Safety of Engensis in Participants With Amyotrophic Lateral Sclerosis

• Register: ClinicalTrials.gov
• Last refreshed on: 14 November 2022
• Main ID: NCT04632225
• Date of registration: 29/10/2020
• Prospective Registration: Yes
• Primary sponsor: Helixmith Co., Ltd.
• Public title: Safety of Engensis in Participants With Amyotrophic Lateral Sclerosis REViVALS-1A
• Scientific title: A Phase 2a, Double-Blind, Randomized, Placebo-Controlled, Multicenter Study to Assess the Safety of Engensis in Participants With Amyotrophic Lateral Sclerosis
• Date of first enrolment: February 9, 2021
• Target sample size: 18
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/show/NCT04632225
• Study type: Interventional
• Study design: Allocation: Randomized. Intervention model: Parallel Assignment. Primary purpose: Treatment. Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor).
• Phase: Phase 2

Countries of recruitment

Korea, Republic of, United States

Key inclusion & exclusion criteria

Inclusion Criteria:

1. Clinically definite or probable Amyotrophic Lateral Sclerosis (ALS) or
laboratory-supported probable ALS as defined in the revised El Escorial/Airlie House
diagnostic criteria

2. The site of onset of ALS symptoms is a limb and experiencing symptoms of lower motor
dysfunction (e.g., weakness, atrophy, cramps, poor circulation, etc.) with upper motor
neuron symptoms (e.g., weakness, brisk reflexes, spasticity)

3. Onset of ALS symptoms = 4 years

4. Slow Vital Capacity (SVC) = 50% of predicted value at Screening

5. Not taking riluzole, or on a stable dose (defined as no noted toxicities) for at least
30 days prior to Screening and throughout the study

6. Not taking edaravone or on a maintenance cycle for at least 30 days prior to Screening
and throughout the study

7. For females of childbearing potential, a negative urine pregnancy test at Screening
and on Day 0

8. Male Participants and their female partners must agree to use double-barrier
contraception during the study or provide proof of postmenopausal state (minimum 1
year) or surgical sterility

9. Male Participants must not donate sperm during the study

10. Female Participants must be nonpregnant, nonlactating, and either postmenopausal for
at least 1 year, or surgically sterile for at least 3 months, or agree to use
double-barrier contraception from 28 days prior to randomization (Day 0) and/or their
last confirmed menstrual period prior to study randomization (whichever is longer)
until the end of the study

11. Capable of complying and willing to comply with the requirements and restrictions in
the informed consent form and this protocol

12. Willing to forgo new experimental ALS treatments for at least 6 months following
randomization

Exclusion Criteria:

1. Progressive or degenerative neurological disorder such as Alzheimer's disease,
Parkinson's disease, vascular dementia, multiple sclerosis, and other neurological or
vascular disorders felt by the Investigator to preclude participation

2. Requires tracheotomy ventilation or noninvasive ventilation related to bulbar function

3. Evidence by physical examination, history, or laboratory evaluation of significant
concomitant disease with a life expectancy of < 6 months at Screening

4. INR values >2.0

5. Platelet count <100,000/µL

6. Inflammatory disorder of the blood vessels (inflammatory angiopathy or vasculitis,
such as Buerger's disease)

7. Active infection (chronic infection or severe active infection that may compromise the
Participant's wellbeing or participation in the study in the Investigator's judgment)

8. Chronic inflammatory disease (e.g., Crohn's disease, rheumatoid arthritis)

9. Positive human immunodeficiency virus (HIV) or human T-cell lymphotrophic virus (HTLV)
I/II test at Screening

10. Active acute or chronic hepatitis B

11. Active hepatitis C

12. Immunosuppression due to underlying disease (e.g., rheumatoid arthritis, systemic
lupus erythematosus) or to currently receiving immunosuppressive drugs, (e.g.,
chemotherapy, corticosteroids) or to radiation therapy

13. Stroke or myocardial infarction within 3 months prior to Screening

14. Active deep vein thrombosis

15. Recent history (< 3 years) or presence of cancer except basal cell carcinoma or
squamous cell carcinoma of the skin that was excised and has shown no evidence of
recurrence for at least 1 year

16. Major psychiatric disorder diagnosed in the past 6 months that has not been stabilized
or in the Investigator's opinion would not allow the patient to participate in the
scheduled procedures

17. Use of an investigational drug for the treatment of ALS in the past 30 days or 5
half-lives (if available), whichever is longer, or previous participation in a
clinical study with Engensis

18. Stem cell administration for investigational treatment of ALS or other conditions in
the 6 months prior to Screening



Age minimum: 18 Years
Age maximum: 80 Years
Gender: All

Health Condition(s) or Problem(s) studied

Amyotrophic Lateral Sclerosis

Intervention(s)

Biological: Engensis

Other: Placebo

Primary Outcome(s)

Safety of intramuscular (IM) injections of Engensis in Participants with Amyotrophic Lateral Sclerosis (ALS) compared to Placebo [Time Frame: From the Day 0 Visit to the Day 180 Visit]

Secondary ID(s)

VMALS-002-2

Source(s) of Monetary Support

Please refer to primary and secondary sponsors