Department of Neurology, Medical University Innsbruck, Innsbruck, Austria.
Mov Disord. 2014 Jun;29(7):935-9. doi: 10.1002/mds.25836. Epub 2014 Feb 11.
Erythropoietin (EPO) derivatives have been found to increase frataxin levels in Friedreich's ataxia (FRDA) in vitro. This multicenter, double-blind, placebo-controlled, phase II clinical trial aimed to evaluate the safety and tolerability of Lu AA24493 (carbamylated EPO; CEPO).
Thirty-six ambulatory FRDA patients harboring >400 GAA repeats were 2:1 randomly assigned to either CEPO in a fixed dose (325 µg thrice-weekly) or placebo. Safety and tolerability were assessed up to 103 days after baseline. Secondary outcome measures of efficacy (exploration of biomarkers and ataxia ratings) were performed up to 43 days after baseline.
All patients received six doses of study medication. Adverse events were equally distributed between CEPO and placebo. There was no evidence for immunogenicity of CEPO after multiple dosing. Biomarkers, such as frataxin, or measures for oxidative stress and ataxia ratings did not differ between CEPO and placebo.
CEPO was safe and well tolerated in a 2-week treatment phase. Secondary outcome measures remained without apparent difference between CEPO and placebo.
促红细胞生成素（EPO）衍生物已被发现可增加弗里德里希共济失调（FRDA）体外的铁蛋白水平。这项多中心、双盲、安慰剂对照、II 期临床试验旨在评估 Lu AA24493（氨基甲酰化 EPO；CEPO）的安全性和耐受性。
36 名携带 >400 GAA 重复的 FRDA 门诊患者以 2:1 的比例随机分配至 CEPO 固定剂量（每周 3 次，325 µg）或安慰剂。安全性和耐受性评估至基线后 103 天。次要疗效指标（探索生物标志物和共济失调评分）在基线后 43 天进行。
所有患者均接受了 6 剂研究药物。CEPO 和安慰剂之间不良反应分布均匀。多次给药后，CEPO 无免疫原性证据。生物标志物（如铁蛋白）或氧化应激和共济失调评分测量值在 CEPO 和安慰剂之间没有差异。
CEPO 在 2 周的治疗期内安全且耐受良好。次要疗效指标在 CEPO 和安慰剂之间仍无明显差异。
