Amyotrophic Lateral Sclerosis (ALS)
Conditions
Brief summary
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
Interventions
DRUGHuman Allogeneic Induced Pluripotent Stem Cells (iPSCs) -Derived Motor Neuron Progenitor Cells (XS228CN)
Injection, once, 6 months
Sponsors
XellSmart Bio-Pharmaceutical (Suzhou) Co., Ltd.
Study design
Allocation
NON_RANDOMIZED
Intervention model
SEQUENTIAL
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
18 Years to 75 Years
Healthy volunteers
No
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.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| The incidence of XS228CN-related adverse events (AEs) and serious adverse events (SAEs) within 28 days after the last administration in ALS patients | within 28 days after the last administration |
Countries
China
Contacts
Primary ContactMichael LEE
Outcome results
None listed