A Phase 1/2 interventional study of VM202 in Amyotrophic Lateral Sclerosis, sponsored by Helixmith Co., Ltd.. Completed at 1 site in United States. Open to participants aged 21 Years to 75 Years. Per ClinicalTrials.gov, last updated 2025-10-06.
Sponsored by Helixmith Co., Ltd. · Phase 1/2, Interventional, and Treatment
The purpose of this study is to determine the safety and tolerability of intramuscular injections of VM202 at different injection sites in people with amyotrophic lateral sclerosis.
A phase I/II, open label, single center study designed to assess the safety and tolerability of intramuscular injections of VM202 inpatients with Amyotrophic Lateral Sclerosis. Study enrollment will be staged. Enrollment will be halted after the sixth subject qualifies for treatment. A Data Safety Monitoring Board will conduct a safety evaluation after the first patient treated completes the Day 60 follow-up evaluation and the five other sequentially enrolled subjects complete at least the Day 30 follow-up. Enrollment will be suspended until a formal recommendation to proceed (or not proceed) is made by the Data Safety Monitoring Board.
Patients aged ≥ 21 years, but ≤ 75 years diagnosed with clinically definite, clinically probable, or clinically probable-laboratory supported Amyotrophic Lateral Sclerosis.
This study is not powered to detect differences in efficacy measures. However, descriptive statistics (N, mean, median, standard deviation, minimum and maximum values, where applicable) of clinically meaningful endpoints will be tabulated
981 studies on the registry are indexed under Amyotrophic Lateral Sclerosis; 283 are open to participants now.
This study's enrollment of 18 is below the median of 36 across 667 interventional studies indexed under Amyotrophic Lateral Sclerosis.
Browse Amyotrophic Lateral Sclerosis studies →Helixmith Co., Ltd. is the lead sponsor of 19 studies on the registry; none are open to participants now.
Of its 6 completed or terminated interventional studies of FDA-regulated products, 6 (100%) have results posted.
Counted across the registry records on this site, refreshed daily.
Subjects diagnosed with:
Exclusion Criteria:
Total dose of 64 mg of VM202 It will be 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.
Biological: VM202
Number of Subjects With Serious and Non Serious Adverse Events
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.
Time frame: Throughout the nine month follow up
The Revised Amyotrophic Lateral Sclerosis Functional Rating Scale (ALSFRS-R)
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.
Time frame: Screening, on Day 0 before the treatment (injection), on Day 30, Day 60, Day 90, at 6 months and 9 months
Change in Mean Muscle Strength Medical Research Council (MRC) Scores
The 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.
Time frame: Day 0, Day 30, Day 60, Day 90, at 6 months and 9 months
Change From Baseline (Day 0) in Forced Vital Capacity (%)
pulmonary function test that quantifies the volume of air that can forcibly be blown out after full inspiration. It correlates with survival in ALS
Time frame: Day 30, Day 60, Day 90, at 6 months and 9 months
| Milestone | Engensis (VM202) Group 1 | Engensis (VM202) Group 2 |
|---|---|---|
| Started | 9 | 9 |
| Completed | 9 | 8 |
| Not completed | 0 | 1 |
| Withdrew: Adverse event | 0 | 1 |
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.
| Participants | Total |
|---|---|
| Diarrhoea | 1 |
| Gastroesophargeal reflux disease | 1 |
| Small intestinal obstruction | 1 |
| Asthenia | 1 |
| Fatigue | 1 |
| Pyrexia | 1 |
| Bronchitis | 1 |
| Eye infection | 1 |
| Lyme disease | 1 |
| Nasopharyngitis | 4 |
| Tooth abscess | 1 |
| Urinary tract infection | 1 |
| Fall | 11 |
| Muscle strain | 1 |
| Skin abrasion | 1 |
| Skin laceration | 2 |
| Blood pressure increased | 1 |
| Weight decreased | 1 |
| Arthralgia | 1 |
| Myalgia | 1 |
| Headache | 3 |
| Depression | 2 |
| Haematuria | 1 |
| Aspiration | 1 |
| Cough | 1 |
| Dyspnoea | 1 |
| Pneumonia aspiration | 1 |
| Respiratory failure | 1 |
| Respiratory tract congestion | 1 |
| Rash | 1 |
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.
| units on a scale | Engensis (VM202) Group 1 | Engensis (VM202) Group 2 | Overall |
|---|---|---|---|
| Screening | 39.89 ± 3.26 | 38.11 ± 4.62 | 39 ± 3.99 |
| Day 0 | 40.22 ± 2.99 | 37.11 ± 5.25 | 38.67 ± 4.45 |
| Day 30 | 40 ± 3.94 | 35 ± 6.44 | 37.5 ± 5.78 |
| Day 60 | 39.25 ± 3.65 | 33.44 ± 6.46 | 36.18 ± 5.97 |
| Day 90 | 38.22 ± 3.11 | 33.89 ± 6.05 | 36.06 ± 5.17 |
| Month 6 | 35.5 ± 4.5 | 29.25 ± 8.5 | 32.38 ± 7.32 |
| Month 9 | 31.22 ± 4.97 | 25.88 ± 8.51 | 28.71 ± 7.18 |
The 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.
| score on a scale | Engensis (VM202) Group 1 | Engensis (VM202) Group 2 | Overall |
|---|---|---|---|
| Day 30 | -0.67 ± 3.54 | -3.78 ± 5.78 | -2.22 ± 4.92 |
| Day 60 | -0.63 ± 3.81 | -2.89 ± 3.59 | -1.82 ± 3.76 |
| Day 90 | -5.11 ± 4.7 | -4.56 ± 7.97 | -4.83 ± 6.35 |
| Month 6 | -8.25 ± 5.2 | -13.63 ± 7.25 | -10.94 ± 6.7 |
| Month 9 | -17.38 ± 11.39 | -18.63 ± 10.03 | -18.0 ± 10.39 |
pulmonary function test that quantifies the volume of air that can forcibly be blown out after full inspiration. It correlates with survival in ALS
| percentage | Engensis (VM202) Group 1 | Engensis (VM202) Group 2 | Overall |
|---|---|---|---|
| Day 30 | -1.67 ± 5.12 | -4.11 ± 6.15 | -2.89 ± 5.63 |
| Day 60 | -5 ± 7.03 | -8.33 ± 10.3 | -6.76 ± 8.81 |
| Day 90 | -8 ± 7.5 | -13.44 ± 12.45 | -10.72 ± 10.36 |
| Month 6 | -11.13 ± 9.76 | -24 ± 22.17 | -17.56 ± 17.84 |
| Month 9 | -16.63 ± 11.83 | -29.88 ± 18.78 | -23.25 ± 16.64 |
Collected over Day 0 through the Month 9 visit. Non-serious events are listed at a 0% frequency threshold.
| Group | Deaths | Serious | Other |
|---|---|---|---|
| Total | 1/18 (5.6%) | 3/18 (16.7%) | 17/18 (94.4%) |
| Event | Total |
|---|---|
| small intestinal obstructionGastrointestinal disorders | 1/18 |
| BronchitisInfections and infestations | 1/18 |
| Urinary tract infectionInfections and infestations | 1/18 |
| Pneumonia aspirationRespiratory, thoracic and mediastinal disorders | 1/18 |
| Respiratory failureRespiratory, thoracic and mediastinal disorders | 1/18 |
| Event | Total |
|---|---|
| FallInjury, poisoning and procedural complications | 11/18 |
| NasopharyngitisInfections and infestations | 4/18 |
| HeadacheNervous system disorders | 3/18 |
| Skin lacerationInjury, poisoning and procedural complications | 2/18 |
| DepressionPsychiatric disorders | 2/18 |
| diarrhoeaGastrointestinal disorders | 1/18 |
| Gastroesophageal reflux diseaseGastrointestinal disorders | 1/18 |
| AstheniaGeneral disorders | 1/18 |
| FatigueGeneral disorders | 1/18 |
| PyrexiaGeneral disorders | 1/18 |
Safety analysis set
| Age, Continuous(years) | Engensis (VM202) Group 1 | Engensis (VM202) Group 2 | Total |
|---|---|---|---|
| Mean | 49.1 ± 11.9 | 55.8 ± 3.0 | 52.4 ± 9.1 |
| Sex: Female, Male(Participants) | Engensis (VM202) Group 1 | Engensis (VM202) Group 2 | Total |
|---|---|---|---|
| Female | 1 | 2 | 3 |
| Male | 8 | 7 | 15 |
| Race (NIH/OMB)(Participants) | Engensis (VM202) Group 1 | Engensis (VM202) Group 2 | Total |
|---|---|---|---|
| American Indian or Alaska Native | 0 | 0 | 0 |
| Asian | 0 | 0 | 0 |
| Native Hawaiian or Other Pacific Islander | 0 | 0 | 0 |
| Black or African American | 0 | 0 | 0 |
| White | 9 | 9 | 18 |
| More than one race | 0 | 0 | 0 |
| Unknown or Not Reported | 0 | 0 | 0 |
| Region of Enrollment(participants) | Engensis (VM202) Group 1 | Engensis (VM202) Group 2 | Total |
|---|---|---|---|
| United States | 9 | 9 | 18 |
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Amyotrophic Lateral Sclerosis→
Helixmith Co., Ltd.