Clinical trial NCT02356809: Safety and Efficacy Study of Gene Therapy Drug (Neovasculgen) to Treat Secondary Raynaud's Phenomenon
Official title: Safety and Efficacy Study of Pl-vegf165 to Treat Secondary Raynaud's Phenomenon Caused by Systemic Scleroderma
Study type: INTERVENTIONAL
Overall status: UNKNOWN
Phase: PHASE1
PHASE2
Conditions: Secondary Raynaud's Phenomenon
Keywords: Gene therapy, plasmid, vegf, systemic scleroderma,secondary raynaud's phenomenon
Lead sponsor: Human Stem Cell Institute, Russia
Sponsor class: OTHER
Collaborators: name: Russian Academy of Medical Sciences
First posted: 2015-02-05
Enrollment: 30
Sex: ALL
Minimum age (years): 18
Maximum age (years): 65
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

Brief summary: 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

Detailed description: 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.

Eligibility criteria: 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.

Primary outcomes:
measure: Frequency of new digital ulcers
timeFrame: 180 days
measure: Frequency of adverse events
timeFrame: 180 days
Secondary outcomes:
measure: Time of complete healing of digital ulcers
timeFrame: 180 days
measure: Pain scores on the visual analog scale
timeFrame: 180 days
Other outcomes:
Locations:
city: Moscow
country: Russian Federation
facility: Human Stem Cell Institute
Study officials:
References: