Evaluate the Safety and Tolerability of CE211NS21 in Patients With AQP4-IgG-positive NMOSD Relapse

NCT07595965 | Not Yet Recruiting Phase 1 DMC

• 1 other identifier: CE211NS21_NMO123
• Study Type: interventional
• Target: 6
• Locations: 0 countries
• Sites: N/A

Brief 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

Conditions

Neuromyelitis Optica Spectrum Disorder Relapse

Keywords

cell therapy; Phase 1 clinical trial; allogeneic bone marrow-derived mesenchymal stem cells

Outcome Measures

Primary Outcomes (2)

• Dose-Limiting Toxicity

  To evaluate the incidence of dose-limiting toxicity (DLT) of CE211NS21 Injection in patients with NMOSD relapse.

  [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.

  [Time Frame: up to 4 weeks]

Secondary Outcomes (3)

• Functional System Score

  12 weeks, 24 weeks

• 36-item Short Form Survey (SF-36)

  [Time Frame: 12 weeks, 24 weeks]

• Expanded Disability Status Scale (EDSS)

  [Time Frame: 12 weeks, 24 weeks]

Study Arms (1)

CE211NS21

EXPERIMENTAL

baseline (D0), 4-week point (Visit 3), and 16-week point (Visit 6) for intrathecal administration of CE211NS21.

Biological: CE211NS21

Interventions

CE211NS21 BIOLOGICAL

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

CE211NS21

Eligibility Criteria

• Age: 19 Years - 65 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.
• †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.

You may not qualify if:

• 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.
• 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.

Sponsors & Collaborators

• Corestemchemon, Inc.: lead

Study Officials

• sungmin Kim, MD, PhD

  Seoul University hospital

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Kwijoo kim

CONTACT

02-497-3711; kwjkim@csco.co.kr

Yerim jung

CONTACT

02-497-3711; yrjung@csco.co.kr

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: January 6, 2025
• First Posted: May 19, 2026
• Study Start (Estimated): December 1, 2026
• Primary Completion (Estimated): December 30, 2027
• Study Completion (Estimated): December 30, 2028
• Last Updated: May 19, 2026

Record last verified: 2026-05