Trial Outcomes & Findings for Safety of Engensis in Participants With Amyotrophic Lateral Sclerosis (NCT NCT04632225)
NCT ID: NCT04632225
Last Updated: 2025-10-06
Results Overview
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.
Recruitment status
COMPLETED
Study phase
PHASE2
Target enrollment
18 participants
Primary outcome timeframe
From the Day 0 Visit to the Day 180 Visit
Results posted on
2025-10-06
Participant Flow
Participant milestones
| Measure |
Engensis
64 mg Engensis per Treatment Cycle, with each of 3 cycles composed of 2 days of 128 injections each to the right and left target muscles, spaced 2 weeks apart
Engensis: Lyophilized biologic to be reconstituted containing Engensis
|
Placebo
32 mL of Placebo per Treatment Cycle, with each of 3 cycles composed of 2 days of 128 injections each to the right and left target muscles, spaced 2 weeks apart
Placebo: Injectable liquid
|
|---|---|---|
|
Overall Study
STARTED
|
12
|
6
|
|
Overall Study
COMPLETED
|
10
|
4
|
|
Overall Study
NOT COMPLETED
|
2
|
2
|
Reasons for withdrawal
| Measure |
Engensis
64 mg Engensis per Treatment Cycle, with each of 3 cycles composed of 2 days of 128 injections each to the right and left target muscles, spaced 2 weeks apart
Engensis: Lyophilized biologic to be reconstituted containing Engensis
|
Placebo
32 mL of Placebo per Treatment Cycle, with each of 3 cycles composed of 2 days of 128 injections each to the right and left target muscles, spaced 2 weeks apart
Placebo: Injectable liquid
|
|---|---|---|
|
Overall Study
Withdrawal by Subject
|
2
|
2
|
Baseline Characteristics
Safety of Engensis in Participants With Amyotrophic Lateral Sclerosis
Baseline characteristics by cohort
| Measure |
Engensis
n=12 Participants
64 mg Engensis per Treatment Cycle, with each of 3 cycles composed of 2 days of 128 injections each to the right and left target muscles, spaced 2 weeks apart
Engensis: Lyophilized biologic to be reconstituted containing Engensis
|
Placebo
n=6 Participants
32 mL of Placebo per Treatment Cycle, with each of 3 cycles composed of 2 days of 128 injections each to the right and left target muscles, spaced 2 weeks apart
Placebo: Injectable liquid
|
Total
n=18 Participants
Total of all reporting groups
|
|---|---|---|---|
|
Age, Categorical
<=18 years
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
12 Participants
n=5 Participants
|
6 Participants
n=7 Participants
|
18 Participants
n=5 Participants
|
|
Age, Categorical
>=65 years
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Age, Continuous
|
46 years
STANDARD_DEVIATION 14.2 • n=5 Participants
|
52.2 years
STANDARD_DEVIATION 8.45 • n=7 Participants
|
48.1 years
STANDARD_DEVIATION 12.67 • n=5 Participants
|
|
Sex: Female, Male
Female
|
2 Participants
n=5 Participants
|
2 Participants
n=7 Participants
|
4 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
10 Participants
n=5 Participants
|
4 Participants
n=7 Participants
|
14 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
12 Participants
n=5 Participants
|
6 Participants
n=7 Participants
|
18 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 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
|
5 Participants
n=5 Participants
|
2 Participants
n=7 Participants
|
7 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
|
7 Participants
n=5 Participants
|
4 Participants
n=7 Participants
|
11 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
South Korea
|
5 participants
n=5 Participants
|
2 participants
n=7 Participants
|
7 participants
n=5 Participants
|
|
Region of Enrollment
United States
|
7 participants
n=5 Participants
|
4 participants
n=7 Participants
|
11 participants
n=5 Participants
|
PRIMARY outcome
Timeframe: From the Day 0 Visit to the Day 180 VisitPopulation: Treatment-Emergent Adverse Events in ≥10% of Subjects Overall by System Organ Class and Preferred Term (Safety Analysis Population)
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.
Outcome measures
| Measure |
Engensis
n=12 Participants
64 mg Engensis per Treatment Cycle, with each of 3 cycles composed of 2 days of 128 injections each to the right and left target muscles, spaced 2 weeks apart
Engensis: Lyophilized biologic to be reconstituted containing Engensis
|
Placebo
n=6 Participants
32 mL of Placebo per Treatment Cycle, with each of 3 cycles composed of 2 days of 128 injections each to the right and left target muscles, spaced 2 weeks apart
Placebo: Injectable liquid
|
|---|---|---|
|
Safety of Intramuscular Injections of Engensis in Participants With Amyotrophic Lateral Sclerosis Compared to Placebo
Fatigue
|
1 Participants
|
1 Participants
|
|
Safety of Intramuscular Injections of Engensis in Participants With Amyotrophic Lateral Sclerosis Compared to Placebo
Arthralgia
|
2 Participants
|
0 Participants
|
|
Safety of Intramuscular Injections of Engensis in Participants With Amyotrophic Lateral Sclerosis Compared to Placebo
Dysarthria
|
1 Participants
|
1 Participants
|
|
Safety of Intramuscular Injections of Engensis in Participants With Amyotrophic Lateral Sclerosis Compared to Placebo
Muscle contractions involuntary
|
2 Participants
|
1 Participants
|
|
Safety of Intramuscular Injections of Engensis in Participants With Amyotrophic Lateral Sclerosis Compared to Placebo
Headache
|
1 Participants
|
1 Participants
|
|
Safety of Intramuscular Injections of Engensis in Participants With Amyotrophic Lateral Sclerosis Compared to Placebo
Cough
|
3 Participants
|
1 Participants
|
|
Safety of Intramuscular Injections of Engensis in Participants With Amyotrophic Lateral Sclerosis Compared to Placebo
Rhinorrhoea
|
2 Participants
|
1 Participants
|
|
Safety of Intramuscular Injections of Engensis in Participants With Amyotrophic Lateral Sclerosis Compared to Placebo
Diarrhoea
|
2 Participants
|
0 Participants
|
|
Safety of Intramuscular Injections of Engensis in Participants With Amyotrophic Lateral Sclerosis Compared to Placebo
Nausea
|
1 Participants
|
1 Participants
|
|
Safety of Intramuscular Injections of Engensis in Participants With Amyotrophic Lateral Sclerosis Compared to Placebo
COVID-19
|
5 Participants
|
1 Participants
|
|
Safety of Intramuscular Injections of Engensis in Participants With Amyotrophic Lateral Sclerosis Compared to Placebo
Insomnia
|
1 Participants
|
1 Participants
|
|
Safety of Intramuscular Injections of Engensis in Participants With Amyotrophic Lateral Sclerosis Compared to Placebo
Hypertension
|
1 Participants
|
1 Participants
|
|
Safety of Intramuscular Injections of Engensis in Participants With Amyotrophic Lateral Sclerosis Compared to Placebo
Injection Site bruising
|
4 Participants
|
3 Participants
|
|
Safety of Intramuscular Injections of Engensis in Participants With Amyotrophic Lateral Sclerosis Compared to Placebo
Pyrexia
|
3 Participants
|
1 Participants
|
|
Safety of Intramuscular Injections of Engensis in Participants With Amyotrophic Lateral Sclerosis Compared to Placebo
Injection site pain
|
2 Participants
|
1 Participants
|
|
Safety of Intramuscular Injections of Engensis in Participants With Amyotrophic Lateral Sclerosis Compared to Placebo
Asthenia
|
0 Participants
|
2 Participants
|
|
Safety of Intramuscular Injections of Engensis in Participants With Amyotrophic Lateral Sclerosis Compared to Placebo
Muscle spasms
|
1 Participants
|
1 Participants
|
|
Safety of Intramuscular Injections of Engensis in Participants With Amyotrophic Lateral Sclerosis Compared to Placebo
Musculoskeletal stiffness
|
2 Participants
|
0 Participants
|
|
Safety of Intramuscular Injections of Engensis in Participants With Amyotrophic Lateral Sclerosis Compared to Placebo
Back pain
|
2 Participants
|
0 Participants
|
|
Safety of Intramuscular Injections of Engensis in Participants With Amyotrophic Lateral Sclerosis Compared to Placebo
Joint range of motion decreased
|
1 Participants
|
1 Participants
|
|
Safety of Intramuscular Injections of Engensis in Participants With Amyotrophic Lateral Sclerosis Compared to Placebo
Myalgia
|
0 Participants
|
2 Participants
|
OTHER_PRE_SPECIFIED outcome
Timeframe: Day 0 to Day 180Population: Revised Amyotrophic Lateral Sclerosis Function Rating scores - Change from Baseline to Day 180 (ITT Population)
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 of between 0 = worst and 48 = best. The Revised Amyotrophic Lateral Sclerosis Function Rating scores was conducted at Screening, pre-dose on Days 0, 60, and 120, and on Days 30, 84, 144, and 180.
Outcome measures
| Measure |
Engensis
n=12 Participants
64 mg Engensis per Treatment Cycle, with each of 3 cycles composed of 2 days of 128 injections each to the right and left target muscles, spaced 2 weeks apart
Engensis: Lyophilized biologic to be reconstituted containing Engensis
|
Placebo
n=6 Participants
32 mL of Placebo per Treatment Cycle, with each of 3 cycles composed of 2 days of 128 injections each to the right and left target muscles, spaced 2 weeks apart
Placebo: Injectable liquid
|
|---|---|---|
|
Changes in Muscle Function Following Engensis Injections Compared to Placebo
|
-5.9 score on a scale
Standard Deviation 4.2
|
-4.2 score on a scale
Standard Deviation 4.76
|
OTHER_PRE_SPECIFIED outcome
Timeframe: Day 0 to Day 180Population: ITT Population
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)
Outcome measures
| Measure |
Engensis
n=12 Participants
64 mg Engensis per Treatment Cycle, with each of 3 cycles composed of 2 days of 128 injections each to the right and left target muscles, spaced 2 weeks apart
Engensis: Lyophilized biologic to be reconstituted containing Engensis
|
Placebo
n=6 Participants
32 mL of Placebo per Treatment Cycle, with each of 3 cycles composed of 2 days of 128 injections each to the right and left target muscles, spaced 2 weeks apart
Placebo: Injectable liquid
|
|---|---|---|
|
Evaluation of Muscle Strength Changes Following Engensis Injections Compared to Placebo - Hand-Held Dynamometry
Elbow Extension, left
|
-6.424 Pounds (lbs.)
Standard Deviation 11.0173
|
2.350 Pounds (lbs.)
Standard Deviation 1.1590
|
|
Evaluation of Muscle Strength Changes Following Engensis Injections Compared to Placebo - Hand-Held Dynamometry
Abductor Pollicus Brevis, right
|
-2.174 Pounds (lbs.)
Standard Deviation 4.2892
|
0.050 Pounds (lbs.)
Standard Deviation 0.1000
|
|
Evaluation of Muscle Strength Changes Following Engensis Injections Compared to Placebo - Hand-Held Dynamometry
Abductor Digiti Minimi, left
|
-0.17 Pounds (lbs.)
Standard Deviation 1.122
|
0.00 Pounds (lbs.)
Standard Deviation 0.000
|
|
Evaluation of Muscle Strength Changes Following Engensis Injections Compared to Placebo - Hand-Held Dynamometry
Knee Extension, left
|
-14.966 Pounds (lbs.)
Standard Deviation 10.2662
|
-1.725 Pounds (lbs.)
Standard Deviation 4.2366
|
|
Evaluation of Muscle Strength Changes Following Engensis Injections Compared to Placebo - Hand-Held Dynamometry
Shoulder Flexion, left
|
-6.48 Pounds (lbs.)
Standard Deviation 8.317
|
-3.33 Pounds (lbs.)
Standard Deviation 1.552
|
|
Evaluation of Muscle Strength Changes Following Engensis Injections Compared to Placebo - Hand-Held Dynamometry
Shoulder Flexion, right
|
-7.608 Pounds (lbs.)
Standard Deviation 9.2493
|
-2.100 Pounds (lbs.)
Standard Deviation 4.4234
|
|
Evaluation of Muscle Strength Changes Following Engensis Injections Compared to Placebo - Hand-Held Dynamometry
Elbow Flexion, left
|
-10.562 Pounds (lbs.)
Standard Deviation 12.7571
|
-3.250 Pounds (lbs.)
Standard Deviation 4.4004
|
|
Evaluation of Muscle Strength Changes Following Engensis Injections Compared to Placebo - Hand-Held Dynamometry
Elbow Flexion, right
|
-10.68 Pounds (lbs.)
Standard Deviation 11.813
|
-1.68 Pounds (lbs.)
Standard Deviation 3.174
|
|
Evaluation of Muscle Strength Changes Following Engensis Injections Compared to Placebo - Hand-Held Dynamometry
Elbow Extension, right
|
-6.83 Pounds (lbs.)
Standard Deviation 8.925
|
-1.08 Pounds (lbs.)
Standard Deviation 5.171
|
|
Evaluation of Muscle Strength Changes Following Engensis Injections Compared to Placebo - Hand-Held Dynamometry
Wrist Extension, left
|
-7.162 Pounds (lbs.)
Standard Deviation 7.8940
|
4.943 Pounds (lbs.)
Standard Deviation 8.5556
|
|
Evaluation of Muscle Strength Changes Following Engensis Injections Compared to Placebo - Hand-Held Dynamometry
Wrist Extension, right
|
-6.53 Pounds (lbs.)
Standard Deviation 10.081
|
-2.18 Pounds (lbs.)
Standard Deviation 4.350
|
|
Evaluation of Muscle Strength Changes Following Engensis Injections Compared to Placebo - Hand-Held Dynamometry
First Dorsal Interosseous, left
|
-0.712 Pounds (lbs.)
Standard Deviation 1.2620
|
-0.150 Pounds (lbs.)
Standard Deviation 0.3000
|
|
Evaluation of Muscle Strength Changes Following Engensis Injections Compared to Placebo - Hand-Held Dynamometry
First Dorsal Interosseous, right
|
-0.808 Pounds (lbs.)
Standard Deviation 1.0350
|
-0.050 Pounds (lbs.)
Standard Deviation 0.1000
|
|
Evaluation of Muscle Strength Changes Following Engensis Injections Compared to Placebo - Hand-Held Dynamometry
Abductor Pollicus Brevis, left
|
-2.188 Pounds (lbs.)
Standard Deviation 2.7008
|
0.200 Pounds (lbs.)
Standard Deviation 0.4000
|
|
Evaluation of Muscle Strength Changes Following Engensis Injections Compared to Placebo - Hand-Held Dynamometry
Abductor Digiti Minimi, right
|
-1.028 Pounds (lbs.)
Standard Deviation 1.1762
|
0.00 Pounds (lbs.)
Standard Deviation 0.0000
|
|
Evaluation of Muscle Strength Changes Following Engensis Injections Compared to Placebo - Hand-Held Dynamometry
Hip Flexion, left
|
-14.82 Pounds (lbs.)
Standard Deviation 23.235
|
0.15 Pounds (lbs.)
Standard Deviation 23.742
|
|
Evaluation of Muscle Strength Changes Following Engensis Injections Compared to Placebo - Hand-Held Dynamometry
Hip Flexion, right
|
-13.12 Pounds (lbs.)
Standard Deviation 18.239
|
-1.88 Pounds (lbs.)
Standard Deviation 20.747
|
|
Evaluation of Muscle Strength Changes Following Engensis Injections Compared to Placebo - Hand-Held Dynamometry
Knee Flexion, left
|
-14.018 Pounds (lbs.)
Standard Deviation 19.2911
|
-1.575 Pounds (lbs.)
Standard Deviation 6.2809
|
|
Evaluation of Muscle Strength Changes Following Engensis Injections Compared to Placebo - Hand-Held Dynamometry
Knee Flexion, right
|
-12.64 Pounds (lbs.)
Standard Deviation 11.764
|
-1.73 Pounds (lbs.)
Standard Deviation 5.904
|
|
Evaluation of Muscle Strength Changes Following Engensis Injections Compared to Placebo - Hand-Held Dynamometry
Knee Extension, right
|
-13.022 Pounds (lbs.)
Standard Deviation 6.5484
|
0.125 Pounds (lbs.)
Standard Deviation 5.9690
|
|
Evaluation of Muscle Strength Changes Following Engensis Injections Compared to Placebo - Hand-Held Dynamometry
Ankle Dorsiflexion, left
|
-13.28 Pounds (lbs.)
Standard Deviation 18.262
|
-3.80 Pounds (lbs.)
Standard Deviation 3.586
|
|
Evaluation of Muscle Strength Changes Following Engensis Injections Compared to Placebo - Hand-Held Dynamometry
Ankle Dorsiflexion, right
|
-14.30 Pounds (lbs.)
Standard Deviation 15.165
|
-4.03 Pounds (lbs.)
Standard Deviation 4.800
|
OTHER_PRE_SPECIFIED outcome
Timeframe: Day 0 to Day 180Population: The Accurate Test of Limb Isometric Strength was not performed on any subject due to sites not being trained or were unable to conduct the procedure.
As assessed bilaterally by the Accurate Test of Limb Isometric Strength where available. Accurate Test of Limb Isometric Strength - Muscle strength values for each muscle group and change from Baseline were presented, neither descriptive statistics presented by visit and treatment group.
Outcome measures
Outcome data not reported
OTHER_PRE_SPECIFIED outcome
Timeframe: Day 0 to Day 84, and Day 0 to 180Population: Amyotrophic Lateral Sclerosis Assessment Questionnaire. Percent Change from Baseline (ITT Population) for Engensis and Placebo.
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. The Total ALSAQ-40 scores are per Category as percentages of 100% maximum. Each Category percentage is summed for the Intent-to-treat Population. The Change from Baseline is displayed in the Table. ALSAQ-40 was completed at the pre-dose visit (Day 0), and at the Day 84 and Day 180 visits. The Total Change From Baseline is calculated for the Day 84 visit, and the Day 180 visit.
Outcome measures
| Measure |
Engensis
n=12 Participants
64 mg Engensis per Treatment Cycle, with each of 3 cycles composed of 2 days of 128 injections each to the right and left target muscles, spaced 2 weeks apart
Engensis: Lyophilized biologic to be reconstituted containing Engensis
|
Placebo
n=6 Participants
32 mL of Placebo per Treatment Cycle, with each of 3 cycles composed of 2 days of 128 injections each to the right and left target muscles, spaced 2 weeks apart
Placebo: Injectable liquid
|
|---|---|---|
|
Evaluation of Quality of Life Improvement Following Engensis Injections Compared to Placebo - Exploratory Outcome
Day 84 - Physical Mobility
|
1.88 percentage of Change from Baseline
Standard Deviation 20.255
|
-1.5 percentage of Change from Baseline
Standard Deviation 5.755
|
|
Evaluation of Quality of Life Improvement Following Engensis Injections Compared to Placebo - Exploratory Outcome
Day 180 - Physical Mobility
|
13.44 percentage of Change from Baseline
Standard Deviation 24.014
|
11.88 percentage of Change from Baseline
Standard Deviation 17.366
|
|
Evaluation of Quality of Life Improvement Following Engensis Injections Compared to Placebo - Exploratory Outcome
Day 84 - Activities of Daily Living/Independence
|
10.31 percentage of Change from Baseline
Standard Deviation 9.396
|
0.50 percentage of Change from Baseline
Standard Deviation 8.178
|
|
Evaluation of Quality of Life Improvement Following Engensis Injections Compared to Placebo - Exploratory Outcome
Day 180 - Activities of Daily Living/Independence
|
15.94 percentage of Change from Baseline
Standard Deviation 17.369
|
11.25 percentage of Change from Baseline
Standard Deviation 12.332
|
|
Evaluation of Quality of Life Improvement Following Engensis Injections Compared to Placebo - Exploratory Outcome
Day 84 - Eating and Drinking
|
8.34 percentage of Change from Baseline
Standard Deviation 12.596
|
11.66 percentage of Change from Baseline
Standard Deviation 21.732
|
|
Evaluation of Quality of Life Improvement Following Engensis Injections Compared to Placebo - Exploratory Outcome
Day 180 - Eating and Drinking
|
17.73 percentage of Change from Baseline
Standard Deviation 18.623
|
14.58 percentage of Change from Baseline
Standard Deviation 21.886
|
|
Evaluation of Quality of Life Improvement Following Engensis Injections Compared to Placebo - Exploratory Outcome
Day 84 - Communication
|
11.15 percentage of Change from Baseline
Standard Deviation 24.334
|
7.14 percentage of Change from Baseline
Standard Deviation 13.363
|
|
Evaluation of Quality of Life Improvement Following Engensis Injections Compared to Placebo - Exploratory Outcome
Day 180 - Communication
|
24.56 percentage of Change from Baseline
Standard Deviation 27.690
|
11.60 percentage of Change from Baseline
Standard Deviation 25.977
|
|
Evaluation of Quality of Life Improvement Following Engensis Injections Compared to Placebo - Exploratory Outcome
Day 84 - Emotional Functioning
|
7.81 percentage of Change from Baseline
Standard Deviation 24.401
|
6.00 percentage of Change from Baseline
Standard Deviation 6.755
|
|
Evaluation of Quality of Life Improvement Following Engensis Injections Compared to Placebo - Exploratory Outcome
Day 180 - Emotional Functioning
|
20.00 percentage of Change from Baseline
Standard Deviation 21.423
|
11.25 percentage of Change from Baseline
Standard Deviation 11.087
|
OTHER_PRE_SPECIFIED outcome
Timeframe: Day 84 and Day 180 (End of Study or Early Termination)Population: Patient Global Impression of Change - ITT Population
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 difference, 5=Moderately better, and a slight but noticeable change, 6=Better, and a definite improvement that has made a real and worthwhile difference, and 7 = A great deal better, and a considerable improvement that has made all the difference. The test was self-administered on Days 84 and 180.
Outcome measures
| Measure |
Engensis
n=12 Participants
64 mg Engensis per Treatment Cycle, with each of 3 cycles composed of 2 days of 128 injections each to the right and left target muscles, spaced 2 weeks apart
Engensis: Lyophilized biologic to be reconstituted containing Engensis
|
Placebo
n=6 Participants
32 mL of Placebo per Treatment Cycle, with each of 3 cycles composed of 2 days of 128 injections each to the right and left target muscles, spaced 2 weeks apart
Placebo: Injectable liquid
|
|---|---|---|
|
Evaluation of Patient Reported Outcome Improvement Following Engensis Injections Compared to Placebo
Day 84
|
3.1 score on a scale
Standard Deviation 1.81
|
4.6 score on a scale
Standard Deviation 0.89
|
|
Evaluation of Patient Reported Outcome Improvement Following Engensis Injections Compared to Placebo
End of Study / Early Term- Day 180
|
2.9 score on a scale
Standard Deviation 1.46
|
4.0 score on a scale
Standard Deviation 2.00
|
OTHER_PRE_SPECIFIED outcome
Timeframe: Day 84 to Day 180Population: Clinical Global Impression of Change - ITT Population
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.
Outcome measures
| Measure |
Engensis
n=12 Participants
64 mg Engensis per Treatment Cycle, with each of 3 cycles composed of 2 days of 128 injections each to the right and left target muscles, spaced 2 weeks apart
Engensis: Lyophilized biologic to be reconstituted containing Engensis
|
Placebo
n=6 Participants
32 mL of Placebo per Treatment Cycle, with each of 3 cycles composed of 2 days of 128 injections each to the right and left target muscles, spaced 2 weeks apart
Placebo: Injectable liquid
|
|---|---|---|
|
Evaluation of Clinical Reported Outcome Improvement Following Engensis Injections Compared to Placebo
Day 84 - Minimally improved
|
2 Participants
|
3 Participants
|
|
Evaluation of Clinical Reported Outcome Improvement Following Engensis Injections Compared to Placebo
Day 84 - Minimally worse
|
5 Participants
|
1 Participants
|
|
Evaluation of Clinical Reported Outcome Improvement Following Engensis Injections Compared to Placebo
Day 84 - No change
|
3 Participants
|
2 Participants
|
|
Evaluation of Clinical Reported Outcome Improvement Following Engensis Injections Compared to Placebo
End Study/Early term- Minimally improved
|
0 Participants
|
1 Participants
|
|
Evaluation of Clinical Reported Outcome Improvement Following Engensis Injections Compared to Placebo
End Study/Early term- Minimally worse
|
4 Participants
|
0 Participants
|
|
Evaluation of Clinical Reported Outcome Improvement Following Engensis Injections Compared to Placebo
End Study/Early term- Much worse
|
2 Participants
|
1 Participants
|
|
Evaluation of Clinical Reported Outcome Improvement Following Engensis Injections Compared to Placebo
End Study/Early term- No change
|
4 Participants
|
2 Participants
|
OTHER_PRE_SPECIFIED outcome
Timeframe: Day 0 to Day 180Population: Slow Vital Capacity Percent Change from Baseline by Visit (ITT Population)
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.
Outcome measures
| Measure |
Engensis
n=12 Participants
64 mg Engensis per Treatment Cycle, with each of 3 cycles composed of 2 days of 128 injections each to the right and left target muscles, spaced 2 weeks apart
Engensis: Lyophilized biologic to be reconstituted containing Engensis
|
Placebo
n=6 Participants
32 mL of Placebo per Treatment Cycle, with each of 3 cycles composed of 2 days of 128 injections each to the right and left target muscles, spaced 2 weeks apart
Placebo: Injectable liquid
|
|---|---|---|
|
To Determine Effects of Engensis on Respiratory Function Compared to Placebo - Slow Vital Capacity
Day 30
|
-3.31 percentage of Change from Baseline
Standard Deviation 4.191
|
2.53 percentage of Change from Baseline
Standard Deviation 7.501
|
|
To Determine Effects of Engensis on Respiratory Function Compared to Placebo - Slow Vital Capacity
Day 60 (pre-dose)
|
-6.50 percentage of Change from Baseline
Standard Deviation 9.439
|
-3.90 percentage of Change from Baseline
Standard Deviation 9.173
|
|
To Determine Effects of Engensis on Respiratory Function Compared to Placebo - Slow Vital Capacity
Day 84
|
-6.50 percentage of Change from Baseline
Standard Deviation 11.808
|
-2.3 percentage of Change from Baseline
Standard Deviation 9.182
|
|
To Determine Effects of Engensis on Respiratory Function Compared to Placebo - Slow Vital Capacity
Day 120 (pre-dose)
|
-12.76 percentage of Change from Baseline
Standard Deviation 13.363
|
-8.84 percentage of Change from Baseline
Standard Deviation 12.916
|
|
To Determine Effects of Engensis on Respiratory Function Compared to Placebo - Slow Vital Capacity
Day 144
|
-13.46 percentage of Change from Baseline
Standard Deviation 17.605
|
-9.04 percentage of Change from Baseline
Standard Deviation 15.925
|
|
To Determine Effects of Engensis on Respiratory Function Compared to Placebo - Slow Vital Capacity
Day 180
|
-17.80 percentage of Change from Baseline
Standard Deviation 17.308
|
-1.75 percentage of Change from Baseline
Standard Deviation 10.046
|
OTHER_PRE_SPECIFIED outcome
Timeframe: Day 0 to Day 180Population: Participants that Experienced Tracheostomy during the study - ITT Population
Total Participants that experienced Tracheostomy during the study in the Intent-to-treat population.
Outcome measures
| Measure |
Engensis
n=12 Participants
64 mg Engensis per Treatment Cycle, with each of 3 cycles composed of 2 days of 128 injections each to the right and left target muscles, spaced 2 weeks apart
Engensis: Lyophilized biologic to be reconstituted containing Engensis
|
Placebo
n=6 Participants
32 mL of Placebo per Treatment Cycle, with each of 3 cycles composed of 2 days of 128 injections each to the right and left target muscles, spaced 2 weeks apart
Placebo: Injectable liquid
|
|---|---|---|
|
To Determine Effects of Engensis on Respiratory Function Compared to Placebo - Tracheostomy
|
0 Participants
|
0 Participants
|
OTHER_PRE_SPECIFIED outcome
Timeframe: Day 0 to Day 180Population: Time to All-Cause Death - ITT Population
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.
Outcome measures
| Measure |
Engensis
n=12 Participants
64 mg Engensis per Treatment Cycle, with each of 3 cycles composed of 2 days of 128 injections each to the right and left target muscles, spaced 2 weeks apart
Engensis: Lyophilized biologic to be reconstituted containing Engensis
|
Placebo
n=6 Participants
32 mL of Placebo per Treatment Cycle, with each of 3 cycles composed of 2 days of 128 injections each to the right and left target muscles, spaced 2 weeks apart
Placebo: Injectable liquid
|
|---|---|---|
|
To Determine Effects of Engensis on Survival Compared to Placebo - Deaths
|
0 Participants
|
0 Participants
|
Adverse Events
Engensis
Serious events: 1 serious events
Other events: 10 other events
Deaths: 0 deaths
Placebo
Serious events: 0 serious events
Other events: 5 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
Engensis
n=12 participants at risk
64 mg Engensis per Treatment Cycle, with each of 3 cycles composed of 2 days of 128 injections each to the right and left target muscles, spaced 2 weeks apart
Engensis: Lyophilized biologic to be reconstituted containing Engensis
|
Placebo
n=6 participants at risk
32 mL of Placebo per Treatment Cycle, with each of 3 cycles composed of 2 days of 128 injections each to the right and left target muscles, spaced 2 weeks apart
Placebo: Injectable liquid
|
|---|---|---|
|
Infections and infestations
Bronchitis
|
8.3%
1/12 • Number of events 1 • From Day 0 to Day 180 or Early Termination
Treatment Emergent Adverse Events by System Organ Class and Preferred Term Safety Analysis Population
|
0.00%
0/6 • From Day 0 to Day 180 or Early Termination
Treatment Emergent Adverse Events by System Organ Class and Preferred Term Safety Analysis Population
|
Other adverse events
| Measure |
Engensis
n=12 participants at risk
64 mg Engensis per Treatment Cycle, with each of 3 cycles composed of 2 days of 128 injections each to the right and left target muscles, spaced 2 weeks apart
Engensis: Lyophilized biologic to be reconstituted containing Engensis
|
Placebo
n=6 participants at risk
32 mL of Placebo per Treatment Cycle, with each of 3 cycles composed of 2 days of 128 injections each to the right and left target muscles, spaced 2 weeks apart
Placebo: Injectable liquid
|
|---|---|---|
|
General disorders
Injection site bruising
|
33.3%
4/12 • Number of events 7 • From Day 0 to Day 180 or Early Termination
Treatment Emergent Adverse Events by System Organ Class and Preferred Term Safety Analysis Population
|
50.0%
3/6 • Number of events 9 • From Day 0 to Day 180 or Early Termination
Treatment Emergent Adverse Events by System Organ Class and Preferred Term Safety Analysis Population
|
|
General disorders
Pyrexia
|
25.0%
3/12 • Number of events 3 • From Day 0 to Day 180 or Early Termination
Treatment Emergent Adverse Events by System Organ Class and Preferred Term Safety Analysis Population
|
16.7%
1/6 • Number of events 1 • From Day 0 to Day 180 or Early Termination
Treatment Emergent Adverse Events by System Organ Class and Preferred Term Safety Analysis Population
|
|
General disorders
Injection site pain
|
16.7%
2/12 • Number of events 13 • From Day 0 to Day 180 or Early Termination
Treatment Emergent Adverse Events by System Organ Class and Preferred Term Safety Analysis Population
|
16.7%
1/6 • Number of events 1 • From Day 0 to Day 180 or Early Termination
Treatment Emergent Adverse Events by System Organ Class and Preferred Term Safety Analysis Population
|
|
General disorders
Asthenia
|
0.00%
0/12 • From Day 0 to Day 180 or Early Termination
Treatment Emergent Adverse Events by System Organ Class and Preferred Term Safety Analysis Population
|
33.3%
2/6 • Number of events 4 • From Day 0 to Day 180 or Early Termination
Treatment Emergent Adverse Events by System Organ Class and Preferred Term Safety Analysis Population
|
|
General disorders
Fatigue
|
8.3%
1/12 • Number of events 1 • From Day 0 to Day 180 or Early Termination
Treatment Emergent Adverse Events by System Organ Class and Preferred Term Safety Analysis Population
|
16.7%
1/6 • Number of events 2 • From Day 0 to Day 180 or Early Termination
Treatment Emergent Adverse Events by System Organ Class and Preferred Term Safety Analysis Population
|
|
Musculoskeletal and connective tissue disorders
Arthralgia
|
16.7%
2/12 • Number of events 3 • From Day 0 to Day 180 or Early Termination
Treatment Emergent Adverse Events by System Organ Class and Preferred Term Safety Analysis Population
|
0.00%
0/6 • From Day 0 to Day 180 or Early Termination
Treatment Emergent Adverse Events by System Organ Class and Preferred Term Safety Analysis Population
|
|
Musculoskeletal and connective tissue disorders
Muscle spasms
|
8.3%
1/12 • Number of events 2 • From Day 0 to Day 180 or Early Termination
Treatment Emergent Adverse Events by System Organ Class and Preferred Term Safety Analysis Population
|
16.7%
1/6 • Number of events 1 • From Day 0 to Day 180 or Early Termination
Treatment Emergent Adverse Events by System Organ Class and Preferred Term Safety Analysis Population
|
|
Musculoskeletal and connective tissue disorders
Musculoskeletal stiffness
|
16.7%
2/12 • Number of events 3 • From Day 0 to Day 180 or Early Termination
Treatment Emergent Adverse Events by System Organ Class and Preferred Term Safety Analysis Population
|
0.00%
0/6 • From Day 0 to Day 180 or Early Termination
Treatment Emergent Adverse Events by System Organ Class and Preferred Term Safety Analysis Population
|
|
Musculoskeletal and connective tissue disorders
Back pain
|
16.7%
2/12 • Number of events 2 • From Day 0 to Day 180 or Early Termination
Treatment Emergent Adverse Events by System Organ Class and Preferred Term Safety Analysis Population
|
0.00%
0/6 • From Day 0 to Day 180 or Early Termination
Treatment Emergent Adverse Events by System Organ Class and Preferred Term Safety Analysis Population
|
|
Musculoskeletal and connective tissue disorders
Joint range of motion decreased
|
8.3%
1/12 • Number of events 1 • From Day 0 to Day 180 or Early Termination
Treatment Emergent Adverse Events by System Organ Class and Preferred Term Safety Analysis Population
|
16.7%
1/6 • Number of events 1 • From Day 0 to Day 180 or Early Termination
Treatment Emergent Adverse Events by System Organ Class and Preferred Term Safety Analysis Population
|
|
Musculoskeletal and connective tissue disorders
Myalgia
|
0.00%
0/12 • From Day 0 to Day 180 or Early Termination
Treatment Emergent Adverse Events by System Organ Class and Preferred Term Safety Analysis Population
|
33.3%
2/6 • Number of events 2 • From Day 0 to Day 180 or Early Termination
Treatment Emergent Adverse Events by System Organ Class and Preferred Term Safety Analysis Population
|
|
Nervous system disorders
Muscle contractions involuntary
|
16.7%
2/12 • Number of events 3 • From Day 0 to Day 180 or Early Termination
Treatment Emergent Adverse Events by System Organ Class and Preferred Term Safety Analysis Population
|
16.7%
1/6 • Number of events 1 • From Day 0 to Day 180 or Early Termination
Treatment Emergent Adverse Events by System Organ Class and Preferred Term Safety Analysis Population
|
|
Nervous system disorders
Dysarthria
|
8.3%
1/12 • Number of events 2 • From Day 0 to Day 180 or Early Termination
Treatment Emergent Adverse Events by System Organ Class and Preferred Term Safety Analysis Population
|
16.7%
1/6 • Number of events 1 • From Day 0 to Day 180 or Early Termination
Treatment Emergent Adverse Events by System Organ Class and Preferred Term Safety Analysis Population
|
|
Nervous system disorders
Headache
|
8.3%
1/12 • Number of events 2 • From Day 0 to Day 180 or Early Termination
Treatment Emergent Adverse Events by System Organ Class and Preferred Term Safety Analysis Population
|
16.7%
1/6 • Number of events 1 • From Day 0 to Day 180 or Early Termination
Treatment Emergent Adverse Events by System Organ Class and Preferred Term Safety Analysis Population
|
|
Respiratory, thoracic and mediastinal disorders
Cough
|
25.0%
3/12 • Number of events 4 • From Day 0 to Day 180 or Early Termination
Treatment Emergent Adverse Events by System Organ Class and Preferred Term Safety Analysis Population
|
16.7%
1/6 • Number of events 1 • From Day 0 to Day 180 or Early Termination
Treatment Emergent Adverse Events by System Organ Class and Preferred Term Safety Analysis Population
|
|
Respiratory, thoracic and mediastinal disorders
Rhinorrhea
|
16.7%
2/12 • Number of events 2 • From Day 0 to Day 180 or Early Termination
Treatment Emergent Adverse Events by System Organ Class and Preferred Term Safety Analysis Population
|
16.7%
1/6 • Number of events 1 • From Day 0 to Day 180 or Early Termination
Treatment Emergent Adverse Events by System Organ Class and Preferred Term Safety Analysis Population
|
|
Gastrointestinal disorders
Diarrhoea
|
16.7%
2/12 • Number of events 2 • From Day 0 to Day 180 or Early Termination
Treatment Emergent Adverse Events by System Organ Class and Preferred Term Safety Analysis Population
|
0.00%
0/6 • From Day 0 to Day 180 or Early Termination
Treatment Emergent Adverse Events by System Organ Class and Preferred Term Safety Analysis Population
|
|
Gastrointestinal disorders
Nausea
|
8.3%
1/12 • Number of events 1 • From Day 0 to Day 180 or Early Termination
Treatment Emergent Adverse Events by System Organ Class and Preferred Term Safety Analysis Population
|
16.7%
1/6 • Number of events 1 • From Day 0 to Day 180 or Early Termination
Treatment Emergent Adverse Events by System Organ Class and Preferred Term Safety Analysis Population
|
|
Infections and infestations
COVID-19
|
41.7%
5/12 • Number of events 5 • From Day 0 to Day 180 or Early Termination
Treatment Emergent Adverse Events by System Organ Class and Preferred Term Safety Analysis Population
|
16.7%
1/6 • Number of events 1 • From Day 0 to Day 180 or Early Termination
Treatment Emergent Adverse Events by System Organ Class and Preferred Term Safety Analysis Population
|
|
Psychiatric disorders
Insomnia
|
8.3%
1/12 • Number of events 1 • From Day 0 to Day 180 or Early Termination
Treatment Emergent Adverse Events by System Organ Class and Preferred Term Safety Analysis Population
|
16.7%
1/6 • Number of events 1 • From Day 0 to Day 180 or Early Termination
Treatment Emergent Adverse Events by System Organ Class and Preferred Term Safety Analysis Population
|
|
Vascular disorders
Hypertension
|
8.3%
1/12 • Number of events 1 • From Day 0 to Day 180 or Early Termination
Treatment Emergent Adverse Events by System Organ Class and Preferred Term Safety Analysis Population
|
16.7%
1/6 • Number of events 1 • From Day 0 to Day 180 or Early Termination
Treatment Emergent Adverse Events by System Organ Class and Preferred Term Safety Analysis Population
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place