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

18. mai 2026 oppdatert av: Corestemchemon, Inc.

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

Studieoversikt

Status

Har ikke rekruttert ennå

Intervensjon / Behandling

Detaljert beskrivelse

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.

Studietype

Intervensjonell

Registrering (Antatt)

6

Fase

  • Fase 1

Kontakter og plasseringer

Denne delen inneholder kontaktinformasjon for de som utfører studien, og informasjon om hvor denne studien blir utført.

Studiekontakt

Studer Kontakt Backup

Deltakelseskriterier

Forskere ser etter personer som passer til en bestemt beskrivelse, kalt kvalifikasjonskriterier. Noen eksempler på disse kriteriene er en persons generelle helsetilstand eller tidligere behandlinger.

Kvalifikasjonskriterier

Alder som er kvalifisert for studier

  • Voksen
  • Eldre voksen

Tar imot friske frivillige

Nei

Beskrivelse

Inclusion Criteria:

  1. Male or female adults aged 19 years or older and 65 years or younger (by full age) at the time of obtaining informed consent.
  2. 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.
  3. Patients diagnosed with anti-aquaporin-4-immunoglobulin G (AQP4-IgG) positive neuromyelitis optica spectrum disorder based on serum testing at the time of screening.
  4. 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.

  5. Patients who can come to outpatient visits alone or with caregiver assistance
  6. 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:
  7. 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.
  8. 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.

Exclusion Criteria:

  1. 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.
  2. Patients with a history of any other malignancy within 5 years prior to screening.
  3. Patients who are HIV positive.
  4. Patients deemed unsuitable for participation in this clinical trial by the investigator based on active hepatitis (HBV, HCV) test results.
  5. Patients with acute or severe infections.
  6. Patients for whom lumber puncture is contraindicated.
  7. Patients with a history of major neurological diseases other than the target disease (including a history of polio).
  8. Patients suspected of having demyelinating diseases other than the target disease or progressive multifocal leukoencephalopathy (PML).
  9. 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).
  10. Patients who have undergone major surgery requiring general anesthesia within 4 weeks prior to screening.
  11. 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.
  12. Patients who have previously received other cell therapies.
  13. Patients with hypersensitivity to bovine proteins, penicillin, or streptomycin antibiotics.
  14. Patients with a history of hypersensitivity to any components of the investigational product.

    <Related to contraindicated concomitant medications>

  15. Patients who require high-dose steroids exceeding 0.5 mg/kg/day or pulse steroid therapy at the baseline*.
  16. 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>

  17. 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>
  18. Pregnant or breastfeeding women, or those with a positive pregnancy test at screening.
  19. 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).
  20. Patients deemed unsuitable for participation in this clinical trial by the investigator for any other reasons.

Studieplan

Denne delen gir detaljer om studieplanen, inkludert hvordan studien er utformet og hva studien måler.

Hvordan er studiet utformet?

Designdetaljer

  • Primært formål: Behandling
  • Tildeling: N/A
  • Intervensjonsmodell: Enkeltgruppeoppdrag
  • Masking: Ingen (Open Label)

Våpen og intervensjoner

Deltakergruppe / Arm
Intervensjon / Behandling
Eksperimentell: CE211NS21
baseline (D0), 4-week point (Visit 3), and 16-week point (Visit 6) for intrathecal administration of CE211NS21.
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

Hva måler studien?

Primære resultatmål

Resultatmål
Tiltaksbeskrivelse
Tidsramme
Dose-Limiting Toxicity
Tidsramme: [Time Frame: up to 4 weeks]
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)
Tidsramme: [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.
[Time Frame: up to 4 weeks]

Sekundære resultatmål

Resultatmål
Tiltaksbeskrivelse
Tidsramme
Functional System Score
Tidsramme: 12 weeks, 24 weeks
To evaluate functional impairment in patients with NMOSD relapse using the Functional System Score.
12 weeks, 24 weeks
36-item Short Form Survey (SF-36)
Tidsramme: [Time Frame: 12 weeks, 24 weeks]
To evaluate health-related quality of life in patients with NMOSD relapse using SF-36.
[Time Frame: 12 weeks, 24 weeks]
Expanded Disability Status Scale (EDSS)
Tidsramme: [Time Frame: 12 weeks, 24 weeks]
To measure disability level in patients with NMOSD relapse using EDSS.
[Time Frame: 12 weeks, 24 weeks]

Samarbeidspartnere og etterforskere

Det er her du vil finne personer og organisasjoner som er involvert i denne studien.

Etterforskere

  • Hovedetterforsker: sungmin Kim, MD, PhD, Seoul University Hospital

Studierekorddatoer

Disse datoene sporer fremdriften for innsending av studieposter og sammendragsresultater til ClinicalTrials.gov. Studieposter og rapporterte resultater gjennomgås av National Library of Medicine (NLM) for å sikre at de oppfyller spesifikke kvalitetskontrollstandarder før de legges ut på det offentlige nettstedet.

Studer hoveddatoer

Studiestart (Antatt)

1. desember 2026

Primær fullføring (Antatt)

30. desember 2027

Studiet fullført (Antatt)

30. desember 2028

Datoer for studieregistrering

Først innsendt

6. januar 2025

Først innsendt som oppfylte QC-kriteriene

18. mai 2026

Først lagt ut (Faktiske)

19. mai 2026

Oppdateringer av studieposter

Sist oppdatering lagt ut (Faktiske)

19. mai 2026

Siste oppdatering sendt inn som oppfylte QC-kriteriene

18. mai 2026

Sist bekreftet

1. mai 2026

Mer informasjon

Begreper knyttet til denne studien

Legemiddel- og utstyrsinformasjon, studiedokumenter

Studerer et amerikansk FDA-regulert medikamentprodukt

Nei

Studerer et amerikansk FDA-regulert enhetsprodukt

Nei

produkt produsert i og eksportert fra USA

Ja

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