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This is an exploratory, randomized, single-blind, placebo-controlled, multiple-dose, dose-escalation clinical study. The study aims to evaluate the safety and preliminary efficacy of nebulized inhalation of Human Umbilical Cord Mesenchymal Stem Cell-Derived Extracellular Vesicles (hUCMSC-EVs) in patients with interstitial lung disease (ILD).
This study is designed to include three sequential dose cohorts-low, medium, and high-with dose escalation proceeding in an ascending order. Eligible subjects will be enrolled and randomized sequentially, with a planned enrollment of 8 subjects per cohort. Within each cohort, subjects will be randomly assigned in a 3:1 ratio to receive either hUCMSC-EVs or saline placebo.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Low-dose group | Experimental |
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| Moderate-dose group | Experimental |
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| High-dose group | Experimental |
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| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Human Umbilical Cord Mesenchymal Stem Cell-Derived Extracellular Vesicles (hUCMSC-EVs) | Biological | hUCMSC-EVs , nebulized BID × 7d/cycle, 3 cycles |
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| Measure | Description | Time Frame |
|---|---|---|
| Incidence and Severity of Adverse Events | From first dose to Week 12 |
| Measure | Description | Time Frame |
|---|---|---|
| Change from Baseline in Forced Vital Capacity (FVC) | Forced Vital Capacity (FVC) will be assessed using standardized spirometry in accordance with American Thoracic Society (ATS)/European Respiratory Society (ERS) guidelines. Measurements will be performed at Baseline (prior to first dose) and at scheduled post-baseline visits. Spirometry will be conducted using a calibrated spirometer. Results will be summarized as absolute change from baseline and percent predicted normal values. |
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Key Inclusion Criteria:
Key Exclusion Criteria:
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| Name | Role | Phone | Extension | |
|---|---|---|---|---|
| Tong, M.D | Contact | 86-13764089607 | tong.lin@zs-hospital.sh.cn |
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| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Zhongshan Hospital, Fudan University | Shanghai | China |
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| ID | Term |
|---|---|
| D017563 | Lung Diseases, Interstitial |
| ID | Term |
|---|---|
| D008171 | Lung Diseases |
| D012140 | Respiratory Tract Diseases |
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The study is designed as a dose-escalation trial comprising three sequential dose cohorts: low-dose, medium-dose, and high-dose. Escalation will proceed from the lowest to the highest dose level. Within each cohort, subjects will be randomized in a 3:1 ratio to receive either investigational product or placebo.
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| Normal Saline Placebo | Biological | Nebulized inhalation BID × 7d per cycle, 3 cycles |
|
| From first dose to Week 24 |
| Change from Baseline in Diffusing Capacity of the Lung for Carbon Monoxide (DLCO) | Diffusing Capacity of the Lung for Carbon Monoxide (DLCO) will be assessed using the single-breath method in accordance with ATS/ERS guidelines. Hemoglobin-corrected DLCO values will be recorded if applicable. Measurements will be performed at Baseline and at scheduled post-baseline visits. Results will be summarized as absolute change from baseline and percent predicted normal values. | From first dose to Week 24 |
| Change from Baseline in 6-Minute Walk Test (6MWT) Distance | From first dose to Week 24 |
| Change from Baseline in St. George's Respiratory Questionnaire (SGRQ) Total Score | The St. George's Respiratory Questionnaire (SGRQ) is a disease-specific instrument designed to measure health-related quality of life in patients with chronic respiratory diseases. The Total Score is calculated as a weighted sum of the domain scores and ranges from 0 to 100, with higher scores indicating worse health-related quality of life. A change of 4 units is generally considered the minimal clinically important difference (MCID). | From first dose to Week 24 |