Nebulized UC-EVs for Recurrent or Refractory IMIDs With Lung Involvement
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.
研究概览
地位
详细说明
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
研究类型
注册 (估计的)
阶段
- 第一阶段早期
联系人和位置
学习联系方式
- 姓名:Qiubai Li, MD
- 电话号码:+86 13995671635
- 邮箱:qiubaili@hust.edu.cn
研究联系人备份
- 姓名:Jingna Li, MD
- 电话号码:+86 19801116153
- 邮箱:lijingna0615@126.com
学习地点
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Beijing、中国
- Peking Union Medical College Hospital
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接触:
- Wen Zhang, MD
- 电话号码:+86 13661131264
- 邮箱:zhangwen91@sina.com
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参与标准
资格标准
适合学习的年龄
- 成人
- 年长者
接受健康志愿者
描述
Inclusion Criteria:
- Age 18 to 80 years, inclusive, regardless of sex.
- Good compliance and willingness to receive study treatment and complete follow-up visits and assessments as required by the protocol.
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).
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.
- Able to understand the study purpose, procedures, and possible discomforts, and willing to provide written informed consent.
- 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:
- 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.
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.
- Allergic constitution or history of potentially life-threatening drug allergy.
- 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.
- History of organ transplantation or currently awaiting organ transplantation.
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.
- 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.
- Prior treatment with stem cells or extracellular vesicles.
- 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.
- Self-harm or suicidal ideation within 1 year before screening.
- History of alcohol or drug abuse within 6 months before screening.
- Major surgery or clinically significant surgery within 6 months before screening, or planned major surgery during the study.
- 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.
- History of psychiatric illness, epilepsy, or other central nervous system disease.
- Severe systemic disease, including but not limited to myocardial infarction, unstable angina, heart failure, liver cirrhosis, or acute glomerulonephritis.
- Participation in any other clinical trial within 3 months before screening or within 5 half-lives of the investigational product, whichever is longer.
- Pregnancy or breastfeeding.
- Poor compliance or inability to complete study procedures.
- Any condition that, in the investigator's opinion, may increase participant risk or interfere with interpretation of study results.
学习计划
研究是如何设计的?
设计细节
- 主要用途:治疗
- 分配:不适用
- 介入模型:顺序分配
- 屏蔽:无(打开标签)
武器和干预
参与者组/臂 |
干预/治疗 |
|---|---|
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实验性的: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.
其他名称:
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研究衡量的是什么?
主要结果指标
结果测量 |
措施说明 |
大体时间 |
|---|---|---|
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Incidence and Severity of Adverse Events and Serious Adverse Events
大体时间:From first dose through Week 52
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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.
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From first dose through Week 52
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Maximum Tolerated Dose and Recommended Phase 2 Dose of Nebulized UC-EVs
大体时间:Through the first 14-day treatment cycle for each dose cohort
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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.
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Through the first 14-day treatment cycle for each dose cohort
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次要结果测量
结果测量 |
措施说明 |
大体时间 |
|---|---|---|
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Change From Baseline in Health Assessment Questionnaire Score
大体时间:Baseline through Week 52
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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.
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Baseline through Week 52
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Change From Baseline in Patient Global Impression of Severity Score
大体时间:Baseline through Week 52
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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.
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Baseline through Week 52
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Change From Baseline in St. George's Respiratory Questionnaire Score
大体时间:Baseline through Week 52
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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.
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Baseline through Week 52
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Change From Baseline in Leicester Cough Questionnaire Score
大体时间:Baseline through Week 52
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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.
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Baseline through Week 52
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Change From Baseline in Forced Vital Capacity at Week 24
大体时间:Baseline to Week 24
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Absolute change from baseline in forced vital capacity (FVC), measured in milliliters, at Week 24.
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Baseline to Week 24
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Percent Change From Baseline in Percent Predicted Diffusing Capacity of the Lung for Carbon Monoxide at Week 24
大体时间:Baseline to Week 24
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Percent change from baseline in percent predicted single-breath diffusing capacity of the lung for carbon monoxide (DLCO SB % predicted) at Week 24.
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Baseline to Week 24
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Change From Baseline in Maximum Pulmonary Nodule Size on Chest HRCT at Week 24
大体时间:Baseline to Week 24
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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.
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Baseline to Week 24
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Change From Baseline in Bronchial Wall Thickness on Chest HRCT at Week 24
大体时间:Baseline to Week 24
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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.
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Baseline to Week 24
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Change From Baseline in Quantitative Lung Fibrosis and Quantitative Ground-Glass Opacity Scores on Chest HRCT at Week 24
大体时间:Baseline to Week 24
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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.
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Baseline to Week 24
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Change From Baseline in Quantitative Interstitial Lung Disease Score on Chest HRCT at Week 24
大体时间:Baseline to Week 24
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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.
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Baseline to Week 24
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Change From Baseline in Disease-Related Serologic Markers (for IgG4-RD patients only)
大体时间:Baseline through Week 52
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Absolute change from baseline in serum IgG4 level (mg/L) for participants with IgG4-related disease.
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Baseline through Week 52
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Change From Baseline in Disease-Related Serologic Markers (for SSc patients only)
大体时间:Baseline through Week 52
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Absolute change from baseline in systemic sclerosis-related autoantibody titers (U/mL) for participants with systemic sclerosis.
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Baseline through Week 52
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Change From Baseline in erythrocyte sedimentation rate
大体时间:Baseline through Week 52
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Absolute change from baseline in erythrocyte sedimentation rate (ESR) (mm/h) level during the study.
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Baseline through Week 52
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Change From Baseline in C-reactive protein
大体时间:Baseline through Week 52
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Absolute change from baseline in C-reactive protein (CRP) (mg/L) levels during the study.
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Baseline through Week 52
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其他结果措施
结果测量 |
措施说明 |
大体时间 |
|---|---|---|
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Change From Baseline in Bronchoalveolar Lavage Fluid Cellular and Cytokine Profiles
大体时间:Baseline and Week 12
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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.
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Baseline and Week 12
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合作者和调查者
赞助
调查人员
- 首席研究员:Qiubai Li, MD、Department of Rheumatology, Wuhan Union Hospital, Tongji Medical College of Huazhong University of Science and Technology
研究记录日期
研究主要日期
学习开始 (估计的)
初级完成 (估计的)
研究完成 (估计的)
研究注册日期
首次提交
首先提交符合 QC 标准的
首次发布 (实际的)
研究记录更新
最后更新发布 (实际的)
上次提交的符合 QC 标准的更新
最后验证
更多信息
与本研究相关的术语
其他研究编号
- UCEV-IMID 2.0
计划个人参与者数据 (IPD)
计划共享个人参与者数据 (IPD)?
IPD 计划说明
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