Safety and Preliminary Efficacy of hUCMSC-EVs in Interstitial Lung Disease

Phase 1Not Yet RecruitingNCT07570836

Shanghai Zhongshan Hospital24 enrolled

Overview

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.

Study Type

INTERVENTIONAL

Allocation

RANDOMIZED

Purpose

TREATMENT

Masking

SINGLE

Enrollment

Conditions

Interstitial Lung Disease (ILD)

Interventions

Eligibility

Sex: ALLMin age: 18 Years

Medical Language ↔ Plain English

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.

Outcomes

Primary Outcomes

Incidence and Severity of Adverse Events

Time frame: From first dose to Week 12

Secondary Outcomes

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.

Time frame: 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.

Time frame: From first dose to Week 24

Change from Baseline in 6-Minute Walk Test (6MWT) Distance

Time frame: 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).

Time frame: From first dose to Week 24

Safety and Preliminary Efficacy of hUCMSC-EVs in Interstitial Lung Disease

Overview

Conditions

Interventions

Eligibility

Locations (1)

Outcomes

Primary Outcomes

Secondary Outcomes

Central Contacts