Trial Outcomes & Findings for Phase 3 Gene Therapy for Painful Diabetic Neuropathy (NCT NCT02427464)
NCT ID: NCT02427464
Last Updated: 2025-10-09
Results Overview
Participants rated their 24-hour average daily pain intensity score using an 11-point numerical rating scale from 0 (no pain) to 10 (worst possible pain) in a Daily Pain and Sleep Interference Diary
Recruitment status
COMPLETED
Study phase
PHASE3
Target enrollment
507 participants
Primary outcome timeframe
The Pain and Sleep Interference diary was completed by participants for at least 5 assessments during a 7-day period at Screening (the mean 24-hour score was the reference/baseline score) and within 14 days prior to Day 90 visit.
Results posted on
2025-10-09
Participant Flow
Participants recruited using standard methods were randomized 2:1 to Engensis or Placebo and stratified by gabapentinoid use versus non-gabapentinoid concomitant medications.
Participant milestones
| Measure |
Engensis (VM202)
Participants randomized to the Engensis (VM202) treatment arm received the following intramuscular injections in each calf:
* Day 0 - 16 injections of 0.5mL of VM202 / calf
* Day 14 - 16 injections of 0.5mL of VM202 / calf
* Day 90 - 16 injections of 0.5mL of VM202 / calf
* Day 104 - 16 injections of 0.5mL of VM202 / calf
Engensis (VM202): gene therapy
|
Placebo
Participants randomized to the Placebo treatment arm received the following intramuscular injections in each calf
* Day 0 - 16 injections of 0.5mL of VM202 vehicle / calf
* Day 14 - 16 injections of 0.5mL of VM202 vehicle / calf
* Day 90 - 16 injections of 0.5mL of VM202 vehicle / calf
* Day 104 - 16 injections of 0.5mL of VM202 vehicle / calf
placebo
|
|---|---|---|
|
Overall Study
STARTED
|
338
|
169
|
|
Overall Study
Intent-to-Treat Population
|
336
|
164
|
|
Overall Study
COMPLETED
|
288
|
145
|
|
Overall Study
NOT COMPLETED
|
50
|
24
|
Reasons for withdrawal
| Measure |
Engensis (VM202)
Participants randomized to the Engensis (VM202) treatment arm received the following intramuscular injections in each calf:
* Day 0 - 16 injections of 0.5mL of VM202 / calf
* Day 14 - 16 injections of 0.5mL of VM202 / calf
* Day 90 - 16 injections of 0.5mL of VM202 / calf
* Day 104 - 16 injections of 0.5mL of VM202 / calf
Engensis (VM202): gene therapy
|
Placebo
Participants randomized to the Placebo treatment arm received the following intramuscular injections in each calf
* Day 0 - 16 injections of 0.5mL of VM202 vehicle / calf
* Day 14 - 16 injections of 0.5mL of VM202 vehicle / calf
* Day 90 - 16 injections of 0.5mL of VM202 vehicle / calf
* Day 104 - 16 injections of 0.5mL of VM202 vehicle / calf
placebo
|
|---|---|---|
|
Overall Study
Adverse Event
|
6
|
1
|
|
Overall Study
Lost to Follow-up
|
16
|
5
|
|
Overall Study
Physician Decision
|
2
|
0
|
|
Overall Study
Withdrawal by Subject
|
10
|
8
|
|
Overall Study
Noncompliance
|
16
|
10
|
Baseline Characteristics
Phase 3 Gene Therapy for Painful Diabetic Neuropathy
Baseline characteristics by cohort
| Measure |
Engensis (VM202)
n=336 Participants
Subjects randomized to the Engensis (VM202) treatment arm received the following intramuscular injections in each calf:
* Day 0 - 16 injections of 0.5mL of VM202 / calf
* Day 14 - 16 injections of 0.5mL of VM202 / calf
* Day 90 - 16 injections of 0.5mL of VM202 / calf
* Day 104 - 16 injections of 0.5mL of VM202 / calf
Engensis (VM202): gene therapy
|
Placebo
n=164 Participants
Subjects in the placebo control group received the following intramuscular injections in each calf:
* Day 0 - 16 injections of 0.5mL of VM202 vehicle / calf
* Day 14 - 16 injections of 0.5mL of VM202 vehicle / calf
* Day 90 - 16 injections of 0.5mL of VM202 vehicle / calf
* Day 104 - 16 injections of 0.5mL of VM202 vehicle / calf
placebo
|
Total
n=500 Participants
Total of all reporting groups
|
|---|---|---|---|
|
Age, Continuous
|
60.8 years
STANDARD_DEVIATION 9.09 • n=93 Participants
|
61.6 years
STANDARD_DEVIATION 9.09 • n=4 Participants
|
61.0 years
STANDARD_DEVIATION 9.09 • n=27 Participants
|
|
Sex: Female, Male
Female
|
143 Participants
n=93 Participants
|
45 Participants
n=4 Participants
|
188 Participants
n=27 Participants
|
|
Sex: Female, Male
Male
|
193 Participants
n=93 Participants
|
119 Participants
n=4 Participants
|
312 Participants
n=27 Participants
|
|
Race/Ethnicity, Customized
White
|
247 Participants
n=93 Participants
|
125 Participants
n=4 Participants
|
372 Participants
n=27 Participants
|
|
Race/Ethnicity, Customized
Black or African American
|
71 Participants
n=93 Participants
|
29 Participants
n=4 Participants
|
100 Participants
n=27 Participants
|
|
Race/Ethnicity, Customized
Asian
|
11 Participants
n=93 Participants
|
4 Participants
n=4 Participants
|
15 Participants
n=27 Participants
|
|
Race/Ethnicity, Customized
American Indian or Alaska Native
|
1 Participants
n=93 Participants
|
1 Participants
n=4 Participants
|
2 Participants
n=27 Participants
|
|
Race/Ethnicity, Customized
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=93 Participants
|
0 Participants
n=4 Participants
|
0 Participants
n=27 Participants
|
|
Race/Ethnicity, Customized
Other
|
5 Participants
n=93 Participants
|
4 Participants
n=4 Participants
|
9 Participants
n=27 Participants
|
|
Race/Ethnicity, Customized
Unknown
|
1 Participants
n=93 Participants
|
1 Participants
n=4 Participants
|
2 Participants
n=27 Participants
|
|
Region of Enrollment
United States
|
336 participants
n=93 Participants
|
164 participants
n=4 Participants
|
500 participants
n=27 Participants
|
PRIMARY outcome
Timeframe: The Pain and Sleep Interference diary was completed by participants for at least 5 assessments during a 7-day period at Screening (the mean 24-hour score was the reference/baseline score) and within 14 days prior to Day 90 visit.Population: Overall number of Intent-to-Treat population with available data for this assessment
Participants rated their 24-hour average daily pain intensity score using an 11-point numerical rating scale from 0 (no pain) to 10 (worst possible pain) in a Daily Pain and Sleep Interference Diary
Outcome measures
| Measure |
Engensis (VM202)
n=312 Participants
Participants randomized to the Engensis (VM202) treatment arm received the following intramuscular injections in each calf:
* Day 0 - 16 injections of 0.5mL of VM202 / calf
* Day 14 - 16 injections of 0.5mL of VM202 / calf
* Day 90 - 16 injections of 0.5mL of VM202 / calf
* Day 104 - 16 injections of 0.5mL of VM202 / calf
Engensis (VM202): gene therapy
|
Placebo
n=154 Participants
Participants randomized to the Placebo treatment arm received the following intramuscular injections in each calf
* Day 0 - 16 injections of 0.5mL of VM202 vehicle / calf
* Day 14 - 16 injections of 0.5mL of VM202 vehicle / calf
* Day 90 - 16 injections of 0.5mL of VM202 vehicle / calf
* Day 104 - 16 injections of 0.5mL of VM202 vehicle / calf
placebo
|
|---|---|---|
|
Change in the Average 24 Hour Pain Score From Baseline to Day 90
|
-1.80 units on a scale
Standard Deviation 2.05
|
-1.57 units on a scale
Standard Deviation 2.07
|
PRIMARY outcome
Timeframe: Baseline to Day 90Population: Intent-to-Treat population
Number of participants with at least a 50 percent reduction in average 24-hour pain score, using an 11-point numerical rating scale from 0 (no pain) to 10 (worst possible pain) in the Daily Pain and Sleep Interference Diary
Outcome measures
| Measure |
Engensis (VM202)
n=336 Participants
Participants randomized to the Engensis (VM202) treatment arm received the following intramuscular injections in each calf:
* Day 0 - 16 injections of 0.5mL of VM202 / calf
* Day 14 - 16 injections of 0.5mL of VM202 / calf
* Day 90 - 16 injections of 0.5mL of VM202 / calf
* Day 104 - 16 injections of 0.5mL of VM202 / calf
Engensis (VM202): gene therapy
|
Placebo
n=164 Participants
Participants randomized to the Placebo treatment arm received the following intramuscular injections in each calf
* Day 0 - 16 injections of 0.5mL of VM202 vehicle / calf
* Day 14 - 16 injections of 0.5mL of VM202 vehicle / calf
* Day 90 - 16 injections of 0.5mL of VM202 vehicle / calf
* Day 104 - 16 injections of 0.5mL of VM202 vehicle / calf
placebo
|
|---|---|---|
|
Participants With at Least at 50 Percent Reduction in Average 24-hour Pain Score From Baseline to Day 90
|
69 Participants
|
28 Participants
|
PRIMARY outcome
Timeframe: Baseline to Day 270Population: Safety population
Number of Participants with at least one treatment-emergent adverse events.
Outcome measures
| Measure |
Engensis (VM202)
n=332 Participants
Participants randomized to the Engensis (VM202) treatment arm received the following intramuscular injections in each calf:
* Day 0 - 16 injections of 0.5mL of VM202 / calf
* Day 14 - 16 injections of 0.5mL of VM202 / calf
* Day 90 - 16 injections of 0.5mL of VM202 / calf
* Day 104 - 16 injections of 0.5mL of VM202 / calf
Engensis (VM202): gene therapy
|
Placebo
n=161 Participants
Participants randomized to the Placebo treatment arm received the following intramuscular injections in each calf
* Day 0 - 16 injections of 0.5mL of VM202 vehicle / calf
* Day 14 - 16 injections of 0.5mL of VM202 vehicle / calf
* Day 90 - 16 injections of 0.5mL of VM202 vehicle / calf
* Day 104 - 16 injections of 0.5mL of VM202 vehicle / calf
placebo
|
|---|---|---|
|
Number of Participants With Treatment-emergent Adverse Events.
|
241 Participants
|
111 Participants
|
SECONDARY outcome
Timeframe: Baseline to Day 180Population: Intent-to-Treat population
Participants rated their 24-hour average daily pain intensity score using an 11-point numerical rating scale from 0 (no pain) to 10 (worst possible pain) in the Daily Pain and Sleep Interference Diary
Outcome measures
| Measure |
Engensis (VM202)
n=336 Participants
Participants randomized to the Engensis (VM202) treatment arm received the following intramuscular injections in each calf:
* Day 0 - 16 injections of 0.5mL of VM202 / calf
* Day 14 - 16 injections of 0.5mL of VM202 / calf
* Day 90 - 16 injections of 0.5mL of VM202 / calf
* Day 104 - 16 injections of 0.5mL of VM202 / calf
Engensis (VM202): gene therapy
|
Placebo
n=164 Participants
Participants randomized to the Placebo treatment arm received the following intramuscular injections in each calf
* Day 0 - 16 injections of 0.5mL of VM202 vehicle / calf
* Day 14 - 16 injections of 0.5mL of VM202 vehicle / calf
* Day 90 - 16 injections of 0.5mL of VM202 vehicle / calf
* Day 104 - 16 injections of 0.5mL of VM202 vehicle / calf
placebo
|
|---|---|---|
|
Change in the Average 24-hour Pain Score From Baseline to Day 180
|
-2.59 units on a scale
Standard Deviation 2.38
|
-2.14 units on a scale
Standard Deviation 2.36
|
SECONDARY outcome
Timeframe: Baseline to Day 180Population: Intent-to-Treat population
The number of participants with at least a 50% reduction in average 24-hour pain score using an 11-point numerical rating scale from 0 (no pain) to 10 (worst possible pain) in the Daily Pain and Sleep Interference Diary
Outcome measures
| Measure |
Engensis (VM202)
n=336 Participants
Participants randomized to the Engensis (VM202) treatment arm received the following intramuscular injections in each calf:
* Day 0 - 16 injections of 0.5mL of VM202 / calf
* Day 14 - 16 injections of 0.5mL of VM202 / calf
* Day 90 - 16 injections of 0.5mL of VM202 / calf
* Day 104 - 16 injections of 0.5mL of VM202 / calf
Engensis (VM202): gene therapy
|
Placebo
n=164 Participants
Participants randomized to the Placebo treatment arm received the following intramuscular injections in each calf
* Day 0 - 16 injections of 0.5mL of VM202 vehicle / calf
* Day 14 - 16 injections of 0.5mL of VM202 vehicle / calf
* Day 90 - 16 injections of 0.5mL of VM202 vehicle / calf
* Day 104 - 16 injections of 0.5mL of VM202 vehicle / calf
placebo
|
|---|---|---|
|
Participants With at Least a 50 Percent Reduction in Average 24-hour Pain Score From Baseline to Day 180
|
113 Participants
|
42 Participants
|
Adverse Events
Engensis (VM202)
Serious events: 32 serious events
Other events: 237 other events
Deaths: 0 deaths
Placebo
Serious events: 16 serious events
Other events: 102 other events
Deaths: 1 deaths
Serious adverse events
| Measure |
Engensis (VM202)
n=332 participants at risk
Participants randomized to the Engensis (VM202) treatment arm received the following intramuscular injections in each calf:
* Day 0 - 16 injections of 0.5mL of VM202 / calf
* Day 14 - 16 injections of 0.5mL of VM202 / calf
* Day 90 - 16 injections of 0.5mL of VM202 / calf
* Day 104 - 16 injections of 0.5mL of VM202 / calf
Engensis (VM202): gene therapy
|
Placebo
n=161 participants at risk
Participants randomized to the Placebo treatment arm received the following intramuscular injections in each calf
* Day 0 - 16 injections of 0.5mL of VM202 vehicle / calf
* Day 14 - 16 injections of 0.5mL of VM202 vehicle / calf
* Day 90 - 16 injections of 0.5mL of VM202 vehicle / calf
* Day 104 - 16 injections of 0.5mL of VM202 vehicle / calf
placebo
|
|---|---|---|
|
Infections and infestations
Appendicitis
|
0.00%
0/332 • 270 days
All-Cause Mortality, Serious and Other Adverse Events were monitored for the Safety Population
|
0.62%
1/161 • 270 days
All-Cause Mortality, Serious and Other Adverse Events were monitored for the Safety Population
|
|
Infections and infestations
Cellulitis of male external genital organ
|
0.00%
0/332 • 270 days
All-Cause Mortality, Serious and Other Adverse Events were monitored for the Safety Population
|
0.62%
1/161 • 270 days
All-Cause Mortality, Serious and Other Adverse Events were monitored for the Safety Population
|
|
Infections and infestations
Cholecystitis infective
|
0.00%
0/332 • 270 days
All-Cause Mortality, Serious and Other Adverse Events were monitored for the Safety Population
|
0.62%
1/161 • 270 days
All-Cause Mortality, Serious and Other Adverse Events were monitored for the Safety Population
|
|
Infections and infestations
Diverticulitis
|
0.30%
1/332 • 270 days
All-Cause Mortality, Serious and Other Adverse Events were monitored for the Safety Population
|
0.00%
0/161 • 270 days
All-Cause Mortality, Serious and Other Adverse Events were monitored for the Safety Population
|
|
Infections and infestations
Infected bite
|
0.00%
0/332 • 270 days
All-Cause Mortality, Serious and Other Adverse Events were monitored for the Safety Population
|
0.62%
1/161 • 270 days
All-Cause Mortality, Serious and Other Adverse Events were monitored for the Safety Population
|
|
Cardiac disorders
Acute coronary syndrome
|
0.30%
1/332 • 270 days
All-Cause Mortality, Serious and Other Adverse Events were monitored for the Safety Population
|
0.62%
1/161 • 270 days
All-Cause Mortality, Serious and Other Adverse Events were monitored for the Safety Population
|
|
Cardiac disorders
Acute myocardial infarction
|
0.30%
1/332 • 270 days
All-Cause Mortality, Serious and Other Adverse Events were monitored for the Safety Population
|
0.62%
1/161 • 270 days
All-Cause Mortality, Serious and Other Adverse Events were monitored for the Safety Population
|
|
Cardiac disorders
Angina pectoris
|
0.60%
2/332 • 270 days
All-Cause Mortality, Serious and Other Adverse Events were monitored for the Safety Population
|
0.00%
0/161 • 270 days
All-Cause Mortality, Serious and Other Adverse Events were monitored for the Safety Population
|
|
Cardiac disorders
Coronary artery disease
|
0.60%
2/332 • 270 days
All-Cause Mortality, Serious and Other Adverse Events were monitored for the Safety Population
|
0.00%
0/161 • 270 days
All-Cause Mortality, Serious and Other Adverse Events were monitored for the Safety Population
|
|
Cardiac disorders
Angina unstable
|
0.30%
1/332 • 270 days
All-Cause Mortality, Serious and Other Adverse Events were monitored for the Safety Population
|
0.00%
0/161 • 270 days
All-Cause Mortality, Serious and Other Adverse Events were monitored for the Safety Population
|
|
Cardiac disorders
Atrial fibrillation
|
0.30%
1/332 • 270 days
All-Cause Mortality, Serious and Other Adverse Events were monitored for the Safety Population
|
0.00%
0/161 • 270 days
All-Cause Mortality, Serious and Other Adverse Events were monitored for the Safety Population
|
|
Cardiac disorders
Atrioventricular block complete
|
0.00%
0/332 • 270 days
All-Cause Mortality, Serious and Other Adverse Events were monitored for the Safety Population
|
0.62%
1/161 • 270 days
All-Cause Mortality, Serious and Other Adverse Events were monitored for the Safety Population
|
|
Cardiac disorders
Cardiac failure acute
|
0.30%
1/332 • 270 days
All-Cause Mortality, Serious and Other Adverse Events were monitored for the Safety Population
|
0.00%
0/161 • 270 days
All-Cause Mortality, Serious and Other Adverse Events were monitored for the Safety Population
|
|
Cardiac disorders
Cardiac failure congestive
|
0.00%
0/332 • 270 days
All-Cause Mortality, Serious and Other Adverse Events were monitored for the Safety Population
|
0.62%
1/161 • 270 days
All-Cause Mortality, Serious and Other Adverse Events were monitored for the Safety Population
|
|
Cardiac disorders
Myocardial infarction
|
0.30%
1/332 • 270 days
All-Cause Mortality, Serious and Other Adverse Events were monitored for the Safety Population
|
0.00%
0/161 • 270 days
All-Cause Mortality, Serious and Other Adverse Events were monitored for the Safety Population
|
|
Infections and infestations
Cellulitis
|
0.60%
2/332 • 270 days
All-Cause Mortality, Serious and Other Adverse Events were monitored for the Safety Population
|
0.00%
0/161 • 270 days
All-Cause Mortality, Serious and Other Adverse Events were monitored for the Safety Population
|
|
Infections and infestations
Infected skin ulcer
|
0.00%
0/332 • 270 days
All-Cause Mortality, Serious and Other Adverse Events were monitored for the Safety Population
|
0.62%
1/161 • 270 days
All-Cause Mortality, Serious and Other Adverse Events were monitored for the Safety Population
|
|
Infections and infestations
Influenza
|
0.30%
1/332 • 270 days
All-Cause Mortality, Serious and Other Adverse Events were monitored for the Safety Population
|
0.00%
0/161 • 270 days
All-Cause Mortality, Serious and Other Adverse Events were monitored for the Safety Population
|
|
Infections and infestations
Post procedural cellulitis
|
0.30%
1/332 • 270 days
All-Cause Mortality, Serious and Other Adverse Events were monitored for the Safety Population
|
0.00%
0/161 • 270 days
All-Cause Mortality, Serious and Other Adverse Events were monitored for the Safety Population
|
|
Infections and infestations
Pyelonephritis
|
0.30%
1/332 • 270 days
All-Cause Mortality, Serious and Other Adverse Events were monitored for the Safety Population
|
0.00%
0/161 • 270 days
All-Cause Mortality, Serious and Other Adverse Events were monitored for the Safety Population
|
|
Infections and infestations
Sepsis
|
0.30%
1/332 • 270 days
All-Cause Mortality, Serious and Other Adverse Events were monitored for the Safety Population
|
0.00%
0/161 • 270 days
All-Cause Mortality, Serious and Other Adverse Events were monitored for the Safety Population
|
|
Infections and infestations
Urinary tract infection
|
0.30%
1/332 • 270 days
All-Cause Mortality, Serious and Other Adverse Events were monitored for the Safety Population
|
0.00%
0/161 • 270 days
All-Cause Mortality, Serious and Other Adverse Events were monitored for the Safety Population
|
|
Injury, poisoning and procedural complications
Hand fracture
|
0.00%
0/332 • 270 days
All-Cause Mortality, Serious and Other Adverse Events were monitored for the Safety Population
|
0.62%
1/161 • 270 days
All-Cause Mortality, Serious and Other Adverse Events were monitored for the Safety Population
|
|
Injury, poisoning and procedural complications
Head injury
|
0.30%
1/332 • 270 days
All-Cause Mortality, Serious and Other Adverse Events were monitored for the Safety Population
|
0.00%
0/161 • 270 days
All-Cause Mortality, Serious and Other Adverse Events were monitored for the Safety Population
|
|
Injury, poisoning and procedural complications
Humerus fracture
|
0.30%
1/332 • 270 days
All-Cause Mortality, Serious and Other Adverse Events were monitored for the Safety Population
|
0.00%
0/161 • 270 days
All-Cause Mortality, Serious and Other Adverse Events were monitored for the Safety Population
|
|
Injury, poisoning and procedural complications
Joint dislocation
|
0.00%
0/332 • 270 days
All-Cause Mortality, Serious and Other Adverse Events were monitored for the Safety Population
|
0.62%
1/161 • 270 days
All-Cause Mortality, Serious and Other Adverse Events were monitored for the Safety Population
|
|
Injury, poisoning and procedural complications
Lumbar vertebral fracture
|
0.00%
0/332 • 270 days
All-Cause Mortality, Serious and Other Adverse Events were monitored for the Safety Population
|
0.62%
1/161 • 270 days
All-Cause Mortality, Serious and Other Adverse Events were monitored for the Safety Population
|
|
Injury, poisoning and procedural complications
Post procedural haematoma
|
0.00%
0/332 • 270 days
All-Cause Mortality, Serious and Other Adverse Events were monitored for the Safety Population
|
0.62%
1/161 • 270 days
All-Cause Mortality, Serious and Other Adverse Events were monitored for the Safety Population
|
|
Injury, poisoning and procedural complications
Road traffic accident
|
0.30%
1/332 • 270 days
All-Cause Mortality, Serious and Other Adverse Events were monitored for the Safety Population
|
0.00%
0/161 • 270 days
All-Cause Mortality, Serious and Other Adverse Events were monitored for the Safety Population
|
|
Musculoskeletal and connective tissue disorders
Arthralgia
|
0.30%
1/332 • 270 days
All-Cause Mortality, Serious and Other Adverse Events were monitored for the Safety Population
|
1.2%
2/161 • 270 days
All-Cause Mortality, Serious and Other Adverse Events were monitored for the Safety Population
|
|
Musculoskeletal and connective tissue disorders
Neuropathic arthropathy
|
0.30%
1/332 • 270 days
All-Cause Mortality, Serious and Other Adverse Events were monitored for the Safety Population
|
0.00%
0/161 • 270 days
All-Cause Mortality, Serious and Other Adverse Events were monitored for the Safety Population
|
|
Musculoskeletal and connective tissue disorders
Osteoarthritis
|
0.00%
0/332 • 270 days
All-Cause Mortality, Serious and Other Adverse Events were monitored for the Safety Population
|
0.62%
1/161 • 270 days
All-Cause Mortality, Serious and Other Adverse Events were monitored for the Safety Population
|
|
Musculoskeletal and connective tissue disorders
Osteochondrosis
|
0.30%
1/332 • 270 days
All-Cause Mortality, Serious and Other Adverse Events were monitored for the Safety Population
|
0.00%
0/161 • 270 days
All-Cause Mortality, Serious and Other Adverse Events were monitored for the Safety Population
|
|
Nervous system disorders
Cerebral infarction
|
0.00%
0/332 • 270 days
All-Cause Mortality, Serious and Other Adverse Events were monitored for the Safety Population
|
0.62%
1/161 • 270 days
All-Cause Mortality, Serious and Other Adverse Events were monitored for the Safety Population
|
|
Nervous system disorders
Diabetic hyperosmolar coma
|
0.00%
0/332 • 270 days
All-Cause Mortality, Serious and Other Adverse Events were monitored for the Safety Population
|
0.62%
1/161 • 270 days
All-Cause Mortality, Serious and Other Adverse Events were monitored for the Safety Population
|
|
Nervous system disorders
Encephalopathy
|
0.00%
0/332 • 270 days
All-Cause Mortality, Serious and Other Adverse Events were monitored for the Safety Population
|
0.62%
1/161 • 270 days
All-Cause Mortality, Serious and Other Adverse Events were monitored for the Safety Population
|
|
Nervous system disorders
Hypertensive encephalopathy
|
0.30%
1/332 • 270 days
All-Cause Mortality, Serious and Other Adverse Events were monitored for the Safety Population
|
0.00%
0/161 • 270 days
All-Cause Mortality, Serious and Other Adverse Events were monitored for the Safety Population
|
|
Nervous system disorders
Metabolic encephalopathy
|
0.00%
0/332 • 270 days
All-Cause Mortality, Serious and Other Adverse Events were monitored for the Safety Population
|
0.62%
1/161 • 270 days
All-Cause Mortality, Serious and Other Adverse Events were monitored for the Safety Population
|
|
Nervous system disorders
Mononeuropathy
|
0.30%
1/332 • 270 days
All-Cause Mortality, Serious and Other Adverse Events were monitored for the Safety Population
|
0.00%
0/161 • 270 days
All-Cause Mortality, Serious and Other Adverse Events were monitored for the Safety Population
|
|
Nervous system disorders
Transient ischaemic attack
|
0.30%
1/332 • 270 days
All-Cause Mortality, Serious and Other Adverse Events were monitored for the Safety Population
|
0.00%
0/161 • 270 days
All-Cause Mortality, Serious and Other Adverse Events were monitored for the Safety Population
|
|
Gastrointestinal disorders
Melaena
|
0.30%
1/332 • 270 days
All-Cause Mortality, Serious and Other Adverse Events were monitored for the Safety Population
|
0.00%
0/161 • 270 days
All-Cause Mortality, Serious and Other Adverse Events were monitored for the Safety Population
|
|
Gastrointestinal disorders
Oesophageal varices haemorrhage
|
0.00%
0/332 • 270 days
All-Cause Mortality, Serious and Other Adverse Events were monitored for the Safety Population
|
0.62%
1/161 • 270 days
All-Cause Mortality, Serious and Other Adverse Events were monitored for the Safety Population
|
|
Gastrointestinal disorders
Pancreatitis
|
0.30%
1/332 • 270 days
All-Cause Mortality, Serious and Other Adverse Events were monitored for the Safety Population
|
0.00%
0/161 • 270 days
All-Cause Mortality, Serious and Other Adverse Events were monitored for the Safety Population
|
|
Metabolism and nutrition disorders
Diabetic ketoacidosis
|
0.30%
1/332 • 270 days
All-Cause Mortality, Serious and Other Adverse Events were monitored for the Safety Population
|
0.00%
0/161 • 270 days
All-Cause Mortality, Serious and Other Adverse Events were monitored for the Safety Population
|
|
Metabolism and nutrition disorders
Hyperglycaemia
|
0.30%
1/332 • 270 days
All-Cause Mortality, Serious and Other Adverse Events were monitored for the Safety Population
|
0.00%
0/161 • 270 days
All-Cause Mortality, Serious and Other Adverse Events were monitored for the Safety Population
|
|
Metabolism and nutrition disorders
Hypoglycaemia
|
0.30%
1/332 • 270 days
All-Cause Mortality, Serious and Other Adverse Events were monitored for the Safety Population
|
0.00%
0/161 • 270 days
All-Cause Mortality, Serious and Other Adverse Events were monitored for the Safety Population
|
|
Metabolism and nutrition disorders
Hypokalaemia
|
0.00%
0/332 • 270 days
All-Cause Mortality, Serious and Other Adverse Events were monitored for the Safety Population
|
0.62%
1/161 • 270 days
All-Cause Mortality, Serious and Other Adverse Events were monitored for the Safety Population
|
|
Metabolism and nutrition disorders
Lactic acidosis
|
0.00%
0/332 • 270 days
All-Cause Mortality, Serious and Other Adverse Events were monitored for the Safety Population
|
0.62%
1/161 • 270 days
All-Cause Mortality, Serious and Other Adverse Events were monitored for the Safety Population
|
|
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Adenocarcinoma
|
0.30%
1/332 • 270 days
All-Cause Mortality, Serious and Other Adverse Events were monitored for the Safety Population
|
0.00%
0/161 • 270 days
All-Cause Mortality, Serious and Other Adverse Events were monitored for the Safety Population
|
|
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Invasive ductal breast carcinoma
|
0.30%
1/332 • 270 days
All-Cause Mortality, Serious and Other Adverse Events were monitored for the Safety Population
|
0.00%
0/161 • 270 days
All-Cause Mortality, Serious and Other Adverse Events were monitored for the Safety Population
|
|
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Rectal cancer
|
0.30%
1/332 • 270 days
All-Cause Mortality, Serious and Other Adverse Events were monitored for the Safety Population
|
0.00%
0/161 • 270 days
All-Cause Mortality, Serious and Other Adverse Events were monitored for the Safety Population
|
|
Respiratory, thoracic and mediastinal disorders
Acute respiratory failure
|
0.00%
0/332 • 270 days
All-Cause Mortality, Serious and Other Adverse Events were monitored for the Safety Population
|
0.62%
1/161 • 270 days
All-Cause Mortality, Serious and Other Adverse Events were monitored for the Safety Population
|
|
Respiratory, thoracic and mediastinal disorders
Chronic obstructive pulmonary disease
|
0.30%
1/332 • 270 days
All-Cause Mortality, Serious and Other Adverse Events were monitored for the Safety Population
|
0.00%
0/161 • 270 days
All-Cause Mortality, Serious and Other Adverse Events were monitored for the Safety Population
|
|
Respiratory, thoracic and mediastinal disorders
Respiratory failure
|
0.00%
0/332 • 270 days
All-Cause Mortality, Serious and Other Adverse Events were monitored for the Safety Population
|
0.62%
1/161 • 270 days
All-Cause Mortality, Serious and Other Adverse Events were monitored for the Safety Population
|
|
Vascular disorders
Hypertensive emergency
|
0.30%
1/332 • 270 days
All-Cause Mortality, Serious and Other Adverse Events were monitored for the Safety Population
|
0.00%
0/161 • 270 days
All-Cause Mortality, Serious and Other Adverse Events were monitored for the Safety Population
|
|
Vascular disorders
Orthostatic hypotension
|
0.00%
0/332 • 270 days
All-Cause Mortality, Serious and Other Adverse Events were monitored for the Safety Population
|
0.62%
1/161 • 270 days
All-Cause Mortality, Serious and Other Adverse Events were monitored for the Safety Population
|
|
Vascular disorders
Peripheral artery stenosis
|
0.30%
1/332 • 270 days
All-Cause Mortality, Serious and Other Adverse Events were monitored for the Safety Population
|
0.00%
0/161 • 270 days
All-Cause Mortality, Serious and Other Adverse Events were monitored for the Safety Population
|
|
General disorders
Chest pain
|
0.60%
2/332 • 270 days
All-Cause Mortality, Serious and Other Adverse Events were monitored for the Safety Population
|
0.00%
0/161 • 270 days
All-Cause Mortality, Serious and Other Adverse Events were monitored for the Safety Population
|
|
Investigations
Influenza A virus test positive
|
0.30%
1/332 • 270 days
All-Cause Mortality, Serious and Other Adverse Events were monitored for the Safety Population
|
0.00%
0/161 • 270 days
All-Cause Mortality, Serious and Other Adverse Events were monitored for the Safety Population
|
|
Investigations
Lipase increased
|
0.30%
1/332 • 270 days
All-Cause Mortality, Serious and Other Adverse Events were monitored for the Safety Population
|
0.00%
0/161 • 270 days
All-Cause Mortality, Serious and Other Adverse Events were monitored for the Safety Population
|
|
Renal and urinary disorders
Acute kidney injury
|
0.30%
1/332 • 270 days
All-Cause Mortality, Serious and Other Adverse Events were monitored for the Safety Population
|
0.00%
0/161 • 270 days
All-Cause Mortality, Serious and Other Adverse Events were monitored for the Safety Population
|
|
Renal and urinary disorders
Hydronephrosis
|
0.30%
1/332 • 270 days
All-Cause Mortality, Serious and Other Adverse Events were monitored for the Safety Population
|
0.00%
0/161 • 270 days
All-Cause Mortality, Serious and Other Adverse Events were monitored for the Safety Population
|
|
Renal and urinary disorders
Ureterolithiasis
|
0.30%
1/332 • 270 days
All-Cause Mortality, Serious and Other Adverse Events were monitored for the Safety Population
|
0.00%
0/161 • 270 days
All-Cause Mortality, Serious and Other Adverse Events were monitored for the Safety Population
|
|
Ear and labyrinth disorders
Vertigo
|
0.00%
0/332 • 270 days
All-Cause Mortality, Serious and Other Adverse Events were monitored for the Safety Population
|
0.62%
1/161 • 270 days
All-Cause Mortality, Serious and Other Adverse Events were monitored for the Safety Population
|
|
Eye disorders
Vitreous haemorrhage
|
0.30%
1/332 • 270 days
All-Cause Mortality, Serious and Other Adverse Events were monitored for the Safety Population
|
0.00%
0/161 • 270 days
All-Cause Mortality, Serious and Other Adverse Events were monitored for the Safety Population
|
|
Hepatobiliary disorders
Cholecystitis
|
0.00%
0/332 • 270 days
All-Cause Mortality, Serious and Other Adverse Events were monitored for the Safety Population
|
0.62%
1/161 • 270 days
All-Cause Mortality, Serious and Other Adverse Events were monitored for the Safety Population
|
|
Psychiatric disorders
Schizoaffective disorder
|
0.00%
0/332 • 270 days
All-Cause Mortality, Serious and Other Adverse Events were monitored for the Safety Population
|
0.62%
1/161 • 270 days
All-Cause Mortality, Serious and Other Adverse Events were monitored for the Safety Population
|
Other adverse events
| Measure |
Engensis (VM202)
n=332 participants at risk
Participants randomized to the Engensis (VM202) treatment arm received the following intramuscular injections in each calf:
* Day 0 - 16 injections of 0.5mL of VM202 / calf
* Day 14 - 16 injections of 0.5mL of VM202 / calf
* Day 90 - 16 injections of 0.5mL of VM202 / calf
* Day 104 - 16 injections of 0.5mL of VM202 / calf
Engensis (VM202): gene therapy
|
Placebo
n=161 participants at risk
Participants randomized to the Placebo treatment arm received the following intramuscular injections in each calf
* Day 0 - 16 injections of 0.5mL of VM202 vehicle / calf
* Day 14 - 16 injections of 0.5mL of VM202 vehicle / calf
* Day 90 - 16 injections of 0.5mL of VM202 vehicle / calf
* Day 104 - 16 injections of 0.5mL of VM202 vehicle / calf
placebo
|
|---|---|---|
|
Skin and subcutaneous tissue disorders
Skin ulcer
|
2.1%
7/332 • 270 days
All-Cause Mortality, Serious and Other Adverse Events were monitored for the Safety Population
|
2.5%
4/161 • 270 days
All-Cause Mortality, Serious and Other Adverse Events were monitored for the Safety Population
|
|
General disorders
Oedema peripheral
|
2.7%
9/332 • 270 days
All-Cause Mortality, Serious and Other Adverse Events were monitored for the Safety Population
|
1.2%
2/161 • 270 days
All-Cause Mortality, Serious and Other Adverse Events were monitored for the Safety Population
|
|
Injury, poisoning and procedural complications
Injection Site Reaction (Grade 1 or 2)
|
13.9%
46/332 • 270 days
All-Cause Mortality, Serious and Other Adverse Events were monitored for the Safety Population
|
11.2%
18/161 • 270 days
All-Cause Mortality, Serious and Other Adverse Events were monitored for the Safety Population
|
|
Infections and infestations
Upper respiratory tract infection
|
6.6%
22/332 • 270 days
All-Cause Mortality, Serious and Other Adverse Events were monitored for the Safety Population
|
7.5%
12/161 • 270 days
All-Cause Mortality, Serious and Other Adverse Events were monitored for the Safety Population
|
|
Infections and infestations
Nasopharyngitis
|
5.1%
17/332 • 270 days
All-Cause Mortality, Serious and Other Adverse Events were monitored for the Safety Population
|
2.5%
4/161 • 270 days
All-Cause Mortality, Serious and Other Adverse Events were monitored for the Safety Population
|
|
Infections and infestations
Bronchitis
|
2.7%
9/332 • 270 days
All-Cause Mortality, Serious and Other Adverse Events were monitored for the Safety Population
|
1.2%
2/161 • 270 days
All-Cause Mortality, Serious and Other Adverse Events were monitored for the Safety Population
|
|
Musculoskeletal and connective tissue disorders
Pain in extremity
|
5.4%
18/332 • 270 days
All-Cause Mortality, Serious and Other Adverse Events were monitored for the Safety Population
|
3.1%
5/161 • 270 days
All-Cause Mortality, Serious and Other Adverse Events were monitored for the Safety Population
|
|
Musculoskeletal and connective tissue disorders
Arthralgia
|
4.2%
14/332 • 270 days
All-Cause Mortality, Serious and Other Adverse Events were monitored for the Safety Population
|
4.3%
7/161 • 270 days
All-Cause Mortality, Serious and Other Adverse Events were monitored for the Safety Population
|
|
Musculoskeletal and connective tissue disorders
Muscle spasms
|
3.0%
10/332 • 270 days
All-Cause Mortality, Serious and Other Adverse Events were monitored for the Safety Population
|
6.2%
10/161 • 270 days
All-Cause Mortality, Serious and Other Adverse Events were monitored for the Safety Population
|
|
Musculoskeletal and connective tissue disorders
Back pain
|
3.6%
12/332 • 270 days
All-Cause Mortality, Serious and Other Adverse Events were monitored for the Safety Population
|
3.1%
5/161 • 270 days
All-Cause Mortality, Serious and Other Adverse Events were monitored for the Safety Population
|
|
Injury, poisoning and procedural complications
Fall
|
2.1%
7/332 • 270 days
All-Cause Mortality, Serious and Other Adverse Events were monitored for the Safety Population
|
5.0%
8/161 • 270 days
All-Cause Mortality, Serious and Other Adverse Events were monitored for the Safety Population
|
|
Eye disorders
Diabetic retinopathy
|
2.1%
7/332 • 270 days
All-Cause Mortality, Serious and Other Adverse Events were monitored for the Safety Population
|
6.2%
10/161 • 270 days
All-Cause Mortality, Serious and Other Adverse Events were monitored for the Safety Population
|
|
Eye disorders
Cataract
|
2.7%
9/332 • 270 days
All-Cause Mortality, Serious and Other Adverse Events were monitored for the Safety Population
|
1.9%
3/161 • 270 days
All-Cause Mortality, Serious and Other Adverse Events were monitored for the Safety Population
|
|
Nervous system disorders
Headache
|
4.2%
14/332 • 270 days
All-Cause Mortality, Serious and Other Adverse Events were monitored for the Safety Population
|
1.9%
3/161 • 270 days
All-Cause Mortality, Serious and Other Adverse Events were monitored for the Safety Population
|
|
Gastrointestinal disorders
Diarrhoea
|
2.7%
9/332 • 270 days
All-Cause Mortality, Serious and Other Adverse Events were monitored for the Safety Population
|
1.9%
3/161 • 270 days
All-Cause Mortality, Serious and Other Adverse Events were monitored for the Safety Population
|
|
Gastrointestinal disorders
Nausea
|
2.7%
9/332 • 270 days
All-Cause Mortality, Serious and Other Adverse Events were monitored for the Safety Population
|
1.2%
2/161 • 270 days
All-Cause Mortality, Serious and Other Adverse Events were monitored for the Safety Population
|
|
Metabolism and nutrition disorders
Hyperglycaemia
|
2.7%
9/332 • 270 days
All-Cause Mortality, Serious and Other Adverse Events were monitored for the Safety Population
|
1.2%
2/161 • 270 days
All-Cause Mortality, Serious and Other Adverse Events were monitored for the Safety Population
|
|
Metabolism and nutrition disorders
Hypoglycaemia
|
2.7%
9/332 • 270 days
All-Cause Mortality, Serious and Other Adverse Events were monitored for the Safety Population
|
1.2%
2/161 • 270 days
All-Cause Mortality, Serious and Other Adverse Events were monitored for the Safety Population
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee Investigator Agreement .....
- Publication restrictions are in place
Restriction type: OTHER