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Critical Limb IschemiaPhase 1NCT00696124

Safety Study of Gene Therapy in Treating Critical Leg Ischemia

A Phase 1 study testing VM202 2 mg, VM202 4 mg and VM202 8 mg in people with Critical Limb Ischemia, run by Helixmith Co., Ltd. at 1 site in United States.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record
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Trial summary

The purpose of this study is to determine the safety, tolerability and preliminary efficacy of intramuscular injections of VM202 for subjects with critical limb ischemia. Subjects selected for this study will have critical limb ischemia that has not responded to standard therapy with symptoms including pain at rest… Read more about this study

Who is it for?
Ages 20 to 90 years
Conditions the study is for
Critical Limb Ischemia
What is being tested?
VM202 2 mg, VM202 4 mg and VM202 8 mg
Type of treatment
Injection or infusion
Could I receive a placebo?
No, everyone receives an active treatment
How many people
12 participants
Where
1 siteUnited States
Study dates
3 Apr 2007to 11 Dec 2023
Phase
Phase 1
Are there results?
Yes, posted in the registrySummary in the doctors' view

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 20 to 90 years
  • All sexes
  • No healthy volunteers
  • Adult, Older adult

Who may be able to join

  • Male or female, between 20 and 90 years of age
  • Have critical limb ischemia (Rutherford Class 4 and 5) and considered not a candidate for bypass graft surgery or percutaneous angioplasty due to co-morbid conditions, failure of previous surgical or interventional procedures or caliber of grafting arteries. Critical Limb ischemia is defined as
  • Stable symptoms on standard therapy including anti-platelet agents, vascular rheologic agents, cilostazol, anticoagulant and pain medication for 30 days.
  • Pain at rest and/or ischemic ulcers for a minimum of 4 weeks.
  • Have diagnostic angiography of the affected limb in the last 12 months demonstrating a significant occlusion of one more of the following arteries: iliac, superficial femoral, popliteal, and one or more infra-popliteal arteries.
  • Have a resting ankle systolic pressure (in either the dorsalis pedis or posterior tibial arteries) of less than or equal to 60mmHg or a resting toe systolic pressure of less than or equal to 40 mmHg in the affected limb.
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  • Be willing to maintain current drug therapy for peripheral arterial disease throughout the course of the study including anti-platelet and statin inhibitor treatment
  • 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 serum 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.

Who cannot join

  • Subjects who have undergone a revascularization procedure or sympathectomy within 12 weeks prior to study entry that remains patent. A failed revascularization procedure in the previous 4 weeks is acceptable.
  • Subjects with grade 3 (hemorrhages, exudates) or grade 4 (papilledema) retinopathy.
  • Subjects currently receiving immunosuppressive medications, chemotherapy, radiation therapy.
  • Subject with aorta-iliac occlusion (greater than 75%).
  • Subjects that will require amputation within 4 weeks of randomization.
  • 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
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  • Subjects with history of drug (defined as illicit drug use) or a history of alcohol abuse (defined as regular or daily consumption of more than 4 alcoholic drinks per day) within the past 3 months.
  • Subjects with a current history or new screening finding of malignant neoplasm except for basal cell carcinoma of the skin and squamous cell carcinoma of the skin (if excised and no evidence of recurrence).
  • Subjects with evidence of active infection (e.g. cellulitis, osteomyelitis) or deep ulceration exposing bone or tendon in the extremity planned for treatment.
  • Subjects with a clinically significant abnormality in routine hematology, urinalysis, chemistry, liver function or other laboratory tests, including HIV, Hepatitis B, Cytomegalovirus, hepatitis C virus, Venereal Disease Research Laboratory test, prostate-specific antigen, and chorio-embryonic antigen, or signs of malignant neoplasm by radiological imaging tests, including chest radiography at Screening or Day 1. Specific laboratory exclusion criteria include the following:
  • Hemoglobin less than 9.0 g/dL
  • White Blood Cell count less than 3,000 cells/mm3
  • Platelet count less than 75,000 platelets/uL
  • Fasting glucose greater than 250 mg/dL
  • AST and/or ALT greater than 3X upper limit of normal
  • Subjects with any other condition that in the opinion of the investigator might put the subject at risk or interfere with his/her participation.
  • Subjects unwilling or unable to comply with the protocol or to cooperate fully with the investigator or site personnel.
  • Subjects that have received any other investigational drug within the 30 days prior to study drug administration or will receive such a drug during the time frame of this study.
  • Subjects with uncontrolled hypertension defined as systolic blood pressure greater than 200 mmHg or diastolic blood pressure greater than 115 mmHg at Baseline evaluation.
  • Subjects with advanced liver disease including decompensated cirrhosis, jaundice, ascites, or bleeding varices.

Trial locations and contacts

1 site in 1 country.

United States1 site

  • Minneapolis Heart Institute Foundation/ Abbott Northwestern Hospital

    Minneapolis, Minnesota, 55407

Other trials in United States

About this study

Official title: Phase 1, Dose-Escalation Study to Assess the Safety and Tolerability of VM202 (Engensis) in Subjects With Critical Limb Ischemia

The purpose of this study is to determine the safety, tolerability and preliminary efficacy of intramuscular injections of VM202 for subjects with critical limb ischemia.

Subjects selected for this study will have critical limb ischemia that has not responded to standard therapy with symptoms including pain at rest and/or ischemic ulcers.

Read the detailed description

The study will consist of four (4) cohorts with a total of 3 subjects enrolled in each cohort to VM202.For each dose cohort, VM202 will be administered as a local intramuscular injection in 2 divided doses with a 2-week interval between the injections. Preliminary efficacy (hemodynamic assessments), safety and tolerability will be evaluated at Baseline (screening) and at designated time points throughout the study.

After the first subject in each cohort completed Day 30 (±2 days), and before the Day 15 dosing of the other 2 subjects in the same cohort, an interim safety evaluation was performed with the submission of safety data to the Data Safety Monitoring Committee.

All four dose cohorts will be followed for up to 5 years from the time of the first dose of study drug administration.

Being tested
VM202 2 mg, VM202 4 mg, VM202 8 mg, VM202 16 mg
Keywords
lower leg ischemia, peripheral artery disease
Medical subject headings
Chronic Limb-Threatening Ischemia, Peripheral Arterial Disease

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • VM202 2 mgBiological

    2 mg intramuscular injection with the first half of the total dose given on Day 1 and the second half on Day 15

  • VM202 4 mgBiological

    4 mg intramuscular injection, with half of the total dose given on Day 1 and the second half given on Day 15

  • VM202 8 mgBiological

    8 mg intramuscular injection. The first half of the total dose given on Day 1 and the second half on Day 15.

  • VM202 16 mgBiological

    16 mg dose intramuscular injection. The first half of the total dose given on Day 1 and the second half on Day 15.

Study groups in NCT00696124
Study groupRoleWhat they receive
Cohort 1Experimental2 mg dose VM202. The first half of the total dose given on Day 1 and the second half on Day 15.
Receives: Biological: VM202 2 mg
Cohort 2Experimental4 mg dose VM202. The first half of the total dose given on Day 1 and the second half on Day 15.
Receives: Biological: VM202 4 mg
Cohort 3Experimental8 mg dose VM202. The first half of the total dose given on Day 1 and the second half on Day 15.
Receives: Biological: VM202 8 mg
Cohort 4Experimental16 mg dose VM202. The first half of the total dose given on Day 1 and the second half on Day 15.
Receives: Biological: VM202 16 mg

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Treatment-Emergent Adverse Events.

    Treatment-emergent adverse events defined as adverse events after the first dose of Engensis (Day 1) through Day 365

    Measured: Day 1 to Day 365

Secondary outcomes (4)

  1. Change From Baseline in Pain Visual Analog Scale

    Pain intensity was assessed by subjects marking a place on a 100 mm Visual Analog Scale ranging from 0 = no pain to 100 = worst possible pain. The distance from 0 to the mark was to be measured in millimeters (0 to 100 mm).

    Measured: Days 15, 28, 59, 91, 180, and 365

  2. Change From Baseline in Ankle Brachial Index

    The Ankle Brachial Index is the ratio of the systolic blood pressure at the ankle to the systolic blood pressure in the upper arm (brachial). Outcome measure is the Change in Baseline from Day 0 (Baseline) to Actual visit Days (Days 15, 28, 59, 91, 180 and 365).

    Measured: Days 15, 28, 59, 91, 180, and 365

  3. Change From Baseline in Toe Brachial Index

    Toe brachial index is the ratio of the systolic blood pressure of the toes to the systolic blood pressure in the upper arm (brachial). Outcome measure is the Change in Ratio for Baseline from Day 0 (Baseline) to Actual visit Days (Days 15, 28, 59, 91, 180 and 365).

    Measured: Baseline and Days 1,15,28,59,91,180,and 365

  4. Change From Baseline in Transcutaneous Oxygen Pressure

    At Screening, transcutaneous oxygen pressure was measured at pre-defined locations on the anterior and posterior calf and dorsum of the foot. The limb/chest Transcutaneous Oxygen Pressure index was calculated by using the lower of the distal limb measurements

    Measured: Days 1 to 365

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted12 Jun 2008 (estimated)
  2. Study start3 Apr 2007
  3. Primary completion19 Oct 2009
  4. Study completion11 Dec 2023

Resources

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
Primary purpose
Treatment
Allocation
Non-randomised
Intervention model
Single group
Masking
None (open label)
Enrolment
12 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT00696124
Sponsor's protocol ID
US 06-1-001
Status last verified
Aug 2025
First submitted
10 Jun 2008
Last updated
6 Oct 2025
Data monitoring committee
Yes

Publications

  • Henry TD, Hirsch AT, Goldman J, Wang YL, Lips DL, McMillan WD, Duval S, Biggs TA, Keo HH. Safety of a non-viral plasmid-encoding dual isoforms of hepatocyte growth factor in critical limb ischemia patients: a phase I study. Gene Ther. 2011 Aug;18(8):788-94. doi: 10.1038/gt.2011.21. Epub 2011 Mar 24.

    PubMed 21430785

Questions about this trial

What is this clinical trial studying?

A Phase 1 study testing VM202 2 mg, VM202 4 mg and VM202 8 mg in people with Critical Limb Ischemia, run by Helixmith Co., Ltd. at 1 site in United States.

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 10 inclusion and 20 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: finished, as of the sponsor's last update on 6 Oct 2025. 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 United States. 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 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.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

Yes. Results are posted in the registry (first posted 18 Oct 2024). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Treatment-Emergent Adverse Events. (time frame: Day 1 to Day 365). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT00696124, which also lists 1 linked publication.

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 6 Oct 2025.

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