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

XellSmart Bio-Pharmaceutical (Suzhou) Co., Ltd.

Status and phase: Not yet enrolling; Phase 1

Conditions: Amyotrophic Lateral Sclerosis (ALS)

Treatments: Drug: Human Allogeneic Induced Pluripotent Stem Cells (iPSCs) -Derived Motor Neuron Progenitor Cells (XS228CN)

Study type: Interventional

Funder types: Industry

Identifiers

NCT07118319

2025LP01221 (Registry Identifier)

XS228-Allo-ALS-CN1/2-P01

Details and patient eligibility

About

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

Enrollment: 12 estimated patients

Sex: All

Ages: 18 to 75 years old

Volunteers: No Healthy Volunteers

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.

Trial design

Primary purpose: Treatment

Allocation: Non-Randomized

Interventional model: Sequential Assignment

Masking: None (Open label)

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12 participants in 2 patient groups

5.0×10^7 cells/person/dose

Experimental group

Description:

intrathecal injection

Treatment:

Drug: Human Allogeneic Induced Pluripotent Stem Cells (iPSCs) -Derived Motor Neuron Progenitor Cells (XS228CN)

Drug: Human Allogeneic Induced Pluripotent Stem Cells (iPSCs) -Derived Motor Neuron Progenitor Cells (XS228CN)

1.0×10^8 cells/person/dose

Experimental group

Description:

intrathecal injection

Treatment:

Drug: Human Allogeneic Induced Pluripotent Stem Cells (iPSCs) -Derived Motor Neuron Progenitor Cells (XS228CN)

Drug: Human Allogeneic Induced Pluripotent Stem Cells (iPSCs) -Derived Motor Neuron Progenitor Cells (XS228CN)