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

Secondary Raynaud's Phenomenon Clinical Trial

Official title:

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

Clinical Trial Details — Status: Not yet recruiting

Administrative data

• NCT number: NCT02356809
• Other study ID #: RAT
• Status: Not yet recruiting
• Phase: Phase 1/Phase 2
• First received: January 26, 2015
• Last updated: February 2, 2015
• Start date: March 2015
• Est. completion date: August 2015

Study information

• Verified date: January 2015
• Source: Human Stem Cell Institute, Russia
• Contact: n/a
• Is FDA regulated: No
• Health authority: Russian Federation: Ethics Committee of the Institute of Rheumatology
• Study type: Interventional

Clinical 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

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.

Recruitment information / eligibility

• Status: Not yet recruiting
• Enrollment: 30
• Est. completion date: August 2015
• Est. primary completion date: March 2015
• Accepts healthy volunteers: No
• Gender: Both
• Age group: 18 Years to 65 Years

Eligibility

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.

Study Design

Allocation: Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Parallel Assignment, Masking: Open Label, Primary Purpose: Treatment

Related Conditions & MeSH terms

• Neoplasm Metastasis
• Raynaud Disease
• Scleroderma, Diffuse
• Scleroderma, Systemic
• Secondary Raynaud's Phenomenon

Intervention

Drug:
• 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.

Locations

Country	Name	City	State
Russian Federation	Human Stem Cell Institute	Moscow

Sponsors (2)

Lead Sponsor	Collaborator
Human Stem Cell Institute, Russia	Russian Academy of Medical Sciences

Country where clinical trial is conducted

Russian Federation, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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	180 days	No
Primary	Frequency of adverse events		180 days	Yes
Secondary	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	180 days	No
Secondary	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.	180 days	No