Amyotrophic Lateral Sclerosis (ALS)Phase 1NCT07118319
The Safety, Tolerability and Preliminary Efficacy of Derived Motor Neuron Progenitor Cells (XS228CN) in Subjects With Amyotrophic Lateral Sclerosis
A Phase 1 study testing Human Allogeneic Induced Pluripotent Stem Cells (iPSCs) -Derived Motor Neuron Progenitor Cells (XS228CN) in people with Amyotrophic Lateral Sclerosis (ALS), run by XellSmart Bio-Pharmaceutical (Suzhou) Co., Ltd. at 1 site in China.
Trial 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 Read more about this study
- Who is it for?
- Ages 18 to 75 years
- Conditions the study is for
- Amyotrophic Lateral Sclerosis (ALS)
- What is being tested?
- Human Allogeneic Induced Pluripotent Stem Cells (iPSCs) -Derived Motor Neuron Progenitor Cells (XS228CN)
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 12 participants
- Where
- 1 siteChina
- Study dates
- 15 Sep 2025to 30 Sep 2028
- Phase
- Phase 1
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 75 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 18-75 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.
Who cannot join
- 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;
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- 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;
- History of severe trauma or surgery and may affect the assessment judged by investigator;
- Subjects with contraindications to lumbar puncture, including menifestations of injection site infection or high intracranial pressure.
- Those who have had a malignant tumor within 5 years prior to screening or are undergoing antitumor therapy;
- Have participated in other clinical trials within 3 months prior to screening;
- Pregnant or breastfeeding women;
- Subject who is judged by the investigator to be unsuitable for the clinical trial.
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
China1 site, 1 recruiting
- Recruiting
Peking University Third Hospital
Beijing, Beijing Municipality, 100191
Dongsheng Fan, +86 13701023871, dsfan@sina.com
About this study
Official title: 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
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)
- Being tested
- Human Allogeneic Induced Pluripotent Stem Cells (iPSCs) -Derived Motor Neuron Progenitor Cells (XS228CN)
- Medical subject headings
- Amyotrophic Lateral Sclerosis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Human Allogeneic Induced Pluripotent Stem Cells (iPSCs) -Derived Motor Neuron Progenitor Cells (XS228CN)Drug
Injection, once, 6 months
Human Allogeneic Induced Pluripotent Stem Cells (iPSCs) -Derived Motor Neuron Progenitor Cells (XS228CN)Drug
Injection, every two weeks for four doses, 6 months
| Study group | Role | What they receive |
|---|---|---|
| 5.0×10^7 cells/person/dose | Experimental | intrathecal injection Receives: Drug: Human Allogeneic Induced Pluripotent Stem Cells (iPSCs) -Derived Motor Neuron Progenitor Cells (XS228CN) |
| 1.0×10^8 cells/person/dose | Experimental | intrathecal injection Receives: Drug: Human Allogeneic Induced Pluripotent Stem Cells (iPSCs) -Derived Motor Neuron Progenitor Cells (XS228CN) |
What is being measured
The primary outcome is the main question the study is designed to answer.
The incidence of XS228CN-related adverse events (AEs) and serious adverse events (SAEs) within 28 days after the last administration in ALS patients
Measured: within 28 days after the last administration
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted12 Aug 2025
- Study start15 Sep 2025
- Primary completion31 Jan 2027 (estimated)
- Study completion30 Sep 2028 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Amyotrophic Lateral Sclerosis (ALS)Studies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Sequential
- Masking
- None (open label)
- Enrolment
- 12 participants (estimated)
Sponsor and investigators
- Lead sponsor
- XellSmart Bio-Pharmaceutical (Suzhou) Co., Ltd.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07118319
- Sponsor's protocol ID
- XS228-Allo-ALS-CN1/2-P01
- Other IDs
- 2025LP01221
- Status last verified
- Jul 2025
- First submitted
- 24 Jul 2025
- Last updated
- 21 Nov 2025
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 21 Nov 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- 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
- ClinicalTrials.gov ID
- NCT07118319
- Acronym / sponsor ID
- XS228-Allo-ALS-CN1/2-P01
- Conditions
- Amyotrophic Lateral Sclerosis (ALS)
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 12 participants (estimated)
- Lead sponsor
- XellSmart Bio-Pharmaceutical (Suzhou) Co., Ltd.Industry (pharma, biotech, devices)
- Study start
- 15 Sep 2025 (actual)
- Primary completion
- 31 Jan 2027 (estimated)
- Study completion
- 30 Sep 2028 (estimated)
- First posted
- 12 Aug 2025 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Sequential
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| 5.0×10^7 cells/person/dose | Experimental | Drug: Human Allogeneic Induced Pluripotent Stem Cells (iPSCs) -Derived Motor Neuron Progenitor Cells (XS228CN)intrathecal injection |
| 1.0×10^8 cells/person/dose | Experimental | Drug: Human Allogeneic Induced Pluripotent Stem Cells (iPSCs) -Derived Motor Neuron Progenitor Cells (XS228CN)intrathecal injection |
Who may qualify
- Ages
- 18 to 75 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (5 listed)
- 18-75 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.
Key exclusion criteria (21 listed)
- 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;
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Human Allogeneic Induced Pluripotent Stem Cells (iPSCs) -Derived Motor Neuron Progenitor Cells (XS228CN)Injection, once, 6 months | Drug | 1.0×10^8 cells/person/dose, 5.0×10^7 cells/person/dose |
| Human Allogeneic Induced Pluripotent Stem Cells (iPSCs) -Derived Motor Neuron Progenitor Cells (XS228CN)Injection, every two weeks for four doses, 6 months | Drug | 1.0×10^8 cells/person/dose, 5.0×10^7 cells/person/dose |
Primary outcome
The incidence of XS228CN-related adverse events (AEs) and serious adverse events (SAEs) within 28 days after the last administration in ALS patients
Time frame: within 28 days after the last administration
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 21 Nov 2025.
- Individual participant dataSharing planned: Yes
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing Human Allogeneic Induced Pluripotent Stem Cells (iPSCs) -Derived Motor Neuron Progenitor Cells (XS228CN) in people with Amyotrophic Lateral Sclerosis (ALS), run by XellSmart Bio-Pharmaceutical (Suzhou) Co., Ltd. at 1 site in China.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 5 inclusion and 21 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 21 Nov 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in China. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Michael LEE (+86 21 64027719, CEO@xellsmart.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
The incidence of XS228CN-related adverse events (AEs) and serious adverse events (SAEs) within 28 days after the last administration in ALS patients (time frame: within 28 days after the last administration). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 21 Nov 2025.