]差异1.04%；p=0.8282）。次要组织学参数，MRS,和功能评估与主要评估一致。givinostat组整个大腿和股四头肌的MRI脂肪分数与基线相比没有变化，但是安慰剂的价值增加了，LSMgivinostat-安慰剂在第12个月的差异为-1.35%(p=0.0149)和-1.96%(p=0.0022)，分别。不良事件,最轻度或中度，接受givinostat和安慰剂的患者分别为88.2%和52.9%。
未经评估：该研究未能达到主要终点。然而,来自MRI评估的潜在信号提示givinostat可以预防(或减缓)BMD疾病进展.
UNASSIGNED：年龄在18-65岁之间并经基因检测证实为BMD的男性随机接受2：1至12个月的吉维诺他或安慰剂治疗。
未经批准：在51名患者中，
未经评估：该研究未能达到主要终点。
