Back to resultsSafety and Efficacy Study of Gene Therapy Drug (Neovasculgen) to Treat Secondary Raynaud's Phenomenon
Primary Purpose
Secondary Raynaud's Phenomenon
Status
Unknown status
Phase
Phase 1
Locations
Russian Federation
Study Type
Interventional
Intervention
Neovasculgen
Sponsored by
About this trial
This is an interventional treatment trial for Secondary Raynaud's Phenomenon focused on measuring Gene therapy, plasmid, vegf, systemic scleroderma,secondary raynaud's phenomenon
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.
Sites / Locations
- Human Stem Cell Institute
Arms of the Study
Arm 1
Arm 2
Arm Type
Experimental
No Intervention
Arm Label
pl-vegf165
standard care
Arm Description
Patients of this group will receive 2,4 mg of pl-vegf165 administered by intramuscular injection which is given in the hand
Patients of this group will receive standard therapy accepted in a clinical centre
Outcomes
Primary Outcome Measures
Frequency of new digital ulcers
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
Secondary Outcome Measures
Time of complete healing of digital ulcers
The ability of pl-vegf165 to accelerate the healing of the digital ulcer will be measured by estimating the area of the defect in progress
Pain scores on the visual analog scale
Visual analog scale is a diagnostic tool providing an assessment of the severity and quality of pain experienced by patients.
Full Information
NCT ID
NCT02356809
First Posted
January 26, 2015
Last Updated
February 2, 2015
Sponsor
Human Stem Cell Institute, Russia
Collaborators
Russian Academy of Medical Sciences
1. Study Identification
Unique Protocol Identification Number
NCT02356809
Brief Title
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
2. Study Status
Record Verification Date
January 2015
Overall Recruitment Status
Unknown status
Study Start Date
March 2015 (undefined)
Primary Completion Date
March 2015 (Anticipated)
Study Completion Date
August 2015 (Anticipated)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Human Stem Cell Institute, Russia
Collaborators
Russian Academy of Medical Sciences
4. Oversight
5. Study Description
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.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Secondary Raynaud's Phenomenon
Keywords
Gene therapy, plasmid, vegf, systemic scleroderma,secondary raynaud's phenomenon
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 1, Phase 2
Interventional Study Model
Parallel Assignment
Masking
None (Open Label)
Allocation
Randomized
Enrollment
30 (Anticipated)
8. Arms, Groups, and Interventions
Arm Title
pl-vegf165
Arm Type
Experimental
Arm Description
Patients of this group will receive 2,4 mg of pl-vegf165 administered by intramuscular injection which is given in the hand
Arm Title
standard care
Arm Type
No Intervention
Arm Description
Patients of this group will receive standard therapy accepted in a clinical centre
Intervention Type
Drug
Intervention Name(s)
Neovasculgen
Intervention Description
Neovasculgen is an original gene construction that contains supercoiled plasmid DNA (1.2 mg) that encodes pCMV-vegf165 as the active substance. The drug will be supplied to the study centre as a sterile lyophilisate that will be then dissolved in 2 ml of water for injections immediately prior to administration. The drug will be administered intramuscularly (interosseous muscles of hand) at 4-5 injection sites in the dorsal surface of the hand.
Primary Outcome Measure Information:
Title
Frequency of new digital ulcers
Description
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
Time Frame
180 days
Title
Frequency of adverse events
Time Frame
180 days
Secondary Outcome Measure Information:
Title
Time of complete healing of digital ulcers
Description
The ability of pl-vegf165 to accelerate the healing of the digital ulcer will be measured by estimating the area of the defect in progress
Time Frame
180 days
Title
Pain scores on the visual analog scale
Description
Visual analog scale is a diagnostic tool providing an assessment of the severity and quality of pain experienced by patients.
Time Frame
180 days
10. Eligibility
Sex
All
Minimum Age & Unit of Time
18 Years
Maximum Age & Unit of Time
65 Years
Accepts Healthy Volunteers
No
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.
Facility Information:
Facility Name
Human Stem Cell Institute
City
Moscow
Country
Russian Federation
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Igor Plaksa
Phone
+79112171859
Email
i.plaksa2014@yandex.ru
12. IPD Sharing Statement
Learn more about this trial
Safety and Efficacy Study of Gene Therapy Drug (Neovasculgen) to Treat Secondary Raynaud's Phenomenon
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