Open label study to assess the safety of VM202 in subjects with amyotrophic lateral sclerosis
Open label study to assess the safety of VM202 in subjects with amyotrophic lateral sclerosis
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评估 VM202 在肌萎缩侧索硬化症受试者中的安全性的开放标签研究
DOI：
10.1080/21678421.2016.1259334
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发表时间：
2017
影响因子：
2.8
通讯作者：
J. Kessler
中科院分区：
文献类型：
--
作者：
R. Sufit;S. Ajroud;Patricia Casey;J. Kessler
摘要目的：评估肌萎缩侧索硬化症（ALS）患者肌内注射肝细胞生长因子（HGF）DNA质粒VM 202的安全性并确定疗效指标。研究方法：在第0、7、14和21天，18名参与者通过在上肢和下肢之间交替注射分剂量施用VM 202进行治疗。受试者被随访9个月，以评估可能的不良事件。使用ALS功能评定量表-修订版（ALSFRS-R）以及连续测量肌肉力量、肌肉周长和用力肺活量来评估功能结局。结果：18名参与者中有17人完成了研究。所有参与者均耐受64 mg VM 202，没有与药物相关的严重不良事件（SAE）。12名参与者报告了26例轻度或中度注射部位反应。3名受试者发生了5起与VM 202无关的SAE。1例受试者死于继发于ALS进展的呼吸功能不全。结论：肌内注射VM 202对ALS患者是安全的。未来三个月后再治疗的试验将确定VM 202治疗是否会改变ALS的长期病程。
Abstract Objective: To assess safety and define efficacy measures of hepatocyte growth factor (HGF) DNA plasmid, VM202, administered by intramuscular injections in patients with amyotrophic lateral sclerosis (ALS). Methods: Eighteen participants were treated with VM202 administered in divided doses by injections alternating between the upper and lower limbs on d 0, 7, 14, and 21. Subjects were followed for nine months to evaluate possible adverse events. Functional outcome was assessed using the ALS Functional Rating Scale-Revised (ALSFRS-R) as well as by serially measuring muscle strength, muscle circumference, and forced vital capacity. Results: Seventeen of 18 participants completed the study. All participants tolerated 64 mg of VM202 well with no serious adverse events (SAE) related to the drug. Twelve participants reported 26 mild or moderate injection site reactions. Three participants experienced five SAEs unrelated to VM202. One subject died from respiratory insufficiency secondary to ALS progression. Conclusions: Multiple intramuscular injection of VM202 into the limbs appears safe in ALS subjects. Future trials with retreatment after three months will determine whether VM202 treatment alters the long-term course of ALS.
DOI：
10.1097/00005072-199905000-00005
发表时间：
1999-05-01
影响因子：
3.2
作者：
Martin, LJ
通讯作者：
Martin, LJ