Long-term, Prospective, Non-interventional, Extension of a Phase III, Randomized, Placebo-controlled, Multicenter Study to Assess the Safety & Efficacy of Engensis (VM202) in Subjects With Painful Diabetic Peripheral Neuropathy
试验速览
- 阶段
- 3 期
- 状态
- 已完成
- 入组人数
- 101
- 试验地点
- 14
- 主要终点
- Long-term Safety for Engensis Versus Placebo
研究概览
简要总结
The purpose of this study is to explore the overall safety profile and durability of efficacy of Engensis (VM202) in painful diabetic peripheral neuropathy.
All subjects still in follow-up for the VMDN-003 study or who have completed the Day 270 visit within the prior 90 days will be approached to enroll in the long-term safety extension study.
详细描述
In the phase III VMDN-003 study, subjects received 2 treatments of either Engensis (VM202) or placebo administered as intramuscular injections into bilateral calves on Days 0 and 14, and Days 90 and 104. Primary efficacy was evaluated 90 days following the first injection. The growth potential for Hepatocyte Growth Factor make long-term follow-up important both for safety and efficacy: in order for Engensis to be a candidate for chronic treatment of Painful Diabetic Peripheral Neuropathy, it must be demonstrated not to induce unexpected adverse events with repeated dosing; and the potential for reversal or stabilization of diabetic neuropathy using only one or two treatments of Engensis may make it especially attractive compared to current treatments which must be taken daily for the duration of the disease. A safety extension to the VMDN-003 study is therefore warranted.
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Parallel
- 主要目的
- Treatment
- 盲法
- Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
盲法说明
Double-blind
入排标准
- 年龄范围
- 18 Years 至 75 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Were randomized and dosed in the VMDN-003 study
- •Received all intramuscular injections of study drug on Days 0, 14, 90, and 104 in the VMDN-003 study
- •Were in follow-up for the VMDN-003 study or had completed Day 270 within the last 90 days prior to signing consent
排除标准
- •Were using an investigational drug or treatment
- •Were unable or unwilling to give informed consent
研究组 & 干预措施
Subjects who received Engensis (VM202)
VM202, Engensis
干预措施: Long-Term Follow-Up of Patients who Received Engensis (VM202) (Genetic)
Subjects who received Placebo
Placebo, vehicle
干预措施: Long-Term Follow-Up of Patients who Received Placebo (Drug)
结局指标
主要结局
Long-term Safety for Engensis Versus Placebo
时间窗: Baseline through Day 365
Long-term (6 months) safety in terms of the incidence of Treatment-emergent Adverse Events and Treatment-emergent Serious Adverse Events for Subjects who received Engensis or Placebo (in the prior VMDN-003 study)
次要结局
- The Change in the Average 24-hour Pain Score From Baseline (Day 0 of Study VMDN-003) to Day 365 for Engensis Versus Placebo(Baseline to the Day 365)
- Change in the Average 24-hour Pain Score From Day 270 to Day 365 for Engensis Versus Placebo(Day 270 to Day 365)
- Subgroup Analysis of the Change in the Average 24-hour Pain Score From Baseline (Day 0 of Study VMDN-003) to Day 365 for Engensis Versus Placebo for Subjects Without Gabapentin and/or Pregabalin Use at Baseline(Baseline to Day 365)
- Patient's Global Impression of Change at the Day 365 Visit for Engensis Versus Placebo(At the Day 365 visit)