Trial Outcomes & Findings for Safety Study of Gene Therapy in Treating Critical Leg Ischemia (NCT NCT00696124)
NCT ID: NCT00696124
Last Updated: 2025-10-06
Results Overview
Treatment-emergent adverse events defined as adverse events after the first dose of Engensis (Day 1) through Day 365
Recruitment status
COMPLETED
Study phase
PHASE1
Target enrollment
12 participants
Primary outcome timeframe
Day 1 to Day 365
Results posted on
2025-10-06
Participant Flow
Participant milestones
| Measure |
Cohort 1
Engensis 2 mg (VM202). The 2 mg intramuscular injection was given on Day 1 (first half of the total dose) and Day 15 (the second half of the total dose)
|
Cohort 2
Engensis 4 mg (VM202). The 4 mg intramuscular injection was given on Day 1 (first half of the total dose) and Day 15 (the second half of the total dose)
|
Cohort 3
Engensis 8 mg (VM202). The 8 mg intramuscular injection was given on Day 1 (first half of the total dose) and Day 15 (the second half of the total dose)
|
Cohort 4
Engensis 16 mg (VM202). The 16 mg intramuscular injection was given on Day 1 (first half of the total dose) and Day 15 (the second half of the total dose)
|
|---|---|---|---|---|
|
Overall Study
STARTED
|
3
|
3
|
3
|
3
|
|
Overall Study
COMPLETED
|
3
|
3
|
3
|
3
|
|
Overall Study
NOT COMPLETED
|
0
|
0
|
0
|
0
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
Safety Study of Gene Therapy in Treating Critical Leg Ischemia
Baseline characteristics by cohort
| Measure |
Cohort 1
n=3 Participants
Engensis 2 mg (VM202). The 2 mg intramuscular injection was given on Day 1 (first half of the total dose) and Day 15 (the second half of the total dose)
|
Cohort 2
n=3 Participants
Engensis 4 mg (VM202). The 4 mg intramuscular injection was given on Day 1 (first half of the total dose) and Day 15 (the second half of the total dose)
|
Cohort 3
n=3 Participants
Engensis 8 mg (VM202). The 8 mg intramuscular injection was given on Day 1 (first half of the total dose) and Day 15 (the second half of the total dose)
|
Cohort 4
n=3 Participants
Engensis 16 mg (VM202). The 16 mg intramuscular injection was given on Day 1 (first half of the total dose) and Day 15 (the second half of the total dose)
|
Total
n=12 Participants
Total of all reporting groups
|
|---|---|---|---|---|---|
|
Age, Continuous
|
67.0 years
STANDARD_DEVIATION 15.1 • n=5 Participants
|
70.0 years
STANDARD_DEVIATION 5.3 • n=7 Participants
|
70.3 years
STANDARD_DEVIATION 9.0 • n=5 Participants
|
63.7 years
STANDARD_DEVIATION 20.8 • n=4 Participants
|
67.8 years
STANDARD_DEVIATION 12.6 • n=21 Participants
|
|
Sex: Female, Male
Female
|
1 Participants
n=5 Participants
|
1 Participants
n=7 Participants
|
1 Participants
n=5 Participants
|
2 Participants
n=4 Participants
|
5 Participants
n=21 Participants
|
|
Sex: Female, Male
Male
|
2 Participants
n=5 Participants
|
2 Participants
n=7 Participants
|
2 Participants
n=5 Participants
|
1 Participants
n=4 Participants
|
7 Participants
n=21 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
0 Participants
n=4 Participants
|
0 Participants
n=21 Participants
|
|
Race (NIH/OMB)
Asian
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
0 Participants
n=4 Participants
|
0 Participants
n=21 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
0 Participants
n=4 Participants
|
0 Participants
n=21 Participants
|
|
Race (NIH/OMB)
Black or African American
|
1 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
0 Participants
n=4 Participants
|
1 Participants
n=21 Participants
|
|
Race (NIH/OMB)
White
|
2 Participants
n=5 Participants
|
3 Participants
n=7 Participants
|
3 Participants
n=5 Participants
|
3 Participants
n=4 Participants
|
11 Participants
n=21 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
0 Participants
n=4 Participants
|
0 Participants
n=21 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
0 Participants
n=4 Participants
|
0 Participants
n=21 Participants
|
|
Region of Enrollment
United States
|
3 participants
n=5 Participants
|
3 participants
n=7 Participants
|
3 participants
n=5 Participants
|
3 participants
n=4 Participants
|
12 participants
n=21 Participants
|
PRIMARY outcome
Timeframe: Day 1 to Day 365Population: Safety population included all subjects who received at least one dose of study drug.
Treatment-emergent adverse events defined as adverse events after the first dose of Engensis (Day 1) through Day 365
Outcome measures
| Measure |
Cohort 1
n=3 Participants
Engensis 2 mg (VM202). The 2 mg intramuscular injection was given on Day 1 (first half of the total dose) and Day 15 (the second half of the total dose)
|
Cohort 2
n=3 Participants
Engensis 4 mg (VM202). The 4 mg intramuscular injection was given on Day 1 (first half of the total dose) and Day 15 (the second half of the total dose)
|
Cohort 3
n=3 Participants
Engensis 8 mg (VM202). The 8 mg intramuscular injection was given on Day 1 (first half of the total dose) and Day 15 (the second half of the total dose)
|
Cohort 4
n=3 Participants
Engensis 16 mg (VM202). The 16 mg intramuscular injection was given on Day 1 (first half of the total dose) and Day 15 (the second half of the total dose)
|
|---|---|---|---|---|
|
Treatment-Emergent Adverse Events.
Tongue injury
|
1 Participants
|
0 Participants
|
0 Participants
|
0 Participants
|
|
Treatment-Emergent Adverse Events.
Injury, Poisoning and Procedural Complications
|
1 Participants
|
0 Participants
|
0 Participants
|
0 Participants
|
|
Treatment-Emergent Adverse Events.
Upper respiratory infection
|
0 Participants
|
1 Participants
|
1 Participants
|
0 Participants
|
|
Treatment-Emergent Adverse Events.
Cellulitis
|
1 Participants
|
0 Participants
|
0 Participants
|
0 Participants
|
|
Treatment-Emergent Adverse Events.
Chronic sinusitis
|
0 Participants
|
1 Participants
|
0 Participants
|
0 Participants
|
|
Treatment-Emergent Adverse Events.
Wound infection
|
0 Participants
|
0 Participants
|
1 Participants
|
0 Participants
|
|
Treatment-Emergent Adverse Events.
General Disorders and Administration Site Conditions
|
2 Participants
|
0 Participants
|
1 Participants
|
0 Participants
|
|
Treatment-Emergent Adverse Events.
Chest pain
|
1 Participants
|
0 Participants
|
0 Participants
|
0 Participants
|
|
Treatment-Emergent Adverse Events.
Injection site bruising
|
1 Participants
|
0 Participants
|
0 Participants
|
0 Participants
|
|
Treatment-Emergent Adverse Events.
Pyrexia
|
0 Participants
|
0 Participants
|
1 Participants
|
0 Participants
|
|
Treatment-Emergent Adverse Events.
Musculoskeletal and Connective Tissue Disorders
|
0 Participants
|
1 Participants
|
1 Participants
|
1 Participants
|
|
Treatment-Emergent Adverse Events.
Pain in extremity
|
0 Participants
|
1 Participants
|
1 Participants
|
0 Participants
|
|
Treatment-Emergent Adverse Events.
Muscle spasms
|
0 Participants
|
0 Participants
|
0 Participants
|
1 Participants
|
|
Treatment-Emergent Adverse Events.
Musculoskeletal pain
|
0 Participants
|
0 Participants
|
0 Participants
|
1 Participants
|
|
Treatment-Emergent Adverse Events.
Neoplasms Benign, Malignant and Unspecified (Including Cysts and Polyps)
|
1 Participants
|
1 Participants
|
1 Participants
|
0 Participants
|
|
Treatment-Emergent Adverse Events.
Biliary adenoma
|
0 Participants
|
1 Participants
|
0 Participants
|
0 Participants
|
|
Treatment-Emergent Adverse Events.
Colon cancer
|
0 Participants
|
0 Participants
|
1 Participants
|
0 Participants
|
|
Treatment-Emergent Adverse Events.
Metastases to liver
|
1 Participants
|
0 Participants
|
0 Participants
|
0 Participants
|
|
Treatment-Emergent Adverse Events.
Small cell lung cancer stage unspecified
|
1 Participants
|
0 Participants
|
0 Participants
|
0 Participants
|
|
Treatment-Emergent Adverse Events.
Respiratory, Thoracic and Mediastinal Disorders
|
1 Participants
|
2 Participants
|
0 Participants
|
0 Participants
|
|
Treatment-Emergent Adverse Events.
Rhonchi
|
1 Participants
|
0 Participants
|
0 Participants
|
0 Participants
|
|
Treatment-Emergent Adverse Events.
Throat irritation
|
0 Participants
|
1 Participants
|
0 Participants
|
0 Participants
|
|
Treatment-Emergent Adverse Events.
Wheezing
|
0 Participants
|
1 Participants
|
0 Participants
|
0 Participants
|
|
Treatment-Emergent Adverse Events.
Vascular Disorders
|
1 Participants
|
0 Participants
|
2 Participants
|
0 Participants
|
|
Treatment-Emergent Adverse Events.
Peripheral arterial occlusive disease
|
0 Participants
|
0 Participants
|
2 Participants
|
0 Participants
|
|
Treatment-Emergent Adverse Events.
Haematoma
|
1 Participants
|
0 Participants
|
0 Participants
|
0 Participants
|
|
Treatment-Emergent Adverse Events.
Hypotension
|
0 Participants
|
0 Participants
|
1 Participants
|
0 Participants
|
|
Treatment-Emergent Adverse Events.
Blood and Lymphatic System Disorders
|
0 Participants
|
1 Participants
|
1 Participants
|
0 Participants
|
|
Treatment-Emergent Adverse Events.
Anaemia
|
0 Participants
|
1 Participants
|
1 Participants
|
0 Participants
|
|
Treatment-Emergent Adverse Events.
Cardiac Disorders
|
1 Participants
|
1 Participants
|
0 Participants
|
0 Participants
|
|
Treatment-Emergent Adverse Events.
Bundle branch block left
|
1 Participants
|
0 Participants
|
0 Participants
|
0 Participants
|
|
Treatment-Emergent Adverse Events.
Cyanosis
|
0 Participants
|
1 Participants
|
0 Participants
|
0 Participants
|
|
Treatment-Emergent Adverse Events.
Gastrointestinal Disorders
|
1 Participants
|
0 Participants
|
1 Participants
|
0 Participants
|
|
Treatment-Emergent Adverse Events.
Gastric ulcer
|
0 Participants
|
0 Participants
|
1 Participants
|
0 Participants
|
|
Treatment-Emergent Adverse Events.
Ileus
|
1 Participants
|
0 Participants
|
0 Participants
|
0 Participants
|
|
Treatment-Emergent Adverse Events.
Investigations
|
1 Participants
|
0 Participants
|
1 Participants
|
0 Participants
|
|
Treatment-Emergent Adverse Events.
Blood creatinine increased
|
0 Participants
|
0 Participants
|
1 Participants
|
0 Participants
|
|
Treatment-Emergent Adverse Events.
Blood glucose increased
|
0 Participants
|
0 Participants
|
1 Participants
|
0 Participants
|
|
Treatment-Emergent Adverse Events.
Glucose urine present
|
0 Participants
|
0 Participants
|
1 Participants
|
0 Participants
|
|
Treatment-Emergent Adverse Events.
International normalized ratio increased
|
0 Participants
|
0 Participants
|
1 Participants
|
0 Participants
|
|
Treatment-Emergent Adverse Events.
Prostatic specific antigen increased
|
1 Participants
|
0 Participants
|
0 Participants
|
0 Participants
|
|
Treatment-Emergent Adverse Events.
Skin and Subcutaneous Tissue Disorders
|
0 Participants
|
1 Participants
|
0 Participants
|
1 Participants
|
|
Treatment-Emergent Adverse Events.
Erythema
|
0 Participants
|
1 Participants
|
0 Participants
|
0 Participants
|
|
Treatment-Emergent Adverse Events.
Pruritus
|
0 Participants
|
0 Participants
|
0 Participants
|
1 Participants
|
|
Treatment-Emergent Adverse Events.
Skin ulcer
|
0 Participants
|
0 Participants
|
0 Participants
|
1 Participants
|
|
Treatment-Emergent Adverse Events.
Psychiatric Disorders
|
1 Participants
|
0 Participants
|
0 Participants
|
0 Participants
|
|
Treatment-Emergent Adverse Events.
Confusional state
|
1 Participants
|
0 Participants
|
0 Participants
|
0 Participants
|
|
Treatment-Emergent Adverse Events.
Subjects with at least one TEAE
|
3 Participants
|
3 Participants
|
3 Participants
|
3 Participants
|
|
Treatment-Emergent Adverse Events.
Infections and Infestations
|
2 Participants
|
1 Participants
|
3 Participants
|
1 Participants
|
|
Treatment-Emergent Adverse Events.
Urinary tract infection
|
2 Participants
|
0 Participants
|
1 Participants
|
1 Participants
|
SECONDARY outcome
Timeframe: Days 15, 28, 59, 91, 180, and 365Population: The Intent-to-Treat population included all subjects who received at least one dose of study drug medication and had at least one post-dose assessment
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).
Outcome measures
| Measure |
Cohort 1
n=3 Participants
Engensis 2 mg (VM202). The 2 mg intramuscular injection was given on Day 1 (first half of the total dose) and Day 15 (the second half of the total dose)
|
Cohort 2
n=3 Participants
Engensis 4 mg (VM202). The 4 mg intramuscular injection was given on Day 1 (first half of the total dose) and Day 15 (the second half of the total dose)
|
Cohort 3
n=3 Participants
Engensis 8 mg (VM202). The 8 mg intramuscular injection was given on Day 1 (first half of the total dose) and Day 15 (the second half of the total dose)
|
Cohort 4
n=3 Participants
Engensis 16 mg (VM202). The 16 mg intramuscular injection was given on Day 1 (first half of the total dose) and Day 15 (the second half of the total dose)
|
|---|---|---|---|---|
|
Change From Baseline in Pain Visual Analog Scale
Baseline (Day 0) Actual values only
|
66.7 score on a scale
Standard Deviation 20.79
|
64.3 score on a scale
Standard Deviation 12.90
|
38.3 score on a scale
Standard Deviation 45.79
|
50.3 score on a scale
Standard Deviation 20.31
|
|
Change From Baseline in Pain Visual Analog Scale
Day 15
|
-0.3 score on a scale
Standard Deviation 19.30
|
-20.7 score on a scale
Standard Deviation 22.19
|
1.7 score on a scale
Standard Deviation 9.87
|
7.0 score on a scale
Standard Deviation 19.67
|
|
Change From Baseline in Pain Visual Analog Scale
Day 28
|
4.0 score on a scale
Standard Deviation 14.42
|
-53.7 score on a scale
Standard Deviation 18.88
|
8.7 score on a scale
Standard Deviation 7.23
|
-15.3 score on a scale
Standard Deviation 3.06
|
|
Change From Baseline in Pain Visual Analog Scale
Day 59
|
-8.3 score on a scale
Standard Deviation 30.01
|
-52.0 score on a scale
Standard Deviation 10.39
|
13.0 score on a scale
Standard Deviation 9.00
|
-26.3 score on a scale
Standard Deviation 14.57
|
|
Change From Baseline in Pain Visual Analog Scale
Day 91
|
-3.3 score on a scale
Standard Deviation 29.02
|
-56.3 score on a scale
Standard Deviation 9.07
|
1.0 score on a scale
Standard Deviation 11.36
|
0.0 score on a scale
Standard Deviation 38.11
|
|
Change From Baseline in Pain Visual Analog Scale
Day 180
|
-12.3 score on a scale
Standard Deviation 22.68
|
-63.3 score on a scale
Standard Deviation 12.22
|
31.0 score on a scale
Standard Deviation 55.15
|
-29.7 score on a scale
Standard Deviation 19.55
|
|
Change From Baseline in Pain Visual Analog Scale
Day 365
|
-13.7 score on a scale
Standard Deviation 35.5
|
-63.3 score on a scale
Standard Deviation 13.61
|
37.5 score on a scale
Standard Deviation 58.69
|
4.3 score on a scale
Standard Deviation 9.87
|
SECONDARY outcome
Timeframe: Days 15, 28, 59, 91, 180, and 365Population: Intent-to-treat population, defined as all subjects who received at least one dose of study drug and had at least one post-dose assessment
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).
Outcome measures
| Measure |
Cohort 1
n=3 Participants
Engensis 2 mg (VM202). The 2 mg intramuscular injection was given on Day 1 (first half of the total dose) and Day 15 (the second half of the total dose)
|
Cohort 2
n=3 Participants
Engensis 4 mg (VM202). The 4 mg intramuscular injection was given on Day 1 (first half of the total dose) and Day 15 (the second half of the total dose)
|
Cohort 3
n=3 Participants
Engensis 8 mg (VM202). The 8 mg intramuscular injection was given on Day 1 (first half of the total dose) and Day 15 (the second half of the total dose)
|
Cohort 4
n=3 Participants
Engensis 16 mg (VM202). The 16 mg intramuscular injection was given on Day 1 (first half of the total dose) and Day 15 (the second half of the total dose)
|
|---|---|---|---|---|
|
Change From Baseline in Ankle Brachial Index
Day 15
|
0.10 Ratio Systolic BP-Change ankle to arm
Standard Deviation 0.12
|
0.05 Ratio Systolic BP-Change ankle to arm
Standard Deviation 0.04
|
0.02 Ratio Systolic BP-Change ankle to arm
Standard Deviation 0.02
|
0.04 Ratio Systolic BP-Change ankle to arm
Standard Deviation 0.14
|
|
Change From Baseline in Ankle Brachial Index
Day 28
|
0.0 Ratio Systolic BP-Change ankle to arm
Standard Deviation 0.14
|
0.14 Ratio Systolic BP-Change ankle to arm
Standard Deviation 0.07
|
-0.06 Ratio Systolic BP-Change ankle to arm
Standard Deviation 0.17
|
-0.08 Ratio Systolic BP-Change ankle to arm
Standard Deviation 0.38
|
|
Change From Baseline in Ankle Brachial Index
Day 59
|
0.08 Ratio Systolic BP-Change ankle to arm
Standard Deviation 0.08
|
0.14 Ratio Systolic BP-Change ankle to arm
Standard Deviation 0.14
|
-0.10 Ratio Systolic BP-Change ankle to arm
Standard Deviation 0.17
|
0.13 Ratio Systolic BP-Change ankle to arm
Standard Deviation 0.24
|
|
Change From Baseline in Ankle Brachial Index
Day 91
|
0.14 Ratio Systolic BP-Change ankle to arm
Standard Deviation 0.09
|
0.05 Ratio Systolic BP-Change ankle to arm
Standard Deviation 0.13
|
-0.03 Ratio Systolic BP-Change ankle to arm
Standard Deviation 0.03
|
0.09 Ratio Systolic BP-Change ankle to arm
Standard Deviation 0.32
|
|
Change From Baseline in Ankle Brachial Index
Day 180
|
0.06 Ratio Systolic BP-Change ankle to arm
Standard Deviation 0.14
|
0.07 Ratio Systolic BP-Change ankle to arm
Standard Deviation 0.11
|
-0.05 Ratio Systolic BP-Change ankle to arm
Standard Deviation 0.08
|
0.15 Ratio Systolic BP-Change ankle to arm
Standard Deviation 0.05
|
|
Change From Baseline in Ankle Brachial Index
Day 365
|
0.12 Ratio Systolic BP-Change ankle to arm
Standard Deviation 0.06
|
0.21 Ratio Systolic BP-Change ankle to arm
Standard Deviation 0.15
|
0.13 Ratio Systolic BP-Change ankle to arm
Standard Deviation NA
No SD available for 1 participant
|
0.13 Ratio Systolic BP-Change ankle to arm
Standard Deviation 0.19
|
|
Change From Baseline in Ankle Brachial Index
Baseline (Day 0) Actual values only
|
0.35 Ratio Systolic BP-Change ankle to arm
Standard Deviation 0.10
|
0.35 Ratio Systolic BP-Change ankle to arm
Standard Deviation 0.04
|
0.44 Ratio Systolic BP-Change ankle to arm
Standard Deviation 0.06
|
0.57 Ratio Systolic BP-Change ankle to arm
Standard Deviation 0.43
|
SECONDARY outcome
Timeframe: Baseline and Days 1,15,28,59,91,180,and 365Population: Intent-to-treat population defined as all subjects who received at least one dose of study drug and had at least one post-dose assessment
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).
Outcome measures
| Measure |
Cohort 1
n=3 Participants
Engensis 2 mg (VM202). The 2 mg intramuscular injection was given on Day 1 (first half of the total dose) and Day 15 (the second half of the total dose)
|
Cohort 2
n=3 Participants
Engensis 4 mg (VM202). The 4 mg intramuscular injection was given on Day 1 (first half of the total dose) and Day 15 (the second half of the total dose)
|
Cohort 3
n=3 Participants
Engensis 8 mg (VM202). The 8 mg intramuscular injection was given on Day 1 (first half of the total dose) and Day 15 (the second half of the total dose)
|
Cohort 4
n=3 Participants
Engensis 16 mg (VM202). The 16 mg intramuscular injection was given on Day 1 (first half of the total dose) and Day 15 (the second half of the total dose)
|
|---|---|---|---|---|
|
Change From Baseline in Toe Brachial Index
Baseline (Day 0) Actual values only
|
0.16 Ratio Systolic BP-Change toe to arm
Standard Deviation 0.04
|
0.19 Ratio Systolic BP-Change toe to arm
Standard Deviation 0.01
|
0.18 Ratio Systolic BP-Change toe to arm
Standard Deviation 0.11
|
0.22 Ratio Systolic BP-Change toe to arm
Standard Deviation 0.26
|
|
Change From Baseline in Toe Brachial Index
Day 15
|
0.0 Ratio Systolic BP-Change toe to arm
Standard Deviation 0.07
|
0.07 Ratio Systolic BP-Change toe to arm
Standard Deviation 0.05
|
-0.05 Ratio Systolic BP-Change toe to arm
Standard Deviation 0.13
|
0.22 Ratio Systolic BP-Change toe to arm
Standard Deviation 0.01
|
|
Change From Baseline in Toe Brachial Index
Day 28
|
-0.01 Ratio Systolic BP-Change toe to arm
Standard Deviation 0.09
|
-0.01 Ratio Systolic BP-Change toe to arm
Standard Deviation 0.02
|
-0.02 Ratio Systolic BP-Change toe to arm
Standard Deviation 0.08
|
0.03 Ratio Systolic BP-Change toe to arm
Standard Deviation 0.01
|
|
Change From Baseline in Toe Brachial Index
Day 59
|
0.08 Ratio Systolic BP-Change toe to arm
Standard Deviation 0.09
|
0.05 Ratio Systolic BP-Change toe to arm
Standard Deviation 0.05
|
-0.04 Ratio Systolic BP-Change toe to arm
Standard Deviation 0.09
|
0.14 Ratio Systolic BP-Change toe to arm
Standard Deviation 0.03
|
|
Change From Baseline in Toe Brachial Index
Day 91
|
0.04 Ratio Systolic BP-Change toe to arm
Standard Deviation 0.15
|
0.06 Ratio Systolic BP-Change toe to arm
Standard Deviation 0.04
|
-0.08 Ratio Systolic BP-Change toe to arm
Standard Deviation 0.08
|
0.12 Ratio Systolic BP-Change toe to arm
Standard Deviation 0.06
|
|
Change From Baseline in Toe Brachial Index
Day 180
|
0.05 Ratio Systolic BP-Change toe to arm
Standard Deviation 0.06
|
0.10 Ratio Systolic BP-Change toe to arm
Standard Deviation 0.12
|
0.01 Ratio Systolic BP-Change toe to arm
Standard Deviation 0.12
|
0.01 Ratio Systolic BP-Change toe to arm
Standard Deviation 0.22
|
|
Change From Baseline in Toe Brachial Index
Day 365
|
0.10 Ratio Systolic BP-Change toe to arm
Standard Deviation 0.07
|
0.14 Ratio Systolic BP-Change toe to arm
Standard Deviation 0.11
|
-0.08 Ratio Systolic BP-Change toe to arm
|
0.11 Ratio Systolic BP-Change toe to arm
Standard Deviation 0.03
|
SECONDARY outcome
Timeframe: Days 1 to 365Population: Intent-to-treat population defined as all subjects who received at least one dose of study drug and had at least one post-dose assessment
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
Outcome measures
| Measure |
Cohort 1
n=3 Participants
Engensis 2 mg (VM202). The 2 mg intramuscular injection was given on Day 1 (first half of the total dose) and Day 15 (the second half of the total dose)
|
Cohort 2
n=3 Participants
Engensis 4 mg (VM202). The 4 mg intramuscular injection was given on Day 1 (first half of the total dose) and Day 15 (the second half of the total dose)
|
Cohort 3
n=3 Participants
Engensis 8 mg (VM202). The 8 mg intramuscular injection was given on Day 1 (first half of the total dose) and Day 15 (the second half of the total dose)
|
Cohort 4
n=3 Participants
Engensis 16 mg (VM202). The 16 mg intramuscular injection was given on Day 1 (first half of the total dose) and Day 15 (the second half of the total dose)
|
|---|---|---|---|---|
|
Change From Baseline in Transcutaneous Oxygen Pressure
Baseline (Day 0) Actual values only
|
48.7 mmHg
Standard Deviation 12.10
|
47.0 mmHg
Standard Deviation 18.03
|
38.3 mmHg
Standard Deviation 11.02
|
70.7 mmHg
Standard Deviation 4.51
|
|
Change From Baseline in Transcutaneous Oxygen Pressure
Day 91
|
10.0 mmHg
Standard Deviation 8.72
|
4.3 mmHg
Standard Deviation 13.87
|
2.7 mmHg
Standard Deviation 13.32
|
-1.3 mmHg
Standard Deviation 5.13
|
|
Change From Baseline in Transcutaneous Oxygen Pressure
Day 180
|
15.0 mmHg
Standard Deviation 10.54
|
13.0 mmHg
Standard Deviation 6.24
|
10.0 mmHg
Standard Deviation 11.31
|
-16.3 mmHg
Standard Deviation 21.96
|
|
Change From Baseline in Transcutaneous Oxygen Pressure
Day 365
|
9.0 mmHg
Standard Deviation 7.81
|
7.3 mmHg
Standard Deviation 7.64
|
25.5 mmHg
Standard Deviation 2.12
|
-3.7 mmHg
Standard Deviation 7.37
|
Adverse Events
Cohort 1
Serious events: 2 serious events
Other events: 3 other events
Deaths: 1 deaths
Cohort 2
Serious events: 1 serious events
Other events: 3 other events
Deaths: 0 deaths
Cohort 3
Serious events: 3 serious events
Other events: 3 other events
Deaths: 0 deaths
Cohort 4
Serious events: 0 serious events
Other events: 3 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
Cohort 1
n=3 participants at risk
Engensis 2 mg (VM202). The 2 mg intramuscular injection was given on Day 1 (first half of the total dose) and Day 15 (the second half of the total dose)
|
Cohort 2
n=3 participants at risk
Engensis 4 mg (VM202). The 4 mg intramuscular injection was given on Day 1 (first half of the total dose) and Day 15 (the second half of the total dose)
|
Cohort 3
n=3 participants at risk
Engensis 8 mg (VM202). The 8 mg intramuscular injection was given on Day 1 (first half of the total dose) and Day 15 (the second half of the total dose)
|
Cohort 4
n=3 participants at risk
Engensis 16 mg (VM202). The 16 mg intramuscular injection was given on Day 1 (first half of the total dose) and Day 15 (the second half of the total dose)
|
|---|---|---|---|---|
|
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Metastases to Liver
|
33.3%
1/3 • Number of events 1 • Day 1 through Day 365
|
0.00%
0/3 • Day 1 through Day 365
|
0.00%
0/3 • Day 1 through Day 365
|
0.00%
0/3 • Day 1 through Day 365
|
|
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Small Cell Lung Cancer - Stage Unspecified
|
33.3%
1/3 • Number of events 1 • Day 1 through Day 365
|
0.00%
0/3 • Day 1 through Day 365
|
0.00%
0/3 • Day 1 through Day 365
|
0.00%
0/3 • Day 1 through Day 365
|
|
Infections and infestations
Urinary tract infection
|
33.3%
1/3 • Number of events 1 • Day 1 through Day 365
|
0.00%
0/3 • Day 1 through Day 365
|
33.3%
1/3 • Number of events 1 • Day 1 through Day 365
|
0.00%
0/3 • Day 1 through Day 365
|
|
Blood and lymphatic system disorders
Anaemia
|
0.00%
0/3 • Day 1 through Day 365
|
33.3%
1/3 • Number of events 1 • Day 1 through Day 365
|
0.00%
0/3 • Day 1 through Day 365
|
0.00%
0/3 • Day 1 through Day 365
|
|
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Biliary Adenoma
|
0.00%
0/3 • Day 1 through Day 365
|
33.3%
1/3 • Number of events 1 • Day 1 through Day 365
|
0.00%
0/3 • Day 1 through Day 365
|
0.00%
0/3 • Day 1 through Day 365
|
|
Infections and infestations
Wound infection
|
0.00%
0/3 • Day 1 through Day 365
|
0.00%
0/3 • Day 1 through Day 365
|
33.3%
1/3 • Number of events 1 • Day 1 through Day 365
|
0.00%
0/3 • Day 1 through Day 365
|
|
Vascular disorders
Peripheral Arterial Occlusive Disease
|
0.00%
0/3 • Day 1 through Day 365
|
0.00%
0/3 • Day 1 through Day 365
|
33.3%
1/3 • Number of events 2 • Day 1 through Day 365
|
0.00%
0/3 • Day 1 through Day 365
|
|
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Colon cancer
|
0.00%
0/3 • Day 1 through Day 365
|
0.00%
0/3 • Day 1 through Day 365
|
33.3%
1/3 • Number of events 1 • Day 1 through Day 365
|
0.00%
0/3 • Day 1 through Day 365
|
Other adverse events
| Measure |
Cohort 1
n=3 participants at risk
Engensis 2 mg (VM202). The 2 mg intramuscular injection was given on Day 1 (first half of the total dose) and Day 15 (the second half of the total dose)
|
Cohort 2
n=3 participants at risk
Engensis 4 mg (VM202). The 4 mg intramuscular injection was given on Day 1 (first half of the total dose) and Day 15 (the second half of the total dose)
|
Cohort 3
n=3 participants at risk
Engensis 8 mg (VM202). The 8 mg intramuscular injection was given on Day 1 (first half of the total dose) and Day 15 (the second half of the total dose)
|
Cohort 4
n=3 participants at risk
Engensis 16 mg (VM202). The 16 mg intramuscular injection was given on Day 1 (first half of the total dose) and Day 15 (the second half of the total dose)
|
|---|---|---|---|---|
|
Infections and infestations
Urinary tract infection
|
33.3%
1/3 • Number of events 2 • Day 1 through Day 365
|
0.00%
0/3 • Day 1 through Day 365
|
33.3%
1/3 • Number of events 2 • Day 1 through Day 365
|
33.3%
1/3 • Number of events 1 • Day 1 through Day 365
|
|
Infections and infestations
Upper respiratory tract infection
|
0.00%
0/3 • Day 1 through Day 365
|
33.3%
1/3 • Number of events 1 • Day 1 through Day 365
|
33.3%
1/3 • Number of events 1 • Day 1 through Day 365
|
0.00%
0/3 • Day 1 through Day 365
|
|
Infections and infestations
Cellulitis
|
33.3%
1/3 • Number of events 1 • Day 1 through Day 365
|
0.00%
0/3 • Day 1 through Day 365
|
0.00%
0/3 • Day 1 through Day 365
|
0.00%
0/3 • Day 1 through Day 365
|
|
General disorders
Chest pain
|
33.3%
1/3 • Number of events 1 • Day 1 through Day 365
|
0.00%
0/3 • Day 1 through Day 365
|
0.00%
0/3 • Day 1 through Day 365
|
0.00%
0/3 • Day 1 through Day 365
|
|
General disorders
Injection site bruising
|
33.3%
1/3 • Number of events 1 • Day 1 through Day 365
|
0.00%
0/3 • Day 1 through Day 365
|
0.00%
0/3 • Day 1 through Day 365
|
0.00%
0/3 • Day 1 through Day 365
|
|
General disorders
Pyrexia
|
0.00%
0/3 • Day 1 through Day 365
|
0.00%
0/3 • Day 1 through Day 365
|
33.3%
1/3 • Number of events 1 • Day 1 through Day 365
|
0.00%
0/3 • Day 1 through Day 365
|
|
Musculoskeletal and connective tissue disorders
Pain in Extremity
|
0.00%
0/3 • Day 1 through Day 365
|
33.3%
1/3 • Number of events 1 • Day 1 through Day 365
|
33.3%
1/3 • Number of events 2 • Day 1 through Day 365
|
0.00%
0/3 • Day 1 through Day 365
|
|
Musculoskeletal and connective tissue disorders
Muscle spasms
|
0.00%
0/3 • Day 1 through Day 365
|
0.00%
0/3 • Day 1 through Day 365
|
0.00%
0/3 • Day 1 through Day 365
|
33.3%
1/3 • Number of events 1 • Day 1 through Day 365
|
|
Musculoskeletal and connective tissue disorders
Musculoskeletal pain
|
0.00%
0/3 • Day 1 through Day 365
|
0.00%
0/3 • Day 1 through Day 365
|
0.00%
0/3 • Day 1 through Day 365
|
33.3%
1/3 • Number of events 1 • Day 1 through Day 365
|
|
Respiratory, thoracic and mediastinal disorders
Rhonchi
|
33.3%
1/3 • Number of events 1 • Day 1 through Day 365
|
0.00%
0/3 • Day 1 through Day 365
|
0.00%
0/3 • Day 1 through Day 365
|
0.00%
0/3 • Day 1 through Day 365
|
|
Respiratory, thoracic and mediastinal disorders
Throat irritation
|
0.00%
0/3 • Day 1 through Day 365
|
33.3%
1/3 • Number of events 1 • Day 1 through Day 365
|
0.00%
0/3 • Day 1 through Day 365
|
0.00%
0/3 • Day 1 through Day 365
|
|
Respiratory, thoracic and mediastinal disorders
Wheezing
|
0.00%
0/3 • Day 1 through Day 365
|
33.3%
1/3 • Number of events 1 • Day 1 through Day 365
|
0.00%
0/3 • Day 1 through Day 365
|
0.00%
0/3 • Day 1 through Day 365
|
|
Vascular disorders
Peripheral arterial occlusive disease
|
0.00%
0/3 • Day 1 through Day 365
|
0.00%
0/3 • Day 1 through Day 365
|
33.3%
1/3 • Number of events 2 • Day 1 through Day 365
|
0.00%
0/3 • Day 1 through Day 365
|
|
Vascular disorders
Haematoma
|
33.3%
1/3 • Number of events 1 • Day 1 through Day 365
|
0.00%
0/3 • Day 1 through Day 365
|
0.00%
0/3 • Day 1 through Day 365
|
0.00%
0/3 • Day 1 through Day 365
|
|
Vascular disorders
Hypotension
|
0.00%
0/3 • Day 1 through Day 365
|
0.00%
0/3 • Day 1 through Day 365
|
33.3%
1/3 • Number of events 1 • Day 1 through Day 365
|
0.00%
0/3 • Day 1 through Day 365
|
|
Blood and lymphatic system disorders
Anaemia
|
0.00%
0/3 • Day 1 through Day 365
|
33.3%
1/3 • Number of events 2 • Day 1 through Day 365
|
33.3%
1/3 • Number of events 1 • Day 1 through Day 365
|
0.00%
0/3 • Day 1 through Day 365
|
|
Cardiac disorders
Bundle Branch Block Left
|
33.3%
1/3 • Number of events 1 • Day 1 through Day 365
|
0.00%
0/3 • Day 1 through Day 365
|
0.00%
0/3 • Day 1 through Day 365
|
0.00%
0/3 • Day 1 through Day 365
|
|
Cardiac disorders
Cyanosis
|
0.00%
0/3 • Day 1 through Day 365
|
33.3%
1/3 • Number of events 1 • Day 1 through Day 365
|
0.00%
0/3 • Day 1 through Day 365
|
0.00%
0/3 • Day 1 through Day 365
|
|
Gastrointestinal disorders
Gastric ulcer
|
0.00%
0/3 • Day 1 through Day 365
|
0.00%
0/3 • Day 1 through Day 365
|
33.3%
1/3 • Number of events 1 • Day 1 through Day 365
|
0.00%
0/3 • Day 1 through Day 365
|
|
Gastrointestinal disorders
Ileus
|
33.3%
1/3 • Number of events 1 • Day 1 through Day 365
|
0.00%
0/3 • Day 1 through Day 365
|
0.00%
0/3 • Day 1 through Day 365
|
0.00%
0/3 • Day 1 through Day 365
|
|
Investigations
Blood creatinine increased
|
0.00%
0/3 • Day 1 through Day 365
|
0.00%
0/3 • Day 1 through Day 365
|
33.3%
1/3 • Number of events 1 • Day 1 through Day 365
|
0.00%
0/3 • Day 1 through Day 365
|
|
Investigations
Blood glucose increased
|
0.00%
0/3 • Day 1 through Day 365
|
0.00%
0/3 • Day 1 through Day 365
|
33.3%
1/3 • Number of events 1 • Day 1 through Day 365
|
0.00%
0/3 • Day 1 through Day 365
|
|
Investigations
Glucose urine present
|
0.00%
0/3 • Day 1 through Day 365
|
0.00%
0/3 • Day 1 through Day 365
|
33.3%
1/3 • Number of events 1 • Day 1 through Day 365
|
0.00%
0/3 • Day 1 through Day 365
|
|
Investigations
International normalised ratio increased
|
0.00%
0/3 • Day 1 through Day 365
|
0.00%
0/3 • Day 1 through Day 365
|
33.3%
1/3 • Number of events 1 • Day 1 through Day 365
|
0.00%
0/3 • Day 1 through Day 365
|
|
Investigations
Prostatic specific antigen increased
|
33.3%
1/3 • Number of events 1 • Day 1 through Day 365
|
0.00%
0/3 • Day 1 through Day 365
|
0.00%
0/3 • Day 1 through Day 365
|
0.00%
0/3 • Day 1 through Day 365
|
|
Skin and subcutaneous tissue disorders
Erythema
|
0.00%
0/3 • Day 1 through Day 365
|
33.3%
1/3 • Number of events 1 • Day 1 through Day 365
|
0.00%
0/3 • Day 1 through Day 365
|
0.00%
0/3 • Day 1 through Day 365
|
|
Skin and subcutaneous tissue disorders
Pruritis
|
0.00%
0/3 • Day 1 through Day 365
|
0.00%
0/3 • Day 1 through Day 365
|
0.00%
0/3 • Day 1 through Day 365
|
33.3%
1/3 • Number of events 1 • Day 1 through Day 365
|
|
Skin and subcutaneous tissue disorders
Skin ulcer
|
0.00%
0/3 • Day 1 through Day 365
|
0.00%
0/3 • Day 1 through Day 365
|
0.00%
0/3 • Day 1 through Day 365
|
33.3%
1/3 • Number of events 1 • Day 1 through Day 365
|
|
Injury, poisoning and procedural complications
Tongue injury
|
33.3%
1/3 • Number of events 1 • Day 1 through Day 365
|
0.00%
0/3 • Day 1 through Day 365
|
0.00%
0/3 • Day 1 through Day 365
|
0.00%
0/3 • Day 1 through Day 365
|
|
Psychiatric disorders
Confusional state
|
33.3%
1/3 • Number of events 2 • Day 1 through Day 365
|
0.00%
0/3 • Day 1 through Day 365
|
0.00%
0/3 • Day 1 through Day 365
|
0.00%
0/3 • Day 1 through Day 365
|
|
Infections and infestations
Chronic Sinusitis
|
0.00%
0/3 • Day 1 through Day 365
|
33.3%
1/3 • Number of events 1 • Day 1 through Day 365
|
0.00%
0/3 • Day 1 through Day 365
|
0.00%
0/3 • Day 1 through Day 365
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place