Clinical trial: Evaluatiton the Efficacy and Safety of Mutiple Lenzumestrocel (Neuronata-R® Inj.) Treatment in Patients With ALS (NCT04745299)

Eligibility criteria:
[Inclusion Criteria]

          1. Subjects who show both upper motor neuron signs and lower motor neuron signs at the
             same time in neurological tests.

          2. Among subjects diagnosed with familial or sporadic ALS, subjects falling into
             clinically definite ALS, probable ALS and probable ALS-lab supported according to The
             revised World Federation of Neurology El Escorial Criteria[Rix Brooks, 2000], during
             17-weeks period prior to the administration and ALSFRS-R score (progression rate) of
             1.03 ± 50%/month (meaning mean value in that total period).

          3. For subjects who are under Riluzole treatment, those who have received stable dose of
             Riluzole for more than 28 days before screening visit.

          4. Subjects with duration of disease of no more than 2 years from the first diagnosis
             date.

          5. Subjects whose ALSFRS-R scores are in the range of 31~46 at the time of screening
             (P-V0).

        [Exclusion Criteria]

          1. Subjects who received tracheostomy or use ventilators (including positive pressure
             ventilators; subjects who use non-invasive ventilation for sleep apnea may be allowed
             after review) at the time of screening (P-V0).

          2. Subjects who received gastrotomy at the time of screening (P-V0).

          3. Subjects for whom clinical efficacy evaluation is not possible because pulmonary
             functional tests cannot be conducted at the time of screening (P-V0) or subjects whose
             forced vital capacity is found to be not greater than 40%of the expected value.

          4. Subjects who fall into above Class II according to the New York Heart Association's
             functional classification, who have showed myocardial infarction, unstable arrhythmia
             and/or other significant cardiovascular diseases such as unstable angina in the past 3
             months, or who show electrocardiographic signs of myocardial infarction or angina at
             the time of screening (P-V0) or who received stent insertion or coronary artery bypass
             grafting.

          5. Subjects who have received other investigational products or edaravone within 3 month
             or 5 half-lives at the time of screening (P-V0) and lead in period visit L-V1
             (evaluated by whichever is longer).

          6. Subjects who have experienced epileptic seizure.

          7. Subjects with severe renal disorder (serum creatinine: not less than 2.0 mg/dL).

          8. Subjects with severe hepatic disorder (ALT, AST, or bilirubin: over 2.0 times of the
             normal upper limit).

          9. Subjects who show hemorrhagic tendency at the time of screening (PT and aPTT > 1.5 x
             ULN)

         10. Subjects who are found to have active viral infections (HBsAg, HCV Ab, HIV Ab, CMV
             IgM, EBV IgM, HSV IgM and Treponema pallidum) at the time of screening.

         11. Subjects with hypersensitivity to antibiotics (penicillin or streptomycin).

         12. Subjects who have ever received any cell therapy product for the same disease.

         13. Subjects with any malignant tumor in the past 5 years before screening, except
             malignant tumors with very low risk of metastasis or death.
No condition on gender to be admitted to the trial.
No healthy subjects accepted to join the trial.
Subject must be at least 25 Years old.
Subject must be at most 75 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