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

NCT ID: NCT07118319

Last Updated: 2025-11-21

Basic Information

• Recruitment Status: RECRUITING
• Clinical Phase: PHASE1
• Total Enrollment: 12 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2025-09-15
• Study Completion Date: 2028-09-30

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

Conditions

Amyotrophic Lateral Sclerosis (ALS)

Study Design

• Allocation Method: NON_RANDOMIZED
• Intervention Model: SEQUENTIAL
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

5.0×10^7 cells/person/dose

intrathecal injection

Group Type: EXPERIMENTAL

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

Intervention Type: DRUG

Injection, once, 6 months

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

Intervention Type: DRUG

Injection, every two weeks for four doses, 6 months

1.0×10^8 cells/person/dose

intrathecal injection

Group Type: EXPERIMENTAL

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

Intervention Type: DRUG

Injection, once, 6 months

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

Intervention Type: DRUG

Injection, every two weeks for four doses, 6 months

Interventions

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

Injection, once, 6 months

Intervention Type: DRUG

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

Injection, every two weeks for four doses, 6 months

Intervention Type: DRUG

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.

• Minimum Eligible Age: 18 Years
• Maximum Eligible Age: 75 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

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

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Locations

Peking University Third Hospital

Beijing, Beijing Municipality, China

Site Status: RECRUITING

Countries

China

Central Contacts

Michael LEE

Role: CONTACT

CEO@xellsmart.com

+86 21 64027719

Facility Contacts

Dongsheng Fan

Role: primary

dsfan@sina.com

+86 13701023871

Other Identifiers

2025LP01221

Identifier Type: REGISTRY

Identifier Source: secondary_id

XS228-Allo-ALS-CN1/2-P01

Identifier Type: -

Identifier Source: org_study_id