The Safety, Tolerability and Preliminary Efficacy of Derived Motor Neuron Progenitor Cells (XS228CN) in Subjects With Amyotrophic Lateral Sclerosis

A Phase I Study to Assess the Safety, Tolerability and Preliminary Efficacy of Human Allogeneic Induced Pluripotent Stem Cells (iPSCs) -Derived Motor Neuron Progenitor Cells (XS228CN) in Subjects With Amyotrophic Lateral Sclerosis

A Phase I Study to Assess the Safety, Tolerability and Preliminary Efficacy of Derived Motor Neuron Progenitor Cells (XS228CN) in Subjects with Amyotrophic Lateral Sclerosis

Inclusion Criteria:

1. 18-75 years of age (inclusive of 18 and 75 years), regardless of gender;
2. Diagnosed of definite or probable ALS according to the revised EI Escorial criteria;
3. Respiratory function FVC at baseline was ≥70% of the predicted value (FVC%);
4. Patients with birth-potential (both male and female) must agree to use effective non-drug contraceptive measures from the time of signing the informed consent until 6 months after the conclusion of the trial;
5. Volunteer to participate in the clinical study, understand and sign the informed consent form.

Exclusion Criteria:

1. Subject has signs and symptoms of neuromuscular weakness, and other causes of muscle weakness cannot be ruled out;
2. Baseline body mass index (BMI) < 18.5 kg/m²;
3. Primary lateral sclerosis presenting only with upper motor neuron symptoms;
4. Significant psychiatric disorders that the investigator assesses may affect evaluation;
5. Diseases causing neurological or muscular dysfunction, such as metabolic muscle diseases or myasthenia gravis;
6. Diagnosed autoimmune diseases with uncontrolled severe arthritis or other conditions (e.g., lameness) that the investigator assesses may affect evaluation;
7. Acute active infections requiring antibiotics, antivirals, or antifungals that occurred within the 2 weeks prior to screening and are not controlled;
8. Subject diagnosed with active pulmonary tuberculosis or treated for suspected tuberculosis;
9. Diagnosed severe pulmonary diseases that the investigator assesses may affect evaluation;
10. Poorly controlled hypertension;
11. Previous or detected cardiac abnormalities;
12. A history of cirrhosis, chronic hepatitis, or liver function at screening;
13. A history of chronic kidney disease;
14. Previous history of bleeding, abnormal clotting, or being treated with anticoagulation;
15. Active hepatitis B, active hepatitis C, human immunodeficiency virus (HIV) antibody or treponema pallidum antibody positive at screening;
16. History of severe trauma or surgery and may affect the assessment judged by investigator;
17. Subjects with contraindications to lumbar puncture, including menifestations of injection site infection or high intracranial pressure.
18. Those who have had a malignant tumor within 5 years prior to screening or are undergoing antitumor therapy;
19. Have participated in other clinical trials within 3 months prior to screening;
20. Pregnant or breastfeeding women;
21. Subject who is judged by the investigator to be unsuitable for the clinical trial.