Evaluation the Efficacy and Safety of Mutiple Lenzumestrocel (Neuronata-R® Inj.) Treatment in Patients With ALS

A regiszterben nem jelölt feltételek

• \[Inclusion Criteria\]
• Subjects who show both upper motor neuron signs and lower motor neuron signs at the same time in neurological tests.
• 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).
• For subjects who are under Riluzole treatment, those who have received stable dose of Riluzole for more than 28 days before screening visit.
• Subjects with duration of disease of no more than 2 years from the first diagnosis date.
• Subjects whose ALSFRS-R scores are in the range of 31\~46 at the time of screening (P-V0).
  • \[Exclusion Criteria\]
• 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).
• Subjects who received gastrotomy at the time of screening (P-V0).
• 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.
• 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.
• 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).
• Subjects who have experienced epileptic seizure.
• Subjects with severe renal disorder (serum creatinine: not less than 2.0 mg/dL).
• Subjects with severe hepatic disorder (ALT, AST, or bilirubin: over 2.0 times of the normal upper limit).
• Subjects who show hemorrhagic tendency at the time of screening (PT and aPTT \> 1.5 x ULN)
• 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.
• Subjects with hypersensitivity to antibiotics (penicillin or streptomycin).
• Subjects who have ever received any cell therapy product for the same disease.
• Subjects with any malignant tumor in the past 5 years before screening, except malignant tumors with very low risk of metastasis or death.