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

2026년 5월 18일 업데이트: 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

연구 개요

상태

아직 모집하지 않음

상세 설명

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.

연구 유형

중재적

등록 (추정된)

6

단계

  • 1단계

연락처 및 위치

이 섹션에서는 연구를 수행하는 사람들의 연락처 정보와 이 연구가 수행되는 장소에 대한 정보를 제공합니다.

연구 연락처

연구 연락처 백업

참여기준

연구원은 적격성 기준이라는 특정 설명에 맞는 사람을 찾습니다. 이러한 기준의 몇 가지 예는 개인의 일반적인 건강 상태 또는 이전 치료입니다.

자격 기준

공부할 수 있는 나이

  • 성인
  • 고령자

건강한 자원 봉사자를 받아들입니다

아니

설명

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.

공부 계획

이 섹션에서는 연구 설계 방법과 연구가 측정하는 내용을 포함하여 연구 계획에 대한 세부 정보를 제공합니다.

연구는 어떻게 설계됩니까?

디자인 세부사항

  • 주 목적: 치료
  • 할당: 해당 없음
  • 중재 모델: 단일 그룹 할당
  • 마스킹: 없음(오픈 라벨)

무기와 개입

참가자 그룹 / 팔
개입 / 치료
실험적: 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

연구는 무엇을 측정합니까?

주요 결과 측정

결과 측정
측정값 설명
기간
Dose-Limiting Toxicity
기간: [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)
기간: [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]

2차 결과 측정

결과 측정
측정값 설명
기간
Functional System Score
기간: 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)
기간: [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)
기간: [Time Frame: 12 weeks, 24 weeks]
To measure disability level in patients with NMOSD relapse using EDSS.
[Time Frame: 12 weeks, 24 weeks]

공동 작업자 및 조사자

여기에서 이 연구와 관련된 사람과 조직을 찾을 수 있습니다.

수사관

  • 수석 연구원: sungmin Kim, MD, PhD, Seoul University Hospital

연구 기록 날짜

이 날짜는 ClinicalTrials.gov에 대한 연구 기록 및 요약 결과 제출의 진행 상황을 추적합니다. 연구 기록 및 보고된 결과는 공개 웹사이트에 게시되기 전에 특정 품질 관리 기준을 충족하는지 확인하기 위해 국립 의학 도서관(NLM)에서 검토합니다.

연구 주요 날짜

연구 시작 (추정된)

2026년 12월 1일

기본 완료 (추정된)

2027년 12월 30일

연구 완료 (추정된)

2028년 12월 30일

연구 등록 날짜

최초 제출

2025년 1월 6일

QC 기준을 충족하는 최초 제출

2026년 5월 18일

처음 게시됨 (실제)

2026년 5월 19일

연구 기록 업데이트

마지막 업데이트 게시됨 (실제)

2026년 5월 19일

QC 기준을 충족하는 마지막 업데이트 제출

2026년 5월 18일

마지막으로 확인됨

2026년 5월 1일

추가 정보

이 연구와 관련된 용어

기타 연구 ID 번호

  • CE211NS21_NMO123

약물 및 장치 정보, 연구 문서

미국 FDA 규제 의약품 연구

아니

미국 FDA 규제 기기 제품 연구

아니

미국에서 제조되어 미국에서 수출되는 제품

예

이 정보는 변경 없이 clinicaltrials.gov 웹사이트에서 직접 가져온 것입니다. 귀하의 연구 세부 정보를 변경, 제거 또는 업데이트하도록 요청하는 경우 register@clinicaltrials.gov. 문의하십시오. 변경 사항이 clinicaltrials.gov에 구현되는 즉시 저희 웹사이트에도 자동으로 업데이트됩니다. .

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