Trial Outcomes & Findings for Safety and Efficacy Study for the Treatment of Painful Diabetic Neuropathy (NCT NCT01475786)
NCT ID: NCT01475786
Last Updated: 2025-10-06
Results Overview
The difference in the mean change of the 24 hour pain score was compared between the treatment groups and the placebo arm to determine treatment effect. The average pain scores were obtained from the Daily Pain and Sleep Interference Diary (recorded daily by subjects for 7 days during Screening prior to the first round of injections and again, before the 6 month follow-up). Subjects rated their 24-hor daily pain intensity according to the 11-point numerical rating scale from 0 (no pain) to 10 (worst possible pain).
Recruitment status
COMPLETED
Study phase
PHASE2
Target enrollment
104 participants
Primary outcome timeframe
seven (7) days before 9 Month visit
Results posted on
2025-10-06
Participant Flow
Participant milestones
| Measure |
High Dose 32mg Engensis (VM202)
Subjects in the High Dose Group (16 mg VM202 / leg) received the following intramuscular injections in each calf for a total of 32 mg Engensis (VM202):
Day 0 - 32 injections of 0.5 mL of VM202 / calf (8 mg of VM202 / calf)
Day 14 - 32 injections of 0.5 mL of VM202 / calf (8 mg of VM202 / calf) For a total of 32mg VM202
|
Low Dose 16mg Engensis (VM202) and Placebo
Subjects in the Low Dose Group (8 mg/leg) received the following intramuscular injections in each calf for a total of 16mg Engensis (VM202):
: Day 0 - 32 injections / calf 16 injections of 0.5mL of VM202 / calf - (4 mg of VM202 / calf) and 16 injections of Placebo 0.5mL / calf
Day 14 - 32 injections / calf 16 injections of 0.5mL of VM202 / calf - (4 mg of VM202 / calf) and 16 injections of Placebo 0.5mL / calf
|
Control - Placebo (Normal Saline)
Subjects in the Placebo Group received 32 injections / calf of 0.5 mL Placebo on Day 0 and Day 14
|
|---|---|---|---|
|
Overall Study
STARTED
|
43
|
40
|
21
|
|
Overall Study
COMPLETED
|
41
|
36
|
19
|
|
Overall Study
NOT COMPLETED
|
2
|
4
|
2
|
Reasons for withdrawal
| Measure |
High Dose 32mg Engensis (VM202)
Subjects in the High Dose Group (16 mg VM202 / leg) received the following intramuscular injections in each calf for a total of 32 mg Engensis (VM202):
Day 0 - 32 injections of 0.5 mL of VM202 / calf (8 mg of VM202 / calf)
Day 14 - 32 injections of 0.5 mL of VM202 / calf (8 mg of VM202 / calf) For a total of 32mg VM202
|
Low Dose 16mg Engensis (VM202) and Placebo
Subjects in the Low Dose Group (8 mg/leg) received the following intramuscular injections in each calf for a total of 16mg Engensis (VM202):
: Day 0 - 32 injections / calf 16 injections of 0.5mL of VM202 / calf - (4 mg of VM202 / calf) and 16 injections of Placebo 0.5mL / calf
Day 14 - 32 injections / calf 16 injections of 0.5mL of VM202 / calf - (4 mg of VM202 / calf) and 16 injections of Placebo 0.5mL / calf
|
Control - Placebo (Normal Saline)
Subjects in the Placebo Group received 32 injections / calf of 0.5 mL Placebo on Day 0 and Day 14
|
|---|---|---|---|
|
Overall Study
Adverse Event
|
0
|
1
|
0
|
|
Overall Study
Lost to Follow-up
|
1
|
2
|
1
|
|
Overall Study
Withdrawal by Subject
|
0
|
1
|
1
|
|
Overall Study
Subject discontinued prior to study treatment
|
1
|
0
|
0
|
Baseline Characteristics
Safety and Efficacy Study for the Treatment of Painful Diabetic Neuropathy
Baseline characteristics by cohort
| Measure |
High Dose 32mg Engensis (VM202)
n=42 Participants
Subjects in the High Dose Group (16 mg VM202 / leg) received the following intramuscular injections in each calf for a total of 32 mg Engensis (VM202):
Day 0 - 32 injections of 0.5 mL of VM202 / calf (8 mg of VM202 / calf)
Day 14 - 32 injections of 0.5 mL of VM202 / calf (8 mg of VM202 / calf) For a total of 32mg VM202
|
Low Dose 16mg Engensis (VM202) and Placebo
n=40 Participants
Subjects in the Low Dose Group (8 mg/leg) received the following intramuscular injections in each calf for a total of 16mg Engensis (VM202):
: Day 0 - 32 injections / calf 16 injections of 0.5mL of VM202 / calf - (4 mg of VM202 / calf) and 16 injections of Placebo 0.5mL / calf
Day 14 - 32 injections / calf 16 injections of 0.5mL of VM202 / calf - (4 mg of VM202 / calf) and 16 injections of Placebo 0.5mL / calf
|
Control - Placebo (Normal Saline)
n=21 Participants
Subjects in the Placebo Group received 32 injections / calf of 0.5 mL Placebo on Day 0 and Day 14
|
Total
n=103 Participants
Total of all reporting groups
|
|---|---|---|---|---|
|
Age, Continuous
|
60.6 years
STANDARD_DEVIATION 10.2 • n=5 Participants
|
60.2 years
STANDARD_DEVIATION 7.2 • n=7 Participants
|
60.5 years
STANDARD_DEVIATION 8.3 • n=5 Participants
|
60.4 years
STANDARD_DEVIATION 8.7 • n=4 Participants
|
|
Sex: Female, Male
Female
|
9 Participants
n=5 Participants
|
11 Participants
n=7 Participants
|
6 Participants
n=5 Participants
|
26 Participants
n=4 Participants
|
|
Sex: Female, Male
Male
|
33 Participants
n=5 Participants
|
29 Participants
n=7 Participants
|
15 Participants
n=5 Participants
|
77 Participants
n=4 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
1 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
1 Participants
n=4 Participants
|
|
Race (NIH/OMB)
Asian
|
6 Participants
n=5 Participants
|
5 Participants
n=7 Participants
|
6 Participants
n=5 Participants
|
17 Participants
n=4 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
|
|
Race (NIH/OMB)
Black or African American
|
3 Participants
n=5 Participants
|
7 Participants
n=7 Participants
|
2 Participants
n=5 Participants
|
12 Participants
n=4 Participants
|
|
Race (NIH/OMB)
White
|
32 Participants
n=5 Participants
|
27 Participants
n=7 Participants
|
12 Participants
n=5 Participants
|
71 Participants
n=4 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=5 Participants
|
1 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
1 Participants
n=4 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
1 Participants
n=5 Participants
|
1 Participants
n=4 Participants
|
|
Region of Enrollment
United States
|
42 participants
n=5 Participants
|
40 participants
n=7 Participants
|
21 participants
n=5 Participants
|
103 participants
n=4 Participants
|
PRIMARY outcome
Timeframe: seven (7) days before 9 Month visitPopulation: The Efficacy population included all subjects who received the correct dose of study drug based on the randomization schedule at both Days 0 and 14, have the Day 180 assessment, and did not have any protocol violations or major deviations
The difference in the mean change of the 24 hour pain score was compared between the treatment groups and the placebo arm to determine treatment effect. The average pain scores were obtained from the Daily Pain and Sleep Interference Diary (recorded daily by subjects for 7 days during Screening prior to the first round of injections and again, before the 6 month follow-up). Subjects rated their 24-hor daily pain intensity according to the 11-point numerical rating scale from 0 (no pain) to 10 (worst possible pain).
Outcome measures
| Measure |
High Dose 32mg Engensis (VM202)
n=36 Participants
Subjects in the High Dose Group (16 mg VM202 / leg) received the following intramuscular injections in each calf for a total of 32 mg Engensis (VM202):
Day 0: 32 injections of 0.5 mL of VM202 / calf (8 mg of VM202 / calf)
Day 14: 32 injections of 0.5 mL of VM202 / calf (8 mg of VM202 / calf) For a total of 32mg VM202
|
Low Dose 16mg Engensis (VM202) and Placebo
n=31 Participants
Subjects in the Low Dose Group (8 mg/leg) received the following intramuscular injections in each calf for a total of 16mg Engensis (VM202):
: Day 0: 32 injections / calf 16 injections of 0.5mL of VM202 / calf - (4 mg of VM202 / calf) and 16 injections of Placebo 0.5mL / calf
Day 14: 32 injections / calf 16 injections of 0.5mL of VM202 / calf - (4 mg of VM202 / calf) and 16 injections of Placebo 0.5mL / calf
|
Control - Placebo (Normal Saline)
n=17 Participants
Subjects in the Placebo Group received 32 injections / calf of 0.5 mL Placebo on Day 0 and Day 14
|
|---|---|---|---|
|
The Primary Study Endpoint Was the Change in Average 24-hour Pain Score From Baseline to the 6-month Follow-up.
|
-1.94 units on a scale
Standard Deviation 1.96
|
-2.78 units on a scale
Standard Deviation 2.23
|
-1.59 units on a scale
Standard Deviation 1.89
|
Adverse Events
High Dose 32mg Engensis (VM202)
Serious events: 4 serious events
Other events: 33 other events
Deaths: 0 deaths
Low Dose 16mg Engensis (VM202) and Placebo
Serious events: 3 serious events
Other events: 22 other events
Deaths: 0 deaths
Control - Placebo (Normal Saline)
Serious events: 3 serious events
Other events: 13 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
High Dose 32mg Engensis (VM202)
n=43 participants at risk
Subjects in the High Dose Group (16 mg VM202 / leg) received the following intramuscular injections in each calf for a total of 32 mg Engensis (VM202):
Day 0: 32 injections of 0.5 mL of VM202 / calf (8 mg of VM202 / calf)
Day 14: 32 injections of 0.5 mL of VM202 / calf (8 mg of VM202 / calf) For a total of 32mg VM202
|
Low Dose 16mg Engensis (VM202) and Placebo
n=39 participants at risk
Subjects in the Low Dose Group (8 mg/leg) received the following intramuscular injections in each calf for a total of 16mg Engensis (VM202):
: Day 0: 32 injections / calf 16 injections of 0.5mL of VM202 / calf - (4 mg of VM202 / calf) and 16 injections of Placebo 0.5mL / calf
Day 14: 32 injections / calf 16 injections of 0.5mL of VM202 / calf - (4 mg of VM202 / calf) and 16 injections of Placebo 0.5mL / calf
|
Control - Placebo (Normal Saline)
n=21 participants at risk
Subjects in the Placebo Group received 32 injections / calf of 0.5 mL Placebo on Day 0 and Day 14
|
|---|---|---|---|
|
Cardiac disorders
Left ventricular dysfunction
|
2.3%
1/43 • Baseline (Day 0) to Day 270
|
0.00%
0/39 • Baseline (Day 0) to Day 270
|
0.00%
0/21 • Baseline (Day 0) to Day 270
|
|
Gastrointestinal disorders
Omental infarction
|
0.00%
0/43 • Baseline (Day 0) to Day 270
|
2.6%
1/39 • Baseline (Day 0) to Day 270
|
0.00%
0/21 • Baseline (Day 0) to Day 270
|
|
General disorders
Chest pain
|
2.3%
1/43 • Baseline (Day 0) to Day 270
|
0.00%
0/39 • Baseline (Day 0) to Day 270
|
0.00%
0/21 • Baseline (Day 0) to Day 270
|
|
Hepatobiliary disorders
Cholecystitis
|
0.00%
0/43 • Baseline (Day 0) to Day 270
|
2.6%
1/39 • Baseline (Day 0) to Day 270
|
0.00%
0/21 • Baseline (Day 0) to Day 270
|
|
Immune system disorders
Asthma
|
2.3%
1/43 • Baseline (Day 0) to Day 270
|
0.00%
0/39 • Baseline (Day 0) to Day 270
|
0.00%
0/21 • Baseline (Day 0) to Day 270
|
|
Infections and infestations
Gastroenteritis
|
0.00%
0/43 • Baseline (Day 0) to Day 270
|
0.00%
0/39 • Baseline (Day 0) to Day 270
|
4.8%
1/21 • Baseline (Day 0) to Day 270
|
|
Infections and infestations
Localised infection
|
0.00%
0/43 • Baseline (Day 0) to Day 270
|
0.00%
0/39 • Baseline (Day 0) to Day 270
|
4.8%
1/21 • Baseline (Day 0) to Day 270
|
|
Infections and infestations
Pneumonia
|
0.00%
0/43 • Baseline (Day 0) to Day 270
|
0.00%
0/39 • Baseline (Day 0) to Day 270
|
4.8%
1/21 • Baseline (Day 0) to Day 270
|
|
Injury, poisoning and procedural complications
Rib fracture
|
0.00%
0/43 • Baseline (Day 0) to Day 270
|
0.00%
0/39 • Baseline (Day 0) to Day 270
|
4.8%
1/21 • Baseline (Day 0) to Day 270
|
|
Metabolism and nutrition disorders
Hyperglycaemia
|
0.00%
0/43 • Baseline (Day 0) to Day 270
|
2.6%
1/39 • Baseline (Day 0) to Day 270
|
0.00%
0/21 • Baseline (Day 0) to Day 270
|
|
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Malignant melanoma
|
2.3%
1/43 • Baseline (Day 0) to Day 270
|
0.00%
0/39 • Baseline (Day 0) to Day 270
|
0.00%
0/21 • Baseline (Day 0) to Day 270
|
|
Nervous system disorders
Spontaneous cerebrospinal fluid leak
|
2.3%
1/43 • Baseline (Day 0) to Day 270
|
0.00%
0/39 • Baseline (Day 0) to Day 270
|
0.00%
0/21 • Baseline (Day 0) to Day 270
|
|
Psychiatric disorders
Drug abuse
|
0.00%
0/43 • Baseline (Day 0) to Day 270
|
0.00%
0/39 • Baseline (Day 0) to Day 270
|
4.8%
1/21 • Baseline (Day 0) to Day 270
|
Other adverse events
| Measure |
High Dose 32mg Engensis (VM202)
n=43 participants at risk
Subjects in the High Dose Group (16 mg VM202 / leg) received the following intramuscular injections in each calf for a total of 32 mg Engensis (VM202):
Day 0: 32 injections of 0.5 mL of VM202 / calf (8 mg of VM202 / calf)
Day 14: 32 injections of 0.5 mL of VM202 / calf (8 mg of VM202 / calf) For a total of 32mg VM202
|
Low Dose 16mg Engensis (VM202) and Placebo
n=39 participants at risk
Subjects in the Low Dose Group (8 mg/leg) received the following intramuscular injections in each calf for a total of 16mg Engensis (VM202):
: Day 0: 32 injections / calf 16 injections of 0.5mL of VM202 / calf - (4 mg of VM202 / calf) and 16 injections of Placebo 0.5mL / calf
Day 14: 32 injections / calf 16 injections of 0.5mL of VM202 / calf - (4 mg of VM202 / calf) and 16 injections of Placebo 0.5mL / calf
|
Control - Placebo (Normal Saline)
n=21 participants at risk
Subjects in the Placebo Group received 32 injections / calf of 0.5 mL Placebo on Day 0 and Day 14
|
|---|---|---|---|
|
Infections and infestations
Localised infection
|
9.3%
4/43 • Baseline (Day 0) to Day 270
|
7.7%
3/39 • Baseline (Day 0) to Day 270
|
4.8%
1/21 • Baseline (Day 0) to Day 270
|
|
Infections and infestations
Sinusitis
|
0.00%
0/43 • Baseline (Day 0) to Day 270
|
12.8%
5/39 • Baseline (Day 0) to Day 270
|
0.00%
0/21 • Baseline (Day 0) to Day 270
|
|
Infections and infestations
Upper respiratory tract infection
|
2.3%
1/43 • Baseline (Day 0) to Day 270
|
2.6%
1/39 • Baseline (Day 0) to Day 270
|
9.5%
2/21 • Baseline (Day 0) to Day 270
|
|
Infections and infestations
Bronchitis
|
0.00%
0/43 • Baseline (Day 0) to Day 270
|
2.6%
1/39 • Baseline (Day 0) to Day 270
|
9.5%
2/21 • Baseline (Day 0) to Day 270
|
|
Infections and infestations
Nasopharyngitis
|
7.0%
3/43 • Baseline (Day 0) to Day 270
|
0.00%
0/39 • Baseline (Day 0) to Day 270
|
0.00%
0/21 • Baseline (Day 0) to Day 270
|
|
Infections and infestations
Pneumonia
|
2.3%
1/43 • Baseline (Day 0) to Day 270
|
0.00%
0/39 • Baseline (Day 0) to Day 270
|
9.5%
2/21 • Baseline (Day 0) to Day 270
|
|
Musculoskeletal and connective tissue disorders
Pain in extremity
|
9.3%
4/43 • Baseline (Day 0) to Day 270
|
2.6%
1/39 • Baseline (Day 0) to Day 270
|
4.8%
1/21 • Baseline (Day 0) to Day 270
|
|
Musculoskeletal and connective tissue disorders
Ligament sprain
|
0.00%
0/43 • Baseline (Day 0) to Day 270
|
5.1%
2/39 • Baseline (Day 0) to Day 270
|
9.5%
2/21 • Baseline (Day 0) to Day 270
|
|
Musculoskeletal and connective tissue disorders
Muscle spasms
|
4.7%
2/43 • Baseline (Day 0) to Day 270
|
5.1%
2/39 • Baseline (Day 0) to Day 270
|
4.8%
1/21 • Baseline (Day 0) to Day 270
|
|
Musculoskeletal and connective tissue disorders
Arthralgia
|
4.7%
2/43 • Baseline (Day 0) to Day 270
|
0.00%
0/39 • Baseline (Day 0) to Day 270
|
4.8%
1/21 • Baseline (Day 0) to Day 270
|
|
Gastrointestinal disorders
Diarrhoea
|
2.3%
1/43 • Baseline (Day 0) to Day 270
|
0.00%
0/39 • Baseline (Day 0) to Day 270
|
14.3%
3/21 • Baseline (Day 0) to Day 270
|
|
Gastrointestinal disorders
Nausea
|
7.0%
3/43 • Baseline (Day 0) to Day 270
|
2.6%
1/39 • Baseline (Day 0) to Day 270
|
0.00%
0/21 • Baseline (Day 0) to Day 270
|
|
Injury, poisoning and procedural complications
Contusion
|
7.0%
3/43 • Baseline (Day 0) to Day 270
|
0.00%
0/39 • Baseline (Day 0) to Day 270
|
0.00%
0/21 • Baseline (Day 0) to Day 270
|
|
Injury, poisoning and procedural complications
Limb injury
|
2.3%
1/43 • Baseline (Day 0) to Day 270
|
5.1%
2/39 • Baseline (Day 0) to Day 270
|
0.00%
0/21 • Baseline (Day 0) to Day 270
|
|
Eye disorders
Cataract
|
4.7%
2/43 • Baseline (Day 0) to Day 270
|
2.6%
1/39 • Baseline (Day 0) to Day 270
|
0.00%
0/21 • Baseline (Day 0) to Day 270
|
|
General disorders
Chest pain
|
4.7%
2/43 • Baseline (Day 0) to Day 270
|
2.6%
1/39 • Baseline (Day 0) to Day 270
|
0.00%
0/21 • Baseline (Day 0) to Day 270
|
|
General disorders
Oedema peripheral
|
4.7%
2/43 • Baseline (Day 0) to Day 270
|
2.6%
1/39 • Baseline (Day 0) to Day 270
|
0.00%
0/21 • Baseline (Day 0) to Day 270
|
|
Respiratory, thoracic and mediastinal disorders
Cough
|
2.3%
1/43 • Baseline (Day 0) to Day 270
|
2.6%
1/39 • Baseline (Day 0) to Day 270
|
4.8%
1/21 • Baseline (Day 0) to Day 270
|
|
Vascular disorders
Hypertension
|
4.7%
2/43 • Baseline (Day 0) to Day 270
|
0.00%
0/39 • Baseline (Day 0) to Day 270
|
4.8%
1/21 • Baseline (Day 0) to Day 270
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place