Interstitial Lung Disease (ILD)
Conditions
Brief summary
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.
Interventions
BIOLOGICALHuman Umbilical Cord Mesenchymal Stem Cell-Derived Extracellular Vesicles (hUCMSC-EVs)
hUCMSC-EVs , nebulized BID × 7d/cycle, 3 cycles
BIOLOGICALNormal Saline Placebo
Nebulized inhalation BID × 7d per cycle, 3 cycles
Sponsors
Shanghai Zhongshan Hospital
Study design
Allocation
RANDOMIZED
Intervention model
SEQUENTIAL
Primary purpose
TREATMENT
Masking
SINGLE (Subject)
Intervention model description
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.
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
No
Inclusion criteria
Key Inclusion Criteria: * Age 18 years and older, regardless of gender. * Diagnosed with interstitial lung disease (ILD). * High-resolution computed tomography (HRCT) findings within 3 months prior to randomization must be consistent with interstitial pulmonary imaging changes. * Pulmonary function test within the protocol-defined acceptable range prior to randomization. * Good compliance, able to understand and cooperate with the required examinations and procedures, and willing to attend follow-up visits as scheduled. * Voluntary participation and signing of written informed consent form. Key
Exclusion criteria
* Inability to tolerate nebulized inhalation therapy. * Requirement for oxygen supplementation \> 15 hours per day during the screening period. * Allergic diathesis or a history of life-threatening drug hypersensitivity. * History of malignancy within 5 years. * Active hepatitis B, hepatitis C, or HIV infection. * Active systemic infection requiring antibiotic therapy. * Clinically significant laboratory abnormalities at screening. * Concomitant significant or severe respiratory disease. * History of psychiatric illness, epilepsy, or other central nervous system disorders. * Concomitant severe cardiovascular, hepatic, or renal systemic disease. * Pregnancy, planned pregnancy in the near future, or breastfeeding. * Unwillingness to use effective contraception during the study period. * Any condition that, in the opinion of the investigator, may increase the risk to the participant or interfere with the study outcomes.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Incidence and Severity of Adverse Events | From first dose to Week 12 |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Change from Baseline in Forced Vital Capacity (FVC) | From first dose to Week 24 | 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. |
| Change from Baseline in Diffusing Capacity of the Lung for Carbon Monoxide (DLCO) | From first dose to Week 24 | 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. |
| 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 | From first dose to Week 24 | 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). |
Countries
China
Contacts
CONTACTTong, M.D
Outcome results
None listed