Trial · NCT01064440
Safety and Efficacy Study Using Gene Therapy for Critical Limb Ischemia
NCT01064440, Phase 2, Completed, Helixmith Co., Ltd.; results first posted 2024-09-19
3 sourcestrials-2026-11-12-0.5.0
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ConductScience Library. Safety and Efficacy Study Using Gene Therapy for Critical Limb Ischemia. Trials, record NCT01064440. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT01064440
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Safety and Efficacy Study Using Gene Therapy for Critical Limb Ischemia
- NCT number
- NCT01064440
- Status
- Completed
- Phases
- Phase 2
- Enrollment
- 52 participants, actual
- Lead sponsor
- Helixmith Co., Ltd.
- Conditions
- Critical Limb Ischemia
- Interventions
- Low Dose VM202, High Dose VM202, Placebo
- First posted
- 2010-02-08
- Last updated
- 2025-10-06
- Results first posted
- 2024-09-19
Source ClinicalTrials.gov
Registered outcomes12
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | 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. | Baseline - Days 0, 14, 28, 42, 49, 90, 180, 270 and 365 | 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. |
| Primary | Change From Baseline in Visual Analog Scale (VAS) for Pain | Days 0, 90, 180, 270, and 365 | 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" |
| Secondary | Change From Baseline in Tissue Oxygenation (TcPO2) for the Dorsal Surface of the Foot Following Engensis (VM202) or Placebo | Day 0 to Days 180, 270, and 365 | 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. |
| Secondary | Change From Baseline in Hemodynamic Assessment for Ankle Brachial-Index (mmHg) for the Index Leg | Days 0, 28, 90, 180, 270, and 365 | 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. |
| Secondary | Change From Baseline in Perfusion of the Occluded Target Artery by Magnetic Resonance Angiogram (MRA) | Day 0 to Days 180 and 270 | 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. |
| Secondary | Subjects With 100% Wound Healing | Days 0, 14, 28, 42, 49, 90, 180, 270, and 365 | 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 |
| Secondary | Change From Baseline in the Vascular Quality of Life Total Score | Days 0, 90, 270, and 365 | 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. |
| Secondary | Number of Subjects With Major, Lower Leg, Amputations During the Trial | Day 0 through Day 365 | The number and percentage of subjects with major amputations during the trial |
| Secondary | The Number of Deaths During the Trial | Day 0 to Day 365 | The number and percentage of subjects who died during the trial |
| Secondary | Change From Baseline in Visual Analog Scale (VAS) for Pain at 9 Months- by Sex | Days 0 (baseline), 9 months (Day 270) | 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). |
1 to 10 of 12Source ClinicalTrials.gov
See all 12 registered outcomesOutcomes12
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | 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. | Baseline - Days 0, 14, 28, 42, 49, 90, 180, 270 and 365 | The Safety Population included all subjects who received at least 1 study drug injection of Engensis or Placebo | Participants | COUNT_OF_PARTICIPANTS |
| PRIMARY | Change From Baseline in Visual Analog Scale (VAS) for Pain | Days 0, 90, 180, 270, and 365 | The Per-Protocol (PP) Population included all subjects who received the correct dose of study drug, had the 9-month (Day 270) VAS assessment, and did not have any protocol violations or major deviations. The PP Population was determined in a blinded review before database lock. Subjects were analyzed according to the treatment to which they were randomized. Primary efficacy analyses were performed on the PP population. | units on a scale | MEAN |
| SECONDARY | Change From Baseline in Tissue Oxygenation (TcPO2) for the Dorsal Surface of the Foot Following Engensis (VM202) or Placebo | Day 0 to Days 180, 270, and 365 | The Intent-to-Treat population included all subjects who were randomized regardless of whether treatment was received | mmHg | MEAN |
| SECONDARY | Change From Baseline in Hemodynamic Assessment for Ankle Brachial-Index (mmHg) for the Index Leg | Days 0, 28, 90, 180, 270, and 365 | Intent-to-treat population: includes all subjects who were randomized regardless of whether treatment was received | mm Hg | MEAN |
| SECONDARY | Change From Baseline in Perfusion of the Occluded Target Artery by Magnetic Resonance Angiogram (MRA) | Day 0 to Days 180 and 270 | ITT population or Per protocol population - Note: Only 1 subject had both a baseline and post-treatment MRA performed. Unfortunately, no quantitative perfusion data were collected. | ||
| SECONDARY | Subjects With 100% Wound Healing | Days 0, 14, 28, 42, 49, 90, 180, 270, and 365 | Intent-to-Treat population | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | Change From Baseline in the Vascular Quality of Life Total Score | Days 0, 90, 270, and 365 | Per-protocol population | score on a scale | MEAN |
| SECONDARY | Number of Subjects With Major, Lower Leg, Amputations During the Trial | Day 0 through Day 365 | Intent-to-treat population | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | The Number of Deaths During the Trial | Day 0 to Day 365 | Intent-to-treat population | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | Change From Baseline in Visual Analog Scale (VAS) for Pain at 9 Months- by Sex | Days 0 (baseline), 9 months (Day 270) | The Per-Protocol (PP) Population included all subjects who received the correct dose of study drug, had the 9-month (Day 270) VAS assessment, and did not have any protocol violations or major deviations. The PP Population was determined in a blinded review before database lock. Subjects were analyzed according to the treatment to which they were randomized. Primary efficacy analyses were performed on the PP population. | units on a scale | MEAN |
1 to 10 of 12Source ClinicalTrials.gov
See all 12 outcomesResults by group274
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| 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. | Low Dose Engensis (8 mg) | Infections and infestations | 8 | 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. | High Dose Engensis (16 mg) | Infections and infestations | 10 | 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. | Placebo | Infections and infestations | 6 | 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. | Low Dose Engensis (8 mg) | Cellulitis | 3 | 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. | High Dose Engensis (16 mg) | Cellulitis | 1 | 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. | Placebo | Cellulitis | 1 | 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. | Low Dose Engensis (8 mg) | Gangrene | 0 | 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. | High Dose Engensis (16 mg) | Gangrene | 3 | 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. | Placebo | Gangrene | 2 | 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. | Low Dose Engensis (8 mg) | Urinary tract infection | 2 | Participants |
1 to 10 of 274Source ClinicalTrials.gov
See all 274 results by groupParticipant flow9
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | Low Dose Engensis (8 mg) | 21 | |
| Overall Study | STARTED | High Dose Engensis (16 mg) | 20 | |
| Overall Study | STARTED | Placebo | 11 | |
| Overall Study | COMPLETED | Low Dose Engensis (8 mg) | 18 | |
| Overall Study | COMPLETED | High Dose Engensis (16 mg) | 17 | |
| Overall Study | COMPLETED | Placebo | 8 | |
| Overall Study | NOT COMPLETED | Low Dose Engensis (8 mg) | 3 | |
| Overall Study | NOT COMPLETED | High Dose Engensis (16 mg) | 3 | |
| Overall Study | NOT COMPLETED | Placebo | 3 |
Source ClinicalTrials.gov
Adverse events192
Each adverse event the trial posted, group by group: the participants affected out of those at risk.
| Type | Event | Organ system | Group | Affected | At risk | Events |
|---|---|---|---|---|---|---|
| other | Cellulitis | Infections and infestations | Engensis 8 mg | 3 | 21 | 3 |
| other | Cellulitis | Infections and infestations | Engensis 16 mg | 1 | 20 | 1 |
| other | Cellulitis | Infections and infestations | Placebo | 1 | 11 | 1 |
| other | Urinary tract infection | Infections and infestations | Engensis 8 mg | 2 | 21 | 2 |
| other | Urinary tract infection | Infections and infestations | Engensis 16 mg | 1 | 20 | 2 |
| other | Urinary tract infection | Infections and infestations | Placebo | 2 | 11 | 3 |
| other | Wound infection | Infections and infestations | Engensis 8 mg | 1 | 21 | 1 |
| other | Wound infection | Infections and infestations | Engensis 16 mg | 2 | 20 | 3 |
| other | Wound infection | Infections and infestations | Placebo | 0 | 11 | 0 |
| other | Cystitis | Infections and infestations | Engensis 8 mg | 0 | 21 | 0 |
1 to 10 of 192Source ClinicalTrials.gov
See all 192 adverse eventsAdverse event totals9
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Engensis 8 mg | 1 | 21 |
| Total, all-cause mortality | Engensis 16 mg | 0 | 20 |
| Total, all-cause mortality | Placebo | 1 | 11 |
| Total, other adverse events | Engensis 8 mg | 18 | 21 |
| Total, other adverse events | Engensis 16 mg | 18 | 20 |
| Total, other adverse events | Placebo | 10 | 11 |
| Total, serious adverse events | Engensis 8 mg | 9 | 21 |
| Total, serious adverse events | Engensis 16 mg | 11 | 20 |
| Total, serious adverse events | Placebo | 6 | 11 |
Source ClinicalTrials.gov
Papers1
The papers the trial lists on ClinicalTrials.gov.
| PMID | Listed as |
|---|---|
| 26649448 | BACKGROUND |
Source PubMed
Grants0
The grants NIH RePORTER links to the trial, newest funded first: NIH's, and some of AHRQ's, CDC's, FDA's and VA's. Each shows its title, principal investigators, organization and funder in its newest year.
None in this release.
Trial comparisons0
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
None in this release.
Tests0
The ConductCognition tests this trial's registered outcomes name, as ScienceGraph matched them in trial-tests-2026-11-12-0.2.1.
None in this release.
Data dates
The day each source was copied for this release.
Sources 3
- ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
- ClinicalTrials.govNo license stated
- NIH RePORTER's clinical studies and project filesPublic domain
Each source's terms
- ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
- ClinicalTrials.govnone stated
- NIH RePORTER's clinical studies and project filesPublic domain (NIH RePORTER)
ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.
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