Safety and Efficacy Study of Gene Therapy for Acute Myocardial Infarction in Korea

NCT03404024 | Withdrawn Phase 2 DMC

• 1 other identifier: VMCAD-002
• Study Type: interventional
• Target: N/A
• Locations: 1 country
• Sites: 4

Brief Summary

The purpose of this study is to evaluate the safety and clinical efficacy of VM202RY injected via transendocardial route using C-Cathez® catheter (Celyad, S.A., Belgium) in subjects with AMI.

• Stage 1: Evaluation of safety and tolerability of VM202RY injection
• Stage 2: Evaluation of safety and efficacy of VM202RY injection

Conditions

Ischemic Heart Disease; Acute Myocardial Infarction

Keywords

gene therapy

Outcome Measures

Primary Outcomes (2)

• Stage 1: MTD (maximum tolerated dose)

  • MTD is defined as the dose level below the dose at which ≥ 33% of participants experienced DLT (dose limiting toxicity). The DLT assessment will be conducted on day 14. Toxicities more severe than grade 3 on the WHO toxicity scale will be designated as DLT. The dose where severe adverse events occur according to the Spilker classification will also be designated as DLT. The assessment will be conducted from the lowest dose to higher doses incrementally. MTD will be measured in milligrams (mg).

  6 months

• Stage 2: LVEF (left ventricular ejection fraction) measured by cardiac MRI

  • The change in LVEF measured by cardiac MRI in VM202RY and placebo will be compared at 6 months after the treatment of VM202RY or Placebo by transendocardial injections using C-CATHez® catheter. LVEF will be measured in percentage (%).

  6 months

Secondary Outcomes (16)

• Change in left ventricular diameter (cardiac MRI)

  3 and 6 months

• Change in left ventricular diameter (TTE)

  3 and 6 months

• Change in left ventricular volume (cardiac MRI)

  3 and 6 months

• Change in left ventricular volume (TTE)

  3 and 6 months

• Change in cardiac output (cardiac MRI)

  3 and 6 months

Study Arms (6)

Stage 1-Low dose VM202RY

EXPERIMENTAL

Patients in this group will receive total 1mg of VM202RY. (4 sites of 0.25mg/0.5 mL VM202RY)

Biological: Stage 1-Low dose VM202RY; Device: C-Cathez® Catheter

Stage 1-Middle dose VM202RY

EXPERIMENTAL

Patients in this group will receive total 2mg of VM202RY. (8 sites of 0.25mg/0.5 mL VM202RY)

Biological: Stage 1-Middle dose VM202RY; Device: C-Cathez® Catheter

Stage 1-High dose VM202RY

EXPERIMENTAL

Patients in this group will receive total 3mg of VM202RY. (12 sites of 0.25mg/0.5 mL VM202RY)

Biological: Stage 1-High dose VM202RY; Device: C-Cathez® Catheter

Stage 2-Placebo

PLACEBO COMPARATOR

Patients in this group will receive 6mL of VM202RY vehicle. (12 sites of 0.5mL 0.9% NaCl, 1.1% sucrose)

Drug: Stage 2-Placebo; Device: C-Cathez® Catheter

Stage 2-Low dose VM202RY

EXPERIMENTAL

Patients in this group will receive total 6mL of VM202RY and VM202RY vehicle. (The dose of VM202RY can be one of the three candidate-0.5mg VM202RY/1mg VM202RY/1.5mg VM202RY based on the tolerated dose result from Stage 1.)

Biological: Stage 2-Low dose VM202RY; Device: C-Cathez® Catheter

Stage 2-High dose VM202RY

EXPERIMENTAL

Patients in this group will receive total 6mL of VM202RY and VM202RY vehicle. (The dose of VM202RY can be one of the three candidate-1mg VM202RY/2mg VM202RY/3mg VM202RY based on the tolerated dose result from Stage 1.)

Biological: Stage 2-High dose VM202RY; Device: C-Cathez® Catheter

Interventions

Stage 1-Low dose VM202RY BIOLOGICAL

Day 0: 1mg of VM202RY (4 sites of 0.25mg/0.5mL VM202RY)

Also known as: DNA Plasmid, Hepatocyte growth factor (HGF)-X7

Stage 1-Low dose VM202RY

Stage 1-Middle dose VM202RY BIOLOGICAL

Day 0: 2mg of VM202RY (8 sites of 0.25mg/0.5mL VM202RY)

Also known as: DNA Plasmid, HGF-X7

Stage 1-Middle dose VM202RY

Stage 1-High dose VM202RY BIOLOGICAL

Day 0: 3mg of VM202RY (12 sites of 0.25mg/0.5mL VM202RY)

Also known as: DNA Plasmid, HGF-X7

Stage 1-High dose VM202RY

Stage 2-Placebo DRUG

Day 0: 6mL of VM202RY vehicle (12 sites of 0.5mL 1.1% sucrose/0.9% NaCl)

Also known as: VM202RY vehicle, Sodium chloride, sucrose

Stage 2-Placebo

Stage 2-Low dose VM202RY BIOLOGICAL

Day 0: 6mL of VM202RY and VM202RY vehicle (total 12 site injections, low dose candidate-0.5mg VM202RY, 1mg VM202RY, 1.5mg VM202RY)

Also known as: DNA plasmid, HGF-X7

Stage 2-Low dose VM202RY

Stage 2-High dose VM202RY BIOLOGICAL

Day 0: 6mL of VM202RY and VM202RY vehicle (total 12 site injections, high dose candidate-1mg VM202RY, 2mg VM202RY, 3mg VM202RY)

Also known as: DNA Plasmid, HGF-X7

Stage 2-High dose VM202RY

C-Cathez® Catheter DEVICE

Day 0 (Stage 1-Low dose VM202RY): 1mg of VM202RY (4 sites of 0.25mg/0.5mL VM202RY) Day 0 (Stage 1-Middle dose VM202RY): 2mg of VM202RY (8 sites of 0.25mg/0.5mL VM202RY) Day 0 (Stage 1-High dose VM202RY): 3mg of VM202RY (12 sites of 0.25mg/0.5mL VM202RY) Day 0 (Stage 2-Placebo): 6mL of VM202RY vehicle (12 sites of 0.5mL 1.1% sucrose/0.9% NaCl) Day 0 (Stage 2-Low dose VM202RY): 6mL of VM202RY and VM202RY vehicle (total 12 site injections, low dose candidate-0.5mg VM202RY, 1mg VM202RY, 1.5mg VM202RY) Day 0 (Stage 2-High dose VM202RY): 6mL of VM202RY and VM202RY vehicle (total 12 site injections, high dose candidate-1mg VM202RY, 2mg VM202RY, 3mg VM202RY)

Stage 1-High dose VM202RY; Stage 1-Low dose VM202RY; Stage 1-Middle dose VM202RY; Stage 2-High dose VM202RY; Stage 2-Low dose VM202RY; Stage 2-Placebo

Eligibility Criteria

• Age: 19 Years - 75 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Age ≥ 19 years to ≤ 75 years
• Patients who underwent percutaneous coronary intervention (PCI) for the anterior wall infarction of left ventricle and had the elapsed time of 30 ± 2 days from the PCI, regardless of success or failure of myocardial revascularization in the anterior wall
• Patients with \> 20% to ≤ 45% of left ventricular ejection fraction via trans-thoracic echocardiography within 7 days prior to the study drug or placebo injection
• Left ventricular wall thickness ≥ 8 mm via trans-thoracic echocardiography (however, the subject shall be included if 50% or greater of the left ventricular anterior wall is ≥8mm or injection site other than the left ventricular anterior wall is ≥ 8mm.)
• If female of childbearing potential, negative urine pregnancy test at screening and using acceptable method of birth control during the study; if male, using barrier method of birth control during study
• Be capable of understanding and complying with the protocol and signing the informed consent document prior to being subjected to any study related procedures.

You may not qualify if:

• Severe systolic heart failure, NYHA Class III or IV
• New York Heart Association (NYHA) functional class IV
• History of recurrent ventricular tachycardia or cariogenic shock following PCI
• Stroke or transient ischemic attack (TIA) within 180 days
• Uncontrolled hypertension defined as systolic blood pressure ≥ 180 mmHg or diastolic ≥ 110 mmHg at screening and/or on the day of study drug or placebo injection
• Sustained ventricular tachyarrhythmia or recurrent ventricular tachycardia
• Implantation of automatic implantable cardioverter defibrillator (AICD)
• On extracorporeal membrane oxygenator (ECMO)
• History of ventricular fibrillation after PCI
• Permanent pacemaker implantation (temporary pacemaker may be enrolled)
• Subjects with aortic stenosis of moderate or greater degree, or with prosthetic aortic valve who may not be appropriate to use the C-CATHez® catheter due to the risk of injury during the interventional procedure through the valve
• Atherosclerotic or other disease of the aorto-iliac system that would impede the safe passage of the C-CATHez®
• Subjects with any serious comorbidities that the investigators deemed to be inappropriate to be enrolled
• Patients with a recent history (\< 5 years) of, or new screening finding of malignant neoplasm except basal cell carcinoma or squamous cell carcinoma of the skin (if excised and no evidence of recurrence); patients with family history of colon cancer in any first degree relative are excluded unless they have undergone a colonoscopy in the last 12 months with negative findings
• Elevated prostate-specific antigen (PSA) despite not having prostate cancer history

Sponsors & Collaborators

• Helixmith Co., Ltd.: lead

Study Sites (4)

GangNeung Asan Hospital

Gangneung, South Korea

Location

Chonnam National University Hospital

Gwangju, South Korea

Location

Ewha Womans University Medical Center

Seoul, South Korea

Location

KyungHee University Medical Center

Seoul, South Korea

Location

MeSH Terms

Conditions

Myocardial Ischemia

Interventions

Hepatocyte Growth Factor; Sodium Chloride; Sucrose

Condition Hierarchy (Ancestors)

Heart Diseases; Cardiovascular Diseases; Vascular Diseases

Intervention Hierarchy (Ancestors)

Cytokines; Intercellular Signaling Peptides and Proteins; Peptides; Amino Acids, Peptides, and Proteins; Proteins; Biological Factors; Chlorides; Hydrochloric Acid; Chlorine Compounds; Inorganic Chemicals; Sodium Compounds; Disaccharides; Oligosaccharides; Polysaccharides; Carbohydrates; Sugars

Study Officials

• Wook Bum Pyun, MD, PhD

  Ewha Womans University

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: August 16, 2017
• First Posted: January 19, 2018
• Study Start: January 25, 2018
• Primary Completion: August 20, 2019
• Study Completion: August 20, 2019
• Last Updated: September 25, 2025

Record last verified: 2025-08

Data Sharing

• IPD Sharing: Will not share

Locations

KR (4)