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RECRUITINGINTERVENTIONAL

Clinical Study of Induced Pluripotent Stem Cells Derived Motor Neuron Precursor Cell Therapy for Amyotrophic Lateral Sclerosis (ALS)

Clinical Study of Human Induced Pluripotent Stem Cells Derived Motor Neuron Precursor(iPSC-MNP) Cells for the Treatment of Amyotrophic Lateral Sclerosis (ALS)

Important: This information is not medical advice. Talk to your doctor about whether a clinical trial is right for you.

About This Trial

Amyotrophic lateral sclerosis (ALS) is a severe neurodegenerative disease in the human motor system characterized by the selective involvement of spinal cord anterior horn cells, brainstem motor nuclei, and the corticospinal tract. It predominantly presents as concurrent damage to upper and lower motor neurons. Induced pluripotent stem cells (iPSCs) are a type of induced pluripotent stem cell derived from autologous or allogeneic cell sources. They can differentiate into various functional cell types, including specific motor neuron cells. iPSCs are used for stem cell replacement therapy. iPSCs hold significant clinical potential for ALS treatment. The iPSC database with human leukocyte antigen characteristics may represent a promising technology. This technology has the potential to obtain high-quality cell products and reduce the risk of graft rejection. Moreover, human iPSCs have demonstrated a certain degree of efficacy in the transplantation of neural stem/progenitor cells derived from ALS rodent models. The potential mechanisms of iPSC therapy for ALS include: the differentiated motor neuron precursor cells can replace damaged motor neurons, and restore motor conduction function; by secreting neurotrophic factors, they protect neurons; through immune regulation, they inhibit inflammatory reactions, and slow the progression of ALS. Xellsmart Biomedical (Suzhou) Co., Ltd. is developing an injectable solution for ALS treatment using human iPSC-derived motor neuron precursor cells to address the pressing need for ALS therapy.

Who May Be Eligible (Plain English)

Who May Qualify: 1. Patients themselves or their legal guardians must consent to undergo this treatment protocol and sign the willing to sign a consent form Form (ICF). 2. Age between 18 and 60 years, inclusive, with no gender restrictions. 3. Diagnosed with ALS according to the World Federation of Neurology criteria, and the initial diagnosis date is between 6 to 24 months before the screening date. 4. Patients who have received standard treatment in the past with poor efficacy or disease progression. 5. Forced Vital Capacity (FVC) should be ≥50%. 6. During any night of the screening period, the total time with peripheral blood oxygen saturation \<90% should not exceed 2%. 7. Patients should be deemed by the investigator to be in good nutritional status, with a Body Mass Index (BMI) ≥18.5. 8. Male patients and their spouses, as well as women of childbearing age, should agree to implement effective contraceptive measures from the time of signing the ICF until one year after the start of treatment. 9. Patients should be able to cooperate in the collection and preservation of medical history data and the visit process. Who Should NOT Join This Trial: 1. Patients with symptoms of neuromuscular weakness but cannot be conclusively determined to have ALS. 2. Patients diagnosed with severe cognitive impairment, clinical dementia, or major psychiatric disorders, including but not limited to schizophrenia, bipolar disorder, or severe depression, according to the Diagnostic and Statistical Manual of Mental Disorders, Fifth Edition (DSM-V). 3. Patients with any disease that impairs nerve or muscle function, such as peripheral neuropathy or metabolic myopathy. 4. Patients with a history of malignant tumors or a previous diagnosis of malignancy. 5. Within the two weeks preceding the screening period, patients who experienced acute active infections requiring treatment with antibiotics, antiviral drugs, or antifungal medications. ...See full criteria on ClinicalTrials.gov Always talk to your doctor about whether this trial is right for you.

Original Eligibility Criteria

View original clinical language
Inclusion Criteria: 1. Patients themselves or their legal guardians must consent to undergo this treatment protocol and sign the Informed Consent Form (ICF). 2. Age between 18 and 60 years, inclusive, with no gender restrictions. 3. Diagnosed with ALS according to the World Federation of Neurology criteria, and the initial diagnosis date is between 6 to 24 months before the screening date. 4. Patients who have received standard treatment in the past with poor efficacy or disease progression. 5. Forced Vital Capacity (FVC) should be ≥50%. 6. During any night of the screening period, the total time with peripheral blood oxygen saturation \<90% should not exceed 2%. 7. Patients should be deemed by the investigator to be in good nutritional status, with a Body Mass Index (BMI) ≥18.5. 8. Male patients and their spouses, as well as women of childbearing age, should agree to implement effective contraceptive measures from the time of signing the ICF until one year after the start of treatment. 9. Patients should be able to cooperate in the collection and preservation of medical history data and the visit process. Exclusion Criteria: 1. Patients with symptoms of neuromuscular weakness but cannot be conclusively determined to have ALS. 2. Patients diagnosed with severe cognitive impairment, clinical dementia, or major psychiatric disorders, including but not limited to schizophrenia, bipolar disorder, or severe depression, according to the Diagnostic and Statistical Manual of Mental Disorders, Fifth Edition (DSM-V). 3. Patients with any disease that impairs nerve or muscle function, such as peripheral neuropathy or metabolic myopathy. 4. Patients with a history of malignant tumors or a previous diagnosis of malignancy. 5. Within the two weeks preceding the screening period, patients who experienced acute active infections requiring treatment with antibiotics, antiviral drugs, or antifungal medications. 6. ALS patients with concomitant respiratory failure. 7. Patients who have previously undergone any allogeneic cell therapy or organ transplantation. 8. Patients who have Participated in other clinical trials within the three months prior to screening. 9. Patients with a history of tracheostomy or those using mechanical ventilatory support. 10. Patients with a documented history of severe allergic reactions to general anesthesia drugs or previous severe allergic reactions for other reasons. 11. Patients with intracranial organic diseases causing increased intracranial pressure. 12. Patients with elevated liver function test results during the screening period, such as total bilirubin \>1.5 times the upper limit of normal (ULN), or aspartate aminotransferase (AST) or alanine aminotransferase (ALT) \>3 times ULN. 13. Patients who have abnormal kidney function test results during the screening period, such as serum creatinine \>1.5 mg/dL or an estimated creatinine clearance rate \<60 mL/min calculated by the Cockcroft and Gault formula. 14. Other clinically significant laboratory abnormalities during the screening period. 15. Patients with hepatitis A, active hepatitis B (HBsAg positive and HBV DNA ≥500 IU/ml, excluding drug- or other-caused hepatitis), active hepatitis C (anti-HCV antibody positive and HCV RNA positive), hepatitis E, human immunodeficiency virus (HIV) antibody positive, or syphilis treponemal antibody positive. 16. Patients with impaired consciousness. 17. Coagulation abnormalities (prothrombin time \[PT\] or international normalized ratio \[INR\] \>1.5 times ULN; activated partial thromboplastin time \[APTT\] \>1.5 times ULN) or those currently receiving anticoagulation therapy. 18. Poorly controlled hypertension, with systolic blood pressure \>160 mmHg and/or diastolic blood pressure \>100 mmHg after treatment. 19. Severe diabetes with late complications; patients with other diseases affecting limb mobility (e.g., limping, osteoarthritis, rheumatoid arthritis, gout, etc.). 20. Patients who have undergone surgery or experienced trauma (including fractures) in the past month. 21. Pregnant or breastfeeding women. 22. Patients who, in the opinion of the investigator, have poorly controlled systemic diseases or other conditions that make them unsuitable for participation in this clinical study.

Treatments Being Tested

DRUG

iPSC-MNP

Using iPSC-MNP Cells for the Treatment of ALS Patients

Locations (1)

Shang hai East Hospital
Shanghai, Shanghai Municipality, China