This 24-week, Phase I/II, double-blind, randomized, placebo-controlled study investigated the safety and efficacy of extended-release albuterol in late-onset Pompe disease stably treated with enzyme replacement therapy at the standard dose for 4.9 (1.0-9.4) years and with no contraindications to intake of albuterol. Twelve of 13 participants completed the study. No serious adverse events were related to albuterol, and transient minor drug-related adverse events included muscle spasms and tremors. For the albuterol group, forced vital capacity in the supine position increased by 10% (p < .005), and forced expiratory volume in one second increased by 8% (p < .05); the six-minute walk test increased 25 m (p < .05; excluding one participant unable to complete muscle function testing); the Gross Motor Function Measure increased by 8% (p < .005) with the greatest increases in the Standing (18%; p < .05) and Walking, Running, and Jumping (11%; p < .005) subtests. No significant improvements would be expected in patients with late-onset Pompe disease who were stably treated with enzyme replacement therapy. The placebo group demonstrated no significant increases in performance on any measure. These data support a potential benefit of extended-release albuterol as adjunctive therapy in carefully selected patients with late-onset Pompe disease based on ability to take albuterol on enzyme replacement therapy (NCT01885936).

中文翻译：

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沙丁胺醇治疗庞贝病的I / II期I / II期临床试验中改善了肌肉功能。

这项为期24周，I / II期，双盲，随机，安慰剂对照的研究调查了缓释沙丁胺醇在以标准剂量4.9（1.0 -9.4）岁，没有沙丁胺醇的禁忌症。13名参与者中的12名完成了研究。没有与沙丁胺醇有关的严重不良事件，短暂的与药物相关的不良事件包括肌肉痉挛和震颤。对于沙丁胺醇组，仰卧位强制肺活量增加10％（p <.005），一秒钟内强制呼气量增加8％（p <.05）；六分钟步行测试增加了25 m（p <.05；不包括一名无法完成肌肉功能测试的参与者）；运动功能总指标提高了8％（p <。005）的子项测试中，站立（18％; p <.05）和步行，跑步和跳跃的增幅最大（11％; p <.005）。接受酶替代疗法稳定治疗的迟发性庞贝病患者预期不会有明显改善。安慰剂组在任何方面均未显示出明显的性能提升。这些数据基于在酶替代疗法上服用沙丁胺醇的能力，为精心选择的迟发性庞贝病患者提供了缓释沙丁胺醇作为辅助治疗的潜在益处（NCT01885936）。接受酶替代疗法稳定治疗的迟发性庞贝病患者预期不会有明显改善。安慰剂组在任何方面均未显示出明显的性能提升。这些数据基于在酶替代疗法上服用沙丁胺醇的能力，为精心选择的迟发性庞贝病患者提供了缓释沙丁胺醇作为辅助治疗的潜在益处（NCT01885936）。接受酶替代疗法稳定治疗的迟发性庞贝病患者预期不会有明显改善。安慰剂组在任何方面均未显示出明显的性能提升。这些数据基于在酶替代疗法上服用沙丁胺醇的能力，为精心选择的迟发性庞贝病患者提供了缓释沙丁胺醇作为辅助治疗的潜在益处（NCT01885936）。