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

NCT ID: NCT02356809

Last Updated: 2015-02-05

Basic Information

• Recruitment Status: UNKNOWN
• Clinical Phase: PHASE1/PHASE2
• Total Enrollment: 30 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2015-03-31
• Study Completion Date: 2015-08-31

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.

Conditions

Secondary Raynaud's Phenomenon

Study Design

• Allocation Method: RANDOMIZED
• Intervention Model: PARALLEL
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

pl-vegf165

Patients of this group will receive 2,4 mg of pl-vegf165 administered by intramuscular injection which is given in the hand

Group Type: EXPERIMENTAL

Neovasculgen

Intervention Type: DRUG

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.

standard care

Patients of this group will receive standard therapy accepted in a clinical centre

Group Type: NO_INTERVENTION

No interventions assigned to this group

Interventions

Neovasculgen

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.

Intervention Type: DRUG

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.

• Minimum Eligible Age: 18 Years
• Maximum Eligible Age: 65 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Russian Academy of Medical Sciences

OTHER

Sponsor Role: collaborator

Artgen Biotech

OTHER

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Locations

Human Stem Cell Institute

Moscow, , Russia

Countries

Russia

Facility Contacts

Igor Plaksa

Role: primary

i.plaksa2014@yandex.ru

+79112171859

Other Identifiers

RAT

Identifier Type: -

Identifier Source: org_study_id