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

NCT07118319 | Recruiting Phase 1 FDA Drug

• 2 other identifiers: XS228-Allo-ALS-CN1/2-P012025LP01221
• Study Type: interventional
• Target: 12
• Locations: 1 country
• Sites: 1

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)

Outcome Measures

Primary Outcomes (1)

• 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

Study Arms (2)

5.0×10^7 cells/person/dose

EXPERIMENTAL

intrathecal injection

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

1.0×10^8 cells/person/dose

EXPERIMENTAL

intrathecal injection

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

Interventions

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

Injection, once, 6 months

1.0×10^8 cells/person/dose; 5.0×10^7 cells/person/dose

Eligibility Criteria

• Age: 18 Years - 75 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• years of age (inclusive of 18 and 75 years), regardless of gender;
• Diagnosed of definite or probable ALS according to the revised EI Escorial criteria;
• Respiratory function FVC at baseline was ≥70% of the predicted value (FVC%);
• 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;
• Volunteer to participate in the clinical study, understand and sign the informed consent form.

You may not qualify if:

• Subject has signs and symptoms of neuromuscular weakness, and other causes of muscle weakness cannot be ruled out;
• Baseline body mass index (BMI) \< 18.5 kg/m²;
• Primary lateral sclerosis presenting only with upper motor neuron symptoms;
• Significant psychiatric disorders that the investigator assesses may affect evaluation;
• Diseases causing neurological or muscular dysfunction, such as metabolic muscle diseases or myasthenia gravis;
• Diagnosed autoimmune diseases with uncontrolled severe arthritis or other conditions (e.g., lameness) that the investigator assesses may affect evaluation;
• Acute active infections requiring antibiotics, antivirals, or antifungals that occurred within the 2 weeks prior to screening and are not controlled;
• Subject diagnosed with active pulmonary tuberculosis or treated for suspected tuberculosis;
• Diagnosed severe pulmonary diseases that the investigator assesses may affect evaluation;
• Poorly controlled hypertension;
• Previous or detected cardiac abnormalities;
• A history of cirrhosis, chronic hepatitis, or liver function at screening;
• A history of chronic kidney disease;
• Previous history of bleeding, abnormal clotting, or being treated with anticoagulation;
• Active hepatitis B, active hepatitis C, human immunodeficiency virus (HIV) antibody or treponema pallidum antibody positive at screening;

Sponsors & Collaborators

• XellSmart Bio-Pharmaceutical (Suzhou) Co., Ltd.: lead

Study Sites (1)

Peking University Third Hospital

Beijing, Beijing Municipality, 100191, China

RECRUITING

MeSH Terms

Conditions

Amyotrophic Lateral Sclerosis

Condition Hierarchy (Ancestors)

Spinal Cord Diseases; Central Nervous System Diseases; Nervous System Diseases; Motor Neuron Disease; Neurodegenerative Diseases; TDP-43 Proteinopathies; Neuromuscular Diseases; Proteostasis Deficiencies; Metabolic Diseases; Nutritional and Metabolic Diseases

Central Study Contacts

Michael LEE

CONTACT

+86 21 64027719; CEO@xellsmart.com

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SEQUENTIAL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: July 24, 2025
• First Posted: August 12, 2025
• Study Start: September 15, 2025
• Primary Completion (Estimated): January 31, 2027
• Study Completion (Estimated): September 30, 2028
• Last Updated: November 21, 2025

Record last verified: 2025-07

Data Sharing

• IPD Sharing: Will share

Locations

CN (1)