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

18. Mai 2026 aktualisiert von: 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

Studienübersicht

Status

Noch keine Rekrutierung

Intervention / Behandlung

Detaillierte Beschreibung

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.

Studientyp

Interventionell

Einschreibung (Geschätzt)

6

Phase

  • Phase 1

Kontakte und Standorte

Dieser Abschnitt enthält die Kontaktdaten derjenigen, die die Studie durchführen, und Informationen darüber, wo diese Studie durchgeführt wird.

Studienkontakt

Studieren Sie die Kontaktsicherung

Teilnahmekriterien

Forscher suchen nach Personen, die einer bestimmten Beschreibung entsprechen, die als Auswahlkriterien bezeichnet werden. Einige Beispiele für diese Kriterien sind der allgemeine Gesundheitszustand einer Person oder frühere Behandlungen.

Zulassungskriterien

Studienberechtigtes Alter

  • Erwachsene
  • Älterer Erwachsener

Akzeptiert gesunde Freiwillige

Nein

Beschreibung

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.

Studienplan

Dieser Abschnitt enthält Einzelheiten zum Studienplan, einschließlich des Studiendesigns und der Messung der Studieninhalte.

Wie ist die Studie aufgebaut?

Designdetails

  • Hauptzweck: Behandlung
  • Zuteilung: N / A
  • Interventionsmodell: Einzelgruppenzuweisung
  • Maskierung: Keine (Offenes Etikett)

Waffen und Interventionen

Teilnehmergruppe / Arm
Intervention / Behandlung
Experimental: 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

Was misst die Studie?

Primäre Ergebnismessungen

Ergebnis Maßnahme
Maßnahmenbeschreibung
Zeitfenster
Dose-Limiting Toxicity
Zeitfenster: [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)
Zeitfenster: [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]

Sekundäre Ergebnismessungen

Ergebnis Maßnahme
Maßnahmenbeschreibung
Zeitfenster
Functional System Score
Zeitfenster: 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)
Zeitfenster: [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)
Zeitfenster: [Time Frame: 12 weeks, 24 weeks]
To measure disability level in patients with NMOSD relapse using EDSS.
[Time Frame: 12 weeks, 24 weeks]

Mitarbeiter und Ermittler

Hier finden Sie Personen und Organisationen, die an dieser Studie beteiligt sind.

Ermittler

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

Studienaufzeichnungsdaten

Diese Daten verfolgen den Fortschritt der Übermittlung von Studienaufzeichnungen und zusammenfassenden Ergebnissen an ClinicalTrials.gov. Studienaufzeichnungen und gemeldete Ergebnisse werden von der National Library of Medicine (NLM) überprüft, um sicherzustellen, dass sie bestimmten Qualitätskontrollstandards entsprechen, bevor sie auf der öffentlichen Website veröffentlicht werden.

Haupttermine studieren

Studienbeginn (Geschätzt)

1. Dezember 2026

Primärer Abschluss (Geschätzt)

30. Dezember 2027

Studienabschluss (Geschätzt)

30. Dezember 2028

Studienanmeldedaten

Zuerst eingereicht

6. Januar 2025

Zuerst eingereicht, das die QC-Kriterien erfüllt hat

18. Mai 2026

Zuerst gepostet (Tatsächlich)

19. Mai 2026

Studienaufzeichnungsaktualisierungen

Letztes Update gepostet (Tatsächlich)

19. Mai 2026

Letztes eingereichtes Update, das die QC-Kriterien erfüllt

18. Mai 2026

Zuletzt verifiziert

1. Mai 2026

Mehr Informationen

Begriffe im Zusammenhang mit dieser Studie

Andere Studien-ID-Nummern

  • CE211NS21_NMO123

Arzneimittel- und Geräteinformationen, Studienunterlagen

Studiert ein von der US-amerikanischen FDA reguliertes Arzneimittelprodukt

Nein

Studiert ein von der US-amerikanischen FDA reguliertes Geräteprodukt

Nein

Produkt, das in den USA hergestellt und aus den USA exportiert wird

Ja

Diese Informationen wurden ohne Änderungen direkt von der Website clinicaltrials.gov abgerufen. Wenn Sie Ihre Studiendaten ändern, entfernen oder aktualisieren möchten, wenden Sie sich bitte an register@clinicaltrials.gov. Sobald eine Änderung auf clinicaltrials.gov implementiert wird, wird diese automatisch auch auf unserer Website aktualisiert .

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