Open label study to assess the safety of VM202 in subjects with amyotrophic lateral sclerosis

IF 2.5 4区 医学 Q2 CLINICAL NEUROLOGY Amyotrophic Lateral Sclerosis and Frontotemporal Degeneration Pub Date : 2017-02-06 DOI:10.1080/21678421.2016.1259334
R. Sufit, S. Ajroud-Driss, Patricia Casey, J. Kessler
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引用次数: 31

Abstract

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.
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评估VM202在肌萎缩侧索硬化症患者中的安全性的开放标签研究
摘要目的:评价肌内注射肝细胞生长因子(HGF)DNA质粒VM202治疗肌萎缩侧索硬化症(ALS)的安全性并确定有效性措施。方法:18名参与者在第0天、第7天、第14天和第21天通过上肢和下肢交替注射的方式分剂量服用VM202。受试者接受了9个月的随访,以评估可能的不良事件。使用ALS功能评定量表修订版(ALSFRS-R)以及通过连续测量肌肉力量、肌肉周长和强迫肺活量来评估功能结果。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的VM202,没有与该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。三名参与者经历了五次与VM202无关的SAE。一名受试者死于ALS进展继发的呼吸功能不全。结论:四肢多次肌内注射VM202对ALS患者是安全的。未来三个月后再治疗的试验将确定VM202治疗是否会改变ALS的长期病程。
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来源期刊
CiteScore
5.40
自引率
10.70%
发文量
64
期刊介绍: Amyotrophic Lateral Sclerosis and Frontotemporal Degeneration is an exciting new initiative. It represents a timely expansion of the journal Amyotrophic Lateral Sclerosis in response to the clinical, imaging pathological and genetic overlap between ALS and frontotemporal dementia. The expanded journal provides outstanding coverage of research in a wide range of issues related to motor neuron diseases, especially ALS (Lou Gehrig’s disease) and cognitive decline associated with frontotemporal degeneration. The journal also covers related disorders of the neuroaxis when relevant to these core conditions.
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