Adults 18 to 80, any sex, with Amyotrophic Lateral Sclerosis. Patients with the condition only — healthy volunteers not accepted.
Results — posted to ClinicalTrials.gov
Per-arm endpoint measurements with 95% confidence intervals where reported. Source: trial results section.
Safety of Intramuscular Injections of Engensis in Participants With Amyotrophic Lateral Sclerosis Compared to PlaceboPrimary· From the Day 0 Visit to the Day 180 Visit
Incidence of treatment-emergent adverse events in more than or equal to 10% of subjects, and treatment-emergent serious adverse events after injections, injection site reactions, and clinically significant laboratory values for Engensis compared to Placebo.
Injection Site bruising
Group
Value
95% CI
Engensis
4
Placebo
3
Pyrexia
Group
Value
95% CI
Engensis
3
Placebo
1
Injection site pain
Group
Value
95% CI
Engensis
2
Placebo
1
Asthenia
Group
Value
95% CI
Engensis
0
Placebo
2
Fatigue
Group
Value
95% CI
Engensis
1
Placebo
1
Arthralgia
Group
Value
95% CI
Engensis
2
Placebo
0
Muscle spasms
Group
Value
95% CI
Engensis
1
Placebo
1
Musculoskeletal stiffness
Group
Value
95% CI
Engensis
2
Placebo
0
Changes in Muscle Function Following Engensis Injections Compared to PlaceboSecondary· Day 0 to Day 180
Change from Baseline (Day 0) in total mean Revised Amyotrophic Lateral Sclerosis Function Rating scores, subscores for Fine and Gross Motor Functions and Bulbar Function, and slope of the total score.
The Revised Amyotrophic Lateral Sclerosis Function Rating scores 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
Group
Value
95% CI
Engensis
-5.9
± 4.2
Placebo
-4.2
± 4.76
Evaluation of Muscle Strength Changes Following Engensis Injections Compared to Placebo - Hand-Held DynamometrySecondary· Day 0 to Day 180
As assessed bilaterally by Hand-Held Dynamometry in muscles in the upper and lower extremities.
Muscle Strength as Measured by Handheld Dynamometry (lbs.) and Change from Baseline to Day 180 by Muscle Group (ITT Population)
Shoulder Flexion, left
Group
Value
95% CI
Engensis
-6.48
± 8.317
Placebo
-3.33
± 1.552
Shoulder Flexion, right
Group
Value
95% CI
Engensis
-7.608
± 9.2493
Placebo
-2.100
± 4.4234
Elbow Flexion, left
Group
Value
95% CI
Engensis
-10.562
± 12.7571
Placebo
-3.250
± 4.4004
Elbow Flexion, right
Group
Value
95% CI
Engensis
-10.68
± 11.813
Placebo
-1.68
± 3.174
Elbow Extension, left
Group
Value
95% CI
Engensis
-6.424
± 11.0173
Placebo
2.350
± 1.1590
Elbow Extension, right
Group
Value
95% CI
Engensis
-6.83
± 8.925
Placebo
-1.08
± 5.171
Wrist Extension, left
Group
Value
95% CI
Engensis
-7.162
± 7.8940
Placebo
4.943
± 8.5556
Wrist Extension, right
Group
Value
95% CI
Engensis
-6.53
± 10.081
Placebo
-2.18
± 4.350
Evaluation of Quality of Life Improvement Following Engensis Injections Compared to Placebo - Exploratory OutcomeSecondary· Day 0 to Day 84, and Day 0 to 180
The Amyotrophic Lateral Sclerosis Assessment Questionnaire has 40 items (ALSAQ-40), and is a standardized Quality of Life assessment.
The Participant completes the 40 questions in the ALSAQ-40 with 5 categories: Physical Mobility, Activities of Daily Living and Independence, Eating and Drinking, Communication, and Emotional Reactions. Each question has 5 responses to select from: 0-Never (Best Case), 1-Rarely, 2-Sometime, 3-Often, and 4-Always (Worst Case).
Note for each question there is a Minimum of 0 (Best), to the Maximum 4 (Worst). Decreasing scores indicates improvement of symptoms.
T
Day 84 - Physical Mobility
Group
Value
95% CI
Engensis
1.88
± 20.255
Placebo
-1.5
± 5.755
Day 180 - Physical Mobility
Group
Value
95% CI
Engensis
13.44
± 24.014
Placebo
11.88
± 17.366
Day 84 - Activities of Daily Living/Independence
Group
Value
95% CI
Engensis
10.31
± 9.396
Placebo
0.50
± 8.178
Day 180 - Activities of Daily Living/Independence
Group
Value
95% CI
Engensis
15.94
± 17.369
Placebo
11.25
± 12.332
Day 84 - Eating and Drinking
Group
Value
95% CI
Engensis
8.34
± 12.596
Placebo
11.66
± 21.732
Day 180 - Eating and Drinking
Group
Value
95% CI
Engensis
17.73
± 18.623
Placebo
14.58
± 21.886
Day 84 - Communication
Group
Value
95% CI
Engensis
11.15
± 24.334
Placebo
7.14
± 13.363
Day 180 - Communication
Group
Value
95% CI
Engensis
24.56
± 27.690
Placebo
11.60
± 25.977
Evaluation of Patient Reported Outcome Improvement Following Engensis Injections Compared to PlaceboSecondary· Day 84 and Day 180 (End of Study or Early Termination)
The subject's impression of change after treatment was measured with the Patient Global Impression of Change questionnaire through use of the electronic Patient Reported Outcome . This questionnaire measures the subject's perception of how treatment has affected their level of activity, symptoms, emotions, and overall quality of life.
Each descriptor is ranked on an increasing improvement scale; where 1 = No change (or condition has got worse), 2=Almost the same, hardly any change at all, 3=A little better, but no noticeable change, 4=Somewhat better, but the change has not made any real diff
Day 84
Group
Value
95% CI
Engensis
3.1
± 1.81
Placebo
4.6
± 0.89
End of Study / Early Term- Day 180
Group
Value
95% CI
Engensis
2.9
± 1.46
Placebo
4.0
± 2.00
Evaluation of Clinical Reported Outcome Improvement Following Engensis Injections Compared to PlaceboSecondary· Day 84 to Day 180
The Clinical Global Impression of Change is a validated instrument completed by observers as an assessment of Quality of Life. The Clinical Global Impression of Change is an 8-point scale with scores ranging from Marked Improvement, Moderate Improvement, Minimal Improvement, Slight Improvement, and Unchanged (or Worse), along with an efficacy index with questions in a matrix for therapeutic effect and side effects.The test was completed on Days 84 and 180/ Early Termination.
Day 84 - Minimally improved
Group
Value
95% CI
Engensis
2
Placebo
3
Day 84 - Minimally worse
Group
Value
95% CI
Engensis
5
Placebo
1
Day 84 - No change
Group
Value
95% CI
Engensis
3
Placebo
2
End Study/Early term- Minimally improved
Group
Value
95% CI
Engensis
0
Placebo
1
End Study/Early term- Minimally worse
Group
Value
95% CI
Engensis
4
Placebo
0
End Study/Early term- Much worse
Group
Value
95% CI
Engensis
2
Placebo
1
End Study/Early term- No change
Group
Value
95% CI
Engensis
4
Placebo
2
To Determine Effects of Engensis on Respiratory Function Compared to Placebo - Slow Vital CapacitySecondary· Day 0 to Day 180
Slow vital capacity is a pulmonary function test that quantifies the volume of air that can be slowly exhaled after slow maximum inhalation, and was to be measured at Screening, pre-dose on Days 60 and 120, and on Days 30, 84, 144, and 180.
Day 30
Group
Value
95% CI
Engensis
-3.31
± 4.191
Placebo
2.53
± 7.501
Day 60 (pre-dose)
Group
Value
95% CI
Engensis
-6.50
± 9.439
Placebo
-3.90
± 9.173
Day 84
Group
Value
95% CI
Engensis
-6.50
± 11.808
Placebo
-2.3
± 9.182
Day 120 (pre-dose)
Group
Value
95% CI
Engensis
-12.76
± 13.363
Placebo
-8.84
± 12.916
Day 144
Group
Value
95% CI
Engensis
-13.46
± 17.605
Placebo
-9.04
± 15.925
Day 180
Group
Value
95% CI
Engensis
-17.80
± 17.308
Placebo
-1.75
± 10.046
To Determine Effects of Engensis on Respiratory Function Compared to Placebo - TracheostomySecondary· Day 0 to Day 180
Total Participants that experienced Tracheostomy during the study in the Intent-to-treat population.
Group
Value
95% CI
Engensis
0
Placebo
0
To Determine Effects of Engensis on Survival Compared to Placebo - DeathsSecondary· Day 0 to Day 180
Participants that died during the study, for all causes including Treatment Emergent Adverse Events, or Treatment Emergent Serious Adverse Events, leading to study discontinuation, or study drug withdrawal in any subject.
Group
Value
95% CI
Engensis
0
Placebo
0
Adverse events — posted to ClinicalTrials.gov
Time frame: From Day 0 to Day 180 or Early Termination.
Reporting threshold: 0%.
Adverse-event reports describe events observed during the trial — not all are caused by the drug.
The purpose of this study was to evaluate the safety of intramuscular administration of Engensis in Participants with Amyotrophic Lateral Sclerosis as compared to Placebo. Safety will be assessed by incidences of treatment-emergent adverse events, treatment-emergent serious adverse events, injection site reactions and other adverse events of special interest, and the clinically significant laboratory values after injections of Engensis compared to Placebo. Exploratory endpoints include assessment of muscle function using the Revised Amyotrophic Lateral Sclerosis Functional Rating Scale subscores for Fine and Gross Motor Function; muscle strength by quantitative testing using handheld dynamometry and the Accurate Test of Limb Isometric Strength where available; quality of life using the Amyotrophic Lateral Sclerosis Assessment Questionnaire-40; patient global impression of change, clinical global impression of change, and clinical global impression of severity; and evaluation of lung function using Slow Vital Capacity. Muscle biopsies will be performed during the study for future biomarker analyses.
Publications & conference data
8 peer-reviewed publications reference this trial (live from Europe PMC):
NCT05176093 — A 6-Month Extension Study to Assess the Long-Term Safety of Engensis in Amyotrophic Lateral Sclerosis
· Phase 2
· completed
NCT04873232 — A 6-Month Extension Study of VMDN-003-2 to Assess Engensis in Participants With Painful Diabetic Peripheral Neuropathy
· Phase 3
· completed
NCT04469270 — Study to Assess Safety and Efficacy of Engensis in Painful Diabetic Peripheral Neuropathy
· Phase 3
· completed
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Other Helixmith Co., Ltd. trials
Trials by the same sponsor.
NCT05176093 — A 6-Month Extension Study to Assess the Long-Term Safety of Engensis in Amyotrophic Lateral Sclerosis
· Phase 2
· completed
NCT04873232 — A 6-Month Extension Study of VMDN-003-2 to Assess Engensis in Participants With Painful Diabetic Peripheral Neuropathy
· Phase 3
· completed
NCT05552625 — The Efficacy and Safety of TADIOS as an Adjuvant Therapy in Patients Diagnosed With Mild to Moderate COVID-19
· Phase 2, PHASE3
· withdrawn
NCT04469270 — Study to Assess Safety and Efficacy of Engensis in Painful Diabetic Peripheral Neuropathy
· Phase 3
· completed
NCT05361031 — The Safety and Tolerability of Engensis (VM202) in Patients With Charcot-Marie-Tooth Disease Subtype 1A (CMT1A)
· Phase 1, PHASE2
· completed
Publications: Europe PMC API search by NCT ID, retrieved 10 June 2026
Drug + disease cross-links: matched in real time against Drug Landscape's normalised drug + company + condition tables
Sponsor: as reported to ClinicalTrials.gov by Helixmith Co., Ltd.
Last refreshed: 6 October 2025
Drug Landscape aggregates and links these public records for informational use only. Always verify against the primary source before clinical or regulatory decisions. Canonical URL: https://druglandscape.com/trial/NCT04632225.