Trial Outcomes & Findings for Safety Study of VM202 to Treat Amyotrophic Lateral Sclerosis (NCT NCT02039401)
NCT ID: NCT02039401
Last Updated: 2025-10-06
Results Overview
Adverse events (including serious adverse events, and adverse events leading to treatment discontinuation) throughout the 9 months follow-up. Descriptive statistics will be used to characterize safety parameters.
Recruitment status
COMPLETED
Study phase
PHASE1/PHASE2
Target enrollment
18 participants
Primary outcome timeframe
Throughout the nine month follow up
Results posted on
2025-10-06
Participant Flow
Participant milestones
| Measure |
Engensis (VM202) Group 1
Total dose of 64 mg of VM202, administered over the course of four visits: Day 0, Day 7, Day 14, and Day 21. As in all previous VM202 studies, final dose of VM202 for each target muscle group is divided and administered 2 weeks apart. Group 1 received Engensis IM injections beginning with the lower limbs, with injection location (lower vs upper limbs) alternating weekly
VM202
|
Engensis (VM202) Group 2
Total dose of 64 mg of VM202, administered over the course of four visits: Day 0, Day 7, Day 14, and Day 21. As in all previous VM202 studies, final dose of VM202 for each target muscle group is divided and administered 2 weeks apart. Group 2 received Engensis IM injections beginning with the upper limbs, with injection location (lower vs upper limbs) alternating weekly
VM202
|
|---|---|---|
|
Overall Study
STARTED
|
9
|
9
|
|
Overall Study
COMPLETED
|
9
|
8
|
|
Overall Study
NOT COMPLETED
|
0
|
1
|
Reasons for withdrawal
| Measure |
Engensis (VM202) Group 1
Total dose of 64 mg of VM202, administered over the course of four visits: Day 0, Day 7, Day 14, and Day 21. As in all previous VM202 studies, final dose of VM202 for each target muscle group is divided and administered 2 weeks apart. Group 1 received Engensis IM injections beginning with the lower limbs, with injection location (lower vs upper limbs) alternating weekly
VM202
|
Engensis (VM202) Group 2
Total dose of 64 mg of VM202, administered over the course of four visits: Day 0, Day 7, Day 14, and Day 21. As in all previous VM202 studies, final dose of VM202 for each target muscle group is divided and administered 2 weeks apart. Group 2 received Engensis IM injections beginning with the upper limbs, with injection location (lower vs upper limbs) alternating weekly
VM202
|
|---|---|---|
|
Overall Study
Adverse Event
|
0
|
1
|
Baseline Characteristics
Safety Study of VM202 to Treat Amyotrophic Lateral Sclerosis
Baseline characteristics by cohort
| Measure |
Engensis (VM202) Group 1
n=9 Participants
Total dose of 64 mg of VM202, administered over the course of four visits: Day 0, Day 7, Day 14, and Day 21. As in all previous VM202 studies, final dose of VM202 for each target muscle group is divided and administered 2 weeks apart. Group 1 received Engensis IM injections beginning with the lower limbs, with injection location (lower vs upper limbs) alternating weekly
VM202
|
Engensis (VM202) Group 2
n=9 Participants
Total dose of 64 mg of VM202, administered over the course of four visits: Day 0, Day 7, Day 14, and Day 21. As in all previous VM202 studies, final dose of VM202 for each target muscle group is divided and administered 2 weeks apart. Group 2 received Engensis IM injections beginning with the upper limbs, with injection location (lower vs upper limbs) alternating weekly
VM202
|
Total
n=18 Participants
Total of all reporting groups
|
|---|---|---|---|
|
Age, Continuous
|
49.1 years
STANDARD_DEVIATION 11.9 • n=5 Participants
|
55.8 years
STANDARD_DEVIATION 3.0 • n=7 Participants
|
52.4 years
STANDARD_DEVIATION 9.1 • n=5 Participants
|
|
Sex: Female, Male
Female
|
1 Participants
n=5 Participants
|
2 Participants
n=7 Participants
|
3 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
8 Participants
n=5 Participants
|
7 Participants
n=7 Participants
|
15 Participants
n=5 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Asian
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 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
|
|
Race (NIH/OMB)
Black or African American
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
White
|
9 Participants
n=5 Participants
|
9 Participants
n=7 Participants
|
18 Participants
n=5 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Region of Enrollment
United States
|
9 participants
n=5 Participants
|
9 participants
n=7 Participants
|
18 participants
n=5 Participants
|
PRIMARY outcome
Timeframe: Throughout the nine month follow upPopulation: Safety analysis set
Adverse events (including serious adverse events, and adverse events leading to treatment discontinuation) throughout the 9 months follow-up. Descriptive statistics will be used to characterize safety parameters.
Outcome measures
| Measure |
Total
n=18 Participants
All subjects in Groups 1 and 2
|
Engensis (VM202) Group 2
Total dose of 64 mg of VM202, administered over the course of four visits: Day 0, Day 7, Day 14, and Day 21. As in all previous VM202 studies, final dose of VM202 for each target muscle group is divided and administered 2 weeks apart. Group 2 received Engensis IM injections beginning with the upper limbs, with injection location (lower vs upper limbs) alternating weekly
VM202
|
Overall
Total subjects from Groups 1 and 2
|
|---|---|---|---|
|
Number of Subjects With Serious and Non Serious Adverse Events
Urinary tract infection
|
1 Participants
|
—
|
—
|
|
Number of Subjects With Serious and Non Serious Adverse Events
Fall
|
11 Participants
|
—
|
—
|
|
Number of Subjects With Serious and Non Serious Adverse Events
Muscle strain
|
1 Participants
|
—
|
—
|
|
Number of Subjects With Serious and Non Serious Adverse Events
Aspiration
|
1 Participants
|
—
|
—
|
|
Number of Subjects With Serious and Non Serious Adverse Events
Diarrhoea
|
1 Participants
|
—
|
—
|
|
Number of Subjects With Serious and Non Serious Adverse Events
Gastroesophargeal reflux disease
|
1 Participants
|
—
|
—
|
|
Number of Subjects With Serious and Non Serious Adverse Events
Small intestinal obstruction
|
1 Participants
|
—
|
—
|
|
Number of Subjects With Serious and Non Serious Adverse Events
Asthenia
|
1 Participants
|
—
|
—
|
|
Number of Subjects With Serious and Non Serious Adverse Events
Fatigue
|
1 Participants
|
—
|
—
|
|
Number of Subjects With Serious and Non Serious Adverse Events
Pyrexia
|
1 Participants
|
—
|
—
|
|
Number of Subjects With Serious and Non Serious Adverse Events
Bronchitis
|
1 Participants
|
—
|
—
|
|
Number of Subjects With Serious and Non Serious Adverse Events
Eye infection
|
1 Participants
|
—
|
—
|
|
Number of Subjects With Serious and Non Serious Adverse Events
Lyme disease
|
1 Participants
|
—
|
—
|
|
Number of Subjects With Serious and Non Serious Adverse Events
Nasopharyngitis
|
4 Participants
|
—
|
—
|
|
Number of Subjects With Serious and Non Serious Adverse Events
Tooth abscess
|
1 Participants
|
—
|
—
|
|
Number of Subjects With Serious and Non Serious Adverse Events
Skin abrasion
|
1 Participants
|
—
|
—
|
|
Number of Subjects With Serious and Non Serious Adverse Events
Skin laceration
|
2 Participants
|
—
|
—
|
|
Number of Subjects With Serious and Non Serious Adverse Events
Blood pressure increased
|
1 Participants
|
—
|
—
|
|
Number of Subjects With Serious and Non Serious Adverse Events
Weight decreased
|
1 Participants
|
—
|
—
|
|
Number of Subjects With Serious and Non Serious Adverse Events
Arthralgia
|
1 Participants
|
—
|
—
|
|
Number of Subjects With Serious and Non Serious Adverse Events
Myalgia
|
1 Participants
|
—
|
—
|
|
Number of Subjects With Serious and Non Serious Adverse Events
Headache
|
3 Participants
|
—
|
—
|
|
Number of Subjects With Serious and Non Serious Adverse Events
Depression
|
2 Participants
|
—
|
—
|
|
Number of Subjects With Serious and Non Serious Adverse Events
Haematuria
|
1 Participants
|
—
|
—
|
|
Number of Subjects With Serious and Non Serious Adverse Events
Cough
|
1 Participants
|
—
|
—
|
|
Number of Subjects With Serious and Non Serious Adverse Events
Dyspnoea
|
1 Participants
|
—
|
—
|
|
Number of Subjects With Serious and Non Serious Adverse Events
Pneumonia aspiration
|
1 Participants
|
—
|
—
|
|
Number of Subjects With Serious and Non Serious Adverse Events
Respiratory failure
|
1 Participants
|
—
|
—
|
|
Number of Subjects With Serious and Non Serious Adverse Events
Respiratory tract congestion
|
1 Participants
|
—
|
—
|
|
Number of Subjects With Serious and Non Serious Adverse Events
Rash
|
1 Participants
|
—
|
—
|
SECONDARY outcome
Timeframe: Screening, on Day 0 before the treatment (injection), on Day 30, Day 60, Day 90, at 6 months and 9 monthsPopulation: Safety analysis set
The Amyotrophic Lateral Sclerosis Function Rating Scale includes twelve questions that ask the physician to rate his/her impression of the patient's level of functional impairment in performing one of twelve common tasks (e.g., climbing stairs). Each task is rated on a five-point scale from 0 = can't do, to 4 = normal ability. Individual item scores are summed to produce a reported score of between 0 = worst and 48 = best.
Outcome measures
| Measure |
Total
n=9 Participants
All subjects in Groups 1 and 2
|
Engensis (VM202) Group 2
n=9 Participants
Total dose of 64 mg of VM202, administered over the course of four visits: Day 0, Day 7, Day 14, and Day 21. As in all previous VM202 studies, final dose of VM202 for each target muscle group is divided and administered 2 weeks apart. Group 2 received Engensis IM injections beginning with the upper limbs, with injection location (lower vs upper limbs) alternating weekly
VM202
|
Overall
n=18 Participants
Total subjects from Groups 1 and 2
|
|---|---|---|---|
|
The Revised Amyotrophic Lateral Sclerosis Functional Rating Scale (ALSFRS-R)
Screening
|
39.89 units on a scale
Standard Deviation 3.26
|
38.11 units on a scale
Standard Deviation 4.62
|
39 units on a scale
Standard Deviation 3.99
|
|
The Revised Amyotrophic Lateral Sclerosis Functional Rating Scale (ALSFRS-R)
Day 0
|
40.22 units on a scale
Standard Deviation 2.99
|
37.11 units on a scale
Standard Deviation 5.25
|
38.67 units on a scale
Standard Deviation 4.45
|
|
The Revised Amyotrophic Lateral Sclerosis Functional Rating Scale (ALSFRS-R)
Day 30
|
40 units on a scale
Standard Deviation 3.94
|
35 units on a scale
Standard Deviation 6.44
|
37.5 units on a scale
Standard Deviation 5.78
|
|
The Revised Amyotrophic Lateral Sclerosis Functional Rating Scale (ALSFRS-R)
Day 90
|
38.22 units on a scale
Standard Deviation 3.11
|
33.89 units on a scale
Standard Deviation 6.05
|
36.06 units on a scale
Standard Deviation 5.17
|
|
The Revised Amyotrophic Lateral Sclerosis Functional Rating Scale (ALSFRS-R)
Day 60
|
39.25 units on a scale
Standard Deviation 3.65
|
33.44 units on a scale
Standard Deviation 6.46
|
36.18 units on a scale
Standard Deviation 5.97
|
|
The Revised Amyotrophic Lateral Sclerosis Functional Rating Scale (ALSFRS-R)
Month 6
|
35.5 units on a scale
Standard Deviation 4.5
|
29.25 units on a scale
Standard Deviation 8.5
|
32.38 units on a scale
Standard Deviation 7.32
|
|
The Revised Amyotrophic Lateral Sclerosis Functional Rating Scale (ALSFRS-R)
Month 9
|
31.22 units on a scale
Standard Deviation 4.97
|
25.88 units on a scale
Standard Deviation 8.51
|
28.71 units on a scale
Standard Deviation 7.18
|
SECONDARY outcome
Timeframe: Day 0, Day 30, Day 60, Day 90, at 6 months and 9 monthsThe Medical Research Council (MRC) Scale is a validated instrument used in assessing muscle strength. It uses the numeral grades 0-5 to characterize muscle strength as follows: 0 - No contraction;1 - Flicker or trace contraction; 2 - Active movement, with gravity eliminated; 3 - Active movement against gravity; 4 - Active movement against gravity and resistance; 5 - Normal power The MRC scale was used to assess muscle strength in the muscle groups injected with Engensis.
Outcome measures
| Measure |
Total
n=9 Participants
All subjects in Groups 1 and 2
|
Engensis (VM202) Group 2
n=9 Participants
Total dose of 64 mg of VM202, administered over the course of four visits: Day 0, Day 7, Day 14, and Day 21. As in all previous VM202 studies, final dose of VM202 for each target muscle group is divided and administered 2 weeks apart. Group 2 received Engensis IM injections beginning with the upper limbs, with injection location (lower vs upper limbs) alternating weekly
VM202
|
Overall
n=18 Participants
Total subjects from Groups 1 and 2
|
|---|---|---|---|
|
Change in Mean Muscle Strength Medical Research Council (MRC) Scores
Day 90
|
-5.11 score on a scale
Standard Deviation 4.7
|
-4.56 score on a scale
Standard Deviation 7.97
|
-4.83 score on a scale
Standard Deviation 6.35
|
|
Change in Mean Muscle Strength Medical Research Council (MRC) Scores
Month 9
|
-17.38 score on a scale
Standard Deviation 11.39
|
-18.63 score on a scale
Standard Deviation 10.03
|
-18.0 score on a scale
Standard Deviation 10.39
|
|
Change in Mean Muscle Strength Medical Research Council (MRC) Scores
Day 30
|
-0.67 score on a scale
Standard Deviation 3.54
|
-3.78 score on a scale
Standard Deviation 5.78
|
-2.22 score on a scale
Standard Deviation 4.92
|
|
Change in Mean Muscle Strength Medical Research Council (MRC) Scores
Day 60
|
-0.63 score on a scale
Standard Deviation 3.81
|
-2.89 score on a scale
Standard Deviation 3.59
|
-1.82 score on a scale
Standard Deviation 3.76
|
|
Change in Mean Muscle Strength Medical Research Council (MRC) Scores
Month 6
|
-8.25 score on a scale
Standard Deviation 5.2
|
-13.63 score on a scale
Standard Deviation 7.25
|
-10.94 score on a scale
Standard Deviation 6.7
|
SECONDARY outcome
Timeframe: Day 30, Day 60, Day 90, at 6 months and 9 monthsPopulation: Safety analysis set
pulmonary function test that quantifies the volume of air that can forcibly be blown out after full inspiration. It correlates with survival in ALS
Outcome measures
| Measure |
Total
n=9 Participants
All subjects in Groups 1 and 2
|
Engensis (VM202) Group 2
n=9 Participants
Total dose of 64 mg of VM202, administered over the course of four visits: Day 0, Day 7, Day 14, and Day 21. As in all previous VM202 studies, final dose of VM202 for each target muscle group is divided and administered 2 weeks apart. Group 2 received Engensis IM injections beginning with the upper limbs, with injection location (lower vs upper limbs) alternating weekly
VM202
|
Overall
n=18 Participants
Total subjects from Groups 1 and 2
|
|---|---|---|---|
|
Change From Baseline (Day 0) in Forced Vital Capacity (%)
Day 30
|
-1.67 percentage
Standard Deviation 5.12
|
-4.11 percentage
Standard Deviation 6.15
|
-2.89 percentage
Standard Deviation 5.63
|
|
Change From Baseline (Day 0) in Forced Vital Capacity (%)
Month 9
|
-16.63 percentage
Standard Deviation 11.83
|
-29.88 percentage
Standard Deviation 18.78
|
-23.25 percentage
Standard Deviation 16.64
|
|
Change From Baseline (Day 0) in Forced Vital Capacity (%)
Day 60
|
-5 percentage
Standard Deviation 7.03
|
-8.33 percentage
Standard Deviation 10.3
|
-6.76 percentage
Standard Deviation 8.81
|
|
Change From Baseline (Day 0) in Forced Vital Capacity (%)
Day 90
|
-8 percentage
Standard Deviation 7.5
|
-13.44 percentage
Standard Deviation 12.45
|
-10.72 percentage
Standard Deviation 10.36
|
|
Change From Baseline (Day 0) in Forced Vital Capacity (%)
Month 6
|
-11.13 percentage
Standard Deviation 9.76
|
-24 percentage
Standard Deviation 22.17
|
-17.56 percentage
Standard Deviation 17.84
|
Adverse Events
Total
Serious events: 3 serious events
Other events: 17 other events
Deaths: 1 deaths
Serious adverse events
| Measure |
Total
n=18 participants at risk
All subjects in Groups 1 and 2
|
|---|---|
|
Gastrointestinal disorders
small intestinal obstruction
|
5.6%
1/18 • Number of events 1 • Day 0 through the Month 9 visit
|
|
Infections and infestations
Bronchitis
|
5.6%
1/18 • Number of events 1 • Day 0 through the Month 9 visit
|
|
Infections and infestations
Urinary tract infection
|
5.6%
1/18 • Number of events 1 • Day 0 through the Month 9 visit
|
|
Respiratory, thoracic and mediastinal disorders
Pneumonia aspiration
|
5.6%
1/18 • Number of events 1 • Day 0 through the Month 9 visit
|
|
Respiratory, thoracic and mediastinal disorders
Respiratory failure
|
5.6%
1/18 • Number of events 1 • Day 0 through the Month 9 visit
|
Other adverse events
| Measure |
Total
n=18 participants at risk
All subjects in Groups 1 and 2
|
|---|---|
|
Gastrointestinal disorders
diarrhoea
|
5.6%
1/18 • Number of events 1 • Day 0 through the Month 9 visit
|
|
Gastrointestinal disorders
Gastroesophageal reflux disease
|
5.6%
1/18 • Number of events 1 • Day 0 through the Month 9 visit
|
|
General disorders
Asthenia
|
5.6%
1/18 • Number of events 1 • Day 0 through the Month 9 visit
|
|
General disorders
Fatigue
|
5.6%
1/18 • Number of events 1 • Day 0 through the Month 9 visit
|
|
General disorders
Pyrexia
|
5.6%
1/18 • Number of events 1 • Day 0 through the Month 9 visit
|
|
Infections and infestations
Eye infection
|
5.6%
1/18 • Number of events 1 • Day 0 through the Month 9 visit
|
|
Infections and infestations
Lyme disease
|
5.6%
1/18 • Number of events 1 • Day 0 through the Month 9 visit
|
|
Infections and infestations
Nasopharyngitis
|
22.2%
4/18 • Number of events 4 • Day 0 through the Month 9 visit
|
|
Infections and infestations
Tooth abscess
|
5.6%
1/18 • Number of events 1 • Day 0 through the Month 9 visit
|
|
Injury, poisoning and procedural complications
Fall
|
61.1%
11/18 • Number of events 21 • Day 0 through the Month 9 visit
|
|
Injury, poisoning and procedural complications
Muscle strain
|
5.6%
1/18 • Number of events 1 • Day 0 through the Month 9 visit
|
|
Injury, poisoning and procedural complications
Skin abraision
|
5.6%
1/18 • Number of events 1 • Day 0 through the Month 9 visit
|
|
Injury, poisoning and procedural complications
Skin laceration
|
11.1%
2/18 • Number of events 2 • Day 0 through the Month 9 visit
|
|
Investigations
Blood pressure increased
|
5.6%
1/18 • Number of events 1 • Day 0 through the Month 9 visit
|
|
Investigations
Weight decreased
|
5.6%
1/18 • Number of events 1 • Day 0 through the Month 9 visit
|
|
Musculoskeletal and connective tissue disorders
Arthralgia
|
5.6%
1/18 • Number of events 1 • Day 0 through the Month 9 visit
|
|
Musculoskeletal and connective tissue disorders
Myalgia
|
5.6%
1/18 • Number of events 1 • Day 0 through the Month 9 visit
|
|
Nervous system disorders
Headache
|
16.7%
3/18 • Number of events 3 • Day 0 through the Month 9 visit
|
|
Psychiatric disorders
Depression
|
11.1%
2/18 • Number of events 2 • Day 0 through the Month 9 visit
|
|
Renal and urinary disorders
Haematuria
|
5.6%
1/18 • Number of events 1 • Day 0 through the Month 9 visit
|
|
Respiratory, thoracic and mediastinal disorders
Aspiration
|
5.6%
1/18 • Number of events 1 • Day 0 through the Month 9 visit
|
|
Respiratory, thoracic and mediastinal disorders
Cough
|
5.6%
1/18 • Number of events 1 • Day 0 through the Month 9 visit
|
|
Respiratory, thoracic and mediastinal disorders
Dyspnoea
|
5.6%
1/18 • Number of events 1 • Day 0 through the Month 9 visit
|
|
Respiratory, thoracic and mediastinal disorders
Respiratory tract congestion
|
5.6%
1/18 • Number of events 1 • Day 0 through the Month 9 visit
|
|
Skin and subcutaneous tissue disorders
Rash
|
5.6%
1/18 • Number of events 1 • Day 0 through the Month 9 visit
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place