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Nebulized UC-EVs for Recurrent or Refractory IMIDs With Lung Involvement

2026년 9월 4일 업데이트: Qiubai Li

A Phase I, Multicenter, Single-Arm, Dose-Escalation Study of Nebulized Human Umbilical Cord Mesenchymal Stromal Cell-Derived Extracellular Vesicles in Patients With Recurrent or Refractory Immune-Mediated Inflammatory Diseases With Lung Involvement

Immune-mediated inflammatory diseases (IMIDs) are systemic inflammatory disorders driven by dysregulated immune responses that can affect multiple organs, including the lungs. Examples include Behçet disease, IgG4-related disease, and systemic sclerosis (SSc). IMID-related lung involvement may cause progressive lung damage, impaired lung function, disability, and increased mortality, but effective targeted treatment options remain limited.

This clinical trial will study nebulized human umbilical cord mesenchymal stromal cell-derived extracellular vesicles, also called UC-EVs, in adults with recurrent or refractory IMIDs with lung involvement.

연구 개요

상세 설명

This trial is an investigator initiated, multicenter, open-label, single-arm, dose-escalation trial. The main goal is to learn whether nebulized UC-EVs are safe and tolerable in adults with recurrent or refractory IMIDs with lung involvement.]. Researchers will also look for early signs of whether UC-EVs may help improve lung function, lung imaging findings, symptoms, and disease-related laboratory tests.

The study will include patients aged 18 and 80 years with IMID-related lung involvement. Eligible participants will have a diagnosis of IgG4-RD or SSc for at least 6 months. They must be receiving stable standard-of-care (SOC) treatment for the required period, as described in the inclusion criteria. They must also have adequate organ function and no history of severe allergy, as described in the eligibility criteria.

Eligible participants will:

  • Inhale nebulized UC-EVs once daily for 7 days, followed by 7 days without treatment
  • Repeat this 2-week treatment cycle 6 times, for a total treatment period of about 12 weeks
  • Have study visits during treatment and follow-up visits through week 52
  • Have safety checks, lung function tests, chest HRCT scans, blood tests, and symptom assessments
  • Record symptoms, adverse events, and any rescue treatment in a study diary

연구 유형

중재적

등록 (추정된)

18

단계

  • 초기 1단계

연락처 및 위치

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연구 연락처

연구 연락처 백업

연구 장소

      • Beijing, 중국
        • Peking Union Medical College Hospital
        • 연락하다:

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. Age 18 to 80 years, inclusive, regardless of sex.
  2. Good compliance and willingness to receive study treatment and complete follow-up visits and assessments as required by the protocol.
  3. For participants with IgG4-related disease:

    • Diagnosis of IgG4-related disease for at least 6 months, according to the 2019 ACR/EULAR Classification Criteria or the 2020 Revised Comprehensive Diagnostic Criteria for IgG4-related Disease.
    • Evidence of IgG4-related lung involvement, including but not limited to bronchial wall thickening, bronchovascular bundle thickening, pulmonary nodules, or interstitial lung disease on chest high-resolution computed tomography (HRCT).
  4. For participants with systemic sclerosis:

    • Diagnosis of systemic sclerosis for at least 6 months, according to the 2013 ACR/EULAR Classification Criteria.
    • Evidence of lung involvement on chest HRCT, including bronchial involvement, pulmonary nodules considered related to the underlying disease, and/or interstitial lung disease, with or without impaired pulmonary function.
  5. Able to understand the study purpose, procedures, and possible discomforts, and willing to provide written informed consent.
  6. Female participants of childbearing potential and male participants, including their female partners, must use highly effective contraception from screening until at least 6 months after the last dose. Participants must have no plan for pregnancy, sperm donation, or egg donation from screening until at least 6 months after the last dose.

Exclusion Criteria:

  1. Diagnosis before screening of any connective tissue disease other than IgG4-related disease or systemic sclerosis, or diagnosis of another interstitial lung disease, such as idiopathic pulmonary fibrosis or interstitial pneumonia with autoimmune features.
  2. Inadequate organ function reserve, including any of the following:

    • Respiratory system: pulmonary arterial hypertension with pulmonary artery systolic pressure greater than 55 mmHg by echocardiography or mean pulmonary arterial pressure greater than 40 mmHg by right heart catheterization; severe impairment of diffusing capacity of the lung for carbon monoxide, defined as DLCO less than 30% of predicted; respiratory failure, defined as arterial oxygen tension less than 8 kPa or less than 60 mmHg and/or arterial carbon dioxide tension greater than 6.7 kPa or greater than 50 mmHg at rest without oxygen supplementation.
    • Renal function: creatinine clearance less than 30 mL/min/1.73 m².
    • Cardiac function: clinical symptoms of refractory congestive heart failure; left ventricular ejection fraction less than 35% by myocardial scintigraphy or echocardiography; chronic atrial fibrillation requiring oral anticoagulation; uncontrolled ventricular arrhythmia; or pericardial effusion causing hemodynamic instability by echocardiography.
    • Liver function: persistent alanine aminotransferase, aspartate aminotransferase, or bilirubin greater than 3 times the upper limit of normal, or severe hepatic impairment, Child-Pugh class C.
  3. Allergic constitution or history of potentially life-threatening drug allergy.
  4. Clinically significant chronic or recurrent infection, defined as 3 or more infections of the same type within 1 year, or recent severe infection, such as pneumonia or sepsis. Participants will also be excluded if, within 1 month before baseline, they required inhaled, intramuscular, or systemic antibiotic treatment, systemic antiviral treatment, hospitalization, or prolonged hospitalization for infection.
  5. History of organ transplantation or currently awaiting organ transplantation.
  6. Positive screening test results for hepatitis B, hepatitis C, human immunodeficiency virus, or syphilis, as defined below:

    • Hepatitis B surface antigen positive.
    • Hepatitis B surface antigen negative but hepatitis B core antibody positive, with hepatitis B virus DNA above the upper limit of normal.
    • Hepatitis C virus antibody positive, with hepatitis C virus RNA above the upper limit of normal.
    • Human immunodeficiency virus antibody positive.
    • Treponema pallidum antibody positive with positive rapid plasma reagin or toluidine red unheated serum test.
  7. Possible active tuberculosis infection based on interferon-gamma release assay, such as QuantiFERON-TB Gold or T-SPOT.TB, clinical symptoms, and/or chest imaging. Participants with evidence of previously active tuberculosis that has been adequately treated may be enrolled after investigator assessment. Participants with latent tuberculosis may continue screening or be enrolled after at least 4 weeks of anti-tuberculosis treatment, if there is no liver injury before baseline and the investigator determines that the risk is controlled.
  8. Prior treatment with stem cells or extracellular vesicles.
  9. Any documented active or suspected malignancy, or history of malignancy, within 5 years before screening, except adequately treated basal cell carcinoma of the skin, squamous cell carcinoma in situ of the skin, or carcinoma in situ of the cervix.
  10. Self-harm or suicidal ideation within 1 year before screening.
  11. History of alcohol or drug abuse within 6 months before screening.
  12. Major surgery or clinically significant surgery within 6 months before screening, or planned major surgery during the study.
  13. Any of the following cardiovascular or cerebrovascular events within 6 months before screening: unstable angina requiring hospitalization, myocardial infarction, coronary artery bypass grafting, percutaneous coronary intervention, moderate to severe congestive heart failure defined as New York Heart Association class III or IV, atrial or ventricular arrhythmia requiring hospitalization, pacemaker or defibrillator implantation, cerebrovascular accident such as stroke, or planned coronary artery bypass grafting or revascularization during the study.
  14. History of psychiatric illness, epilepsy, or other central nervous system disease.
  15. Severe systemic disease, including but not limited to myocardial infarction, unstable angina, heart failure, liver cirrhosis, or acute glomerulonephritis.
  16. Participation in any other clinical trial within 3 months before screening or within 5 half-lives of the investigational product, whichever is longer.
  17. Pregnancy or breastfeeding.
  18. Poor compliance or inability to complete study procedures.
  19. Any condition that, in the investigator's opinion, may increase participant risk or interfere with interpretation of study results.

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: 순차적 할당
  • 마스킹: 없음(오픈 라벨)

무기와 개입

참가자 그룹 / 팔
개입 / 치료
실험적: UC-EVs arm

This open-label, dose-escalation study with up to three dose levels of nebulized UC-EVs. The maximum tolerated dose (MTD) of nebulized UC-EVs will be determined using dose-escalation 3+3 design.

Dose Level 1: 5×10^9 particles

Dose Level 2: 1×10^10 particles

Dose Level 3: 2×10^10 particles

UC-EVs are extracellular vesicles derived from human umbilical cord mesenchymal stromal cells. Participants will receive UC-EVs by nebulized inhalation once daily for 7 consecutive days, followed by 7 days without treatment. Each 14-day cycle will be repeated for 6 cycles over approximately 12 weeks.
다른 이름들:
  • UC-EVs

연구는 무엇을 측정합니까?

주요 결과 측정

결과 측정
측정값 설명
기간
Incidence and Severity of Adverse Events and Serious Adverse Events
기간: From first dose through Week 52
The incidence and severity of adverse events (AEs) and serious adverse events (SAEs) will be assessed in participants who receive at least one dose of nebulized UC-EVs. AEs and SAEs will be collected throughout the study and graded according to the protocol-defined safety assessment criteria.
From first dose through Week 52
Maximum Tolerated Dose and Recommended Phase 2 Dose of Nebulized UC-EVs
기간: Through the first 14-day treatment cycle for each dose cohort
The maximum tolerated dose (MTD), if determinable, and the recommended Phase 2 dose (RP2D) of nebulized UC-EVs will be determined based on dose-limiting toxicities (DLTs), overall safety and tolerability, and available clinical and laboratory safety data during the dose-escalation phase.
Through the first 14-day treatment cycle for each dose cohort

2차 결과 측정

결과 측정
측정값 설명
기간
Change From Baseline in Health Assessment Questionnaire Score
기간: Baseline through Week 52
Absolute change from baseline in Health Assessment Questionnaire (HAQ) score during the study. HAQ is a participant-reported outcome measure used to assess physical function and daily activity. The total score ranges from 0 to 3, with higher scores indicating worse physical function and greater difficulty in daily activities. A decrease from baseline indicates improvement.
Baseline through Week 52
Change From Baseline in Patient Global Impression of Severity Score
기간: Baseline through Week 52
Absolute change from baseline in Patient Global Impression of Severity (PGI-S) score during the study. PGI-S is used to assess the participant's overall perception of disease severity. The score ranges from 0 to 7, with higher scores indicating more severe disease symptoms. A decrease from baseline indicates improvement.
Baseline through Week 52
Change From Baseline in St. George's Respiratory Questionnaire Score
기간: Baseline through Week 52
Absolute change from baseline in St. George's Respiratory Questionnaire (SGRQ) score during the study. SGRQ is a participant-reported outcome measure used to assess respiratory symptoms, activity limitation, and disease impact. The total score ranges from 0 to 100, with higher scores indicating worse respiratory health status. A decrease from baseline indicates improvement.
Baseline through Week 52
Change From Baseline in Leicester Cough Questionnaire Score
기간: Baseline through Week 52
Absolute change from baseline in Leicester Cough Questionnaire (LCQ) score during the study. LCQ is a participant-reported outcome measure used to assess cough-related quality of life. The total score ranges from 3 to 21, with higher scores indicating better cough-related quality of life. An increase from baseline indicates improvement.
Baseline through Week 52
Change From Baseline in Forced Vital Capacity at Week 24
기간: Baseline to Week 24
Absolute change from baseline in forced vital capacity (FVC), measured in milliliters, at Week 24.
Baseline to Week 24
Percent Change From Baseline in Percent Predicted Diffusing Capacity of the Lung for Carbon Monoxide at Week 24
기간: Baseline to Week 24
Percent change from baseline in percent predicted single-breath diffusing capacity of the lung for carbon monoxide (DLCO SB % predicted) at Week 24.
Baseline to Week 24
Change From Baseline in Maximum Pulmonary Nodule Size on Chest HRCT at Week 24
기간: Baseline to Week 24
For participants with pulmonary nodules at baseline, absolute change from baseline in the size of the largest pulmonary nodule on chest high-resolution computed tomography (HRCT), measured in millimeters, at Week 24.
Baseline to Week 24
Change From Baseline in Bronchial Wall Thickness on Chest HRCT at Week 24
기간: Baseline to Week 24
For participants with bronchial wall thickening at baseline, absolute change from baseline in bronchial wall thickness on chest HRCT, measured in millimeters, at Week 24.
Baseline to Week 24
Change From Baseline in Quantitative Lung Fibrosis and Quantitative Ground-Glass Opacity Scores on Chest HRCT at Week 24
기간: Baseline to Week 24
For participants with interstitial lung disease at baseline, absolute change from baseline in quantitative lung fibrosis (QLF) score and quantitative ground-glass opacity (QGGO) score on chest HRCT at Week 24. Scores are expressed as percentages.
Baseline to Week 24
Change From Baseline in Quantitative Interstitial Lung Disease Score on Chest HRCT at Week 24
기간: Baseline to Week 24
For participants with interstitial lung disease at baseline, absolute change from baseline in quantitative interstitial lung disease (QILD) score on chest HRCT at Week 24. The score is expressed as a percentage.
Baseline to Week 24
Change From Baseline in Disease-Related Serologic Markers (for IgG4-RD patients only)
기간: Baseline through Week 52
Absolute change from baseline in serum IgG4 level (mg/L) for participants with IgG4-related disease.
Baseline through Week 52
Change From Baseline in Disease-Related Serologic Markers (for SSc patients only)
기간: Baseline through Week 52
Absolute change from baseline in systemic sclerosis-related autoantibody titers (U/mL) for participants with systemic sclerosis.
Baseline through Week 52
Change From Baseline in erythrocyte sedimentation rate
기간: Baseline through Week 52
Absolute change from baseline in erythrocyte sedimentation rate (ESR) (mm/h) level during the study.
Baseline through Week 52
Change From Baseline in C-reactive protein
기간: Baseline through Week 52
Absolute change from baseline in C-reactive protein (CRP) (mg/L) levels during the study.
Baseline through Week 52

기타 결과 측정

결과 측정
측정값 설명
기간
Change From Baseline in Bronchoalveolar Lavage Fluid Cellular and Cytokine Profiles
기간: Baseline and Week 12
In participants who voluntarily undergo bronchoscopy and bronchoalveolar lavage fluid (BALF) collection, changes from baseline in BALF cellular characteristics and key inflammatory or immune-related cytokine levels will be assessed to explore local lung inflammation and immune microenvironment changes after UC-EV treatment.
Baseline and Week 12

공동 작업자 및 조사자

여기에서 이 연구와 관련된 사람과 조직을 찾을 수 있습니다.

스폰서

수사관

  • 수석 연구원: Qiubai Li, MD, Department of Rheumatology, Wuhan Union Hospital, Tongji Medical College of Huazhong University of Science and Technology

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 10월 14일

기본 완료 (추정된)

2028년 7월 30일

연구 완료 (추정된)

2029년 7월 30일

연구 등록 날짜

최초 제출

2026년 8월 29일

QC 기준을 충족하는 최초 제출

2026년 9월 4일

처음 게시됨 (실제)

2026년 9월 8일

연구 기록 업데이트

마지막 업데이트 게시됨 (실제)

2026년 9월 8일

QC 기준을 충족하는 마지막 업데이트 제출

2026년 9월 4일

마지막으로 확인됨

2026년 7월 1일

추가 정보

이 연구와 관련된 용어

개별 참가자 데이터(IPD) 계획

개별 참가자 데이터(IPD)를 공유할 계획입니까?

아니요

IPD 계획 설명

Individual participant data will not be publicly shared because this is an early-phase, small-sample study involving patients with rare immune-mediated inflammatory diseases and detailed clinical, imaging, laboratory, safety, and exploratory biomarker data. Even after de-identification, there may remain a risk of participant re-identification. De-identified aggregate data will be reported in publications and/or trial registry results as appropriate.

약물 및 장치 정보, 연구 문서

미국 FDA 규제 의약품 연구

아니

미국 FDA 규제 기기 제품 연구

아니

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. .

구독하다