XS228CN motor neuron progenitor cell therapy for ALS: safety, tolerability and early efficacy

Is this trial a fit for you?

A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.

What this trial studies

This is a Phase 1 interventional trial testing an allogeneic iPSC-derived motor neuron progenitor cell product (XS228CN) in adults with definite or probable ALS. Participants aged 18–75 with baseline FVC ≥70% will receive the cell product and be closely monitored for safety, tolerability, respiratory function, and standard clinical measures of ALS progression. The primary goal is to characterize adverse events and tolerability; investigators will also collect preliminary signals of efficacy. The study is sponsored by XellSmart Bio-Pharmaceutical and conducted at Peking University Third Hospital in Beijing.

Why it matters

Who can join: the full rules

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.

Where it's running

• Peking University Third Hospital
  Beijing, Beijing Municipality, China Recruiting

Study contacts

• Study coordinator: Michael LEE
• Email: CEO@xellsmart.com
• Phone: +86 21 64027719

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How to take part

1. Review the eligibility criteria above with your treating physician.
2. Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
3. Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.