Safety and Efficacy Study of Pl-vegf165 to Treat Secondary Raynaud's Phenomenon Caused by Systemic Scleroderma
试验速览
- 阶段
- 1 期
- 发起方
- 入组人数
- 30
- 试验地点
- 1
- 主要终点
- Frequency of new digital ulcers
研究概览
简要总结
The purpose of this study is to determine whether pl-vegf165 (Neovasculgen) is effective in the treatment of digital ulcers related to secondary Raynaud's phenomenon associated with systemic scleroderma
详细描述
This is a randomized, open, comparing clinical trial assessing the therapeutic efficacy of pl-vegf165 (Neovasculgen) in treating scleroderma-associated Raynaud's syndrome. Each patient of clinical group will undergo several intramuscular injections with a treatment dose of pl-vegf165 (Neovasculgen) in hand where digital ulcers locate. In the control group patients will receive only standard of care without gene therapy drug.
Study participants at the first study visit will complete study questionnaires, their hands will be assessed clinically for digital ulceration, and their hands will undergo non-invasive capillaroscopy. After this initial assessment, the patients of clinical group will undergo intramuscular injections of pl-vegf165 (Neovasculgen) in a hand altered with digital ulcers.
Patient will report the severity of their Raynaud's symptoms weekly over the six month study period. At three month post-injection, the patient will complete study questionnaires, their hands will be assessed clinically for digital ulceration, and their hands will undergo non-invasive capillaroscopy. At six months post-injection, the patient will again complete study questionnaires, their hands will be assessed clinically for digital ulceration, and their hands will undergo non-invasive capillaroscopy.
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
- None
入排标准
- 年龄范围
- 18 Years 至 65 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •obtained voluntary informed consent for participation in the clinical study
- •presence of systemic scleroderma features satisfied to criteria of American College of Rheumatology classification
- •presence at least one active digital ulcer at baseline
排除标准
- •presence of another systemic connective tissue disease;
- •absence at least one active digital ulcer at baseline;
- •smoking within 3 months or smoking cessation using nicotine products;
- •subjects currently taking sildenafil, tadalafil or vardenafil;
- •history of sympathectomy over previous 12 months
- •not able or unwilling to give voluntary informed consent for the study or follow requirements of the clinical study;
- •decompensated chronic visceral diseases;
- •clinically significant laboratory abnormalities;
- •HIV, HBV and HCV antibodies in serum;
- •alcohol or drug addiction;
- •participation in other clinical studies (or administration of study products) within 3 months prior the study;
- •conditions limiting study compliance (dementia, psycho-neurological diseases, drug addiction, alcoholism, etc.);
- •malignancies including post-surgical period with chemo- and (or) radiation therapy);
- •vascular malformations;
- •pregnancy or breastfeeding.
研究组 & 干预措施
pl-vegf165
Patients of this group will receive 2,4 mg of pl-vegf165 administered by intramuscular injection which is given in the hand
干预措施: Neovasculgen (Drug)
结局指标
主要结局
Frequency of new digital ulcers
时间窗: 180 days
To determine the ability of pl-vegf165 to prevent development of new digital ulcers the frequency of new digital ulcers will be measured in both groups and then compared
Frequency of adverse events
时间窗: 180 days
次要结局
- Pain scores on the visual analog scale(180 days)
- Time of complete healing of digital ulcers(180 days)