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CompletedNCT01064440Updated Oct 6, 2025Results posted

Safety and Efficacy Study Using Gene Therapy for Critical Limb Ischemia

A Phase 2 interventional study of Low Dose VM202 and High Dose VM202 in Critical Limb Ischemia, sponsored by Helixmith Co., Ltd.. Completed at 16 sites in 2 countries. Open to participants aged 18 Years to 90 Years. Per ClinicalTrials.gov, last updated 2025-10-06.

Sponsored by Helixmith Co., Ltd. · Phase 2, Interventional, and Treatment

Phase
Phase 2
Study type
Interventional
Enrollment
52
Allocation
Randomized
Ages
18 Years to 90 Years
Sex
All
01

Study summary

The purpose of this study is to evaluate whether intramuscular injections of VM202 into the calf is safe and effective in the treatment of critical limb ischemia.

Read the detailed description

In the absence of revascularization options, most patients with CLI require amputation within 6 months. Patients requiring major amputation face a diminished quality of life, an unfavorable natural history and need extensive resources for their post-amputation rehabilitation and course. The 1-year amputation-free survival rate for patients diagnosed with CLI is 45%; the mortality rate is approximately 25% and may be as high as 45% in those who have undergone amputation. Management of this end-stage disease process consumes a significant amount of healthcare resources. Clearly, new therapeutic approaches are required.

Hepatocyte growth factor (HGF) has been shown to be a potent angiogenic growth factor stimulating the growth of endothelial cells and migration of vascular smooth muscle cells. Because of its pluripotent capabilities, increasing the availability of HGF in ischemic tissues to achieve therapeutic angiogenesis has been a growing area of research.

This study will use VM202, which is a DNA plasmid that contains novel genomic cDNA hybrid human HGF coding sequence (HGF-X7) expressing two isoforms of HGF, HGF 728 and HGF 723. As there are currently no approved drugs that can reverse CLI and as most patients have exhausted surgical and endovascular intervention options, inducing angiogenesis in the affected limb with VM202 may result in an increase in tissue perfusion, which, in turn improve wound healing, reduce pain and improve limb salvage rates.

02

Conditions studied

  • Critical Limb Ischemia

Keywords

  • Painful legs
  • Ischemic legs
  • Treatment for Claudication
  • Gene therapy
03

In context

Chronic Limb-Threatening Ischemia

343 studies on the registry are indexed under Chronic Limb-Threatening Ischemia; 70 are open to participants now.

This study's enrollment of 52 is above the median of 45 across 233 interventional studies indexed under Chronic Limb-Threatening Ischemia.

Browse Chronic Limb-Threatening Ischemia studies →

Lead sponsor

Helixmith Co., Ltd. is the lead sponsor of 19 studies on the registry; none are open to participants now.

Of its 6 completed or terminated interventional studies of FDA-regulated products, 6 (100%) have results posted.

Counted across the registry records on this site, refreshed daily.

04

Who can participate

Ages eligible
18 Years to 90 Years
Sexes eligible
All
Accepts healthy volunteers
No

Inclusion criteria

  • Male or female, between 18 and 90 years of age;

  • Diagnosis of critical limb ischemia (Rutherford Class 4 or 5), including:

    • A resting ankle systolic pressure (in either the dorsalis pedis or posterior tibial arteries) of ≤ 70 mmHg in the affected limb; or
    • A resting toe systolic pressure of ≤ 50 mmHg in the affected limb; or
    • For patients in which measurement of ankle systolic pressure is not feasible (e.g. vessel calcification and non-compressibility); TcPO2 ≤ 30 mmHg;
  • Poor or suboptimal candidate for bypass graft surgery or percutaneous angioplasty;
  • Pain at rest, and/or ischemic ulcers, and/or focal gangrene (\< 3 cm2) for a minimum of 2 weeks,
  • Significant stenosis (≥ 75%) of one or more of the following arteries: superficial femoral, popliteal, or two or more infra-popliteal arteries as verified by angiography within 12 months prior to enrollment;
  • Be willing to maintain current drug therapy for peripheral arterial disease throughout the course of the study including an anti-platelet and statin treatment unless not tolerated;
  • Clinically stable on optimized medical regimen for >30 days
  • Be capable of understanding and complying with the protocol and signing the informed consent document prior to being subjected to any study related procedures;
  • Women who are surgically sterile or at least 1 year postmenopausal or who have been practicing adequate contraception for at least 12 weeks prior to entering the study. If the subject is of child-bearing potential, she must have a negative urine pregnancy test result prior to study enrollment and must agree to repeat pregnancy screening tests during the study. If the subject or the subject's partner(s) is of child bearing potential, the subject and the subject's partner(s) must agree to use a "double barrier" method of birth control while participating in this study.

Exclusion criteria

Exclusion Criteria:

  • Subjects who have undergone a successful revascularization procedure or sympathectomy within 12 weeks prior to study entry. A clinically unsuccessful revascularization procedure is defined as one in which:

    • the target vessel re-occludes (≥50%, as verified by a second angiogram. Duplex ultrasonography can be used to determine vessel patency if the patient cannot tolerate a second angiogram), or
    • the target vessel remains patent, but there is no resolution of symptoms 6 weeks after the procedure (e.g. no evidence of ulcer healing, no improvement in pressures, no reduction in resting pain);
  • Subjects that will require an amputation in the target leg within 4 weeks of randomization;
  • Subjects with evidence of active infection (e.g., cellulitis, osteomyelitis) or deep ulceration exposing bone or tendon in the extremity planned for treatment;
  • Heart Failure with a NYHA classification of III or IV;
  • Stroke (NIH scale >2) or myocardial infarction within last 3 months;
  • Unstable angina
  • Uncontrolled hypertension defined as sustained systolic blood pressure (SBP) > 200 mmHg or diastolic BP (DBP) > 110 mmHg at baseline/screening evaluation;
  • Ophthalmologic conditions pertinent to proliferative retinopathy or conditions that preclude standard ophthalmologic examination;
  • Inflammatory disorder of the blood vessels (inflammatory angiopathy, such as Buerger's disease);
  • Subjects with advanced liver disease including decompensated cirrhosis, jaundice, ascites or bleeding varices;
  • Subjects currently receiving immunosuppressive medications chemotherapy, or radiation therapy;
  • Positive HIV or HTLV at screening;
  • Active Hepatitis B or C infection as determined by Hepatitis B surface antibody (HBsAb), Hepatitis B core antibody (IgG and IgM; HBcAb), Hepatitis B surface antigen (HBsAg) and Hepatitis C antibodies (Anti-HCV), at Screening;
  • Specific laboratory values at Screening including: Hemoglobin \< 8.0 g/dL, WBC \< 3,000 cells per microliter, platelet count \<75,000/mm3, AST and/or ALT > 3 times the upper limit of normal or any other clinically significant lab abnormality which in the opinion of the investigator should be exclusionary;
  • 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 PSA unless prostate cancer has been excluded;
  • Subjects with any co- morbid conditions likely to interfere with assessment of safety or efficacy or with an estimated life expectancy of less than 6 months
  • Subjects requiring > 81 mg daily of acetylsalicylic acid; If > 81 mg are taken at screening, subjects may be enrolled if willing/able to switch to another medication;
  • Subjects requiring regular COX-2 inhibitor drug(s) or high dose steroids (excepting inhaled steroids);
  • Major psychiatric disorder in past 6 months;
  • History of drug or alcohol abuse / dependence in the past 2 years;
  • Use of an investigational drug or treatment in past 12 months; concurrent participation in investigational protocol or unapproved therapeutics and
  • Unable or unwilling to give informed consent.
05

Study design

Phase
Phase 2
Primary purpose
Treatment
Allocation
Randomized
Intervention model
Parallel assignment
Masking
Quadruple (Participant, Care provider, Investigator, Outcomes assessor)
Enrollment
52 participants (actual)

Study arms

  • Experimental
    Low Dose VM202

    Patients in this group received 8mg total of VM202. Day 0: 4mg of VM202 (16 injections of 0.5ml of VM202) Day14: 4mg of VM202 (16 injections of 0.5ml of VM202) Day 28: 16 injections of 0.5ml of normal saline Day 42: 16 injections of 0.5ml of normal saline

    Biological: Low Dose VM202 · Other: Placebo

  • Experimental
    High Dose VM202

    Patients in this treatment group received a total of 16mg VM202. Day 0: 4mg of VM202 (16 injections of 0.5ml of VM202) Day14: 4mg of VM202 (16 injections of 0.5ml of VM202) Day 28: 4mg of VM202 (16 injections of 0.5ml of VM202) Day42: 4mg of VM202 (16 injections of 0.5ml of VM202)

    Biological: High Dose VM202

  • Sham comparator
    Placebo

    Patients in this group received a total of 8ml normal saline. Day 0: 16 injections of 0.5ml of normal saline Day 14: 16 injections of 0.5ml of normal saline Day 28: 16 injections of 0.5ml of normal saline Day 42: 16 injections of 0.5ml of normal saline

    Other: Placebo

Interventions

  • BiologicalLow Dose VM202

    Day 0: 4mg of VM202 (16 injections of 0.5ml of VM202) Day14: 4mg of VM202 (16 injections of 0.5ml of VM202)

    Also known as: DNA Plasmid, HGF-X7

  • BiologicalHigh Dose VM202

    Day 0: 4mg of VM202 (16 injections of 0.5ml of VM202) Day 14: 4mg of VM202 (16 injections of 0.5ml of VM202) Day 28: 4mg of VM202 (16 injections of 0.5ml of VM202) Day 42: 4mg of VM202 (16 injections of 0.5ml of VM202)

    Also known as: DNA Plasmid, HGF-X7

  • OtherPlacebo

    Day 0: 16 injections of 0.5ml of normal saline Day 14: 16 injections of 0.5ml of normal saline Day 28: 16 injections of 0.5ml of normal saline Day 42: 16 injections of 0.5ml of normal saline

    Also known as: Normal Saline

06

What researchers measure

Primary outcomes

  1. Number of Participants With Treatment-emergent Adverse Events Following Intramuscular Administration of 8 and 16 mg Engensis (VM202) or Placebo in Subjects With Critical Limb Ischemia.

    The number of participants with treatment-emergent adverse events (TEAEs), defined as adverse events occurring after the first injection of Engensis (VM202), was assessed in moderate or high-risk Critical Limb Ischemia subjects.

    Time frame: Baseline - Days 0, 14, 28, 42, 49, 90, 180, 270 and 365

  2. Change From Baseline in Visual Analog Scale (VAS) for Pain

    The Visual Analog Scale (VAS) for Pain scoring instrument is a 10 cm line, oriented horizontally, with the left end score of "0" indicating "no pain", and the right end score of "10" representing "pain as bad as it can be"

    Time frame: Days 0, 90, 180, 270, and 365

Secondary outcomes

  1. Change From Baseline in Tissue Oxygenation (TcPO2) for the Dorsal Surface of the Foot Following Engensis (VM202) or Placebo

    Tissue Oxygenation (TcPO2) measurement is reported for the dorsum of the foot. The change in baseline for the TcPO2 measured in the dorsal surface of the foot results are reported for each of the 3 study groups: 8 mg, or 16 mg for the Engensis (VM202) group, or the Placebo group. Because of the indication being peripheral vascular disease, the dorsal surface of the foot was decided by the sponsor to be a good representative of the lower extremity for any of the other measured sites.

    Time frame: Day 0 to Days 180, 270, and 365

  2. Change From Baseline in Hemodynamic Assessment for Ankle Brachial-Index (mmHg) for the Index Leg

    Change in the Resting Ankle-Brachial Index (ABI) from Baseline (Day 0) for the Index Leg to Days 180, 270, and 365. Note that by default, Day 0 has no change from baseline. Days 28 and 90 time point data was not included,as they were not relevant to assess efficacy, because of the delayed effect of Engensis, the investigational product.

    Time frame: Days 0, 28, 90, 180, 270, and 365

  3. Change From Baseline in Perfusion of the Occluded Target Artery by Magnetic Resonance Angiogram (MRA)

    The quantitative blood flow of the occluded target artery and the volumetric analysis of the newly developed artery by Magnetic Resonance Angiogram (MRA) were recorded. Note that "no change from baseline table of data" is not presented because there was only one subject with both a Baseline and Post Treatment value for Magnetic Resonance Angiogram (MRA) measurement.

    Time frame: Day 0 to Days 180 and 270

  4. Subjects With 100% Wound Healing

    The length and width (in cm) was based on photographs and measurements of ulcers. If a ulcer was determined to be 100% healed, the area of the ulcer was set to 0

    Time frame: Days 0, 14, 28, 42, 49, 90, 180, 270, and 365

  5. Change From Baseline in the Vascular Quality of Life Total Score

    The Vascular Quality of Life Total Score (VascuQol) questionnaire has 25 questions that reviewed five domains: activity level (8 items), symptoms (4 items), pain (4 items), emotional (7 items), and social (2 items). The total score is the total of the non-missing scored divided by the number of responded questions. The Vascular Quality of Life Total Score (VascuQol) scale is a 7-point scale with "1" as the worst change from baseline score, and "7" is the least change from baseline score.

    Time frame: Days 0, 90, 270, and 365

  6. Number of Subjects With Major, Lower Leg, Amputations During the Trial

    The number and percentage of subjects with major amputations during the trial

    Time frame: Day 0 through Day 365

  7. The Number of Deaths During the Trial

    The number and percentage of subjects who died during the trial

    Time frame: Day 0 to Day 365

  8. Change From Baseline in Visual Analog Scale (VAS) for Pain at 9 Months- by Sex

    The VAS scoring instrument is a 10 cm line, oriented horizontally, with the left end indicating "no pain" (score = 0 mm, better outcome) and the right end representing "pain as bad as it can be (score = 100 mm, worse outcome).

    Time frame: Days 0 (baseline), 9 months (Day 270)

  9. Change From Baseline in Visual Analog Scale (VAS) for Pain at 9 Months- by Renal Dysfunction Status

    The VAS scoring instrument is a 10 cm line, oriented horizontally, with the left end indicating "no pain" (score = 0 mm, better outcome) and the right end representing "pain as bad as it can be (score = 100 mm, worse outcome).

    Time frame: Days 0 (baseline), 9 months (Day 270)

  10. Change From Baseline in Visual Analog Scale (VAS) for Pain at 9 Months- by Diabetes Status

    The VAS scoring instrument is a 10 cm line, oriented horizontally, with the left end indicating "no pain" (score = 0 mm, better outcome) and the right end representing "pain as bad as it can be (score = 100 mm, worse outcome).

    Time frame: Days 0 (baseline), 9 months (Day 270)

07

Results

Posted Sep 19, 2024

Participant flow

Participant flow — Overall Study
MilestoneLow Dose Engensis (8 mg)High Dose Engensis (16 mg)Placebo
Started212011
Completed18178
Not completed333
Withdrew: Lost to follow-up111
Withdrew: Non-compliance010
Withdrew: Death101
Withdrew: Withdrawal by subject011
Withdrew: Other: amputation100

Outcome measures

PrimaryNumber of Participants With Treatment-emergent Adverse Events Following Intramuscular Administration of 8 and 16 mg Engensis (VM202) or Placebo in Subjects With Critical Limb Ischemia.

The number of participants with treatment-emergent adverse events (TEAEs), defined as adverse events occurring after the first injection of Engensis (VM202), was assessed in moderate or high-risk Critical Limb Ischemia subjects.

Time frame:
Baseline - Days 0, 14, 28, 42, 49, 90, 180, 270 and 365
Reported as:
Count of participants · Participants
Number of Participants With Treatment-emergent Adverse Events Following Intramuscular Administration of 8 and 16 mg Engensis (VM202) or Placebo in Subjects With Critical Limb Ischemia.
ParticipantsLow Dose Engensis (8 mg)High Dose Engensis (16 mg)Placebo
Infections and infestations8106
Cellulitis311
Gangrene032
Urinary tract infection212
Wound infection120
Cystitis020
Sinusitis020
Tooth Abscess002
Gastrointestinal disorders977
Diarrhoea322
Constipation213
Nausea222
Gastritis121
Vomiting112
Hiatus hernia020
General disorders and administration site conditions1265
Oedema peripheral321
Injection site haematoma202
Pyrexia021
Musculoskeletal and connective tissue disorders984
Pain in extremity462
Muscle spasms401
Arthralgia310
Vascular disorders974
Peripheral arterial occlusive disease241
Arterial thrombosis limb020
Metabolism and nutrition disorders565
Hypoglycaemia222
Decreased appetite022
Hyperglycaemia112
Skin and subcutaneous tissue disorders835
skin ulcer412
blister012
Injury, poisoning and procedureal complications834
Contusion212
Procedural pain311
Limb injury120
Nervous system disorders173
Headache021
Phantom pain120
Dizziness002
Renal and urinary disorders722
Renal failure acute510
Respiratory, thoracic and mediastinal disorders650
Chronic obstructive pulmonary disease120
Dyspnoea120
Psychiatric disorders451
Anxiety220
Mental status changes120
Depression020
Blood and lymphatic system disorders513
Anaemia413
Cardiac disorders342
Acute myocardial infarction020
PrimaryChange From Baseline in Visual Analog Scale (VAS) for Pain

The Visual Analog Scale (VAS) for Pain scoring instrument is a 10 cm line, oriented horizontally, with the left end score of "0" indicating "no pain", and the right end score of "10" representing "pain as bad as it can be"

Time frame:
Days 0, 90, 180, 270, and 365
Reported as:
Mean · units on a scale
Change From Baseline in Visual Analog Scale (VAS) for Pain
units on a scaleEngensis 8 mgEngensis 16 mgPlacebo
Baseline (Day 0) - Actual values only40.5 ± 31.362.3 ± 22.560.6 ± 30.1
Day 90-13.0 ± 15.1-32.7 ± 19.8-29.0 ± 19.9
Day 180-17.2 ± 25.6-28.9 ± 31.1-29.0 ± 28.5
Day 270-8.2 ± 22.3-34.5 ± 39.6-38.6 ± 41.1
Day 365-17.6 ± 30.3-29.1 ± 21.7-45.8 ± 30.8
SecondaryChange From Baseline in Tissue Oxygenation (TcPO2) for the Dorsal Surface of the Foot Following Engensis (VM202) or Placebo

Tissue Oxygenation (TcPO2) measurement is reported for the dorsum of the foot. The change in baseline for the TcPO2 measured in the dorsal surface of the foot results are reported for each of the 3 study groups: 8 mg, or 16 mg for the Engensis (VM202) group, or the Placebo group. Because of the indication being peripheral vascular disease, the dorsal surface of the foot was decided by the sponsor to be a good representative of the lower extremity for any of the other measured sites.

Time frame:
Day 0 to Days 180, 270, and 365
Reported as:
Mean · mmHg
Change From Baseline in Tissue Oxygenation (TcPO2) for the Dorsal Surface of the Foot Following Engensis (VM202) or Placebo
mmHgEngensis 8 mgEngensis 16 mgPlacebo
Baseline (Day 0) Actual values only33.4 ± 19.435.0 ± 18.440.3 ± 21.5
Day 1805.9 ± 19.77.1 ± 16.6-9.6 ± 13.5
Day 2703.6 ± 19.01.7 ± 17.8-7.1 ± 14.4
Day 3654.1 ± 20.513.4 ± 15.6-9.1 ± 15.3
SecondaryChange From Baseline in Hemodynamic Assessment for Ankle Brachial-Index (mmHg) for the Index Leg

Change in the Resting Ankle-Brachial Index (ABI) from Baseline (Day 0) for the Index Leg to Days 180, 270, and 365. Note that by default, Day 0 has no change from baseline. Days 28 and 90 time point data was not included,as they were not relevant to assess efficacy, because of the delayed effect of Engensis, the investigational product.

Time frame:
Days 0, 28, 90, 180, 270, and 365
Reported as:
Mean · mm Hg
Change From Baseline in Hemodynamic Assessment for Ankle Brachial-Index (mmHg) for the Index Leg
mm HgEngensis 8 mgEngensis 16 mgPlacebo
Day 1800.009 ± 0.130-0.004 ± 0.2390.112 ± 0.267
Day 2700.006 ± 0.1810.010 ± 0.2760.042 ± 0.216
Day 365-0.012 ± 0.123-0.011 ± 0.277-0.010 ± 0.142
SecondaryChange From Baseline in Perfusion of the Occluded Target Artery by Magnetic Resonance Angiogram (MRA)

The quantitative blood flow of the occluded target artery and the volumetric analysis of the newly developed artery by Magnetic Resonance Angiogram (MRA) were recorded. Note that "no change from baseline table of data" is not presented because there was only one subject with both a Baseline and Post Treatment value for Magnetic Resonance Angiogram (MRA) measurement.

Time frame:
Day 0 to Days 180 and 270
Change From Baseline in Perfusion of the Occluded Target Artery by Magnetic Resonance Angiogram (MRA)
MeasureEngensis 8 mgEngensis 16 mgPlacebo
Baseline———
Day 180———
Day 270———
SecondarySubjects With 100% Wound Healing

The length and width (in cm) was based on photographs and measurements of ulcers. If a ulcer was determined to be 100% healed, the area of the ulcer was set to 0

Time frame:
Days 0, 14, 28, 42, 49, 90, 180, 270, and 365
Reported as:
Count of participants · Participants
Subjects With 100% Wound Healing
ParticipantsEngensis 8 mgEngensis 16 mgPlacebo
Day 14130
Day 28140
Day 42240
Day 49240
Day 90440
Day 180840
Day 270730
Day 365530
SecondaryChange From Baseline in the Vascular Quality of Life Total Score

The Vascular Quality of Life Total Score (VascuQol) questionnaire has 25 questions that reviewed five domains: activity level (8 items), symptoms (4 items), pain (4 items), emotional (7 items), and social (2 items). The total score is the total of the non-missing scored divided by the number of responded questions. The Vascular Quality of Life Total Score (VascuQol) scale is a 7-point scale with "1" as the worst change from baseline score, and "7" is the least change from baseline score.

Time frame:
Days 0, 90, 270, and 365
Reported as:
Mean · score on a scale
Change From Baseline in the Vascular Quality of Life Total Score
score on a scaleEngensis 8 mgEngensis 16 mgPlacebo
Baseline (Day 0) - Actual values only3.57 ± 1.133.20 ± 1.153.50 ± 1.60
Day 900.53 ± 1.251.40 ± 1.062.27 ± 1.39
Day 2700.55 ± 1.161.26 ± 1.532.30 ± 1.34
Day 3650.88 ± 1.360.89 ± 1.162.07 ± 1.23
SecondaryNumber of Subjects With Major, Lower Leg, Amputations During the Trial

The number and percentage of subjects with major amputations during the trial

Time frame:
Day 0 through Day 365
Reported as:
Count of participants · Participants
Number of Subjects With Major, Lower Leg, Amputations During the Trial
ParticipantsEngensis 8 mgEngensis 16 mgPlacebo
Number of Subjects With Major, Lower Leg, Amputations During the Trial331
SecondaryThe Number of Deaths During the Trial

The number and percentage of subjects who died during the trial

Time frame:
Day 0 to Day 365
Reported as:
Count of participants · Participants
The Number of Deaths During the Trial
ParticipantsLow Dose Engensis (8 mg)High Dose Engensis (16 mg)Placebo
The Number of Deaths During the Trial101
SecondaryChange From Baseline in Visual Analog Scale (VAS) for Pain at 9 Months- by Sex

The VAS scoring instrument is a 10 cm line, oriented horizontally, with the left end indicating "no pain" (score = 0 mm, better outcome) and the right end representing "pain as bad as it can be (score = 100 mm, worse outcome).

Time frame:
Days 0 (baseline), 9 months (Day 270)
Reported as:
Mean · units on a scale
Change From Baseline in Visual Analog Scale (VAS) for Pain at 9 Months- by Sex
units on a scaleEngensis 8 mgEngensis 16 mgPlacebo
Males - Baseline (Day 0) - Actual values only39.1 ± 31.270.9 ± 14.460.6 ± 30.1
Males - Day 270-6.0 ± 22.2-43.8 ± 36.2-38.6 ± 41.1
Females - Baseline (Day 0) - Actual values only44.3 ± 36.253.7 ± 27.0—
Females Day 270-14.4 ± 24.7-25.3 ± 43.9—
SecondaryChange From Baseline in Visual Analog Scale (VAS) for Pain at 9 Months- by Renal Dysfunction Status

The VAS scoring instrument is a 10 cm line, oriented horizontally, with the left end indicating "no pain" (score = 0 mm, better outcome) and the right end representing "pain as bad as it can be (score = 100 mm, worse outcome).

Time frame:
Days 0 (baseline), 9 months (Day 270)
Reported as:
Mean · units on a scale
Change From Baseline in Visual Analog Scale (VAS) for Pain at 9 Months- by Renal Dysfunction Status
units on a scaleEngensis 8 mgEngensis 16 mgPlacebo
Has Renal Dysfunction - Baseline (Day 0) - Actual values only13.5 ± 9.059.0 ± 26.852.0 ± 38.2
Has Renal Dysfunction - Day 270-4.0 ± 11.1-37.1 ± 45.2-27.5 ± 61.5
No Renal Dysfunction - Baseline (Day 0) - Actual values only47.2 ± 31.464.6 ± 20.866.3 ± 30.9
No Renal Dysfunction - Day 270-9.3 ± 24.6-32.6 ± 38.7-46.0 ± 35.7
SecondaryChange From Baseline in Visual Analog Scale (VAS) for Pain at 9 Months- by Diabetes Status

The VAS scoring instrument is a 10 cm line, oriented horizontally, with the left end indicating "no pain" (score = 0 mm, better outcome) and the right end representing "pain as bad as it can be (score = 100 mm, worse outcome).

Time frame:
Days 0 (baseline), 9 months (Day 270)
Reported as:
Mean · units on a scale
Change From Baseline in Visual Analog Scale (VAS) for Pain at 9 Months- by Diabetes Status
units on a scaleEngensis 8 mgEngensis 16 mgPlacebo
Has Diabetes - Baseline (Day 0) - Actual values only32.6 ± 28.063.8 ± 21.877.0 ± 2.8
Has Diabetes - Day 270-4.2 ± 21.3-48.2 ± 29.3-46.5 ± 34.6
No Diabetes - Baseline (Day 0) - Actual values only56.3 ± 34.860.8 ± 25.249.7 ± 36.8
No Diabetes - Day 270-16.3 ± 24.4-20.8 ± 46.3-33.3 ± 51.6

Adverse events

Non-serious events are listed at a 5% frequency threshold.

Adverse event summary by group
GroupDeathsSeriousOther
Engensis 8 mg1/21 (4.8%)9/21 (42.9%)18/21 (85.7%)
Engensis 16 mg0/20 (0%)11/20 (55%)18/20 (90%)
Placebo1/11 (9.1%)6/11 (54.5%)10/11 (90.9%)
Most frequent serious events
Showing 10 of 36
Most frequent serious events
EventEngensis 8 mgEngensis 16 mgPlacebo
Peripheral arterial occlusive diseaseVascular disorders1/214/201/11
GangreneInfections and infestations0/213/202/11
Renal failure acuteRenal and urinary disorders3/211/200/11
Renal failure acuteRenal and urinary disorders3/211/200/11
Arterial thrombosis limbVascular disorders0/212/200/11
Acute myocardial infarctionCardiac disorders0/212/200/11
Peripheral ischaemiaVascular disorders2/211/201/11
OsteomyelitisInfections and infestations2/210/200/11
Arterial stenosis limbVascular disorders0/210/201/11
CelulitisInfections and infestations0/210/201/11
Most frequent other events
Showing 10 of 28
Most frequent other events
EventEngensis 8 mgEngensis 16 mgPlacebo
ConstipationGastrointestinal disorders2/211/203/11
Urinary tract infectionInfections and infestations2/211/202/11
Tooth abscessInfections and infestations0/210/202/11
DiarrhoeaGastrointestinal disorders3/212/202/11
NauseaGastrointestinal disorders2/212/202/11
VomitingGastrointestinal disorders1/211/202/11
CellulitisInfections and infestations3/211/201/11
Wound infectionInfections and infestations1/212/200/11
CystitisInfections and infestations0/212/200/11
SinusitisInfections and infestations0/212/200/11

Baseline characteristics

Safety population

Age, Continuous
Age, Continuous(years)Engensis 8 mgEngensis 16 mgPlaceboTotal
Mean65.9 ± 10.767.2 ± 10.964.3 ± 14.5NA ± NA
Sex: Female, Male
Sex: Female, Male(Participants)Engensis 8 mgEngensis 16 mgPlaceboTotal
Female77519
Male1413633
Race (NIH/OMB)
Race (NIH/OMB)(Participants)Engensis 8 mgEngensis 16 mgPlaceboTotal
American Indian or Alaska Native0000
Asian2136
Native Hawaiian or Other Pacific Islander0000
Black or African American47213
White1311630
More than one race0000
Unknown or Not Reported2103
Region of Enrollment
Region of Enrollment(participants)Engensis 8 mgEngensis 16 mgPlaceboTotal
South Korea2121
United States1919951
08

Study locations

16 sites
  • Cardiology PC
    Birmingham, Alabama 35211, United States
  • Vascular and Interventional Specialist of Orange County
    Orange, California 92868, United States
  • Northwestern Memorial Hospital
    Chicago, Illinois 60611, United States
  • St. Vincent Medical Group
    Indianapolis, Indiana 46290, United States
  • Boston University School of Medicine
    Boston, Massachusetts 02118, United States
  • University of Minnesota
    Minneapolis, Minnesota 55454, United States
  • Saint Louis University
    St Louis, Missouri 63110, United States
  • UNC School of Medicine
    Chapel Hill, North Carolina 27599, United States
  • Jobst Vascular
    Toledo, Ohio 43506, United States
  • University of Oklahoma HSC
    Oklahoma City, Oklahoma 73104, United States
  • Texas Heart Institute
    Houston, Texas 77030, United States
  • The Methodist Hospital
    Houston, Texas 77030, United States
  • University of Utah
    Salt Lake City, Utah 84132, United States
  • Seoul National University
    Seoul, Jongno-gu 110-744, South Korea
  • Yonsei University Health System. Severance Cardiovascular Hospital
    Seoul, Seodaemun-gu 120-752, South Korea
  • Ewha Womans University Medical Center
    Seoul, YangCheon-ku 158-710, South Korea
09

References and documents

Publications

  • Kibbe MR, Hirsch AT, Mendelsohn FO, Davies MG, Pham H, Saucedo J, Marston W, Pyun WB, Min SK, Peterson BG, Comerota A, Choi D, Ballard J, Bartow RA, Losordo DW, Sherman W, Driver V, Perin EC. Safety and efficacy of plasmid DNA expressing two isoforms of hepatocyte growth factor in patients with critical limb ischemia. Gene Ther. 2016 Mar;23(3):306-12. doi: 10.1038/gt.2015.110. Epub 2015 Dec 8. PubMed 26649448 ↗

Study documents

  • Study protocol · Jul 21, 2011
  • Statistical analysis plan · Oct 2, 2013

Documents are hosted by the registry — open the source record to download them.

10

Updates

Tracking since Sep 25, 2026
No changes since tracking began. The registry record was last updated on Oct 6, 2025, before this site started recording changes on Sep 25, 2026. Its history is on ClinicalTrials.gov ↗
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Registry details

Key details

Study ID
NCT01064440
Lead sponsor
Helixmith Co., Ltd.
Responsible party
Sponsor
First posted
Feb 8, 2010
Start date
Jul 9, 2010
Primary completion
Aug 5, 2013
Completion
Nov 17, 2023
Results posted
Sep 19, 2024
Last update
Oct 6, 2025

Study contacts

Emerson Perin, MD
principal investigator · Texas Heart Institute

Oversight

Data monitoring committee
Yes
View the source record on ClinicalTrials.gov ↗

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This study is completed, as verified in Aug 2025. You cannot join it, but the record below documents what was studied.

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Helixmith Co., Ltd.

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