ClinicalTrials.gov record NCT07806045
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
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AI-written from the registry record, not the study's own wording. May contain errors or omissions. Check the registry text on this page and the original record ↗ before acting on it.
This Phase I, open-label, single-arm dose-escalation study tests nebulized human umbilical cord mesenchymal stromal cell‑derived extracellular vesicles (UC‑EVs) in adults with recurrent or refractory immune‑mediated inflammatory diseases (IMIDs) that involve the lungs, specifically IgG4‑related disease and systemic sclerosis. Participants inhale UC‑EVs daily in 7‑day on / 7‑day off cycles repeated for six 14‑day cycles (about 12 weeks) and are followed for safety through Week 52. The trial aims to find the maximum tolerated dose and collect early data on lung function, imaging, symptoms, and laboratory markers.
What it is studying. Whether nebulized UC‑EVs are safe and tolerable in adults with recurrent or refractory IMIDs with lung involvement, and to collect early information on possible effects on lung function, chest imaging, symptoms, and disease‑related laboratory tests.
What the researchers hope to learn. Determine the safety and tolerability of nebulized UC‑EVs, identify the maximum tolerated dose and a recommended Phase 2 dose, and collect early clinical and laboratory data on lung function, chest imaging, symptoms, and disease‑related biomarkers in people with IgG4‑related disease or systemic sclerosis with lung involvement.
What this study is about
The study's own summary, as published on ClinicalTrials.gov. Underlined terms have a short explanation.
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.
Interventions being studied
Being studied in this trial does not mean the treatment or procedure is approved or available for this condition.
- Biological
- nebulized human umbilical cord mesenchymal stromal cell-derived extracellular vesicles
Eligibility
The criteria exactly as listed on ClinicalTrials.gov. Only the study team can say whether they apply to you.
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.
Study description and what participation involves
The study's detailed description, as published on ClinicalTrials.gov.
Detailed description from the registry
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
AI summaryWhat participation may involve, in plain languageShow ▾Hide ▴
AI-written from the registry record, not the study's own wording. May contain errors or omissions. Check the registry text on this page and the original record ↗ before acting on it.
Participants will inhale nebulized UC-EVs once daily for 7 days followed by 7 days without treatment; this 14-day cycle is repeated six times (about 12 weeks). Treatment is given in a dose-escalation design with up to three dose levels (3+3). You will have study visits during treatment and follow-up visits through Week 52 for safety checks, lung function tests, chest high-resolution CT (HRCT) scans, blood tests, and symptom assessments. You will also keep a study diary to record symptoms, adverse events, and any rescue treatments.
How the study is structured. Interventional, open‑label, single‑arm dose‑escalation study (3+3 design) with no masking. Up to three dose levels of nebulized UC‑EVs are tested (5×10^9, 1×10^10, and 2×10^10 particles). Participants inhale UC‑EVs once daily for seven consecutive days followed by seven days without treatment; each 14‑day cycle is repeated six times (~12 weeks). Safety checks, lung function tests, chest HRCT, blood tests, symptom assessments, and study diaries are used; participants are followed through Week 52.
Questions you could ask
These questions are suggestions for a conversation with the study team or your own clinician, not advice.
- This is an early-phase study, mainly about safety and dose. What is already known about this treatment?
- The registry says recruitment has not started. When is it expected to open, and can I register interest now?
- The listed sites are in China. Is any of them reachable for me, and does the study help with travel?
- Which of the eligibility criteria would need to be checked with tests before I could join?
More on IgG4-related disease
Important dates
- Study start
- Oct 14, 2026 (estimated)
- Primary completion
- Jul 30, 2028 (estimated)
- Study completion
- Jul 30, 2029 (estimated)
- Target enrollment
- 18 participants (estimated)
- Last updated on registry
- Sep 8, 2026
- Data verified
- Oct 10, 2026
Sponsor and research team
The lead sponsor funds and oversees the trial. A pharmaceutical or biotech company sponsor typically means the study is testing that company's drug or device; a hospital, university, or government agency usually indicates investigator-initiated or publicly funded research.
- Lead sponsor
- Qiubai Li
- Collaborators
- Peking Union Medical College Hospital
- Shandong Qilu Stem Cells Engineering Co., Ltd.
Central study contact
If you're interested in participating, the central contact can answer general questions about the study — including eligibility requirements and how to find a recruiting site near you. Actual eligibility is determined by the site's medical team.
- Qiubai Li, MD 📞 +86 13995671635 ✉ qiubaili@hust.edu.cn
- Jingna Li, MD 📞 +86 19801116153 ✉ lijingna0615@126.com
Participating sites (1)
- Peking Union Medical College Hospital
Beijing · China
- Wen Zhang, MD · +86 13661131264 · zhangwen91@sina.com
Terms used on this page · explanations by MedicalUpdates.ai
- refractory
- A condition that has not responded to a treatment. 'Relapsed' means it responded and then came back; 'refractory' means it did not respond in the first place.
- follow-up
- The period after treatment or enrolment during which participants are observed or contacted, for example to record side effects or outcomes.
- computed tomography
- An X-ray scan that builds detailed cross-section images of the body. 'High-resolution' CT (HRCT) is used to look closely at the lungs.
- written informed consent
- The process of explaining a study, including its risks and what participation involves, so that a person can decide freely whether to take part. Consent is usually documented in writing, and a participant can choose to leave the study later.
- childbearing potential
- Studies often ask participants who could become pregnant, or could father a child, to use contraception during the study and for a period afterwards, because the effect of the treatment on a pregnancy is unknown.
- before screening
- The checks done before enrolment to see whether a person qualifies, such as tests, scans or a review of medical history.
- echocardiography
- An ultrasound scan of the heart. A transesophageal echocardiogram is done through a probe passed into the food pipe for a closer view.
- diffusing capacity
- A breathing test that measures how well the lungs pass oxygen from the air into the blood.
- creatinine clearance
- A measure of kidney function, calculated from a blood creatinine level together with age, weight and sex.
- upper limit of normal
- The highest value of a blood test that a laboratory considers normal. Criteria such as '≤ 3 × ULN' mean the result must be no more than three times that value.
- baseline
- Measurements taken at the start of the study, before any study treatment, so that later results can be compared with them.
- Prior treatment
- Treatment a person has already had for the condition. A 'line of therapy' is one course of treatment; studies often require, or limit, the number of prior lines.
- 5 half-lives
- The time it takes for the amount of a drug in the body to fall by half. 'Five half-lives' is often used as the point at which a previous drug has essentially left the body.
- investigational product
- A treatment that is being tested and has not been approved for this use. It may be approved for a different condition. Being studied does not mean it works or is available outside the study.
- Inclusion Criteria
- The conditions a person must meet to be considered for the study, for example a confirmed diagnosis, an age range or a test result.
- Exclusion Criteria
- The conditions that rule a person out even if they meet the inclusion criteria, for example other illnesses, certain medicines or pregnancy.
- open-label
- No masking is used, so the assigned intervention is not deliberately hidden from participants or study staff.
- single-arm
- All participants receive the same treatment or follow the same plan. There is no comparison group within the study.
- dose-escalation
- Groups of participants receive increasing doses of a treatment, one group after another, to find a dose that can be given safely.
- eligibility criteria
- The rules the study team uses to decide who may take part. They are usually split into inclusion criteria and exclusion criteria.
- adverse events
- Any unwanted medical problem that happens during a study, whether or not it was caused by the treatment. A 'serious' adverse event is one that meets specific criteria, such as causing death, being life-threatening, requiring or prolonging hospital admission, causing significant disability, or another medically important outcome.
The study record on this page is reproduced from ClinicalTrials.gov, a database of the U.S. National Library of Medicine, where it was submitted by the study sponsor. MedicalUpdates.ai does not own this content, is not affiliated with or endorsed by ClinicalTrials.gov, NIH, or the U.S. Government, and does not verify the record. Plain-language notes, the glossary and the questions above are added by this site; text in the boxes marked "AI summary" was written by an AI model and may contain errors. Registry records change over time. Check the original record for the current version and discuss eligibility or participation with a qualified healthcare professional.
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