Neuromyelitis Optica Spectrum Disorder RelapsePhase 1NCT07595965
Evaluate the Safety and Tolerability of CE211NS21 in Patients With AQP4-IgG-positive NMOSD Relapse
A Phase 1 study testing CE211NS21 in people with Neuromyelitis Optica Spectrum Disorder Relapse, run by Corestemchemon, Inc..
Trial summary
To evaluate the safety and tolerability of allogeneic bone marrow-derived mesenchymal stem cells (CE211NS21) in patients with severe anti-aquaporin-4-immunoglobulin G positive neuromyelitis optica spectrum disorder (AQP4-IgG-positive NMOSD) relapse Read more about this study
- Who is it for?
- Ages 19 to 65 years
- Conditions the study is for
- Neuromyelitis Optica Spectrum Disorder Relapse
- What is being tested?
- CE211NS21
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 6 participants
- Where
- No sites listed yet
- Study dates
- 1 Dec 2026to 30 Dec 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 19 to 65 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Male or female adults aged 19 years or older and 65 years or younger (by full age) at the time of obtaining informed consent.
- Patients who have a bone marrow donor who is 19 years or older and 70 years or younger (by full age) among their blood relatives.
- Patients diagnosed with anti-aquaporin-4-immunoglobulin G (AQP4-IgG) positive neuromyelitis optica spectrum disorder based on serum testing at the time of screening.
- Patients who have experienced a severe relapse† (first attack or acute relapse) within 28 days prior to screening and meet the following criteria. However, in patients with both transverse myelitis and optic neuritis, the criteria for the site of relapse are applied.
- <Subgroup of Transverse Myelitis> Expanded disability status scale (EDSS) scale of 4.0 or higher and 8.5 or lower.
- <Subgroup of Optic Neuritis> Best corrected visual acuity (BCVA) of 20/200 or lower in one or both eyes.
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- †Relapse: New onset of neurological symptoms related to neuromyelitis optica or worsening of pre-existing neurological symptoms, objective changes on neurological examination (clinical findings or MRI findings) persisting for 24 hours or more, or the new onset of neurological symptoms or worsening of neurological symptoms requiring treatment.
- Patients who can come to outpatient visits alone or with caregiver assistance
- Women of childbearing potential who have not undergone sterilization must agree to use appropriate contraception* until 12 months after the administration of the investigational product, and must provide evidence of not being in a childbearing state at the time of screening by meeting one of the following criteria:
- For male patients who have not undergone a vasectomy: The patient must agree to use a barrier method of contraception (e.g., condoms) and ensure that he and his partner use appropriate contraception* until 12 months after the administration of the investigational product, and must agree to refrain from donating sperm.
- Patients who have been fully informed about this clinical trial, have voluntarily agreed to participate, and have given written consent to comply with the clinical trial's requirements.
Who cannot join
- Patients with any of the following cardiovascular diseases at the time of screening:
- Myocardial infarction, unstable arrhythmia, and/or unstable angina within 6 months.
- QTc interval ≥ 450 milliseconds or clinically significant changes on the electrocardiogram.
- Congestive heart failure of New York Heart Association (NYHA) Class II or higher.
- Stroke or transient ischemic attack (TIA) within 6 months.
- Patients with a history of any other malignancy within 5 years prior to screening.
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- Patients who are HIV positive.
- Patients deemed unsuitable for participation in this clinical trial by the investigator based on active hepatitis (HBV, HCV) test results.
- Patients with acute or severe infections.
- Patients for whom lumber puncture is contraindicated.
- Patients with a history of major neurological diseases other than the target disease (including a history of polio).
- Patients suspected of having demyelinating diseases other than the target disease or progressive multifocal leukoencephalopathy (PML).
- Patients with a history of active infection within 4 weeks prior to screening (patients with conditions such as onychomycosis or dental caries may be allowed to participate in this clinical trial if deemed suitable by the investigator).
- Patients who have undergone major surgery requiring general anesthesia within 4 weeks prior to screening.
- Patients who are legally incapacitated, have active psychiatric disorders, or have severe neurological or psychiatric problems that could affect the conduct of the clinical trial.
- Patients who have previously received other cell therapies.
- Patients with hypersensitivity to bovine proteins, penicillin, or streptomycin antibiotics.
- Patients with a history of hypersensitivity to any components of the investigational product.
- <Related to contraindicated concomitant medications>
- Patients who require high-dose steroids exceeding 0.5 mg/kg/day or pulse steroid therapy at the baseline*.
- Patients who have received immunosuppressants or immunomodulators other than concomitantly allowable immunosuppressive therapy (IST), or any other investigational products within 12 weeks prior to screening (or within 5 times the medication's half-life).
- <Related to laboratory tests>
- Patients whose laboratory test results at screening fall into any of the following categories:
- ANC <1,500/mm³
- Platelet count <100,000/mm³
- Hemoglobin <9.0 g/dL (Patients may be eligible if hemoglobin level recovers to 9.0 g/dL or higher. However, transfusions within 7 days prior to screening to meet this criterion are not allowed.)
- Serum creatinine >2.0xUNL
- Total bilirubin >2.0xUNL
- AST, ALT >3.0xUNL <Others>
- Pregnant or breastfeeding women, or those with a positive pregnancy test at screening.
- Patients who have been enrolled in another clinical trial within 12 weeks prior to screening (however, participation is allowed for those enrolled in non-interventional observational studies without the administration of investigational products).
- Patients deemed unsuitable for participation in this clinical trial by the investigator for any other reasons.
Trial locations and contacts
No sites are listed yet. Contact the study team or a site near you to ask about taking part.
About this study
Official title: A Phase I Clinical Trial to Evaluate the Safety and Tolerability of Allogeneic Bone Marrow-derived Mesenchymal Stem Cells in Patients With Severe Anti-aqua Porin-4-immunoglobulin G Positive Neuromyelitis Optica Spectrum Disorder Relapse
To evaluate the safety and tolerability of allogeneic bone marrow-derived mesenchymal stem cells (CE211NS21) in patients with severe anti-aquaporin-4-immunoglobulin G positive neuromyelitis optica spectrum disorder (AQP4-IgG-positive NMOSD) relapse
Read the detailed description
CE211NS21 is an allogeneic myeloid-derived mesenchymal stem cell therapy that secretes various growth factors and cytokines and regulates immune activity to rejuvenate myelin, and this clinical trial aims to confirm the safety and tolerability of CE211NS21 in phase 1 clinical trials in patients with anti-aquaporin4 antibody-positive optic spectrum category disease.
In terms of risk assessment, the safety was confirmed when 1x10^6 cells/kg of CE211NS21 was administered to C57BL6 mice three times at intervals of two weeks, and safety and tolerability were confirmed when 1x10^6 cells/kg of allogeneic myeloid-derived mesenchymal stem cells (CS20BR08) were administered in the intrathecal twice at 28 days intervals in therapeutic use in NMOSD patients.
When CE211NS21 was administered to C57BL6 mice intrathecal, the lethal dose exceeded the high dose of 1x10^6 cells/kg, and the non-toxic dose (NOAEL) was 1x10^6 cells/head/20uL when administered repeatedly three times at intervals of two weeks into the intrathecal. Therefore, in this clinical trial, CE211NS21 (step 1 dose 1.0x10^6 cells/kg, step 2x10^6 cells/kg) was set to be administered three times in consideration of the toxicity test result of three repeated doses and the safety of up to three doses in therapeutic clinical trials.
And the administration administers the first or second-stage dose into the intrathecal at baseline, at 4 weeks and at 16 weeks.
- Being tested
- CE211NS21
- Keywords
- cell therapy, Phase 1 clinical trial, allogeneic bone marrow-derived mesenchymal stem cells
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
CE211NS21Biological
baseline (D0), 4-week point (Visit 3), and 16-week point (Visit 6) for intrathecal administration of CE211NS21, Step 1 dose : 1x10^6 cells/kg Step 2 dose : 2x10^6 cells/kg; The Duration of follow up study following the administration of CE211NS21 is 5 years
| Study group | Role | What they receive |
|---|---|---|
| CE211NS21 | Experimental | baseline (D0), 4-week point (Visit 3), and 16-week point (Visit 6) for intrathecal administration of CE211NS21. Receives: Biological: CE211NS21 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Dose-Limiting Toxicity
To evaluate the incidence of dose-limiting toxicity (DLT) of CE211NS21 Injection in patients with NMOSD relapse.
Measured: [Time Frame: up to 4 weeks]
Adverse Events (AEs)
To evaluate the safety and tolerability of CE211NS21 Injection in patients with NMOSD relapse by assessing treatment-emergent adverse events.
Measured: [Time Frame: up to 4 weeks]
Secondary outcomes (3)
Functional System Score
To evaluate functional impairment in patients with NMOSD relapse using the Functional System Score.
Measured: 12 weeks, 24 weeks
36-item Short Form Survey (SF-36)
To evaluate health-related quality of life in patients with NMOSD relapse using SF-36.
Measured: [Time Frame: 12 weeks, 24 weeks]
Expanded Disability Status Scale (EDSS)
To measure disability level in patients with NMOSD relapse using EDSS.
Measured: [Time Frame: 12 weeks, 24 weeks]
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted19 May 2026
- Study start1 Dec 2026 (estimated)
- Primary completion30 Dec 2027 (estimated)
- Study completion30 Dec 2028 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Neuromyelitis Optica Spectrum Disorder RelapseStudies 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
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 6 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Corestemchemon, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- sungmin Kim, MD, PhD, Principal investigator, Seoul University hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07595965
- Sponsor's protocol ID
- CE211NS21_NMO123
- Status last verified
- May 2026
- First submitted
- 6 Jan 2025
- Last updated
- 19 May 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 19 May 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase I Clinical Trial to Evaluate the Safety and Tolerability of Allogeneic Bone Marrow-derived Mesenchymal Stem Cells in Patients With Severe Anti-aqua Porin-4-immunoglobulin G Positive Neuromyelitis Optica Spectrum Disorder Relapse
- ClinicalTrials.gov ID
- NCT07595965
- Acronym / sponsor ID
- CE211NS21_NMO123
- Keywords
- cell therapy, Phase 1 clinical trial, allogeneic bone marrow-derived mesenchymal stem cells
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Not yet recruiting
- Enrolment
- 6 participants (estimated)
- Lead sponsor
- Corestemchemon, Inc.Industry (pharma, biotech, devices)
- Study start
- 1 Dec 2026 (estimated)
- Primary completion
- 30 Dec 2027 (estimated)
- Study completion
- 30 Dec 2028 (estimated)
- First posted
- 19 May 2026 (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.
To evaluate the safety and tolerability of allogeneic bone marrow-derived mesenchymal stem cells (CE211NS21) in patients with severe anti-aquaporin-4-immunoglobulin G positive neuromyelitis optica spectrum disorder (AQP4-IgG-positive NMOSD) relapse
Detailed description
CE211NS21 is an allogeneic myeloid-derived mesenchymal stem cell therapy that secretes various growth factors and cytokines and regulates immune activity to rejuvenate myelin, and this clinical trial aims to confirm the safety and tolerability of CE211NS21 in phase 1 clinical trials in patients with anti-aquaporin4 antibody-positive optic spectrum category disease.
In terms of risk assessment, the safety was confirmed when 1x10^6 cells/kg of CE211NS21 was administered to C57BL6 mice three times at intervals of two weeks, and safety and tolerability were confirmed when 1x10^6 cells/kg of allogeneic myeloid-derived mesenchymal stem cells (CS20BR08) were administered in the intrathecal twice at 28 days intervals in therapeutic use in NMOSD patients.
When CE211NS21 was administered to C57BL6 mice intrathecal, the lethal dose exceeded the high dose of 1x10^6 cells/kg, and the non-toxic dose (NOAEL) was 1x10^6 cells/head/20uL when administered repeatedly three times at intervals of two weeks into the intrathecal. Therefore, in this clinical trial, CE211NS21 (step 1 dose 1.0x10^6 cells/kg, step 2x10^6 cells/kg) was set to be administered three times in consideration of the toxicity test result of three repeated doses and the safety of up to three doses in therapeutic clinical trials.
And the administration administers the first or second-stage dose into the intrathecal at baseline, at 4 weeks and at 16 weeks.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| CE211NS21 | Experimental | Biological: CE211NS21baseline (D0), 4-week point (Visit 3), and 16-week point (Visit 6) for intrathecal administration of CE211NS21. |
Who may qualify
- Ages
- 19 to 65 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (11 listed)
- Male or female adults aged 19 years or older and 65 years or younger (by full age) at the time of obtaining informed consent.
- Patients who have a bone marrow donor who is 19 years or older and 70 years or younger (by full age) among their blood relatives.
- Patients diagnosed with anti-aquaporin-4-immunoglobulin G (AQP4-IgG) positive neuromyelitis optica spectrum disorder based on serum testing at the time of screening.
- Patients who have experienced a severe relapse† (first attack or acute relapse) within 28 days prior to screening and meet the following criteria. However, in patients with both transverse myelitis and optic neuritis, the criteria for the site of relapse are applied.
- <Subgroup of Transverse Myelitis> Expanded disability status scale (EDSS) scale of 4.0 or higher and 8.5 or lower.
Key exclusion criteria (32 listed)
- Patients with any of the following cardiovascular diseases at the time of screening:
- Patients with a history of any other malignancy within 5 years prior to screening.
- Patients who are HIV positive.
- Patients deemed unsuitable for participation in this clinical trial by the investigator based on active hepatitis (HBV, HCV) test results.
- Patients with acute or severe infections.
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 |
|---|---|---|
| CE211NS21baseline (D0), 4-week point (Visit 3), and 16-week point (Visit 6) for intrathecal administration of CE211NS21, Step 1 dose : 1x10^6 cells/kg Step 2 dose : 2x10^6 cells/kg; The Duration of follow up study following the administration of CE211NS21 is 5 years | Biological | CE211NS21 |
Primary outcome
Dose-Limiting Toxicity
Time frame: [Time Frame: up to 4 weeks]
To evaluate the incidence of dose-limiting toxicity (DLT) of CE211NS21 Injection in patients with NMOSD relapse.
Adverse Events (AEs)
Time frame: [Time Frame: up to 4 weeks]
To evaluate the safety and tolerability of CE211NS21 Injection in patients with NMOSD relapse by assessing treatment-emergent adverse events.
3 secondary outcomes
Functional System Score
Time frame: 12 weeks, 24 weeks
36-item Short Form Survey (SF-36)
Time frame: [Time Frame: 12 weeks, 24 weeks]
Expanded Disability Status Scale (EDSS)
Time frame: [Time Frame: 12 weeks, 24 weeks]
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 19 May 2026.
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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.
sungmin Kim, MD, PhD principal investigator
Seoul University hospital
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing CE211NS21 in people with Neuromyelitis Optica Spectrum Disorder Relapse, run by Corestemchemon, Inc..
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 11 inclusion and 32 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: opening soon, as of the sponsor's last update on 19 May 2026. Status can change between updates, so confirm with the study contact before making plans.
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 Kwijoo kim (02-497-3711, kwjkim@csco.co.kr), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Dose-Limiting Toxicity (time frame: [Time Frame: up to 4 weeks]). The study lists 2 primary outcomes in all. 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 19 May 2026.