Amyotrophic lateral sclerosis (ALS)
Conditions
Sponsors
Tomii Ryo
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: Japanese ALS patients fulfilling the following inclusion criteria and not meeting any of the exclusion criteria (1) Patients with "definite" or "probable" ALS according to the Revised El Escorial criteria (Airlie House 1998) (2) Patients with ALS severity grade 1 or 2 according to the Japan ALS severity classification. (3) Patients with the ALSFRS-R scores of 2 or more for all items, with scores of 4 for all breathing-related items (i.e., dyspnea, orthopnea, respiratory insufficiency). (4) Patients with %FVC of 80% or more. (5) Patients within 2 years of ALS onset. (6) Patients expected to be able to complete the study. (7) Patients aged 18 to 75 years at the time of informed consent. (8) Patients with written consent provided in person or by the legally acceptable representative or proxy signer (The study contents shall be explained to the patient and informed consent shall be obtained from the patient in person. If the patient is not capable of giving adequate consent, the consent form shall be signed by the patient's legally acceptable representative. If the patient is not capable of writing, the patient's willingness shall be confirmed verbally, and the consent form shall be signed by the patient's proxy signer.)
Exclusion criteria
Exclusion criteria: (1) Patients with uncontrollable psychiatric symptoms. (2) Patients with dementia. (3) Patients with infection with HBV*, HCV, HIV, HTLV-1, HPVB19, or syphilis. *If only HBs antibody is positive due to HBV vaccination, the patient will be allowed to participate in this study. (4) Patients with infection with Mycoplasma, CMV, and EBV, and are currently symptomatic. (5) Patients with a history of COVID-19 within the past 3 months or current sequelae from COVID-19. (6) Patients with decreased peripheral blood cells (white blood cell count <2,000/uL, hemoglobin <10.0 g/dL, platelet count <100,000/uL). (7) Patients with current or past history of any of the following: Malignant neoplasms (except those considered cured with sufficient time elapsed from the date of last diagnosis), severe blood or hematopoietic organ diseases or immune system disorders, severe psychiatric or behavioral disorders, severe nervous system diseases, severe congenital malformations or deformities, or chromosomal abnormalities (8) Patients with a history of allergy to penicillin or streptomycin or other serious allergies (e.g., shock, anaphylactoid symptoms). (9) Patients with poor general condition, for example due to the following diseases: Example: endocrine and metabolic diseases, psychiatric disorders, neurological diseases, cardiovascular diseases (e.g., uncontrolled cardiac failure, moderate or severe valvular disease, uncontrolled atrial fibrillation affecting circulation, atrial or ventricular thrombus unamenable to pharmacotherapy, history of myocardial infarction or angina pectoris treated with percutaneous coronary intervention performed within 12 months, other severe arrhythmias), respiratory diseases, gastrointestinal diseases, musculoskeletal or connective tissue diseases, renal or urogenital diseases. (e.g., maintenance dialysis), trauma/poisoning or effects of other external factors (10) Patients with a more than 70% stenosis of coronary artery or a dissecting vessel regardless of the degree of stenosis. (11) Patients with current or past history of intracranial lesions (e.g., severe asymptomatic lesions, severe old lesions, severe white matter lesions, severe multiple lesions, severe microhemorrhage or hemosiderin deposits, vascular lesions such as moyamoya disease or cerebral aneurysms, severe intracranial bleeding). (12) Patients with a more than 70% stenosis of a blood vessel in the head or neck region or a dissecting vessel regardless of the degree of stenosis (except for complete occlusion). The condition does not change even after revascularization. (13) Patients with strong atherosclerotic change or calcification of blood vessels in the head or neck region and coronary artery. (14) Artificial devices (e.g., pacemakers, artificial valves) are inserted into the cardiovascular system. (15) Patients not anticipated to have controlled blood pressure (systolic less than140 mmHg, diastolic less than 90 mmHg) even with treatment just before administration of the investigational product. (16) Patients currently participating in any other clinical trial. (17) Patients who are receiving or who received cell therapy. (18) Women who are pregnant, breastfeeding, or who may be pregnant or plan to become pregnant during study participation, or men who wish their partner to become pregnant during study participation. (19) Patients who started, increased or decreased the dose of ALS medication (e.g., edaravone, riluzole) within 1 month (20)
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Change in the ALSFRS-R score from 85 days after enrollment to 253 days after enrollment in Primary Analysis Population | — |
Secondary
| Measure | Time frame |
|---|---|
| [Safety] Aggregation of adverse events and defects [Efficacy] Change in ALSFRS-R score Change in %FVC Change in MMT Change in ALSAQ-40 Change in Kings staging Change in MiToS system Percentage of dropouts due to death, ventilator placement, or tracheostomy after enrollment and the number of days to dropout | — |
Contacts
Public ContactRyo Tomii
Nipro Corporation
Outcome results
None listed