Secondary Raynaud's PhenomenonPhase 1/2NCT02356809
Safety and Efficacy Study of Gene Therapy Drug (Neovasculgen) to Treat Secondary Raynaud's Phenomenon
A Phase 1/2 study testing Neovasculgen in people with Secondary Raynaud's Phenomenon, run by Artgen Biotech at 1 site in Russia.
Trial 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 Read more about this study
- Who is it for?
- Ages 18 to 65 years
- Conditions the study is for
- Secondary Raynaud's Phenomenon
- What is being tested?
- Neovasculgen
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 30 participants
- Where
- 1 siteRussia
- Study dates
- Mar 2015to Aug 2015
- Phase
- Phase 1/2
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 65 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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
Who cannot join
- 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;
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- 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.
Trial locations and contacts
1 site in 1 country.
Russia1 site
Human Stem Cell Institute
Moscow
Igor Plaksa, +79112171859, i.plaksa2014@yandex.ru
About this study
Official title: 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
Read the 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.
- Conditions
- Secondary Raynaud's Phenomenon
- Being tested
- Neovasculgen
- Keywords
- Gene therapy, plasmid, vegf, systemic scleroderma,secondary raynaud's phenomenon
- Medical subject headings
- Raynaud Disease, Scleroderma, Systemic
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
NeovasculgenDrug
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.
| Study group | Role | What they receive |
|---|---|---|
| pl-vegf165 | Experimental | Patients of this group will receive 2,4 mg of pl-vegf165 administered by intramuscular injection which is given in the hand Receives: Drug: Neovasculgen |
| standard care | No intervention | Patients of this group will receive standard therapy accepted in a clinical centre |
What is being measured
The primary outcome is the main question the study is designed to answer.
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
Measured: 180 days
Frequency of adverse events
Measured: 180 days
Secondary outcomes (2)
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
Measured: 180 days
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.
Measured: 180 days
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted5 Feb 2015 (estimated)
- Study startMar 2015
- Primary completionMar 2015 (estimated)
- Study completionAug 2015 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Secondary Raynaud's PhenomenonStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1/2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 30 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Artgen Biotech
- Sponsor type
- University, hospital or other
- Collaborators
- Russian Academy of Medical Sciences
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02356809
- Sponsor's protocol ID
- RAT
- Status last verified
- Jan 2015
- First submitted
- 26 Jan 2015
- Last updated
- 5 Feb 2015
- Results posted
- No
Study at a glance
From the registry record, last updated by the sponsor on 5 Feb 2015. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Safety and Efficacy Study of Pl-vegf165 to Treat Secondary Raynaud's Phenomenon Caused by Systemic Scleroderma
- ClinicalTrials.gov ID
- NCT02356809
- Acronym / sponsor ID
- RAT
- Conditions
- Secondary Raynaud's Phenomenon
- Keywords
- Gene therapy, plasmid, vegf, systemic scleroderma,secondary raynaud's phenomenon
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 30 participants (estimated)
- Lead sponsor
- Artgen BiotechUniversity, hospital or other
- Collaborators
- Russian Academy of Medical Sciences
- Study start
- Mar 2015
- Primary completion
- Mar 2015 (estimated)
- Study completion
- Aug 2015 (estimated)
- First posted
- 5 Feb 2015 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| pl-vegf165 | Experimental | Drug: NeovasculgenPatients of this group will receive 2,4 mg of pl-vegf165 administered by intramuscular injection which is given in the hand |
| standard care | No intervention | –Patients of this group will receive standard therapy accepted in a clinical centre |
Who may qualify
- Ages
- 18 to 65 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (3 listed)
- 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
Key exclusion criteria (15 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| NeovasculgenNeovasculgen 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. | Drug | pl-vegf165 |
Primary outcome
Frequency of new digital ulcers
Time frame: 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
Time frame: 180 days
2 secondary outcomes
Time of complete healing of digital ulcers
Time frame: 180 days
Pain scores on the visual analog scale
Time frame: 180 days
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 5 Feb 2015.
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A Study of LIQ861 (Inhaled Treprostinil) in Adults With Systemic Sclerosis Who Have Symptomatic Raynaud's Phenomenon Attacks
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing Neovasculgen in people with Secondary Raynaud's Phenomenon, run by Artgen Biotech at 1 site in Russia.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 3 inclusion and 15 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: status not confirmed, as of the sponsor's last update on 5 Feb 2015. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in Russia. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this combined phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
Each site lists its own contact: choose the nearest one under Trial locations. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Frequency of new digital ulcers (time frame: 180 days). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 5 Feb 2015.