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

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

This study will test whether XS228CN iPSC-derived motor neuron progenitor cells are safe and tolerable in adults with definite or probable ALS who have preserved lung function.

Quick facts

• Phase: PHASE1
• Study type: Interventional
• Enrollment: 12 (estimated)
• Ages: 18 Years to 75 Years
• Sex: All
• Sponsor: XellSmart Bio-Pharmaceutical (Suzhou) Co., Ltd. (industry)
• Locations: 1 site (Beijing, Beijing Municipality)
• Trial ID: NCT07118319 on ClinicalTrials.gov

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.

Who should consider this trial

Good fit: Adults 18–75 years old with definite or probable ALS by the revised El Escorial criteria, baseline FVC ≥70% predicted, who can consent and agree to required contraception if applicable are the intended participants.

Not a fit: Patients with significant respiratory impairment (FVC <70%), primary lateral sclerosis, other neuromuscular causes of weakness, low BMI (<18.5 kg/m²), or major psychiatric conditions are excluded and are unlikely to benefit from this Phase 1 intervention.

Why it matters

Potential benefit: If successful, this approach could provide a new cell-based therapy that slows motor neuron loss and helps preserve function in people with ALS.

How similar studies have performed: Related cell- and stem-cell–based early-phase trials in ALS have generally shown acceptable safety but only mixed or limited evidence of clinical benefit, so this approach is novel but builds on prior exploratory work.

Eligibility criteria

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 this trial is running

Beijing, Beijing Municipality

• Peking University Third Hospital — Beijing, Beijing Municipality, China (RECRUITING)

Study contacts

• Study coordinator: Michael LEE

Conditions: Amyotrophic Lateral Sclerosis