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IgG4 Related DiseaseEarly Phase 1NCT07806045

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

A Early Phase 1 study testing nebulized human umbilical cord mesenchymal stromal cell-derived extracellular vesicles in people with IgG4 Related Disease and Systemic Sclerosis (SSc), run by Qiubai Li at 1 site in China.

Not yet recruiting

Opening soon. The study is approved but enrolment has not started yet. Sites may already take enquiries.

Sites and contacts
Official record
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Trial summary

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). Read more about this study

Who is it for?
Ages 18 to 80 years
What is being tested?
nebulized human umbilical cord mesenchymal stromal cell-derived extracellular vesicles
Could I receive a placebo?
No, everyone receives an active treatment
How many people
18 participants
Where
1 siteChina
Study dates
14 Oct 2026to 30 Jul 2029
Phase
Early Phase 1
Are there results?
Not yet: the study is under way

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 18 to 80 years
  • All sexes
  • No healthy volunteers
  • Adult, Older adult

Who may be able to join

  • 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:
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  • 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.

Who cannot join

  • 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.
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  • 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.

Trial locations and contacts

1 site in 1 country. Contact the study team or a site near you to ask about taking part.

China1 site
  • Peking Union Medical College Hospital

    Beijing

    Wen Zhang, MD, +86 13661131264, zhangwen91@sina.com

Other trials in China

About this study

Official title: 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.

Read the detailed description

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
Being tested
nebulized human umbilical cord mesenchymal stromal cell-derived extracellular vesicles
Keywords
IgG4 Related Disease, Systemic Sclerosis (SSc), Extracellular Vescicles, Umbilial Cord Mesenchymal Stromal Cell
Medical subject headings
Immunoglobulin G4-Related Disease, Scleroderma, Systemic

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • nebulized human umbilical cord mesenchymal stromal cell-derived extracellular vesiclesBiological

    Also called UC-EVs

    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.

Study groups in NCT07806045
Study groupRoleWhat they receive
UC-EVs armExperimentalThis 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
Receives: Biological: nebulized human umbilical cord mesenchymal stromal cell-derived extracellular vesicles

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Incidence and Severity of Adverse Events and Serious Adverse Events

    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.

    Measured: From first dose through Week 52

  2. Maximum Tolerated Dose and Recommended Phase 2 Dose of Nebulized UC-EVs

    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.

    Measured: Through the first 14-day treatment cycle for each dose cohort

Secondary outcomes (14)
  1. Change From Baseline in Health Assessment Questionnaire Score

    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.

    Measured: Baseline through Week 52

  2. Change From Baseline in Patient Global Impression of Severity Score

    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.

    Measured: Baseline through Week 52

  3. Change From Baseline in St. George's Respiratory Questionnaire Score

    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.

    Measured: Baseline through Week 52

  4. Change From Baseline in Leicester Cough Questionnaire Score

    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.

    Measured: Baseline through Week 52

  5. Change From Baseline in Forced Vital Capacity at Week 24

    Absolute change from baseline in forced vital capacity (FVC), measured in milliliters, at Week 24.

    Measured: Baseline to Week 24

  6. Percent Change From Baseline in Percent Predicted Diffusing Capacity of the Lung for Carbon Monoxide at 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.

    Measured: Baseline to Week 24

  7. Change From Baseline in Maximum Pulmonary Nodule Size on Chest HRCT at 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.

    Measured: Baseline to Week 24

  8. Change From Baseline in Bronchial Wall Thickness on Chest HRCT at 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.

    Measured: Baseline to Week 24

  9. Change From Baseline in Quantitative Lung Fibrosis and Quantitative Ground-Glass Opacity Scores on Chest HRCT at 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.

    Measured: Baseline to Week 24

  10. Change From Baseline in Quantitative Interstitial Lung Disease Score on Chest HRCT at 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.

    Measured: Baseline to Week 24

  11. Change From Baseline in Disease-Related Serologic Markers (for IgG4-RD patients only)

    Absolute change from baseline in serum IgG4 level (mg/L) for participants with IgG4-related disease.

    Measured: Baseline through Week 52

  12. Change From Baseline in Disease-Related Serologic Markers (for SSc patients only)

    Absolute change from baseline in systemic sclerosis-related autoantibody titers (U/mL) for participants with systemic sclerosis.

    Measured: Baseline through Week 52

  13. Change From Baseline in erythrocyte sedimentation rate

    Absolute change from baseline in erythrocyte sedimentation rate (ESR) (mm/h) level during the study.

    Measured: Baseline through Week 52

  14. Change From Baseline in C-reactive protein

    Absolute change from baseline in C-reactive protein (CRP) (mg/L) levels during the study.

    Measured: Baseline through Week 52

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted8 Sep 2026
  2. Study start14 Oct 2026 (estimated)
  3. Primary completion30 Jul 2028 (estimated)
  4. Study completion30 Jul 2029 (estimated)

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Early Phase 1
Primary purpose
Treatment
Allocation
Not applicable
Intervention model
Sequential
Masking
None (open label)
Enrolment
18 participants (estimated)
IDs, dates and publications
ClinicalTrials.gov ID
NCT07806045
Sponsor's protocol ID
UCEV-IMID 2.0
Status last verified
Jul 2026
First submitted
29 Aug 2026
Last updated
8 Sep 2026
Results posted
No
Data monitoring committee
Yes
FDA-regulated drug
No
FDA-regulated device
No
Individual data sharing
No: 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.

Questions about this trial

What is this clinical trial studying?

A Early Phase 1 study testing nebulized human umbilical cord mesenchymal stromal cell-derived extracellular vesicles in people with IgG4 Related Disease and Systemic Sclerosis (SSc), run by Qiubai Li at 1 site in China.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 10 inclusion and 28 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: opening soon, as of the sponsor's last update on 8 Sep 2026. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 1 site in China. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Early Phase 1 (sometimes called Phase 0) studies give a very small amount of a treatment to a few people, mainly to see how the body handles it.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

This study is still under way. Results are not available in the source record yet.

How can a doctor refer a patient or contact the research team?

Contact Qiubai Li, MD (+86 13995671635, qiubaili@hust.edu.cn), or a site near the patient. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Incidence and Severity of Adverse Events and Serious Adverse Events (time frame: From first dose through Week 52). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.

Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 8 Sep 2026.

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