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

18 de maio de 2026 atualizado por: 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

Visão geral do estudo

Status

Ainda não está recrutando

Intervenção / Tratamento

Descrição detalhada

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.

Tipo de estudo

Intervencional

Inscrição (Estimado)

6

Estágio

  • Fase 1

Contactos e Locais

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Contato de estudo

Estude backup de contato

Critérios de participação

Os pesquisadores procuram pessoas que se encaixem em uma determinada descrição, chamada de critérios de elegibilidade. Alguns exemplos desses critérios são a condição geral de saúde de uma pessoa ou tratamentos anteriores.

Critérios de elegibilidade

Idades elegíveis para estudo

  • Adulto
  • Adulto mais velho

Aceita Voluntários Saudáveis

Não

Descrição

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.

Plano de estudo

Esta seção fornece detalhes do plano de estudo, incluindo como o estudo é projetado e o que o estudo está medindo.

Como o estudo é projetado?

Detalhes do projeto

  • Finalidade Principal: Tratamento
  • Alocação: N / D
  • Modelo Intervencional: Atribuição de grupo único
  • Mascaramento: Nenhum (rótulo aberto)

Armas e Intervenções

Grupo de Participantes / Braço
Intervenção / Tratamento
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

O que o estudo está medindo?

Medidas de resultados primários

Medida de resultado
Descrição da medida
Prazo
Dose-Limiting Toxicity
Prazo: [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)
Prazo: [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]

Medidas de resultados secundários

Medida de resultado
Descrição da medida
Prazo
Functional System Score
Prazo: 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)
Prazo: [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)
Prazo: [Time Frame: 12 weeks, 24 weeks]
To measure disability level in patients with NMOSD relapse using EDSS.
[Time Frame: 12 weeks, 24 weeks]

Colaboradores e Investigadores

É aqui que você encontrará pessoas e organizações envolvidas com este estudo.

Patrocinador

Investigadores

  • Investigador principal: sungmin Kim, MD, PhD, Seoul University Hospital

Datas de registro do estudo

Essas datas acompanham o progresso do registro do estudo e os envios de resumo dos resultados para ClinicalTrials.gov. Os registros do estudo e os resultados relatados são revisados ​​pela National Library of Medicine (NLM) para garantir que atendam aos padrões específicos de controle de qualidade antes de serem publicados no site público.

Datas Principais do Estudo

Início do estudo (Estimado)

1 de dezembro de 2026

Conclusão Primária (Estimado)

30 de dezembro de 2027

Conclusão do estudo (Estimado)

30 de dezembro de 2028

Datas de inscrição no estudo

Enviado pela primeira vez

6 de janeiro de 2025

Enviado pela primeira vez que atendeu aos critérios de CQ

18 de maio de 2026

Primeira postagem (Real)

19 de maio de 2026

Atualizações de registro de estudo

Última Atualização Postada (Real)

19 de maio de 2026

Última atualização enviada que atendeu aos critérios de controle de qualidade

18 de maio de 2026

Última verificação

1 de maio de 2026

Mais Informações

Termos relacionados a este estudo

Outros números de identificação do estudo

  • CE211NS21_NMO123

Informações sobre medicamentos e dispositivos, documentos de estudo

Estuda um medicamento regulamentado pela FDA dos EUA

Não

Estuda um produto de dispositivo regulamentado pela FDA dos EUA

Não

produto fabricado e exportado dos EUA

Sim

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