Clinical trial: Safety of Engensis in Participants With Amyotrophic Lateral Sclerosis (NCT04632225)

Eligibility 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
No condition on gender to be admitted to the trial.
No healthy subjects accepted to join the trial.
Subject must be at least 18 Years old.
Subject must be at most 80 Years

Patient description (medical language): A 47-year-old man comes to the clinic for the follow up of his neuromuscular disease. He experienced gradual, progressive weakness of the left upper extremity over the last year.  Over the last few months, he has also noticed weakness in the right upper extremity.  BP is 120/75, PR is 80 and temperature is 37 C. Reflexes are brisk in the upper extremities, and the plantar responses are extensor.  Mild gait ataxia is present. The patient is under treatment of Riluzole 50 mg BID with the diagnosis of ALS.

Patient description (patient's own words): I've been suffering from a neuromuscular disease for a while now, and I went to my doctor's office. I'm now a 47-year-old man and over the past year I experienced a progressive and gradual weakness of my left upper extremity, and over the past month, I also noticed a weakness over my right upper extremity. My heart rate was 120/75, and my  PR was 80 with 37°C for temperature. My reflexes are not good in my upper extremities, and I have trouble with my balance. I'm also under Exservan 50 mg for my sclerosis.

Does the patient match the trial criteria? Entailment