Safety and Efficacy Study of Gene Therapy Drug (Neovasculgen) to Treat Secondary Raynaud's Phenomenon

Safety and Efficacy Study of Pl-vegf165 to Treat Secondary Raynaud's Phenomenon Caused by Systemic Scleroderma

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

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.

Inclusion Criteria:

• 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

Exclusion Criteria:

• 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.